🎙️ Biotech Blueprint brings you weekly video updates on the latest biotech and pharma news, plus in-depth podcast interviews with industry leaders. You can find us on YouTube, Spotify, and Apple Podcasts. If you want to be featured on our podcast, get in touch.

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VIDEO SUMMARY

THIS WEEK’S KEY TAKEAWAYS 🔑

The biggest story this week is a modality. The FDA approved Veppanu on May 1, the first PROTAC protein degrader ever cleared by the agency, in ER-positive, HER2-negative, ESR1-mutated metastatic breast cancer. The Phase 3 numbers are modest: median progression-free survival of 5 months versus 2.1 months on fulvestrant in the ESR1-mutated subgroup, and the broader trial population missed statistical significance entirely, which is why the label is restricted to patients with a confirmed ESR1 mutation identified by a companion diagnostic blood test. None of that matters as much as the fact that targeted protein degradation now has a regulatory template. PROTACs work by recruiting cellular machinery to destroy a disease-causing protein entirely rather than just blocking it, and the field has been waiting years for proof that the FDA would accept this pharmacology at the registration level.

The second theme running through the week is precision medicine moving into common diseases. Two siRNA deals landed within 48 hours. Madrigal paid Arrowhead $25M upfront and up to $975M in milestones for an siRNA targeting a single genetic variant, the PNPLA3 I148M mutation, that drives MASH progression in about 30% of patients with moderate to advanced fibrosis. The Phase 1 data showed 46% liver fat reduction in patients carrying two copies of the variant and zero effect in those carrying one, which is about as clean a genetic signal as you will see in a complex metabolic disease. GSK followed the next day with a $1B deal for SiranBio’s ALK7-targeting siRNA, designed to reduce visceral fat while preserving lean mass, a profile that could complement GLP-1s rather than compete with them. Both are siRNA, both treat conditions historically owned by small molecules, and both are explicit bets that genetically defined patient subgroups are worth pursuing even when the overall population is large.

Obesity is now an earnings story. Novo Nordisk reported oral Wegovy pulling in roughly $355M in its first full quarter on the market, about double consensus, on 1.3M prescriptions. Total Q1 sales were up 32% on a constant currency basis. None of the bear cases on GLP-1 pricing pressure are showing up in actual results.

Cytokinetics had an important week. Aficamten hit both primary endpoints in non-obstructive hypertrophic cardiomyopathy, a form of the disease where there is currently no approved disease-modifying therapy and where BMS’s Camzyos has repeatedly failed. The placebo-adjusted efficacy improvements are modest on paper, but the trial swept the secondary endpoints too, and the indication is wide open. A safety flag around cardiac function will mean echo monitoring in any approved label, but that's routine in cardiology.

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BIOTECH/PHARMA NEWS 🧬

🔹 The FDA approved Arvinas’ Veppanu (vepdegestrant) on May 1, eight months ahead of its scheduled review date, for adults with ER-positive, HER2-negative, ESR1-mutated metastatic breast cancer after at least one prior line of endocrine therapy. In the Phase 3 VERITAC-2 trial, median progression-free survival was 5 months on vepdegestrant versus 2.1 months on fulvestrant in the ESR1-mutated subgroup, a 43% reduction in progression or death risk. The broader trial population missed statistical significance, confining the label to ESR1-mutated patients identified by the Guardant360 companion diagnostic blood test. Arvinas and Pfizer are not launching directly and are seeking a third-party commercialization partner, an unusual setup that likely reflects the narrow, biomarker-gated patient population. This is the first PROTAC heterobifunctional protein degrader ever approved anywhere, and it gives every other degrader program in development a regulatory roadmap.

🔹 Cytokinetics reported that aficamten hit both primary endpoints in the Phase 3 ACACIA-HCM trial in non-obstructive hypertrophic cardiomyopathy. Quality of life score improved 11.4 points versus 8.4 on placebo, and peak oxygen uptake improved 0.64 mL/kg/min versus a 0.03 decline on placebo. Secondary endpoints including functional class improvement and a key cardiac stress marker all hit with p less than 0.001. The safety flag: 10% of aficamten patients dropped their left ventricular ejection fraction below 50% versus 1% on placebo, with two serious heart failure events linked to that drop. Echo monitoring will be in any label. Cytokinetics has not given a regulatory submission date but says it will discuss findings with the FDA shortly.

🔹 Entrada Therapeutics reported early Phase 1/2 data for ENTR-601-44 in Duchenne muscular dystrophy patients with exon-44 amenable mutations. The 6 mg/kg cohort showed a mean dystrophin increase of 2.36% from a 4.00% baseline and a statistically significant improvement on a timed Rise from floor test, with no serious adverse events. Drug exposure in younger patients came in below adult levels, so the next cohort is now dosing at 12 mg/kg with more data expected by year end. The numbers are small and early, but a functional signal on top of a biomarker signal in a disease with very few options is enough to keep watching.

🔹 Madrigal licensed ARO-PNPLA3 from Arrowhead for $25M upfront and up to $975M in milestones, adding a precision siRNA alongside Rezdiffra, the only approved MASH drug. ARO-PNPLA3 targets the PNPLA3 I148M variant that drives liver fat accumulation in MASH, showing 46% liver fat reduction in patients carrying two copies of the variant and essentially no effect in those carrying one. Roughly 30% of MASH patients with moderate to advanced fibrosis carry two copies. The asset was originally co-developed with J&J before being deprioritized in 2023. Madrigal plans a Phase 2 combination trial with Rezdiffra, which starts to build a precision medicine MASH platform rather than a single asset story.

🔹 GSK agreed to pay up to $1B in milestones to license SA030, an siRNA targeting ALK7, from China-based SiranBio. ALK7 inhibition is designed to reduce visceral abdominal fat while preserving lean muscle mass, addressing one of the meaningful criticisms of GLP-1s, significant lean mass loss alongside fat loss. SiranBio retains China rights and runs the Phase 1 and GSK takes everything else. Two China-sourced siRNA assets in big pharma’s hands in 48 hours signals the modality is now platform and mature enough that large companies are licensing in rather than building from scratch.

🔹 Novo Nordisk reported first quarter sales of roughly $15.2B, up 32% on a constant currency basis, with oral Wegovy pulling in approximately $355M in its first full quarter, about double analyst consensus on roughly 1.3M prescriptions. Full year guidance was narrowed upward. Novo’s first mover advantage in the oral GLP-1 format is showing up in the numbers despite Lilly’s Foundayo entering the market in April.

🔹 Pfizer reported Q1 revenue of $14.45B, up 5% year over year, with Eliquis growing 13% to $2.17B and Comirnaty falling 59% to $232M. Full year guidance was reaffirmed. With the Metsera acquisition closed at roughly $7 billion enterprise value, Pfizer now owns MET-097i, a weekly and monthly injectable GLP-1 heading into Phase 3, plus a monthly amylin and an oral GLP-1 in Phase 1. The Lilly/Novo duopoly formally has a third serious contender.

🔹 Seaport Therapeutics and Hemab Therapeutics both priced upsized IPOs at $18, the top of their ranges, raising a combined $556M. April was the strongest biotech IPO month in five years. Institutional appetite for late stage biotech is back, but only for programs with clean data and a credible near term catalyst.

🔹 Q1 earnings data points. Vertex reported $2.99B in Q1 revenue with Casgevy at $43M and new pain drug Journavx at $29M on over 350k prescriptions in its first quarter. Bristol Myers Squibb reported Camzyos approaching 25k US patients. Regeneron reported Dupixent global sales of $4.9B, up 33%, while the overall Eylea franchise declined 10% as the newer HD formulation cannibalizes the original.

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WATCHLIST 👀

• Revolution Medicines: ASCO plenary LBA5 (daraxonrasib in 2L PDAC) on May 31, the dominant near-term oncology catalyst. Merck-Revolution acquisition talks reportedly broke down on price in late January at the $28B-$32B level

• IDEAYA Biosciences: ASCO LBA on darovasertib + crizotinib in HLA-A*02:01-negative uveal melanoma; NDA filing H2 2026

• Aficamten: Cytokinetics regulatory path discussion with FDA following ACACIA-HCM positive readout. Market expects label-expansion path with echo monitoring

• Veppanu launch: Arvinas-Pfizer third party commercialization partner selection; Q1 prescription metrics will set the bar for PROTAC class commercialization

• MET-097i (Pfizer-Metsera): VESPER-5 Phase 3 obesity readouts beginning across 10 pivotal studies in 2026

• Foundayo (Lilly orforglipron): Q2 prescription run rate, the Q2 test of oral GLP-1 demand against Wegovy oral

• Travere Therapeutics: Sparsentan FSGS PDUFA outcome (April 28, 2026) still not visibly resolved. Flagged as unverified for second consecutive week

• Intellia lonvo-z: Full HAELO Phase 3 safety table at upcoming conference. Rolling BLA submission progress

• United Therapeutics: Ralinepag NDA targeted H2 2026 (PAH). Tyvaso IPF regulatory path post-TETON-1

• Seaport (SPTX), Hemab: First-week trading prints will signal whether the IPO window stays open or tightens

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Have a great rest of your week and thanks for reading Biotech Blueprint!

DISCLAIMER: This publication is for informational and educational purposes only and does not constitute investment, legal, medical, or tax advice, a solicitation, or an offer to buy/sell any security. Information is believed reliable but no warranty is made as to accuracy or completeness; views may change without notice. Do your own research and consult qualified professionals.

DISCLOSURE & CONFLICTS: The author may hold positions in securities mentioned and may change positions at any time without notice. No compensation is received from companies mentioned, and there are no known material business relationships unless explicitly stated. Content may reference clinical data and regulatory events; always consult primary sources and a licensed clinician for medical decisions. Past performance is not indicative of future results.

LIABILITY: Use of this content is at your own risk. The author assumes no responsibility for any losses arising from reliance on this material.

Cover image credit: https://dmpkservice.wuxiapptec.com/articles/5-an-overview-of-protac-technology-and-dmpk-research-strategy/ Example of PROTAC structure.

🎙️ Biotech Blueprint brings you weekly video updates on the latest biotech and pharma news, plus in-depth podcast interviews with industry leaders. You can find us on YouTube, Spotify, and Apple Podcasts. If you want to be featured on our podcast, get in touch.

🌐 For consulting inquiries, please email us or visit BiotechBlueprintConsulting.com.

👥 If you haven’t already, please connect with me or follow me on LinkedIn.

VIDEO SUMMARY

THIS WEEK’S KEY TAKEAWAYS 🔑

This week’s defining clinical event came on April 27, when Intellia Therapeutics reported that a single dose of lonvoctocogene-z, an in vivo CRISPR therapy, reduced hereditary angioedema attack rate by 87% versus placebo in Phase 3, with 62% of recipients attack-free or off other prophylactic therapy compared to 11% on placebo. This is the first Phase 3 win for any in vivo CRISPR therapy, and it matters because in vivo gene editing has faced persistent questions about whether it could deliver durable, tolerable results in a chronic non-cancer indication at the registration level. This trial answered that. Rolling regulatory submission has started, with a US launch targeted for the first half of 2027.

Two days later, Italy’s Chiesi agreed to acquire KalVista Pharmaceuticals for $1.9 billion for Ekterly, an oral on-demand hereditary angioedema rescue therapy barely nine months into commercial launch. Ekterly is rescue treatment. Lonvoctocogene-z is one shot prophylaxis. They serve different moments in disease management, but the long run question is whether a patient population that can functionally eliminate attacks with a single CRISPR dose will keep reaching for a rescue pill at the same rate. Chiesi paid a rare disease premium two days after the prophylaxis ceiling moved. Whether that was disciplined portfolio building or a misjudgment of modality risk, the market will render a verdict over the next few years.

The week’s M&A story was Sun Pharma’s $11.75 billion acquisition of Organon, the largest biopharma deal of 2026. Organon was spun out of Merck in 2021 with established brands and biosimilars, and Sun is buying scale in mature markets and manufacturing depth. The deal signals that Indian generics and emerging market specialty pharma are now serious players in large cap consolidation, and Section 232 tariff dynamics that disadvantage import-dependent biosimilar manufacturers may have added urgency to building US-domiciled scale.

On the regulatory side, the FDA’s advisory committee voted 6 to 3 against the clinical benefit of AstraZeneca’s camizestrant in a circulating tumor DNA-triggered first-line breast cancer switch setting, the agency’s first such meeting in nine months. The progression-free survival data was strong, a hazard ratio of roughly 0.44. The committee said no anyway, citing immature overall survival, confounded secondary endpoints, and a safety profile that’s hard to justify in a clinically stable population. The precedent is important: a compelling progression-free survival signal triggered by a liquid biopsy biomarker alone is not sufficient to move stable patients to a new therapy.

Earnings closed the week with Lilly posting $19.8 billion in first quarter revenue, up 56% year-over-year, with Mounjaro and Zepbound combining for $12.76 billion. Guidance was raised to $82 to $85 billion for the full year. Foundayo’s early launch data was mixed. 3,707 prescriptions in week two versus roughly 18k for oral Wegovy at the same stage, though Lilly noted 20k patients already on the drug by the earnings call. Volume growth across the franchise is more than offsetting pricing concessions, and the obesity narrative remains intact heading into ASCO season.

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BIOTECH/PHARMA NEWS 🧬

🔹 Intellia Therapeutics reported that a single dose of lonvoctocogene-z, an in vivo CRISPR therapy, reduced hereditary angioedema attack rate by 87% versus placebo in Phase 3, with 62% of recipients attack-free or off other prophylactic therapy compared to 11% on placebo. Safety was clean, with no serious treatment-related adverse events in the topline. This is the first Phase 3 win for any in vivo CRISPR therapy, and it matters because in vivo gene editing, where the editing happens inside the body rather than in a lab dish, has faced persistent questions about whether it could deliver durable, tolerable results in a chronic non-cancer indication at the registration level. This trial, with a proper placebo control and a clean safety readout, is the clearest answer yet. Rolling regulatory submission has started, with a US launch targeted for the first half of 2027.

🔹 Italy’s Chiesi agreed to acquire KalVista Pharmaceuticals for $27 per share in cash, roughly $1.9B and a 36% premium to KalVista’s 30 day average price. KalVista’s only marketed asset is Ekterly, an oral on-demand plasma kallikrein inhibitor approved in July 2025 for hereditary angioedema attacks, barely nine months into commercial launch. The timing is uncomfortable. Ekterly is rescue therapy. Lonvoctocogene-z is one shot prophylaxis. They serve different moments in disease management, but if patients can functionally eliminate attacks with a single CRISPR dose, the lon run volume outlook for rescue therapy gets harder to underwrite. Chiesi paid the rare disease premium two days after the prophylaxis ceiling moved. That timing will be debated for a while.

🔹 The FDA’s Oncologic Drugs Advisory Committee voted 6 to 3 on April 30 against the clinical benefit of AstraZeneca’s camizestrant in a specific first-line breast cancer setting, the agency’s first such meeting in 9 months. The SERENA-6 trial randomized patients who developed an ESR1 mutation during first-line hormone therapy plus a CDK4/6 inhibitor to either switch to camizestrant or stay on their current regimen. Progression free survival was 16.0 to 16.6 months on camizestrant versus 9.2 months on continued therapy, a hazard ratio of roughly 0.44, which is a large effect. The committee wasn’t convinced: secondary endpoints were confounded by protocol-mandated switching, overall survival is immature, and a safety profile including neutropenia, visual disturbances, and a potential cardiac signal with ribociclib is hard to justify in a clinically stable population. The vote is non-binding, but the message is that a strong progression-free survival signal triggered by a circulating tumor DNA biomarker alone is not enough to move stable patients to a new therapy.

🔹 Sun Pharma signed a definitive agreement to acquire Organon for $14 per share in cash, an enterprise value of $11.75B, the largest biopharma deal of 2026. Organon was spun out of Merck in 2021 with a portfolio of established brands and biosimilars. The combined company would have pro forma revenue of roughly $12.4B. This is about scale in established markets and a deeper biosimilars manufacturing base, and it signals that Indian generics and emerging market specialty pharma companies are now serious players in large cap consolidation in a way that Western companies have largely sat out. Section 232 tariff dynamics, which structurally disadvantage import-dependent biosimilar manufacturers, may have added strategic urgency to building US-domiciled scale.

🔹 Teva agreed to acquire Emalex Biosciences for $700M upfront plus up to $200M in milestones for ecopipam, a first in class dopamine D1 receptor antagonist that hit its Phase 3 primary endpoint in pediatric Tourette syndrome, with an NDA filing planned for the second half of 2026. Existing Tourette treatments carry significant tolerability burden in kids, largely through D2-mediated metabolic effects. A selective D1 mechanism that sidesteps that is genuinely differentiated, if the safety holds in broader use. The deal fits Teva’s ongoing effort to build a credible branded neuroscience franchise alongside its legacy generics business.

🔹 Eli Lilly reported Q1 revenue of $19.8B, up 56% year-over-year, with adjusted earnings per share of $8.55 versus consensus of $6.66. Mounjaro and Zepbound combined for $12.76B in the quarter, and Lilly raised full year revenue guidance to $82 to $85B. Foundayo, the oral GLP-1 launched April 1, wasn’t broken out separately, but launch data told a nuanced story: early IQVIA prescription tracking showed 3,707 scripts in week two, roughly five times fewer than oral Wegovy at the same stage, sending shares lower. Lilly pushed back on the earnings call, noting 20k patients were already on the drug and that around 8k prescribers had written Foundayo scripts, roughly a third of whom had never written an oral GLP-1 before. The broader earnings verdict is that GLP-1 pricing concerns haven’t materially dented the franchise. Volume growth is more than offsetting any pricing concession.

🔹 Boehringer Ingelheim reported Phase 3 results for survodutide, a once-weekly injectable glucagon/GLP-1 dual agonist licensed from Zealand Pharma, showing 16.6% weight loss versus 3.2% on placebo at 76 weeks in people with obesity or overweight without type 2 diabetes. The headline number is solid, though the topline release leaves tolerability questions open. Discontinuation rates hit 24.6% in Phase 2, and Phase 3 didn’t clearly address whether that improved. Full data will be presented at the American Diabetes Association meeting in June. No filing timeline has been announced, but Boehringer is positioning survodutide as a potential first dual GLP-1/glucagon agonist to reach the market.

🔹 The FDA launched a real time clinical trials initiative with AstraZeneca and Amgen as initial partners, targeting 20 to 40% reductions in trial timelines by receiving endpoint and safety data continuously rather than at completion. A request for information is open through May 29. Worth watching, but the operational burden of real-time reporting will favor larger sponsors with sophisticated data infrastructure over most mid-cap biotechs.

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WATCHLIST 👀

• Viridian Therapeutics REVEAL-2 (elegrobart, chronic thyroid eye disease): Phase 3 topline expected Q2 2026; possibly May

• Novo Nordisk Q1 2026 earnings - May 6: first commentary on competitive response to Foundayo and Mounjaro/Zepbound

• Vertex Q1 2026 earnings - May 4: Casgevy uptake and Alyftrek commentary

• Pfizer Q1 2026 earnings - May 5: Comirnaty and Paxlovid trajectory plus Vyndamax franchise commentary post-settlement

• Catalyst Pharmaceuticals (CPRX) Q1 earnings - May 11: possible Angelini bid commentary

• Merck IDVYNSO HIV launch - May 11: first week prescription read versus Biktarvy switch volumes

• Seaport Therapeutics IPO pricing: terms set at $16-$18 for $201M raise; pricing expected this week

• Hemab Therapeutics IPO pricing: terms set targeting $200M at $715M market cap

• ASCO 2026, May 29-June 2, Chicago: plenary session features Revolution daraxonrasib LBA5 (May 31) plus IDEAYA darovasertib LBA. Five plenary abstracts confirmed: PROTEUS, SARC041, LIBRETTO-432, HARMONi-6, and RASolute 302

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Have a great rest of your week and thanks for reading Biotech Blueprint!

DISCLAIMER: This publication is for informational and educational purposes only and does not constitute investment, legal, medical, or tax advice, a solicitation, or an offer to buy/sell any security. Information is believed reliable but no warranty is made as to accuracy or completeness; views may change without notice. Do your own research and consult qualified professionals.

DISCLOSURE & CONFLICTS: The author may hold positions in securities mentioned and may change positions at any time without notice. No compensation is received from companies mentioned, and there are no known material business relationships unless explicitly stated. Content may reference clinical data and regulatory events; always consult primary sources and a licensed clinician for medical decisions. Past performance is not indicative of future results.

LIABILITY: Use of this content is at your own risk. The author assumes no responsibility for any losses arising from reliance on this material.

Cover image credit: iStock, avgust01

🎙️ Biotech Blueprint brings you weekly video updates on the latest biotech and pharma news, plus in-depth podcast interviews with industry leaders. You can find us on YouTube, Spotify, and Apple Podcasts. If you want to be featured on our podcast, get in touch.

🌐 For consulting inquiries, please email us or visit BiotechBlueprintConsulting.com.

👥 If you haven’t already, please connect with me or follow me on LinkedIn.

VIDEO SUMMARY

THIS WEEK’S KEY TAKEAWAYS 🔑

Merck’s Idvynso got FDA approval on April 21, a few days ahead of its scheduled review date, for virologically suppressed adults with HIV-1 looking to switch regimens. The significance isn’t the timing but what the drug represents, because for the first time, a suppressed patient can move to a two drug regimen that contains neither an integrase inhibitor nor tenofovir. Both drug classes are effective but carry long term baggage. Integrase inhibitors with weight gain and resistance accumulation risk, and tenofovir with renal and bone signals in susceptible patients. Idvynso won’t displace Biktarvy overnight, but it establishes a non-integrase standard of care in the switch setting that didn’t exist before.

Replimune had the opposite kind of week. The company received its second complete response letter for RP1 plus nivolumab in advanced melanoma, again on the same grounds: the trial couldn’t isolate what RP1 was actually contributing on top of a checkpoint inhibitor that already works well on its own. Replimune then announced layoffs of more than 200 people, roughly 60% of its workforce.

Kailera Therapeutics raised $625 million in its Nasdaq IPO, the largest biotech debut on record, for a Phase 2 obesity drug licensed from China’s Hengrui Pharma. That number says something about where investors sit right now. Lilly and Novo define the current GLP-1 battlefield, but the race for next gen differentiated mechanisms is still wide open, and the market is clearly willing to fund it at scale.

Beeline Medicines launched with $300 million in Series A funding built on five programs licensed from Bristol Myers Squibb, led by afimetoran, an oral TLR7/8 inhibitor in Phase 1b/2 for lupus with FDA Fast Track Designation. Same logic as J&J’s divestiture of bota-vec to MeiraGTx last week. These aren’t unwanted assets. Large companies are just concluding that focused independent teams run early precision immunology programs better than internal commercial machinery does.

AACR wrapped up April 22 in San Diego with Revolution Medicines presenting updated first-line daraxonrasib data alongside the already confirmed Phase 3 overall survival results. ASCO abstract titles dropped April 21, full data embargoed until May 29 through June 2 in Chicago, with the daraxonrasib Phase 3 RASolute 302 dataset confirmed for a plenary oral on May 31. The next six weeks will be the most data dense stretch of the year for precision oncology, and Revolution’s ASCO presentation is the one everyone is watching.

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BIOTECH/PHARMA NEWS 🧬

🔹 The FDA approved Idvynso on April 21 for virologically suppressed adults with HIV-1 looking to switch off their current regimen. The once-daily single tablet pairs doravirine, a non-nucleoside reverse transcriptase inhibitor, with islatravir, which works through a distinct mechanism from conventional HIV drugs. Two Phase 3 trials supported the approval: a head-to-head against Gilead's Biktarvy showed identical efficacy at Week 48, and a switch trial showed 96% of Idvynso recipients maintained viral suppression. The clinical argument is straightforward: long-term exposure to the drug class in Biktarvy carries associations with weight gain and metabolic effects, and tenofovir carries renal and bone signals in some patients. Idvynso removes both in a single pill. Commercial availability begins May 11, and the switch population is large. The real launch question is formulary access.

🔹 Replimune received a second complete response letter for RP1 in combination with nivolumab for advanced melanoma, and the FDA's concern hasn't changed: a single-arm trial can't isolate what the drug is actually contributing on top of nivolumab alone. The company followed the news with layoffs of more than 200 employees, roughly 60% of its workforce. The broader lesson for the oncolytic virus field is hard to ignore: compelling early signal in open-label studies does not survive FDA scrutiny if the registration design can't answer the additive benefit question. Any future combination program will need a randomized arm comparing the backbone therapy alone. That's the bar now.

🔹 Kailera Therapeutics raised $625M in its Nasdaq IPO, the largest biotech debut on record, surpassing Moderna's 2018 raise. The company's lead program is oral ribupatide, a GLP-1/GIP dual agonist currently in Phase 2 for obesity, licensed from China's Hengrui Pharma. That China angle matters: Hengrui has one of the more productive GLP-1 pipelines outside the US, and Kailera is essentially a vehicle to bring that chemistry into the American development and regulatory system. The size of the raise signals that investors aren't waiting for the Lilly versus Novo battle to resolve before backing the next wave of obesity drugs.

🔹 Beeline Medicines launched April 15 with $300M in Series A financing, built around five programs licensed from Bristol Myers Squibb targeting immune-mediated diseases. The lead asset is afimetoran, an oral TLR7/8 inhibitor in Phase 1b/2 for lupus with FDA Fast Track Designation, with a Phase 2 readout expected in the second half of 2026. The pipeline also includes a TYK2 inhibitor and an IL-2/CD25 fusion protein. The structure mirrors what we've seen elsewhere recently: a large pharma deciding a set of early-stage programs is better run in a leaner, focused vehicle than inside a commercial organization. BMS keeps some economics, Beeline gets the operational flexibility. Whether afimetoran can compete in lupus against established options like belimumab and anifrolumab depends on tolerability, and the Phase 2 data will be the first real read on that.

🔹 The FDA approved Sanofi/Regeneron’s Dupixent (dupilumab) for chronic spontaneous urticaria, a condition causing persistent unpredictable hives, in children aged 2 to 11 who don't respond adequately to antihistamines. This is Dupixent's ninth allergy-related indication. The approval was supported by Phase 3 data from the LIBERTY-CUPID program in adolescents and adults plus pharmacokinetic and safety data in the target pediatric age group. No single pediatric label expansion moves the needle much on a drug already doing over $16B a year, but Sanofi and Regeneron have been methodical about this: build formulary positioning across the entire atopic disease spectrum, one indication at a time.

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CLINICAL TRIAL UPDATES 📊

🔹 Doravirine / Islatravir (Merck) - Phase 3, HIV-1. The two trials behind the Idvynso approval are covered above. The clinical read is clean and the non-inferiority bar against Biktarvy is met. The standard for any competitive HIV switch study is now a head to head against an approved non-integrase inhibitor regimen at Week 48, and Merck cleared it.

🔹 Daraxonrasib (Revolution Medicines) - Phase 1/2, first-line metastatic pancreatic cancer. Revolution presented early first-line data at AACR this week, though full efficacy numbers remain embargoed until the slides are released. What’s notable is that Revolution has submitted for a plenary presentation at ASCO for the Phase 3 dataset, which signals internal confidence in the full data package. The scientific question worth watching is whether the drug’s multi-variant RAS targeting works as well in first-line patients, whose tumors haven’t yet been shaped by chemotherapy selection pressure, as it does in the second-line setting where the landmark results came from. Still early, still small cohorts, but the first-line ambition is clearly alive.

WATCHLIST 👀

• ASCO 2026 abstracts: Full abstracts release May 21; meeting runs May 29 to June 2 in Chicago

• Daraxonrasib (Revolution Medicines): Phase 3 RASolute 302 plenary presentation, Abstract LBA5, May 31, ASCO 2026

• Darovasertib + crizotinib (IDEAYA): Phase 2/3 OptimUM-02 late-breaking oral presentation, ASCO 2026; NDA submission targeted H2 2026

• Viridian elegrobart: REVEAL-2 readout (chronic thyroid eye disease) expected Q2 2026; BLA targeted Q1 2027

• Lilly Foundayo T2D NDA: Filing targeted end of Q2 2026; ACHIEVE-4 MACE HR 0.84 and all-cause mortality HR 0.43 (not multiplicity-controlled) are the supporting data

• Beeline afimetoran (SLE): Phase 2 completion targeted H2 2026; pivotal decision to follow

• Novartis pelacarsen (Lp(a)HORIZON): Lp(a) lowering cardiovascular outcomes trial; timing on readout not confirmed but widely described as a high-stakes event for 2026; watch for announcement

• IDVYNSO commercial launch: Available in pharmacies after May 11; payer formulary positioning and switch uptake versus Biktarvy will be early launch metrics to watch

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Have a great rest of your week and thanks for reading Biotech Blueprint!

DISCLAIMER: This publication is for informational and educational purposes only and does not constitute investment, legal, medical, or tax advice, a solicitation, or an offer to buy/sell any security. Information is believed reliable but no warranty is made as to accuracy or completeness; views may change without notice. Do your own research and consult qualified professionals.

DISCLOSURE & CONFLICTS: The author may hold positions in securities mentioned and may change positions at any time without notice. No compensation is received from companies mentioned, and there are no known material business relationships unless explicitly stated. Content may reference clinical data and regulatory events; always consult primary sources and a licensed clinician for medical decisions. Past performance is not indicative of future results.

LIABILITY: Use of this content is at your own risk. The author assumes no responsibility for any losses arising from reliance on this material.

Cover image credit: iStock, avgust01

🎙️ Biotech Blueprint brings you weekly video updates on the latest biotech and pharma news, plus in-depth podcast interviews with industry leaders. You can find us on YouTube, Spotify, and Apple Podcasts. If you want to be featured on our podcast, get in touch.

🌐 For consulting inquiries, please email us or visit BiotechBlueprintConsulting.com.

👥 If you haven’t already, please connect with me or follow me on LinkedIn.

VIDEO SUMMARY

THIS WEEK’S KEY TAKEAWAYS 🔑

April 13 may turn out to be one of the more important single days in pancreatic cancer research in a generation. Revolution Medicines posted a landmark Phase 3 result in second-line metastatic pancreatic cancer, a disease where chemotherapy produces median overall survival of roughly six to seven months, and where nothing has meaningfully moved that number in over a decade. The trial posted 13.2 months, with a hazard ratio of 0.40, meaning a 60% reduction in the risk of death versus chemotherapy, delivered as a once daily oral pill. If this holds through full data review, daraxonrasib becomes the standard of care for RAS-mutant second-line pancreatic cancer, which is essentially all of it, and Revolution becomes a serious acquisition target.

On the same morning, IDEAYA Biosciences reported a clean Phase 3 readout in uveal melanoma, a rare eye cancer with no approved first-line treatment for the relevant patient subgroup. The overall response rate was 37.1% versus 5.8% for investigator’s choice, with a hazard ratio of 0.42 on progression-free survival. Smaller population than pancreatic cancer, but an equally unambiguous result. An NDA filing is targeted for the second half of 2026. Two clean results in two notoriously hard indications on the same morning is not something that happens often.

The Foundayo story moved forward on two fronts. The FDA asked Lilly for post-marketing studies covering liver injury, thyroid cancer risk, and long-term cardiovascular outcomes, which is standard for a new mechanism being deployed at massive scale, not a red flag. Lilly responded with ACHIEVE-4, their largest and longest orforglipron trial to date: 2,749 patients with type 2 diabetes and elevated cardiovascular risk, non-inferior cardiovascular outcomes versus insulin glargine, no liver signal, 8.1 kg of weight loss versus a 1.4 kg gain on the comparator, and all-cause mortality 57% lower with Foundayo, though that last number wasn’t controlled for multiple comparisons and should be read carefully. The T2D filing is on track for Q2.

On the policy side, the FDA scheduled an advisory committee review to reconsider restrictions on several compounded peptides that were flagged for safety concerns back in 2023. The practical read is that compounding boundaries directly affect demand for branded alternatives, safety narratives around entire drug classes, and how aggressively telehealth platforms can operate. When those boundaries get revisited, it creates real uncertainty for companies that have been planning around the existing framework. On the capital side, the market is open but selective. Revolution pulled in a $2B financing on the back of its Phase 3 data in a single move, while smaller players like MeiraGTx had to get creative with asset reacquisitions and targeted equity raises. Lilly also added to its oncology portfolio, acquiring CrossBridge Bio for up to $300M to get its hands on a dual-payload TROP2 antibody drug conjugate at the pre-IND stage. Small deal, but consistent with a deliberate pattern of building out targeted delivery capabilities before the competitive window closes.

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BIOTECH/PHARMA NEWS 🧬

🔹 In an unusual reversal of the typical deal direction, MeiraGTx announced on April 16 that it is acquiring botaretigene sparoparvovec (bota-vec) back from J&J for $25M upfront, with a single milestone payment due on U.S. approval and royalties to J&J starting in mid-2029. Bota-vec is an AAV-based gene therapy targeting RPGR-associated X-linked retinitis pigmentosa (XLRP), a rare inherited retinal disease causing progressive vision loss in an estimated 20k patients in the U.S. and EU with no approved treatment. The FDA has granted Fast Track and Orphan Drug Designations; the EU has granted PRIME and ATMP designations. MeiraGTx targets regulatory filings in the U.S. and EU in 2027. For MeiraGTx, this clears the path to commercialize a program it originated.

🔹 The FDA scheduled advisory committee review to reconsider restrictions on several compounded peptides that had been placed in a category associated with significant safety concerns in 2023. The agency is moving to reclassify multiple substances, with meetings planned in 2026 and early 2027. This is a policy volatility signal for biopharma and telehealth alike, because compounding boundaries influence demand flows, safety narratives, and future enforcement assumptions.

🔹 Lilly announced on April 14 the acquisition of CrossBridge Bio, a Houston-based startup spun out of UTHealth Houston, for up to $300M in cash (upfront plus a development milestone). CrossBridge’s lead asset is CBB-120, a TROP2-targeting antibody-drug conjugate (ADC) that delivers two payloads simultaneously: a topoisomerase 1 inhibitor and an ATR kinase inhibitor. The crowded TROP2 ADC space already has two approved single-payload drugs in Trodelvy and Datroway, and the bet with CBB-120 is that hitting two biological targets simultaneously generates more durable responses and makes it harder for tumors to develop resistance. IND filing for CBB-120 is targeted for 2026. This is Lilly’s latest oncology platform add, following its ongoing expansion into degraders and conjugate hybrids. Section 232 tariff exemptions for ADCs further advantage this category. The deal is expected to close in Q2 2026.

🔹 Roche said it will launch a new global Phase 3 trial for Elevidys in early ambulatory Duchenne muscular dystrophy patients, randomizing about 100 participants to Elevidys or placebo over 72 weeks with time-to-rise as the primary endpoint. The study is designed to support a resubmission in Europe after the prior negative opinion from the EMA. This resets expectations for a longer and more data-intensive European path and reinforces that gene therapy commercialization is now a multi-cycle evidence process.

🔹 Replimune began layoffs after receiving a second complete response letter on RP1 in advanced melanoma, including a reported reduction of 63 employees this month, while management continues to argue the therapy’s activity profile remains clinically meaningful. This is the execution penalty in real time when accelerated approval strategy fails to align with regulator expectations.

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CLINICAL TRIAL UPDATES 📊

🔹 Daraxonrasib (Revolution Medicines) - Phase 3 [RASolute 302], 2L metastatic pancreatic ductal adenocarcinoma (PDAC). In what may be the most important oncology data package of 2026 so far, Revolution Medicines announced on April 13 that its RAS(ON) multi-selective inhibitor daraxonrasib met both primary endpoints in the RASolute 302 Phase 3 trial: statistically significant improvement in both progression-free survival and overall survival (OS) versus investigator’s choice chemotherapy in approximately 460 previously treated metastatic PDAC patients. Median OS was 13.2 months for daraxonrasib versus 6.7 months for chemotherapy. That is nearly double the survival achieved by gemcitabine/nab-paclitaxel or FOLFOX in the second-line setting. The drug targets RAS(ON) mutations, which drive tumor growth in approximately 90% of pancreatic cancer cases, making this a broadly applicable result. Safety was manageable with no new signals. Revolution plans to submit an NDA using an expedited national priority review pathway enabling a one-to-two month review timeline. A first-line PDAC Phase 3 (RASolute 303) is already enrolling. Shares surged on the news.

🔹 Darovasertib + crizotinib (IDEAYA Biosciences / Servier) - Phase 2/3 [OptimUM-02], 1L HLA-A*02:01-negative metastatic uveal melanoma. The regimen met its primary endpoint in 313 patients with median progression-free survival 6.9 months versus 3.1 months. Objective response rate was 37.1% versus 5.8%, including five complete responses in the combination arm. This remains one of the cleanest efficacy signals this week in a hard to treat setting, with overall survival maturity still the key open variable.

🔹 Orforglipron / Foundayo (Eli Lilly) - Phase 3 [ACHIEVE-4], type 2 diabetes with obesity and elevated cardiovascular risk. Lilly dropped the largest and longest Foundayo (orforglipron) trial to date, ACHIEVE-4, covering 2,749 patients with type 2 diabetes and elevated cardiovascular risk across 15 countries. Against insulin glargine, Foundayo met cardiovascular non-inferiority, cut weight by 8.1 kg versus a 1.4 kg gain on the comparator, and reduced A1C by 1.6% versus 1.0%. The headline number that will get attention: all-cause mortality was 57% lower with Foundayo, though that result wasn’t controlled for multiple comparisons so it needs to be interpreted carefully. Lilly plans to file for a type 2 diabetes indication by end of Q2 2026.

WATCHLIST 👀

• Merck DOR/ISL (doravirine/islatravir) - PDUFA April 28; if approved, first once-daily two-drug HIV regimen without an integrase inhibitor; watch for any impact on Gilead’s Biktarvy franchise

• Viridian Therapeutics / elegrobart - REVEAL-2 (chronic thyroid eye disease) topline data expected Q2 2026; REVEAL-1 (active TED) was positive; BLA filing targeted Q1 2027

• IDEAYA / darovasertib+crizotinib - NDA filing H2 2026; first potential approval in first-line uveal melanoma

• Revolution Medicines / daraxonrasib - NDA submission timing TBD; ASCO 2026 data presentation upcoming; RASolute 303 (1L PDAC) now enrolling

• Eli Lilly / Foundayo (T2D) - NDA submission by end of Q2 2026; FDA will evaluate against ACHIEVE-4 data and post-marketing study framework from obesity approval

• Section 232 tariffs - July 31, 2026 effective date for Annex III companies; ADC/cell gene/orphan/plasma-derived biologics remain exempt; watch for pre-July deal acceleration in tariff-exposed modalities

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DISCLAIMER: This publication is for informational and educational purposes only and does not constitute investment, legal, medical, or tax advice, a solicitation, or an offer to buy/sell any security. Information is believed reliable but no warranty is made as to accuracy or completeness; views may change without notice. Do your own research and consult qualified professionals.

DISCLOSURE & CONFLICTS: The author may hold positions in securities mentioned and may change positions at any time without notice. No compensation is received from companies mentioned, and there are no known material business relationships unless explicitly stated. Content may reference clinical data and regulatory events; always consult primary sources and a licensed clinician for medical decisions. Past performance is not indicative of future results.

LIABILITY: Use of this content is at your own risk. The author assumes no responsibility for any losses arising from reliance on this material.

Cover image credit: Pancreatic cancer, illustration Nemes Laszl | Science Photo Library | Getty Images

🎙️ Biotech Blueprint brings you weekly video updates on the latest biotech and pharma news, plus in-depth podcast interviews with industry leaders. You can find us on YouTube, Spotify, and Apple Podcasts. If you want to be featured on our podcast, get in touch.

🌐 For consulting inquiries, please email us or visit BiotechBlueprintConsulting.com.

👥 If you haven’t already, please connect with me or follow me on LinkedIn.

VIDEO SUMMARY

THIS WEEK’S KEY TAKEAWAYS 🔑

Gilead committed another $3.15 billion upfront to acquire Tubulis, an antibody-drug conjugate company, bringing its total dealmaking this year to roughly $11 billion across three transactions. Meanwhile, the backstory on Merck’s Terns acquisition finally came out via SEC filings. Merck originally offered $61 per share, updated trial data on TERN-701 showed a lower response rate than previously disclosed, the competing bidder walked away, and the deal settled at $53 after Terns negotiated up from Merck’s revised $50 offer. The final price still looks cheap to most analysts, but the process showed that even highly anticipated assets get repriced when data gets more nuanced in due diligence.

Lilly launched Foundayo with self-pay pricing starting at $149 per month, and the commercial argument centers on convenience - no food or water timing restrictions, fast fulfillment, and an accessible entry price. The competition between Lilly and Novo Nordisk has shifted from clinical trial comparisons to adherence, payer contracts, and logistics, and that dynamic will play out over years.

On the science side, Roche and C4 Therapeutics expanded their collaboration into degrader antibody conjugates, combining targeted delivery with payload biology that destroys disease-causing proteins rather than just blocking them. Still preclinical, but the investment in the concept is notable. Clinical results elsewhere were mixed. Sanofi’s lunsekimig won in airway disease but missed in atopic dermatitis, and Insmed dropped another indication for Brinsupri after a mid-stage failure. Capital is going to programs with tight translational logic and away from broad indication expansion without it.

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BIOTECH/PHARMA NEWS 🧬

🔹 Gilead agreed to acquire Tubulis for $3.15B upfront plus up to $1.85B in milestones, adding clinical stage and preclinical antibody drug conjugate programs and expanding Gilead’s internal payload and linker capabilities. The transaction values Tubulis at up to $5B and is expected to close in Q2. This is another signal that large cap oncology buyers are paying for technical depth in chemistry and design, not just target novelty. Gilead has now committed about $11B across three deals this year, which makes the company’s diversification push harder to dismiss as incremental portfolio maintenance.

🔹 The CDC’s acting director Jay Bhattacharya has delayed publication of a CDC report showing that updated COVID vaccines cut the risk of emergency department visits by about 50% and hospitalizations by roughly 55% in healthy adults last winter. The study, based on data from September through December and originally scheduled for publication on March 19 in the CDC’s Morbidity and Mortality Weekly Report, used a methodology called test-negative design, a well-established approach the agency has relied on for years to evaluate respiratory virus vaccines. Bhattacharya has raised concerns about that methodology, and HHS framed the delay as routine leadership review. Former CDC official Dan Jernigan pushed back, arguing the findings simply don’t align with the direction RFK Jr. has taken on vaccines, noting that suppressing evidence of vaccine effectiveness while simultaneously rolling back COVID shot recommendations for children and pregnant women sends a deeply contradictory public health message.

🔹 Merck’s $6.7B acquisition of Terns Pharmaceuticals got more interesting this week when SEC filings revealed the real negotiation behind the headline number. Four companies were originally bidding, and Merck’s initial offer was $61 per share, about $1B more than the final price. What changed: updated data from the CARDINAL trial showed that TERN-701’s major molecular response rate at 24 weeks came in lower than the 64% figure Terns had presented at a December conference. Merck cut its offer to $50, the competing bidder walked away entirely, and Terns negotiated back up to $53. William Blair, which had previously floated the possibility of a counterbid, now says a competing offer is unlikely and expects the deal to close in early May.

🔹 Lilly formally launched Foundayo (orforglipron) in the U.S. after FDA approval last week, opening prescriptions immediately with shipping starting April 6. Lilly highlighted ATTAIN 1 data showing average weight loss of 12.4% among trial completers at the highest dose, and self pay pricing that starts at $149 per month on the lowest dose. The oral GLP-1 race is now live, not theoretical. Novo Nordisk had a three month head start in oral obesity, and Lilly responded with a convenience argument that Foundayo can be taken without food or water restrictions. This does not automatically dethrone Novo, but it changes the commercial battlefield from pure efficacy comparison to adherence friction, fulfillment speed, and net pricing strategy.

🔹 Roche and C4 Therapeutics expanded their long running targeted protein degradation relationship with a new degrader antibody conjugate collaboration. C4T receives $20M upfront, potential milestone payments exceeding $1B across discovery, regulatory, and commercial events, and tiered royalties if products launch. This is a concrete sign that oncology modality convergence is accelerating. Antibody drug conjugates already validated targeted delivery, and degraders promise catalytic biology beyond occupancy inhibition. The combination can expand target space if safety and therapeutic index hold up in humans. It is still early, still unproven clinically, but strategically this is where next cycle optionality is being built.

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CLINICAL TRIAL UPDATES 📊

🔹 Lunsekimig (Sanofi) - Phase 2 across asthma, chronic rhinosinusitis with nasal polyps, and atopic dermatitis. Sanofi reported a split outcome: positive reads in asthma and chronic rhinosinusitis with nasal polyps, but failure in atopic dermatitis. Public coverage has not yet provided full endpoint level tables and denominator level detail across all cohorts, so the current read remains directional rather than complete. One clean signal in airway disease does not grant automatic credibility in dermatology. The next key step is whether Sanofi narrows to the strongest biology defined populations or continues broad indication expansion with a higher execution burden.

🔹 Subcutaneous Tepezza (Amgen) - Phase 3 in thyroid eye disease. Amgen said a subcutaneous formulation of Tepezza was comparable to the infused version in a pivotal setting. Full numerical disclosure remains limited in open access summaries, but the route of administration signal is clear. Convenience is now part of competitive defense in thyroid eye disease as oral and subcutaneous challengers, including Viridian Therapeutics, move closer to market. Improved administration logistics can protect incumbent share even before head to head superiority data exist.

🔹 Brinsupri (Insmed) - mid stage hidradenitis suppurativa program discontinued. Insmed stopped Brinsupri in hidradenitis suppurativa after a mid stage miss, adding to a prior discontinuation in sinus inflammation. Serial indication failures force portfolio triage and tighter capital allocation. In this market, investors are not rewarding broad anti inflammatory expansion without strong translational logic and a visible efficacy gradient early in development. Next for Insmed is focus and redeployment into assets with cleaner probability weighted paths.

WATCHLIST 👀

• Oral obesity net pricing: whether Lilly and Novo keep matching low entry prices or shift to tighter dose tier economics as payer negotiations deepen.

• Gilead post close integration: timeline updates for Tubulis programs and whether management changes development sequencing this quarter.

• Merck TERN-701 clinical disclosures: deeper molecular response and safety detail ahead of broader investor scrutiny on best in class claims.

• DAC modality validation: first meaningful preclinical to clinical translation markers from Roche and C4T’s degrader antibody conjugate strategy.

• Immunology indication narrowing: Sanofi’s development path decision after lunsekimig’s mixed read across three diseases.

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Have a great rest of your week and thanks for reading Biotech Blueprint!

DISCLAIMER: This publication is for informational and educational purposes only and does not constitute investment, legal, medical, or tax advice, a solicitation, or an offer to buy/sell any security. Information is believed reliable but no warranty is made as to accuracy or completeness; views may change without notice. Do your own research and consult qualified professionals.

DISCLOSURE & CONFLICTS: The author may hold positions in securities mentioned and may change positions at any time without notice. No compensation is received from companies mentioned, and there are no known material business relationships unless explicitly stated. Content may reference clinical data and regulatory events; always consult primary sources and a licensed clinician for medical decisions. Past performance is not indicative of future results.

LIABILITY: Use of this content is at your own risk. The author assumes no responsibility for any losses arising from reliance on this material.

Cover image credit: Hannah Beier (Reuters)

🎙️ Biotech Blueprint brings you weekly video updates on the latest biotech and pharma news, plus in-depth podcast interviews with industry leaders. You can find us on YouTube, Spotify, and Apple Podcasts. If you want to be featured on our podcast, get in touch.

🌐 For consulting inquiries, please email us or visit BiotechBlueprintConsulting.com.

👥 If you haven’t already, please connect with me or follow me on LinkedIn.

VIDEO SUMMARY

THIS WEEK’S KEY TAKEAWAYS 🔑

Eli Lilly paired a fast regulatory win for Foundayo (orforglipron) with a large strategic acquisition of Centessa, while Biogen bought Apellis for near term commercial cash flow and nephrology field infrastructure. Those moves all point to thee fact that large buyers are still willing to pay real money, but mostly when uncertainty is constrained and launch math is visible.

Yesterday, the White House moved to impose 100% tariffs on a subset of branded pharmaceutical imports from countries without qualifying trade arrangements, with carveouts and phased implementation language shaping the near term impact. The immediate business effect is not simple demand destruction. It is supply chain repricing risk, gross margin uncertainty, and fresh pressure to localize active pharmaceutical ingredient and finished dose manufacturing for politically exposed products. Even before full implementation details settle, boards now have to model trade policy as a core variable.

The obesity narrative also got more strategic in the last 48 hours. Lilly now has the first approved oral small molecule GLP-1 option for chronic weight management that can be taken without food or water timing constraints. Novo Nordisk quickly challenged comparative claims in public, which is exactly what a mature two player market looks like when convenience and efficacy are both investable endpoints. This no longer reads like a single winner category. It reads like a multiyear channel battle across adherence, payer access, and real world persistence.

Clinical data reinforced a stricter quality bar. Immunovant reported that batoclimab failed both pivotal thyroid eye disease studies on the primary endpoint, while Beam continued to publish early but encouraging base editing data in sickle cell disease. The market is being pretty blunt. Late stage misses get punished immediately, and early platform wins only matter if they keep turning into durable efficacy with strong execution.

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BIOTECH/PHARMA NEWS 🧬

🔹 U.S. administration signed an order setting 100% tariffs on certain imported branded drugs and active pharmaceutical ingredients from countries without qualifying trade deals, with carveouts that appear designed to limit immediate disruption while preserving political leverage. For operating teams, the implication is not just customs cost pass through. It is contract manufacturing renegotiation, product specific sourcing triage, and potential gross to net pressure if pricing actions face payer resistance. Investors now need to underwrite policy implementation risk product by product, not simply at sector level.

🔹 Eli Lilly consolidated an unusually strong week by combining regulatory speed and strategic M&A. The FDA approved Foundayo (orforglipron) for chronic weight management in adults with obesity, or overweight with at least one weight related comorbidity, and did so 50 days after filing under the Commissioner’s National Priority Voucher program. Lilly then announced the acquisition of Centessa for $38/share in cash, valuing the transaction at about $6.3B upfront, plus a contingent value right worth up to $9 per share linked to orexin receptor 2 program milestones. The strategic logic is that obesity generated scale is being recycled into adjacent, high value chronic disease franchises where first movers can still build durable share.

🔹 Merck signed a collaboration that could reach $838M in milestones for antibody candidates generated against multiple undisclosed targets, with economics weighted to downstream achievements. The headline matters less than the structure. Large pharma still wants external biologics throughput, but prefers options and staged risk transfer over heavy upfront checks for preclinical stories. That framework raises the execution burden for platform companies: it is no longer enough to show technical novelty, you need repeatable asset quality and partner relevant timelines.

🔹 Biogen agreed to acquire Apellis for $41 per share in cash, approximately $5.6B upfront, with additional contingent value rights tied to Syfovre sales milestones. The deal adds two marketed complement pathway products and an established nephrology commercial footprint while Biogen advances internal kidney programs. Apellis reported $690M in 2025 net product revenues across Syfovre and Empaveli, which gives Biogen immediate top line contribution rather than a long precommercial wait. In this funding tape, that is exactly the type of asset profile that clears internal return hurdles for large buyers.

🔹 IO Biotech moved toward bankruptcy after failing to secure a buyer or financing path following pivotal disappointment in its cancer vaccine program. The company disclosed intent to seek bankruptcy protection, underscoring how quickly a late stage miss plus weak capital access can collapse strategic optionality. This is the opposite side of the same market that rewarded large cap buyers this week. Capital exists, but it is selective and often binary around data credibility and near term commercial visibility.

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CLINICAL TRIAL UPDATES 📊

🔹 Batoclimab (Immunovant) - Phase 3, thyroid eye disease: Immunovant reported that both pivotal studies, TIDE-1 and TIDE-2, missed the primary endpoint of at least a 2 millimeter reduction in proptosis at Week 24 versus placebo. The company also said one secondary endpoint, diplopia responder rate, was not statistically significant in either study, while another secondary endpoint, Clinical Activity Score of 0 or 1, reached statistical significance in one trial but not the other. Safety was described as generally consistent with prior batoclimab experience, with no newly identified signal that altered overall interpretation. This was not a clean enough efficacy package to support a credible TED path for batoclimab, and management signaled focus would remain on next generation FcRn assets.

🔹 BEAM-101 (risto-cel) (Beam Therapeutics) - Phase 1/2, sickle cell disease: Beam announced publication of BEACON data in The New England Journal of Medicine, reinforcing the early thesis that base editing can durably raise fetal hemoglobin and reduce vaso occlusive events in treated patients. Patient counts remain limited and follow up is still maturing, so this is not yet a registrational level dataset. But the translational signal continues to hold up across company updates and peer reviewed presentation, which keeps the platform competitive in the broader one time treatment race for hemoglobinopathies. The practical next step is scaling enrollment and durability follow up while demonstrating manufacturing consistency at higher throughput.

WATCHLIST 👀

• U.S. pharma tariff implementation details: country scope, product exemptions, and active pharmaceutical ingredient treatment will drive near term margin modeling and supply chain changes.

• Orca Bio, Orca-T: FDA extended review to July 6 after additional chemistry, manufacturing, and controls submission.

• Viridian Therapeutics, REVEAL-2: chronic thyroid eye disease Phase 3 readout expected in Q2, key for commercial positioning after REVEAL-1 debate.

• Lilly vs Novo oral obesity positioning: comparative messaging fight will likely shift quickly toward payer contracts and persistence data.

• Biogen integration of Apellis assets: early commercial execution and nephrology channel retention will signal whether the deal thesis converts into growth.

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Have a great rest of your week and thanks for reading Biotech Blueprint!

DISCLAIMER: This publication is for informational and educational purposes only and does not constitute investment, legal, medical, or tax advice, a solicitation, or an offer to buy/sell any security. Information is believed reliable but no warranty is made as to accuracy or completeness; views may change without notice. Do your own research and consult qualified professionals.

DISCLOSURE & CONFLICTS: The author may hold positions in securities mentioned and may change positions at any time without notice. No compensation is received from companies mentioned, and there are no known material business relationships unless explicitly stated. Content may reference clinical data and regulatory events; always consult primary sources and a licensed clinician for medical decisions. Past performance is not indicative of future results.

LIABILITY: Use of this content is at your own risk. The author assumes no responsibility for any losses arising from reliance on this material.

Cover image credit: Andrew Leyden/NurPhoto via AP

🎙️ Biotech Blueprint brings you weekly video updates on the latest biotech and pharma news, plus in-depth podcast interviews with industry leaders. You can find us on YouTube, Spotify, and Apple Podcasts. If you want to be featured on our podcast, get in touch.

🌐 For consulting inquiries, please email us or visit BiotechBlueprintConsulting.com.

👥 If you haven’t already, please connect with me or follow me on LinkedIn.

VIDEO SUMMARY

THIS WEEK’S KEY TAKEAWAYS 🔑

The biggest theme this week was capital flowing toward rare and hard to treat diseases, and the market rewarding science that actually solves delivery problems rather than just finding new targets. Merck’s $6.7 billion grab of Terns Pharmaceuticals is the headline M&A move, and it’s an interesting one because the price looks low by almost every analyst’s math. TERN-701 is an oral chronic myeloid leukemia drug with early data compelling enough that Leerink is penciling in $6.2 billion in peak annual sales, yet Merck paid a 6% premium to last close, one of the thinnest acquisition premiums in years. Whether that reflects shrewd negotiating or a valuation gap that other companies might exploit with a counterbid is the question the market is now actively debating. For Merck, the strategic logic is simple as Keytruda’s patent clock is ticking and they need new pillars.

On the science side, two stories stood out for platform reasons, not just drug reasons. Denali’s FDA accelerated approval of Avlayah for Hunter syndrome is the first time a therapy has been approved specifically to cross the blood-brain barrier using the transferrin receptor as a molecular Trojan horse. The disease itself is devastating and rare, affecting roughly 500 Americans, but the approval is really a proof-of-concept for Denali’s TransportVehicle delivery platform, which has more programs in the clinic. Similarly, Sarepta’s 25% single-day jump came from early siRNA data in two untreatable muscular dystrophies showing the company may have cracked muscle delivery, the longstanding Achilles heel of gene silencing approaches.

The governance subplot running in the background is the CDC leadership vacuum. The Trump administration missed its own deadline to name a permanent director, and the agency has now cycled through multiple acting leaders since the firing of Susan Monarez in August 2025, apparently for resisting RFK Jr.’s vaccine policy agenda. It’s easy to write this off as Washington noise, but prolonged instability at the CDC has real downstream consequences for public health infrastructure, outbreak response capacity, and the regulatory environment that drug and vaccine makers operate in.

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BIOTECH/PHARMA NEWS 🧬

🔹 The Trump administration missed its own Wednesday deadline to name a permanent CDC director, meaning NIH chief Jay Bhattacharya will keep steering the agency but loses the acting director title in what is becoming a running governance problem. About six candidates are reportedly under vetting, including Johns Hopkins cardiologist Joseph Marine and former Kentucky governor Ernie Fletcher, but the White House is taking its time. The CDC has been effectively leaderless for most of Trump’s second term: Susan Monarez lasted barely a month as director before being fired in August 2025, apparently for refusing to implement vaccine policy changes ordered by HHS Secretary RFK Jr., and the agency has cycled through acting leadership ever since.

🔹 Sarepta Therapeutics jumped 25% after dropping first-in-human data from two phase 1/2 trials targeting rare, currently untreatable muscular dystrophies. SRP-1001 targets facioscapulohumeral muscular dystrophy type 1 (FSHD1), caused by toxic overexpression of the DUX4 protein that progressively destroys skeletal muscle in about 16,000 Americans, while SRP-1003 goes after myotonic dystrophy type 1 (DM1), the most common adult-onset muscular dystrophy affecting around 40,000 Americans, which wrecks not just muscle but also the heart, lungs, and central nervous system. Both drugs use small interfering RNA, or siRNA, a gene-silencing approach that blocks production of the disease-causing protein or toxic RNA before it can do damage. The historical problem with siRNA in muscle disease has been that the drug degrades before reaching muscle cells, but Sarepta's platform uses an integrin-targeted ligand that actively ferries the siRNA into muscle tissue. The early data showed dose-dependent drug exposure in muscle, proof-of-concept knockdown of the target after a single dose, and no dose-limiting toxicities, which is exactly what you want to see at this stage.

🔹 Merck agreed to acquire Terns Pharmaceuticals for $53 per share in cash, roughly $6.7B in equity value or about $5.7B net of cash, to get its hands on TERN-701, an oral allosteric tyrosine kinase inhibitor targeting the BCR::ABL1 fusion protein that drives chronic myeloid leukemia, a slow-growing blood cancer. The drug works differently from older inhibitors by binding to a distinct pocket on the ABL protein, and early phase 1/2 data from the CARDINAL trial showed major and deep molecular responses by week 24, including in heavily pretreated patients who had already failed other allosteric inhibitors, with a clean tolerability profile and no meaningful blood pressure signals. The deal is strategically obvious for Merck. Keytruda faces patent expiration and the company needs new revenue pillars, and Leerink analysts project TERN-701 could peak at $6.2B in annual sales by 2040 if it captures newly diagnosed patients. That projection makes the $5.7B net price look thin, and Wall Street noticed: the 6% premium to last close is among the lowest paid for a public drugmaker since 2018, sparking chatter that AbbVie or BMS could table a competing bid.

🔹 The FDA granted accelerated approval to Denali Therapeutics’ Avlayah for Hunter syndrome, a rare X-linked lysosomal storage disorder affecting roughly 500 Americans, almost all boys, that progressively strips patients of their ability to speak, walk, and think. The approval is a genuine step forward because existing enzyme replacement therapies cannot cross the blood brain barrier, leaving the neurological devastation of the disease untreated for nearly 20 years. Avlayah gets around this using Denali’s TransportVehicle platform, which fuses the therapeutic enzyme to an engineered fragment that hijacks the transferrin receptor, a natural transport system at the blood-brain barrier, to ferry the drug into the brain. In the phase 1/2 trial it drove a 91% reduction in cerebrospinal fluid heparan sulfate by week 24, with 93% of patients reaching normal levels, and that biomarker served as the surrogate endpoint for accelerated approval. The ongoing phase 2/3 COMPASS trial is required to confirm clinical benefit.

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CLINICAL TRIAL UPDATES 📊

🔹 Insmed had an important win in lung infection. In its phase 3b ENCORE trial, adding Arikayce (amikacin liposome inhalation suspension) to standard azithromycin plus ethambutol in newly diagnosed Mycobacterium avium complex lung disease improved symptoms and drove much higher culture conversion than placebo on top of the same backbone. The respiratory symptom score improved more at Month 13 (17.8 points vs 14.7, p=0.0299) and culture conversion was the headline, 87.8% by Month 6 vs 57.0% with placebo, with durability holding through Month 15. Safety looked consistent with what we already know for inhaled amikacin, with more voice and airway issues and a higher discontinuation rate, but no new red flags. This was the post-marketing confirmatory work tied to the 2018 accelerated approval, and Insmed now plans a supplemental filing in the second half of 2026 to both expand the label into earlier disease and convert the refractory indication to traditional approval.

🔹 Apogee popped after one year data on zumilokibart in atopic dermatitis suggested something patients actually care about: staying controlled without living on an injection schedule. In the phase 2 APEX part A maintenance readout, responses held up through 52 weeks with dosing every three months or even every six months, and they’re claiming responses kept improving over time rather than plateauing. It’s still phase 2 and the key induction readout (APEX Part B at 16 weeks) lands in Q2 2026, but it seems the drug can offer strong control with two to four dosing days per year.

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DISCLAIMER: This publication is for informational and educational purposes only and does not constitute investment, legal, medical, or tax advice, a solicitation, or an offer to buy/sell any security. Information is believed reliable but no warranty is made as to accuracy or completeness; views may change without notice. Do your own research and consult qualified professionals.

DISCLOSURE & CONFLICTS: The author may hold positions in securities mentioned and may change positions at any time without notice. No compensation is received from companies mentioned, and there are no known material business relationships unless explicitly stated. Content may reference clinical data and regulatory events; always consult primary sources and a licensed clinician for medical decisions. Past performance is not indicative of future results.

LIABILITY: Use of this content is at your own risk. The author assumes no responsibility for any losses arising from reliance on this material.

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🎙️ Biotech Blueprint brings you weekly video updates on the latest biotech and pharma news, plus in-depth podcast interviews with industry leaders. You can find us on YouTube, Spotify, and Apple Podcasts. If you want to be featured on our podcast, get in touch.

🌐 For consulting inquiries, please email us or visit BiotechBlueprintConsulting.com.

👥 And if you haven’t already, please connect with me or follow me on LinkedIn.

VIDEO SUMMARY

THIS WEEK’S KEY TAKEAWAYS 🔑

Let’s start with TrumpRx. Right now it does not actually guarantee the world’s lowest prices, but it basically discounts for a specific slice of people, mainly cash-pay patients, and only on a limited list of drugs. The NY Times ran a price check and a lot of branded drugs still come out cheaper overseas. That is not because those countries have better coupon codes, but because they negotiate harder and then spread costs through the system.

On the policy front, the RFK Jr. vaccine overhaul hit a legal wall. A federal judge in Massachusetts paused the January memo that reduced routine childhood schedule and also froze the new ACIP appointments and everything they voted on. The practical implication is that vaccine guidance is now in limbo again, but this at least puts a temporary brake on more abrupt changes while the legal process plays out.

Clinically and commercially, there were two very different kinds of “big wins.” J&J’s oral Icotyde got its FDA approval in moderate-to-severe plaque psoriasis. And it does not need to beat AbbVie’s Skyrizi on peak efficacy to matter. If a once daily pill pulls even a modest chunk of patients off endless topical cycling, it expands the treated market and changes what first-line can look like in the real world. Meanwhile, Eli Lilly put up another serious set of numbers with retatrutide in type 2 diabetes. Strong A1C drops, unusually large weight loss for this population plus low discontinuation rates. You do not have to crown it the “best” today to see the strategic problem for everyone else - Lilly just keeps showing up with another credible next thing.

Finally, Aldeyra’s dry eye program got a Complete Response Letter again, and this time the FDA basically said the efficacy story is not consistent enough to trust. No safety or manufacturing issues, just the hardest kind of rejection to spin because it goes straight at the signal.

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BIOTECH/PHARMA NEWS 🧬

🔹 TrumpRx is running into a reality check: a discount website is not the same thing as “the lowest prices in the world.” A new New York Times investigation (with German outlets) compared TrumpRx prices to what patients in Germany and other wealthy countries effectively pay and found many branded, on-patent drugs are still cheaper abroad, sometimes dramatically, because those systems negotiate harder and then spread the cost through insurance. TrumpRx can still be a real help for a narrow group (people paying cash, for a limited list of roughly a few dozen drugs), but it is not a systemic price reset, and it does not touch the biggest driver of U.S. drug spending: what insurers and government programs pay for high-cost specialty meds. The bigger takeaway for biotech is that Most favored nation pricing as a slogan is easy, but turning it into broad, durable savings without shifting costs somewhere else is the hard part.

🔹 J&J just picked up an important plaque psoriasis approval. The FDA cleared Icotyde (icotrokinra), a once daily interleukin-23 receptor antagonist, for moderate-to-severe disease in adults and adolescents 12+ who are candidates for systemic therapy or phototherapy. The pivotal ICONIC phase 3 program (about 2,500 patients) hit primary endpoints with a favorable safety profile, and the Street is already framing this as the first serious “oral first line” threat to injectable incumbents like AbbVie’s Skyrizi, not necessarily because it beats Skyrizi on raw efficacy, but because an effective oral option can pull more patients off endless topical cycling and expand the treated market. For Protagonist it means a $50M approval milestone now, 6% to 10% royalties, and up to $580M more in future milestones.

🔹 Aldeyra got hit with a Complete Response Letter for reproxalap in dry eye disease, and the FDA’s message was basically “the efficacy story is not consistent enough to approve.” The agency didn’t flag safety or manufacturing, but it said the totality of the trials doesn’t support effectiveness and that the mixed results make the positives hard to trust. What’s unusual here is the FDA did not explicitly ask for another trial, and Aldeyra says it doesn’t plan to run one right now, instead pushing for a fast Type A meeting to figure out what, exactly, would get this over the line.

🔹 A federal judge in Massachusetts just paused the RFK Jr. vaccine overhaul. Judge Brian Murphy issued a preliminary injunction blocking the January memo that cut the routine childhood schedule from 17 diseases down to 11, and he also stayed the appointments of the newly installed ACIP members, along with all votes they have taken. The judge’s point was procedural but consequential. HHS sidestepped the normal expert-driven process and replaced the committee without the usual screening, which he said likely violates governing law. Practically, this throws vaccine guidance into limbo again, because the panel “as currently constituted” cannot operate while the injunction stands.

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CLINICAL TRIAL UPDATES 📊

🔹 Eli Lilly reported phase 3 results for retatrutide, its “triple agonist” that hits GLP-1, GIP, and glucagon receptors. In TRANSCEND-T2D-1, the drug cut hemoglobin A1C by about 1.7% to 2.0% at 40 weeks across doses and delivered eye-catching weight loss for a type 2 diabetes population: 16.8% on the highest dose in the on-treatment analysis (15.3% including discontinuations), with discontinuations for side effects reported in the low single digits. This doesn’t automatically dethrone tirzepatide on glucose control, but it does reinforce Lilly’s core advantage in obesity and diabetes: it keeps showing up with another credible “next thing,” and the bar for everyone else keeps moving higher.

🔹 Aspen Neuroscience put out twelve-month data from its early ASPIRO trial for Parkinson’s using a personalized cell therapy made from each patient’s own skin cells. In the first eight treated patients (four lower dose, four higher dose), they reported no serious surgery-related adverse events, no severe graft-induced dyskinesia, and imaging consistent with the cells surviving and engrafting. On the “does this feel meaningfully better?” front, both cohorts showed numerical improvements across movement scores, daily function, and quality of life measures, with some patients also cutting their levodopa-equivalent dose. It’s still a tiny, open-label dataset, so you can’t call this proof yet, but it’s a real step forward, and the next big tell will be whether they can replicate anything like this in a larger, more controlled phase 3.

🔹 Rhythm’s phase 3 EMANATE trial for setmelanotide (Imcivree) in several MC4R-pathway rare obesity subtypes missed its prespecified primary endpoint (placebo-adjusted BMI change at 52 weeks across four genetic substudies), which is why the stock dipped. The company is trying to salvage the readout with post hoc signals suggesting meaningful BMI reductions in the heterozygous POMC/PCSK1 and SRC1 (NCOA1) groups, with no new safety issues, but this still reads as “not clean enough” for an immediate label expansion, and it shifts attention to Rhythm’s next-generation melanocortin 4 receptor agonists (bivamelagon and RM-718) for what comes next.

🔹 Candel reported “survival tail” update in its mid-stage non-small cell lung cancer study for CAN-2409 (aglatimagene besadenovec) plus valacyclovir on top of continued checkpoint inhibitors, in patients who already had an inadequate response to prior checkpoint therapy. With another year of follow-up, the company says 50% of 46 per-protocol patients were still alive at 24 months (up from 39% at the prior data cut), which is the kind of number that gets attention in a post-checkpoint setting where expectations are usually grim. They also leaned on biomarker work showing a more inflamed tumor environment after treatment and a broader T cell receptor response, and they’re using the dataset to justify a pivotal phase 3 in non-squamous disease starting in Q2 2026.

🔹 Eledon posted early but eye-catching islet transplant data in long-standing type 1 diabetes. In a 12-patient, investigator-initiated trial at the University of Chicago, 10 of the 12 patients who were at least four weeks post-transplant were insulin-independent, with no reported graft rejection and no new donor-specific human leukocyte antigen antibodies. The bigger “so what” is the regimen: tegoprubart is an anti-CD40 ligand antibody used in a calcineurin-inhibitor-free immunosuppression approach, and the team highlighted the absence of the classic calcineurin inhibitor baggage (kidney toxicity, hypertension, neurotoxicity) in this small cohort. Still early, still small, but it keeps the idea alive that you might protect islet grafts without paying the tacrolimus tax.

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Have a great rest of your week and thanks for reading Biotech Blueprint!

DISCLAIMER: This publication is for informational and educational purposes only and does not constitute investment, legal, medical, or tax advice, a solicitation, or an offer to buy/sell any security. Information is believed reliable but no warranty is made as to accuracy or completeness; views may change without notice. Do your own research and consult qualified professionals.

DISCLOSURE & CONFLICTS: The author may hold positions in securities mentioned and may change positions at any time without notice. No compensation is received from companies mentioned, and there are no known material business relationships unless explicitly stated. Content may reference clinical data and regulatory events; always consult primary sources and a licensed clinician for medical decisions. Past performance is not indicative of future results.

LIABILITY: Use of this content is at your own risk. The author assumes no responsibility for any losses arising from reliance on this material.

Image credit: Getty Images.

Hi everyone, today’s edition is just a quick note. For personal and family reasons, I’m taking a short break from publishing for the next two weeks. I plan to be back with This Week in Biotech on March 20.

🎙️ Biotech Blueprint brings you weekly video updates on the latest biotech and pharma news, plus in-depth podcast interviews with industry leaders. You can find us on YouTube, Spotify, and Apple Podcasts. If you want to be featured on our podcast, get in touch.

🌐 For consulting inquiries, please email me or visit BiotechBlueprintConsulting.com.

👥 And if you haven’t already, please connect with me or follow me on LinkedIn.

In the meantime, here are a few reader favorite episodes to catch up on.

1. Podcast (November 2025): Sensible Biotechnologies
   I sat down with Miro Gasparek (CEO) and Krishna Motheramgari (Principal Computational Scientist). Sensible is an Oxford-born, Y Combinator-backed startup betting that cell-based production will shape the next decade of mRNA.

2. Podcast (December 2025): NImmune Biopharma
   I spoke with Dr. Josep Bassaganya-Riera (Founder and CEO) about building a late-stage immunology company in 2025, including raising capital in a tighter market, designing Phase 3 programs that match today’s regulatory expectations, and using artificial intelligence in ways that actually change decisions.

3. Special episode (September 2025): mRNA therapeutics mini-series (part 1)
   I teamed up with Hartaj Singh (Biotech Capital Compass) to cover how ribonucleic acid became medicine, the engineering breakthroughs behind the coronavirus disease vaccines, and what might come next in infectious disease and oncology. We also discussed programs from Moderna and BioNTech, including personalized cancer vaccines.

Thanks for your patience and for reading with Biotech Blueprint.

Subscribe now

DISCLAIMER: This publication is for informational and educational purposes only and does not constitute investment, legal, medical, or tax advice, a solicitation, or an offer to buy/sell any security. Information is believed reliable but no warranty is made as to accuracy or completeness; views may change without notice. Do your own research and consult qualified professionals.

DISCLOSURE & CONFLICTS: The author may hold positions in securities mentioned and may change positions at any time without notice. No compensation is received from companies mentioned, and there are no known material business relationships unless explicitly stated. Content may reference clinical data and regulatory events; always consult primary sources and a licensed clinician for medical decisions. Past performance is not indicative of future results.

LIABILITY: Use of this content is at your own risk. The author assumes no responsibility for any losses arising from reliance on this material.

IN COLLABORATION WITH CYTIVA

Succeeding in an industry under pressure

60% of biopharma firms are missing market share goals. Find out why, and learn what leaders are doing to deliver.

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🎙️ Biotech Blueprint brings you weekly video updates on the latest biotech and pharma news, plus in-depth podcast interviews with industry leaders. You can find us on YouTube, Spotify, and Apple Podcasts. If you want to be featured on our podcast, get in touch.

🌐 For consulting inquiries, please email us or visit BiotechBlueprintConsulting.com.

👥 And if you haven’t already, please connect with me or follow me on LinkedIn.

VIDEO SUMMARY

THIS WEEK’S KEY TAKEAWAYS 🔑

Yesterday the FDA commissioner went on CNBC and drew a pretty blunt line. Without mentioning a company by name, he said that if a rare disease therapy requires drilling a burr hole into someone’s skull, don’t expect the agency to shrug off the procedure risk just because the need is high. The market took it personally and uniQure’s stock dropped about 30%. And while he may have been talking about a different program, investors heard the broader point loud and clear - the FDA’s patience for invasive, high-morbidity delivery is thinning, and nobody wants to be the next test case.

Vaccine policy is getting more unstable in ways that actually affect schedules, coverage, and manufacturer planning. The CDC pulled its February vaccine advisory committee meeting after the lawsuit pressure (by AAP and other medical groups), rescheduled it for March 18-19, and the agenda now includes COVID-19 vaccine injuries and long COVID. Mainstream medical groups are treating this like an emergency, not a normal policy disagreement, because they think the process is being reshaped in a way that will push recommendations toward lower vaccination rates.

Gene therapy had a reality check this week. BioMarin pulling Roctavian after failing to find a buyer is the clearest sign yet that one and done cures are still commercially fragile. It is not just the science, it is reimbursement, eligibility, logistics, and a standard of care that keeps getting better. And the uncomfortable takeaway is that approval is no longer the finish line, it is just the beginning for a very hard commercialization marathon.

Gilead buying Arcellx feels like a simple call. Anito-cel looks legit, and Gilead doesn’t want to share the upside anymore. But they’re still hedging with that earnout, basically agreeing to pay extra if the drug really sells. And Novo Nordisk finally did the obesity head-to-head everyone’s been debating (CagriSema vs Lilly’s Zepbound) and it lost. Not by a ton, but in obesity, close still counts as losing, at least for now in investors’ eyes. If Novo wants to change the conversation, it has to win on something people actually feel, like fewer dropouts, better tolerability, better outcomes, or a higher-dose version that clearly beats tirzepatide.

And lastly there’s Grail. A large National Health Service trial in the UK missed its primary endpoint, which is a real hit to the idea that this test is ready for population-wide screening. Yes, they can highlight secondary signals and argue the picture could improve with more digging, but the market reaction is understandable. These tests only matter if they reliably move diagnoses earlier at scale, in messy real-world care with real follow-up, not just in theory. That’s the standard now.

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BIOTECH/PHARMA NEWS 🧬

🔹 uniQure sold off after FDA Commissioner Marty Makary said on CNBC the agency will not approve rare-disease therapies that require invasive delivery with meaningful procedure-related morbidity, citing a “burr hole” brain-delivery example and a trial showing no benefit. Investors took it as a warning shot at AMT-130, though at least one analyst argued the remarks may fit an already-rejected intraventricular program more closely than uniQure’s.

🔹 CRISPR Therapeutics shares popped on fresh takeover chatter, with traders circulating a Betaville “uncooked” alert suggesting Vertex may be exploring an acquisition. Nothing is confirmed, but the logic is obvious: Vertex already co-developed Casgevy with CRISPR, so buying CRISPR would consolidate control over the partnership and pipeline while the market is quick to reprice on any whiff of M&A.

🔹 ACIP meeting update: After major medical groups sued to stop the CDC’s vaccine advisory committee meeting, the CDC pulled the late-February dates and has now rescheduled ACIP for March 18-19, 2026. The Federal Register agenda adds discussions and possible votes on COVID-19 vaccine injuries and long COVID, alongside ACIP recommendation methodology, with written public comments open March 2-12. The lawsuit is still the backdrop here, and the practical effect is the same: when the advisory process is being challenged and reshaped in real time, vaccine guidance, coverage expectations, and manufacturer planning all get harder.

🔹 BioMarin is pulling Roctavian, its hemophilia A gene therapy, after a multi-month search for a buyer went nowhere. The company will take roughly a $240M hit tied mainly to inventory write offs and asset impairments, underscoring how hard it has been to make one time gene therapies work commercially at scale. Roctavian was approved in 2023 and still grew to $36M in 2025 sales, but uptake stayed limited amid reimbursement friction, eligibility constraints, and strong competition from established hemophilia options.

🔹 Most Favored Nation pricing is back in the spotlight, and a new trade group is forming around the fear that it hits smaller innovators differently than big pharma. Fierce Biotech reports that 10 mid-sized biotechs have launched the Midsized Biotech Alliance of America to oppose MFN, arguing that a diversified pharma can absorb price caps across a portfolio, while a one asset biotech cannot. If MFN becomes a blunt tool rather than a targeted policy, the predictable second-order effect is fewer big, expensive phase 3 bets and more early partnering or asset sales, which means less upside but also less innovation risk taken in-house. Lower prices are the point; the open question is whether MFN can deliver them without quietly shrinking the pipeline that produces tomorrow’s drugs.

🔹 Sarepta Therapeutics said CEO Doug Ingram will step down by the end of 2026, with the board launching a search for a successor and Ingram staying on until a new chief is named. He said the decision was driven by family circumstances: his wife and son were recently diagnosed with myotonic dystrophy type 1, a disease Sarepta began working on in 2024 via a partnership. Ingram’s tenure featured rapid growth on the back of multiple Duchenne muscular dystrophy approvals, including the Elevidys gene therapy, but also major controversy and scrutiny tied to gene therapy safety.

🔹 Vir Biotechnology jumped after signing a global development and commercialization deal with Astellas to advance VIR-5500 in metastatic prostate cancer. The economics are meaningful for a company of Vir’s size: $335M in upfront and near term payments, up to $1.37B in milestones, and tiered double digit royalties outside the U.S., with Astellas covering 60% of development costs.

🔹 MacroGenics’ lead program lorigerlimab is now under an FDA partial clinical hold: no new patients can be enrolled in its Phase 2 LINNET trial after one patient died following Grade 4 neutropenia and septic shock, and three other patients had severe events (including Grade 4 thrombocytopenia and Grade 4 myocarditis). 41 patients have been dosed so far, and current participants can stay on treatment while the company works with the agency on protocol changes and enhanced monitoring to try to lift the hold.

🔹 Gilead agreed to buy Arcellx for $7.8B and giving Gilead full control of anitocabtagene autoleucel (anito-cel), their BCMA-directed CAR-T therapy for multiple myeloma. The contingent payment only triggers if anito-cel reaches $6B in cumulative global net sales from launch through the end of 2029, so Gilead is paying for upside but trying to keep discipline. The near term key question is regulatory: the FDA has accepted the BLA for anito-cel in fourth line relapsed or refractory multiple myeloma with a Dec. 23, 2026 action date, and Gilead says the deal becomes earnings per share accretive in 2028 and beyond if approved.

🔹 Vanda said the FDA approved Bysanti (milsaperidone) for bipolar I disorder and schizophrenia, sending the stock up about 44% after hours. Milsaperidone is the active metabolite of iloperidone, which Vanda previously sold as Fanapt, and Vanda says studies found the two drugs are bioequivalent. The company is also studying Bysanti in phase 3 as an add on treatment for major depressive disorder.

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CLINICAL TRIAL UPDATES 📊

🔹 Protara’s phase 2 ADVANCED-2 update for TARA-002 in high risk non-muscle invasive bladder cancer looked mixed on durability, which is why the stock sold off despite solid early complete response rates. In the Bacillus Calmette-Guérin (BCG) unresponsive cohort, complete response was 68% at six months but only 33% at 12 months. In the BCG naïve cohort, complete response was 67% at six months and 58% at 12 months, roughly in line with the prior data cut. Safety stayed clean with no grade 3 or higher treatment-related adverse events, and the company is still guiding to start its registrational BCG naïve trial in the second half of 2026 while finishing enrollment in the unresponsive registrational cohort later in 2026.

🔹 Novo Nordisk’s obesity head-to-head trial landed cleanly and not in Novo’s favor. In the open label phase 3 REDEFINE 4 trial (84 weeks, 809 adults with obesity plus comorbidities), CagriSema (cagrilintide 2.4 mg plus semaglutide 2.4 mg) missed the primary endpoint of non-inferiority versus Eli Lilly’s tirzepatide (Zepbound) 15 mg on weight loss. On-treatment weight loss was 23.0% for CagriSema versus 25.5% for tirzepatide, and with discontinuations included it was 20.2% versus 23.6%. Novo highlighted a safe, well-tolerated profile with mostly mild to moderate gastrointestinal effects that eased over time and argued the amylin add on still delivers clinically meaningful lift beyond GLP-1 alone, but the commercial reality is that obesity markets reward the winner, not close. With CagriSema already filed in the U.S. with a decision expected in late 2026, Novo now needs differentiation that changes behavior, such as better persistence and tolerability, stronger outcomes beyond weight, or a higher dose path.

🔹 Grail’s NHS-Galleri trial in the UK missed its primary endpoint. Adding annual Galleri screening did not significantly reduce combined stage 3 to 4 cancers in a 142k person randomized study. Grail highlighted secondary signals including fewer stage 4 diagnoses in a pre-specified 12 cancers with highest mortality group and plans deeper analyses ahead of ASCO 2026, but the miss is a hit to the “population screening” story.

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Have a great rest of your week and thanks for reading Biotech Blueprint!

DISCLAIMER: This publication is for informational and educational purposes only and does not constitute investment, legal, medical, or tax advice, a solicitation, or an offer to buy/sell any security. Information is believed reliable but no warranty is made as to accuracy or completeness; views may change without notice. Do your own research and consult qualified professionals.

DISCLOSURE & CONFLICTS: The author may hold positions in securities mentioned and may change positions at any time without notice. No compensation is received from companies mentioned, and there are no known material business relationships unless explicitly stated. Content may reference clinical data and regulatory events; always consult primary sources and a licensed clinician for medical decisions. Past performance is not indicative of future results.

LIABILITY: Use of this content is at your own risk. The author assumes no responsibility for any losses arising from reliance on this material.

🎙️ Biotech Blueprint brings you weekly video updates on the latest biotech and pharma news, plus in-depth podcast interviews with industry leaders. You can find us on YouTube, Spotify, and Apple Podcasts. If you want to be featured on our podcast, get in touch.

🌐 For consulting inquiries, please email us or visit BiotechBlueprintConsulting.com.

👥 And if you haven’t already, please connect with me or follow me on LinkedIn.

VIDEO SUMMARY

THIS WEEK’S KEY TAKEAWAYS 🔑

In a New England Journal of Medicine piece, FDA leadership said the new default for most approvals will be one adequate, well-controlled pivotal trial plus “confirmatory evidence,” rather than two pivotal trials. In theory, that can be a rational modernization. A second trial does not rescue a weak control arm, sloppy endpoints, or a fragile statistical plan. In practice, the market will care less about the philosophy and more about the execution. A policy shift this large, announced without clear implementation guidance, invites the one thing drug developers and investors dislike most - ambiguity about what will be accepted, when, and by whom.

That uncertainty showed up in real time with Moderna. Last week, its mRNA flu vaccine drew a Refusal to File over a comparator dispute. This week, it was accepted back into review after a Type A meeting and an amended application, with an August 5, 2026 target action date. Moderna’s revised path also shifts to an age-tiered approach, starting with adults 50 and older and adding a post marketing trial in older adults. Process and policy are now moving parts, and that will shape how sponsors design trials, allocate capital, and decide which programs are worth the regulatory risk.

Major medical groups asked a federal judge to block the CDC’s ACIP meeting and pause implementation of recent immunization guidance changes. The late-February meeting has been pulled from the calendar, and reporting is mixed on whether it will be rescheduled in mid-March. The plaintiffs are treating this as an emergency, not a routine policy dispute. They argue the committee’s remade composition and process could produce recommendations that further lower vaccination rates, and they want to stop the next round of guidance before it lands.

Clinically, Compass Pathways delivered a Phase 3 win in treatment-resistant depression. COMP360, a synthetic psilocybin, beat a very low dose control (1mg) on the Montgomery Åsberg Depression Rating Scale at week six. The company is talking about rapid onset and a generally tolerable safety profile, and it is now pushing toward FDA discussions and a targeted Q4 filing.

Finally, China is becoming a bigger driver of global biotech deal flow. Out licensing hit a record $137.7B in 2025, and early 2026 is pointing to bigger average deal sizes and higher upfront payments as global pharma shops for pipeline help ahead of major patent expirations. The simplest read is that China is no longer just a bargain source of external innovation. It is becoming a core hunting ground, especially in modalities like antibody-drug conjugates, where the density of licensable assets keeps rising.

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BIOTECH/PHARMA NEWS 🧬

🔹 Makary and Prasad just reset the default on FDA evidence standards. In a New England Journal of Medicine piece, they said the agency’s new baseline will be one adequate, well-controlled pivotal trial plus “confirmatory evidence,” not two. They argue two trials can create false confidence if the design is weak, and that focusing reviewer time on one high quality study could speed development and cut costs, with extra studies still required when mechanisms are fuzzy, endpoints are surrogate, or designs are otherwise fragile. Former FDA leaders and industry voices are warning that announcing a major policy shift via an opinion style article without detailed guidance, stakeholder input, or implementation specifics could reduce predictability and rattle investors, payers, and global regulators who usually treat FDA as the reference standard.

🔹 Sensei Biotherapeutics is effectively rebooting via an all stock merger with Faeth Therapeutics plus a $200M private placement, shifting the company’s center of gravity to Faeth’s lead program PIKTOR, an all oral dual node approach in the PI3K–AKT–mTOR pathway (serabelisib + sapanisertib). The near-term focus is a phase 2 study in second-line advanced endometrial cancer and a planned phase 1b in hormone receptor-positive, HER2-negative advanced breast cancer, with key milestones expected before end of 2026. The pitch is simple: multi-node inhibition aimed at avoiding the classic PI3K trap where efficacy gains come with intolerability, supported by early signals that included complete responses in heavily pretreated endometrial cancer. Sensei will also use some of the new capital to finish its ongoing phase 1/2 VISTA antibody study (solnerstotug).

🔹 ImmunityBio jumped after the European Commission granted conditional approval for Anktiva plus BCG in BCG-unresponsive non-muscle invasive bladder cancer with carcinoma in situ, with or without papillary tumors. It gives the company commercial access across the full EU (plus Iceland, Norway, and Liechtenstein), and ImmunityBio is pitching it as the first immunotherapy authorized in Europe for this setting, where the fallback has often been radical cystectomy. The label is conditional because the approval leaned on a single arm phase 2/3 dataset, so the company now owes European regulators longer term safety and durability followup to keep the authorization.

🔹 Moderna got a fast reversal at FDA on mRNA-1010. After last week’s Refusal to File over a “best available standard of care” comparator dispute, FDA has now accepted the amended biologics license application for review and set an Aug. 5, 2026 target action date. Moderna says the turnaround followed a Type A meeting and a revised plan that splits the filing by age, with an initial push toward adults 50+ and a post-marketing requirement for an additional trial in older adults.

🔹 The American Academy of Pediatrics, the American Public Health Association, and others asked a federal judge in Massachusetts to block the CDC’s next ACIP meeting and pause recent immunization guidance changes. Since the suit landed, the late-February meeting has been pulled. Reuters reported it will not be held with no new date announced, while Bloomberg reported CDC is considering a mid-March reschedule. The plaintiffs argue HHS Secretary RFK Jr.’s overhaul was unlawful and risks lowering vaccination rates. The Department of Justice has pushed back that the challengers are trying to stifle debate and that HHS has broad discretion, while Judge Brian Murphy flagged the uncomfortably tight timeline for a decision.

🔹 The FDA issued a Complete Response Letter for Disc Medicine’s bitopertin in erythropoietic protoporphyria, effectively saying accelerated approval will have to wait for phase 3. The nuance is FDA agreed the phase 2 trials showed a clear biomarker effect (lowering whole-blood metal-free protoporphyrin), but said the phase 2 package did not demonstrate that the size of that biomarker change tracked with clinical benefit on the sunlight-exposure endpoints used in the studies. Disc now pivots to the APOLLO phase 3 readout expected in the fourth quarter of 2026 as the likely path to traditional approval.

🔹 China biotech out-licensing hit a record $137.7B in 2025, and early 2026 activity points to larger average deal sizes and higher upfront checks as global pharma shops for pipeline replenishment ahead of major patent expirations. The message is that China is increasingly a core sourcing market for innovative assets, with particular strength in high demand modalities like antibody-drug conjugates.

🔹 AC Immune said Janssen (J&J) has temporarily paused enrollment in the [hase 2b ReTain trial of their anti-phospho-tau vaccine (ACI-35.030 / JNJ-2056) in preclinical Alzheimer’s, while Janssen reviews trial logistics including recruitment. The company emphasized the pause is voluntary and not tied to new safety findings, and that the trial’s pre-specified interim immunogenicity threshold was met. AC Immune also said the pause does not change its cash runway, which it still expects to extend into Q3 2027 (excluding any milestones).

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CLINICAL TRIAL UPDATES 📊

🔹 Compass Pathways jumped after its second pivotal phase 3 study of COMP360, its synthetic psilocybin therapy, hit the primary endpoint in treatment resistant depression, showing a statistically significant 3.8 point advantage on the Montgomery Åsberg Depression Rating Scale at week 6 versus a very low dose control. The company also highlighted fast onset starting the next day, a generally tolerable safety profile with mostly mild to moderate events, and plans to meet with the FDA on a rolling application ahead of a targeted Q4 filing.

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Have a great rest of your week and thanks for subscribing to Biotech Blueprint!

DISCLAIMER: This publication is for informational and educational purposes only and does not constitute investment, legal, medical, or tax advice, a solicitation, or an offer to buy/sell any security. Information is believed reliable but no warranty is made as to accuracy or completeness; views may change without notice. Do your own research and consult qualified professionals.

DISCLOSURE & CONFLICTS: The author may hold positions in securities mentioned and may change positions at any time without notice. No compensation is received from companies mentioned, and there are no known material business relationships unless explicitly stated. Content may reference clinical data and regulatory events; always consult primary sources and a licensed clinician for medical decisions. Past performance is not indicative of future results.

LIABILITY: Use of this content is at your own risk. The author assumes no responsibility for any losses arising from reliance on this material.

🎙️ Biotech Blueprint brings you weekly video updates on the latest biotech and pharma news, plus in-depth podcast interviews with industry leaders. You can find us on YouTube, Spotify, and Apple Podcasts. If you want to be featured on our podcast, get in touch.

🌐 For consulting inquiries, please visit BiotechBlueprintConsulting.com.

👥 And if you haven’t already, please connect with me or follow me on LinkedIn.

VIDEO SUMMARY

THIS WEEK’S KEY TAKEAWAYS 🔑

This week’s biggest signal came from FDA. Moderna’s flu vaccine received a Refusal to File not for safety or efficacy, but for a comparator dispute. The company used a licensed standard dose flu shot, which it says FDA initially accepted, and it also included older adult evidence, including a separate phase 3 comparison against a high dose vaccine in those 65 and older. When a filing can turn on shifting views of what counts as the best available standard of care, the regulatory bar starts to feel like a moving target. Reports that leadership overruled career reviewers only deepen the impression that the rulebook is getting less predictable.

Hims’ $49 compounded semaglutide pill came and went almost instantly, but the signal it sent is more durable. The company briefly reset what people think an obesity pill should cost to under $100 a month, then got hit from both sides. FDA and HHS escalated scrutiny of compounded GLP-1 ingredients and mass marketing, and Novo filed a patent suit that aims to shut down copycat versions in both pill and injectable forms. Novo and Lilly now have to explain why the real thing costs more after a $49 almost version showed up overnight, and FDA has to decide if compounding is still a temporary shortage fix or if it is turning into a permanent side door to the same drugs.

BridgeBio’s Phase 3 in achondroplasia is the cleanest win this week. Statistically strong growth data plus a meaningful nudge toward body proportionality, packaged as an oral, mechanism-targeted option that fits real life better than injections. Upstream’s asthma data is the opposite lesson. The drug worked, but the stock collapsed because the best result points to more frequent dosing, which weakens the long acting convenience thesis investors were underwriting. And Regenxbio’s CRL is the sobering counterpoint to all of it. In ultra rare diseases, regulators can still demand cleaner population definition, better controls, and more convincing surrogate endpoints, even when the human need is obvious and the development story is a decade long.

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BIOTECH/PHARMA NEWS 🧬

🔹 Sanofi pushed out CEO Paul Hudson, with his last day set for Feb. 17, and named Merck KGaA chief Belén Garijo as the next CEO, effective after the Apr. 29 shareholder meeting, with Olivier Charmeil serving as interim. The board is effectively admitting the R&D reboot has been too slow, with Dupixent still doing the heavy lifting after underwhelming pipeline updates and with vaccine sales facing added pressure in the U.S. Shares were down about 3.5% on the day as investors weighed Garijo’s lower profile and mixed R&D track record against the clock ticking on the next growth engine.

🔹 AbbVie sued HHS after CMS picked Botox for the next round of Medicare Part D price negotiations, arguing the agency crossed a statutory line rather than just picking another political fight. AbbVie’s cleanest hook is plain text. Botox contains human serum albumin sourced from human blood plasma, so it should qualify as a plasma-derived product that Congress excluded from the IRA negotiation program. The company is also repeating familiar constitutional arguments that have largely failed for other drugmakers, including that the program compels speech by forcing companies to describe the negotiated price as fair and that it amounts to an unlawful taking. Botox is not just a cosmetic vanity line item either, it is used in migraines and other therapeutic indications and generated about $4.7B in U.S. sales in 2025, which helps explain why AbbVie is willing to litigate even as it has already modeled some pricing pressure into longer term forecasts.

🔹 Regenxbio shares slid after FDA issued a complete response letter for RGX-121 (clemidsogene lanparvovec), its one time gene therapy for Hunter syndrome, knocking the accelerated approval bid off track and sending the stock down about 15% premarket. The agency questioned whether the study eligibility criteria cleanly captured the neuronopathic population, whether the natural history external control was truly comparable, and whether the cerebrospinal fluid biomarker CSF HS D2S6 is an acceptable surrogate endpoint reasonably likely to predict clinical benefit. The letter lays out a menu of painful options in an ultra rare disease, including a new study, dosing more patients with longer follow up, or adding an untreated control arm, and the company says it will request a Type A meeting and try to resubmit with additional expert input and longer term clinical data.

🔹 Moderna said FDA’s CBER issued a Refusal to File letter for its investigational mRNA flu vaccine mRNA-1010, meaning the agency will not even start reviewing the BLA, despite Moderna using a Priority Review Voucher and despite no cited safety or efficacy concerns. CBER’s stated reason was the phase 3 comparator. Moderna used a licensed standard dose flu shot, and FDA said that did not reflect the “best available standard of care” in the US, which Moderna argues contradicts earlier FDA feedback and the fact that it also submitted supportive older adult data including a separate phase 3 comparison versus a high dose vaccine in 65+. Moderna has requested a Type A meeting to clarify the path forward, says its 2026 guidance is unchanged, and notes mRNA-1010 has been accepted for review in the EU, Canada, and Australia. If comparator standards can shift this late, sponsors either overbuild trials to avoid getting bounced, or they stop taking the risk at all and shift investment elsewhere. On Wednesday, STAT added an important detail. Three FDA officials told STAT that career reviewers were prepared to proceed and that David Kaslow, head of the vaccine office, wrote a memo arguing the agency should start the review, but CBER director Vinay Prasad overruled staff and signed the refusal memo himself, which is unusual and shifts this from a routine filing dispute to a signal about who is setting the bar and how predictable that bar is.

🔹 Hims pulled its $49 compounded semaglutide pill almost immediately, and now Novo is escalating the fight in court. Novo filed a patent infringement lawsuit against Hims tied to Wegovy in both pill and injectable forms, asking for a permanent ban on compounded versions that Novo says infringe its patents and seeking damages. The legal move lands on top of the regulatory squeeze already building: the FDA signaled a broader crackdown on compounded GLP-1 ingredients and mass marketed “copycats,” and the U.S. HHS said it referred Hims to the Department of Justice for potential violations. Hims is trying to flip the politics, calling the suit a “blatant attack” on Americans who rely on compounded meds and framing it as Big Pharma using the courts to limit choice. Novo rose about 5% and Hims dropped roughly 18% to 20%.

🔹 Eli Lilly and Innovent just signed a new oncology and immunology R&D partnership. Innovent will run discovery through phase 2 proof of concept in China, then Lilly gets an exclusive license to develop and commercialize worldwide outside Greater China, while Innovent keeps Greater China rights. The money is big: $350M upfront plus up to about $8.5B in milestones, plus tiered royalties. It is also their seventh deal together, which reads less like a one off and more like Lilly formalizing Innovent as a repeatable early development engine. The tell will be how many programs are actually included and how quickly they move from China phase 2 into global phase 3.

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CLINICAL TRIAL UPDATES 📊

🔹 BridgeBio popped after its pivotal phase 3 PROPEL 3 trial of oral infigratinib in children with achondroplasia hit its primary endpoint at 52 weeks, improving annualized height velocity vs placebo with a least squares mean treatment difference of +1.74 centimeters per year and a mean treatment difference of +2.10 centimeters per year. The study also cleared a key secondary endpoint on height Z-score (achondroplasia reference population), and the more interesting nuance is the exploratory readout on body proportionality. In children under 8 years old, infigratinib showed the first statistically significant improvement versus placebo on an upper to lower body proportionality measure, which gets closer to the real lived burden of the disease than raw height alone. Safety looked clean for a year long pediatric study with no discontinuations or serious adverse events tied to drug, and only three mild, transient hyperphosphatemia cases without dose changes. BridgeBio is now aiming for second half of 2026 submissions in the U.S. and EU and is accelerating a phase 3 path in hypochondroplasia, positioning this as a potentially best in class, needle free, mechanism targeted option in a market currently defined by injections and tradeoffs.

🔹 Upstream Bio shares got cut in half even as its phase 2 VALIANT trial in severe asthma because the efficacy landed hardest on the more frequent dosing schedule investors were not hoping for. Verekitug, a monoclonal antibody that blocks the thymic stromal lymphopoietin receptor, met the primary endpoint by reducing the annualized asthma exacerbation rate, with a 56% reduction on 100 mg every 12 weeks and a 39% reduction on 400 mg every 24 weeks versus placebo, plus clinically meaningful improvements in lung function and exhaled nitric oxide on both regimens. The market issue is the product profile because the company has been selling verekitug as a long acting, low burden biologic, so the read through is that the most compelling efficacy may come with quarterly injections rather than a true twice yearly convenience story. Safety looked clean, with adverse events mostly mild to moderate and more than 90% of eligible patients rolling into the long term extension.

🔹 Priovant Therapeutics, the Pfizer and Roivant-created autoimmune biotech, posted unusually clean phase 2 data for brepocitinib in cutaneous sarcoidosis, a disfiguring inflammatory skin disease with no FDA-approved therapies. In the 31-patient BEACON trial, the drug drove a 22.3 point mean improvement at week 16 on the Cutaneous Sarcoidosis Activity and Morphology Instrument Activity score versus a 0.7 point change on placebo, and every patient on brepocitinib hit at least a 10 point improvement versus 14% on placebo (with separation showing up as early as week 4). On a tougher clinician bar, 69% reached “clear” or “almost clear” on the Investigator’s Global Assessment compared with 0% on placebo, with all adverse events graded mild to moderate. Management plans to take the package to the FDA and start a phase 3 program in 2026, adding a third pivotal track for a dual TYK2 and JAK1 inhibitor that is already further along in dermatomyositis and non-infectious uveitis.

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VIDEO SUMMARY

THIS WEEK’S KEY TAKEAWAYS 🔑

Hims & Hers escalated the GLP-1 pricing fight by launching a compounded semaglutide pill at direct-to-consumer prices ($49 first month, then $99/month). Novo Nordisk went straight to illegal copycat framing, with the FDA commissioner publicly warning the agency will move against mass marketed compounded products that imply equivalence to approved drugs. I don’t think the question is “will people buy it?” They will. The question is what patients think they are buying, and how long this can exist before enforcement and lawsuits show up. If regulators let this stand, every future obesity pill becomes a price war on day one. If they don’t, this pressure doesn’t go away, it just moves to the next loophole.

Novo’s downbeat 2026 outlook, guiding sales and operating profit down 5% to 13%, put a big red circle around US net price erosion. Lilly got the opposite reaction. A strong quarter and bullish guidance reminded investors what it looks like when volume can still outrun price pressure. Lilly also kept pushing the access story with the $50 a month Medicare copay headline and the rise of Zepbound vials for self pay, a smart distribution move that also shows where the category is headed (simpler, cheaper entry points).

Outside obesity, the same theme showed up in different costumes: middlemen and cliffs. CVS, through its pharmacy benefit manager, is explicitly using formularies to accelerate the next biosimilar wave by swapping branded bone drugs for cheaper alternatives starting 4/1, building on its Humira playbook. Novartis, meanwhile, is staring at a patent cliff year and telling you up front that generics will chew a multi billion dollar hole in sales, so 2026 becomes a live test of whether its newer growth brands can compound fast enough to cover the gap.

On the clinical side, Ultragenyx posted durable long term gene therapy data in Sanfilippo syndrome type A, and with its resubmission in, the next real catalyst is the FDA decision expected in Q3 2026.

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BIOTECH/PHARMA NEWS 🧬

🔹 Hims & Hers said it will offer a compounded semaglutide pill for weight loss at $49 for the first month and $99/month after that on a five month plan, immediately reigniting the GLP-1 pricing war narrative. Novo Nordisk said the move is illegal mass compounding and promised legal and regulatory action, while FDA Commissioner Makary warned the agency will move against companies mass marketing “illegal copycat drugs” that imply equivalence to FDA approved medicines. Novo fell about 8% and Lilly about 6%, and Hims reversed and was down about 10% after hours after the FDA comments. Novo’s CEO also sharpened the scientific and messaging fight, calling the $49 pill a “waste of money” and arguing its branded oral depends on proprietary absorption tech, while Hims says it uses a different approach to support absorption. Consumers want oral GLP1s at DTC prices, and the first company that can deliver that inside the rules will own the next leg of the category.

🔹 Alvotech cleared a key de-risking step on an Entyvio biosimilar for inflammatory bowel disease. Its pivotal PK study for AVT80 (subcutaneous vedolizumab) hit primary endpoints in healthy volunteers, supporting planned filings for both its SC (AVT80) and IV (AVT16) versions of a biosimilar to Takeda’s $6.4B Entyvio franchise used in ulcerative colitis and Crohn’s disease.

🔹 CVS is using its commercial formularies to force the next biosimilar wave. Starting April 1, it plans to prefer denosumab biosimilars over Amgen’s Prolia and to prefer teriparatide generics over Lilly’s Forteo, framing it as 50%+ lower cost per prescription and a continuation of the pharmacy benefit manager playbook that already pushed most Humira users onto biosimilars.

🔹 The FDA hit Aquestive with a CRL for Anaphylm (sublingual epinephrine film) over human-factors and packaging use issues, not the drug itself. The agency flagged problems like difficulty opening the pouch and incorrect film placement in an emergency setting. Importantly, no CMC deficiencies were cited, and management says it can run an updated human-factors validation plus a small PK bridge study in parallel and resubmit as early as Q3 2026, keeping EU and Canada filings on track for 2H 2026.

🔹 Eli Lilly had its best day in 9 months after a Q4 beat and a bullish 2026 guide, with investors also keying on management’s framing of a $50/month Medicare copay for obesity drugs as a demand catalyst as early as July 1. On the call, Lilly highlighted growth in its LillyDirect channel (over 1M users added in 2025) and said Zepbound vials are now a meaningful entry point for self-pay, representing about one third of new patient starts. The company acknowledged pricing headwinds in 2026 but argued volume expansion and smoother access pathways can offset the hit.

🔹 Novartis is bracing for its biggest patent cliff in years and expects roughly $4B of generic-driven sales erosion in 2026. Guidance calls for low single digit sales growth but a low single digit decline in core operating profit, with Entresto already pressured in Q4 by U.S. generics and higher revenue deductions. The debate for 2026 is straightforward: can growth brands like Kisqali, Kesimpta, Pluvicto and Scemblix compound fast enough to outrun the hole as legacy exclusivity fades.

🔹 Novo Nordisk sold off after it pre-released a downbeat 2026 outlook, guiding sales and operating profit down 5% to 13%. The issue is not demand for GLP-1s, it is U.S. net pricing, where competition, discounts, and compounded semaglutide are tightening economics into a price fight. Novo pointed to strong early traction for its oral Wegovy launch, but the market is treating that as incremental volume that does not yet offset repricing of the base business.

🔹 AbbVie beat Q4 estimates, but the stock dropped anyway because the beat came from Humira, not Rinvoq, its newer immunology drug. Humira reported about $1.25B vs $0.99B expected, while Rinvoq and Skyrizi were essentially on plan (Rinvoq $2.37B vs $2.38B; Skyrizi $5.01B vs $4.9B). When a declining product supplies the upside, the market discounts it. Investors want the handoff to Rinvoq/Skyrizi to outperform.

🔹 Regeneron is keeping the cash engine running while pushing a heavy 2026 pipeline cadence. Q4 revenue rose 3% to $3.9B as Dupixent (Sanofi-reported) grew 34% to $4.9B and Eylea HD U.S. sales climbed to $506M, even as legacy Eylea continues to slide. Management expects at least four FDA decisions in 2026 and plans 18 new phase 3 starts, while guiding higher R&D spend and warning Eylea biosimilar pressure could intensify in 2H 2026.

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🔹 Ultragenyx shared long term UX111 data in Sanfilippo syndrome A showing durable biomarker suppression and sustained separation from natural history out to 8.5 years. CSF heparan sulfate fell 64% median (most >50%), and earlier-treated children showed a +23.2-point Bayley III cognitive raw score benefit versus comparators, with supportive communication/motor trends. Safety remained consistent (mainly liver enzyme elevations). With a July 2025 CRL and a late January resubmission, this sets up a potential Q3 2026 FDA decision if timelines hold.

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DISCLAIMER: This publication is for informational and educational purposes only and does not constitute investment, legal, medical, or tax advice, a solicitation, or an offer to buy/sell any security. Information is believed reliable but no warranty is made as to accuracy or completeness; views may change without notice. Do your own research and consult qualified professionals.

DISCLOSURE & CONFLICTS: The author may hold positions in securities mentioned and may change positions at any time without notice. No compensation is received from companies mentioned, and there are no known material business relationships unless explicitly stated. Content may reference clinical data and regulatory events; always consult primary sources and a licensed clinician for medical decisions. Past performance is not indicative of future results.

LIABILITY: Use of this content is at your own risk. The author assumes no responsibility for any losses arising from reliance on this material.

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VIDEO SUMMARY

THIS WEEK’S KEY TAKEAWAYS 🔑

This week showed how quickly “defensive” biopharma revenue stops looking defensive when policy and patient behavior shift at the same time. Sanofi basically said US vaccine demand is soft because patients are confused and uptake is falling. Flu and Covid held up, but Beyfortus (pediatric RSV antibody) and pediatric combos didn’t, and management is guiding to slightly negative vaccine growth in 2026. The sector’s most reliable cash flows are now exposed to the same forces as everything else: trust, messaging, and who sets the default in the clinic. This week made it uncomfortably clear that even the safest looking franchises depend on fragile inputs like federal guidance and public trust.

That trust theme showed up again in gene therapy, but with even sharper edges. The FDA froze two Regenxbio AAV programs after a brain tumor was found on routine MRI in one child treated 4 years ago, with causality still undetermined. The scientific signal regulators can’t ignore is the reported vector integration event tied to PLAG1 overexpression, even if it ends up being coincidence. The human reality families can’t ignore is the other math: 41 treated patients with years of clean follow-up versus one uncertain case, and a pause that reportedly hit one program just days from a potential FDA decision. The correct move may still be “hold,” but the standard should be speed and specificity - what data would actually distinguish causality from background risk, and what’s the fastest path to generate it without turning rare disease into collateral damage.

This week, CMS named 15 new drugs for Medicare price negotiation for 2028, and for the first time the list includes Part B drugs, not just pharmacy (Part D). That’s not a symbolic change, but a widening funnel for pricing pressure onto large, durable franchises (think Botox, Xolair, Cosentyx, Biktarvy, Trulicity). If you hold mature winners, you now have to underwrite not just competition and patent cycles, but government’s growing willingness to set prices and live with the consequences.

Innovation delivered both relief and reality. Intellia got the FDA to lift the hold on its ATTR polyneuropathy study after proposing tighter liver monitoring, but the cardiomyopathy program remains on hold, and that is where the upside lives. Roche’s CT-388 looks legitimately competitive, with weight loss still trending at 48 weeks, which is enough to justify Phase 3. Investors barely blinked because the bar in obesity has moved. Now the fight is tolerability, durability, body composition, and who can actually get reimbursed.

AstraZeneca’s $15B China push is a clear bet on speed: faster trials, deeper manufacturing, and a dense partnering ecosystem. The uneasy part is that scale cuts both ways. It can accelerate the pipeline, but it also increases exposure to policy shifts that can show up suddenly and matter a lot.

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BIOTECH/PHARMA NEWS 🧬

🔹 Sanofi’s vaccine franchise is hitting a real demand headwind in the US: management said “confusion” around vaccination and falling uptake is showing up in results, with Q4 vaccine sales down 2.5% and the company guiding to slightly negative vaccine growth in 2026, even as influenza vaccines beat expectations during a heavy flu season. The mix matters: flu and COVID vaccines were up (Q4 +31.5%), but Beyfortus (RSV antibody) fell (Q4 -14.9%) and core pediatric combos (polio, pertussis, Hib) also declined (Q4 -9.5%), leaving total vaccine sales weaker despite broader company momentum (FY 2025 sales +9.9% at constant exchange rates; Dupixent +25.2% to €15.7B). Sanofi is still leaning into vaccines strategically, pointing to the Vicebio acquisition and the pending Dynavax deal as a bet that today’s demand wobble is cyclical not structural, but the near-term setup is straightforward: if US uptake stays soft, “defensive” vaccine revenues look less defensive, and you should expect more emphasis on adult immunization, international rollout, and pipeline optionality to offset policy-driven noise.

🔹 AstraZeneca said it will invest $15B in China through 2030 to expand R&D and manufacturing, leaning into China as both its second largest market and a faster innovation engine. The plan spans drug discovery, clinical development and new and expanded sites across Wuxi, Taizhou, Qingdao and Beijing, with an explicit push into higher value modalities like cell therapy and radioconjugates and more partnering with local biotechs, all announced alongside Keir Starmer’s Beijing visit. Read it as a bet that China speed plus AZ execution can keep the pipeline fed, but it also raises the obvious geopolitical and policy headline risk of building deeper dependence on a strategic hub.

🔹 Regenxbio reported a delayed safety signal that regulators have to treat as potentially platform-wide until proven otherwise. The FDA put RGX-111 on clinical hold after a routine brain MRI found an intraventricular central nervous system tumor in a 5 year old who had been dosed about four years earlier, and early analysis flagged an AAV vector genome integration event linked to overexpression of a proto-oncogene (PLAG1), even though causality is not established and the child is asymptomatic. The agency then extended the hold to RGX-121 as a precaution because of product and population similarities, despite RGX-121 having a long follow-up story in more than 30 patients, including some treated nearly seven years ago.

🔹 CMS just named the next 15 drugs headed into Medicare price negotiations for 2028, and this round matters because it pulls in Part B drugs for the first time, not just pharmacy (Part D) products. The list spans some of the biggest “known knowns” in biopharma spend, including Trulicity, Biktarvy, Cosentyx, Botox, Xolair, and Xeljanz (plus others like Entyvio, Kisqali, Verzenio, Lenvima, and Orencia). Collectively, these drugs accounted for about $27.0B in Medicare Part B and Part D spending over Nov 2024 to Oct 2025, across roughly 1.78M Medicare users, so the intent is clearly to keep widening the aperture of where pricing pressure can land.

🔹 Intellia got a partial green light on its in vivo CRISPR program nexiguran ziclumeran (“nex-z”) in transthyretin amyloidosis: the FDA lifted the clinical hold on MAGNITUDE-2 (the polyneuropathy study) after Intellia proposed new risk mitigation steps, including enhanced liver lab monitoring, and bumped planned enrollment to about 60 patients. The bigger overhang remains MAGNITUDE (the cardiomyopathy study), which is still on hold, and that is the real value driver. The pause traces back to October 29, 2025, after a patient dosed in MAGNITUDE developed severe liver toxicity meeting the protocol’s stopping criteria and subsequently died, so the market is essentially waiting to see what the FDA will require for the older, higher-risk cardiomyopathy population.

🔹 On January 26, The American Academy of Pediatrics (AAP) published its 2026 schedule and, for the first time in decades, it is no longer fully harmonized with the federal schedule. The headline divergence is simple: AAP continues to recommend routine COVID-19 and influenza vaccination starting at six months, while the updated CDC schedule shifts COVID-19 and flu into “shared clinical decision making” rather than a routine recommendation. For biopharma, it is less about new science and more about a messy implementation year. Pediatricians and states are already signaling they will lean on AAP guidance (for example, California has explicitly told providers to follow AAP’s schedule). Vaccine demand becomes more geography- and provider-driven, which raises U.S. policy risk for Covid and flu franchises (Moderna, Pfizer/BioNTech, Novavax; and the flu complex) even if AAP keeps the medical “default” intact in much of clinical practice.

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🔹 Roche posted encouraging phase 2 obesity data for CT-388, a once-weekly dual GLP-1/GIP agonist acquired via the Carmot deal. In patients who stayed on the regimen for 48 weeks, placebo-adjusted weight loss reached 22.5% at the highest dose. Using a more real world analysis that counts people who missed doses or fell off schedule, the placebo-adjusted effect was 18.3%. Roche highlighted that weight loss did not appear to plateau by week 48 and said the readout validates two phase 3 trials slated to start this quarter, though investor reaction was muted given how crowded the category is and how far a launch still is.

🔹 Sarepta reported positive topline three year functional results from Part 1–treated patients in the phase 3 EMBARK study of Elevidys in ambulatory Duchenne muscular dystrophy. Elevidys-treated patients had mean North Star Ambulatory Assessment (NSAA) scores above baseline at year 3 while the external controls declined. On timed function, Sarepta said Elevidys showed a 73% slowing of decline on time-to-rise (TTR) and about 70% slowing on the 10 meter walk/run (10MWR), with the functional gap versus external controls widening between year 2 and year 3. The company said no new treatment-related safety signals were observed in this ambulatory dataset.

PUBLIC HEALTH SPOTLIGHT 🔍

🔹 Two confirmed Nipah infections in India’s West Bengal, reportedly in healthcare workers, triggered tighter border health measures across parts of Asia. Officials traced 196 contacts and said all were asymptomatic and tested negative, so near term public risk is still considered low. Nipah is a bat linked zoonotic virus that can cause encephalitis and has had high fatality rates in past outbreaks (often cited around 40% to 75%), but it typically requires prolonged close contact and does not spread easily. With no approved vaccine or specific treatment, the real takeaway is platform readiness: surveillance and sequencing, rapid diagnostics, infection control capacity, and vaccine R&D that can move quickly from sequence to clinic.

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DISCLAIMER: This publication is for informational and educational purposes only and does not constitute investment, legal, medical, or tax advice, a solicitation, or an offer to buy/sell any security. Information is believed reliable but no warranty is made as to accuracy or completeness; views may change without notice. Do your own research and consult qualified professionals.

DISCLOSURE & CONFLICTS: The author may hold positions in securities mentioned and may change positions at any time without notice. No compensation is received from companies mentioned, and there are no known material business relationships unless explicitly stated. Content may reference clinical data and regulatory events; always consult primary sources and a licensed clinician for medical decisions. Past performance is not indicative of future results.

LIABILITY: Use of this content is at your own risk. The author assumes no responsibility for any losses arising from reliance on this material.

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VIDEO SUMMARY

THIS WEEK’S KEY TAKEAWAYS 🔑

This week felt like the exhale after the JPMorgan Healthcare Conference, with fewer headlines than typical.

First, Moderna and Merck added real long term follow up to the personalized neoantigen mRNA vaccine story in melanoma. A five year follow up that still shows about a 49% reduction in the risk of recurrence or death is the kind of durability clinicians respect and investors will actually underwrite. Moderna entered 2026 under heavy skepticism and elevated short interest, and the stock is starting to catch momentum as management leans harder into oncology and cardiovascular programs while stepping back from funding new phase 3 vaccine studies in the current US policy climate.

On the deal side, GSK is making a clean product profile bet in food allergy. The wager is that ozureprubart can match the anti-IgE effect that Xolair (Genentech and Novartis) established, while stretching dosing to about once every 12 weeks. If that holds, it is a meaningful quality of life win for kids and parents, and a very classic acquisition logic (validated target, better dosing, bigger addressable market). In parallel, BMS’s Janux partnership is another sign that tumor activated T cell engagers still draw big pharma interest. The deal structure is also pretty standard for early oncology programs as Janux runs the preclinical work through the IND, BMS takes over once it is ready for the clinic, and most of the economics sit in milestones and royalties tied to success.

Finally, two clinical updates are worth flagging. Corcept said its Phase 3 ROSELLA study hit overall survival in platinum resistant ovarian cancer, with no biomarker requirement and no meaningful added safety burden versus chemotherapy alone, setting up a high visibility FDA decision in July. Corvus also put up notably strong phase 1 cohort 4 atopic dermatitis data for an oral agent in a placebo controlled cohort, with responses deepening through eight weeks and a clean early safety profile, which is exactly the kind of package that earns a serious phase 2 run.

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BIOTECH/PHARMA NEWS 🧬

🔹 Bristol Myers Squibb signed a tumor activated T cell engager deal with Janux Therapeutics worth up to about $850M ($50M upfront and near term, plus up to roughly $800M in milestones and tiered royalties). Janux will take the asset through preclinical work up to the IND application, then BMS will run the human trials and commercialization, with Janux staying involved through completion of the first phase 1 study. The target is not disclosed, but BMS says it is a validated solid tumor antigen expressed across multiple cancer types. The timing is notable for Janux after a choppy stretch in late 2025 when phase 1 data for its lead program disappointed investors, and for BMS, which has been active in T cell engager partnering but has also walked away from prior initiatives.

🔹 GSK is buying RAPT Therapeutics for about $2.2B, paying $58 per share in cash in a deal expected to close in Q1 2026, and it is basically a one asset bet on making anti-immunoglobulin E (anti-IgE) therapy for food allergy far less burdensome. The prize is ozureprubart, a long acting anti-IgE monoclonal antibody in phase 2b for prophylactic protection against food allergens, aiming to rival Xolair, the first FDA approved medicine to reduce allergic reactions from accidental food exposure, but with dosing every 12 weeks instead of every two to four. Phase 2b data is expected in 2027, with phase 3 plans anticipated, and GSK gets rights outside mainland China, Macau, Taiwan, and Hong Kong.

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CLINICAL TRIAL UPDATES 📊

🔹 Moderna and Merck reported long term weight behind the personalized mRNA cancer vaccine story, reporting five year follow up from KEYNOTE-942 in high risk resected stage 3 and 4 melanoma where intismeran autogene (V940, also called mRNA-4157) plus Keytruda cut the risk of recurrence or death by 49% versus Keytruda alone (HR 0.510, 95% confidence interval 0.294 to 0.887), basically matching the three year effect. Each dose is built from a patient’s tumor mutations and encodes up to 34 neoantigens to train T cells to hunt residual disease. Moderna and Merck are now trying to scale this into a post-Covid platform with eight phase 2 and phase 3 studies across tumors including non-small cell lung cancer, bladder cancer, and renal cell carcinoma, with adjuvant melanoma phase 3 fully enrolled and more analyses ahead. Wall Street is already pricing the optionality, with chatter about Keytruda-like pricing around $200k and multibillion peak melanoma sales if phase 3 holds, but the real gate is operational. Can personalized manufacturing run fast and reliably enough to fit oncology workflows?

By late 2025 Moderna was the most shorted stock in the S&P 500 by percent of float, and now 2026 is starting with momentum too, with shares up about 29% over the past five sessions and printing a new 52 week high even as the CEO said Moderna will not fund new vaccine phase 3 programs for now amid US policy and regulatory pushback, instead leaning harder into cancer and heart disease.

Here is a link to last year’s deep dive on Moderna’s neoantigen vaccines:

🔹 Corcept says its phase 3 ROSELLA trial in platinum resistant ovarian cancer hit overall survival: relacorilant plus nab paclitaxel cut the risk of death by 35% versus nab paclitaxel alone (HR 0.65, p=0.0004), with median overall survival 16.0 months versus 11.9 months, a 4.1 month gain, and no meaningful increase in adverse events versus chemo alone. ROSELLA also met its other dual primary endpoint, progression free survival by blinded independent central review, with a 30% reduction in risk of progression (HR 0.70, p=0.008), and the company is leaning into a key commercial angle: no biomarker selection required. The NDA is already under FDA review with a PDUFA date of July 11, 2026, and the EMA application is also under review, setting up a high visibility mid year decision point.

🔹 Corvus dropped phase 1 Cohort 4 data for soquelitinib in moderate to severe atopic dermatitis and the numbers are punchy for an oral immunology drug in a small, placebo controlled set. In Cohort 4, patients were randomized 1:1 to soquelitinib 200 mg twice daily or placebo for 56 days. By day 56, mean EASI improvement was 72% on drug versus 40% on placebo (p=0.035), and the separation widened from day 28 to day 56. On responder endpoints, 75% of soquelitinib patients hit EASI 75, 25% hit EASI 90, and 33% reached IGA 0 or 1, versus 20%, 0%, and 0% on placebo. Corvus also pointed to real world relevance: responses looked similar in patients who had prior systemic therapy, including those described as resistant to dupilumab and JAK inhibitors, which is the population where a new oral option can realistically take share. Safety looked clean, with no new safety signals, no severe or serious adverse events, and only Grade 1 to 2 adverse events without dose interruptions. That tees up a phase 2 start in Q1 2026, expected to enroll about 200 patients.

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DISCLAIMER: This publication is for informational and educational purposes only and does not constitute investment, legal, medical, or tax advice, a solicitation, or an offer to buy/sell any security. Information is believed reliable but no warranty is made as to accuracy or completeness; views may change without notice. Do your own research and consult qualified professionals.

DISCLOSURE & CONFLICTS: The author may hold positions in securities mentioned and may change positions at any time without notice. No compensation is received from companies mentioned, and there are no known material business relationships unless explicitly stated. Content may reference clinical data and regulatory events; always consult primary sources and a licensed clinician for medical decisions. Past performance is not indicative of future results.

LIABILITY: Use of this content is at your own risk. The author assumes no responsibility for any losses arising from reliance on this material.

🎙️ Biotech Blueprint brings you weekly video updates on the latest biotech and pharma news, plus in-depth podcast interviews with industry leaders. You can find us on YouTube, Spotify, and Apple Podcasts.

SPONSOR CONTENT BY ARCHES DATA

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VIDEO SUMMARY

THIS WEEK’S KEY TAKEAWAYS 🔑

Washington is back in the drug pricing conversation. President Trump’s “Great Healthcare Plan” wants to pull some U.S. drug prices closer to what other wealthy countries pay through “most favored nation” pricing, squeeze middlemen economics by targeting pharmacy benefit manager kickbacks, and push transparency so patients can actually see prices and coverage in plain English. The part to watch is execution as lower prices only translate into better access if savings show up at the pharmacy counter and in out of pocket costs.

NVIDIA and Eli Lilly are building an AI co-innovation lab and planning up to one billion dollars over five years for talent, compute, and infrastructure. The pitch is a closed loop engine that links automated wet labs with modeling so experiments generate data, data improves models, and models choose the next experiments.

Eikon’s initial public offering filing is an early sign that the market may be warming to new biotech listings again, with the real test coming at pricing and trading. In parallel, Beam laid out a cleaner 2026 plan with a longer runway into 2029 and a stated path toward a biologics license application filing for its sickle cell program as early as the end of 2026. The common thread is that the market is rewarding credible timelines again, but only when the milestones are concrete and close enough to touch.

And none of it matters until regulators say it does. Atara took a second Complete Response Letter for Ebvallo, with the agency now saying the single arm dataset is no longer sufficient to demonstrate efficacy, even as manufacturing issues were described as resolved. On the flip side, ImmunityBio is pairing real commercial momentum with fresh lung cancer proof points: rising Anktiva revenue in bladder cancer, new Saudi approvals for lung cancer and bladder cancer, and updated lung cancer data that supports the company’s argument that Anktiva can help restore immune competence when added to checkpoint inhibitors.

Gene therapy put up more evidence that it can compete in large indications, not just ultra rare ones. Ocugen posted an interim look in geographic atrophy with lesion growth reduction and clean early safety, while Krystal showed delivery and expression signals in the lung for cystic fibrosis. Regenxbio added longer follow up in Duchenne and kept its accelerated approval timeline in view. The message is breadth as delivery is improving and the modality is creeping into mainstream markets.

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🔹 Trump unveiled the “Great Healthcare Plan,” a broad framework that asks Congress to do three things: lock in “most favored nation” drug pricing deals meant to bring some United States prescription prices closer to what other wealthy countries pay, change exchange subsidies by sending assistance directly to eligible people rather than insurers while also funding the program that lowers out of pocket costs for some lower income enrollees, and force more transparency and less middleman economics by targeting pharmacy benefit manager kickbacks and requiring insurers and Medicare or Medicaid participating providers to post clearer pricing and coverage information.

President Trump’s Great Healthcare Plan is a framework urging Congress to lock in his Most Favored Nation drug pricing agreements, expand over-the-counter access for verified safe medicines, and redirect federal subsidy dollars away from insurers and directly to eligible Americans. It also proposes funding the Affordable Care Act cost-sharing reduction program, which the White House says would save taxpayers at least $36B and cut premiums for the most common Obamacare plans by over 10% per the Congressional Budget Office, while banning pharmacy benefit manager kickbacks that flow to brokerage middlemen. The plan would require insurers to present coverage and rates in plain English and disclose claim payouts versus profits, denial rates, and routine-care wait times, and it would require any provider or insurer that takes Medicare or Medicaid to post pricing and fees prominently.

🔹 NVIDIA and Eli Lilly are launching an AI co-innovation lab in the Bay Area and plan to invest up to $1B over five years in talent, compute, and infrastructure. The lab will use NVIDIA BioNeMo and next-gen NVIDIA architecture (Vera Rubin) to build a “closed-loop” discovery setup that links automated wet labs with computational modeling so experiments and models continuously improve each other. They also plan to push robotics and physical AI beyond discovery into manufacturing, using tools like digital twins to optimize production and supply chains.

🔹 ImmunityBio paired commercial momentum with more lung cancer proof points: the company said preliminary Q4 2025 net product revenue was $38.3M (up 20% vs. Q3 and 431% year over year), driven by continued Anktiva uptake in bladder cancer, with full-year 2025 preliminary net product revenue of $113M (the company says 700% year over year) and unit volume up 750% vs. 2024. It ended 2025 with $242.8M in cash and marketable securities and flagged recent Saudi approvals as incremental catalysts, including Anktiva plus a checkpoint inhibitor for metastatic non-small-cell lung cancer and Anktiva plus standard bladder therapy for high risk, treatment resistant non-muscle invasive bladder cancer. On the clinical side, updated lung cancer data suggested Anktiva may help rebuild immune competence when added to checkpoint inhibitors like Keytruda or Opdivo: in a first-line study, the combo increased absolute lymphocyte counts vs. checkpoint inhibitor alone, and in a later-line single-arm study, 77% of patients restored or maintained lymphocyte counts above a predefined threshold, with greater lymphocyte recovery tracking with longer survival.

🔹 Fortress jumped after the FDA approved Zycubo (copper histidinate, formerly CUTX-101), the first U.S.-approved copper replacement therapy for pediatric Menkes disease, a rare and often fatal disorder. Under Cyprium’s deal with Sentynl (Zydus), Cyprium will receive tiered royalties on net sales plus up to $129M in milestones, and it will also receive the Rare Pediatric Disease Priority Review Voucher issued with the approval.

🔹 Atara sank after the FDA issued a second Complete Response Letter for Ebvallo (tabelecleucel) in EBV-positive post-transplant lymphoproliferative disease. This time, the FDA said the single arm ALLELE study is no longer adequate to show efficacy and that design, conduct, and analysis issues limit interpretability, even though the agency confirmed the prior GMP deficiency is now resolved and raised no new safety concerns. Atara and partner Pierre Fabre said they will seek a Type A meeting to clarify a path forward, but the decision reintroduces major regulatory uncertainty for a product already approved in Europe (as Ebvallo).

🔹 Beam Therapeutics rallied after outlining a cleaner 2026 plan and longer cash runway. It said it ended 2025 with about $1.25B in cash and expects funding into 2029, supporting a biologics license application filing for risto-cel in sickle cell disease as early as year end 2026. Beam also said it aligned with the FDA on a potential accelerated approval path for BEAM-302 in alpha-1 antitrypsin deficiency using biomarker endpoints, with updated phase 1/2 data and a pivotal plan due by the end of the first quarter of 2026, plus more 2026 data for BEAM-301 and a new liver program reveal in the first half of 2026.

🔹 Moderna’s stock has pushed back above $40 per share, a level it only touched briefly in January 2025 and otherwise has not held since late 2024. The move came as the company reiterated a 2026 plan for up to 10% revenue growth by building a “seasonal vaccine” business, while continuing aggressive cost cuts and positioning its cash engine to fund the next wave of oncology and rare disease programs. Management also pointed to about $1.9B in expected 2025 revenue, a lower 2025 operating expense outlook, and an estimated about $8.1B year end cash balance, with a stated goal of reaching cash breakeven in 2028, plus multiple 2026 clinical and regulatory catalysts. CEO Stéphane Bancel framed it as stabilization plus discipline: fewer expenses, a clearer commercial lane, and enough runway to keep taking shots on the pipeline.

🔹 Eikon Therapeutics filed on January 9 to go public on Nasdaq under EIKN, a decent read through on whether biotech’s rebound is strong enough to reopen the IPO window. The Merck-vet-led company (Roger Perlmutter, with Roy Baynes as CMO) is selling investors on a late stage oncology pipeline led by EIK1001, a systemic TLR7/8 agonist in a global phase 2/3 melanoma trial with pembrolizumab and additional NSCLC studies, plus PARP1 inhibitors EIK1003/1004 in phase 1/2 across multiple solid tumors, with proceeds aimed at advancing these programs through key 2026 milestones.

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CLINICAL TRIAL UPDATES 📊

🔹 Ocugen said its one-time gene therapy OCU410 for geographic atrophy (dry AMD) cut lesion growth by 46% at 12 months in an interim phase 2 look (about half of patients evaluated so far), with no treatment-related serious adverse events reported across phase 1/2. The company plans to share full phase 2 data later this year and start phase 3 in 2026.

🔹 Regenxbio shares rose after it reported new 18 month phase 1/2 data for its Duchenne gene therapy RGX-202. In four patients treated at the pivotal dose, all exceeded the expected disease trajectory on the North Star Ambulatory Assessment, improving an average of 7.4 points versus the established progression model (and 6.6 points at 12 months). Regenxbio said it expects pivotal topline results in early Q2 2026 and plans to file a BLA under the accelerated approval pathway in mid 2026.

🔹 Vir Biotechnology shared an update in chronic hepatitis delta: updated phase two SOLSTICE data showed its combination of tobevibart and elebsiran was well tolerated and drove hepatitis delta virus ribonucleic acid to “target not detected” in 88% of evaluable participants at 96 weeks, versus lower rates on antibody alone.

🔹 Krystal Biotech shared a positive interim update from its phase 1 CORAL-1 study of KB407, an inhaled gene therapy for cystic fibrosis designed to deliver normal, full length CFTR protein to the lungs. In the highest dose cohort, lung biopsies confirmed delivery and expression in all six patients with successful bronchoscopies, with about 29% to 42% of conducting airway cells showing transduction signals. Safety looked consistent with earlier cohorts, with mostly mild to moderate, transient adverse events and one serious asthma event judged related to the bronchoscopy procedure rather than the drug. Krystal has submitted the design for a registrational repeat-dosing study, CORAL-3, to the FDA and expects to start enrollment in the first half of 2026 after alignment.

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Have a great rest of your week and thanks for reading Biotech Blueprint!

DISCLAIMER: This publication is for informational and educational purposes only and does not constitute investment, legal, medical, or tax advice, a solicitation, or an offer to buy/sell any security. Information is believed reliable but no warranty is made as to accuracy or completeness; views may change without notice. Do your own research and consult qualified professionals.

DISCLOSURE & CONFLICTS: The author may hold positions in securities mentioned and may change positions at any time without notice. No compensation is received from companies mentioned, and there are no known material business relationships unless explicitly stated. Content may reference clinical data and regulatory events; always consult primary sources and a licensed clinician for medical decisions. Past performance is not indicative of future results.

LIABILITY: Use of this content is at your own risk. The author assumes no responsibility for any losses arising from reliance on this material.

🎙️ Biotech Blueprint brings you weekly video updates on the latest biotech and pharma news, plus in-depth podcast interviews with industry leaders. You can find us on YouTube, Spotify, and Apple Podcasts.

SPONSOR CONTENT BY ARCHES DATA

We help biotech CEOs and leadership teams pressure test strategy through rigorous data science, forecasting, and scenario analysis. From deep dive custom research to complex market modeling, we provide the evidence you need to lead with confidence.

Book a consultation HERE.

Visit our website: archesdata.com.

VIDEO SUMMARY

THIS WEEK’S KEY TAKEAWAYS 🔑

On Monday (Jan 5th) HHS said the CDC is reshaping the childhood immunization schedule, shifting several familiar vaccines out of “recommended for all” and into “high risk” or “shared decision making.” Even if insurance still covers everything, the behavioral effect is the point. More friction means fewer routine shots, more confusion for parents, and a higher chance of outbreaks. For vaccine makers and anyone building around prevention, this injects demand uncertainty straight into strategy.

On the data front, two readouts stood out because they can change standards of care. Jazz’s Ziihera delivered phase 3 first line results in HER2-positive gastroesophageal adenocarcinoma that beat trastuzumab plus chemotherapy on time before progression. The arm that added tislelizumab reached a median overall survival of 26.4 months. In immune disease, Alumis reported phase 3 plaque psoriasis results for envudeucitinib, an oral inhibitor of tyrosine kinase two, with unusually high rates of near complete and complete skin clearance by six months. The market cared because it is an oral drug posting efficacy that starts to resemble injectable biologics.

Chronic hepatitis B also got a meaningful catalyst. GSK and Ionis reported positive phase 3 topline results for bepirovirsen, showing a statistically significant lift in “functional cure” when added to standard nucleoside therapy. Translation: today’s care often means long term suppression with low cure rates. A finite course that can produce sustained loss of surface antigen and undetectable viral DNA would be a genuine shift, and GSK is already pointing to regulatory filings starting in the first quarter of 2026.

Mergers took center stage this week. Revolution Medicines became the takeover rumor of the week, with the narrative shifting from AbbVie to Merck and a floated $28 to $32 billion range, though nothing is signed and neither company is confirming. Separately, Amgen’s up to $840 million buyout of preclinical Dark Blue Therapeutics and Lilly’s up to $950 million collaboration with InduPro both signal the same thing: big pharma is still willing to pay early for differentiated mechanisms and enabling platforms, then scale them internally.

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🔹 Generic oseltamivir (Tamiflu) is showing regional stockouts, with ASHP flagging demand-driven supply strain and some sites already shifting to conservation or substitution. At the same time, CDC’s outpatient flu-like illness indicator hit 8.2% for the week ending Dec 27, 2025, a level CDC notes is the highest for that metric in decades, with flu A(H3N2) viruses predominant. The practical point is that antivirals are most valuable when started early, especially for hospitalized and high risk patients, so shortages tend to push clinicians toward alternatives (baloxavir, zanamivir, IV peramivir) rather than change the overall clinical playbook.

🔹 Vanda said the FDA rejected its attempt to expand Hetlioz (tasimelteon) into jet lag disorder, sending the stock down about 11% premarket. The agency told Vanda its sNDA could not be approved in its current form because the evidence did not adequately show effectiveness for real world jet lag. FDA’s core issue was that Vanda’s main studies used controlled phase-advance sleep shifts (5h and 8h bedtime changes), which the FDA argued are not close enough to actual jet travel conditions. Vanda disagrees, arguing those models are a standard surrogate for circadian misalignment and that its package includes supportive evidence including simulated and transatlantic travel outcomes. This re-review followed a court ordered reset of a prior refusal and an October 2025 framework agreement for an expedited decision.

🔹 Eli Lilly struck a discovery and licensing collaboration with InduPro Therapeutics worth up to $950M plus an undisclosed equity investment, betting that “spatial biology” can help it build more tumor selective multispecific cancer drugs. InduPro will run early discovery on up to three undisclosed targets using its AI and machine learning-enabled membrane interactomics platform, which maps which surface proteins sit close together on tumor cells, then proposes co-target pairs meant to improve the safety and potency of bispecific antibody drug conjugates and multispecific T cell engagers. The timing matters: InduPro only formed in 2022, raised an $85M Series A in mid 2024, and just announced a separate Sanofi partnership and equity check last month, so this is another fast vote of confidence from big pharma in a platform that is still preclinical.

🔹 Revolution Medicines jumped on a Wall Street Journal report that AbbVie was in advanced talks to buy the company, then AbbVie told Reuters it was not in discussions and the premium faded. Now the reporting points to Merck instead with a rumored $28B to $32B price range, though nothing is signed and Revolution is not commenting.

🔹 The Department of HHS says the CDC, led by Acting Director Jim O’Neill, is immediately reshaping the U.S. childhood immunization schedule after a Trump-ordered peer-country review: a smaller set stays “recommended for all” (including measles, mumps, rubella, polio, pertussis, tetanus, diphtheria, Haemophilus influenzae type b, pneumococcal disease, varicella, and human papillomavirus), while several familiar shots like influenza, COVID-19, rotavirus, hepatitis A, hepatitis B, RSV, and meningococcal disease shift to “high-risk” or “shared clinical decision-making” (doctor/parent consult). Officials stress insurance will still cover everything with no cost-sharing, but pediatric and public health experts argue the real effect is behavioral: fewer automatic reminders, less routine stocking, more parental confusion, and higher odds of preventable outbreaks plus a messy state-by-state patchwork as school requirements diverge.

🔹 Amgen agreed to acquire U.K.-based Dark Blue Therapeutics for up to $840M, pulling an Oxford-linked, preclinical oncology program into its own research organization. The key asset, DBT-3757, is a small molecule targeted protein degrader aimed at MLLT1 and MLLT3 (proteins implicated in certain subsets of acute myeloid leukemia) with early data in leukemia models suggesting a differentiated mechanism and potential for both single agent and combination use. The broader read is that Amgen is still willing to buy mechanism-led science before the clinical price tag kicks in, especially in hard diseases where new biology can matter.

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🔹 Alumis said its next gen oral TYK2 inhibitor envudeucitinib delivered strong phase 3 results in moderate to severe plaque psoriasis. At 16 weeks, about 3/4 patients achieved at least a 75% improvement in skin severity and area (PASI 75), and about 3/5 were rated clear or almost clear by investigators (sPGA 0/1), both versus placebo. By 24 weeks, roughly 2/3 reached near complete clearance (PASI 90) and more than 2/5 had completely clear skin (PASI 100) on average across the two trials, and the drug beat apremilast on every psoriasis clearance benchmark at Week 24. Safety looked consistent with earlier studies and no new safety signals were flagged; Alumis says it plans to file for FDA approval in the second half of 2026.

🔹 GSK and Ionis reported positive topline phase 3 results for bepirovirsen in chronic hepatitis B, with both B-Well 1 and B-Well 2 meeting the primary endpoint and showing a statistically significant, clinically meaningful increase in “functional cure” when bepirovirsen was added to standard nucleos(t)ide analogue therapy versus standard therapy alone. Functional cure here means sustained loss of hepatitis B surface antigen and undetectable hepatitis B virus DNA for at least 24 weeks after finishing a finite treatment course, a big deal in a disease where current cures are rare and many patients stay on lifelong suppression. The trials enrolled more than 1,800 patients across 29 countries, showed a safety and tolerability profile consistent with prior studies, and looked even stronger in patients starting with lower hepatitis B surface antigen levels. GSK plans global regulatory filings starting in the first quarter of 2026, and if approved this could become a first finite six month backbone therapy for chronic hepatitis B, with real downstream implications for liver cancer risk.

🔹 Immuneering sold off even as it posted what look like strong phase 2a signals for atebimetinib plus modified gemcitabine and nab paclitaxel in first line pancreatic cancer. The company reported 64% overall survival at 12 months versus a 35% benchmark from historical standard of care data, along with a 39% overall response rate and 8.5 month median progression free survival versus 5.5 months, and it highlighted a tolerability profile where only neutropenia and anemia crossed the 10% threshold for grade 3 events. The market reaction looks like relative competition math, since investors are comparing cross trial headlines to other first line contenders like Verastem’s avutometinib + defactinib and Revolution Medicines’ daraxonarasib, even though follow up times and trial designs differ and Immuneering itself flags there is no head to head comparison. Next catalyst is whether this survival separation holds as the cohort expands beyond 50 patients and as the company moves toward its planned phase 3 start in mid 2026.

🔹 Jazz said its bispecific HER2 antibody Ziihera (zanidatamab) just delivered “practice-changing” phase 3 first line data in HER2-positive locally advanced or metastatic gastroesophageal adenocarcinoma, beating the long-standing Herceptin (trastuzumab) plus chemotherapy control. Both Ziihera + chemo and Ziihera + Tevimbra + chemo cut the risk of progression or death by about 35% and pushed median progression-free survival to 12.4 months versus 8.1 months. The median overall survival reached 26.4 months with the Ziihera + Tevimbra regimen (24.4 months for Ziihera + chemo). Diarrhea was the main tradeoff (higher grade ≥3 rates but few discontinuations). If these numbers hold up through regulatory review, this looks like the long-awaited HER2 upgrade in upper gastrointestinal cancer.

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Have a great rest of your week and thanks for reading Biotech Blueprint!

DISCLAIMER: This publication is for informational and educational purposes only and does not constitute investment, legal, medical, or tax advice, a solicitation, or an offer to buy/sell any security. Information is believed reliable but no warranty is made as to accuracy or completeness; views may change without notice. Do your own research and consult qualified professionals.

DISCLOSURE & CONFLICTS: The author may hold positions in securities mentioned and may change positions at any time without notice. No compensation is received from companies mentioned, and there are no known material business relationships unless explicitly stated. Content may reference clinical data and regulatory events; always consult primary sources and a licensed clinician for medical decisions. Past performance is not indicative of future results.

LIABILITY: Use of this content is at your own risk. The author assumes no responsibility for any losses arising from reliance on this material.

Welcome back to This Week in Biotech by Biotech Blueprint, edition 83, covering biotech and pharma news from December 19th, 2025 through January 1st, 2026.

🎙️ Biotech Blueprint brings you weekly video updates on the latest biotech and pharma news, plus in-depth podcast interviews with industry leaders. You can find us on YouTube, Spotify, and Apple Podcasts.

SPONSOR CONTENT BY ARCHES DATA

We help biotech CEOs and leadership teams pressure test strategy through rigorous data science, forecasting, and scenario analysis. From deep dive custom research to complex market modeling, we provide the evidence you need to lead with confidence.

Book a consultation HERE.

Visit our website: archesdata.com.

VIDEO SUMMARY

THIS WEEK’S KEY TAKEAWAYS 🔑

Despite the holidays, there were several important developments in biotech and pharma over the past two weeks. On the commercial side, Novo Nordisk widened its lead in obesity with FDA approval of oral Wegovy. This step removes the needle from the equation. At a relatively aggressive self-pay price, the pill is about unlocking the next wave of demand among patients who never wanted injections in the first place. In obesity, convenience is now a competitive weapon.

Large pharma also leaned into durability over novelty. Sanofi’s agreement to acquire Dynavax is a straightforward bet on adult vaccination as a stable, cash-generating category, anchored by Heplisav B’s two dose hepatitis B regimen and Sanofi’s global vaccines platform. At the same time, U.S. vaccine policy is clearly entering a noisier phase. There is momentum in Washington to revisit parts of the childhood vaccination framework and how immunization shows up in government quality measures. But big federal changes can’t happen by announcement alone. They still require evidence, process, and legal backing, which slows how much actually changes in practice.

Policy pressure extended beyond vaccines. A federal judge temporarily blocked a proposed 340B rebate pilot, preserving the current discount structure after finding the program was likely rushed and inadequately justified. Separately, new Most Favored Nation drug pricing agreements tied discounted prices to tariff relief and domestic manufacturing commitments. Even though the initial scope is limited, the structure matters. Pricing, access, and supply chain policy are increasingly being negotiated together, adding another layer of complexity for companies planning U.S. launches.

Clinical outcomes reminded investors why biotech remains a high variance business. Several FDA approvals, including treatments from Cytokinetics, Agios, and Vanda, validated years of development work and opened new commercial chapters. At the same time, late-stage setbacks at Ultragenyx, Biohaven, and Outlook were a reminder of how quickly biotech valuations can reset when pivotal data miss or regulators stay unconvinced.

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🔹 A federal judge has temporarily blocked a proposed 340B rebate pilot, halting its planned January 1 rollout after finding the program was likely unlawful under the Administrative Procedure Act. In a December 29 preliminary injunction, a U.S. District Court judge in Maine ruled that the Health Resources and Services Administration failed to adequately justify shifting 10 Medicare price-negotiated drugs into a rebate-based 340B model, citing a “threadbare” administrative record and insufficient analysis of the financial and operational harm to hospitals. The lawsuit, brought by hospital groups including the American Hospital Association, argued the pilot would impose major upfront costs and administrative burden on 340B providers. While not a final ruling, the judge found a strong likelihood the challengers will prevail and that allowing the program to proceed would cause irreparable harm—preserving the status quo for now.

🔹 Vanda Pharmaceuticals (VNDA) jumped after the FDA approved Nereus (tradipitant), an oral neurokinin-1 receptor antagonist licensed from Eli Lilly, for the acute prevention of motion-induced vomiting—the first new pharmacologic option for motion sickness in more than 40 years. Approval was supported by multiple late-stage trials showing a statistically significant reduction in vomiting with a safety profile compatible with single-dose use, and followed the FDA’s recent reclassification of motion sickness as an acute condition, which lifted a long-standing regulatory overhang. While motion sickness is a modest standalone market, analysts see peak U.S. sales north of $100 million and view the decision as a regulatory de-risking event for tradipitant’s broader pipeline, including GLP-1–associated nausea and gastroparesis.

🔹 Gilead agreed to acquire RP-3467, a polymerase theta inhibitor from Repare Therapeutics, for up to $30M, adding another targeted DNA-repair asset to its oncology pipeline. The deal includes $25 million upfront plus a $5 million milestone tied to technology transfer and boosts Repare’s cash balance ahead of its pending take-private transaction with XenoTherapeutics, lifting expected shareholder proceeds to roughly $2.20 per share. RP-3467 is an early-stage drug designed to exploit synthetic lethality in tumors with BRCA mutations and is currently being tested in a Phase 1 study across multiple solid tumors, both alone and in combination with AstraZeneca and Merck’s PARP inhibitor olaparib. While modest in size, the transaction underscores continued pharma interest in precision oncology assets even as broader biotech dealmaking remains selective.

🔹 Sanofi has agreed to acquire Dynavax Technologies for $15.50 per share in cash, valuing the vaccines specialist at roughly $2.2B and marking one of the clearest large pharma vaccine deals of 2025. The acquisition brings Heplisav-B, Dynavax’s marketed adult hepatitis B vaccine, into Sanofi’s vaccines portfolio, alongside an early stage shingles program currently in phase 1/2 development. Strategically, the deal strengthens Sanofi’s push into adult immunization, adding a differentiated two dose hepatitis B vaccine with faster protection than legacy regimens, while giving Dynavax global scale it could not reach alone. Sanofi will fund the transaction entirely with cash and plans to close in the first quarter of 2026.

🔹 The FDA approved Novo Nordisk’s oral Wegovy (semaglutide), making it the first and only oral GLP-1 therapy approved for chronic weight management in adults. The decision was based on the Phase 3 OASIS 4 trial, where once-daily 25 mg tablets delivered ~17% average weight loss at 64 weeks under on-treatment analysis (~14% regardless of discontinuation), materially narrowing the efficacy gap with injectable GLP-1s while eliminating needles. Notably, the pill also carries an indication to reduce major adverse cardiovascular events in patients with established cardiovascular disease—an edge no competing oral obesity drug currently offers. With a $149/month self-pay entry price, the oral expansion reinforces Novo’s dominance as competition in obesity intensifies heading into 2026.

🔹 The FDA approved Agios Pharmaceuticals’ AQVESME (mitapivat) for the treatment of anemia in adults with alpha- or beta-thalassemia, marking the first and only FDA-approved therapy to address anemia in both transfusion-dependent and non-transfusion-dependent patients. The decision was backed by the phase 3 ENERGIZE and ENERGIZE-T trials, which showed meaningful improvements in hemoglobin, fatigue, and transfusion burden across a broad, real world population. Commercially, the approval expands mitapivat into a much larger rare disease market, though uptake will be shaped by the newly implemented REMS program tied to hepatocellular injury risk, with U.S. launch expected in late January 2026.

🔹 Biotech delivered some of the most extreme winners of 2025, even as the broader healthcare sector posted a relatively muted year. The S&P Health Care Index rose about 12% year-to-date by mid-December, but beneath that surface a handful of biotech stocks generated explosive, data-driven returns. The clear standout was Abivax, which surged roughly 1,500-1,600% after positive late stage data for its oral ulcerative colitis drug obefazimod, making it the top performing biotech stock of the year. That rally has since evolved into strategic optionality. In a Truist Securities investor survey, Abivax emerged as the most likely biotech acquisition target in 2026, cited by 44% of respondents, far ahead of any peer, with speculation centered on potential interest from Eli Lilly.

🔹 Cytokinetics won FDA approval for Myqorzo, a first-in-class “cardiac myosin inhibitor” for adults with symptomatic obstructive hypertrophic cardiomyopathy, a condition where an abnormally thick heart muscle blocks blood flow out of the heart and makes everyday activity feel like you’re running uphill. The decision was driven by the phase 3 SEQUOIA-HCM study, where patients treated for 24 weeks showed a meaningful improvement in exercise capacity versus placebo, including higher peak oxygen uptake (a standard measure of cardiopulmonary fitness). Commercially, this is a milestone moment: it’s Cytokinetics’ first approved drug, with U.S. launch expected in the second half of January 2026. The trade off to watch is the label’s boxed warning for risk of heart failure. Because the drug deliberately reduces heart contractility, patients need echocardiogram monitoring and access is gated through a Risk Evaluation and Mitigation Strategy program, which can slow early uptake but also clarifies who should (and shouldn’t) get it.

🔹 The White House announced nine new Most Favored Nation (MFN) drug pricing agreements with major pharmaceutical companies, including Amgen, BMS, Gilead, Merck, Novartis, and Sanofi. Under the deals, select medicines will be sold at steep discounts through a new TrumpRx direct-to-consumer platform launching in January and made available to all state Medicaid programs. In exchange, participating companies avoid pharmaceutical import tariffs for three years, commit to large U.S. manufacturing investments, and agree that future innovative drugs will also be subject to MFN pricing, marking one of the most aggressive federal interventions yet into U.S. drug pricing and launch economics.

🔹 BioMarin will acquire Amicus Therapeutics for $4.8B in cash ($14.50 per share), adding two marketed rare-disease drugs (Galafold for Fabry disease and Pombiliti + Opfolda for Pompe disease) which generated $599M in revenue over the past year. The deal, expected to close in Q2 2026, is projected to accelerate BioMarin’s revenue immediately and be earnings accretive within 12 months, with greater impact from 2027 onward. BioMarin will fund the acquisition with cash and debt, while a separate settlement resolves U.S. patent litigation around Galafold, supporting exclusivity through 2037.

🔹 U.S. vaccine policy is heading into uncertain territory as RFK Jr. pushes to scale back childhood vaccination recommendations, but faces clear legal limits on how fast and how far he can go. While the administration has already moved to stop using childhood and prenatal vaccination rates as a measure of health care quality in government programs, any attempt to overhaul the national vaccine schedule would require a formal, evidence-based process rather than a simple announcement. That reality likely explains why a late December plan to align U.S. vaccine recommendations with Denmark’s was abruptly shelved. For vaccine makers and public health officials alike, the message is clear: major changes are being discussed, but the legal system will strongly shape what actually happens in 2026.

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CLINICAL TRIAL UPDATES 📊

🔹 Ultragenyx (RARE) shares fell sharply after its osteogenesis imperfecta program setrusumab (UX143) failed to meet the primary endpoint in both late-stage trials. Neither the Phase 2/3 ORBIT study (ages 5–25, placebo-controlled) nor the Phase 3 COSMIC study (ages 2–<7, bisphosphonate-controlled) demonstrated a statistically significant reduction in annualized clinical fracture rate, despite both trials showing strong, statistically significant improvements in bone mineral density. The company said it will conduct additional analyses to better understand the disconnect between bone density gains and fracture outcomes and announced plans to implement significant expense reductions. The readout represents a major setback for Ultragenyx’s rare bone disease portfolio and weighed heavily on its partner Mereo BioPharma, which holds EU and UK rights to the asset.

🔹 Biohaven shares fell after its phase 2 study of BHV-7000 in major depressive disorder failed to meet its primary endpoint, with the drug showing no statistically meaningful improvement versus placebo on the Montgomery-Åsberg Depression rating scale, a standard measure of depressive symptoms, over six weeks. While Biohaven pointed to signals favoring BHV-7000 in patients with more severe baseline depression, the company characterized these findings as exploratory and said it does not plan to pursue further psychiatric trials, opting instead to focus resources on immunology, obesity, epilepsy, and oncology. The result underscores the persistent difficulty of translating novel depression mechanisms into broad clinical efficacy and removes BHV-7000 from Biohaven’s central growth narrative.

🔹 Outlook Therapeutics shares collapsed after the FDA issued a Complete Response Letter for its resubmitted BLA for ONS-5010/LYTENAVA (bevacizumab-vikg) in wet AMD, reiterating that a single adequate, well-controlled efficacy study is insufficient for approval and again requesting additional confirmatory evidence. Crucially, the agency did not specify what form that confirmation should take, leaving the company in regulatory limbo. Outlook says it will pursue “all available pathways,” but with U.S. approval now pushed further out, the near-term narrative shifts to how costly and time-consuming the FDA’s missing requirement may be—and whether European commercialization can sustain the program in the interim

🔹 Altimmune reported positive 48 week phase 2b data from its IMPACT trial showing that pemvidutide, a dual glucagon and GLP-1 agonist, produced statistically significant improvements in non-invasive markers of liver fibrosis and inflammation in patients with metabolic dysfunction-associated steatohepatitis, while maintaining a favorable safety profile. Both dose arms showed continued antifibrotic improvement from 24 to 48 weeks, meaningful reductions in liver stiffness and fibrosis scores versus placebo, and modest but sustained weight loss without plateauing at the higher dose, with fewer treatment discontinuations than placebo and no serious treatment-related adverse events. Importantly, Altimmune said it has aligned with the FDA on the design of a registrational phase 3 trial in patients with moderate to advanced fibrosis, which it plans to initiate in 2026, positioning pemvidutide as a differentiated late-stage contender in the crowded MASH landscape.

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Have a great rest of your week and thanks for reading Biotech Blueprint!

DISCLAIMER: This publication is for informational and educational purposes only and does not constitute investment, legal, medical, or tax advice, a solicitation, or an offer to buy/sell any security. Information is believed reliable but no warranty is made as to accuracy or completeness; views may change without notice. Do your own research and consult qualified professionals.

DISCLOSURE & CONFLICTS: The author may hold positions in securities mentioned and may change positions at any time without notice. No compensation is received from companies mentioned, and there are no known material business relationships unless explicitly stated. Content may reference clinical data and regulatory events; always consult primary sources and a licensed clinician for medical decisions. Past performance is not indicative of future results.

LIABILITY: Use of this content is at your own risk. The author assumes no responsibility for any losses arising from reliance on this material.

📣 This is the final This Week in Biotech edition of 2025. Happy holidays from Biotech Blueprint! We’ll be back on January 2, 2026, covering everything from December 19 through January 1.

Instead of recapping the biggest biotech developments of 2025 here, I’ll be publishing an article over the holiday break, alongside a podcast recorded with Biotech Capital Compass. With so much news breaking in the past week, I didn’t want to overload this issue.

🎙️ Biotech Blueprint brings you weekly video updates on the latest biotech and pharma news, plus in-depth podcast interviews with industry leaders. You can find us on YouTube, Spotify, and Apple Podcasts.

In the latest episode, I sat down with NImmune founder and CEO Josep Bassaganya-Riera to talk about omilancor, the gut-restricted oral Inflammatory bowel disease drug now in phase 3. We cover LANCL2 biology, their biomarker aimed at boosting response rates, and the TITAN-X platform that helps guide real development decisions. A really interesting look at how you build an oral immunology company in 2025.

VIDEO SUMMARY

THIS WEEK’S KEY TAKEAWAYS 🔑

The clearest scientific win came from Takeda, which delivered convincing late stage data for its once daily psoriasis pill, zasocitinib. For Takeda, the timing is critical. Major patent expirations loom later this decade, and zasocitinib now looks less like a pipeline asset and more like a future growth engine.

In oncology, J&J showed how incumbents defend leadership without reinventing the science. FDA approval of a subcutaneous formulation of Rybrevant doesn’t change survival curves (those were already strong) but it meaningfully improves how care is delivered. Shorter administration times and fewer infusion reactions matter to patients and health systems alike. As competition in lung cancer intensifies, differentiation is increasingly about how drugs are used, not just whether they work.

The most consequential policy signal came from CEPI stepping in to fund Moderna’s pandemic influenza vaccine after U.S. federal support evaporated. The science never faltered but the political environment did. CEPI’s investment keeps the world’s first mRNA-based pandemic flu vaccine moving toward phase 3. Pandemic preparedness is becoming more international, more distributed, and less dependent on U.S. government leadership. That has long term implications for how future outbreak responses are financed and governed.

Public health policy also took a more uncertain turn. The CDC’s decision to relax universal newborn hepatitis B vaccination guidance marks a notable departure from decades of prevention messaging. While officials stress informed choice and unchanged access, past experience shows that weakening universal recommendations often leads to lower vaccination rates in practice.

Finally, a quieter but deeply important update: newborn screening will now include Duchenne muscular dystrophy. Earlier diagnosis won’t cure the disease, but it fundamentally alters the clinical trajectory by enabling earlier monitoring and access to therapy before irreversible damage sets in. This is public health working as intended by shifting care upstream, where it has the most impact.

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BIOTECH/PHARMA NEWS 🧬

🔹 CEPI will invest up to $54.3M to fund a pivotal phase 3 trial of Moderna’s mRNA-based H5 avian influenza vaccine, mRNA-1018, effectively reviving the program after the U.S. government terminated a much larger BARDA contract earlier this year. The study is expected to begin in early 2026 and would be the first mRNA vaccine to enter a phase 3 trial for pandemic influenza, leveraging prior positive phase 1/2 immunogenicity data in healthy adults. As part of the agreement, Moderna committed to reserving 20% of manufacturing capacity for affordable supply to low- and middle-income countries in the event of a pandemic, underscoring CEPI’s push for rapid, equitable access amid growing uncertainty around U.S. federal support for pandemic preparedness.

🔹 J&J won FDA approval for a subcutaneous version of its EGFR-targeting antibody Rybrevant, developed with Halozyme, enabling a much faster, injection-based alternative to IV dosing for first line EGFR mutant non small cell lung cancer when combined with lazertinib. The approval is supported by phase 3 MARIPOSA data showing a statistically significant overall survival benefit versus AstraZeneca’s Tagrisso, with J&J projecting median survival beyond four years in this chemotherapy-free regimen. The new formulation cuts administration time from hours to minutes and sharply reduces infusion-related reactions, reinforcing J&J’s push to differentiate on convenience and durability as competition in EGFR mutant lung cancer intensifies.

🔹 Incyte said the European Commission approved Minjuvi (tafasitamab), partnered with Xencor, as a new late-line option for adults with relapsed or refractory follicular lymphoma, expanding the drug’s footprint beyond its earlier EU approval in diffuse large B cell lymphoma. The decision clears Minjuvi for use alongside lenalidomide and rituximab after at least one prior systemic therapy, based on Phase 3 inMIND data showing a statistically significant and clinically meaningful improvement in progression-free survival versus standard therapy alone. The approval marks a second European indication for the CD19 antibody and reinforces Incyte’s strategy of incrementally broadening Minjuvi’s role across B cell lymphomas using combination regimens rather than standalone monotherapy.

🔹 The CDC formally adopted a shift in its newborn hepatitis B vaccination guidance, ending its decades-long recommendation that all infants receive a birth dose if their mothers test negative for the virus. Under the new policy, vaccination for these newborns moves to shared clinical decision making between parents and healthcare providers, with the first dose deferred until at least two months of age if the birth dose is skipped, while infants born to hepatitis B-positive or untested mothers will still receive immediate vaccination. The change has sparked significant controversy among public health experts, who warn it could reverse hard won declines in pediatric hepatitis B infections, even as federal officials emphasize informed consent and note that insurance coverage and vaccine access remain unchanged.

🔹 Sarepta shares rose after HHS announced it will add Duchenne muscular dystrophy (DMD) and metachromatic leukodystrophy to the recommended newborn screening panel, a move that could shift diagnosis from around age 4-5 years to shortly after birth. Earlier detection is seen as clinically meaningful in DMD, where irreversible muscle damage begins in infancy, and could accelerate access to care and approved therapies. The decision also has commercial implications for Sarepta, which has marketed and pipeline DMD therapies.

🔹 BioCryst won approval for an oral pellet formulation of Orladeyo, extending its hereditary angioedema franchise to children ages 2 to 12 and making it the first targeted oral prophylactic option for pediatric patients who previously relied on injections or infusions. Meanwhile, Amgen secured FDA approval to expand Uplizna into generalized myasthenia gravis, adding a third U.S. indication for the CD19-targeting antibody and positioning it as a twice yearly option for durable symptom control in a difficult autoimmune disease. Both approvals meaningfully widen access by extending proven therapies to new patient groups.

🔹 Last Friday, reports briefly suggested the FDA might add a “black box” warning to Covid vaccines, but follow up from Bloomberg says the agency has no such plans. Officials emphasized that earlier stories were premature, even as the FDA continues routine safety reviews already reflected in existing labels. While the episode is concerning and highlights growing uncertainty around vaccine policy under new leadership, there is no immediate change to vaccine labeling, recommendations, or the underlying safety data.

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CLINICAL TRIAL UPDATES 📊

🔹 Takeda reported strong late stage results for zasocitinib, a once daily pill for plaque psoriasis developed using AI. In two large phase 3 studies, more than half of treated patients had skin that was clear or almost clear after 16 weeks, and about 30% achieved completely clear skin, with responses continuing to improve over time. The drug was generally well tolerated and outperformed placebo and an existing oral competitor. Takeda plans to file for regulatory approval in 2026, positioning zasocitinib as a potential oral alternative in a market still dominated by injectable biologics, and a key revenue opportunity as the company approaches major patent expirations later this decade.

🔹 Pyxis Oncology fell nearly 47% after releasing phase 1 data for its ADC micvotabart pelidotin (MICVO) in recurrent/metastatic head and neck cancer. While the company highlighted a 46% response rate and 92% disease control at the selected monotherapy dose, investors appeared focused on the safety profile, with Grade ≥3 treatment-related adverse events in 56% of patients and a 28% discontinuation rate. Early combination data with Keytruda looked numerically stronger (71% ORR in seven patients), but remains highly preliminary. With durability data not expected until mid-2026, the readout reinforced how unforgiving the market remains for early stage oncology programs where efficacy comes paired with meaningful toxicity.

🔹 Insmed fell after abandoning brensocatib in chronic rhinosinusitis without nasal polyps, following a phase 2b miss where the drug failed to beat placebo on symptom improvement despite a clean safety profile. The program was discontinued immediately, though brensocatib remains approved for bronchiectasis and in development for other indications. Insmed paired the setback with the acquisition of a phase 2-ready monoclonal antibody, INS1148, to rebuild its respiratory and inflammation pipeline.

🔹 DBV Technologies surged after its phase 3 VITESSE trial showed that the VIASKIN Peanut patch significantly desensitized children aged 4-7 with peanut allergy, with 46.6% meeting responder criteria at 12 months versus 14.8% on placebo, easily clearing the pre-specified efficacy bar. The treatment demonstrated a favorable safety profile consistent with prior studies, driven mainly by mild to moderate local skin reactions and very low rates of treatment related anaphylaxis. With the largest food allergy immunotherapy trial to date now positive and Breakthrough Therapy Designation already in hand, DBV plans to submit a BLA in the first half of 2026, positioning VIASKIN Peanut as a potentially first-in-class, noninvasive option for pediatric peanut allergy. The stock (DBVT) is up 25% on the news.

🔹 Vistagen shares collapsed after its phase 3 PALISADE-3 trial showed that intranasal fasedienol failed to significantly reduce anxiety versus placebo in a public speaking challenge for social anxiety disorder, missing both its primary and secondary endpoints despite earlier positive signals in phase 2 and a prior phase 3 study. The drug’s safety and tolerability remained consistent with past trials, but the lack of efficacy erased the investment case, prompting Vistagen to announce cost-cutting measures to extend its cash runway into 2027 and triggering an analyst downgrade as the company reassesses next steps with regulators.

🔹 Gilead said its investigational single-tablet HIV regimen combining lenacapavir (capsid inhibitor) with bictegravir (INSTI) hit its mark again: in the phase 3 ARTISTRY-2 switch study, moving virologically suppressed patients from Biktarvy to the BIC/LEN combo was statistically non-inferior at week 48, with secondary endpoints also supporting comparable control. With ARTISTRY-1 already positive, Gilead is now framing the two trials as the package for regulatory submissions, positioning BIC/LEN as a “next step” option for suppressed adults that pairs a high resistance barrier backbone with a first-in-class mechanism, without introducing new safety surprises.

🔹 argenx will discontinue its phase 3 UplighTED trials of subcutaneous efgartigimod in thyroid eye disease after an independent monitoring committee recommended stopping for futility at a pre-specified interim analysis. The decision was driven by a lack of efficacy rather than safety, with the company noting a favorable tolerability profile and no new safety signals. Shares fell modestly on the news, but the setback is contained: efgartigimod remains a cornerstone asset for argenx in approved indications like generalized myasthenia gravis and chronic inflammatory demyelinating polyneuropathy.

🔹 Arcus Biosciences halted its phase 3 anti-TIGIT program after failing to improve survival in gastric cancer, adding another late stage strike against the TIGIT class as the company pivots capital to its HIF-2 alpha and inflammation pipeline.

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Have a great rest of your week and thanks for reading Biotech Blueprint!

DISCLAIMER: This publication is for informational and educational purposes only and does not constitute investment, legal, medical, or tax advice, a solicitation, or an offer to buy/sell any security. Information is believed reliable but no warranty is made as to accuracy or completeness; views may change without notice. Do your own research and consult qualified professionals.

DISCLOSURE & CONFLICTS: The author may hold positions in securities mentioned and may change positions at any time without notice. No compensation is received from companies mentioned, and there are no known material business relationships unless explicitly stated. Content may reference clinical data and regulatory events; always consult primary sources and a licensed clinician for medical decisions. Past performance is not indicative of future results.

LIABILITY: Use of this content is at your own risk. The author assumes no responsibility for any losses arising from reliance on this material.

📣 With the holidays approaching, This Week in Biotech will skip Dec 26 edition. Our final issue of the year drops next Friday, Dec 19, covering the biggest biotech developments of 2025, and we’ll be back Jan 2, 2026 with themes and companies to watch next year.

🌐 Visit our updated website, biotechblueprintconsulting.com, where we’re posting new visuals, reports, and details on our consulting work.

🎙️ Biotech Blueprint brings you weekly video updates on the latest biotech and pharma news, plus in-depth podcast interviews with industry leaders. You can find us on YouTube, Spotify, and Apple Podcasts.

In the latest episode, I sat down with NImmune founder and CEO Josep Bassaganya-Riera to talk about omilancor, the gut-restricted oral Inflammatory bowel disease drug now in Phase 3. We covered LANCL2 biology, their biomarker aimed at boosting response rates, and the TITAN-X platform that helps guide real development decisions. A really interesting look at how you build an oral immunology company in 2025. Below are the accompanying Substack article and podcast.

VIDEO SUMMARY

THIS WEEK’S KEY TAKEAWAYS 🔑

This week, the Federal Reserve delivered its second rate cut since September, shifting the economy into an easing cycle, which has historically been biotech’s strongest performance environment. Falling rates typically allow valuations to re-expand, reopen financing windows, and bring more investors back into the sector. After two cuts already and more likely in 2026, the headwind that suppressed the sector for nearly two years has flipped into a genuine tailwind.

On the public health front, the U.S. is sending mixed signals. A CDC analysis of nearly 100k children showed the updated Covid vaccine sharply reduced ER visits, especially in infants and toddlers, the age group with the highest hospitalization rates after seniors. Yet uptake remains stuck at 13%, and national guidance has pivoted from universal recommendation to “ask your doctor,” raising concern that evidence and policy are moving in opposite directions. At the same time, industry leaders are warning about the broader consequences of this skepticism. Moderna co-founder Noubar Afeyan warned that the U.S. is in danger of losing its lead in vaccine and mRNA science just as China accelerates its biotech ambitions.

Meanwhile, this week brought some of the most striking clinical data of the year. Eli Lilly’s triple agonist retatrutide produced bariatric surgery level weight loss and dramatic relief from knee osteoarthritis, a rare pairing in a single therapy. Biohaven reported a 52% response rate across tough solid tumors with its next generation Trop2 antibody-drug conjugate, including a clean pulmonary safety signal that has eluded first wave competitors. On the neuro front, AC Immune showed early but promising signs of disease stabilization in Parkinson’s through an alpha-synuclein vaccine approach. And in immunology, Kymera reported that its oral STAT6 degrader meaningfully reduced inflammation across skin and blood, with early improvements also seen in asthma and allergy symptoms.

Outside the headline catalysts, the week also brought mixed pipeline and business updates. Rezolute’s congenital hyperinsulinism drug failed phase 3, Roche’s oral estrogen receptor degrader modestly outperformed standard endocrine therapy in early breast cancer, and Mirum expanded its rare liver portfolio with the acquisition of Bluejay Therapeutics.

Continue reading for more stories.

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BIOTECH/PHARMA NEWS 🧬

🔹 The Fed’s December 10 rate cut (its second since September) officially moves us into a real easing cycle, and historically that’s when biotech performs best. Over 45 years of data, the sector has delivered about +47% median annualized returns during periods of falling rates, compared with –5% during rate hikes. Lower rates make future earnings more valuable, reopen funding channels, and generally bring buyers back into the market. The impact usually unfolds over the next one to three quarters as valuations rise, the IPO window cracks open, and dealmaking picks up. With two cuts already in place and more expected into 2026, the macro drag that weighed on biotech from 2022 to 2024 has flipped into a meaningful tailwind.

🔹 A large CDC analysis of 98k children across nine states found that the 2024-25 Covid vaccine significantly reduced Covid-related ER and urgent care visits. For kids 9 months to 4 years old, the shot cut the risk by 76%. For those 5 to 17, the reduction was 56%, even in a population with plenty of prior infections and older vaccine doses. Infants remain the pediatric group with the highest Covid hospitalization rates outside seniors. Only about 13% of children received the updated vaccine, and that low uptake now intersects with a major policy shift: national guidance has moved from a universal recommendation to an “ask your doctor” approach under the new health leadership.

🔹 Moderna co-founder and chairman Noubar Afeyan warned that the U.S. is in “big danger” of losing its edge in vaccine and mRNA technology as NIH funding ebbs and political and public skepticism rises, just as China ramps biotech investment without similar resistance. Speaking to Bloomberg TV, he defended the safety of mRNA vaccines from Moderna, Pfizer, and BioNTech amid an ongoing FDA review of potential vaccine-related deaths, pointing to extensive data and estimates that COVID shots prevented nearly 20 million deaths globally in their first year, including more than one million in the U.S., and urging regulators to focus on evidence rather than sentiment. Afeyan’s comments come as Moderna’s shares are down 32% over the past year on falling COVID vaccine sales and over reliance on a shrinking franchise.

🔹 Mirum Pharmaceuticals will acquire privately held Bluejay Therapeutics for $250M in cash, $370M in stock and up to $200M in sales milestones, adding full global rights to brelovitug, a fully human monoclonal antibody in phase 3 for chronic hepatitis delta virus, the most severe form of viral hepatitis with no approved U.S. treatments and about 230k patients across the U.S. and Europe. Brelovitug, which carries Breakthrough Therapy, PRIME and Orphan designations, has shown strong antiviral activity and liver enzyme improvements in phase 2 and could support a biologics license application and potential launch in 2027. Mirum is funding part of the deal via a $200M private placement, effectively levering its existing rare liver commercial footprint (LIVMARLI, CHOLBAM, CTEXLI) into a second major franchise.

🔹 On Friday (12/5), ACIP voted 8-3 to end the universal newborn hepatitis B dose for infants born to mothers who test negative, replacing it with “shared decision-making,” while maintaining the birth dose for infants of mothers who are positive or whose status is unknown. The move reverses a policy credited with a 99% drop in pediatric hepatitis B since 1991 and drew immediate pushback from medical groups concerned infections will rise. After two days of procedural confusion and rewritten ballots, the recommendation now goes to acting CDC director Jim O’Neill, who can accept or reject it. Sen. Bill Cassidy, a hepatologist, has already urged him not to adopt the change.

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CLINICAL TRIAL UPDATES 📊

🔹 Eli Lilly’s “triple agonist” retatrutide just posted the kind of phase 3 results that reshape an entire category: in adults with obesity and knee osteoarthritis, the highest dose drove 29% weight loss among adherent patients (71 lbs on average) and even 24% weight loss when counting dropouts, numbers that edge into bariatric-surgery territory. Knee pain also fell 75%, and more than 1 in 8 patients ended the trial completely pain-free, a meaningful signal in a population where cartilage loss normally means a slow march toward joint replacement. The usual incretin side effects (nausea, diarrhea, vomiting) were common but mostly manageable. Discontinuations were higher at the top dose, with some patients stopping due to excessive weight loss.

🔹 Biohaven’s Trop2-targeting antibody-drug conjugate BHV-1510, paired with Regeneron’s anti-PD-1 antibody Libtayo, delivered eye catching phase 1 signals in heavily pretreated solid tumors: among 23 patients, the combo hit a 52% confirmed response rate overall, including 60% in non-small cell lung cancer, 100% in endometrial cancer (one complete response), and 50% in urothelial cancer, with most patients having already failed prior PD-1/PD-L1 therapy. The safety profile looks meaningfully differentiated from first wave Trop2 antibody-drug conjugates, with low rates of neutropenia, diarrhea, and alopecia, and importantly, no interstitial lung disease so far, though oral mucositis and stomatitis remain a class tax and drove most higher-grade events.

🔹 Rezolute’s phase 3 sunRIZE trial in congenital hyperinsulinism failed to meet its primary and key secondary endpoints, with the top ersodetug dose showing a 45% reduction in weekly hypoglycemia events versus a 40% improvement on placebo and no statistically significant benefit on continuous glucose monitoring time-in-hypoglycemia. Safety was generally acceptable aside from two hypersensitivity reactions, but the efficacy signal was too weak to support the program, leading the company to halt development in this indication while still planning an FDA discussion under its Breakthrough Therapy designation. Shares collapsed nearly 90%, and partner Xoma was hit as well on the loss of future royalty expectations.

🔹 AC Immune reported interim phase 2 data for its alpha-synuclein-targeting immunotherapy ACI-7104.056 in early Parkinson’s disease, showing the shot generated strong antibody responses in 100% of treated patients and produced signs of stabilized disease across multiple biomarker and clinical measures after 12-18 months of treatment. Markers tied to pathology and neurodegeneration (cerebrospinal fluid alpha-synuclein and neurofilament light chain) remained steady in the treatment arm but worsened on placebo, while motor scores also showed a trend toward stabilization. Safety looked clean, with no serious adverse events and mostly mild injection site reactions. The company now plans to meet regulators to explore accelerating development ahead of final part 1 data in mid-2026.

🔹 Roche reported additional phase 3 data showing its oral selective estrogen receptor degrader giredestrant cut the risk of invasive disease recurrence or death by 30% in more than 4k patients with early stage, estrogen receptor positive breast cancer, an area where up to a third of patients eventually relapse on today’s endocrine therapies. Three year invasive disease free survival reached 92.4% on giredestrant versus 89.6% on standard treatment, with a favorable safety profile and fewer discontinuations. The trial positions giredestrant as the first oral endocrine therapy in decades to outperform standard care in the adjuvant setting, though questions remain about how it will be used alongside CDK4/6 inhibitors.

🔹 Kymera’s shares jumped 50% after early phase 1b data for KT-621 showed strong activity in moderate to severe eczema. The oral STAT6 degrader reduced STAT6 levels by more than 90% in both blood and skin lesions, and delivered meaningful clinical improvements. About a 63% drop in Eczema Area and Severity Index scores and a 40% reduction in itch after four weeks with no serious or treatment-related safety issues. Patients with asthma and allergic rhinitis also saw early benefits, hinting at broader potential across type 2 inflammatory diseases. Phase 2b eczema data are expected in 2027, with an asthma trial starting next year.

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Have a great rest of your week and thanks for reading Biotech Blueprint!

DISCLAIMER: This publication is for informational and educational purposes only and does not constitute investment, legal, medical, or tax advice, a solicitation, or an offer to buy/sell any security. Information is believed reliable but no warranty is made as to accuracy or completeness; views may change without notice. Do your own research and consult qualified professionals.

DISCLOSURE & CONFLICTS: The author may hold positions in securities mentioned and may change positions at any time without notice. No compensation is received from companies mentioned, and there are no known material business relationships unless explicitly stated. Content may reference clinical data and regulatory events; always consult primary sources and a licensed clinician for medical decisions. Past performance is not indicative of future results.

LIABILITY: Use of this content is at your own risk. The author assumes no responsibility for any losses arising from reliance on this material.