🎙️ 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 most interesting thing that happened this week wasn’t a clinical readout or a deal. It was the FDA reversing itself on two products in two days. On Wednesday, uniQure said the agency will accept three year data from a roughly 50 patient Phase 1/2 study as the primary basis for an accelerated approval filing of AMT-130, its gene therapy for Huntington’s disease. The FDA had previously asked for more. A day later, the agency’s vaccines advisory committee voted unanimously that Moderna’s mRNA flu vaccine has a favorable benefit-risk profile, for a product the same agency had refused to even review earlier this year. Both reversals came after Marty Makary and Vinay Prasad left.

It would be easy to read this as the FDA going soft. It’s not. Under acting commissioner Kyle Diamantas, the agency is being selectively flexible on programs where there’s no current alternative. There’s nothing approved for Huntington’s, which is a uniformly fatal disease. mRNA gives the US a faster way to update flu vaccines as strains drift. But in other areas, the bar hasn’t moved at all. Fulcrum’s sickle cell program got sidelined last week on a safety question, and AstraZeneca’s camizestrant in breast cancer is still frozen after a negative advisory vote in April. So the agency isn’t lowering its standards across the board but it seems to be making case by case decisions about where unmet need is high enough to accept less data. For developers, that makes regulatory strategy harder to plan, because the rules now depend on which category your program lands in.

The other big theme is that M&A keeps happening every single week. Biogen bought an immunology startup for up to $1 billion. Jazz signed a T cell engager deal worth up to $4.1 billion. Lilly bought a non-opioid pain developer in what’s now their fifteenth deal of 2026. PwC reported first quarter pharma deal value above $65 billion and called the biopharma ecosystem “back to full health.” Large caps are buying discovery speed and modality breadth instead of building it in house, and they’re doing it before Section 232 pharma tariffs kick in on July 31.

The IPO market is the cleanest bull signal in biotech right now. Kardigan raised $400 million in an upsized offering this week and closed up 38% on its first day of trading. That makes four biotechs to raise at least $400 million via IPO in 2026, the most in any single year since 2021.

BIOTECH/PHARMA NEWS 🧬

🔹 uniQure said the FDA will accept three year data from its Phase 1/2 study of AMT-130 as the primary basis for a Biologics License Application under accelerated approval. The company plans to file in Q3 2026. AMT-130 is an AAV gene therapy delivered directly into the striatum via neurosurgery, designed to lower the mutant huntingtin protein that drives Huntington’s disease. There is no approved therapy for Huntington’s, which makes a filing on a roughly 50 patient dataset a clear signal that the agency will accept a small package when the disease is fatal and untreated. The FDA also indicated it’s open to a concurrent standard of care control in the confirmatory study rather than a sham surgical arm, which is a meaningful concession given the ethics and cost of sham neurosurgery. Shares jumped on the news. The open questions are durability of effect and whether a 50 patient base can carry a label in a heterogeneous, slowly progressing disease.

🔹 The FDA’s vaccines advisory committee voted unanimously that the benefit-risk profile of mFluSiva, Moderna’s mRNA trivalent seasonal flu vaccine, is favorable. The FDA target decision date is August 5. This completes a striking turnaround. The same agency declined to review the same shot earlier this year, and the briefing documents released ahead of the meeting were supportive. If approved, mFluSiva would be the first mRNA-based seasonal influenza vaccine in the US. The practical implication is that mRNA’s core advantage, speed of reformulation against drifting strains, finally gets a US regulatory anchor in flu, the largest vaccine market by volume. For Moderna, this is the proof point that the platform extends beyond COVID. Vaccine politics remain the wild card between a unanimous panel and a final signature.

🔹 Jazz Pharmaceuticals and AbCellera signed a collaboration to discover next-generation T cell engaging multispecific antibodies for gastrointestinal and other solid tumors. AbCellera takes $56M upfront across the first two programs, plus $28M on initiation of a third, with up to $792M per program in option and milestone payments and tiered royalties, totaling up to $4.1B across as many as five programs. T cell engagers have largely worked in blood cancers and struggled in solid tumors, where the targets are less clean and the toxicity harder to manage. Jazz is paying for AbCellera’s antibody engineering platform to solve that problem rather than buying a finished asset. The small upfront and heavily backloaded structure is the standard template for platform deals where the science still has to prove itself.

🔹 Biogen agreed to acquire RayThera, a private developer of small molecule anti-inflammatory drugs, for up to $1B in upfront and primarily clinical and regulatory milestones. The lead candidate is expected to enter Phase 1 in early Q3 2026. This is Biogen’s second sizable deal of the year after the roughly $5.6B Apellis acquisition in March, and it continues a deliberate push into immunology as the company diversifies away from a neurology franchise that has produced two Phase 2b misses in twelve months. The deal structure, small disclosed upfront with most of the value contingent, fits a preclinical stage asset. What Biogen is really buying is optionality in immune-mediated disease at a price that limits downside if the lead program stalls.

🔹 Lilly acquired 4E Therapeutics, an Austin neuroscience company developing oral MNK inhibitors for chronic pain, on undisclosed terms. The lead compound, 4ET1103, targets the MNK-eIF4E signaling pathway in peripheral sensory neurons and is the first MNK inhibitor for pain to reach the clinic. This is Lilly’s second non-opioid pain acquisition in as many years, following the up to $1 billion SiteOne deal in May 2025, which targeted sodium ion channels. It’s also their fifteenth or so deal of 2026 and pushes the year’s committed acquisition capital past $18B. The chronic pain market has been waiting two decades for a credible alternative to opioids, and Lilly is now building optionality across multiple non-opioid mechanisms.

🔹 Kardigan, a cardiovascular drug developer founded by former MyoKardia executives, raised $400M in an upsized IPO, selling 25M shares at $16, the top of its range. Shares closed up 38% at $22, giving the company a market value near $2B. MyoKardia developed Camzyos and was acquired by Bristol Myers Squibb for $13B, so the founder pedigree is direct. Kardigan has now raised close to $1B total in two years, including a $254M Series B in October. It’s the fourth biotech to raise at least $400M via IPO this year, the most in a single year since 2021. The 38% pop on debut is the signal worth watching. The 2026 IPO class is holding its value in the aftermarket, which is what separates a real funding window from a temporary one.

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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.

Image source: pharmaphorum.com

🎙️ 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 belonged to metabolism. The American Diabetes Association meeting in New Orleans pushed the obesity conversation into a new phase. For three years the story has been Lilly versus Novo and this week it became a four player field. AstraZeneca put real Phase 2 numbers behind its oral GLP-1 pill elecoglipron, 10.5% weight loss at 26 weeks rising to 11.8% at 36 with no plateau, and committed to a sprawling Phase 3 program anchoring what it now frames as a $5 billion cardiometabolic franchise. AstraZeneca is now the third large cap with a credible oral incretin in pivotal development, behind Lilly’s Foundayo and alongside Pfizer’s Metsera assets. The efficacy isn’t class leading, but you don’t need to be best in class to compete in this market anymore. You just need to be good enough, which says a lot about how big the opportunity has become.

The most important obesity development this week wasn’t about a drug. More US employers are planning to drop GLP-1 coverage for weight loss in 2027 because too many of their employees are taking the drugs. Real world persistence is the problem nobody likes to talk about: adherence falls from 65% at 120 days to 34% at one year. The efficacy race, retatrutide at 28.3% in 80 weeks, CagriSema at 23% in 84, is happening on top of a reimbursement system that’s starting to give way. The next phase of this market isn’t about who loses the most weight, but who can stay on the drug long enough, and affordably enough, for the weight loss to matter.

Outside of obesity, the week had its share of platform wins and program failures. Novartis got an early payoff from its $12 billion Avidity acquisition with a clean biomarker win for an antisense oligonucleotide conjugate in muscular dystrophy, then doubled down on molecular glues with a second Orionis deal. Takeda’s zasocitinib beat BMS’s marketed Sotyktu head-to-head in psoriasis, the first time one TYK2 inhibitor has shown statistical superiority over another. Meanwhile Sanofi halted a Phase 3 in a rare neuropathy for insufficient efficacy, and Gilead and Merck’s Trodelvy plus Keytruda combination missed in lung cancer even as their once weekly HIV pill succeeded.

Parabilis priced the largest venture-backed biotech IPO in history at $670 million, beating Kailera’s $625 million record from two months ago. The same week, Summit Therapeutics pulled a $500 million share sale a day after announcing it, even though its PD-1/VEGF bispecific ivonescimab headlined the ASCO plenary just two weeks ago. It seems that new stories with strong science are getting funded, but existing companies that haven’t delivered on past wins aren’t.

BIOTECH/PHARMA NEWS 🧬

🔹 AstraZeneca used the American Diabetes Association meeting to convert a licensed asset into a franchise commitment. In the Phase 2 VISTA obesity trial, elecoglipron at 75 mg produced 10.5% mean weight loss at 26 weeks versus 0.6% on placebo, with the curve still climbing to 11.8% at 36 weeks. In the SOLSTICE Type 2 diabetes trial, the same dose cut HbA1c by 1.9% from baseline versus 0.2% on placebo, with 90% of treated patients reaching HbA1c below 7%. No liver safety signals, infrequent discontinuations. The efficacy is roughly in line with, not ahead of, Lilly’s oral orforglipron, which is why some analysts called the pill “relatively underwhelming.” That reading misses the strategy. AstraZeneca is launching the EMBOLD trials in obesity and the ELUMINATE trials in diabetes, including cardiovascular and kidney outcomes studies and a combination with dapagliflozin. A small molecule oral GLP-1 that’s merely competitive on weight but pairs naturally with AstraZeneca’s existing cardio-renal portfolio is a different commercial proposition than a standalone weight loss drug. The obesity market now has four serious players: Lilly, Novo, Pfizer-Metsera, and AstraZeneca. And the fight is no longer about who can produce the biggest weight loss number. It’s about who can win on convenience, combination opportunities, and payer access.

🔹 A growing share of US employers are planning to drop GLP-1 coverage for weight loss in 2027, because too many employees are taking the drugs and the costs have become unsustainable. Real world persistence makes this worse: adherence falls from 65% at 120 days to 34% at one year. The efficacy race between Lilly’s retatrutide and Novo’s CagriSema is happening on top of a reimbursement system that’s starting to give way. The next phase of the market isn’t going to be about who loses the most weight. It’s about who can be taken long enough, and affordably enough, for the weight loss to actually matter.

🔹 Novartis booked two wins on its platform acquisition strategy in one week. First, the biomarker cohort of the FORTITUDE Phase 1/2 trial of del-brax in facioscapulohumeral muscular dystrophy met its primary and key secondary endpoints, with reductions in disease biomarkers indicating strong target engagement and reduced muscle damage. Del-brax is the asset that came through Novartis’s $12B Avidity acquisition that closed in February, and the result validates that price tag while a 200-patient Phase 3 enrolls across the US and Europe. The drug is an antisense oligonucleotide conjugate, extending the modality’s category-defining run into neuromuscular disease after recent wins in hereditary angioedema and metabolic disease. Second, Novartis extended its molecular glue partnership with Orionis Biosciences, paying $40M upfront and up to $1.4B in milestones to apply Orionis’s discovery platform to hard targets. Molecular glues are the cheaper, orally deliverable cousin of the degrader modality that crossed its regulatory threshold this spring with the Veppanu approval. Novartis is buying optionality across both validated and emerging modalities rather than building each in house.

🔹 Gilead and Merck delivered a win and miss this week. The win: the Phase 3 ISLEND-1 and ISLEND-2 trials of a once weekly oral islatravir + lenacapavir regimen met their primary efficacy endpoint with safety comparable to daily standard of care in virologically suppressed patients. This would be the first oral once weekly HIV treatment, a meaningful convenience step that protects Gilead’s core antiviral business against the long acting injectable threat. The miss: the EVOKE-03 trial of Trodelvy + Keytruda versus Keytruda alone in first line PD-L1-high non small cell lung cancer was stopped early, posting a numerical but not statistically significant progression free survival improvement. The miss carries read through beyond Gilead. It lands one week after Merck’s own TROP2 antibody drug conjugate sac-TMT hit in first line lung cancer in a China-only trial, raising the question of whether the TROP2-ADC + checkpoint combination actually works in lung cancer, or whether last week’s win reflects something specific about the comparator and the patient population.

🔹 Parabilis Medicines priced the largest venture-backed biotech IPO on record, selling 33.5M shares at $20 for $670M, beating Kailera’s $625M record set in April. The company, formerly FogPharma and founded by Gregory Verdine, develops helix-shaped peptide drugs called Helicons designed to hit previously undruggable proteins. About $150M will fund the lead asset zolucatetide, a beta-catenin inhibitor heading to Phase 3 in desmoid tumors in the first half of 2027. The IPO extends a 2026 streak of 12 biotech debuts raising more than $4.1B. The same day Parabilis priced, Summit Therapeutics pulled a $500M share sale it had announced just one day earlier, citing market conditions, even though its PD-1/VEGF bispecific ivonescimab headlined the ASCO plenary just two weeks ago. Investors will pay top dollar for fresh precommercial stories and balk at adding to positions that haven’t rewarded prior good news.

🔹 Sanofi halted its Phase 3 MOBILIZE trial of riliprubart in chronic inflammatory demyelinating polyneuropathy after an independent monitoring committee found the study unlikely to show sufficient efficacy. The trial enrolled patients refractory to standard of care. No safety signals were identified. The companion VITALIZE Phase 3 in patients treated with intravenous immunoglobulin will be evaluated separately, but the failure removes one path for a complement-targeting therapy Sanofi had viewed as a potential blockbuster. CIDP is the same indication argenx and others are pursuing, and the futility readout reinforces that mechanistic rationale in autoimmune neurology isn’t translating cleanly into efficacy in refractory patients.

🔹 Takeda’s oral TYK2 inhibitor zasocitinib beat Bristol Myers Squibb’s marketed Sotyktu head-to-head in plaque psoriasis, achieving statistical superiority on all primary and secondary endpoints. At 16 weeks, more than a third of zasocitinib patients achieved complete skin clearance, more than double the rate seen on Sotyktu. This is the second approved therapy zasocitinib has topped, following an earlier win against Amgen’s Otezla. Takeda paid roughly $4B for the asset through its Nimbus Therapeutics deal, and a head-to-head superiority claim against the only marketed oral TYK2 inhibitor is the strongest possible commercial setup. Takeda is targeting a 2026 FDA filing with launch expected in 2027. Sotyktu’s positioning as the premium oral psoriasis option is now directly threatened by a next-generation molecule with cleaner data, before zasocitinib has even reached the market.

🔹 Lilly presented the full TRIUMPH-1 dataset behind its May topline at the ADA meeting. At 80 weeks, retatrutide produced mean weight loss of 28.3% (about 70 pounds) at 12 mg and 25.9% (about 64 pounds) at 9 mg in obesity without diabetes. The triple agonist hitting glucagon, GLP-1, and GIP receptors also showed superior HbA1c and weight reductions versus placebo in the TRANSCEND-T2D-1 diabetes study, with added improvements in systolic blood pressure and lipids. The 28% efficacy ceiling retatrutide set is now reinforced with full peer-presented data, and it reframes every competitor. Novo’s CagriSema, at 23% over 84 weeks, sits a tier below, and Novo’s chief scientific officer publicly conceded the jury is still out on how CagriSema stacks up against the tri-agonist. The retatrutide program now spans diabetes, obesity, sleep apnea, MASH, and cardiometabolic outcomes, making it less a drug and more a metabolic platform.

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BIOTECH/PHARMA NEWS 🧬

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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.

Image source: Angus Liu/Fierce Pharma

🎙️ 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 🔑

ASCO 2026 was the meeting where Chinese oncology stopped being a sourcing story and became a science story. For the first time in the society’s 61 year history, a drug that originated in China took the plenary stage. Akeso’s ivonescimab, a bispecific antibody that targets both PD-1 and VEGF simultaneously, beat an active PD-1 inhibitor head to head in first line squamous lung cancer and cut the risk of death by 34%. The comparator wasn’t placebo or chemotherapy. It was an approved checkpoint inhibitor, which makes this a Chinese asset setting a new global standard, published in The Lancet the same day. Behind it came two more positive China-run Phase 3 oncology readouts the same week: Bristol Myers Squibb’s bispecific antibody-drug conjugate cutting progression risk by 71% in triple-negative breast cancer, and Innovent’s claudin-targeted conjugate hitting its endpoint in gastric cancer. The center of gravity in oncology drug origination has visibly shifted east.

Washington noticed. Days after ASCO ended, a bipartisan House bill moved to fold biotechnology into the Comprehensive Outbound Investment National Security Act, which would subject US investment in Chinese biotech to national security review. The bill follows the Pfizer-Metsera and BMS-Hengrui deals from the past two months, and the framing is that Chinese drug development progress is now a security exposure. If it passes, it would put friction on exactly the licensing channel that Lilly, Merck, AstraZeneca, and BMS have been using to buy discovery speed at a discount. The cheapest, fastest moving early stage oncology data in the world is increasingly Chinese, and the US is now trying to restrict the capital that brings it onshore. Any large cap company actively negotiating a China asset deal just picked up a new timing risk.

Revolution Medicines was the Western story of the week. Daraxonrasib confirmed a doubling of overall survival in previously treated metastatic pancreatic cancer at the ASCO plenary, 13.2 months versus 6.7 months on chemotherapy, hazard ratio 0.40. Pancreatic cancer is one of the indications that has resisted progress for a generation, and the result drew a standing ovation. The more interesting signal is commercial. Patients and oncologists are already pressing for access to a drug that hasn’t been approved yet, which is something most oncology programs never have to deal with.

Lilly kept buying modality breadth while pushing back on pricing pressure. An RNA exon-editing deal with Ascidian worth up to $1.9B, a Korean GLP-2 obesity asset from Hanmi, a five program option deal with China’s Haisco, an ultimatum to 340B hospitals on discount data sharing, and a billion dollar cut to planned German manufacturing in response to German pricing reform. And in immunology, Abivax posted the best Phase 3 ulcerative colitis efficacy of the cycle and the stock got crushed anyway, because the safety table had cancer cases in it. A reminder that efficacy doesn’t clear the gate on its own when a chronic, non-life-threatening disease meets an oncogenic signal.

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🔹 Akeso’s ivonescimab became the first China-originated oncology drug ever selected for the ASCO plenary, with Phase 3 HARMONi-6 data showing it reduced the risk of death by 34% in first line advanced squamous non-small cell lung cancer versus tislelizumab, an active PD-1 inhibitor. Median overall survival hit 27.9 months, a 4.2 month improvement after 21.4 months of follow-up. Ivonescimab is a bispecific antibody that targets PD-1 and VEGF at the same time, and the results were published in The Lancet simultaneously with the presentation. This is the first regimen to show a survival edge over a checkpoint inhibitor in first line squamous disease. The geographic caveat stands. HARMONi-6 enrolled entirely in China, and Summit Therapeutics holds the US and ex-China rights and still needs Western confirmatory trials to convince the FDA. But the mechanism question is settled in a way it wasn’t before. A PD-1/VEGF bispecific can outperform the checkpoint inhibitors that currently define standard of care.

🔹 Bristol Myers Squibb’s izalontamab brengitecan, the EGFR-by-HER3 bispecific antibody-drug conjugate the company paid $800M for in 2023, posted positive Phase 3 data in the China-based PANKU-Breast02 study in previously treated metastatic triple-negative breast cancer. The drug cut the risk of progression or death by 71% versus chemotherapy and produced a 51.7% response rate against 20.5% for chemo, hitting both progression-free and overall survival co-primary endpoints. This is the first bispecific antibody-drug conjugate to clear dual primary endpoints in this setting. The competitive read for the marketed TROP2 conjugates, Gilead’s Trodelvy and the recently approved Datroway from AstraZeneca and Daiichi Sankyo, is direct. They now face a dual-target conjugate with a more potent payload landing harder on response rate.

🔹 Representatives John Moolenaar and Debbie Dingell introduced a bipartisan House bill to add biotechnology to the Comprehensive Outbound Investment National Security Act, which would require national security review of US investment in Chinese biotech. The bill follows the Pfizer-Metsera and Bristol Myers-Hengrui deals from the past two months and frames Chinese drug development progress as a security exposure. The practical effect, if it passes, would be friction on exactly the licensing channel that Lilly, Merck, AstraZeneca, and Bristol Myers Squibb have been using to buy discovery speed at a discount. ASCO proved the science is real. Washington responded by trying to gate the money. Companies mid-negotiation on China assets now carry a regulatory timing risk that didn’t exist a quarter ago.

🔹 Revolution Medicines confirmed the pivotal Phase 3 RASolute 302 result at the ASCO plenary: median overall survival of 13.2 months on oral daraxonrasib versus 6.7 months on chemotherapy in previously treated metastatic pancreatic cancer, a hazard ratio of 0.40 with p less than 0.0001. Progression-free survival was 7.2 months versus 3.6. Daraxonrasib is a multi-selective RAS inhibitor dosed at 300 mg once daily, and the survival doubling in an indication where median survival has barely moved in decades drew a standing ovation from the audience. The more telling signal is commercial. STAT reported that patients and oncologists are already pressing for access to a drug that hasn’t been approved, and analyst conversations have shifted from whether daraxonrasib works to how far the franchise expands into earlier lines and first-line use. That is a quality of problem most oncology programs never reach.

🔹 Lilly had another busy week of M&A on multiple fronts. The headline deal was an RNA exon-editing collaboration with Ascidian Therapeutics worth up to $1.9B for genetic kidney diseases. Lilly also took most rights to Hanmi’s GLP-2 obesity candidate sonefpeglutide, optioned up to five programs from China’s Haisco, and extended its delivery technology pact with Camurus. The GLP-2 mechanism is a new metabolic lever distinct from the GLP-1/GIP axis that defines Zepbound, fitting the pattern of buying adjacency rather than more of the same. At the same time, Lilly issued a data sharing ultimatum to 340B hospitals, demanding proof they are not double dipping discounts or losing access to them, and cut its planned German manufacturing investment by at least $1B in response to German drug pricing reform. Boehringer Ingelheim made a similar cut on the same day. Lilly is spending freely on modality breadth while using its scale to push back hard on any policy that compresses net pricing.

🔹 Alnylam signed a three year AI deal worth up to $2B with Inceptive Nucleics, with $30M upfront in cash and equity, to build RNA foundation models. Inceptive was founded by Jakob Uszkoreit, one of the co-authors of the original transformer architecture paper that underpins modern large language models. The aim is to design and prioritize RNAi therapeutics computationally rather than empirically. The deal sits alongside NewLimit’s $435M round for epigenetic reprogramming, with Lilly Ventures participating, as evidence that AI and biology capital is now flowing to incumbents buying compute capability, not just to startups selling it. The small upfront, large milestone ceiling structure treats AI design as a high option, low commitment bet.

🔹 Abivax’s obefazimod, an oral miR-124 enhancer in ulcerative colitis, hit its Phase 3 ABTECT maintenance primary endpoint cleanly. The 25 mg and 50 mg doses produced clinical remission at Week 44 in 50.8% and 51.3% of patients respectively versus 10.4% on placebo, a roughly 40-point placebo-adjusted gap that is among the strongest ever reported in ulcerative colitis, with all key secondary endpoints met. The market sold the stock hard anyway, because the safety table reported cancer cases among treated patients, concentrated at the high dose. This is the immunology evidentiary bar in its purest form: efficacy alone doesn’t clear the gate when a chronic, non-life-threatening disease meets an oncogenic safety question. Abivax, a reported takeover target, plans an FDA filing in Q4 2026, and the label fight will be about dose and malignancy monitoring, not whether the drug works.

🔹 Celcuity presented full Phase 3 VIKTORIA-1 data showing its gedatolisib triplet extended median progression-free survival to 11.1 months versus 5.6 months for alpelisib plus fulvestrant in PIK3CA-mutated, HR-positive/HER2-negative advanced breast cancer, a 50% reduction in progression risk. The doublet matched it at 11.3 months. An investigator called it a potential new standard of care, and gedatolisib, a multi-target inhibitor of the PI3K/AKT/mTOR pathway, clearly beat the existing PI3K inhibitor on the market. The stock fell anyway, because expectations had been set at “transformative” and an 11-month median against an old comparator read as merely good. A useful reminder that in crowded breast cancer, the bar for a clean Phase 3 win is now set by investor narrative as much as by the curve.

🔹 Roche disclosed that giredestrant, its oral selective estrogen receptor degrader, missed in first-line metastatic breast cancer, and is redirecting the program into a new adjuvant Phase 3 rather than abandoning it. The result extends a difficult stretch for the oral SERD class, coming weeks after AstraZeneca’s camizestrant drew a 6-3 ODAC vote against its first-line switch strategy. The pattern is consistent: oral SERDs are struggling to prove first-line benefit against entrenched endocrine backbones, and the survivors are retreating to narrower biomarker-defined or earlier-stage settings where the comparator is weaker.

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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.

Image source: Angus Liu/Fierce Pharma

🎙️ 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 🔑

GSK and Ionis published Phase 3 data in the New England Journal of Medicine showing that bepirovirsen, an antisense oligonucleotide, functionally cured roughly one in five chronic hepatitis B patients across two trials totaling 1,838 patients. The standard of care for the past two decades has been indefinite antiviral therapy that suppresses the virus without ever clearing it. Twenty percent functional cure rates after 24 weeks of dosing is not a marginal improvement. Combined with Madrigal-Arrowhead’s PNPLA3 deal earlier this month and Intellia’s CRISPR Phase 3 win in hereditary angioedema, oligonucleotide modalities have now stacked three category-defining wins in five weeks across virology, metabolic disease, and rare disease.

Lilly’s $3.8 billion vaccine buying spree is the most strategically interesting move the company has made all year. The official framing was about preventing disease at the source rather than treating it later, which reads as polite cover for the real story. Lilly is using GLP-1 cash flow to build franchise breadth it has never had. Curevo’s Phase 2 shingles asset goes directly at GSK’s $4 billion Shingrix franchise. LimmaTech’s Phase 1 Staphylococcus aureus vaccine targets surgical site infections, an indication with no approved vaccine. Vaccine Co.’s preclinical Epstein-Barr virus candidate is a bet on multiple sclerosis prevention. This is Lilly’s tenth acquisition of 2026, and roughly half of those deals are in modalities Lilly does not currently run at scale: gene editing, non-viral delivery, vaccines, dual-payload ADCs. You can read the strategy in the deal flow: buy delivery and infrastructure now, deploy retatrutide cash on indication coverage later.

CVS Caremark reversed its 2025 Wegovy-only obesity formulary this week, adding Foundayo as a covered option starting June 1 and restoring Zepbound as a preferred option October 1, with Wegovy’s products remaining covered. Lilly now has obesity drug coverage across all three major pharmacy benefit managers. The bear case had been that Foundayo’s launch would lag because PBM access was structurally tilted toward Novo, and indeed Foundayo had been pacing at about 30% of oral Wegovy’s six week prescription trajectory. That handicap is now lifted. Lilly shares moved up about 6% on the news. The obesity fight is back to being about efficacy, convenience, and net pricing.

Daiichi Sankyo’s Datroway became the first TROP2 antibody-drug conjugate approved in first-line triple-negative breast cancer this week, with overall survival of 23.7 months versus 18.7 months on chemotherapy. That is the first time any targeted therapy has beaten chemotherapy on survival in this patient population. ADCs are now first line therapy in tumors that have been chemotherapy-only for decades, which is the same trajectory Enhertu took in HER2-positive breast cancer: second-line approval, then label expansion, then first line displacement.

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🔹 GSK and Ionis released full Phase 3 data on May 28 for bepirovirsen in chronic hepatitis B, alongside publication in the New England Journal of Medicine. In B-Well 1, 20% of patients on bepirovirsen plus antivirals achieved functional cure, defined as sustained loss of hepatitis B surface antigen with undetectable viral DNA, versus 0% on placebo plus antivirals. B-Well 2 showed 19%. Cure rates climbed to 25-28% in patients with lower baseline viral protein levels. Patients with undetectable disease at week 48 discontinued standard antiviral therapy. Bepirovirsen is an antisense oligonucleotide developed by Ionis and licensed to GSK. The FDA decision is due October 26. Chronic hepatitis B affects roughly 254M people globally, and the current standard of care, nucleoside analogs, suppresses the virus but rarely eliminates it. A 20% cure rate with a 24-week course is a category redefining number. The open question is pricing and access in the regions where the disease burden is heaviest (China, sub-Saharan Africa, and Southeast Asia).

🔹 Lilly announced three vaccine acquisitions on May 26 worth up to $3.83B in milestone-contingent value. Curevo (up to $1.5B) has a Phase 2 next-generation shingles vaccine using a synthetic adjuvant that has shown comparable immune response with fewer side effects than Shingrix. LimmaTech (up to $780M) has a Phase 1 Staphylococcus aureus vaccine for surgical site infection prevention. Vaccine Co. (up to $1.55B) is preclinical with an Epstein-Barr virus vaccine, which has growing scientific support as a multiple sclerosis prevention target. Upfront amounts were not disclosed. This is Lilly’s tenth M&A move of 2026 and brings 2026 deal spend above $5B across vaccines, gene editing, non-viral DNA delivery, and dual payload ADCs. The strategic frame is that Lilly is treating GLP-1 cash flow as a one-time opportunity to build modality and indication breadth before the next cycle.

🔹 CVS Caremark reversed its 2025 Wegovy-only obesity formulary on May 28, adding Foundayo (oral orforglipron) effective June 1 and restoring Zepbound as a preferred option October 1. Wegovy stays covered. Lilly’s obesity drugs now have coverage across all three major pharmacy benefit managers. The structural handicap on Foundayo’s launch, which had been running at about 30% of oral Wegovy’s early prescription pace, is now lifted. Lilly shares moved up about 6% on the announcement. The commercial competition resolves back to a head-to-head on efficacy, convenience, and net pricing, and Lilly’s retatrutide submissions later this year only widen the efficacy gap.

🔹 Verve Therapeutics, now owned by Lilly, presented Phase 1 data for VERVE-102 at the European Atherosclerosis Society Congress on May 26, with simultaneous publication in the New England Journal of Medicine. The trial enrolled 35 patients across six dose levels. LDL cholesterol reductions ranged from 9% to 62% across doses, with PCSK9 protein reductions of 55% to 88%. Durability of effect held for up to 18 months after a single infusion, the longest follow-up disclosed for any in vivo base editing therapy. No treatment-related adverse events. The top dose effect approaches what Amgen’s Repatha delivers as a chronic biologic that costs about $6k a year. Lilly plans to launch Phase 2 by the end of 2026. The regulatory question has shifted from whether the technology works to whether 18 month durability is sufficient to support a one time administration label rather than periodic redosing.

🔹 Apogee Therapeutics secured up to $1.3B in non-dilutive royalty financing from Blackstone Life Sciences to fund three Phase 3 trials of zumilokibart, an anti-IL-13 antibody, in atopic dermatitis. Phase 2 data showed 66% of mid-dose patients achieved 75% skin clearance versus 23% on placebo. The drug’s structural differentiator is dosing frequency: 2 to 4 injections per year versus every 2 to 4 weeks for Dupixent and Ebglyss. Trials begin in the second half of 2026. The fact that Blackstone is willing to underwrite three Phase 3 trials non-dilutively says something about how the eczema biologic market is now structurally bid for differentiated dosing rather than just efficacy.

🔹 Daiichi Sankyo and AstraZeneca’s Datroway received FDA approval on May 22 in first line metastatic triple negative breast cancer for patients ineligible for checkpoint inhibitor therapy. The Phase 3 trial showed median overall survival of 23.7 months versus 18.7 months on chemotherapy, a hazard ratio of 0.79. This is the first TROP2 antibody-drug conjugate approved in first-line triple-negative breast cancer and the first targeted therapy to show an overall survival benefit over chemotherapy in this population. Datroway now competes against Gilead’s Trodelvy and pushes ADC platforms further into first-line settings that have been chemotherapy-only for decades.

🔹 Biogen and Denali announced that BIIB122, an LRRK2 inhibitor, missed its primary endpoint in the Phase 2b LUMA trial in idiopathic Parkinson’s disease. The partners are ending development in that population. The trial tested whether LRRK2 inhibition slowed motor and clinical progression in idiopathic patients regardless of LRRK2 mutation status, and the answer was clearly no. The parallel LIGHTHOUSE trial in LRRK2 mutation-positive patients continues. For Denali, the commercial story remains AVLAYAH, their brain-penetrant Hunter syndrome therapy launched in April. For Biogen, this is the second neurology Phase 2b miss in twelve months and reinforces a pattern of large cap neurology platforms failing on symptom-progression endpoints despite biomarker rationale.

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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.

Image source: 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.

Treating a new realm of diseases

Beyond traditional limits: explore the drug delivery technology opening new doors in genomic medicine.

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VIDEO SUMMARY

THIS WEEK’S KEY TAKEAWAYS 🔑

The number that defines this week is 28.3%. That is the average body weight lost on the 12 mg dose of Lilly’s retatrutide in TRIUMPH-1, the first successful Phase 3 readout for the GIP/GLP-1/glucagon triple agonist that has been the most watched obesity asset in the pipeline for two years. A second pivotal trial in patients with knee osteoarthritis hit 28.7% plus a 75.8% reduction in pain scores. We have officially entered the territory where a pill or injection produces weight loss comparable to bariatric surgery. The bear case on incretin pricing pressure may eventually arrive, but the front of the efficacy curve just moved again, and Novo Nordisk’s CagriSema and Pfizer’s Metsera assets now have to be measured against a 30% ceiling rather than a 25% one. The wrinkle worth holding onto is the discontinuation curve. 11.3% of patients on the 12 mg dose dropped out for adverse events versus 4.9% on placebo, which is why the 9 mg arm is probably the commercial sweet spot.

The other big story this week is about how pharma is choosing to build, not what it is building. The same Lilly that posted the retatrutide data also acquired Engage Bio for up to $202 million, its seventh M&A move of 2026, to pick up a non-viral DNA delivery platform. Bristol Myers Squibb signed a strategic agreement with Anthropic to deploy Claude across more than 30,000 employees in a workflow-embedded configuration that goes well beyond a chatbot. Incyte struck two AI deals the same week. The emerging pattern is that large pharma is buying discovery infrastructure now, and enterprise AI has crossed from “nice to have” into table stakes.

Two weeks after Marty Makary’s exit, the FDA story has moved from leadership churn into something more operational. Endpoints published an investigation into the coordinated campaign of biotech executives, lobbyists, and former FDA officials that pushed him out, with Biohaven CEO Vlad Coric as a central figure after his company’s pushback on a late-April complete response letter. On the same day, the acting head of NIAID reportedly stepped down, and NIH Director Jay Bhattacharya was grilled by Senate Appropriations on the 2027 budget. The leadership vacuum is widening, not narrowing. If you have a PDUFA date or a Type B meeting on the calendar this summer, you should probably plan for delays.

Outside obesity and US policy, the most strategically important readout came out of China. Merck partner Kelun-Biotech’s sac-TMT combined with Keytruda cut the risk of tumor progression or death by 65% versus Keytruda alone in first-line PD-L1-positive non-small cell lung cancer. This is the first Phase 3 trial of an antibody-drug conjugate combined with a checkpoint inhibitor to win in first line lung cancer. Keytruda loses patent exclusivity in 2028, and Merck’s strategic response is to convert it into a backbone for combination regimens. Nobody really cares anymore that the asset came from China. The next real test is whether the Western confirmatory study running in parallel reproduces the result.

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🔹 Lilly reported positive Phase 3 topline results for retatrutide, its triple-agonist obesity drug, in adults with obesity or overweight plus at least one weight-related comorbidity without diabetes. After 80 weeks, the 12 mg dose drove 28.3% weight loss, roughly 70 pounds on average, versus 2.2% on placebo. The 9 mg dose delivered about 24% and the 4 mg dose about 19%. Discontinuation rates due to adverse events were 4.1%, 6.9%, and 11.3% across the three doses versus 4.9% on placebo, with nausea, diarrhea, and vomiting as the dominant side effects. A second trial in patients with obesity and knee osteoarthritis hit 28.7% weight loss and a 75.8% reduction in WOMAC pain scores at the high dose. Seven additional retatrutide Phase 3 trials read out through 2026 across diabetes, sleep apnea, MASH, chronic low back pain, and cardiovascular outcomes. Lilly is no longer fighting on the same efficacy frontier as Novo’s CagriSema, which posted 22.7% in a comparable population last year. The likely commercial read is that retatrutide expands the obesity market rather than cannibalizing Zepbound, because the 12 mg discontinuation rate creates real-world dose ceiling effects. Lilly will not run pricing off the 12 mg label.

🔹 Lilly also closed its acquisition of Engage Bio for up to $202M, picking up a non-viral DNA delivery platform that combines engineered DNA payloads with lipid nanoparticles. The aim is to solve the persistent problems with gene therapy delivery: potency, tolerability, and the ability to redose, all of which have constrained the AAV-based approaches Sarepta and others have relied on. This is Lilly’s seventh acquisition of 2026 and continues a clear strategic pattern: the company is treating genetic medicine as a delivery problem rather than a payload problem.

🔹 Endpoints News published an investigation this week describing a multi-month coordinated effort by biotech executives, lobbyists, and former FDA officials that contributed to Marty Makary’s departure on May 12, with Biohaven CEO Vlad Coric featured prominently after Biohaven’s late-April complete response letter on a spinocerebellar ataxia program. The broader regulatory landscape is also fraying. The acting head of NIAID reportedly stepped down, and NIH Director Jay Bhattacharya faced bipartisan Senate scrutiny on the 2027 budget. The practical implications include real delay risk for PDUFA decisions, Type B meeting outcomes, and advisory committee scheduling for at least the rest of the quarter, with small-cap companies under $200 million in cash and active regulatory engagement the most exposed.

🔹 Kelun-Biotech released the OptiTROP-Lung05 Phase 3 abstract ahead of ASCO 2026, reporting a 65% reduction in the risk of tumor progression or death for sac-TMT combined with Keytruda versus Keytruda alone in previously untreated PD-L1-positive non-small cell lung cancer. Sac-TMT is a TROP2-directed antibody-drug conjugate carrying a topoisomerase I inhibitor payload, and Merck licensed ex-China rights from Kelun back in 2022. This is the first Phase 3 of any antibody-drug conjugate plus checkpoint inhibitor combination to hit in first line lung cancer. The competitive read matters as AstraZeneca and Daiichi Sankyo’s datopotamab deruxtecan has struggled to convert Phase 3 signals into a first line label, and Merck now has a credible path to turn Keytruda into a combination backbone heading into its 2028 patent expiration. The enrollment was China-only, and Western confirmatory trials are running in parallel, so the question of whether the regional efficacy translates is real. The 65% magnitude is large enough that even a substantial regional discount would still be commercially material.

🔹 BMS signed a strategic agreement with Anthropic to deploy Claude across more than 30k employees globally, with use cases spanning scientific literature summarization, clinical protocol drafting, internal data querying, and routine documentation. The deal also includes Claude Code for research and drug development functions. Combined with Incyte’s two AI deals the same week, it seems that enterprise AI is now baseline infrastructure for top-20 pharma rather than a differentiated capability.

🔹 Immunovant reported Week 16 results from its IMVT-1402 trial in difficult-to-treat rheumatoid arthritis, with ACR20, ACR50, and ACR70 response rates of 72.7%, 54.5%, and 35.8% in patients who had failed at least two prior advanced therapies, including both JAK and anti-TNF inhibitors. Response rates held in the subgroup that had failed both classes. Shares moved on the print. The proof-of-concept readout in cutaneous lupus is expected in the second half of 2026 and will be the next decisive print for the company’s autoimmune blockbuster thesis.

🔹 BioMarin’s Voxzogo hit the primary endpoint in a Phase 3 trial in hypochondroplasia, with annualized growth velocity 2.33 cm per year above placebo at Week 52, opening a second pediatric short-stature indication beyond the original achondroplasia label. Filings are planned for the second half of 2026. Two days earlier, BMN 401, an enzyme therapy acquired in the 2025 Inozyme buyout, missed its radiographic clinical endpoint in ENPP1 deficiency despite hitting on the biomarker co-primary, and will require FDA discussion on surrogate to clinical translation.

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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.

You can listen to this episode on YouTube, Spotify or Apple Podcasts.

In the latest joint podcast episode by Biotech Blueprint × Biotech Capital Compass, we zoom in on Moderna and where the company stands in 2026. We work through the Q1 earnings beat, the unprecedented FDA refusal-to-file on mRNA-1010, the CMV Phase 3 failure, the Vertex cystic fibrosis wind down, and the individualized neoantigen therapy (INT) program that has moved to the center of the pipeline. The short companion piece below picks up one thread from the conversation, which is the pattern across Moderna’s wins and losses, and what it suggests about where the platform actually works.

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Here’s a link to our previous mRNA podcast episodes:

The successes are all acute. COVID-19, RSV via mRESVIA, the seasonal flu candidate, INT. Each one needs a burst of antigen or a pulse of immune training, and then the body takes it from there. The disappointments are the opposite. CMV is a latent virus that hides intracellularly for life and needs durable, often mucosal immunity to keep suppressed. The Vertex-partnered inhaled mRNA program for CF patients without CFTR protein required continuous replacement of a protein the body cannot make on its own. Both programs were asking the platform to do something it isn’t really built to do.

The biology is straightforward. An mRNA-LNP delivers its cargo into a cell, the ribosomes translate it for a day or two, cytosolic nucleases degrade the mRNA, the LNP clears, and detectable protein is gone within a week or two. Modified nucleosides like N1-methylpseudouridine stretch the translation window a bit and dampen innate sensing, but they do not change the fundamental kinetics. mRNA is, by design, a short-burst technology. That is exactly what you want for a vaccine. It is much harder to make it the right tool for sustained protein replacement, or for the kind of tissue-resident memory T cell response you would want against a virus that is going to live in your body forever.

Hartaj framed the underlying question on the episode this way:

“…One of the things that Moderna is having to go through right now is figure out where is it really that the mRNA modality can work.”

— Hartaj Singh (Biotech Capital Compass)

One way to read the last year of Moderna news is that the platform has been answering that question for them. The successes mark out the shape of what it does well and the failures mark out where it doesn’t. The strategic pivot toward oncology and acute respiratory, and away from latent viruses and chronic protein replacement, looks less like a retreat under cash pressure and more like the company drawing the right roadmap.

That makes INT (mRNA-4157) the cleanest test of where this goes. Each product codes for up to 34 patient-specific neoantigens, selected from tumor sequencing by predicting which mutated peptides will bind that patient’s HLA molecules and present on the tumor surface where cytotoxic T cells can find them. Melanoma is the natural first indication because it carries one of the highest tumor mutational burdens of any solid tumor, which gives the selection pipeline more candidates to choose from. Non-small cell lung cancer extends the test to a much larger patient population. INT plays to what mRNA is good at. A focused immune-training event, then the patient’s T cells do the work. If the melanoma signal holds in NSCLC, the read above is probably right. If it doesn’t, mRNA’s reach is narrower than even this piece is suggesting.

Everything else from the episode is near-term noise. The Q1 earnings beat was helpful but ambiguous, since Q1 is seasonally weak and expectations had been cut. The August 5 PDUFA decision on mRNA-1010 will tell us more about whether the regulatory environment is functionally hostile to mRNA respiratory vaccines under this administration than it will about the trial itself.

One quick teaser for next time. The thread we deliberately did not pull on this week is the role of machine learning in INT. The FDA has indicated it wants the neoantigen selection algorithm submitted as part of the regulatory package, which is the first time we are aware of an AI/ML system being treated as a regulated component of a therapy rather than as a tool sitting upstream of it. That is a bigger story than it sounds, and it gets its own episode.

Three Takeaways

1. Moderna’s successes and failures cluster along one axis. Acute, episodic, pulse-style indications on one side. Sustained, latent, chronic indications on the other.

2. The strategic pivot reads more cleanly as realignment. The company is moving toward indications where the biology fits.

3. INT is the test. The 2026 readouts will tell us whether the platform is broadly useful in oncology, or whether even oncology turns out to be narrower than we hope.

Listen to the full episode on YouTube, Spotify, or Apple Podcasts. Our next episode picks up the thread on AI, machine learning, and what it means when the algorithm itself becomes part of the drug.

Thanks for reading Biotech Blueprint!

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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.

Photo credit: Moderna

🎙️ 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 🔑

Three large stories defined the week. Bristol Myers Squibb committed up to $15.2B to license 13 early stage assets from China's Hengrui Pharma, betting that external discovery at Chinese R&D speed is the fastest path to pipeline. On the same day, FDA Commissioner Marty Makary resigned after weeks of pressure, adding a new layer of regulatory unpredictability to an already unstable agency. And on Thursday, Regenxbio posted a clean Phase 3 win in Duchenne muscular dystrophy, landing in the middle of Sarepta’s ongoing Elevidys safety crisis.

The BMS-Hengrui deal is really about the China R&D speed differential. BMS executives explicitly cited that clinical trial filing timelines in China run 50-70% faster than the rest of the world, and they structured the deal accordingly. $600M upfront, another $350M in fixed payments over 30 months, four Hengrui oncology and hematology assets ex-China, four BMS immunology assets going the other direction, and five jointly discovered programs. The specific molecular targets weren’t disclosed. Chinese discovery engines are now treated as outsourceable infrastructure by large pharma, not competitive risk. Pfizer’s Kailera deal, Merck’s Terns acquisition, and now this are all expressions of the same logic.

Makary’s resignation is the story with the most immediate practical consequences for drug developers. His tenure produced unpredictable rejections, including Replimune’s second FDA rejection in late April that cost the company 60% of its workforce, high profile leadership departures including CDER director George Tidmarsh and oncology chief Richard Pazdur, who joined and left within three weeks, and sustained public industry frustration. Acting commissioner Kyle Diamantas comes from the food side of the FDA with no drug review track record. For companies midway through regulatory review, the risk is silence because Type B meetings might stall through summer while a permanent successor is found.

Regenxbio’s RGX-202 result is the cleanest dataset of the week. In 30 boys with Duchenne muscular dystrophy, a single dose of the gene therapy drove mean microdystrophin expression of 71.1%, with 93% of patients crossing the 10% threshold that served as the primary endpoint. More importantly, the trial formally bridged biomarker to function: microdystrophin expression at week 12 was statistically significantly correlated with functional improvement at one year on a standardized motor assessment. That biomarker-to-function link is what prior Duchenne gene therapy submissions have been missing, and it's what gives this dataset a credible accelerated approval path rather than just scientific interest. Sarepta’s Elevidys currently carries a boxed warning for liver injury after three patient deaths and lost its platform technology designation. RGX-202 gives prescribers a real second option for the first time.

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BIOTECH/PHARMA NEWS 🧬

🔹 Bristol Myers Squibb announced a global strategic collaboration with Hengrui Pharma on May 12 covering 13 early stage programs across oncology, hematology, and immunology. BMS pays $600M upfront, $175M on the first anniversary, and a contingent $175M in 2028, with total deal value reaching $15.2B if all milestones are hit. BMS gets exclusive ex-China rights to four Hengrui oncology and hematology assets. Hengrui gets four BMS immunology assets inside Greater China, and five programs will be jointly discovered with Hengrui leading early clinical work. Specific targets were not disclosed. The deal closes Q3 2026 pending antitrust clearance. This is the largest single pharma-China licensing deal of 2026 and the clearest signal yet that big pharma has stopped treating Chinese R&D as a competitive threat and started treating it as a supply chain.

🔹 FDA Commissioner Marty Makary resigned on May 12, with Deputy Commissioner for Food Kyle Diamantas named acting head. The immediate trigger was reportedly a dispute with the White House over fruit-flavored vape approvals, but the structural pressure had been building for months: CDER director George Tidmarsh departed, Richard Pazdur joined CDER and left within three weeks, and a string of high profile rejections including Replimune’s second complete response letter generated sustained industry pushback. The Biotechnology Innovation Organization issued a same day statement calling for organizational stability. Diamantas has no drug review background, and a permanent successor has not been named. For companies in active regulatory review, the near term risk is delays in Type B meeting feedback and PDUFA decisions while the commissioner's office transitions. Industry executives have reportedly floated Pazdur as a permanent replacement, though his recent departure complicates that path.

🔹 REGENXBIO reported positive pivotal Phase 3 data for RGX-202, an AAV8-delivered microdystrophin gene therapy for Duchenne muscular dystrophy, on May 14. In 30 ambulatory boys, 93% achieved microdystrophin expression above 10% at week 12, with mean expression of 71.1% across the full cohort. The headline number that matters most for the regulatory path: microdystrophin expression at week 12 was statistically significantly correlated with functional improvement on the North Star Ambulatory Assessment at one year, the first time a Duchenne gene therapy has formally bridged biomarker to function in a registration dataset. Two serious adverse events were reported, a case of subacute myocarditis that resolved and an asymptomatic liver enzyme elevation that normalized. REGENXBIO is pursuing accelerated approval with a 2027 commercial launch target. Sarepta’s Elevidys carries a boxed warning for serious liver injury after three patient deaths and is approved only for ambulatory patients aged 4 and older. RGX-202’s safety profile to date is the commercial differentiator.

🔹 Isomorphic Labs, the Alphabet-backed DeepMind spinout focused on AI drug design, closed a $2.1B Series B on May 12 led by Thrive Capital, with participation from Alphabet, GV, Temasek, and both the UK Sovereign AI Fund and Abu Dhabi’s MGX. This is the largest AI biotech private round on record. The capital funds continued development of the company’s AI drug design engine and its own therapeutic pipeline, with first-in-human trials targeted for late 2026. Isomorphic retains active partnerships with Novartis, Lilly, and J&J. This is no longer a company positioning itself as a service layer for pharma. It’s funded to be an integrated drug discoverer and developer. Whether AI-designed molecules clear Phase 1 this year is the credibility test for the entire category.

🔹 argenx received FDA approval on May 8 expanding Vyvgart and Vyvgart Hytrulo to all adult patients with generalized myasthenia gravis, including antibody negative patients who previously had no on label biologic options. The Phase 3 ADAPT SERON trial in 119 antibody-negative patients hit its primary endpoint with p=0.0068. argenx reported Q1 product net sales of $1.3B, up 63% year-over-year, its seventeenth consecutive quarter of growth. The label expansion removes the antibody status gating step that had been slowing prescribing decisions in community neurology. Vyvgart is now the only treatment approved for the full generalized myasthenia gravis patient population.

🔹 Italy’s Angelini Pharma agreed to acquire Catalyst Pharmaceuticals for $31.50 per share in cash, roughly $4.1B and a 28% premium, giving Angelini its first US foothold through Catalyst’s rare neurology portfolio: FIRDAPSE, the only FDA-approved treatment for Lambert-Eaton myasthenic syndrome, AGAMREE, an alternative steroid for Duchenne, and US rights to FYCOMPA for partial-onset seizures. This is the second European mid cap pharma company to buy a US-marketed rare neurology asset in as many weeks, after Chiesi’s KalVista deal, and the pattern likely reflects urgency to build US-marketed inventory ahead of Section 232 pharma tariffs taking effect July 31.

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WATCHLIST 👀

• ASCO 2026 Plenary, May 31: Daraxonrasib LBA5 from Revolution Medicines (Phase 3 RASolute 302, 2L metastatic PDAC, OS 13.2m vs 6.7m, HR 0.40). Final analysis and additional subgroup detail. Plenary slot is the highest-profile oncology platform of 2026.

• FDA permanent commissioner nomination: Trump has named Diamantas only as acting. Pazdur was floated by industry but already departed. Watch for nomination signals from HHS and Senate HELP Committee

• REGENXBIO BLA submission for RGX-202: Accelerated approval pathway flagged. FDA surrogate endpoint discussion is the gating step. Likely Q3 2026 filing.

• EHA 2026 Congress, Stockholm, June 11-14: First clinical data from Ajax Therapeutics’s AJ1-11095 (Type II JAK2 inhibitor for myelofibrosis), the asset Lilly is acquiring for up to $2.3B (closing expected H2 2026)

• Cytokinetics aficamten NDA filing timeline: Investor day or topline regulatory commentary at HFSA expected; ACACIA-HCM full data presentation at upcoming cardiology meeting.

• BMS-Hengrui assets disclosed at closing: Specific molecular targets, modality breakdown, and any GLP-1 or ADC programs included in the 13 asset portfolio

• Q2 2026 earnings season, late July-early August: First quarter under MFN drug pricing implementation; first commentary on Section 232 pharma tariff impact (effective July 31)

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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: Anna Moneymaker/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 🔑

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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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.

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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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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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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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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: 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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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: Shutterstock.com

🎙️ 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.

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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.

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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CLINICAL TRIAL UPDATES 📊

🔹 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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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.