Trump cuts IVF & Medicaid drug prices; Novo buys Omeros; Lilly's GLP-1 orforglipron shines — This Week in Biotech #74
Plus: BioCryst-Astria rounds out HAE, Praxis’ essential tremor pill succeeds in Phase 3, and Rocket’s LAD-I gene therapy returns to FDA review (Oct 10-16, 2025).
Hi and welcome to This Week in Biotech by Biotech Blueprint, edition 74, covering biotech & pharma news from October 10th to 16th, 2025.
🎙️ 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.
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THIS WEEK IN BIOTECH VIDEO SUMMARY
THIS WEEK’S KEY TAKEAWAYS 🔑
Trump’s MFN expands and now includes IVF. After Pfizer, AstraZeneca agreed to Most Favored Nation pricing for state Medicaid and direct-to-consumer discounts via TrumpRx.gov (live 2026) alongside a $50B U.S. investment plan. Separately, EMD Serono (Merck KGaA U.S.) will sell IVF meds (Gonal-f, Ovidrel, Cetrotide) at steep cuts starting Jan 2026 and will seek a speedier FDA review for Pergoveris.
Novo Nordisk buys into complement: Omeros deal (up to $2.1B). Novo gets global rights to zaltenibart (OMS906), a MASP-3 inhibitor that blocks the alternative complement pathway. Positive Phase 2 in paroxysmal nocturnal hemoglobinuria. Phase 3 global program next and kidney diseases (IgAN, C3G, aHUS) on deck.
Lilly’s oral GLP-1 keeps winning. Orforglipron hit primary and all key secondaries in two phase 3 T2D trials: vs dapagliflozin (A1C to -1.7% at 40 wks) and as add-on to insulin glargine (A1C to -2.1%). Safety/tolerability consistent with prior studies. Obesity filing in 2025; T2D filings in 2026.
Praxis nails essential tremor (ET). Ulixacaltamide delivered two phase 3 wins: –4.3 on the mADL11 functional scale at week 8, and maintenance of response 55% vs 33% in a randomized-withdrawal test. No drug-related serious AEs; pre-NDA meeting requested with the FDA, aiming to file early 2026.
BioCryst buys Astria to cover both HAE modalities. Cash/stock deal implies $13/share ($700M EV) for navenibart, a long-acting plasma kallikrein antibody for hereditary angioedema (HAE) dosed every 3-6 months. Pairs with Orladeyo (once-daily oral) to give doctors an oral and an injectable option.
Rocket’s gene therapy back on FDA track. FDA accepted the resubmitted BLA for Kresladi (lentiviral therapy for severe LAD-I, a rare immune disorder) with PDUFA Mar 28, 2026. Backed by phase 1/2 showing 100% 12-month survival and fewer serious infections.
If you skim nothing else, these six set the week’s tone: policy pressure translating into practical discount channels, a rare disease license for Novo, continued GLP-1 dominance from Lilly, a breakout CNS readout from Praxis, targeted portfolio shaping at BioCryst, and a key regulatory reset for Rocket.
BIOTECH/PHARMA NEWS 🧬
🔹 TrumpRx & MFN pricing: The White House’s Most Favored Nation (MFN) campaign added AstraZeneca (following Pfizer): state Medicaid gets MFN prices on all AZ drugs (including future launches), and AZ will sell directly to consumers via TrumpRx.gov starting in 2026, alongside a $50B U.S. manufacturing/R&D pledge (new Charlottesville, VA API site; 3,600 jobs). Separately, but part of the same TrumpRx track, EMD Serono (Merck KGaA U.S.) will offer steeply discounted IVF meds (Gonal-f, Ovidrel, Cetrotide) via TrumpRx.gov beginning Jan 2026 (company cites 84% savings for a typical protocol), and will seek expedited FDA review (1-2 months) for Pergoveris to expand options. Administration also moved to let employers offer IVF benefits as an add on. Big picture: meaningful list price relief paths are forming, but watch implementation, employer uptake on IVF benefits, and any legal challenges.
🔹 Novo Nordisk struck a rare-disease deal with Omeros for MASP-3 inhibitor zaltenibart (OMS906), paying $340M upfront/near-term and up to $2.1B total for exclusive global rights. Zaltenibart blocks the alternative complement pathway (upstream at MASP-3) and has positive phase 2 data in paroxysmal nocturnal hemoglobinuria (PHN), a rare blood disorder. Novo plans a global phase 3 in PNH and will explore kidney indications (e.g., IgAN, C3G, aHUS). Omeros keeps tiered royalties and certain preclinical MASP-3 rights. Shares of Omeros more than doubled on the news. Closing targeted for Q4 2025.
🔹 Eli Lilly’s oral, once-daily GLP-1 orforglipron cleared two more phase 3 bars. ACHIEVE-2 (vs. dapagliflozin on metformin) cut A1C up to -1.7% vs -0.8% at 40 weeks. ACHIEVE-5 (add-on to insulin glargine) cut A1C up to -2.1% vs -0.8% placebo, with weight loss and mostly mild GI side effects. Lilly now has head-to-head superiority over two active comparators in T2D (dapagliflozin here; oral semaglutide previously), bolstering the “foundational oral GLP-1” case ahead of an obesity filing by year-end and T2D submissions in 2026 (ACHIEVE-4 readout due early 2026).
🔹 ViiV Healthcare says its long-acting HIV PrEP shot cabotegravir (Apretude) beat Gilead’s lenacapavir on patient/clinician preference and injection-site tolerability in the phase 1 CLARITY crossover study (n=63). After single doses of each, 69% rated cabotegravir injections “totally/very acceptable” vs 48% for lenacapavir, and 90% of participants (86% of HCPs) preferred cabotegravir. Lenacapavir produced more, and more visible, local reactions. Results (presented at EACS 2025) are about acceptability after one dose, not efficacy, but they could shape real-world choice when initiating long acting PrEP.
🔹 Psychedelic-therapy names popped after North Carolina GOP state senator Bob Brinson said the state could “lead the nation” on expanding access for veterans, following a bipartisan event with Rep. Eric Ager that highlighted patient stories and ongoing research. Atai (ATAI) and Compass (CMPS) jumped double digits, with MindMed (MNMD) and Cybin (CYBN) also up. The remarks add momentum to a bipartisan bill that would form a state Psychedelic Medicine Task Force to study access and implementationm another small but notable policy tailwind the sector is trading on.
🔹 Shah Capital (7.2% holder, second-largest) sent Novavax’s board a blistering letter urging an immediate strategic review (including a sale) citing three years of rollout fumbles, weak marketing, costly dilution and a pricey August convertible refi. The fund argues Novavax’s value sits in its COVID/flu combo and Matrix-M adjuvant, which it says would realize greater upside inside a larger pharma, and even floated a $5B takeout as plausible. Novavax said the board remains open to “credible options.” NVAX ticked about 3% higher on the headlines.
🔹 Rocket Pharma pops as FDA resets review clock for Kresladi. The FDA accepted Rocket’s resubmitted BLA for Kresladi (lentiviral gene therapy for severe LAD-I), setting a PDUFA date of Mar 28, 2026. Recall the first filing drew a CRL in June 2024 requesting more data. The package includes phase 1/2 results with 100% 12-month overall survival, meaningful infection reductions, and no treatment-related SAEs. If approved, Rocket would be eligible for a Rare Pediatric Disease PRV. RCKT is up 10-20% on the news.
🔹 BioCryst to buy Astria Therapeutics for $13/share. BioCryst (BCRX) agreed to acquire Astria Therapeutics (ATXS) in a cash and stock deal valuing Astria at an implied $13 per share ($700M EV), a 53% premium to Oct. 13. ATXS jumped 42% premarket, while BCRX fell 9-11%. The prize is navenibart, a long-acting injectable for hereditary angioedema now in phase 3 (every 3-6 months dosing), which pairs with BioCryst’s oral Orladeyo to cover both HAE modalities. BioCryst says it expects to remain non-GAAP profitable and cash-flow positive post-deal, and closing is targeted for Q1 2026.
🔹 After last Friday’s WSJ scoop that J&J is in talks to buy Protagonist Therapeutics, on Monday rumors (Betaville) suggested a consortium led by J&J and possibly Blackstone could bid around $75/share, below where PTGX traded on the news, sending the stock lower. A deal would consolidate the partners’ oral peptide icotrokinra (NDA filed for plaque psoriasis) and add rusfertide for polycythemia vera (with Takeda) as J&J looks to backfill Stelara erosion. Talks are ongoing, terms may change, and no agreement is guaranteed. J&J reports Q3 Tuesday, which could clarify (or not).
CLINICAL TRIAL UPDATES 📊
🔹 Praxis surged after ulixacaltamide delivered in two phase 3 essential tremor studies: Study 1 showed a 4.3-point mADL11 improvement at week 8 with all key secondaries positive. In Study 2’s randomized-withdrawal phase, 55% on drug maintained response vs 33% on placebo. Safety was manageable with no drug-related serious events. Because Essential3 was fully home-based, some discontinuations may overstate real-world tolerability, one reason analysts leaned constructive: Guggenheim lifted its PT to $350 (80% probability of success) and Jefferies to $300 (70% probability of success). A pre-NDA request is in, with an NDA filing planned for early 2026, positioning a potential first-in-class oral for a large, undertreated ET market.
🔹 Vor Bio ticked up after partner RemeGen reported a clean win in a China phase 3 for telitacicept in primary Sjögren’s. The study hit the primary and all secondary endpoints, with 71.8% on 160 mg achieving a ≥3-point ESSDAI drop at week 24 vs 19.3% on placebo, and benefits sustained to 48 weeks. Safety looked placebo-like with no new signals. Vor says it’s evaluating timing for a global phase 3, positioning dual BAFF/APRIL blockade as a potential first disease-modifying therapy in pSjD.
🔹 Arcus Biosciences reported first overall survival data for domvanalimab (anti-TIGIT) plus zimberelimab (anti-PD-1) with chemotherapy as first-line therapy for unresectable/metastatic gastric/GEJ/esophageal adenocarcinoma (n=41) delivered a median overall survival of 26.7 months, median progression-free survival of 12.9 months, and a 59% response rate, with 50% alive at 24 months. Benefits appeared across PD-L1 subgroups, and safety looked in line with PD-1 + chemo (immune-mediated AEs 22%, infusion reactions 7%), supporting the ongoing phase 3 STAR-221 study.
🔹 Tvardi Therapeutics imploded after its oral STAT3 inhibitor TTI-101 failed the phase 2 REVERT trial in idiopathic pulmonary fibrosis: no meaningful Forced Vital Capacity benefit vs placebo and massive dropouts from GI side effects (discontinuations: ~10% placebo vs 57%/62% on 400/800 mg). Management is analyzing the miss while pivoting to TTI-101 in hepatocellular carcinoma and a next-gen STAT3, TTI-109 (HV topline Q1 2026). Stock down >80% in five days. IPF thesis broken, and the platform now must re-earn credibility in oncology.
PUBLIC HEALTH/SCIENCE SPOTLIGHT 🩺
🔹 New York reported the first locally acquired U.S. chikungunya case since 2019. A Nassau County resident was likely infected via a local mosquito bite after domestic travel. Risk is currently low as temps drop, but the CDC has logged 80+ travel-linked U.S. cases this year amid global surges. For prevention, health officials stress repellents, long sleeves, and eliminating standing water, and note there are FDA-approved chikungunya vaccines from Valneva and Bavarian Nordic for travelers at risk.
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