Biotech Heads Into 2026: Obesity, Vaccines, Pricing & Risk — This Week in Biotech #83
A year end / new-year update on what actually moved during the holiday stretch (Dec 19, 2025 – Jan 1, 2026).
Happy New Year 2026!
Welcome back to This Week in Biotech by Biotech Blueprint, edition 83, covering biotech and pharma news from December 19th, 2025 through January 1st, 2026.
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VIDEO SUMMARY
THIS WEEK’S KEY TAKEAWAYS 🔑
Despite the holidays, there were several important developments in biotech and pharma over the past two weeks. On the commercial side, Novo Nordisk widened its lead in obesity with FDA approval of oral Wegovy. This step removes the needle from the equation. At a relatively aggressive self-pay price, the pill is about unlocking the next wave of demand among patients who never wanted injections in the first place. In obesity, convenience is now a competitive weapon.
Large pharma also leaned into durability over novelty. Sanofi’s agreement to acquire Dynavax is a straightforward bet on adult vaccination as a stable, cash-generating category, anchored by Heplisav B’s two dose hepatitis B regimen and Sanofi’s global vaccines platform. At the same time, U.S. vaccine policy is clearly entering a noisier phase. There is momentum in Washington to revisit parts of the childhood vaccination framework and how immunization shows up in government quality measures. But big federal changes can’t happen by announcement alone. They still require evidence, process, and legal backing, which slows how much actually changes in practice.
Policy pressure extended beyond vaccines. A federal judge temporarily blocked a proposed 340B rebate pilot, preserving the current discount structure after finding the program was likely rushed and inadequately justified. Separately, new Most Favored Nation drug pricing agreements tied discounted prices to tariff relief and domestic manufacturing commitments. Even though the initial scope is limited, the structure matters. Pricing, access, and supply chain policy are increasingly being negotiated together, adding another layer of complexity for companies planning U.S. launches.
Clinical outcomes reminded investors why biotech remains a high variance business. Several FDA approvals, including treatments from Cytokinetics, Agios, and Vanda, validated years of development work and opened new commercial chapters. At the same time, late-stage setbacks at Ultragenyx, Biohaven, and Outlook were a reminder of how quickly biotech valuations can reset when pivotal data miss or regulators stay unconvinced.
BIOTECH/PHARMA NEWS 🧬
🔹 A federal judge has temporarily blocked a proposed 340B rebate pilot, halting its planned January 1 rollout after finding the program was likely unlawful under the Administrative Procedure Act. In a December 29 preliminary injunction, a U.S. District Court judge in Maine ruled that the Health Resources and Services Administration failed to adequately justify shifting 10 Medicare price-negotiated drugs into a rebate-based 340B model, citing a “threadbare” administrative record and insufficient analysis of the financial and operational harm to hospitals. The lawsuit, brought by hospital groups including the American Hospital Association, argued the pilot would impose major upfront costs and administrative burden on 340B providers. While not a final ruling, the judge found a strong likelihood the challengers will prevail and that allowing the program to proceed would cause irreparable harm—preserving the status quo for now.
🔹 Vanda Pharmaceuticals (VNDA) jumped after the FDA approved Nereus (tradipitant), an oral neurokinin-1 receptor antagonist licensed from Eli Lilly, for the acute prevention of motion-induced vomiting—the first new pharmacologic option for motion sickness in more than 40 years. Approval was supported by multiple late-stage trials showing a statistically significant reduction in vomiting with a safety profile compatible with single-dose use, and followed the FDA’s recent reclassification of motion sickness as an acute condition, which lifted a long-standing regulatory overhang. While motion sickness is a modest standalone market, analysts see peak U.S. sales north of $100 million and view the decision as a regulatory de-risking event for tradipitant’s broader pipeline, including GLP-1–associated nausea and gastroparesis.
🔹 Gilead agreed to acquire RP-3467, a polymerase theta inhibitor from Repare Therapeutics, for up to $30M, adding another targeted DNA-repair asset to its oncology pipeline. The deal includes $25 million upfront plus a $5 million milestone tied to technology transfer and boosts Repare’s cash balance ahead of its pending take-private transaction with XenoTherapeutics, lifting expected shareholder proceeds to roughly $2.20 per share. RP-3467 is an early-stage drug designed to exploit synthetic lethality in tumors with BRCA mutations and is currently being tested in a Phase 1 study across multiple solid tumors, both alone and in combination with AstraZeneca and Merck’s PARP inhibitor olaparib. While modest in size, the transaction underscores continued pharma interest in precision oncology assets even as broader biotech dealmaking remains selective.
🔹 Sanofi has agreed to acquire Dynavax Technologies for $15.50 per share in cash, valuing the vaccines specialist at roughly $2.2B and marking one of the clearest large pharma vaccine deals of 2025. The acquisition brings Heplisav-B, Dynavax’s marketed adult hepatitis B vaccine, into Sanofi’s vaccines portfolio, alongside an early stage shingles program currently in phase 1/2 development. Strategically, the deal strengthens Sanofi’s push into adult immunization, adding a differentiated two dose hepatitis B vaccine with faster protection than legacy regimens, while giving Dynavax global scale it could not reach alone. Sanofi will fund the transaction entirely with cash and plans to close in the first quarter of 2026.
🔹 The FDA approved Novo Nordisk’s oral Wegovy (semaglutide), making it the first and only oral GLP-1 therapy approved for chronic weight management in adults. The decision was based on the Phase 3 OASIS 4 trial, where once-daily 25 mg tablets delivered ~17% average weight loss at 64 weeks under on-treatment analysis (~14% regardless of discontinuation), materially narrowing the efficacy gap with injectable GLP-1s while eliminating needles. Notably, the pill also carries an indication to reduce major adverse cardiovascular events in patients with established cardiovascular disease—an edge no competing oral obesity drug currently offers. With a $149/month self-pay entry price, the oral expansion reinforces Novo’s dominance as competition in obesity intensifies heading into 2026.
🔹 The FDA approved Agios Pharmaceuticals’ AQVESME (mitapivat) for the treatment of anemia in adults with alpha- or beta-thalassemia, marking the first and only FDA-approved therapy to address anemia in both transfusion-dependent and non-transfusion-dependent patients. The decision was backed by the phase 3 ENERGIZE and ENERGIZE-T trials, which showed meaningful improvements in hemoglobin, fatigue, and transfusion burden across a broad, real world population. Commercially, the approval expands mitapivat into a much larger rare disease market, though uptake will be shaped by the newly implemented REMS program tied to hepatocellular injury risk, with U.S. launch expected in late January 2026.
🔹 Biotech delivered some of the most extreme winners of 2025, even as the broader healthcare sector posted a relatively muted year. The S&P Health Care Index rose about 12% year-to-date by mid-December, but beneath that surface a handful of biotech stocks generated explosive, data-driven returns. The clear standout was Abivax, which surged roughly 1,500-1,600% after positive late stage data for its oral ulcerative colitis drug obefazimod, making it the top performing biotech stock of the year. That rally has since evolved into strategic optionality. In a Truist Securities investor survey, Abivax emerged as the most likely biotech acquisition target in 2026, cited by 44% of respondents, far ahead of any peer, with speculation centered on potential interest from Eli Lilly.
🔹 Cytokinetics won FDA approval for Myqorzo, a first-in-class “cardiac myosin inhibitor” for adults with symptomatic obstructive hypertrophic cardiomyopathy, a condition where an abnormally thick heart muscle blocks blood flow out of the heart and makes everyday activity feel like you’re running uphill. The decision was driven by the phase 3 SEQUOIA-HCM study, where patients treated for 24 weeks showed a meaningful improvement in exercise capacity versus placebo, including higher peak oxygen uptake (a standard measure of cardiopulmonary fitness). Commercially, this is a milestone moment: it’s Cytokinetics’ first approved drug, with U.S. launch expected in the second half of January 2026. The trade off to watch is the label’s boxed warning for risk of heart failure. Because the drug deliberately reduces heart contractility, patients need echocardiogram monitoring and access is gated through a Risk Evaluation and Mitigation Strategy program, which can slow early uptake but also clarifies who should (and shouldn’t) get it.
🔹 The White House announced nine new Most Favored Nation (MFN) drug pricing agreements with major pharmaceutical companies, including Amgen, BMS, Gilead, Merck, Novartis, and Sanofi. Under the deals, select medicines will be sold at steep discounts through a new TrumpRx direct-to-consumer platform launching in January and made available to all state Medicaid programs. In exchange, participating companies avoid pharmaceutical import tariffs for three years, commit to large U.S. manufacturing investments, and agree that future innovative drugs will also be subject to MFN pricing, marking one of the most aggressive federal interventions yet into U.S. drug pricing and launch economics.
🔹 BioMarin will acquire Amicus Therapeutics for $4.8B in cash ($14.50 per share), adding two marketed rare-disease drugs (Galafold for Fabry disease and Pombiliti + Opfolda for Pompe disease) which generated $599M in revenue over the past year. The deal, expected to close in Q2 2026, is projected to accelerate BioMarin’s revenue immediately and be earnings accretive within 12 months, with greater impact from 2027 onward. BioMarin will fund the acquisition with cash and debt, while a separate settlement resolves U.S. patent litigation around Galafold, supporting exclusivity through 2037.
🔹 U.S. vaccine policy is heading into uncertain territory as RFK Jr. pushes to scale back childhood vaccination recommendations, but faces clear legal limits on how fast and how far he can go. While the administration has already moved to stop using childhood and prenatal vaccination rates as a measure of health care quality in government programs, any attempt to overhaul the national vaccine schedule would require a formal, evidence-based process rather than a simple announcement. That reality likely explains why a late December plan to align U.S. vaccine recommendations with Denmark’s was abruptly shelved. For vaccine makers and public health officials alike, the message is clear: major changes are being discussed, but the legal system will strongly shape what actually happens in 2026.
CLINICAL TRIAL UPDATES 📊
🔹 Ultragenyx (RARE) shares fell sharply after its osteogenesis imperfecta program setrusumab (UX143) failed to meet the primary endpoint in both late-stage trials. Neither the Phase 2/3 ORBIT study (ages 5–25, placebo-controlled) nor the Phase 3 COSMIC study (ages 2–<7, bisphosphonate-controlled) demonstrated a statistically significant reduction in annualized clinical fracture rate, despite both trials showing strong, statistically significant improvements in bone mineral density. The company said it will conduct additional analyses to better understand the disconnect between bone density gains and fracture outcomes and announced plans to implement significant expense reductions. The readout represents a major setback for Ultragenyx’s rare bone disease portfolio and weighed heavily on its partner Mereo BioPharma, which holds EU and UK rights to the asset.
🔹 Biohaven shares fell after its phase 2 study of BHV-7000 in major depressive disorder failed to meet its primary endpoint, with the drug showing no statistically meaningful improvement versus placebo on the Montgomery-Åsberg Depression rating scale, a standard measure of depressive symptoms, over six weeks. While Biohaven pointed to signals favoring BHV-7000 in patients with more severe baseline depression, the company characterized these findings as exploratory and said it does not plan to pursue further psychiatric trials, opting instead to focus resources on immunology, obesity, epilepsy, and oncology. The result underscores the persistent difficulty of translating novel depression mechanisms into broad clinical efficacy and removes BHV-7000 from Biohaven’s central growth narrative.
🔹 Outlook Therapeutics shares collapsed after the FDA issued a Complete Response Letter for its resubmitted BLA for ONS-5010/LYTENAVA (bevacizumab-vikg) in wet AMD, reiterating that a single adequate, well-controlled efficacy study is insufficient for approval and again requesting additional confirmatory evidence. Crucially, the agency did not specify what form that confirmation should take, leaving the company in regulatory limbo. Outlook says it will pursue “all available pathways,” but with U.S. approval now pushed further out, the near-term narrative shifts to how costly and time-consuming the FDA’s missing requirement may be—and whether European commercialization can sustain the program in the interim
🔹 Altimmune reported positive 48 week phase 2b data from its IMPACT trial showing that pemvidutide, a dual glucagon and GLP-1 agonist, produced statistically significant improvements in non-invasive markers of liver fibrosis and inflammation in patients with metabolic dysfunction-associated steatohepatitis, while maintaining a favorable safety profile. Both dose arms showed continued antifibrotic improvement from 24 to 48 weeks, meaningful reductions in liver stiffness and fibrosis scores versus placebo, and modest but sustained weight loss without plateauing at the higher dose, with fewer treatment discontinuations than placebo and no serious treatment-related adverse events. Importantly, Altimmune said it has aligned with the FDA on the design of a registrational phase 3 trial in patients with moderate to advanced fibrosis, which it plans to initiate in 2026, positioning pemvidutide as a differentiated late-stage contender in the crowded MASH landscape.
Have a great rest of your week and thanks for reading Biotech Blueprint!
