Welcome back to This Week in Biotech by Biotech Blueprint, edition 122, covering biotech and pharma news from October 9th to 15th, 2026.
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VIDEO SUMMARY
THIS WEEK’S KEY TAKEAWAYS 🔑
Last month I reported four Phase 3 wins that arrived without effect sizes (This Week in Biotech #119), and said the numbers would show up eventually at medical meetings. Merck’s remigromig was the first, and it explains why they were held back. In 984 patients with diabetic macular edema, remigromig gained 9.1 and 8.7 letters of vision against 11.8 for Lucentis, a drug that has been on the market since 2006. Rates of the worst form of diabetic eye disease were about seven times higher, and treatment discontinuations about five times higher. So the trial cleared its bar (showed noninferiority), but it did it with worse safety than a drug that has been available for twenty years. Moderna’s melanoma vaccine is next up. The August topline had no hazard ratio either, and the stock gained 177% in a day on it. Full data at ESMO next Friday (Oct 24).
Bristol Myers did the same thing this week, and in their case the missing numbers matter more. Admilparant hit its Phase 3 endpoint in idiopathic pulmonary fibrosis at 60 mg and missed at 120 mg, with no effect size disclosed for either. A lower dose working better than a higher one is the result that raises the most questions, and without the numbers nobody can tell whether the 60 mg result is real.
On Wednesday, the FDA launched a Rare Disease Framework Initiative meant to make approvals “more predicatble,” and then expanded ImmunityBio’s Anktiva label almost three months early. It did that without a dedicated efficacy analysis in the new population, a year after refusing even to review the filing. The reasoning was that the two tumor types are similar enough that the original evidence carries over, which is defensible but it still means approving a drug for patients it wasn't tested in. Public meeting on the framework is October 28, with recommendations reportedly targeted for February.
OrbiMed closed $3B in new funds, OMass raised $310M privately, and Verastem raised $250M on the same morning as its pancreatic cancer data. Iambic got its AI designed pipeline public at $16 a share, the year’s 25th biotech IPO. Meanwhile two AI discovery partnerships came with small ish upfront payments. Zealand put $30M down on Generate Biomedicines and AstraZeneca $40M on insitro.
There were also a few late stage failures this week. BioNTech’s mRNA immunotherapy BNT113 failed to delay progression in head and neck cancer at an interim look and shares fell about 6%. Vanda’s social anxiety drug missed its Phase 3, 65.6 versus 66.0 on the distress scale, p=0.89. Syndax’s Niktimvo failed in idiopathic pulmonary fibrosis.
BIOTECH/PHARMA NEWS 🧬
🔹 Full one year data for Merck’s remigromig came out at the American Academy of Ophthalmology meeting on October 10. Diabetic macular edema is swelling in the retina that causes vision loss in people with diabetes, and the standard treatments all block a protein called VEGF that drives abnormal blood vessel growth. Remigromig works through a different pathway entirely, which is why Merck paid $1.3B upfront for Eyebiotech in 2024 to get it. The trial randomized 984 patients to remigromig 0.5 mg, 0.8 mg, or Lucentis 0.5 mg, all dosed monthly. Vision gains were +9.1 letters (95% CI 8.1-10.1) and +8.7 letters (7.7-9.8) versus +11.8 (10.7-12.8) for Lucentis. Both doses were noninferior, which is what the trial was designed to show, and no secondary endpoint showed superiority. The safety signal is the bigger problem. Proliferative diabetic retinopathy, the advanced stage of diabetic eye disease, occurred in 6.7% and 6.1% of remigromig patients versus 0.9% on Lucentis. Adverse events led 4.9% and 4.5% to stop treatment versus 0.9%. Merck’s hypothesis is that the drug repairs the blood-retinal barrier rather than suppressing vessel growth, so the higher retinopathy rate may reflect the natural course of the disease rather than drug harm. BMO now sees “limited competitive potential.” The BAROLO trial and year two flexible dosing are what remain. The real case for a new mechanism rests on the 30 to 40% of patients who respond poorly to VEGF blockers, and this trial didn’t test them.
🔹 Bristol Myers Squibb’s admilparant met its Phase 3 primary endpoint in idiopathic pulmonary fibrosis at the 60 mg dose and missed significance at 120 mg. About 1255 adults were randomized 1:1:1 to 60 mg twice daily, 120 mg twice daily, or placebo for at least 52 weeks, with the endpoint being absolute change in forced vital capacity, a measure of how much air a patient can exhale. No effect sizes, confidence intervals or p-values were disclosed for either dose. Discontinuations for side effects were low and similar to placebo. The basic expectation in a dose ranging trial is that more drug means more effect, so when the lower dose works and the higher one doesn’t, the obvious question is whether the 60 mg result is real or came out of the noise. There are several possible explanations, like more side effects at the higher dose causing patients to drop out, but you’d need the actual numbers to tell. Phase 2 had shown a 62% slowing of decline at 60 mg over 26 weeks, which set a high bar. BMS fell 2.6%. Contineum, which has a lower dose drug against the same receptor in Phase 2, fell roughly 25 to 36% the same day. The liver injury risk flagged in an earlier protocol amendment isn’t addressed in the topline at all. ALOFT-PPF, in other progressive fibrotic lung disease, reads out in early 2027.
🔹 The FDA launched the Rare Disease Framework Initiative on October 14. The idea is to write down standards for rare disease drug development that account for how small these patient populations actually are, covering the whole path from the first meeting with the agency through whatever a company has to keep studying after approval. There’s a public meeting at White Oak on October 28 and recommendations are reportedly targeted for February, though the agency set no timeline itself. Leerink’s Joseph Schwartz said that the FDA “is not lowering the bar” but may be making the path “less of a guessing game.” The same day showed what that might look like. The FDA expanded Anktiva from ImmunityBio to cover papillary-only bladder cancer in patients who’ve stopped responding to BCG, the standard first line treatment. The decision came nearly three months early. The agency didn’t ask for a separate randomized trial. It extended the evidence from the 2024 approval in a different tumor type, reasoning that the two co-occur and are hard to tell apart. In May 2025 the agency refused to even file the application. That’s a defensible scientific argument, and it also means approving a drug for patients it wasn’t tested in. I think we can expect other companies to point to this the next time they want an approval without running the trial.
🔹 Two weeks after a Delaware court found that Yutrepia infringed a United Therapeutics patent, which sent Liquidia down almost 50% and United up about 13% in a day, the companies settled. That covers three patent suits, the antitrust claims and a North Carolina trade-secret case. Liquidia pays a net $380.7M in installments through April 2027 and keeps selling Yutrepia, its inhaled treprostinil, for pulmonary arterial hypertension. But Liquidia has to ask the FDA to remove pulmonary hypertension tied to interstitial lung disease from the label and stop promoting it there, which is the growth market where United’s Tyvaso holds the lead. Liquidia shares rose about 42% mainly because the math works for them: Yutrepia did a preliminary $222.7M in Q3 net sales and Liquidia holds $396.1M in cash, so the company is paying a little over one quarter of revenue to remove the risk of an injunction shutting it down entirely. United gives up a litigation option and keeps its most valuable market. Neither company won outright, and both can now plan their businesses.
🔹 Verastem’s oral KRAS G12D inhibitor VS-7375, which binds both the active and inactive forms of the protein, produced response rates of 36 to 50% in metastatic pancreatic cancer depending on how many prior treatments patients had received, 33% in colorectal and 43% in lung cancer. In 22 pancreatic patients at 900 mg, 6 month progression-free survival was 67%. The lung response rate came from 23 patients at 600 mg. These are small single arm cohorts, so the confidence intervals are wide. The competitive frame is Revolution Medicines, whose Rasonque was approved in August and nearly doubled survival in G12-mutant pancreatic cancer, though FDA documents suggest its effect is more modest in G12D specifically, and RevMed has a G12D-selective drug coming. RBC called Verastem’s profile “competitive.” Verastem has FDA alignment for a first line pancreatic Phase 3 starting by year-end, with colorectal and lung to follow in the first half of 2027. Shares rose about 18 to 20%, and the company raised $250M the same morning alongside announcing a CEO transition.
🔹 Arrowhead gave subcutaneous injections of an RNA interference drug that silences the tau gene to about 64 healthy volunteers. At day 105, tau fell 40% at the lowest dose tested, 69% at the middle dose, and an average of 73% at the highest, with individual reductions up to 85%. Side effects were mild: injection-site reactions, bruising, respiratory infections, headaches. The novelty here is the delivery rather than the target. This is a shot under the skin that reaches neurons behind the blood-brain barrier, where competitors have relied on spinal injections or engineered antibody shuttles to get there. Stifel called it “compelling” while noting tau is not yet a validated Alzheimer’s target and Biogen’s spinal injection tau drug has struggled. These are healthy volunteers, so this is a delivery result, not an efficacy one. Still early and still small, but it keeps alive the idea that the brain can be reached with an ordinary injection. Mild Alzheimer’s patients enroll next, with frontotemporal dementia and progressive supranuclear palsy trials planned.
🔹 OrbiMed raised $3B across two funds, including over $1B for Asia, a week after Forbion’s $2.6B close. Oxford-based OMass raised $310M, among the largest UK private biotech rounds in years, to compete in congenital adrenal hyperplasia against Neurocrine and Vertex’s Crinetics. Ultragenyx sold its second priority review voucher in a month, for about $203M after $210M for the first. On the deal side, Zealand Pharma agreed to pay Generate Biomedicines $30M upfront plus up to $35M in early milestones for AI-designed multispecific proteins in obesity, with nearly $1.2B in later milestones. AstraZeneca put $40M upfront into an insitro discovery deal. Gilead paid Vir $135M upfront and near-term with up to $1.74B more for three masked T cell engagers in solid tumors, a deal built on protein engineering rather than AI. And Iambic priced its IPO at $16 to raise $150M and rose about 13% on day one, the 25th biotech to go public this year. Worth noting Iambic had a molecule already in the clinic, IAM1363, an oral inhibitor of the HER2 growth receptor with Phase 1 activity in heavily pretreated patients, which most AI discovery companies going public don’t.
WATCHLIST 👀
Merck/Moderna intismeran: full INTerpath-001 Phase 3 melanoma data (n=1137) at the ESMO Presidential Symposium on Oct 24. The August topline gave no hazard ratio, so this is the second of September’s no number wins to face a full dataset. Remigromig went first and didn’t survive the check.
GSK/Ionis bepirovirsen: FDA decision in hepatitis B on Oct 26. A functional cure delivered by an antisense drug would be the first of its kind.
FDA Rare Disease Framework: public meeting Oct 28 at White Oak, with recommendations reportedly targeted for February. The Anktiva decision this week is the preview of what the standard might look like in practice.
Capricor deramiocel: FDA decision Nov 22 in Duchenne, on the refiled upper limb indication after July’s 9-3 negative advisory vote.
Have a great rest of your week and thanks for reading Biotech Blueprint!
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Front cover image source: mediamice . com (Article: Merck’s Remigromig Meets Primary Endpoint in Phase IIb/III DME Trial)