Welcome back to This Week in Biotech by Biotech Blueprint, edition 123, covering biotech and pharma news from October 16th to 22nd, 2026.
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VIDEO SUMMARY
THIS WEEK’S KEY TAKEAWAYS 🔑
The Moderna data leaked today, and the stock fell more than 25%. A publication broke the ESMO embargo two days before Saturday’s presentation, results started circulating on social media, and ESMO had to release them early. The number everyone had been waiting for: intismeran plus Keytruda cut the risk of recurrence or death by 42%, hazard ratio 0.58.
That number is fine. BMO called the melanoma data “clinically meaningful and likely approvable.” What fell apart was everything investors had attached to it. In August, Moderna announced this trial hit its endpoints with no hazard ratio, no p-value and no confidence interval, and gained 177% in a day, the largest single day move by any S&P 500 company this century. Eight days after that they raised $2 billion in convertible notes against the new price. With no number in the room, people priced a platform that works across cancers. The actual number tells you about melanoma and nothing about kidney, lung or bladder, where the other trials are still running. BMO’s warning was explicit: don’t extrapolate. Overall survival data isn’t available yet. Moderna is still up 525% on the year.
Two rare disease stocks jumped this week. Disc Medicine’s bitopertin treats a genetic disease that makes sunlight physically painful. The FDA rejected it in February because the company showed the drug lowered the toxic molecule causing the problem, but not that patients actually spent more time outside. On Monday a 183-patient Phase 3 showed both, and the stock rose more than 35%. On Thursday Biohaven said the FDA had accepted a resubmission for Vyglxia, a spinocerebellar ataxia drug it rejected last November, and will convene an advisory panel. Shares jumped about 25%. Neither company has an approval but investors are buying a change in who reviews the file and how, a week after the agency launched its Rare Disease Framework Initiative. If that holds, a lot of rejected rare disease drugs just got more valuable.
GSK got two FDA approvals in 48 hours, both ahead of schedule. Hibsago is the first drug that can get hepatitis B patients off lifelong antivirals, and about one in five reach that. It came six days early with a boxed warning attached. A day before that, Jemperli was approved for a type of rectal cancer where patients can now skip chemotherapy, radiation and surgery entirely.
Roche signed a deal worth up to $15B with Hansoh for preclinical antibody-drug conjugates, its sixth China deal since late August. In the same week, Treasury prepared a proposed rule that would screen US licensing deals with Chinese biotechs.
Three interesting readouts. Merck KGaA paid a 130% premium for Silence Therapeutics because its polycythemia vera drug is dosed every 12 weeks against a weekly competitor. Roivant’s brepocitinib cut treatment failure by about three quarters in two uveitis trials. AbbVie’s lutikizumab won in hidradenitis suppurativa by 8 points. All three disclosed numbers on day one.
BIOTECH/PHARMA NEWS 🧬
🔹 Merck and Moderna's Phase 3 melanoma data was scheduled for the ESMO Presidential Symposium on Saturday. It leaked on Thursday instead, after a publication broke the embargo and the results started circulating on social media, forcing ESMO to release them early. Intismeran autogene plus Keytruda cut the risk of recurrence or death by 42%, hazard ratio 0.58, in patients with resected stage IIB-IV melanoma. Moderna fell more than 25%. Merck fell too. The data itself is good. BMO called it "clinically meaningful and likely approvable." What collapsed was everything investors had built on top of it. The August topline announced that the trial met both endpoints without releasing a hazard ratio, a p-value or a confidence interval, and Moderna gained 177% that day, the largest single session move by any S&P 500 company this century. Eight days later the company raised $2B in convertible notes against the new price. With no number available, the market priced a platform that works across tumor types. The number that arrived says something specific about melanoma and nothing about kidney, lung or bladder cancer, where the other trials are still enrolling. BMO's note was explicit about not extrapolating. Overall survival data isn't available yet. Moderna is still up 525% on the year.
🔹 The FDA approved GSK and Ionis’s Hibsago (bepirovirsen) on Oct. 20, six days ahead of its goal date, as the first antisense therapy for chronic hepatitis B. It had already been approved in Japan in August. It is for adults without cirrhosis, on nucleoside analogue antivirals for at least six months, with suppressed viral DNA and surface antigen at or below 3,000 IU/mL. In the pooled B-Well 1 and 2 trials, 19% of patients reached functional cure versus 0% on placebo, rising to 26% when surface antigen started at or below 1,000. Functional cure means virus and antigen undetectable for 24 weeks after all treatment stops, so patients can come off antivirals they’d otherwise take for life. The catch is monitoring. The drug carries a boxed warning, the FDA’s most serious category, covering liver enzyme flares and low platelets, with weekly or biweekly lab tests. Jefferies says that could slow uptake outside specialist centers, while still modelling about $3B at peak, a meaningful piece of GSK’s £40B 2031 revenue goal. The FDA also authorized a Roche test to select patients. Price is undisclosed pending payer talks. The number that will matter is how many eligible patients hepatologists actually screen in.
🔹 On Oct. 19 the FDA granted accelerated approval to GSK’s Jemperli (dostarlimab) as a treatment for stage 2 or 3 rectal cancer in patients whose tumors have mismatch repair deficiency or high microsatellite instability, a genetic signature that makes tumors unusually responsive to immunotherapy. This is the population from the 2022 Memorial Sloan Kettering study where every single patient’s tumor disappeared. In the 154-patient AZUR-1 trial, 68% had a sustained clinical complete response at 12 months by investigator review, but only 47% by independent central review, which was the primary objective. That 21-point gap between what treating physicians saw and what blinded reviewers saw is the soft spot in this dataset. There have been no recurrences among responders after a median of more than two years. Treatment is nine infusions over six months. Serious adverse reactions hit 20% and discontinuations 3%. Accelerated approval means a confirmatory trial still has to show the responses hold. But the goal it sets for this disease is organ preservation instead of surgery, which for rectal cancer means avoiding a permanent colostomy.
🔹 Roche agreed on Oct. 19 to pay $400M upfront and up to $15B in total for two preclinical antibody-drug conjugates from Hansoh, plus more against targets Roche gets to pick. That’s Roche’s sixth China deal since late August and its 11th since January 2025. The urgency shows up in the Q3 numbers. Sales grew 7% to CHF16.1B, which Jefferies called “cosmetically ahead,” and Xolair came in at CHF848M against a CHF972M forecast as Celltrion’s biosimilar launched in the US. Ocrevus, Tecentriq and Hemlibra all face copies by the early 2030s. Washington is moving the other way. Treasury is poised to propose a rule under the outbound investment law known as the COINS Act that would add scrutiny to US licensing deals with Chinese biotechs. Earlier reporting suggested most deals would survive, with pathogen and military use technology excluded. China ran 81 licensing deals worth up to $110B in the first half of this year. A screen may slow that, but likely not stop it.
🔹 Merck KGaA will buy Silence Therapeutics for $20 a share in cash, about $1.3B, a 130% premium to the October 16 close. The asset is divesiran, a short interfering RNA that silences a gene called TMPRSS6 to raise hepcidin, the hormone that limits how much iron reaches the bone marrow. In polycythemia vera, where the body makes too many red blood cells, restricting iron slows that production. The Phase 2 numbers explain the premium: an 88% responder rate versus 19% on placebo, and patients needed an average of 0.2 blood draws over 36 weeks against 2.1 on placebo. Takeda and Protagonist’s Mimrylo, approved in August, works on the same hormone but is a weekly self-administered injection. Divesiran is dosed every 12 weeks. That’s the whole commercial argument. Merck KGaA is paying for a Phase 3 it still has to fund, and Silence shareholders were facing dilution or a sale, so they took the sale. Close is expected in Q1 of 2027.
🔹 Biohaven said on October 22 that the FDA accepted its resubmission for Vyglxia (troriluzole) in spinocerebellar ataxia and will hold an advisory committee, with a decision expected in Q1 of 2027. The agency rejected the drug last November, finding the key study unconvincing partly on design grounds, even though the data pointed to as much as 70% slower progression. Biohaven also withdrew its European application after evaluators questioned the evidence. The resubmission adds “new positive supportive analyses” but no new trial, which is the important detail. Disc Medicine ran the Phase 3 the FDA asked for. Biohaven is testing whether the same evidence reads differently under new leadership. TD Cowen said the panel “substantially increases the probability of approval.” RBC said it depends on how far the flexibility goes. The stock rose about 25%. An advisory panel cuts both ways, though. An outside committee can say no in public, and this FDA has overruled panels in both directions this year.
🔹 Disc Medicine’s bitopertin hit both primary endpoints in a Phase 3 trial in erythropoietic protoporphyria, a genetic disease where a toxic molecule builds up in red blood cells and causes burning pain when patients are exposed to sunlight. The trial randomized 183 patients aged 12 and up to the oral drug or placebo for six months. Protoporphyrin IX, the molecule causing the problem, fell 41.4% versus placebo (p<0.0001), and pain-free time in sunlight rose 12.2 hours a month at month six (p=0.001). That combination is exactly what the FDA said was missing when it rejected the accelerated filing in February. The agency wanted the biomarker and the actual patient benefit shown in the same trial. Dizziness hit 50% of patients, mostly mild and transient with nothing above grade 3, though that deserves attention in a pill people would take daily for life. Bitopertin has an unusual history: it failed in schizophrenia under Roche before Disc picked it up and moved it into blood disorders. Disc plans to resubmit by year end and raised $450M at $75 a share off the news. Jefferies sizes the market at $1B to $2B, and the only approved alternative, Scenesse, is an implant.
🔹 Two placebo-controlled Phase 3 trials of Roivant’s brepocitinib, an oral JAK1 and TYK2 inhibitor, both hit hard in non-infectious uveitis, an inflammatory eye disease that can cause blindness. Hazard ratio for treatment failure from week 6 to 48 was 0.27 in one trial (n=166) and 0.22 in the other (n=204), both p<0.0001. Cumulative failure at week 48 was about a third on drug versus roughly 78% on placebo. Pooled, 62% reached steroid-free disease control at 48 weeks versus 20%. Every secondary endpoint hit. Humira is currently the only approved non-steroid systemic option, so a pill competing against an injection given in an eye clinic is an easy pitch. The trials didn’t test Humira head to head, so any “better than Humira” claim is cross-trial inference. Stifel called brepocitinib “best-in-disease” and Cantor puts uveitis at about $5B peak. The drug was already approved as Lisraya in dermatomyositis in August. Filing for uveitis is planned for the first half of 2027. Safety detail wasn’t in the topline, and for a JAK inhibitor that’s the next thing to read.
🔹 AbbVie’s lutikizumab, an antibody that blocks both IL-1α and IL-1β, met its primary endpoint in 1,400 patients with hidradenitis suppurativa, a painful skin disease that causes recurring abscesses and tunnels under the skin. At week 16, 31.4% hit the main response measure versus 23.4% on placebo (p≤0.001), with eight ranked secondary endpoints also hitting, including meaningful pain reduction at week 8. No new safety concerns. An 8-point gap is real but modest, and the 23.4% placebo rate is high, which is common in this disease. UCB’s Bimzelx and Novartis’s Cosentyx already compete here, and AbbVie’s own Humira still anchors the category. The case for lutikizumab is a new mechanism for patients who stop responding to IL-17 drugs, which IL-1 biology supports in theory but which needs subgroup data from the full 36-week readout to confirm. AbbVie plans to file next year. Shares barely moved, down 0.6%. Investors heard a positive trial, not a new franchise.
WATCHLIST 👀
Merck/Moderna intismeran: full INTerpath-001 Phase 3 melanoma data (n=1,137) at the ESMO Presidential Symposium on October 24. The August topline had no hazard ratio and moved the stock 177% in a day, so this is the big one.
Treasury China biotech rule: proposed rule under the COINS Act expected as early as Oct. 23. Watch the scope on licensing, equity and NewCo structures.
FDA Rare Disease Framework: public meeting Oct 28 at White Oak. Disc and Biohaven are the live test cases for how far the flexibility actually goes.
Capricor deramiocel: FDA decision Nov 22 in Duchenne.
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