NEJM retracts a registrational trial for the first time, plus Trump's childhood vaccine executive order – This Week in Biotech #113
Amgen's Tavneos pivotal study pulled after 9 patient endpoints were changed post-unblinding, Jazz buys Actio Biosciences, and Replimune's approval label comes in lower than expected (8/7-8/13).
Welcome back to This Week in Biotech by Biotech Blueprint, edition 113, covering biotech and pharma news from August 7 to 13, 2026.
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VIDEO SUMMARY
THIS WEEK’S KEY TAKEAWAYS 🔑
Last week, I reported on Replimune’s Tudriqev getting accelerated approval by the FDA, four days past the deadline and eight days after an advisory committee voted 10-3 in its favor. Tudriqev is given to patients with advanced melanoma along with nivolumab. This week the label was made public and it lists efficacy at a 24.2% objective response rate in 91 patients. However, Replimune had been quoting 33.6% for two years, and the 32.9 month median overall survival that convinced the FDA advisory panel isn’t on the label at all. The number discrepancy comes from the FDA narrowing the efficacy population in the label to only include patients who had at least one tumor lesion that was never injected. That is because Tudriqev is injected directly into tumors, and you can’t tell if the drug is actually working throughout the body unless you can see uninjected tumors shrink too. This was a concern initially raised by the FDA in July briefing documents. So the FDA approved the drug but built its scientific criticism into the label by restricting which patients count for the efficacy claim.
Two more stories that remind us that regulatory approval isn’t the finish line for a drug. Prader-Willi syndrome patient organizations flagged 7 deaths and more than 100 serious adverse events for Neurocrine’s Vykat XR. Vykat XR is a drug that helps with hyperphagia (extreme, unsatisfied hunger and constant thoughts about food) in Prader-Willi patients and was purchased for $2.9 billion from Soleno Therapeutics in April 2026. The groups called for tighter patient selection and monitoring. Also, EU regulators published the assessment behind their June recommendation to pull Amgen’s Tavneos. Turns out the pivotal study “breached good clinical practice guidelines,” and the New England Journal of Medicine retracted the paper on June 29. This is the journal’s first ever retraction of a registrational study that supports an approval. So I dug a bit into the specific reason and it seems that the drug application relied on manipulated data for 9 patients that were changed after the trial was unblinded. The FDA has its own withdrawal proposal pending, which Amgen is contesting.
Lastly, on the policy side, two important things happened this week. Donald Trump signed a new executive order to yet again change the childhood vaccine schedule, however it will likely get tied up in court and won’t survive legal challenges. Merck is the only manufacturer of the only MMR vaccine in the US and has said splitting the shot into three is impractical and would almost certainly require new clinical studies. This executive order does not follow any new science or discoveries.
Secondly, the FDA is proposing that companies will get discounts if they run their phase 1 trials in the US or keep their manufacturing here. Once fees like this get written into law, they stick around, unlike executive orders or tariffs that can be reversed by a new administration. If this proposal passes, it might strengthen US biotech capacity in the long run, but it could also raise drug development costs and slow down how quickly new medicines reach patients because some of the fastest and cheapest infrastructure right now is outside the US.
BIOTECH/PHARMA NEWS 🧬
🔹 Replimmune’s Tudriqev is a genetically modified herpes simplex type 1 virus injected directly into tumors, including deep and visceral lesions under imaging guidance, and given with nivolumab in unresectable advanced cutaneous melanoma that progressed on a regimen blocking PD-1. It’s the first accelerated approval built on a single arm study that FDA reviewers had twice rejected (July 2025 and April 2026). In the 91 patient efficacy-evaluable group, 80% of whom had stage 4 disease and 54% of whom lacked the PD-L1 protein on their tumors, the response rate was 24.2% with a median duration of 14.1 months. Serious adverse reactions occurred in 35% of the 140 patients treated and 2.9% discontinued. List price is about $450k per course, with launch expected within roughly two months. The confirmatory trial, IGNYTE-3, is about 1/3 enrolled and reads out overall survival in 2030, which means four years of commercial revenue will come before the data that justifies it. Analysts model $600M-$1B in peak US sales if it lands. A three year regulatory fight ended in a smaller win than the one Replimune was arguing for.
🔹 The Gold Standard Childhood Vaccine Recommendations executive order, signed August 10, cuts universally recommended childhood shots from 17 to 11, directs that the MMR vaccine be split into three separate shots once domestically available, requires that childhood vaccines be given at separate appointments, moves COVID-19 and influenza vaccination to shared clinical decision making for people who are not high risk, gives the Department of Health and Human Services 90 days to produce an adjusted schedule, and directs the Attorney General to challenge state religious and medical exemption laws. No new evidence accompanied it. Merck makes the only MMR vaccine sold in the US., and the company have said splitting it is impractical. Standalone mumps and rubella products would need new manufacturing lines and new clinical work, for a market the order itself would shrink. Senator Bill Cassidy, who chairs the Senate Health Committee, said Trump lacks the expertise to make the changes. The WHO called the order misguided, and the UK regulator reaffirmed its own schedule on Aug. 12. The direct commercial hit to drugmakers is modest. What is more concerning is that this sets a precedent for setting the childhood immunization schedule by presidential order instead of through the standard advisory committee process.
🔹 The FDA published commitment letters for the next reauthorizations of the Prescription Drug User Fee Act (PDUFA VIII) and the Generic Drug User Fee Amendments (GDUFA IV), covering 2028 through 2032. Included are what the agency calls America First provisions, which are cheaper application fees for sponsors running Phase 1 trials in the US, small business fee waivers only available to US applicants, onshoring incentives on the generic drug side, faster reviews for certain applications, and some new meeting types. Past user fee deals were about paying for faster reviews. This one uses fees to influence where the science actually gets done. The letters still need agency ratification, clearance from HHS and the White House budget office, public comment, and Congress, so terms can change. But once fees like this are placed into statute, they tend to outlast the administration that wrote them, unlike an executive order or a tariff. This lands on the same drug companies that spent 2026 fighting over Section 232 exemptions and China licensing deals. If you're picking a site for your next Phase 1 trial, your finance team is going to have to weigh this in.
🔹 Jazz agreed on August 10 to buy Actio Biosciences for $820M in cash plus up to $500M in regulatory and sales milestones, closing by Q4. The asset is ABS-1230, a first in class oral inhibitor of the KCNT1 ion channel, for a genetic developmental and epileptic encephalopathy that affects roughly 2500 people in the US and has no approved treatment. Jazz also takes a minority stake in a spun out rare neurology company holding ABS-0871. That $820M works out to about $330k per US patient with no clinical data yet in hand, on top of an epilepsy franchise Jazz already runs through Epidiolex. This is Jazz’s second deal of the year after the AbCellera T cell engager alliance in June. Mid cap acquirers keep going after rare disease drugs they can sell through their existing sales teams, rather than platforms they would have to build up separately.
🔹 Three Prader-Willi syndrome organizations issued a joint statement on August 11 flagging 7 deaths and more than 100 serious adverse events reported to the FDA’s adverse event system through July 31, mostly involving edema, respiratory, and cardiac problems. The groups were careful to note the reports don’t establish causation, and spontaneous reporting in a population with high baseline respiratory and cardiac mortality is hard to interpret. But they still called for tighter patient selection and monitoring. Neurocrine stands by the drug. Vykat XR is the first therapy approved to reduce hyperphagia in Prader-Willi syndrome, and Neurocrine paid $2.9B to Soleno in April to get it. Similar story to Celcuity and MapLight last month. FDA approval is one thing, but holding up in the real world is another test.
🔹 The bankrupt gene-editing pioneer Sangamo sold its assets for up to about $264M combined. PTC Therapeutics won ST-920 (isaralgagene civaparvovec), a single infusion gene therapy for Fabry disease, for $111M upfront plus $80M on accelerated approval and $20M on full approval. Lilly took the zinc-finger platform programs for $50M in cash. What’s interesting is how much the final price jumped from the opening bid. Astellas had originally offered $25M upfront and $25M in milestones for the Fabry asset. The competitive auction pushed the final price more than fourfold in a matter of weeks. PTC finishes the rolling submission in Q4 and could launch in 2027 into a Fabry market above $2B. So yes, gene therapy companies are going bankrupt, but their late stage assets are still generating real prices at auction.
🔹 A single 100 mcg oral dose of LSD from Definium Therapeutics cut Hamilton Anxiety Rating Scale scores by 11.6 points from baseline at week 12 versus 6.2 on placebo, a placebo-adjusted difference of 5.4 points at p<0.0001. Separation appeared by day 2 and held. There were no new safety signals and no suicidality signal. 92% of participants cleared the end of session checklist by eight hours, averaging 6.4 hours. The company, which was MindMed until January, now has positive Phase 3 data in both major depression (June) and anxiety off one molecule, and holds breakthrough therapy designation here. Analysts model $1.5-2B in depression alone and up to $5B across three indications. The stock rose 15%. The catch is the same catch as every psychedelic trial. A 5.4 point separation is large for psychiatry, but everyone who received an active dose of LSD knew they took it. The next pivotal trial, Panorama, adds a 50 mcg arm specifically to test whether functional unblinding is doing the work. The September readout from that trial is what will really determine the label.
🔹 Zipalertinib from Taiho Oncology and Cullinan Therapeutics plus chemotherapy beat chemotherapy plus placebo on progression-free survival by enough to trigger early unblinding at a pre-planned interim look in first line EGFR exon 20 insertion lung cancer. The trial was powered to detect a 40% reduction in the risk of progression or death, so the effect cleared that bar. The hazard ratio and enrollment were not disclosed and the full data go to a future meeting. The context makes this story interesting. J&J’s Rybrevant, the incumbent in this genetically defined subset, is an infusion and did over $700M in 2025. AstraZeneca and Dizal’s Zegfrovy, which is oral, cut progression risk 35% as monotherapy. Zipalertinib is also oral, already under FDA review for second line use with a decision due Feb. 27, 2027, and now has front line data to support a label expansion. ArriVent’s firmonertinib reads out later this year. So four drugs are chasing a mutation that appears in only about 2% of non-small cell lung cancer, and the fight is about dosing format more than the underlying mechanism.
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Front cover image source: Associated press