Welcome back to This Week in Biotech by Biotech Blueprint, edition 115, covering biotech and pharma news from August 21 to 27, 2026.
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Yesterday, I published a new podcast episode in collaboration with The Biotech Capital Compass on the Moderna/Merck Phase 3 individualized neoantigen therapy. We get into why melanoma was first cancer picked, why the kidney cancer trial will be the real test, and the machine learning algorithm that selects the neoantigens, which may turn out to be the most interesting regulatory question of the whole thing.
VIDEO SUMMARY
Today’s video has a surprise co-host who invited himself to do the intro :) I didn’t notice until the edits.
THIS WEEK’S KEY TAKEAWAYS 🔑
Another busy week in biotech and pharma. The FDA approved Rasonque (daraxonrasib) on August 26, ONLY 35 days after accepting the application on July 22. The application went through the Commissioner’s National Priority Voucher pilot, which aims to go from application to approval in just 1-2 months, and Revolution Medicines is now selling the first broadly RAS-targeted medicine in metastatic pancreatic cancer at $39,800 per 30 day supply.
In contrast, two days before the approval, the FDA told Capricor its Duchenne cell therapy needed three more months, pushing decision for deramiocel from August 22 to November 22 after an amendment that reframed the indication around upper limb function instead of cardiomyopathy. Capricor’s stock rose 29% on the delay, because the alternative that most analysts expected was rejection. The FDA is reviewing some drugs in five weeks and pushing others back by months. The market is now treating that difference as information to trade on.
The second theme this week is that pricing pressure might be moving down to smaller companies. The White House is expected to announce Most favored nation agreements with several mid-size biotechs as early as Monday. This will likely extend the same structure 17 large pharma companies have already signed, including discounts to state Medicaid programs benchmarked to foreign prices, in exchange for a three year exemption from tariffs.
Companies are increasingly disclosing less information. Lilly has pulled Phase 1 programs from its published pipeline and stopped disclosing most deal values. Moderna’s personalized cancer vaccine selects 34 neoantigens per patient out of thousands of tumor mutations, and the algorithm doing the selecting is proprietary, as is the hazard ratio from the Phase 3 that moved the stock 177% on Aug 19. On August 27 Moderna announced a $2 billion private placement of convertible senior notes due 2032, zero coupon, with capped calls struck at least 150% above the reference price. So a readout that included no effect size at all became two billion dollars of cash at a valuation nobody outside the company can independently check.
Lastly, Genentech paid $190 million upfront in a deal worth up to $2.3 billion for a Korean obesity candidate with a non-incretin mechanism, and Akeso’s ivonescimab beat AstraZeneca’s Imfinzi in a China-only Phase 3.
BIOTECH/PHARMA NEWS 🧬
🔹 The FDA approved Rasonque (daraxonrasib) on Aug 26 for adults with metastatic pancreatic adenocarcinoma who have had at least one prior systemic therapy or who can’t tolerate multiagent chemotherapy. RASolute 302 randomized 500 patients 1:1 to daraxonrasib 300 mg once daily (n=248) or chemotherapy (n=252). Median overall survival was 13.2 months versus 6.7 months, hazard ratio 0.40 (95% CI 0.30-0.53, p<0.0001). Median progression-free survival was 7.2 versus 3.6 months, response rate 31.6% versus 11.2%. The RAS G12-mutant subgroup and the overall population produced nearly identical curves, which is the mechanistic point. This drug inhibits the active, GTP-bound form of RAS broadly, rather than targeting one G12C allele the way earlier KRAS drugs did. That’s why those earlier drugs never became large franchises and why RBC Capital Markets models $11.5B in peak sales here. The label warns on dermatologic and soft tissue toxicity, stomatitis, diarrhea, gastrointestinal perforation and interstitial lung disease, with prophylaxis recommended from day one. More than 2,000 patients had already received the drug through expanded access, so the launch starts with a prescriber base already in place. Second-line metastatic pancreatic cancer has never had a targeted option before this.
🔹 The administration is expected to announce Most favored nation pricing agreements with several mid size biotechs as early as Monday, extending a template that 17 large pharma companies have already signed, including Pfizer, Lilly and Amgen. The deal is outpatient discounts to state Medicaid programs benchmarked to what the company charges abroad, with state participation optional, in return for a three year exemption from pharmaceutical tariffs. For a large company that’s a rounding error against tariff exposure. But this can be much more consequential for a single-product mid cap biotech because it permanently resets the US net price while the tariff relief expires after three years.
🔹 Moderna announced a proposed private placement of $2B of convertible senior notes due 2032 on Aug 27, with a $300M upsize option, for oncology investment and debt repayment. The notes bear no regular interest, the principal doesn’t accrue, and capped calls set the effective dilution cap at a premium of at least 150% to the reference price. Shares fell about 5%. What makes this interesting is the sequencing. On August 19 Moderna gained 177% on an interim Phase 3 in resected melanoma reported with no hazard ratio, no p-value and no confidence interval. Eight days later, that stock move became financing. Zero coupon paper struck 150% above a price the market set on incomplete information is a very good trade for Moderna and a leap of faith for whoever buys it. Meanwhile Lilly has stopped publishing itsphase 1 pipeline and most deal values, and the neoantigen-selection algorithm behind Moderna’s vaccine stays undisclosed to the researchers being asked to evaluate it.
🔹 Roche’s Genentech licensed worldwide rights outside South Korea to Hanmi Pharmaceutical’s HM17321 on Aug 24 for $190M upfront, up to roughly $2.3B in milestones, and tiered royalties. HM17321 is a urocortin-2 analog, a non-incretin mechanism designed to drive weight loss while preserving lean mass. Incretin therapies deliver 15% to 25% total body weight reduction, but a meaningful fraction of that loss is lean tissue. The next competitive front in obesity is body composition rather than the headline weight loss percentage. Hanmi cleared a US investigational new drug application in Nov 2025 and is running Phase 1. Genentech takes over from Phase 2.
🔹 The full CARDIO-TTRansform results landed ahead of the European Society of Cardiology Congress and finally explain the July miss. In 1,432 patients randomized 1:1 to eplontersen 45 mg or placebo subcutaneously every four weeks across 130 sites in 20 countries, the composite of cardiovascular death and recurrent cardiovascular events through week 140 was not met. In patients not on a transthyretin stabilizer, the hazard ratio was significant at 0.71. In patients already on a stabilizer at baseline, which was 57% of each arm, plus another 24% who started one mid trial, there was no treatment effect at all. The plain reading is that once tafamidis or acoramidis is already stabilizing the protein, adding an injectable silencer on top doesn’t help much. That’s a commercial problem more than a biology problem, because the stabilizer-naive population shrinks every quarter. It also sets up a hard test for Alnylam, whose vutrisiran analyses and next gen nucresiran Phase 3 TRITON-CM both go on display in Munich starting Friday. Alnylam argues greater potency and a different trial design will separate it from eplontersen.
🔹 Spyre Therapeutics’ anti-TL1A antibody SPY072 missed its internal bar in a 143 patient, 12 week, placebo-controlled Phase 2 in rheumatoid arthritis. The lower dose reached statistical significance with a 1.9-point reduction in disease activity against 1.3 points for placebo. The higher dose missed the primary objective outright, and that’s what really matters, because a dose response that inverts is hard to interpret as a genuine drug effect. Safety was comparable to placebo. The stock fell 13% and gave up more than $1B of a market capitalization above $9B. TL1A is the target Merck bought Prometheus for, and that Sanofi and Roche both paid up to enter, all on inflammatory bowel disease data. It doesn’t automatically translate to rheumatoid arthritis, where the bar is a crowded field of drugs that already work well. Psoriatic arthritis and axial spondyloarthritis data come in the fourth quarter. The IBD case for TL1A is still fine. The case that it works across every inflammatory disease is not.
🔹 Akeso and Summit’s HARMONi-GI1 trial stopped early at a prespecified interim analysis after ivonescimab plus chemotherapy beat durvalumab plus chemotherapy on overall survival in previously untreated advanced biliary tract cancer, with progression and response endpoints also met. This is the first therapy to beat a checkpoint inhibitor + chemotherapy regimen on survival in this setting, and the first positive Phase 3 for the PD-1 by VEGF bispecific outside lung cancer. Summit rose 14%. However, the trial ran entirely in China, which is a real question for a US filing given how the FDA has handled single country oncology datasets. And no hazard ratio, median survival or patient count came with the announcement, which is becoming routine. Biliary tract cancer is a small indication, so the commercial value here is limited. What matters is the evidence that the mechanism generalizes beyond non-small cell lung cancer, where the ivonescimab argument has been stuck for two years.
Have a great rest of your week and thanks for reading Biotech Blueprint!
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Front cover image source: WSJ