Welcome back to This Week in Biotech by Biotech Blueprint, edition 117, covering biotech and pharma news from September 4th to 10th, 2026.
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VIDEO SUMMARY
THIS WEEK’S KEY TAKEAWAYS 🔑
Novartis’s bet on buying its pipeline instead of building it backfired this week. In just 8 days, the company lost the lead asset from a $12 billion acquisition of Avidity Biosciences this February, and also lost a cardiovascular outcomes trial that has been running for six years. The company also placed a voluntary halt on eight cell therapy trials on August 24 due to three patient deaths. Shares fell about 11% in a single session, the worst day in the company’s history. As of Thursday evening, several major financial institutions have downgraded the stock, mostly to Hold. Novartis’s shareholder Artisan Partners didn’t blame the CEO but criticized the board’s oversight of deals.
Novartis’s pelacarsen failure deserves a closer look, because it might be the most consequential result of the week. Lipoprotein(a) or Lp(a) is elevated in about one in five people worldwide, and Mendelian randomization studies have linked it to cardiovascular risk. Nobody had ever tested whether lowering it actually helps with outcomes. Novartis finally ran that test in 8323 patients, and the answer was no - even though Lp(a) came down, strokes and heart attacks didn’t. So either the theory is wrong, or the drug didn't lower Lp(a) enough. Amgen and Lilly each have a drug that cuts Lp(a) by more than 90%, and they think it’s the second explanation. The idea is that Lp(a) might only cause damage above a certain level, and 72% left patients still above it. Nobody knows whether this is the correct hypothesis, but Amgen and Lilly’s trials are still running.
Back in April, FDA’s oncology advisory committee voted 6 to 3 against approving AstraZeneca’s camizestrant, but they just approved it this week. The drug works differently than most cancer therapies. Normally you switch treatment when a scan shows the tumor has grown. With camizestrant, patients get regular blood tests looking for a resistance mutation, and you switch as soon as it appears, before any scan shows progression. The FDA panel objected because the trial never compared this approach against the standard one of waiting for a scan. Last week I covered internal memos showing a senior FDA official overruled her own review team on Replimune’s melanoma drug. It seems like the agency is weighing outside advice differently than it used to, at least in some cases.
Third, notice who published numbers this week. Roivant gave a p-value, Pharvaris gave an effect size, Beeline gave a response rate. Amgen, AstraZeneca and Bristol Myers announced three major oncology wins and gave nothing. The companies that published full numbers this week were mostly the smaller ones.
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BIOTECH/PHARMA NEWS 🧬
🔹 On September 4 Novartis’s pelacarsen missed its primary endpoint in an 8,323 patient cardiovascular outcomes trial. On Sept 8 del-desiran failed the Phase 3 HARBOR study in myotonic dystrophy type 1, missing on video hand opening time in roughly 150 patients dosed every eight weeks over 54 weeks. Both landed inside two weeks of the company halting eight trials of its rap-cel cell therapy after three patient deaths. Shares fell about 11% on Sept 9, the largest one-day decline on record, cutting market value by about 24B Swiss francs to roughly 215B. Del-desiran was the lead of three antibody oligonucleotide conjugates acquired with Avidity Biosciences for $12B at $72 per share, closed Feb 27. The surviving programs are del-zota, filed for accelerated approval with priority review in Duchenne muscular dystrophy, and del-brax in facioscapulohumeral dystrophy. Artisan Partners managing director David Samra told Reuters that “the party is over” and that successive chairmen had failed the company on acquisitions, while explicitly declining to blame chief executive Vas Narasimhan. Chairman Giovanni Caforio took the job last year.
🔹 The first outcomes trial ever run on an Lp(a)-lowering drug missed its composite primary endpoint of cardiovascular death, non-fatal heart attack, non-fatal stroke and urgent coronary revascularization requiring hospitalization. The trial ran 8,323 patients over six years, tested both the overall population at Lp(a) of at least 70 mg/dL and a subpopulation at 90 mg/dL, and gave everyone guideline-directed lipid and blood pressure therapy on top. Lp(a) came down. Events did not. Novartis’s chief medical officer Shreeram Aradhye said the findings did not demonstrate that lowering translated into reduced risk. Ionis fell 13.1% premarket and loses tiered royalties in the mid-teens to low twenties plus up to $650M in milestones. William Blair notes pelacarsen lowers Lp(a) about 72% while Amgen’s olpasiran and Lilly’s lepodisiran exceed 90%, leaving room for a threshold effect. Citi would “not declare the mechanism dead” but wants full data to separate a near-neutral result from a directional miss. Full results come at a medical meeting.
🔹 The FDA granted accelerated approval to camizestrant, branded Etcamah, on Sept 4, combined with a CDK4/6 inhibitor for hormone receptor positive, HER2 negative advanced breast cancer once an ESR1 mutation is detected during first line therapy. This is the first cancer approval triggered by a resistance mutation found in circulating tumor DNA before imaging shows the disease progressing. In SERENA-6, 315 patients switched on the blood result and reached median progression-free survival of 16 months versus 9.2 months (hazard ratio 0.44, 95% CI 0.31 to 0.60, p<0.00001). Overall survival was immature. The Oncologic Drugs Advisory Committee voted 6-3 against in April, objecting to a nonstandard time zero, no evidence that mutation-guided switching beats waiting for actual progression, and no crossover. The label carries a boxed warning for QTc prolongation (heart rhythm problem).
🔹 Amgen’s Imdelltra (tarlatamab) plus AstraZeneca’s Imfinzi (durvalumab) improved overall survival at a planned interim analysis of DeLLphi-305, a 563-patient open label trial of first line maintenance in extensive stage small cell lung cancer after chemotherapy and durvalumab induction. Progression-free survival and response rate also hit. This is the first checkpoint inhibitor plus T cell engager combination to extend survival in the setting. Median survival with current care is about one year and only 3.6% of these patients are alive at five years. Dana-Farber’s Jacob Sands called the results among the most compelling he has seen. Neither company released a hazard ratio, a median or a confidence interval. The same day, BMS said arlo-cel met its primary endpoint of response rate in the registrational QUINTESSENTIAL trial, which would make it the first GPRC5D-directed CAR-T in quadruple-exposed myeloma, and released no response rate. These are changing claims, but no numbers to check any of them against.
🔹 Regulators placed a partial clinical hold on RISE-2, a late stage focal epilepsy study of Biohaven’s opakalim (BHV-7000), after rodent studies turned up a drug-related metabolite the agency wants explained. Enrolled patients keep dosing, recruitment stops. RISE-3, fully enrolled, is unaffected. Biohaven licensed the Kv7 channel opener to SK Biopharmaceuticals in late August for $350M upfront and up to $795M in total value, and analysts had flagged RISE-2 as likely necessary to support a filing. Biohaven says it disclosed all clinical and nonclinical data before signing and that SK was comfortable with the metabolite. RBC’s Leonid Timashev warned the hold “adds risk” and that the requested data could surface a concerning signal. Shares fell more than 10% and RBC downgraded the stock. Biohaven has now had a rare disease rejection, a spinal muscular atrophy failure and a depression within the past 12 months.
🔹 Beeline Medicines launched in April on a $300M Series A, built entirely around five programs Bristol Myers had shelved. The bet was that big pharma drops assets for portfolio reasons rather than scientific ones. This week, Afimetoran, an oral once daily inhibitor of Toll-like receptors 7 and 8, beat placebo in all three dose groups in a phase 2 lupus trial, with p<0.001 at every dose and safety matching earlier studies. They are heading into pivotal trials in both systemic and cutaneous lupus. Lupus has broken more drug programs than almost any other disease, so a clean dose ordered result there is a real signal. And the model is spreading. Kura Oncology did something similar this week, spinning its own diabetes work into a new company called Caspian Therapeutics with $50M and Lilly backing it.
🔹 Roivant reported Phase 2 results in pulmonary hypertension associated with interstitial lung disease, a condition where scarring in the lungs drives up blood pressure in the pulmonary arteries and the heart eventually fails trying to push against it. Their inhaled drug mosliciguat cut pulmonary vascular resistance by 56.3% placebo-adjusted at week 16 (p<0.0001), a 51.3% drop on drug against a 6.6% rise on placebo, in 135 patients across 87 sites in 20 countries. 6 minute walk distance improved 35.2 meters at week 16 (p=0.0027) and 52.7 meters at week 24, with NT-proBNP down 53.2%. Roivant says the resistance number is the largest reduction reported in any randomized controlled pulmonary hypertension trial. The commercially important detail is tolerability. Cough occurred in 12.1% on drug versus 18.2% on placebo, in a market where the cough burden from inhaled treprostinil is the standing complaint. Shares rose 19%. The Phase 3 PHrontier study is enrolling about 375 patients. A Phase 2 hemodynamic effect this large has failed to translate before, but rarely with the walk distance moving alongside it.
🔹 Pharvaris’s oral bradykinin B2 receptor antagonist deucrictibant extended-release reduced monthly attack rates by 83% versus placebo over 24 weeks in hereditary angioedema prophylaxis, rising to 87% in patients with type 1 or type 2 disease, across 85 patients. Every secondary efficacy endpoint hit statistical significance and adverse events were mild to moderate. Filings begin in the first half of 2027. This is the second pivotal win for the molecule, which already has an on-demand application under FDA review, and it puts a once daily pill against injectables in long term prevention. The same week, Intellia’s lonvo-z, a one time in vivo CRISPR treatment for the same disease, was accepted for priority review with a March 10, 2027 target date and no advisory committee planned. Patients with a lifelong genetic disease are about to be offered a daily tablet or a single infusion, and nobody knows yet which one they want.
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