September has been a rough month already for Novo Nordisk, which canned two more late-stage trials of its embattled IL-6 inhibitor, and Novartis, which took Phase 3 hits in both cardiovascular and muscular dystrophy indications; Ultragenyx’s big bet in Angelman syndrome failed to yield benefit in Phase 3 but Ionis’ Pharmaceuticals nabbed the first targeted approval for Alexander disease; and on the business side, biopharma investment is back in a big way.
The cardiovascular space took another hit this week as Novo Nordisk reported it has ended two more late-stage studies for its investigational IL-6 inhibitor ziltivekimab—causing Oppenheimer analysts to declare that IL-6 “now looks like an invalid target” in cardiovascular disease. Another Phase 3 study of the asset failed in July.
Novo wasn’t the only company to take a clinical hit this week. Novartis reported a double blow, including the failure of a much anticipated Phase 3 trial for del-desiran—one of the key assets from the company’s $12 billion buyout of Avidity Biosciences last year—in myotonic dystrophy type 1. Novartis’ other miss was more bad news for the cardiovascular space, as the antisense therapy pelacarsen showed no significant benefit on cardiovascular risk in a late-stage study. Put that together with the suspension of multiple studies of a CAR T candidate last week after three fatalities, and the Swiss pharma found itself down 12% in premarket trading on Tuesday.
The rare disease space took a hit, too, as Ultragenyx’s high-risk Angelman syndrome bet was unsuccessful, with apazunersen failing to improve cognition in Phase 3. It wasn’t all bad news, though, as the FDA approved Ionis Pharmaceuticals’ Zanvastro as the first targeted treatment for Alexander disease—representing the first marketed product from the biotech’s independent neurology pipeline. And uniQure finally filed for FDA and U.K. approval of its Huntington’s disease gene therapy, AMT-130.
On the business side, the biotech sector’s performance over the past 18 months has “more than made up for a challenging 2+ year period,” Truist Securities said last week. Meanwhile, reverse mergers have risen a whopping 1,600% in Q3 to rival the traditionally more popular IPOs, and 2026 is also shaping up to be a strong year for cross-over investment rounds, according to an analysis by PitchBook.
