Roche's sefaxersen hits phase III endpoint in IgA nephropathy

Wells Fargo initiated coverage of Design Therapeutics with an overweight rating, citing the company's Friedreich ataxia candidate DT-216P2 as having potentially best-in-disease functional improvement based on results from the RESTORE-FA study released in May. The bank set a $26 price target, implying roughly 112% upside based on the October 1 close. Analyst TianQi Hang wrote that the May update showed pharmacokinetics look good, and that blood-FXN protein, muscle-mRNA data, plus an early mFARS signal further de-risk the platform. Hang estimates that the blood FXN protein increase seen after 6 weeks can translate to at least a 2-point mFARS change, and said that if the drug kinetics sustain for 12 weeks, which he believes they will, DT-216P2 could deliver best-in-disease functional benefits. He assigns DT-216P2 a 60% probability of success, with peak sales of approximately $600M in the US and approximately $900M outside it. If approved, DT-216P2 would compete against Biogen's Skyclarys, also known as omaveloxolone, and Hang sees it gaining a peak share of the FA treatment market of 30% in the US and 20% ex-US.

Kodiak Sciences reported new Phase 3 data showing its eye drugs Zenkuda and tabirafusp alfa tedromer matched Eylea on primary endpoints for macular degeneration treatment, putting fresh competitive pressure on Regeneron Pharmaceuticals. The trial reported similar vision outcomes with Kodiak's candidates given roughly every six months versus Eylea's current eight week dosing interval. Regeneron, a US biotech group with a US$78.7b market cap, relies on Eylea as a key product in eye disorders, and the company's investor narrative already flags heavy reliance on Eylea and intensifying branded and biosimilar competition as a key risk to revenue sustainability. Kodiak plans to move closer to potential FDA filings in the fourth quarter of 2026, and the clearest early sign of impact will be Eylea and Eylea HD sales trends, with quarterly volume and pricing data in retinal disorders showing whether eye specialists actually switch away from Regeneron. Attention also remains on how quickly Regeneron can shift its mix toward Dupixent, Eylea HD and newer areas like oncology and obesity.