Today, a brief rundown of news from Dyne Therapeutics and Kymera Therapeutics, as well as updates from Wave Life Sciences and Structure Therapeutics that you may have missed.
Dyne Therapeutics will seek approval of a new type of “exon skipping” drug for Duchenne muscular dystrophy. According to Dyne, treatment with the therapy, z-rostudirsen, led to a more than 5% increase in levels of a muscle-protecting protein in a registrational trial. Drug recipients also displayed improvements, after six months, on multiple indicators of motor function, though the study wasn’t powered to detect a statistical benefit on those measures. Dyne intends to file for an accelerated U.S. clearance next year in Duchenne patients who have mutations in exon 51 of the dystrophin gene. There is some “consternation” from investors, as volatility at the Food and Drug Administration and the failures of multiple confirmatory trials have raised concerns about the regulatory bar for Duchenne medicines, wrote Stifel analyst Paul Matteis. Still, Dyne’s data on functional measures are the “best ever generated” for a drug of its kind in a placebo-controlled trial, he claimed.
Shares of Kymera Therapeutics rose by more than 50% on new study results the company revealed for a protein-degrading drug it’s developing for inflammatory conditions. Kymera disclosed in June that the drug, KT-621, appeared safe and able to degrade a tough-to-reach protein in the blood and skin that’s linked to inflammation. Data announced on Monday build on those results, indicating KT-621 — an oral medication — is impacting markers of disease in atopic dermatitis with “biologic-like or better efficacy,” wrote Mizuho Securities analyst Joseph Catanzaro. One mid-stage trial is ongoing in atopic dermatitis, and a second in asthma should begin next year.
Wave Life Sciences saw its share price nearly double after initial study data suggested one of its drug prospects might have potential treating obesity. When compared to a placebo, a single, low dose of the therapy, an oligonucleotide-based treatment called WVE-007, was associated with a 9% reduction in harmful “visceral” fat, a 4% decline in total body fat, and a slight improvement in lean mass after three months. There have been no study discontinuations or serious treatment-emergent adverse events, either. Wave is planning Phase 2 trials testing WVE-007 as a monotherapy and alongside incretin drugs for weight loss. The results so far “signal this program will be competitive,” Leerink Partners analyst Joseph Schwartz wrote.
Structure Therapeutics also reported positive results for a prospective obesity drug. Called aleniglipron, the treatment is a GLP-1 pill, much like a therapy known as orforglipron that Eli Lilly could soon bring to market. In a Phase 2 trial, a 120 milligram dose of aleniglipron spurred weight loss of 11% after nine months compared to a placebo, “similar” efficacy to Lilly’s drug, wrote Leerink’s David Risinger. And though the drug appeared less tolerable — rates of vomiting were higher than what’s been observed in testing of orforglipron — a low starting dose compared more favorably, Risinger added. Structure said the findings support advancement into Phase 3 trials. Company shares, which debuted at $15 apiece in 2023, nearly doubled and were changing hands Monday morning at over $65 apiece.
