Today, a brief rundown of news from Xenon Pharmaceuticals and Capricor Therapeutics, as well as updates from Pfizer, Regeneron, AbbVie and GSK that you may have missed.
An experimental and closely watched seizure medication has succeeded in a late-stage clinical trial, according to its developer, Xenon Pharmaceuticals. Called azetukalner, the drug is designed to calm overly excited neurons by keeping certain kinds of cellular, ion-transporting tunnels open for longer. Xenon's trial enrolled 380 people with focal onset seizures and found azetukalner was significantly better than a placebo at improving the frequency of their seizures. Twelve weeks into the experiment, median monthly frequency was down 53% among participants given a higher dose of the drug, versus down 10% in the control arm. Paul Matteis, an analyst at the investment firm Stifel, described the results as a "home run" and "way above investor expectations." Xenon said it plans to submit the drug for approval later this year. Company shares were up 50%, to nearly $63 apiece, at market's close Monday. — Jacob Bell
The Food and Drug Administration has resumed a review of Capricor Therapeutics’ cell therapy for Duchenne muscular dystrophy. The agency turned back the treatment for heart-related complications of Duchenne in July and asked for more data, in the process making Capricor one of many companies to recently accuse the regulator of flip-flopping on previous guidance. Capricor has since accumulated those results and, on Tuesday, said the FDA has begun a new evaluation that should conclude by Aug. 22. Capricor shares climbed by about 17% in early trading. — Ben Fidler
An experimental, three-pronged Pfizer drug hit its main goal in a Phase 2 trial in eczema, helping significantly more enrollees than a placebo reduce disease symptoms by 75%, the company said Monday. The high and medium monthly doses of tilrekimig helped 49% and 52% of the participants who got them, respectively, achieve the targeted level of skin clearance after 16 weeks. Pfizer said it will “accelerate” the drug into Phase 3 this year. Tilrekimig is a “trispecific antibody,” binding to three proteins implicated in multiple immune diseases. — Jonathan Gardner
Regeneron Pharmaceuticals on Monday said an obesity medicine it’s developing with Hansoh Pharmaceutical Group spurred up to 19% weight loss after 48 weeks in a Phase 3 trial in China. The medicine, olatorepatide, was also associated with “lower rates” of stomach side effects and treatment discontinuations than what’s been reported in other late-stage studies of drugs like it, Regeneron said. Regeneron licensed most rights to the therapy, which targets the same two gut hormones as Eli Lilly’s Zepbound, last year and plans to start a global, late-stage study in 2026. The data put Regeneron’s drug “on the map,” though its “competitive positioning” in obesity is yet to be determined, wrote RBC Capital Markets analyst Brian Abrahams. — Ben Fidler
An experimental obesity medicine AbbVie licensed last year helped non-obese people lose up to 10% of their body weight over 12 to 13 weeks in a Phase 1 trial, the company said Monday. The study evaluated several dosing levels and regimens of the drug, an amylin-targeting shot developed by Denmark-based Gubra and now called ABBV-295. People taking it achieved 10% weight loss over 12 weeks with one of three weekly doses, while others receiving an every-other-week dose hit that mark at week 13. People who ramped up to a once-monthly regimen after five weeks saw 8% weight loss at week 13. Placebo recipients, by comparison, lost less than 1% of their weight. — Jonathan Gardner
GSK has licensed rights to an experimental liver disease medicine to Italian pharmaceutical company Alfasigma. Under a deal announced Monday, Alfasigma will pay GSK $300 million up front for full rights to the therapy, known as linerixibat and nearing approval for a kind of severe itching associated with the rare condition primary biliary cholangitis. GSK could get another $100 million upon a U.S. approval expected later this month, and could receive up to another $290 million if the medicine wins additional clearances and hits certain sales targets. The deal "sharpens GSK’s focus to deliver our next wave of liver disease innovation," said top scientist Tony Wood, mentioning the company's focus on therapies for hepatitis B, MASH and ALD. — Ben Fidler