Denifanstat

Today, a brief rundown of news involving Otsuka Pharmaceutical and Arvinas, as well as updates from Regenxbio, Vigil Neuroscience and Sagimet Biosciences that you may have missed.Vera Therapeutics lost nearly a third of its market value Friday after Otsuka Pharmaceutical presented late-stage study data on a rival drug its developing for the kidney disease IgA nephropathy. At a medical meeting, Otsuka said its therapy, sibeprenlimab, led to a 51% reduction in proteinuria, a key marker of kidney health, after nine months of treatment. Though cross-trial comparisons can be misleading, Veras therapy led to a 42% reduction in proteinuria compared to placebo at a similar timepoint in its own Phase 3 study, causing investors to sell off company shares. Still, some analysts defended Vera. Jefferies Farzin Haque cautioned not to overinterpret the data and argued the two datasets are not clinically or statistically different for commercial uptake. The Food and Drug Administration could approve Otsukas drug by Nov. 28. On Monday, Vera said it intends to file an accelerated approval application in the fourth quarter. Ben FidlerArvinas and partner Pfizer have filed for a U.S. approval of their experimental protein-degrading drug for breast cancer, Arvinas said Friday. The two submitted the therapy,vepdegestrant, for use in people with estrogen-receptor positive, HER2 negative breast cancer and a mutation in the ESR1 gene. The application is based on Phase 3 datapresented at the American Society of Clinical Oncology meeting this week showing the drug held tumors in check longer than a standard treatment.That benefit was modest, however, and wasnt observed in the overall study population. Delilah AlvaradoRegenxbio released new data Thursday showing a potential impact on muscle function among Duchenne muscular dystrophy patients who received its experimental gene therapy in an early-stage clinical trial. Regenxbio said those given the dose its using in pivotal testing outperformed how historical data suggests they might on multiple widely used assessments of functional benefits. Still, shares fell by double digits, as some key information was missing from the update, the treatments effects appear to be plateauing and there is concern the data may not be strong enough to support an accelerated approval, wrote Jefferies analyst Maury Raycroft. Regenxbio expects to report pivotal data next year and has said it intends to seek a speedy approval afterwards, based on the therapys ability to produce a tiny version of a muscle-protecting protein. Ben FidlerVigil Neuroscience reported Wednesday the failure of a mid-stage trial meant to prove its most advanced drug works as intended. The study enrolled 20 people with ALSP, a rare disorder hallmarked by the erosion of white matter in the brain. All participants received Vigils drug, VGL101, which is designed to boost the TREM2 proteins that help control inflammation in the brain. The company said that while showing favorable safety and tolerability, its treatment had no beneficial effects on important markers of effectiveness or biological activity. As such, a separate, so-called long-term extension study is being discontinued. Sanofi is in the process of acquiring Vigil for access to a different TREM2-targeting medicine. Per terms of that deal, rights to VGL101 will be returned to its original licensor, Amgen. Jacob BellShares of Sagimet Biosciences have climbed nearly 40% since partner Ascletis Bioscience on Wednesday reported the companys drug denifanstat succeeded in a Phase 3 trial in China in people with moderate-to-severe acne. The drug produced compelling efficacy in the trial with only mild-to-moderate side effects, which could have positive implications for its ability to treat metabolic dysfunction-associated steatohepatitis, Leerink Partners analyst Thomas Smith wrote in a research note. A 2019 partnership handed Ascletis rights in greater China to denifanstat. Sagimet could receive up to $122 million in future payments, as well as sales royalties, if the drug is approved. Ben Fidler '