STAT+: We’re reading about an Ultragenyx failure, RevMed for lung cancer, and more

Hello there from STAT’s Andrew Joseph, writing to you from London and filling in for Mr. Pharmalot for the day. We just picked up a new bag of coffee beans that boasts notes of cola, cherry, and grape, so we’ll have to see if we pick up on any of that when we grind them up for tomorrow’s cup of stimulation. For now, though, perhaps some headlines will suffice … An experimental gene therapy for Angelman syndrome from Ultragenyx failed in a Phase 3 study, STAT reports. The medicine showed no benefit compared to a sham treatment in the rare disease, which causes severe intellectual disability and developmental delays. The drug, called GTX-102, had shown powerful results in early trials, raising the hopes of families affected by the devastating condition. The trial failure is also a significant blow to Ultragenyx’s business, and the company said it’s now planning “significant expense reductions.” Just a week after the landmark U.S. approval of a game-changing drug for advanced pancreatic cancer, the same therapy is showing promise against the world’s biggest cancer killer, Nature tells us. In a small trial in non-small cell lung cancer, the drug, Revolutions Medicines’ Rasonque, shrank tumors in more than 30% of participants. Each patient had tumor-promoting mutations in a member of the RAS family of proteins, and had previously tried other therapies. A larger study in which participants with non-small cell lung cancer are randomly assigned to receive either the drug, also called daraxonrasib, or standard chemotherapy is underway. STAT+ Exclusive Story Already have an account? Log in This article is exclusive to STAT+ subscribers Unlock this article — plus in-depth analysis, newsletters, premium events, and news alerts. Already have an account? Log in View All Plans To read the rest of this story subscribe to STAT+. Subscribe