After Study 1’s independent data monitoring committee said the experiment will likely fall short of its primary endpoint with ulixacaltamide in essential tremor, Praxis Precision Medicines Inc. plans to wait for full data from Study 1 as well as Study 2 in the Essential 3 phase III program before deciding on regulatory moves.
In the wake of Sage Therapeutics Inc.’s recent phase II fizzle with SAGE-324 in essential tremor, Wall Street is waiting for better news in the indication, which has racked up failures across several developers. Among those finding favor lately is Praxis Precision Medicines Inc. with Nav-targeting ulixacaltamide, also known as PRAX-628, which Oppenheimer analyst Francois Brisebois said “warrants particular attention.”
Despite numerous marketed anti-seizure medications in the U.S., patients with epilepsy continue to experience breakthrough seizures and tolerability issues compounded with drug interactions and side effects, leaving room for companies like Praxis Precision Medicines Inc. to fill treatment gaps. The Boston-based company presented phase IIa proof-of-concept data March 26 showing that in an evaluation of photo paroxysmal response, its once-daily oral small-molecule treatment, PRAX-628, resulted in a complete response in three epilepsy patients receiving 45 mg of the drug and four patients receiving 15 mg for a combined complete response rate of 88%.
Xenon Pharmaceuticals Inc. took to the stage this week at the J.P. Morgan Healthcare Conference to talk up its pipeline, including the phase III program testing XEN-1101 in focal-onset seizures (FOS), due to complete enrollment in the second half of this year. It’s an indication where such other players as Biohaven Ltd. are busy, too. Several million adults are afflicted with FOS in the U.S., with close to a half-million pediatric patients.
Researchers from Praxis Precision Medicines Inc. presented the discovery and preclinical evaluation of a novel voltage-gated sodium channel (Nav) blocker, PRAX-628, being developed as a potential antiepileptic drug candidate.
