Cenobamate

Praxis said the drug candidate was generally well tolerated, but 23% of patients discontinued the study. \n Praxis Precision Medicines has reported a 56.3% median reduction in seizure frequency from baseline in a midphase trial, encouraging the biotech to push ahead with phase 2/3 plans despite almost a quarter of patients discontinuing the epilepsy trial.This morning\'s readout came from 37 focal onset seizure patients who received the sodium-channel modulator vormatrigine in Praxis’ Radiant study. The drug candidate was generally well tolerated, according to Praxis, but 23% of patients discontinued the study.Of the patients who stuck with the treatment, the Boston-based biotech said (PDF) that 60% experienced at least a 50% reduction in seizure frequency over eight weeks.The company saw improvements early, with 54% of patients hitting the 50% threshold in Week 1, and it tracked a deepening of the responses over time. In the last month of the dataset, 22% of the patients experienced a 100% reduction in seizure frequency, according to an August 4 release.Praxis saw the responses in patients who were receiving other anti-seizure medications. More than 80% of participants were taking a sodium channel blocker and 30% were on SK Life Science’s Xcopri. Steven Petrou, Ph.D., chief scientific officer at Praxis, hailed the consistent efficacy across subgroups of patients on different background therapies as a strength of the study.“The bar in Radiant was especially high. This was the first epilepsy study launched and reported in the U.S. following widespread adoption of [Xcopri],” Petrou said on a call with investors to discuss the data.“This level of consistency, in both efficacy and tolerability, across different treatment backgrounds speaks to vormatrigine’s versatility,” Petrou added. “It reinforces the idea that this is a therapy with broad applicability.”Explaining the discontinuation rate, Petrou said many of the dropouts were linked to a lack of background therapy dose adjustment, despite protocol guidance. Adverse events and discontinuations “were avoided entirely” in six instances where investigators proactively reduced background treatment, the Praxis executive pointed out.  The discontinuation rate may explain the lackluster reaction from investors to this morning\'s readout, with shares in the biotech down nearly 14% to $46.68 in Monday trading from a Friday closing price of $54.09.During the trial, vormatrigine\'s efficacy was not impacted by the dialing back of other medications, Praxis CEO Marcio Souza said on the investor call. Souza used the finding to advance Praxis’ argument that vormatrigine could become a standalone therapy.“I think what we established is that the background therapy is not doing anything but causing side effects, and vormatrigine is getting these patients better,” Souza said. “So a monotherapy study, or a switch to monotherapy, makes a lot of sense.”Praxis is planning to start a monotherapy trial in the first half of 2026. Before then, the company is set to kick off a follow-up to Radiant, enrolling a larger patient population to receive placebo or one of three doses of vormatrigine.

A brain-focused biotechnology company saw a double-digit stock drop Monday morning even as it unveiled clinical trial results that some on Wall Street considered top notch.The mid-stage study recruited adults with either of two epileptic disorders, and gave them all a drug from Praxis Precision Medicines once a day for about two months. According to Praxis, by the end of the treatment period, median seizure frequency had declined 56%.The company also said its drug, now called vormatrigine,appeared fast-acting, with a little over half of the participants experiencing a 50% reduction in frequency in the first week of treatment. Around 22% achieved seizure freedom, meaning they stopped having seizures, at the eight-week mark.Participants were taking an average of two other anti-seizure medications, which, Praxis noted, made it very challenging to show its drug was having a positive effect. More than 80% were on a long-established type of treatment known as sodium channel blockers, with 30% taking a product sold by the South Korea-based developer SK Biopharmaceutical. Sales of that medicine, Xcopri, rose 47% between the first quarter of 2024 and the same period this year, where they reached 133.3 billion won or roughly $96 million.While there are many sodium channel blockers already available for the two types of seizures focal onset and tonic-clonicat the center of Praxis study, they often come with drawbacks. Some elicit side effects like dizziness or drowsiness, or need to be administered through time-consuming titrations. Xcorpi, for instance, has set the efficacy bar in focal onset seizures, yet its slow titration scheme and adverse events have driven clinicians to reserve its use for the most severe, hard-to-treat cases of focal onset seizures,according to TD Cowen analyst Ritu Baral.In a client note last month, Baral, who covers Praxis, wrote that key doctors in the neurology field have repeatedly underscored the need for new sodium channel drugs that avoid these problems. Thats what Praxis is hoping to do with its medicine, which is taken orally.Praxis said vormatriginewas generally well tolerated. Across 61 patients, researchers saw treatment-emergent adverse events in 36. Most were mild or moderate, with the most common being dizziness, drowsiness and headache. There were three cases each of severe and serious adverse events, though they all resolved. Almost one in four participants discontinued the study.Baral, in her note, wrote the best case scenario for Praxis is that its study would show a 55% or greater reduction in median monthly seizure frequency among patients with focal onset seizures. Such a result, Baral predicted, could lead to a 30-40% bump in the companys stock price.Instead, Praxis shares were down 13% by late Monday morning, to trade just under $47 apiece.To Brian Abrahams, an analyst at RBC Capital Markets, the seizure reductions seen in the trial could make vormatrigine “reasonably competitive.“ Abrahams, though, covers Xenon Pharmaceuticals, which is developing its own seizure medication that could rival Praxis'. The analyst argued in a client note Monday that not only is Xenon years ahead in development, but Praxis' data do not yet show a “clear efficacy advantage.“ And even if vormatrigine does eventually win approval, it would likely be used in combination with generics or other branded products like Xenon's azetukalner.Praxis plans to present more detailed data from the trial later this month, at an epilepsy medical conference in Portugal.Praxis released the trial results alongside its latest earnings report. For the three-month period from April through June, the company reported a net loss of $71.1 million, up from the $32.7 million loss recorded during the second quarter last year. '