Praxis Precision Medicines has disclosed a median reduction in the rate of seizures by 56.3% from baseline in an intermediate-phase study. The encouraging results prompted the biotech to press forward with Phase 2/3 plans, even though nearly a quarter of the participants dropped out of the epilepsy trial.
The data came from 37 patients with focal onset seizures who received the sodium-channel modulator vormatrigine in the Radiant trial. According to the company, the drug was typically well-tolerated, but 23% of participants left the study.
From the individuals who continued the therapy, the firm, based in Boston, stated in a document that 60% had a decline of at least 50% in the rate of their seizures during an eight-week period.
The company witnessed early improvements, as 54% of particpants reached the 50% reduction mark in the first week, and it observed that the positive responses grew stronger with time. By the final month of the dataset, 22% of the patients had a complete decrease in seizures, as per an August 4 release.
Praxis noted the positive outcomes among individuals who were concurrently taking other drugs for seizures. More than eight in ten participants were using a sodium channel-blocking substance, and 30% used Xcopri by SK Life Science.
Steven Petrou, Praxis’s chief scientific officer, lauded the consistent effectiveness seen among distinct patient groups with varied background therapies, calling it a key advantage of the study.
Petrou told investors on a data call that the threshold set by Radiant had been particularly high, noting that it was the initial U.S. epilepsy trial conducted and reported after Xcopri became widely used. He further observed that the consistent performance in both effectiveness and tolerability across varied prior treatments clearly demonstrated vormatrigine’s adaptability and underscored its potential to serve as a broadly applicable therapeutic option.
In an explanation of the dropout rate, Petrou stated that numerous withdrawals were linked to an absence of dose modifications for background treatments, even though the trial’s guidelines provided direction.
The Praxis executive noted that quite a lot of negative events and discontinuations were completely averted on six occasions where researchers acted proactively and lowered a patients’ background treatments.
The high rate of patient withdrawals could account for the less-than-stellar investor response to the results. The company’s stock fell by almost 14% to $46.68 during trading on Monday, a decline from the prior Friday’s closing price of just over $54.
Throughout the study, vormatrigine’s effectiveness remained unchanged when other medications were reduced, according to comments made by Praxis CEO Marcio Souza during an investor call. The CEO leveraged this finding to help Praxis’s argument that the drug has the potential to function as a standalone treatment.
Souza remarked that the data demonstrated the existing background treatment was largely ineffective and mainly introduced adverse effects, while vormatrigine appeared to be improving patients’ conditions. Based on these observations, he suggested that pursuing a monotherapy trial or transitioning to monotherapy seemed like a logical course of action.
