Praxis Surges After Epilepsy Drug Trial Halted for Efficacy
A second positive FDA update for another key drug on the same day propelled shares higher, signaling strong momentum in the biotech's late-stage pipeline.
Shares of Praxis Precision Medicines (NASDAQ: PRAX) surged in after-hours trading on Thursday following a cascade of positive clinical and regulatory news that significantly de-risked two of the company’s leading drug candidates. The stock jumped 5.7% to $200.80 in extended trading after the company announced its pivotal trial for an experimental epilepsy treatment was being stopped early due to overwhelming effectiveness.
The primary catalyst was the success of the EMBOLD study for relutrigine, a treatment for rare and severe pediatric seizure disorders known as SCN2A and SCN8A developmental and epileptic encephalopathies (DEEs). An independent Data Monitoring Committee recommended halting the trial ahead of schedule, a rare and powerful signal in clinical development that indicates the drug has demonstrated a clear therapeutic benefit. These genetic disorders currently have no approved treatments and are associated with high mortality rates, positioning relutrigine to address a critical unmet medical need.
Praxis announced it will present the full results at the American Epilepsy Society Annual Meeting on December 6 and plans to meet with the U.S. Food and Drug Administration (FDA) to discuss the data and a path toward a New Drug Application (NDA). The drug has already received Orphan Drug and Rare Pediatric Disease designations from the FDA, which could expedite its review and provide market exclusivity upon approval.
“Stopping a study early for efficacy is a significant positive milestone and a testament to the potential of relutrigine to make a meaningful difference for patients and families devastated by SCN2A and SCN8A-DEEs,” the company stated in its press release.
Fueling the investor optimism was a second major announcement released just minutes earlier. Praxis also disclosed the successful completion of a pre-NDA meeting with the FDA for its other lead asset, ulixacaltamide, a treatment for essential tremor. The company confirmed it is aligned with the agency on the contents of its submission, with plans to file the NDA in early 2026.
This dual dose of positive news provides substantial validation for the company's late-stage pipeline and sent the stock price toward its 52-week high of $206.71. The back-to-back announcements suggest Praxis is rapidly advancing toward becoming a commercial-stage entity.
Wall Street has been closely watching the development of ulixacaltamide. Following positive Phase 3 data in October, analysts at Guggenheim raised their price target on Praxis to $350, citing the drug’s “blockbuster potential,” while Truist Securities increased its target to $360. While the stock’s momentum was briefly challenged by a short-seller report in November questioning trial data, Thursday’s positive FDA update appears to have restored confidence in the program's regulatory path.
With the EMBOLD study success de-risking one major pipeline asset and the ulixacaltamide program now on a clear track for a 2026 submission, Praxis is poised for a transformative period. Investors will be keenly focused on the upcoming epilepsy data presentation and further details on the company's regulatory filings as it moves two promising CNS therapies closer to market.