Praxis Precision Medicines Inc. (PRAX) shares surged 24.81% in pre-market trading Friday, climbing to $237.10 after the Boston-based clinical-stage biopharmaceutical company reported promising trial results that could lead to the first approved treatment for certain devastating genetic epilepsies.
The stock closed Thursday's regular session at $189.97, up 2.05%.
Trial Stopped Early Due to Strong Efficacy
Praxis reported positive results from the registrational cohort of its EMBOLD study, which is evaluating relutrigine for SCN2A and SCN8A developmental and epileptic encephalopathies (DEEs). These are rare genetic conditions that cause severe, treatment-resistant seizures in children.
The Food and Drug Administration has scheduled a meeting in the coming weeks to review the data and discuss next steps. Praxis will decide on the timing of its New Drug Application (NDA) filing for relutrigine after that meeting.
Addressing an Unmet Medical Need
Marcio Souza, president and chief executive officer, said in the release that "SCN2A and SCN8A DEEs are devastating conditions with extremely high mortality due to the debilitating seizure burden they impose on patients, and there are currently no approved treatment options."
On Saturday, the company will present its topline results at the American Epilepsy Society Annual Meeting.
Essential Tremor Program Also Progressing
In a separate announcement Thursday, Praxis said it completed a pre-NDA meeting with the FDA for ulixacaltamide, an essential tremor treatment, and expects to submit the NDA in early 2026 after aligning on its content.
Short seller Culper Research previously challenged the ulixacaltamide trial data, claiming there were endpoint changes and optimistic assumptions for discontinued patients.
Impressive Stock Performance
The stock has surged 349.53% over the past six months, reflecting investor optimism about the company's pipeline.
Praxis Precision Medicines has a market capitalization of $4.75 billion, with its stock trading between $26.70 and $206.36 over the past 52 weeks.