Praxis Submits Two NDAs to FDA, Reports $926M Cash Position and $303M Net Loss

Praxis Precision Medicines, Inc. submitted two new drug applications to the U.S. Food and Drug Administration for ulixacaltamide in essential tremor and for relutrigine in SCN2A and SCN8A developmental and epileptic encephalopathies. The company reported cash and investments of $926 million as of December 31, 2025, and net proceeds of $621 million from a January 2026 public offering that are expected to fund operations into 2028.

Following a positive pre-NDA meeting with the FDA in December 2025, Praxis submitted an NDA for ulixacaltamide for the treatment of essential tremor. Essential tremor is one of the most common movement disorders, affecting approximately seven million patients in the U.S., yet it is inadequately managed and undertreated with no specific drugs developed for essential tremor currently approved, as underscored by the interest from over 200,000 patients in the Essential3 program. Ulixacaltamide was the first investigational therapy to demonstrate positive results in a Phase 3 program in essential tremor and was granted Breakthrough Therapy Designation by the FDA in December 2025.

In October 2025, Praxis announced positive topline results from both Phase 3 studies in the ESSENTIAL3 program. Praxis will present several oral presentations and posters on ulixacaltamide at the upcoming American Academy of Neurology Annual Meeting, taking place April 19 to 22, 2026 in Chicago, IL.

Praxis submitted an NDA for relutrigine for the treatment of SCN2A and SCN8A developmental and epileptic encephalopathies based on the strong efficacy observed in the EMBOLD registrational cohort. The results of the trial were shared at the 2025 AES meeting. Relutrigine is a sodium channel modulator designed to precisely target the hyperexcitable state of sodium-channels, with therapeutic potential across developmental epilepsies. Relutrigine has been granted Breakthrough Therapy Designation and Orphan Drug Designation by the FDA.

Enrollment in the EMERALD study in broad developmental and epileptic encephalopathies is progressing well and is expected to be fully enrolled in the second half of 2026. Assuming successful initial NDA approval of relutrigine, the EMERALD study, if positive, would serve as the basis for a supplemental NDA submission in 2027.

Commercial preparations and pre-launch activities for both ulixacaltamide and relutrigine are well underway and will accelerate through 2026. Praxis is scaling its commercial organization, advancing launch readiness efforts, building inventory, and will initiate its disease awareness campaign in conjunction with the AAN meeting. Praxis has begun preparations for the commercial launch of relutrigine, including hiring key commercial roles, building sufficient inventory for launch and preparing and executing key pre-launch activities, which are expected to accelerate throughout 2026.

The company also provided updates on its pipeline. The full dataset from the RADIANT Phase 2 study for vormatrigine were presented at the 2025 American Epilepsy Society Annual Meeting, positioning vormatrigine as a best-in-disease therapy. Study results showed its fast-acting efficacy without titration, sustained seizure reduction over longer treatment duration, seizure-freedom potential, and favorable DDI, tolerability and safety profiles with once-daily dosing. An estimated 3.5 million people in the U.S. suffer from common epilepsies. Vormatrigine is the most potent sodium-channel modulator ever developed for epilepsy and is designed to precisely target the hyperexcitable state of sodium-channels in adult common epilepsies.

The POWER1 Phase 3 study for focal onset seizures completed enrollment and exceeded its original target; topline results are expected in the second quarter of 2026.

Praxis reported a net loss of $303.3 million for the year ended December 31, 2025, which represents an increase from the $182.8 million net loss reported in 2024. Research and development expenses were $267.1 million for the year ended December 31, 2025, up by $114.7 million compared to the previous year. Fourth-quarter net loss was $88.9 million, reflecting increased development spending.

The president and chief executive officer stated that the other two programs in the clinic, vormatrigine and elsunersen, will both have topline results in the first half of 2026, keeping the company on track for additional NDA submissions in the next two years. Together, these four assets have a revenue potential of over $20 billion.