Praxis Precision Medicines Submits Two NDAs, Reports $926M Cash Position

Praxis Precision Medicines has 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 fund operations into 2028.

Both drug candidates have been granted Breakthrough Therapy Designation by the FDA. The relutrigine NDA submission is based on the strong efficacy observed in the EMBOLD registrational cohort, with results shared at the 2025 AES meeting. Relutrigine has also been granted Orphan Drug Designation by the FDA.

The ulixacaltamide NDA submission follows positive topline results from both Phase 3 studies in the ESSENTIAL3 program announced in October 2025 and a positive pre-NDA meeting with the FDA in December 2025. Ulixacaltamide was the first investigational therapy to demonstrate positive results in a Phase 3 program in 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. The ESSENTIAL3 program drew interest from over 200,000 patients. Propranolol, a beta-blocker, is the only approved pharmacotherapy for essential tremor, offering limited efficacy and poor tolerability and is also contraindicated for comorbidities that affect a significant share of the essential tremor population. In a patient survey, up to 77% of patients felt their essential tremor is inadequately controlled and up to 50% of patients aren't receiving treatment.

Ulixacaltamide is a differentiated and highly selective small molecule inhibitor of T-type calcium channels designed to block abnormal neuronal burst firing in the Cerebello-Thalamo-Cortical circuit correlated with tremor activity. The Breakthrough Therapy Designation for ulixacaltamide was based on the positive topline data from the Essential3 program, consisting of two pivotal Phase 3 studies.

Relutrigine is a sodium channel modulator designed to precisely target the hyperexcitable state of sodium-channels, with therapeutic potential across developmental epilepsies. 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.

Pre-launch activities for ulixacaltamide and relutrigine are 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 American Academy of Neurology meeting. The company 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.

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. Essential3 results will be presented as an oral presentation at the meeting.

The company's other clinical programs include vormatrigine for focal onset seizures and generalized epilepsy, with topline results expected in the first half of 2026. 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 full dataset from the RADIANT Phase 2 study were presented at the 2025 American Epilepsy Society Annual Meeting.

On February 13, 2026, Affinity Asset Advisors disclosed a new position in Praxis Precision Medicines, acquiring 185,000 shares in an estimated $54.53 million trade. The stake represents 3.11% of the fund's 13F reportable assets. As of February 13, 2026, shares of Praxis Precision Medicines were priced at $317.25, up 266.1% over the past year. The company's market capitalization stood at $8.8 billion with revenue of $7.46 million and net income of negative $273.04 million.

Third-quarter research and development expense rose to $65.8 million, and net loss widened to $73.9 million. Management highlighted pro forma cash and investments of approximately $956 million, including proceeds from an October offering, which are expected to fund operations into 2028.