Praxis Precision Medicines Inc

Praxis Precision Medicines, Inc. (NASDAQ:PRAX) reported second quarter 2026 financial results and provided updates on its two lead drug candidates under FDA review. The company ended the quarter with $1.4 billion in cash and marketable securities, up from $926.1 million at the end of 2025, and said its cash runway extends into 2028. Net loss for the quarter was $83.7 million, with research and development expenses of $69.4 million and general and administrative expenses of $27.5 million. The FDA completed mid-cycle reviews for both ulixacaltamide, for essential tremor, and relutrigine, for SCN2A and SCN8A developmental and epileptic encephalopathies, and indicated no advisory committee meetings are planned for either asset. Ulixacaltamide has a PDUFA date of January 29, 2027, and relutrigine has a PDUFA date of December 27, 2026. The company also announced a collaboration with Remagine Labs to develop a transdermal formulation of ulixacaltamide and said it plans to restart the POWER2 and POWER3 studies for vormatrigine in the fourth quarter of 2026.

Praxis Precision Medicines Inc. and Remagine Labs disclosed a research partnership and license agreement to design an iontophoretic transdermal patch for the delivery of ulixacaltamide, a strongly targeted T-type calcium channel inhibitor in development for essential tremor. Both companies will use Remagine's platform for precise, multi-day sustained skin-based dosing to support Praxis's oral program. Separately, on June 29, Praxis announced that the FDA extended the NDA review for relutrigine by three months, moving the PDUFA target date from September 27 to December 27, after the company submitted additional sensitivity analyses labeled as a major amendment. No new clinical trials were requested, and no safety or manufacturing issues were raised.

Praxis Precision Medicines shares rose after the U.S. FDA granted Breakthrough Therapy Designation to Elsunersen for treating seizures in SCN2A-DEE, a rare pediatric neurological disorder. The decision was based on Part A of the EMBRAVE study, where patients treated with Elsunersen experienced a 77% reduction in monthly seizure frequency compared with the sham control group, with improvements maintained for up to one year in the open-label extension. In the Phase 1/2 trial, 71% of treated pediatric patients achieved more than a 50% reduction in seizure frequency, and 57% experienced at least one 28-day seizure-free period during the six-month treatment phase. Praxis is continuing enrollment in its EMBRAVE3 registrational trial, a single-arm, baseline-controlled study of approximately 30 patients, under a streamlined development pathway agreed upon with the FDA. Elsunersen has also received Orphan Drug and Rare Pediatric Disease Designations in the U.S., as well as Orphan Drug and PRIME status in Europe. The stock is currently trading at $299.33, up 6.03%.