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Praxis Extends Relutrigine PDUFA to December 2026

The Tip Desk · August 6, 2026 · 3 min read

The FDA reset the SCN2A/SCN8A encephalopathy decision date after extra sensitivity analyses, while ulixacaltamide kept a January 29, 2027 target.

Praxis Precision Medicines (PRAX) said the FDA extended the review period for relutrigine’s new drug application in SCN2A and SCN8A developmental and epileptic encephalopathies, setting a new PDUFA target action date of December 27, 2026, after the agency deemed additional sensitivity analyses a major amendment.

The Boston-based CNS precision neuroscience biopharmaceutical company completed mid-cycle meetings for both relutrigine and ulixacaltamide HCl. The FDA identified no major safety or efficacy concerns to date on either program and does not plan to request an advisory committee meeting. Bioresearch Monitoring inspections of Praxis for both NDAs were completed with no Form FDA 483 observations.

Ulixacaltamide for essential tremor remains on a PDUFA date of January 29, 2027. The company said launch preparation is accelerating: commercial organization leaders have been hired, and infrastructure, marketing and disease-education programs, medical information capabilities, physician targeting, and a distribution network with commercial inventory build are in progress. In July 2026, Praxis initiated a collaboration with Remagine Labs to develop a transdermal patch of ulixacaltamide for ET.

Commercial and medical teams for relutrigine have been hired, inventory is being built, a patient support program is being established, and the company is engaging payers. Enrollment in the EMERALD study in broad DEEs exceeded the planned target, with approximately 200 patients enrolled across more than 50 genetically defined etiologies. Topline results are anticipated in the fourth quarter of 2026; assuming successful initial NDA approval of relutrigine, a positive EMERALD would support a supplemental NDA in 2027.

Vormatrigine’s POWER1 Phase 2/3 study in highly refractory focal onset seizures did not meet its primary endpoint of reduction in monthly focal seizure frequency from baseline at Week 12. The study met a secondary endpoint, with a significant number of patients achieving at least a 50% reduction in seizure frequency. Praxis is finalizing plans to restart POWER2 and initiate POWER3 in the fourth quarter of 2026.

The FDA granted Breakthrough Therapy Designation to elsunersen based on positive results from the EMBRAVE Part A trial of seizures associated with SCN2A DEE caused by gain-of-function variants—Praxis’ third BTD in twelve months. Elsunersen now holds Breakthrough Therapy, Orphan Drug, and Rare Pediatric Disease designations from the FDA. Enrollment in the EMBRAVE3 registrational trial is progressing, with topline results expected in 2027.

Cash, cash equivalents, and marketable securities were $1.4 billion as of June 30, 2026, compared with $926.1 million at December 31, 2025. The $447.8 million increase was primarily attributable to net proceeds from a January 2026 follow-on public offering and interest income, partially offset by cash used in operations. Cash is expected to fund operations into 2028.

Research and development expenses were $69.4 million for the second quarter of 2026, compared with $63.0 million a year earlier. The $6.4 million increase was primarily attributable to $6.7 million more Solidus spending, $4.4 million more personnel-related costs, and $1.8 million more indirect costs, partly offset by $6.5 million less Cerebrum spending. General and administrative expenses were $27.5 million, up $14.4 million from $13.1 million a year earlier, primarily from $7.2 million more professional expenses and $6.3 million more personnel-related costs.

The company incurred a net loss of $83.7 million in the second quarter of 2026, including $11.3 million of stock-based compensation, compared with $71.1 million a year earlier, including $7.8 million of stock-based compensation. Net loss per share was $2.87, with weighted-average shares of 29.1 million. Other income, net was $13.2 million. Common shares outstanding were 27.9 million as of June 30, 2026.

Praxis acknowledged late-stage discussions with the FDA on both applications and does not intend to communicate regulatory updates until the expected PDUFA dates.

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