NDAs Accepted for Two Key Programs with Set PDUFA Dates
The FDA accepted NDAs for ulixacaltamide (Ulixa) and relutrigine with PDUFA dates set (Ulixa: January 29 next year; relutrigine: September 27). Company is ramping commercial efforts for potential U.S. launches within the next ~8 months.
Strong Clinical Milestones and Readouts Ahead
Completed recruitment for the EMBOLD broad DEE study with top-line results expected in Q4 2026. POWER1 readout for lamotrigine expected later this quarter. POWER2 enrollment progressing and expected to finalize this year with a readout early next year.
EMBRAVE Part A: Robust ASO Efficacy Signal
EMBRAVE Part A (elsunersen) in 9 children showed a 77% placebo-adjusted reduction in monthly seizures and demonstrated disease‑modifying components across multiple domains while maintaining a generally safe and well-tolerated profile.
Large Pipeline and Commercial Potential
Company highlights 4 late-stage assets with >$20 billion in peak sales potential; Ulixa alone is estimated to represent over $10 billion in peak sales (company estimate). Relutrigine expansion to broad DEE could target ~200,000 U.S. patients; vormatrigine targets ~3.5 million people with epilepsy in the U.S.
Robust Balance Sheet and Cash Infusion
Cash, cash equivalents, and marketable securities were $1.4 billion as of March 31, 2026, up from $926 million at December 31, 2025 — an increase of approximately $474 million (+51%). The company expects this cash to fund operations into 2028.
Commercial and Manufacturing Preparations Underway
Commercial leadership in place; field force hiring and training planned pre-launch; distribution network established. Ulixa supply includes two independent drug substance manufacturers and inventory planning at commercial scale (company cited 'metric tons' scale for Ulixa).
Strong Scientific Engagement and External Validation
Company presented 15 scientific presentations at the AAN meeting, including a plenary that received an Abstract of Distinction and Movement Disorder Awards. Large physician (2,300+) and patient (1,300+) observational studies validated Ulixa's profile (efficacy, breadth of benefits, tolerability).
Increased Investment in Commercialization and R&D
Operating expenses in Q1 were approximately $106 million, with R&D $78 million (73.6% of Opex) and SG&A $28 million (26.4%), reflecting deliberate investment to support launches and late-stage programs.