Strong Revenue Growth
Q1 net product revenue of $215.4M, up 17% from $184.7M in Q1 2025; management reiterated full-year net revenue guidance of $1.00B to $1.04B.
Solid Patient Traction and Market Opportunity
Average ~8,500 patients on WAKIX in Q1 and ~8,600 at quarter end; diagnosed narcolepsy market ~80,000 patients (current penetration ~10–11%), indicating significant runway for continued patient growth.
Robust and Advancing Pipeline with Near-Term Milestones
Five ongoing Phase 3 registrational trials across distinct CNS indications. BP-205 (Orexin-2 agonist) on track for Phase 1 SAD PK topline data mid-2026 and U.S. IND mid-2026. Pitolisant GR on track for NDA submission in Q2 2026 (target PDUFA Q1 2027). Pitolisant HD is enrolling in two Phase 3 trials with topline data expected in 2027 (PDUFA 2028). EPX-100 OLE data showed median 50% seizure reduction in Dravet patients; registrational top-line data expected H1 2027 (PDUFA 2028).
IP Protection Progress and Litigation Outcomes Favorable to Date
Settled with 6 of 7 ANDA filers maintaining loss of exclusivity (LOE) until March 2030 (inclusive of 6 months pediatric exclusivity). Acquired Novitium license for an amorphous form with issued patents to 2042 and filed a new infringement suit against AET/Sandoz asserting that patent—management expresses confidence in a multilayered IP estate protecting pitolisant into the 2030s and potentially into the 2040s.
Commercial Investment and Execution
Completed field team expansion (~20% increase across field sales, remote sales and field reimbursement) with hires in place as of April 1. New online provider portal and enhanced reimbursement support to launch in Q2 to accelerate patient starts and dispense success.
Strong Balance Sheet and Focused BD Capacity
Cash and cash equivalents of $870.5M and modest debt of $160M at quarter end; leadership reiterates intent and capacity to deploy capital for business development targeting near-term revenue (2028–2032) and assets in Phase 3/market.