Harmony Biosciences provides comprehensive pipeline update

Harmony Biosciences will be providing a comprehensive pipeline update at its Investor Day event today, showcasing new data from its orexin-2 receptor agonist, next-generation pitolisant programs, and long-term extension data from its pitolisant program in idiopathic hypersomnia. The company will also provide program updates from its strategic acquisitions in Fragile X syndrome, FXS, and rare epilepsies, which have strengthened its late-stage development pipeline. Key Highlights: Orexin-2 agonist program: New data show BP1.15205 is based on a novel chemical scaffold and has demonstrated greater potency compared to all publicly disclosed data on orexin-2 agonists, with the potential to be best-in-class. Pitolisant in Idiopathic Hypersomnia: New data show robust and sustained efficacy of pitolisant in patients with idiopathic hypersomnia in the Long-Term Extension study. Pitolisant-HD program: Preliminary data confirm safety up to five times the highest labeled dose of WAKIX, establishing the safety margins for the pitolisant-HD development program. Fragile X syndrome program: A prespecified ad hoc analysis from the Phase 2 CONNECT-FX in Fragile X syndrome study , Social Avoidance subscale; this and other learnings from this trial have informed the design and selection of the primary endpoint for the Phase 3 RECONNECT study, which is on track for topline data in mid-2025. EPX-100 development program: New safety and tolerability data on EPX-100, topline data in DS is expected in 2026, and a pivotal Phase 3 trial for LGS is on track to initiate later this year.