Harmony Biosciences(HRMY) - 2025 Q4 - Annual Results

Financial Performance - Preliminary unaudited net product revenue for Q4 2025 was approximately $243 million, compared to $201.3 million for the same period in 2024, representing a growth of ~21%[6] - Full year 2025 net product revenue was approximately $868 million, compared to $714.7 million for 2024, indicating a year-on-year growth of ~21%[6] - WAKIX is projected to achieve net revenue between $1.0 billion to $1.04 billion for the full year ending December 31, 2026, on track for blockbuster status in narcolepsy[6] Product Development and Pipeline - The average number of patients using WAKIX increased by ~400 to approximately 8,500 in Q4 2025, marking the third consecutive quarter of ~400+ patient additions[6] - The pitolisant franchise is expected to extend into the 2040s with next-gen formulations, including a gastro-resistant version (Pitolisant GR) anticipated for NDA submission in Q2 2026[6] - Five ongoing Phase 3 registrational clinical trials are in progress for five distinct CNS indications, reinforcing the robustness of the late-stage pipeline[6] - The Phase 3 topline data readout for Pitolisant in Prader-Willi Syndrome (PWS) is expected in the second half of 2026, supporting pediatric exclusivity for WAKIX[6] - The orexin-2 receptor agonist (BP1.15205) is currently in a Phase 1 trial, with clinical data expected in mid-2026[6] - The ZYN002 program in Fragile X syndrome is being phased out, and Harmony is no longer pursuing a 22q deletion syndrome indication[7] Company Strategy and Focus - Harmony Biosciences is positioned as a profitable, self-funding biotech with a strong balance sheet, ready to expand its commercial portfolio and pipeline[4] - Harmony Biosciences is focused on developing therapies for rare neurological diseases with significant unmet medical needs[26] - The company is developing EPX-100 (clemizole hydrochloride) for Dravet syndrome and Lennox-Gastaut syndrome, targeting central 5-hydroxytryptamine receptors[23] - Harmony plans to submit a New Drug Application (NDA) for pitolisant GR and aims to extend the pitolisant franchise into the 2040s[27] - The company emphasizes the importance of market acceptance and clinical utility for its product candidates, including pitolisant[28] - Harmony is committed to exploring the therapeutic potential of pitolisant in additional indications, including pitolisant GR and pitolisant HD[28] - The company is focused on identifying and integrating additional products with significant commercial potential[28] - Harmony acknowledges the risks associated with commercialization efforts and the competitive landscape in the pharmaceutical industry[28] - The company has a vision of combining empathy and innovation to foster therapeutic advancements for patients[26] Disease Focus - Dravet syndrome has an estimated incidence rate of 1 in 15,700, with approximately 85% of cases caused by SCN1A gene mutations[24] - Lennox-Gastaut syndrome affects around 48,000 patients in the U.S., characterized by multiple seizure types and developmental issues[25]