Harmony Biosciences provides updates on WAKIX, pitolisant HD, pitolisant GR, and an orexin-2 receptor agonist.
In a press release issued this month, Jeffrey M. Dayno, MD, president and CEO of Harmony Biosciences Holdings Inc, says, “2025 is set up to be a transformational year for Harmony, as our robust, late-stage pipeline comes into focus and we advance our long-term growth strategy,” including initiating two phase 3 registrational trials with a pitolisant HD [high-dose] formulation in both narcolepsy and idiopathic hypersomnia.
Updates from Harmony’s sleep/wake franchise include:
WAKIX in Narcolepsy
- Net sales were $201.3 million for Q4 2024 and $714.7 million for full year 2024
- 2025 net revenue projected between $820 to $860 million
- Settlement reached in first Abbreviated New Drug Application litigation, giving Novugen Pharma a license to sell its generic product beginning in January 2030 or earlier under certain circumstances
- According to Harmony, this settlement reinforces the strength and durability of the company’s intellectual property portfolio and its vigorous defense of its patent estate
- On track to obtain pediatric exclusivity which, if granted, would add an additional 6 months of regulatory exclusivity
Pitolisant HD
- Higher dose and optimized pharmacokinetic profile designed to address the need for greater efficacy in excessive daytime sleepiness and other symptoms in patients with central disorders of hypersomnolence
- Phase 3 registrational trial in narcolepsy to include endpoint on narcolepsy-related fatigue in pursuit of differentiated label
- Trial to initiate in Q4 2025; topline data readout anticipated in 2027 with potential Prescription Drug User Fee Act date in 2028
- Phase 3 registrational trial in idiopathic hypersomnia to include endpoint on sleep inertia in pursuit of differentiated label
- Trial to initiate in Q4 2025; topline data readout anticipated in 2027 with potential Prescription Drug User Fee Act date in 2028
- Provisional IP filed out to 2044 for narcolepsy and idiopathic hypersomnia
Pitolisant GR (gastro-resistant)
- Pivotal bioequivalence study on track to initiate Q1 2025
- Topline data readout anticipated in Q3 2025 with potential Prescription Drug User Fee Act in 2026
- Provisional IP filed out to 2044
Orexin-2 receptor agonist (BP1.15205)
- Orexin-2 receptor agonist based on a novel chemical scaffold, with potential for once-a-day dosing
- Preclinical safety and efficacy data to be presented at SLEEP 2025
- Investigational Medicinal Product Dossier submission on track for mid-2025; first-in-human study expected to initiate 2H 2025
Harmony’s net product revenues for the quarter ended December 31, 2024, were $201.3 million, compared to $168.4 million for the same period in 2023. The 20% growth versus the same period in 2023 is primarily attributed to strong commercial sales of WAKIX driven by continued organic demand tapping into a large market opportunity (approximately 80,000 patients diagnosed with narcolepsy in the US) and the broad clinical utility of WAKIX across the approximately 9,000 healthcare professionals that the company calls on (about 5,000 of whom do not participate in an oxybate REMS program).
The average number of patients on WAKIX increased by approximately 300 sequentially to approximately 7,100 for the quarter ended December 31, 2024.
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