Harmony Biosciences Holdings, Inc. (NASDAQ: HRMY) has announced robust financial results for the third quarter of 2024, alongside key advancements in its late-stage pipeline. The company reported $186.0 million in net revenue for the quarter, marking a 16% increase compared to the same period in 2023. This performance brings WAKIX's cumulative net revenue to over $2 billion since its launch in November 2019 for adult narcolepsy.
WAKIX Performance and Expansion
WAKIX (pitolisant) continues to be a key revenue driver, with approximately 6,800 patients on the medication by the end of the quarter, reflecting a sequential increase of 250 patients. The drug is approved for excessive daytime sleepiness (EDS) or cataplexy in adult patients with narcolepsy and for the treatment of EDS in pediatric patients 6 years of age and older with narcolepsy.
Pitolisant sNDA Submission for Idiopathic Hypersomnia
Harmony Biosciences is preparing to submit a supplemental New Drug Application (sNDA) to the FDA in Q4 2024 for pitolisant in idiopathic hypersomnia (IH). Data from a long-term extension study demonstrated sustained efficacy, with patients showing a mean improvement of approximately 9 points on the Epworth Sleepiness Scale (ESS) from baseline beyond one year. Sustained efficacy was also observed on the Idiopathic Hypersomnia Severity Scale (IHSS) and Sleep Inertia Questionnaire (SIQ).
Advancing Pitolisant Formulations
The company is developing next-generation formulations of pitolisant, including Pitolisant-HD and Pitolisant-GR. Pitolisant-HD is designed with a higher dose and optimized pharmacokinetic profile, targeting fatigue in narcolepsy. Preliminary safety data supports this program. Pitolisant-GR is a gastro-resistant formulation aimed at minimizing gastrointestinal tolerability issues in narcolepsy patients. A pivotal bioequivalence study and dosing optimization study are planned for Q1 2025, with a PDUFA target in 2026.
Rare Epilepsy Program: EPX-100 and EPX-200
Harmony Biosciences is progressing its rare epilepsy program, with EPX-100 (clemizole hydrochloride) in a Phase 3 registration trial (ARGUS study) for Dravet syndrome. Topline data is expected in 2026. A Phase 3 trial for Lennox-Gastaut syndrome is slated to begin before the end of the year. EPX-200 (lorcaserin hydrochloride), a potent, selective 5HT2C agonist, is in the IND-enabling stage.
Neurobehavioral Program: ZYN-002 for Fragile X Syndrome
The pivotal Phase 3 RECONNECT trial for ZYN-002 in Fragile X syndrome is ongoing, with topline data anticipated in mid-2025. A Phase 3 trial in 22q11.2 deletion syndrome is expected to begin in 2025.
Financial Outlook
Harmony Biosciences reiterates its 2024 net product revenue guidance of $700 million to $720 million. The company's strong financial position is supported by $504.7 million in cash, cash equivalents, and investments as of September 30, 2024.
