Harmony Biosciences (NASDAQ:HRMY) highlighted record quarterly performance for its narcolepsy treatment WAKIX and outlined development plans across its orexin, epilepsy and pitolisant lifecycle-management programs during Piper Sandler’s virtual CNS Symposium.
Peter Anastasiou, the company’s chief operating officer, said WAKIX’s seventh year on the market continued to demonstrate its role in narcolepsy treatment and the company’s commercial capabilities. He said the product’s performance supports investment in pipeline programs, including BP-205, and provides flexibility for potential business-development transactions.
BP-205 Data Support Broad Orexin Development Plans
Harmony reported results from a randomized, double-blind, placebo-controlled single-ascending-dose study in 72 healthy volunteers. Doses ranged from 0.2 milligrams to 6 milligrams. Budur said the study showed a time to maximum concentration of 30 to 75 minutes, dose-proportional exposure across the evaluated range and a half-life of approximately 25 hours.
The company believes those pharmacokinetic characteristics could support once-daily dosing. Budur said once-daily administration could be beneficial for patient convenience and adherence, while the longer half-life could help maintain wakefulness later in the day.
In the single-ascending-dose study, reported adverse events included headache in approximately 10% of participants, fatigue in 4% and diarrhea in 3%. The company said it did not observe cardiovascular, hepatic or visual disturbances. Harmony has completed dosing in a multiple-ascending-dose study, where it observed target-engagement adverse events including insomnia and polyuria. Budur said insomnia was neither severe nor sustained, and no visual adverse events had been seen in the multiple-ascending-dose dataset to date.
Harmony expects to disclose the full multiple-ascending-dose dataset in the fourth quarter. It has opened an investigational new drug application in the U.S. and plans to begin a sleep-deprived healthy-volunteer study, with top-line data expected in early 2027.
Anastasiou said Harmony and partner Bioprojet intend to pursue a broad orexin strategy, potentially including a portfolio of orexin agonists. The company is evaluating potential uses in cognition, attention, mood and fatigue, in addition to narcolepsy and idiopathic hypersomnia. Harmony intends to initiate multiple Phase II studies for BP-205 in mid-2027, though it did not identify the specific indications.
EPX-100 Program Targets Dravet and Lennox-Gastaut Syndromes
Harmony also discussed EPX-100, or clemizole hydrochloride, which is in Phase III development for Dravet syndrome and Lennox-Gastaut syndrome. Anastasiou said the epilepsy markets remain characterized by polypharmacy and continued unmet need for both seizure control and tolerability.
According to Budur, prior open-label extension data in Dravet syndrome showed a median 50% reduction in countable motor seizures over 28 days among patients exposed to clemizole hydrochloride for at least six months. He said 50% of patients experienced a 50% reduction in seizure frequency. The patients were taking at least four antiseizure medicines, with clemizole used as adjunctive therapy.
The company said clemizole’s reported tolerability profile has not indicated a need for echocardiogram or liver-function monitoring. Budur said diarrhea, occurring in approximately 2% of patients, was the only adverse event of note in the data previously presented. Harmony plans to present an additional data cut at the American Epilepsy Society meeting later this year.
Harmony expects top-line data from its Phase III Dravet and Lennox-Gastaut studies in 2027 and anticipates a potential approval in 2028. The company said clemizole is administered twice daily, compared with three-times-daily dosing for bexicaserin, though Budur said comparative efficacy, safety and tolerability will need to be evaluated as additional data emerge.
Pitolisant Formulations Aim to Expand Treatment Options
Harmony also outlined plans for gastro-resistant and higher-dose formulations of pitolisant. The company has an April 27 PDUFA date for pitolisant GR, a gastro-resistant formulation intended to potentially reduce gastrointestinal side effects and allow patients to begin treatment at a therapeutic 17.8-milligram dose rather than titrating from a subtherapeutic dose.
Anastasiou said gastrointestinal symptoms are common among narcolepsy patients and may not necessarily be related to therapy. If approved, Harmony expects to use its existing sales force, marketing organization, specialty-pharmacy network and patient-support hub to commercialize the product. He added that the company has filed utility patents that could provide exclusivity into the 2040s.
Pitolisant HD, an optimized higher-dose formulation, is being evaluated in separate Phase III studies in narcolepsy and idiopathic hypersomnia. Harmony is studying doses up to 60 milligrams of pitolisant hydrochloride. The company expects top-line data in 2027 and a potential PDUFA in 2028.
Budur said the narcolepsy study will also assess fatigue, while the idiopathic hypersomnia study will assess sleep inertia. Anastasiou noted that idiopathic hypersomnia is not currently an indication for WAKIX, making it a potential new patient population for the company.
About Harmony Biosciences (NASDAQ:HRMY)
Harmony Biosciences Holdings, Inc is a commercial‐stage biopharmaceutical company focused on developing and delivering therapies for people with rare neurological and endocrine diseases. Founded in 2017 and headquartered in Plymouth Meeting, Pennsylvania, Harmony Biosciences went public in 2020 and trades on the Nasdaq under the ticker HRMY. The company’s mission centers on identifying and advancing medicines that address critical unmet needs in patient populations underserved by existing treatments.
The company’s flagship product is WAKIX (pitolisant), the first and only histamine H3 receptor antagonist/inverse agonist approved by the U.S.
