PLYMOUTH MEETING, PA — Harmony Biosciences Holdings, Inc. (Nasdaq: HRMY) announced that its Phase 3 registrational INTUNE study evaluating the safety and efficacy of pitolisant in adult patients with idiopathic hypersomnia (IH) recently completed enrollment nine months before its estimated enrollment completion date. Topline results are anticipated in the fourth quarter of 2023.
“Completing enrollment in the INTUNE study now puts us nine months ahead of plan and reflects the strong interest and enthusiasm from patients and healthcare professionals alike, the unmet medical need in the IH community, and the strong momentum of our broader pitolisant development program,” said Harmony’s Chief Medical Officer, Kumar Budur M.D., M.S. “At Harmony, we are harnessing a mechanism-based approach to the development of pitolisant, which is thought to target the histamine system to promote wakefulness in the brain. This approach is driven by our shared conviction that innovative science translates into therapeutic possibilities that have the potential to impact the lives of individuals with rare neurological diseases.”
The INTUNE study is a double-blind, placebo-controlled, randomized withdrawal Phase 3 registrational trial in approximately 200 adult patients with IH being conducted at 58 clinical trial sites across the U.S. The primary objective is to evaluate the safety and efficacy of pitolisant compared with placebo in treating excessive daytime sleepiness (EDS). Secondary objectives include assessing the impact of pitolisant on other important symptoms of IH, such as sleep inertia and cognitive impairment. Other outcome measures include patient impression of overall change in symptoms, investigator assessment of overall disease severity, and functional status. Based on recent insurance claims data, the number of patients diagnosed with IH in the U.S. ranges from 30,000 – 40,000.
Pitolisant is marketed as WAKIX® in the U.S. for the treatment of EDS or cataplexy in adult patients with narcolepsy. Pitolisant is not approved for IH and is currently being evaluated as an investigational agent in adult patients with IH.