Harmony Biosciences LLC will present data on the efficacy and safety of pitolisant, which has been studied for the treatment of both excessive daytime sleepiness (EDS) and cataplexy in adult patients with narcolepsy, at the upcoming 2019 American Academy of Neurology (AAN) annual meeting in Philadelphia, May 4-10. Harmony is a biopharmaceutical company dedicated to developing and commercializing novel treatment options for people living with rare diseases.

“We are pleased to have the opportunity to present data on pitolisant at this year’s AAN annual meeting demonstrating our commitment to investigating treatment options for people living with rare diseases such as narcolepsy,” says Harmony’s chief medical officer, Jeffrey Dayno, MD, in a release. “These data reflect the body of scientific evidence that establishes the efficacy and safety of pitolisant as a first-in-class molecule with a novel mechanism of action and potential new treatment option for patients living with narcolepsy. We also look forward to sharing data on patient and healthcare professional perceptions on the burden of narcolepsy, a rare and chronic debilitating neurologic disorder of sleep-wake state instability that impacts as many as 200,000 Americans.”

Data being presented by Harmony includes:

Oral Presentation: Long-Term Evaluation of Safety and Efficacy of Pitolisant in Narcolepsy: HARMONY 3 (Oral Presentation, May 9, 2:28 p.m., by Jeffrey Dayno, M.D.) Y Dauvilliers, I Arnulf, Z Szakács, C Scart-Grès, I Lecomte, C Caussé, J-C Schwartz.

Harmony III is an open-label, long-term (up to 5 years) study of pitolisant, a highly selective histamine 3 (H3) receptor antagonist/inverse agonist, in patients with narcolepsy. This presentation will focus on findings from a 1-year analysis that assessed the long-term safety and efficacy of pitolisant in the treatment of excessive daytime sleepiness (EDS) and cataplexy in patients with narcolepsy.

Poster Presentation: Efficacy and Safety of Pitolisant in Patients with Narcolepsy: A Review of Clinical Trials (Poster 6-034, May 7, 5:30-6:30 p.m.) Y Dauvilliers, J-C Schwartz, C Davis, J Dayno.

Results from Harmony 1 and Harmony CTP, two randomized, double-blind, placebo-controlled pivotal studies that assessed the efficacy and safety of pitolisant for the treatment of EDS (Harmony 1) and cataplexy (Harmony CTP) in adult patients with narcolepsy, will be included in this presentation.

Poster Presentation: Burden of Narcolepsy: A Survey of Patients and Physicians (Poster 6-036, May 7, 5:30-6:30 p.m.) M Thorpy, J Hopper, A Patroneva.

This presentation will report results from a survey on perceptions of healthcare professionals and patients living with narcolepsy regarding the burden, symptoms and treatment of narcolepsy. Discrepancies between patient and healthcare professional responses will also be described.