Harmony Biosciences LLC, a Paragon Biosciences LLC portfolio company, will present scientific data on its investigational product, pitolisant, which is being studied for the treatment of excessive daytime sleepiness (EDS) and cataplexy in patients with narcolepsy, at SLEEP 2018.

“We are pleased to present data from the clinical development program for pitolisant to the US physician audience, which demonstrate the potential utility of pitolisant in treating patients with narcolepsy who experience symptoms of both EDS and cataplexy,” says Harmony’s chief medical officer, Jeffrey Dayno, MD, in a release. “Pitolisant is a first-in-class molecule with a novel mechanism of action that works as a selective histamine H3-receptor antagonist/inverse agonist and enhances histaminergic transmission in the brain. Histamine is important because it is thought to play a role in sleep-wake state stability which, in patients with narcolepsy, is disrupted, resulting in cataplexy and other manifestations of REM sleep intruding into wakefulness.”

Data that Harmony will be presenting include:

Poster Presentation: Anticataplectic Efficacy of Pitolisant, The First Potent and Highly Selective Histamine H3-Receptor Antagonist/Inverse Agonist (Poster Session P12, June 3, 5-7PM) M Thorpy, J Schwartz, J Lecomte, C Caussé, JM Dayno.
In this report, the anticataplectic results of pitolisant are summarized from Phase III studies.

Poster Presentation: Long-Term Evaluation of Safety and Efficacy of Pitolisant in Narcolepsy: Harmony III Study (Poster Session P12, June 3, 5-7PM) Y Dauvilliers, I Arnulf, Z Szakács, C Scart-Grès, I Lecomte, C Caussé, J Schwartz.
The Harmony III study is assessing long-term safety and efficacy of pitolisant, the first potent and highly selective histamine H3-receptor antagonist/inverse agonist, in the treatment of patients with narcolepsy.

Harmony Biosciences announced earlier this month that it received Breakthrough Therapy and Fast Track designations for pitolisant from the US Food and Drug Administration. Breakthrough Therapy designation was granted for the treatment of cataplexy in patients with narcolepsy and Fast Track designation was granted for the investigation of pitolisant for the treatment of excessive daytime sleepiness in patients with narcolepsy and the treatment of cataplexy in patients with narcolepsy. Harmony, in partnership with Bioprojet, plans to submit a New Drug Application for pitolisant to the FDA in 2018 with the goal of bringing this new treatment to those in the U.S. living with the serious, debilitating neurologic disease of narcolepsy in 2019.