PLYMOUTH MEETING, PA — Harmony Biosciences Holdings, Inc. (Nasdaq: HRMY) has recently unveiled positive topline results from its Phase 2 signal detection study. The study evaluated the safety and efficacy of pitolisant, a medication used to treat excessive daytime sleepiness (EDS) and cataplexy in adults with narcolepsy, in adult patients diagnosed with myotonic dystrophy type 1 (DM1).
The primary efficacy endpoint showed a clinically meaningful improvement in EDS as measured by the Daytime Sleepiness Scale (DSS). A secondary efficacy endpoint also demonstrated significant improvement in fatigue as measured by the Fatigue Severity Scale (FSS). Both EDS and fatigue affect up to 80-90% of DM1 patients and significantly impact their daily functioning.
The topline results of the study include:
- Clinically meaningful improvements in EDS as measured by DSS, Epworth Sleepiness Scale (ESS), and Clinical Global Impression of Severity (CGI-S) of EDS.
- A clear and consistent dose-response was demonstrated, with the higher dose pitolisant group showing a greater response than the lower dose group across the study endpoints.
- The safety and tolerability profile in adult patients with DM1 was consistent with the established safety profile of pitolisant with no new safety signals detected and no serious adverse events reported.
“EDS is the most common non-muscular complaint in patients living with DM1, being present in the majority of patients,” said Dr. Jean-Denis Brisson, Centre Intégré Universitaire De Santé Et De Services Sociaux du Saguenay-Lac-Saint-Jean. “The positive signals from this Phase 2 study are very encouraging and support pitolisant as a potential treatment option for DM1.”
The Phase 2 signal detection study was a randomized, double-blind, placebo-controlled study in adults aged 18-65 with DM1. It was not powered to demonstrate statistical significance.
Further results from the trial, including findings from other secondary outcomes such as the Myotonic Dystrophy Health Index (MDHI), cognitive functions, and the safety and effectiveness of pitolisant from the ongoing Open-Label Extension (OLE) Phase will be shared early next year.
Pitolisant is marketed as WAKIX® in the U.S. and is FDA-approved to treat excessive daytime sleepiness or cataplexy in adults with narcolepsy. Pitolisant is not approved for use in patients with DM1 and is currently being evaluated as an investigational agent in this patient population.
For the latest news on everything happening in Chester County and the surrounding area, be sure to follow MyChesCo on Google News and MSN.