HARMONY BIOSCIENCES ANNOUNCES TOPLINE RESULTS FROM PHASE 2 PROOF-OF-CONCEPT STUDY EVALUATING PITOLISANT FOR EXCESSIVE DAYTIME SLEEPINESS IN PATIENTS WITH PRADER-WILLI SYNDROME
Harmony Biosciences Holdings, Inc. ("Harmony") (Nasdaq: HRMY), a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients with rare neurological diseases, today announced initial topline results from its Phase 2 proof-of-concept study in patients with Prader-Willi syndrome (PWS), which showed a positive signal on improvement in the primary outcome related to excessive daytime sleepiness (EDS)
PLYMOUTH MEETING, Pa., Nov. 1, 2022 /PRNewswire/ -- Harmony Biosciences Holdings, Inc. ("Harmony") (Nasdaq: HRMY), a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients with rare neurological diseases, today announced initial topline results from its Phase 2 proof-of-concept study in patients with Prader-Willi syndrome (PWS), which showed a positive signal on improvement in the primary outcome related to excessive daytime sleepiness (EDS).
The Phase 2 clinical trial was a randomized, double-blind, placebo-controlled study designed to assess the safety and efficacy of pitolisant in patients with PWS. This proof-of-concept study was not powered to demonstrate statistical significance and was designed for signal detection. The study included patients ages 6 to 65 years and patients were randomized 1:1:1 to low dose pitolisant, high dose pitolisant, or placebo treatment groups. Pitolisant dosing was based on three age cohorts (children 6 to < 12; adolescents 12 to < 18; and adults 18 to 65) and another objective of the study was to evaluate for a dose-response to pitolisant in patients with PWS. The primary endpoint of this study was the evaluation of EDS as measured by change from baseline to end of treatment (EOT) on the Epworth Sleepiness Scale for Children and Adolescents (ESS-CHAD) Parent/Caregiver Version.
Topline study results include:
