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HARMONY BIOSCIENCES ANNOUNCES POSITIVE PHASE 2 SIGNAL DETECTION STUDY EVALUATING PITOLISANT FOR EXCESSIVE DAYTIME SLEEPINESS IN PRADER-WILLI SYNDROME AT SLEEP 2023
Clinically meaningful improvements potentially address unmet medical need for treating excessive daytime sleepiness in patients with PWS Phase 3
About this update from Harmony Biosciences Holdings, Inc.
[{"type":"text","content":"Clinically meaningful improvements potentially address unmet medical need for treating excessive daytime sleepiness in patients with PWS\n Phase 3 registrational study anticipated in 2H 2023\n\n\nPLYMOUTH MEETING, Pa., June 6, 2023 /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 the presentation of safety and efficacy data from a Phase 2 proof-of-concept study evaluating pitolisant for the treatment of excessive daytime sleepiness (EDS) in people with Prader-Willi syndrome (PWS) at the 37th Annual Meeting of the Associated Professional Sleep Societies (APSS), known as \"SLEEP 2023.\" The positive signal supports further development to determine if pitolisant has the potential to address an unmet medical need for people with PWS experiencing EDS.\n\n \n \n \n \n \n \n\n \nNotably, both the high- and low-dose pitolisant treatment arms demonstrated greater mean improvement from baseline in the Epworth Sleepiness Scale for Children and Adolescents (ESS-CHAD, Parent / Caregiver version) scores in the overall patient population when compared to placebo. A dose-response was observed with a relatively higher response rate occurring in the high dose pitolisant group compared to the low dose pitolisant group. This proof-of-concept study was not powered to demonstrate statistical significance and was designed for signal detection.\nHarmony is working with the U.S. FDA to discuss the results from this study and to finalize the Phase 3 registrational study, which it expects to initiate in 2H 2023.\n\"The positive signal from our Phase 2 study represents a milestone in our quest to address the unmet medical need surrounding excessive daytime sleepiness in Prader-Willi syndrome, for which there is currently no approved treatment,\" said Kumar Budur, M.D., M.S., Chief Medical Officer at Harmony Biosciences. \"Given the lack of existing treatment options for people with Prader-Willi syndrome, these findings represent a step forward in our efforts to improve the quality of life of individuals living with this condition and our commitment to rigorously pursuing promising new indications for pitolisant beyond narcolepsy.\"\nAmong the 15,000 to 20,000 Americans living...