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HARMONY BIOSCIENCES ANNOUNCES POSITIVE TOPLINE DATA FROM PHASE 2 SIGNAL DETECTION STUDY EVALUATING PITOLISANT IN ADULT PATIENTS WITH MYOTONIC DYSTROPHY TYPE 1
Clinically meaningful improvements demonstrated in excessive daytime sleepiness and fatigue PLYMOUTH MEETING, Pa., Dec. 7, 2023 /PRNewswire/ -- Harmony
About this update from Harmony Biosciences Holdings, Inc.
[{"type":"text","content":"Clinically meaningful improvements demonstrated in excessive daytime sleepiness and fatigue\n\n\nPLYMOUTH MEETING, Pa., Dec. 7, 2023 /PRNewswire/ -- Harmony Biosciences Holdings, Inc. (\"Harmony\" or the \"Company\") (Nasdaq: HRMY) today announced positive topline results from its Phase 2 signal detection study evaluating the safety and efficacy of pitolisant in adult patients with myotonic dystrophy type 1 (DM1).\n\n \n \n \n \n \n \n\n \nA clinically meaningful improvement was demonstrated on the primary efficacy endpoint, the change from baseline to the end of the double-blind period for excessive daytime sleepiness (EDS) as measured by the Daytime Sleepiness Scale (DSS). A clinically meaningful improvement was also demonstrated for fatigue, a secondary efficacy endpoint measured by the Fatigue Severity Scale (FSS). EDS and fatigue occur in up to 80-90% of patients with DM1 and impact daily functioning as much as muscular symptoms (myotonia and muscle weakness). \n\"These strong topline results add to the body of evidence supporting the effectiveness of pitolisant for improving EDS,\" said Jeffrey M. Dayno, M.D., President and CEO of Harmony Biosciences. \"In addition, a positive signal for pitolisant has been demonstrated for fatigue, suggesting it could be a potential new treatment option for this symptom as well. We are encouraged by these results, which underscore our commitment to translating innovative science into therapeutic possibilities for patients living with unmet medical needs.\"\nTopline results include:\nClinically meaningful improvements were demonstrated in EDS as measured by DSS, Epworth Sleepiness Scale (ESS), and Clinical Global Impression of Severity (CGI-S) of EDS.On DSS, the mean change from baseline to end of double-blind period was -2.5 and -1.0 for the higher dose and lower dose pitolisant treatment groups, respectively, compared to -0.2 for placebo.\nOn ESS, the mean change from baseline to end of double-blind period was -4.88 for the higher dose pitolisant treatment group compared to -0.10 for placebo.\nOn CGI-S, the mean change from baseline to end of double-blind period was -0.9 for the higher dose pitolisant treatment group compared to -0.1 for placebo.\nClinically meaningful improvements were demonstrated for fatigue as measured by the Fatigue Severity Scale (FSS).On FSS, the mean change from ...