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Harmony Biosciences Announces Topline Results From Phase 2 Pitolisant Study

Results from Phase 2 Proof-of-Concept Study Evaluating Pitolisant for Excessive Daytime Sleepiness in Patients with Prader-Willi Syndrome

Today, Harmony Biosciences announced topline data on the primary outcome, excessive daytime sleepiness, from their Phase 2 proof-of-concept trial in patients with Prader-Willi syndrome, or PWS.

The study included 65 volunteers, ages 6 to 65, who were randomized 1:1:1 to low-dose pitolisant, high-dose pitolisant, or placebo treatment groups. Overall, patients' daytime sleepiness, as measured on the ESS-CHAD Parent/Caregiver Version improved by 3.7 to 5.5 points, representing a clinically meaningful change. 

These results represent the initial topline data with the full data set expected before the end of the year. The full data set will include the results on the secondary outcomes, including caregiver and clinician global impression scores, as well as measurements of behavioral symptoms, cognitive function, and hyperphagia

In a community-facing statement, Harmony shared: "We are encouraged by this initial data, which will inform our plans to advance our clinical development program of pitolisant in patients with PWS.  The entire team at Harmony Biosciences would like to sincerely thank the people with PWS, their caregivers and families, and the clinical trial site personnel who participated in our study. We are grateful for their time and willingness to participate in clinical research."

For more information, please see Harmony's full press release.

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Topics: Research

Susan Hedstrom


Susan Hedstrom is the Executive Director for the Foundation for Prader-Willi Research. Passionate about finding treatments for PWS, Susan joined FPWR in 2009 shortly after her son, Jayden, was diagnosed with Prader-Willi Syndrome. Rather than accepting PWS as it has been defined, Susan has chosen to work with a team of pro-active and tireless individuals to accelerate PWS research in order to change the future of PWS. Inspired by her first FPWR conference and the team of researchers that were working to find answers for the syndrome, she joined the FPWR team in 2010 and led the development of the One SMALL Step walk program. Under Susan’s leadership, over $15 million has been raised for PWS related research.