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.