Harmony Biosciences’ pitolisant shows behavior improvement in PWS

Harmony Biosciences announced the presentation of new secondary endpoint data, including improvements in behavioral disturbances, from its Phase 2 signal-detection study evaluating pitolisant for the treatment of excessive daytime sleepiness – EDS – in Prader-Willi syndrome – PWS -, at the 2023 Foundation for Prader-Willi Research Symposium and Family Conference. The poster presentation of secondary outcomes data included improvements in behavioral symptoms, especially in the higher-dose pitolisant group. Reductions in EDS severity were also observed as were some improvements in hyperphagia. The overall rate of adverse events was similar for pitolisant and placebo, and the safety/tolerability profile was consistent with the known profile for pitolisant. The results from the Phase 2 signal-detection study informed the protocol design for the upcoming Phase 3 registrational TEMPO study, expected to be initiated in Q4. There are currently 15,000 – 20,000 people in the US living with PWS. Reductions in behavioral disturbances among the youngest age group (6 to less than12 years) were observed across all ABC-2 domains especially in the higher-dose pitolisant group. Reductions in CaGI-S scores were greater for pitolisant compared with placebo in the children. Pitolisant is FDA approved to treat EDS or cataplexy in adult patients with narcolepsy.