PLYMOUTH MEETING, Pa., March 17, 2021 /PRNewswire/ -- Harmony Biosciences Holdings, Inc. ("Harmony") (NASDAQ:HRMY), a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients living with rare neurological disorders who have unmet medical needs, today announced that results of a post-hoc analysis of data from two Phase 3 clinical trials of pitolisant, a histamine 3 (H3) receptor antagonist/inverse agonist, have been published online in Sleep Medicine. The analysis focused on patients in the trials who suffered from a high burden of narcolepsy symptoms, including both excessive daytime sleepiness (EDS) and cataplexy.
The publication, Efficacy of Pitolisant in Patients with High Burden of Narcolepsy Symptoms: Pooled Analysis of Short-Term, Placebo-Controlled Studies reports data pooled from two randomized, placebo-controlled, 7-or 8-week studies in adult patients with narcolepsy, in which pitolisant could be titrated to a maximum dose of 35.6 mg/day. Three independent patient subgroups were analyzed based on the following criteria for high burden of narcolepsy symptoms:
- High burden of EDS as defined by an Epworth Sleepiness Scale (ESS) baseline score ≥16 (n = 118);
- High burden of EDS as defined by a sleep latency ≤8 minutes on a Maintenance of Wakefulness Test (MWT) (n = 105); and,
- High burden of cataplexy as defined by ≥15 cataplexy attacks per week (n = 31).
Change from baseline to the end of treatment was evaluated for pitolisant compared with placebo in each patient subgroup.
The publication reports the following results:
- In the patients with a high burden of EDS as defined by ESS (pitolisant, n=60; placebo, n=58), least-squares mean change from baseline on the ESS was significantly greater for pitolisant (–6.1) compared with placebo (–2.3; p<0.001). In addition, significantly more patients were classified as treatment responders (ESS score reduction ≥3) in the pitolisant group (69 percent) compared with the placebo group (35.1 percent; p=0.001).
- In the patients with a high burden of EDS as defined by the MWT (pitolisant, n=59; placebo, n=46), increase in mean sleep latency on the MWT was significantly greater for pitolisant (6.9 minutes) compared with placebo (3.4 minutes; p=0.017).
- In the patients with a high burden of cataplexy (pitolisant, n=20; placebo, n=11), least-squares mean change in the weekly rate of cataplexy was significantly greater for pitolisant (–14.5; baseline, 23.9; final, 9.4) compared with placebo (–0.1; baseline, 23.1; final, 23.0; p=0.004).
- The adverse event profile from this analysis was consistent with the known safety profile for pitolisant; headache, nausea, and anxiety were the most common adverse events in pitolisant-treated patients.
"Narcolepsy is a chronic, debilitating neurological disorder characterized by sleep-wake state instability that can disrupt daily functioning, especially in patients who experience a high symptom burden," said Harmony's Chief Medical Officer, Jeffrey Dayno, M.D. "The findings reported in this publication highlight that WAKIX® (pitolisant) was efficacious for reducing the symptom burden for both EDS and cataplexy, even in patients with a high burden of the two most common symptoms in narcolepsy."
Pitolisant is marketed as WAKIX in the U.S. for the treatment of EDS or cataplexy in adult patients with narcolepsy.