Harmony Biosciences Highlights Publication of WAKIX® (pitolisant) Efficacy Data in Sleep Medicine

Efficacy of pitolisant evaluated in adult patients with a high burden of narcolepsy symptoms

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