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 initiation of a Phase 2 clinical trial to evaluate the safety and efficacy of pitolisant for excessive daytime sleepiness (EDS) and other non-muscular symptoms in adult patients with type 1 myotonic dystrophy (DM1).
“In addition to the primary symptoms of myotonia and muscle weakness in patients with type 1 myotonic dystrophy, the non-muscular symptoms of excessive daytime sleepiness, fatigue, and cognitive dysfunction are very common in these patients and have a negative impact on daily functioning as much as, or more so, than the primary muscle symptoms of the disease,” said Harmony’s Chief Medical Officer, Jeffrey Dayno, M.D. “Pitolisant’s novel mechanism of action increases histamine transmission in the brain which provides the scientific rationale for its potential clinical utility for the common non-muscular symptoms in patients with DM1. We listened to the needs of patients and caregivers in the myotonic dystrophy patient community and are pleased to have initiated this Phase 2 clinical trial to assess the potential clinical benefit of pitolisant in patients with this rare neurological disease, for which there are currently no approved treatment options.”
Myotonic dystrophy is the most common form of adult-onset muscular dystrophy. It is a genetic disorder inherited in an autosomal-dominant pattern. Latest estimates suggest a prevalence of about one per 2,100 people with the genetic defect for DM1, which is the most common form of this disorder. This equates to about 160,000 people in the U.S. with the genetic defect for DM1. Estimates suggest there are 40,000 people currently diagnosed with DM1 in the U.S., with up to 90% of them reporting EDS and fatigue and over 60% of them experiencing cognitive dysfunction.
The Phase 2 clinical trial is a randomized, double-blind, placebo-controlled study designed to assess the safety and efficacy of pitolisant in patients with DM1 ages 18 – 65 years. Approximately 135 patients will be randomized at baseline to low-dose pitolisant, high-dose pitolisant, or placebo in a 1:1:1 treatment ratio titrated over three weeks, followed by eight weeks of stable dosing. Patients who complete the randomized, controlled phase of the trial will be eligible to participate in an open-label extension phase to assess the long-term safety and effectiveness of pitolisant in patients with DM1.
The primary objective of the trial is to evaluate the effect of pitolisant compared with placebo on EDS. Secondary objectives include assessments of fatigue, specific measures of cognitive function using validated computer-based assessments, and overall disease burden utilizing a disease-specific, patient-reported outcomes instrument for DM1. Topline results are anticipated from the Phase 2 trial in the second half of 2022.
Pitolisant is marketed as WAKIX® in the U.S. for the treatment of EDS or cataplexy in adult patients with narcolepsy.
