Harmony Biosciences Presents Promising Data on Pitolisant for Myotonic Dystrophy Type 1

Harmony Biosciences Holdings

PLYMOUTH MEETING, PAHarmony Biosciences (Nasdaq: HRMY) recently unveiled promising data at the annual meeting of the Associated Professional Sleep Societies, “SLEEP 2024.” The data from a Phase 2 signal detection study suggests that pitolisant may reduce excessive daytime sleepiness (EDS) and fatigue in adults with Myotonic Dystrophy Type 1 (DM1).

“More than 80 percent of DM1 patients experience EDS and fatigue, which patient-reported outcomes research has shown to be nearly as debilitating as the primary symptoms of DM1, namely myotonia and muscle weakness,” said Dr. Kumar Budur, Chief Medical and Scientific Officer of Harmony Biosciences. “The findings from our study are encouraging and point to the potential of pitolisant in treating EDS and fatigue in DM1, as well as other sleep/wake disorders.”

The study showed greater improvement in both EDS and fatigue by Week 11 for patients taking pitolisant compared to those on a placebo. Improvements were measured using the Daytime Sleepiness Scale and the Fatigue Severity Scale. Notably, higher doses of pitolisant demonstrated stronger efficacy signals. Although this study was designed for signal detection and not powered to show statistical significance, the results are promising.

Approximately 40,000 people in the U.S. are diagnosed with DM1, with up to 90% of them reporting EDS and fatigue. These symptoms significantly impact their quality of life.

“Given the potential of pitolisant for treating EDS and fatigue in DM1 patients, we plan to move forward with a pivotal Phase 3 study using the Next-Generation 2 (NG2) formulation of pitolisant,” Budur added. “The NG2 formulation is designed to deliver an optimized pharmacokinetic profile and higher dosage strength. These efforts could ultimately help over 100,000 patients living with unmet medical needs.”

READ:  Bridge Cable Set to Attend ADI Expo and Explore New Telecommunications Trends

Myotonic Dystrophy Type 1 is the most common form of adult-onset muscular dystrophy. It is a genetic disorder inherited in an autosomal-dominant pattern, affecting about one in 2,100 people. This equates to roughly 150,000 individuals in the U.S. with the genetic defect for DM1.

Harmony Biosciences’ WAKIX® (pitolisant) Tablets are already approved in the U.S. for treating excessive daytime sleepiness or cataplexy in adults with narcolepsy. WAKIX has been commercially available since late 2019 and holds orphan drug designation for narcolepsy treatment. The drug’s mechanism involves acting on histamine 3 (H₃) receptors, increasing the release of histamine, which promotes wakefulness.

Pitolisant’s potential to alleviate EDS and fatigue in DM1 patients marks a significant step forward in addressing the complex symptoms associated with this genetic disorder. Harmony Biosciences’ ongoing research and development efforts continue to focus on meeting the needs of patients with sleep and wakefulness disorders.

For the latest news on everything happening in Chester County and the surrounding area, be sure to follow MyChesCo on Google News and Microsoft Start.