Harmony Biosciences has reported positive topline data from its Phase II clinical trial of pitolisant to treat adults with myotonic dystrophy type 1 (DM1).

The double-blind, randomised, placebo-controlled signal detection study analysed pitolisant in adult DM1 patients aged 18 to 65 years. 

It enrolled 30 subjects who were randomised into a 1:1:1 ratio to receive a lower or higher dose of pitolisant or placebo for three weeks. It was followed by stable dosing for eight weeks.

Findings showed a clinically meaningful improvement in excessive daytime sleepiness (EDS), a primary efficacy endpoint of the trial.

It also offered improvement in fatigue, a secondary efficacy endpoint. 

EDS and fatigue are reported in up to 80-90% of DM1 patients and affect their daily functioning.

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Furthermore, treatment with a higher dose of pitolisant offered a clear and uniform dose-response indicating an improved response than the lower dose across the trial endpoints.

The safety and tolerability profile was found to be in line with the proven pitolisant’s safety without any new safety signals reported.

Harmony Biosciences president and CEO Jeffrey Dayno said: “These strong topline results add to the body of evidence supporting the effectiveness of pitolisant for improving EDS. 

“In addition, a positive signal for pitolisant has been demonstrated for fatigue, suggesting it could be a potential new treatment option for this symptom as well. 

“We are encouraged by these results, which underscore our commitment to translating innovative science into therapeutic possibilities for patients living with unmet medical needs.”

Sold as WAKIX in the US, pitolisant is approved by the Food and Drug Administration (FDA) for the treatment of excessive daytime sleepiness or cataplexy in adults with narcolepsy. 

Harmony Biosciences chief medical officer Kumar Budur said: “These positive signals for pitolisant are promising news for the DM1 patient community, where 80% to 90% of the approximately 40,000 diagnosed DM1 patients in the US experience significant EDS and fatigue with no FDA-approved treatments available.

“The strong signals for pitolisant reinforce our confidence in it as a potential treatment option worthy of further study in patients with DM1.”