Harmony Biosciences has initiated a phase 3 trial evaluating the efficacy and safety of pitolisant in adult patients with idiopathic hypersomnia (IH), known as the INTUNE study. Pitolisant, also known by its brand name of WAKIX, a first-in-class medication approved by the US Food and Drug Administration for the treatment of excessive daytime sleepiness or cataplexy in adult patients with narcolepsy and has been commercially available in the United States since Q4 2019. 

“There has been much interest by both the patient community and healthcare professionals in pitolisant for the treatment of IH and we are excited about the initiation of our Phase 3 INTUNE study in adult patients with this rare neurological disorder,” says Harmony’s chief medical officer, Jeffrey Dayno, MD, in a release. “Given the novel mechanism of action of pitolisant, which works through histamine, an important wake-promoting neurotransmitter in the brain, we are looking to translate innovative science into therapeutic possibilities that have the potential to improve the health of people living with IH.”

IH is a rare and chronic neurological disease that is characterized by excessive daytime sleepiness (EDS) despite sufficient or even long sleep time. People living with IH experience significant EDS along with the symptoms of sleep inertia and brain fog. Based on insurance claims data, the number of patients diagnosed with IH in the United States ranges from 30,000 to 40,000.

“We are excited to initiate this Phase 3 trial and we are hopeful these efforts, if successful, will lead to Harmony seeking U.S. regulatory approval for the next indication for WAKIX,” says John C. Jacobs, president and CEO of Harmony Biosciences, in a release. “Patient needs drive our team’s drug development efforts and with this in mind, it is our goal to bring WAKIX forward to help the IH patient community as we have done for patients who are living with narcolepsy.”

The INTUNE study is a double-blind, placebo-controlled, randomized withdrawal study in approximately 200 IH patients which will be conducted at 60 to 80 clinical trial sites across the United States. The primary objective is to evaluate the efficacy and safety of pitolisant compared with placebo in treating excessive daytime sleepiness in adult patients with IH. Secondary objectives of the study include the impact of pitolisant on other important symptoms of IH, such as sleep inertia and cognitive impairment. Other outcomes, including patient impression of overall change in symptoms, investigator assessment of overall disease severity, and functional status will also be evaluated in the study.

“Our mission and values are centered around both supporting people with hypersomnias like IH and scientific discovery. We are excited Harmony is moving forward with a Phase 3 clinical trial in people with idiopathic hypersomnia, which may bring us one step closer to finding an important therapeutic advancement for a community with limited treatment options,” says David Burley, board chair at the Hypersomnia Foundation, in a release.

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