Harmony Biosciences Holdings Inc (NASDAQ: HRMY) has started a Phase 3 study to assess the safety and efficacy of pitolisant. This drug could potentially treat idiopathic hypersomnia in adults. The company has named the study to investigate the therapy INTUNE.
According to the company’s CMO, Dr Jeffery Dayno, the company has realised the excitement around pitolisant by physicians and their patients. For this reason, Harmony Biosciences is glad to proceed to the next level of the trial. Dr Dayno adds that the company wants to leverage the drug as it impacts histamine, a neurotransmitter which promotes wakefulness to help patients with idiopathic hypersomnia.
Idiopathic hypersomnia affects thousands of Americans
Although idiopathic hypersomnia is a rare disease, it affects thousands worldwide. The condition causes patients to sleep excessively during the day even when they slept enough during the night.
Patients will often experience difficulty staying awake. They will also have brain fog characterised by poor alertness, attention and combination. Insurance claims indicate that between 30,000 and 40,000 people in the U.S alone have a diagnosis of idiopathic hypersomnia.
Harmony Biosciences’ CEO and President John C. Jacobs states that the company is excited to begin the study. It hopes to be successful as that would enable it to seek approval from U.S regulators. Jacobs states that their work is inspired by a need to help patients. Harmony Biosciences already found a treatment for narcolepsy and hope to achieve the same with idiopathic hypersomnia.
Objectives of the study
The study is a randomised, placebo-controlled, double-blind withdrawal trial. Researchers will assess 200 patients with idiopathic hypersomnia. Moreover, they will recruit patients from between 60 and 80 sites in the country.
Researchers have determined both the primary and secondary objectives of the trial. The primary purpose involves assessing the safety and efficacy of the drug compared to a placebo. Researchers also have several secondary objectives. For example, the company will evaluate how pitolisant could affect other symptoms of the disorders including cognitive impairment and inertia.
Other secondary objectives will be to estimate the drug’s effect on the patients, functional status and disease severity. The drug has already received approval to treat narcolepsy and cataplexy.