Pitolisant could represent the first new treatment for cataplexy in patients with narcolepsy in over a decade
PLYMOUTH MEETING, PA, May 31, 2018 — Harmony Biosciences, LLC (Harmony), a Paragon Biosciences, LLC, portfolio company, today announced that it will present scientific data on its investigational product, pitolisant, which is being studied for the treatment of excessive daytime sleepiness (EDS) and cataplexy in patients with narcolepsy, at the upcoming 32nd Annual Meeting of the Associated Professional Sleep Societies, known as "SLEEP", in Baltimore, MD from June 3-6.
“We are pleased to present data from the clinical development program for pitolisant to the U.S. physician audience, which demonstrate the potential utility of pitolisant in treating patients with narcolepsy who experience symptoms of both EDS and cataplexy,” said Harmony’s Chief Medical Officer, Jeffrey Dayno, M.D. “Pitolisant is a first-in-class molecule with a novel mechanism of action that works as a selective histamine H3-receptor antagonist/inverse agonist and enhances histaminergic transmission in the brain. Histamine is important because it is thought to play a role in sleep-wake state stability which, in patients with narcolepsy, is disrupted, resulting in cataplexy and other manifestations of REM sleep intruding into wakefulness.”
Data that Harmony will be presenting include:
Poster Presentation: Anticataplectic Efficacy of Pitolisant, The First Potent and Highly Selective Histamine H3-Receptor Antagonist/Inverse Agonist (Poster Session P12, June 3, 5-7 PM) M Thorpy, J Schwartz, J Lecomte, C Caussé , JM Dayno.
Poster Presentation: Long-Term Evaluation of Safety and Efficacy of Pitolisant in Narcolepsy: Harmony III Study (Poster Session P12, June 3, 5-7 PM) Y Dauvilliers, I Arnulf, Z Szakács, C Scart-Grès, I Lecomte , C Caussé, J Schwartz.
Harmony Biosciences announced on May 21, 2018 that the company received Breakthrough Therapy and Fast Track designations for pitolisant from the U.S. Food and Drug Administration. Breakthrough Therapy designation was granted for the treatment of cataplexy in patients with narcolepsy and Fast Track designation was granted for the investigation of pitolisant for the treatment of excessive daytime sleepiness in patients with narcolepsy and the treatment of cataplexy in patients with narcolepsy. Harmony, in partnership with Bioprojet, plans to submit a New Drug Application for pitolisant to the FDA in 2018 with the goal of bringing this new treatment to those in the U.S. living with the serious, debilitating neurologic disease of narcolepsy in 2019.
Pitolisant is an investigational medication in the U.S. that is not currently approved by the FDA. It received orphan designation by the FDA for the treatment of narcolepsy. Pitolisant is the first selective histamine H3-receptor antagonist/inverse agonist; it enhances the activity of histaminergic neurons in the brain, which function to improve a patient’s wakefulness and inhibit attacks of cataplexy. It has been developed by Bioprojet which has marketed the product in Europe since its approval by the European Medicines Agency in 2016. If approved, pitolisant would represent the first new therapy in the U.S. in over a decade for the treatment of both excessive daytime sleepiness and cataplexy in patients with narcolepsy.
Harmony Biosciences has initiated the Pitolisant Expanded Access Clinical Evaluation (PEACE) program, an open-label Expanded Access Program (EAP) intended to provide treatment with pitolisant to adult patients in the U.S. with excessive daytime sleepiness associated with narcolepsy with or without cataplexy. It is open to all qualified healthcare professionals who wish to participate and interested patients who meet the inclusion/exclusion criteria of the protocol. For more information on the PEACE program, visit www.thepeacenarcolepsyprogram.com.
Narcolepsy is a rare, chronic, debilitating neurologic disease of sleep-wake state instability that impacts as many as 200,000 Americans. It is characterized by excessive daytime sleepiness and elements of rapid eye movement (REM) sleep (e.g., cataplexy, sleep paralysis, hallucinations) intruding into wakefulness. In most patients, it is caused by the loss of hypocretin, a neuropeptide in the brain that supports sleep-wake state stability. The disease affects men and women equally with typical symptom onset in adolescence or young adulthood; however, the disease can take up to a decade to be properly diagnosed. Narcolepsy can cause significant burden on patients and their families, affecting their ability to perform routine tasks, limit achievement at school and work, impact social relationships and cause impairment in overall quality of life.
Cataplexy is one of several symptoms of narcolepsy that represent elements of REM sleep state intruding into wakefulness, characterized by sudden temporary loss of muscle tone. Cataplexy can be subtle, such as drooping of eyelids, or severe, such as knee buckling or total body collapse. Often times, symptoms of cataplexy may go unrecognized for a while because of the subtle nature of the symptoms in some patients, variability of how cataplexy is expressed, and/or lack of patient complaints or physician recognition of the symptoms as manifestations of cataplexy. This symptom of narcolepsy can often cause significant impact on a person’s ability to carry out normal daily functions. Up to two-thirds of all narcolepsy patients have Type 1 narcolepsy with cataplexy, one of the most debilitating symptoms of this chronic, rare neurologic disease.
Harmony Biosciences, LLC
Harmony Biosciences, LLC, is a privately-owned biopharmaceutical company headquartered in Plymouth Meeting, PA. The company was established in October 2017, with a vision to provide novel treatment options for people living with rare and orphan diseases with an emphasis on central nervous system disorders, starting with patients who live with narcolepsy. Harmony Biosciences is committed to advancing the understanding of narcolepsy and providing information and resources to individuals who live with, and healthcare professionals who treat patients with narcolepsy. On February 28, 2018, National Rare Disease Awareness Day, Harmony Biosciences launched Know Narcolepsy™, an educational campaign to help increase understanding of the disease and provide a new resource for the community. KnowNarcolepsy.com includes information on narcolepsy and cataplexy as well as powerful patient stories about living with the disease. Harmony Biosciences received the 2017 Deal of the Year Award from Life Sciences PA. For more information on Harmony Biosciences, visit www.harmonybiosciences.com.
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