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Long-term safety and tolerability of pitolisant and durability of effect in both EDS and cataplexy in patients with narcolepsy were evaluated out to five years

Patients with EDS and cataplexy in narcolepsy in the U.S. receive treatment in an open-label expanded access program

PLYMOUTH MEETING, PA, September 5, 2018 — Harmony Biosciences, LLC (Harmony) today announced that it will present scientific data on its investigational product, pitolisant, which has been studied for the treatment of both excessive daytime sleepiness (EDS) and cataplexy in adult patients with narcolepsy, at the upcoming 7th International Symposium on Narcolepsy in Beverly, MA from September 9-13.

“We are pleased to have the opportunity to present new data for pitolisant from a long-term study, which adds to the growing body of evidence from the clinical development program. These data further demonstrate the potential of pitolisant as a first-in-class molecule with a novel mechanism of action for patients living with narcolepsy,” said Harmony’s Chief Medical Officer, Jeffrey Dayno, M.D. “In addition, our expanded access program for pitolisant provides treatment to adult patients in the U.S. with EDS and cataplexy associated with narcolepsy, a rare disorder with significant unmet need.”

Data that Harmony will be presenting include:

• Oral Presentation: An Open-Label, Naturalistic Study to Assess the Long-Term Safety of Pitolisant in Adult Patients with Narcolepsy With or Without Cataplexy: Results from the Harmony III Study Five Year Extension (Oral Presentation, Sept. 11, 9 AM) JM Dayno
  • Harmony III study evaluated the long-term (up to 5 years) safety and tolerability of pitolisant in an open-label naturalistic study that included patients who were both naïve and experienced on pitolisant therapy. This presentation will include findings from the French cohort from this trial that extended out to 5 years. Long-term safety/tolerability data will be presented, along with open-label data on the durability of effect of pitolisant over time on EDS and symptoms of REM dysregulation such as cataplexy, hypnagogic hallucinations and sleep paralysis.
• Poster Presentation: Pitolisant Treatment of Excessive Daytime Sleepiness and Cataplexy in Patients with Narcolepsy: an Open Label, Expanded Access Program in the United States (PEACE) (Poster #41 , Sept. 12, 6 PM) M Thorpy, E Bauer, C Davis, A Patroneva, JM Dayno.
  • This presentation will describe the design and methodology of the program, as well as report out initial data, including patient demographics, baseline characteristics and safety and tolerability results.

The Pitolisant Expanded Access Clinical Evaluation (PEACE) program is 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.

About Pitolisant

Pitolisant is an investigational medication in the U.S. that is not currently approved by the FDA. It was granted orphan designation for the treatment of narcolepsy, Fast Track designation for the treatment of excessive daytime sleepiness (EDS) and cataplexy in patients with narcolepsy, and Breakthrough Therapy designation for the treatment of cataplexy in patients with narcolepsy. Pitolisant is the first selective histamine 3 (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 was developed by Bioprojet which has marketed the product in Europe since its approval by the European Medicines Agency in 2016. The receipt of Breakthrough Therapy and Fast Track designations for pitolisant afforded Harmony the opportunity to request a rolling NDA submission to the FDA, which was granted. Harmony’s goal is to obtain FDA approval to market this new medication in the U.S. in 2019. If approved, pitolisant would represent the first new therapy in the U.S. in over a decade for the treatment of excessive daytime sleepiness (EDS) in patients with narcolepsy, and in fifteen years for the treatment of cataplexy in patients with narcolepsy.

About Narcolepsy

Narcolepsy is a rare, chronic, debilitating neurologic disorder of sleep-wake state instability that impacts up to 200,000 Americans and is primarily characterized by excessive daytime sleepiness, cataplexy, and other manifestations of REM sleep dysregulation, which intrude into wakefulness. In most patients, it is caused by the loss of hypocretin, a neuropeptide in the brain that supports sleep-wake state stability. This disorder affects men and women equally with typical symptom onset in adolescence or young adulthood; however, it 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.

About Cataplexy

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 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 cataplexy (known as Type 1 narcolepsy); cataplexy is one of the most debilitating symptoms of this chronic, rare neurologic disorder.

Harmony Biosciences, LLC

Harmony Biosciences, LLC, is a private 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 living with narcolepsy. Harmony Biosciences is committed to advancing the understanding of narcolepsy and providing information and resources to individuals who live with narcolepsy and healthcare professionals who treat patients with this disorder. 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 about 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.

Harmony Biosciences, LLC Media Contact:
Nancy Leone
215-891-6046
nleone@harmonybiosciences.com