RARE Daily

Harmony Biosciences Adds Novel Asset to Rare Neurological Disease Pipeline

August 9, 2021

Harmony Biosciences has acquired HBS-102, a potential first-in-class molecule with a novel mechanism of action, from rare disease focused biotech ConSynance Therapeutics, for the treatment of narcolepsy and other rare neurological diseases.

Photo: Jeffrey Dayno, chief medical officer of Harmony

Under the terms of the agreement, Harmony will acquire full development and commercialization rights globally, with the exception of Greater China, with financial terms including an upfront payment of $3.5 million and potential development and regulatory milestone payments and royalties.

“The acquisition of HBS-102 represents our first addition to the pipeline beyond WAKIX (pitolisant), and our intention is to continue to pursue additional assets in line with our vision of becoming a leading rare neurological disease company with a robust portfolio of products,” said John Jacobs, president and CEO of Harmony.

Narcolepsy is a rare, chronic, debilitating neurological disease of sleep-wake state instability that impacts approximately 165,000 Americans and is primarily characterized by excessive daytime sleepiness (EDS) and cataplexy—its two cardinal symptoms—along with other manifestations of REM sleep dysregulation (hallucinations and sleep paralysis), which intrude into wakefulness. EDS is the inability to stay awake and alert during the day and is the symptom that is present in all people living with narcolepsy. In most patients, narcolepsy is caused by the loss of hypocretin/orexin, a neuropeptide in the brain that supports sleep-wake state stability. This disease 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. 

HBS-102 is a melanin concentrating hormone receptor 1 (MCHR1) antagonist that has the potential to offer a novel approach to the treatment of narcolepsy including the symptoms of Rapid Eye Movement (REM) sleep dysregulation, such as cataplexy, hallucinations, and sleep paralysis. HBS-102 blocks the activity of melanin concentrating hormone (MCH) neurons, which scientific evidence indicates is the generator of REM sleep and its associated behaviors. Therefore, HBS-102 could potentially reduce REM intrusions into wakefulness and reduce the frequency of cataplexy, hallucinations, and sleep paralysis.

In a preclinical proof-of-concept study, Thomas Scammell, professor in the Department of Neurology, Beth Israel Deaconess Medical Center and Division of Sleep Medicine, Harvard Medical School, and his team demonstrated that an MCHR1 antagonist molecule resulted in a significant reduction in cataplexy events in an orexin knockout mouse model of narcolepsy. Harmony will complete additional work to prepare and submit an Investigational New Drug application with the plan to initiate a phase 2 clinical trial.

“Similar to WAKIX, this asset offers Harmony another opportunity to lead with the science and potentially bring another first-in-class treatment option to patients living with narcolepsy and other rare neurological diseases,” said Jeffrey Dayno, chief medical officer of Harmony. “The majority of people living with narcolepsy experience symptoms of REM dysregulation that have a significant impact on their lives. The acquisition and successful development of HBS-102 could represent a next-generation therapy for narcolepsy patients by offering a novel approach, directly targeting the control center for REM sleep and its associated behaviors.”

WAKIX was approved by the U.S. Food and Drug Administration in 2019 for the treatment of excessive daytime sleepiness or cataplexy in adult patients with narcolepsy.

Author: Rare Daily Staff

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