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NIH awards grant to develop a new drug to treat narcolepsy

Reset Therapeutics, the leader in harnessing the power of the body’s internal clocks to develop therapeutics, has been awarded a five-year cooperative agreement by the National Institutes of Health (NIH) to develop a new drug to treat narcolepsy.  The award was made through the NIH Blueprint Neurotherapeutics Network, which was established in order to bridge the gap in neuroscience-related drug development between academic and industry research.

Narcolepsy affects nearly 500,000 people in the United States, Europe, and Japan. The majority of narcolepsy patients also experience cataplexy (brief but debilitating episodes of sudden-onset muscle weakness). None of the drugs marketed for narcolepsy address the underlying cause of the disease and full restoration of alertness remains a challenge for many narcolepsy patients. Additionally, agents approved for treating the symptoms of narcolepsy typically have limited duration of action and are associated with unwanted side effects. Reset is leveraging its expertise and leadership in circadian rhythms biology to develop orexin receptor agonists. Orexin proteins play a key role in the body’s normal sleep/wake cycles. This approach is designed to address the molecular cause of narcolepsy.

Orexin is expressed in a part of the brain that serves as the “on/off” switch for the body’s internal clocks. The neurons that normally express orexin are missing in patients with narcolepsy, possibly due to autoimmune mechanisms. Consequently, narcolepsy patients have dramatically reduced levels of orexin A protein in their blood and spinal fluid, and 90% of narcolepsy patients have orexin deficiency.

Reset is developing orexin receptor agonists that are designed to mimic the effects of the orexin protein and restore normal balance to the body’s sleep/wake cycles. Daily morning administration of an orexin receptor agonist is expected to prevent the daytime sleepiness and cataplexy that are typical of narcolepsy.

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Date Created: January 8th, 2013
Last Updated: January 8th, 2013