Zevra Therapeutics Announces Submission of IND for KP1077 in Narcolepsy

Zevra Therapeutics, Inc. recently announced the submission of an Investigational New Drug (IND) application seeking authorization from the US FDA to begin a Phase 1 clinical trial of KP1077 in narcolepsy. Once the clinical investigation plan proposed in the IND has been cleared to proceed by the FDA, Zevra plans to initiate its first of several Phase 1 clinical trials of KP1077 as early as the second quarter of 2023.

KP1077 is also currently being evaluated in a Phase 2 clinical trial for the treatment of idiopathic hypersomnia (IH). Those data have the potential to support not only the advancement of KP1077 into a pivotal Phase 3 study in IH, but also a Phase 3 trial in narcolepsy.

“Our research of KP1077 in IH laid the groundwork for us to expand our clinical studies to examine its efficacy in narcolepsy, a chronic neurological sleep disorder,” said Richard W. Pascoe, Chief Executive Officer of Zevra. “We are committed to advancing therapies for rare disorders, and our work to address narcolepsy and the treatment of excessive daytime sleepiness with KP1077 is an essential part of our commitment. Expanding existing trials in ways that support the advancement of therapies that the rare disease community needs is the core of Zevra’s mission.”

Zevra filed an IND application in May 2022 for the treatment of IH with KP1077 and subsequently initiated a Phase 2 IH study in December 2022 (NCT05668754). During a pre-IND meeting with the FDA, Zevra received confirmation that additional non-clinical studies were not needed to advance KP1077 into clinical development due to the abundance of data already available on serdexmethylphenidate (SDX). SDX is the sole active pharmaceutical ingredient in KP1077.

Zevra Therapeutics is a rare disease company melding science, data, and patient need to create transformational therapies for diseases with limited or no treatment options. With unique, data-driven clinical, regulatory, and commercialization strategies, the company is overcoming complex drug development challenges to bring much-needed therapies to patients.

Arimoclomol, Zevra’s orally delivered, first-in-class investigational product candidate for the treatment of Niemann-Pick disease type C (NPC), has been granted orphan drug designation, Fast Track designation, Breakthrough Therapy designation and rare pediatric disease designation for NPC by the US FDA, and orphan medicinal product designation for the treatment of NPC by the European Medicines Agency (EMA).

KP1077 is Zevra’s lead clinical candidate being developed to treat idiopathic hypersomnia (IH) and narcolepsy. KP1077 is composed solely of serdexmethylphenidate (SDX), Zevra’s proprietary prodrug of d-methylphenidate (d-MPH). The FDA has granted KP1077 orphan drug designation for the treatment of IH, and the US Drug Enforcement Agency (DEA) has classified SDX as a Schedule IV controlled substance based on evidence suggesting SDX has a lower potential for abuse when compared to d-MPH, a Schedule II controlled substance. For more information, visit,  zevra.com.