KP1077 Phase 2 Trial Results for Treating Idiopathic Hypersomnia

Zevra Therapeutics, a company specializing in rare disease treatments, has reported preliminary results from a Phase 2 clinical trial of a drug called KP1077. The study focused on its effects on patients with idiopathic hypersomnia (IH), a rare sleep disorder characterized by excessive daytime sleepiness. The trial was a double-blind, placebo-controlled, randomized-withdrawal design that assessed the drug's safety, tolerability, and efficacy.

The drug, also known as serdexmethylphenidate or SDX, showed promising results in improving IH symptoms such as sleep inertia and brain fog, in addition to excessive sleepiness. The study involved a five-week dose optimization phase and a subsequent two-week double-blind phase. The drug was well-tolerated at all tested doses, with the most common side effects being insomnia, headache, anxiety, nausea, and decreased appetite.

Dr. Christopher Drake, the study's principal investigator, highlighted the drug's impact on IH symptom severity and maintained improvements during the trial's phases. Zevra's CEO, Neil McFarlane, expressed gratitude to the participants and emphasized the potential of KP1077 to provide a differentiated treatment option for IH patients.

The positive results from the Phase 2 trial support the initiation of a Phase 3 study, with Zevra planning to seek guidance from the U.S. Food and Drug Administration (FDA) on the design of this pivotal trial. The FDA has granted Orphan Drug Designation to KP1077 for the treatment of IH, and the Drug Enforcement Agency (DEA) has classified its active ingredient as a Schedule IV controlled substance due to its lower abuse potential.

Idiopathic hypersomnia affects an estimated 37,000 diagnosed patients in the United States, though the actual number may be higher due to undiagnosed or misdiagnosed cases. Zevra Therapeutics aims to develop transformational therapies for rare diseases, using a data-driven approach to overcome drug development challenges.

The company's mission is to bring life-changing treatments to those living with rare diseases, and the Phase 2 results for KP1077 represent a significant step towards achieving this goal for IH patients. The final results of the trial are expected to be presented at the SLEEP 2024 annual meeting, offering further insights into the drug's potential as a treatment for this debilitating condition.