Zevra Therapeutics Reports Positive Phase 2 Results for KP1077 in Idiopathic Hypersomnia at SLEEP 2024

Zevra Therapeutics has announced promising results from its Phase 2 clinical trial of KP1077, also known as serdexmethylphenidate (SDX), for the treatment of idiopathic hypersomnia (IH). The study, presented at the SLEEP 2024 conference in Houston, Texas, demonstrated both the safety and tolerability of KP1077 and its significant clinical benefits in patients with IH.

Key findings from the trial revealed that KP1077 was well tolerated at all dose levels, including the highest daily dose of 320 mg. Common adverse events (AEs) included insomnia, headaches, anxiety, decreased appetite, and nausea. These AEs were generally mild and occurred mainly during the titration period, without causing early discontinuation of the treatment.

The trial, which was placebo-controlled and double-blind, also highlighted significant improvements in various patient-reported outcomes. These included reductions in excessive daytime sleepiness (EDS), sleep inertia, and brain fog. Specifically, patients experienced meaningful decreases in their Epworth Sleepiness Scale (ESS) scores, IH Severity Scale (IHSS) scores, Sleep Inertia Visual Analog Scale (SIVAS) scores, and Brain Fog Scale (BFS) scores.

Throughout the trial, patients receiving KP1077 exhibited a notable drop in ESS scores by approximately nine points over the five-week open-label treatment period. This improvement was sustained during the subsequent two-week double-blind withdrawal phase. The mean reductions in ESS, IHSS, SIVAS, and BFS scores at the end of the seven-week treatment period were significant for both once-daily (QD) and twice-daily (BID) dosing regimens.

Additionally, a separate study on the pharmacokinetics of KP1077, also presented at the conference, provided crucial insights into the drug's behavior when administered in the morning versus at night. The study found that a nighttime dose of KP1077 resulted in peak exposure of SDX-derived d-MPH the following morning, which may benefit patients experiencing severe morning sleep inertia and EDS.

The promising results from this Phase 2 trial support KP1077's potential as a new treatment option for IH, which is characterized by severe daytime sleepiness and difficulty waking up despite adequate nighttime sleep. The trial's findings will inform the design of a forthcoming Phase 3 trial, aimed at further establishing the efficacy and safety of KP1077.

KP1077 has already received Orphan Drug Designation from both the U.S. Food and Drug Administration (FDA) and the European Commission for the treatment of IH. This designation typically provides various development benefits for drugs designed to treat rare diseases. KP1077 also has intellectual property protection extending through 2037, potentially beyond.

During the SLEEP 2024 conference, Zevra Therapeutics highlighted its commitment to supporting the IH community, participating in the inaugural Idiopathic Hypersomnia Day and the BeyondSleepy Conference. These initiatives aim to raise awareness and provide education about IH, which affects approximately 37,000 diagnosed individuals in the United States, with the actual patient population likely being larger.

Zevra Therapeutics continues to focus on developing transformative therapies for rare diseases through innovative, data-driven approaches. The company's goal is to address unmet medical needs and improve the lives of patients with conditions like IH. With successful results from the Phase 2 trial, Zevra is optimistic about the potential of KP1077 to become a valuable treatment option for those suffering from this debilitating sleep disorder.