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Zevra Therapeutics, Inc. announced interim findings from the Phase 2 clinical trial of KP1077, today. Aimed at patients with idiopathic hypersomnia (IH), this development is a significant stride in enhancing treatment options.

Safety and Tolerability

The primary objective of the Phase 2 clinical trial was to evaluate the tolerability of KP1077 in IH patients. The interim data has unveiled encouraging results, showcasing that KP1077 is well-tolerated across all doses and dosing regimens examined. Most adverse events reported were of mild severity, aligning with those typically associated with stimulants.

Unique Benefits of KP1077

One remarkable takeaway from the open-label dose titration phase of the trial is the potential advantages of KP1077 as an IH treatment. This discovery holds the promise of offering a distinctive treatment alternative for patients who are underserved by existing therapies.

Promising Secondary and Exploratory Endpoints

The interim data highlights substantial enhancements in patient-reported evaluations of key IH symptoms during the open-label titration phase. These improvements encompass reductions in excessive daytime sleepiness, sleep inertia, and brain fog.

Next Steps

The Phase 2 trial is far from its conclusion. The completion of the double-blind withdrawal phase is anticipated to yield topline data in the first half of 2024. The combined open-label interim and upcoming topline data offers insights into secondary and exploratory endpoints, providing a comprehensive understanding of KP1077’s potential as an IH treatment.

These interim findings will guide the final design of the Phase 3 trial in IH by the end of 2024.

About the KP1077 Phase 2 Trial

The Phase 2 clinical trial adopts a double-blind, placebo-controlled, randomized-withdrawal, dose-optimizing, multi-center approach to assess the efficacy and safety of KP1077 for IH treatment. Zevra will enroll approximately 48 adult IH patients across more than 30 U.S. centers.

About Idiopathic Hypersomnia (IH)

Idiopathic Hypersomnia is a rare sleep disorder characterized by excessive daytime sleepiness. Patients with IH struggle with daytime lapses into sleep, an insatiable urge to sleep even after adequate nighttime rest, and difficulty waking. These symptoms, including severe “brain fog,” often lead to further debilitating issues such as memory lapses, difficulty focusing, and depression.

About SDX and KP1077

Serdexmethylphenidate (SDX) serves as Zevra’s proprietary prodrug of d-methylphenidate (d-MPH) and stands as the sole active pharmaceutical ingredient (API) in KP1077. Zevra is enrolling patients in a Phase 2 clinical trial to assess the safety and efficacy of KP1077 for IH.

KP1077 has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for IH treatment. The U.S. Drug Enforcement Agency (DEA) has classified SDX, the sole API in KP1077, as a Schedule IV controlled substance due to evidence suggesting a lower potential for abuse compared to d-MPH, a Schedule II controlled substance.

About Zevra Therapeutics

Zevra Therapeutics is at the forefront of rare disease therapeutics, amalgamating science, data, and patient needs to develop transformative therapies for conditions with limited or no treatment options. Their unique, data-driven approach to clinical development is set to bring much-needed therapies to the rare disease community, shaping the future of healthcare.

Stay tuned for more updates as Zevra Therapeutics continues to make remarkable progress in the realm of rare disease therapeutics.

Disclaimer: This blog post is for informational purposes only and should not be considered medical advice. Always consult with a healthcare professional for any medical concerns or treatment options.

Read the original announcement HERE.