Zevra Therapeutics Announces Interim Data from the Phase 2 Clinical Trial of KP1077 for Idiopathic Hypersomnia
Zevra Therapeutics Announces Interim Data from the Phase 2 Clinical Trial of KP1077 for Idiopathic Hypersomnia
October 2, 2023 at 7:30 AM EDT
Data generated to date indicate SDX is well-tolerated across all doses and both dosing regimens studied, laying the groundwork for future Phase 3 trial
Topline data from completed trial expected in the first half 2024, with potential to initiate Phase 3 trial by the end of 2024
“The open-label data yielded key insights for the design of the Phase 3 trial and potential unique benefits of KP1077 as a treatment for IH, if approved,” said
The interim data related to the secondary and exploratory endpoints showed marked improvements in patient-reported assessments of key IH symptoms from the open-label titration phase, including excessive daytime sleepiness, sleep inertia, and brain fog. The improvements in symptoms were similar after both once-per-day dosing of KP1077 (in the evening, just before bedtime), and twice-per-day dosing (half the daily dose after awakening in the morning and half the daily dose just before bedtime). The results from the completed Phase 2 trial are expected to inform the final design of the anticipated Phase 3 trial in IH, which is expected to be initiated by the end of 2024.
About the KP1077 Phase 2 Trial:
The Phase 2 clinical trial is a double-blind, placebo-controlled, randomized-withdrawal, dose-optimizing, multi-center study evaluating the efficacy and safety of KP1077 for the treatment of IH. Zevra expects to enroll approximately 48 adult patients with IH in more than 30 centers in the
The primary endpoint is the safety and tolerability of SDX. The major secondary efficacy endpoint is the change in Epworth Sleepiness Scale (ESS) total score. Additional exploratory endpoints include the Patient Global Impression of Severity (PGI-S), the Clinical Global Impression of Severity (CGI-S), change in total score on the Idiopathic Hypersomnia Severity Scale (IHSS), and a new scale to assess the symptoms and severity of “Brain Fog”.
About Idiopathic Hypersomnia (IH):
Idiopathic hypersomnia (IH) is a rare sleep disorder characterized by excessive daytime sleepiness. Patients with IH experience daytime lapses into sleep, or an irrepressible need to sleep that persists even with adequate or prolonged nighttime sleep. Additionally, those with IH have extreme difficulty waking, otherwise known as “sleep inertia,” severe “brain fog”, and often fall asleep unintentionally or at inappropriate times. These symptoms of IH often lead to further, even more debilitating problems such as memory lapses, difficulty maintaining focus, and depression.
It is estimated, based on claims data, that approximately 37,000 patients in
About SDX and KP1077:
Serdexmethylphenidate (SDX) is Zevra’s proprietary prodrug of d-methylphenidate (d-MPH) and the sole active pharmaceutical ingredient (API) in KP1077, Zevra’s lead clinical candidate being developed as a treatment for idiopathic hypersomnia (IH) and narcolepsy. Zevra is currently enrolling a multicenter, dose-optimizing, double-blind, placebo-controlled, randomized-withdrawal Phase 2 clinical trial to evaluate safety and efficacy of KP1077 as a treatment for IH. For more information regarding the Phase 2 trial, visit www.clinicaltrials.gov.
KP1077 has been granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) for the treatment of IH, and the U.S. Drug Enforcement Agency (DEA) has classified SDX, the sole API in KP1077, 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.
About Zevra Therapeutics:
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. With both regulatory and clinical stage product candidates, the Company is building its commercial capability to make new therapies available to the rare disease community.
Cautionary Note Concerning Forward-Looking Statements:
This press release may contain forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include all statements that do not relate solely to historical or current facts, and which can be identified by the use of words such as "may," "will," "expect," "project," "estimate," "anticipate," "plan," "believe," "potential," "should," "continue," "could," "intend," "target," "predict," or the negative versions of those words or other comparable words or expressions, although not all forward-looking statements contain these identifying words or expressions. Forward-looking statements are not guarantees of future actions or performance. These forward-looking statements include without limitation statements regarding the promise and potential impact of our preclinical or clinical trial data, including without limitation the initiation, timing, design, interim data and analysis, and results of any clinical trials or readouts and our strategic and product development objectives, including with respect to becoming a leading, commercially-focused rare disease company. Forward-looking statements are based on information currently available to Zevra and its current plans or expectations. They are subject to several known and unknown uncertainties, risks, and other important factors that may cause our actual results, performance, or achievements to be materially different from any future results, performance, or achievements expressed or implied by the forward-looking statements. These and other important factors are described in detail in the “Risk Factors” section of Zevra’s Annual Report on Form 10-K for the year ended
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