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Phase 1 Phase 2 Phase 3 NDA Submission
Niemann-Pick Disease Type C (NPC) Program
Orally-delivered, first-in-class investigational product candidate for NPC.
Niemann-Pick Disease Type C (NPC)
Arimoclomol NDA resubmission to FDA in Q4 2023
Granted orphan drug designation, fast track designation, and rare pediatric disease designation for NPC by the European Medicines Agency (EMA) and FDA as a breakthrough therapy in NPC.
There are no approved treatments for NPC in the U.S. and there are an estimated 1,800 people with NPC in the U.S. and Europe.Learn More
Rare Sleep Disorders Program
Lead prodrug candidate for idiopathic IH and narcolepsy type I & II
Idiopathic Hypersomnia (IH)
KP1077 IH - Phase 2 trial in IH initiated Dec. 2022; Interim Phase 2 IH data expected by the end of Q3 2023; potential Zevra commercial candidate
Phase 1 clinical trial in healthy volunteers initiated during Q2 2023 and currently enrolling
Serdexmethylphenidate (SDX) is Zevra’s proprietary prodrug of d-methylphenidate (d-MPH) and the sole active pharmaceutical ingredient (API) in KP1077, Zevra’s lead product candidates being developed for the treatment of idiopathic hypersomnia (IH) and narcolepsy, respectively. IH is a rare neurological sleep disorder that can exhibit symptoms similar to narcolepsy. Narcolepsy is a chronic neurological disorder that affects the brain's ability to control sleep-wake cycles. Zevra plans to conduct the clinical development of both product candidates under separate INDs.
KP1077 being investigated for IH has been granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) for the treatment of IH and may be eligible for expedited approval pathways. The U.S. 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.
KP1077 in Idiopathic Hypersomnia
Zevra is currently conducting a multicenter, dose-optimizing, double-blind, placebo-controlled, randomized-withdrawal Phase 2 clinical trial to evaluate the safety and efficacy of KP1077 as a treatment for IH. In addition to assessing the safety of SDX in this study, KP1077 is being investigated for its potential to address excessive daytime sleepiness, sleep inertia and cognitive dysfunctions such as difficulty to focus and memory lapses (also referred to as “brain fog”) associated with IH. The results of this trial will help inform the design of a potential pivotal Phase 3 study of KP1077 in patients with IH and a potential Phase 3 trial of KP1077 in patients with narcolepsy.
KP1077 in Narcolepsy
An Investigational New Drug (IND) application has been submitted for KP1077 in narcolepsy and the U.S. Food and Drug Administration (FDA) has notified Zevra that we may initiate a Phase 1 clinical trial of KP1077N. Zevra plans to initiate the first of several Phase 1 clinical trials of KP1077N as early as in the second quarter of 2023. This study is expected to provide additional information for optimizing the dosing regimen of KP1077 for the treatment of narcolepsy. One of the objectives will be to assess the pharmacokinetics of SDX when dosed in the morning versus at night. The Phase 1 studies are expected to potentially support New Drug Applications (NDAs) for both the IH and narcolepsy submissions.Learn More
Phase 2/3 Trial of Arimoclomol in Niemann-Pick Type C
Arimoclomol has been studied in 500+ people across 10 phase 1, four phase 2 and three phase 2/3 clinical trials, with no significant issues with safety identified. Its capsule formulation is designed to be swallowed whole, but administration is flexible, allowing the drug substance to be mixed with soft foods and liquids or delivered through a gastric feeding tube, if needed. Thus far, our data have shown promising results.
A sub-study of the completed CT-ORZY-NPC-002 trial (NCT02612129) is ongoing, investigating the safety of arimoclomol in children below the age of two with NPC. See clinicaltrials.gov for further information. Note that the trial is listed as “active, not recruiting” since the main trial has been completed.
However, the sub-study is still recruiting.
Ongoing Phase 2 Clinical Study (KP1077.D01) in Adults with Idiopathic Hypersomnia (IH)
NOW ENROLLING adults (18 years and older) with IH
Study KP1077.D01 is being conducted by Zevra to evaluate the safety and efficacy of KP1077 capsules in patients with IH.
KP1077 capsules contain serdexmethylphenidate (SDX), a prodrug of dexmethylphenidate. It is an investigational medication for treating excessive daytime sleepiness (EDS) and other symptoms of IH, including sleep inertia (difficulty of waking up in the morning), and brain fog (lack of focus and mental clarity; forgetfulness and confusion). SDX has a unique, slow release profile that could potentially provide stable control of sleepiness throughout the day, with low abuse potential.
The clinical study consists of a screening period (up to 5 weeks), a 5-week open-label dose optimization period during which the optimal SDX dose will be determined, followed by a 2-week double-blind, randomized withdrawal period.
Topline data from Phase 1 Clinical Trial of KP1077 in Idiopathic Hypersomnia
In a phase 1 clinical trial, KP1077 was found to be well-tolerated.
KP1077’s impact on the heart was compared to immediate-release and long-lasting formulations of Ritalin®, a commonly prescribed stimulant for people with IH. It was found that KP1077 could be safely administered at higher doses than Ritalin. This exposure is expected to result in a pharmacokinetic profile that yields greater benefits for people with IH.
We have initiated a phase 2 clinical trial in patients with IH to further understand KP1077’s efficacy and safety, as well as to assess the symptoms and severity of “brain fog.” We expect to enroll approximately 48 adults with IH in more than 30 centers in the United States.
Part 1 of the trial will consist of a five-week open-label period during which patients will be matched to one of four doses of KP1077. Part 2 of the trial will entail a two-week period, during which two-thirds of participants will continue to receive their optimized dose while the remaining one-third will receive placebo.
For more information, see here.
ADHD Commercial Product
serdexmethylphenidate and dexmethylphenidate
Attention Deficit Hyperactivity Disorder (ADHD) in patients age six and older
Potential to reach one or more milestones during 2023 and continued royalty revenue
Approved by the FDA in 2021, AZSTARYS is a prodrug for the treatment of ADHD in patients age six years or older.
For more information visit azstarys.com.
Publications and Presentations
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