Science & Pipeline
Our expertise, elegant solutions, and transparent data narratives have produced a late-stage rare disease clinical pipeline and two partnered commercial products.
Our expertise, elegant solutions, and transparent data narratives have produced a late-stage rare disease clinical pipeline and two partnered commercial products.
The below product candidates are under investigation.
Orally-delivered, first-in-class investigational product candidate for NPC.
Arimoclomol – PDUFA action date set for September 21, 2024
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 MoreNew chemical entity for the treatment of COL3A1-positive vEDS.
Phase 3 trial ongoing
Granted orphan drug designation and breakthrough therapy designation by the U.S. FDA. Celiprolol is a New Chemical Entity currently in Phase 3 development for the treatment of COL3A1-positive vEDS patients to potentially reduce the risk of arterial and other hollow organ clinical events. The Phase 3 protocol is being conducted under a Special Protocol Assessment (SPA) agreement with the U.S. FDA.
Lead prodrug candidate for idiopathic IH and narcolepsy type I & II
KP1077 IH - Phase 2 trial in IH initiated Dec. 2022; Topline data from the completed trial are expected in the first half of 2024; potential Zevra commercial candidate
Phase 1 clinical trial in healthy volunteers completed
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.
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.
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 MoreThese product candidates are under investigation and their safety and efficacy have not been established. There is no guarantee that these products will receive health authority approval or become commercially available for the uses being investigated.
OLPRUVA is a registered trademark of Acer Therapeutics Inc., a wholly owned subsidiary of Zevra Therapeutics, Inc.
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.
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.
NOW ENROLLING COL3A1 positive vEDS patients (26 years and older)
Study ACER002-301 is being conducted by Zevra to evaluate the efficacy and safety of Celiprolol.
Celiprolol is an adrenoceptor modulator believed to decrease mechanical stress on the vascular wall of large arteries and hollow organs such as the uterus. It is an investigational medication for the treatment of vascular Ehlers Danlos Syndrome (vEDS).
The clinical study intends to enroll 150 COL3A1 positive vEDS patients. 50 patients will receive placebo and 100 patients will receive Celiprolol up to 400 mg/day (200mg bid). The study will conclude when 46 qualifying events (fatal or non-fatal cardiac event such as rupture of dissection, uterine rupture, colon rupture and/or unexplained sudden death)have been reported. This trial will conclude in approximately 40 months.”