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.
EMA MAA Submission: Targeting H2 2025
New 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. For more information.
Lead prodrug candidate for IH and narcolepsy type I & II
Phase 3 trial ready
Phase 3 trial potential
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 completed 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.
Phase 1 clinical trial under the narcolepsy IND was completed for KP1077 in healthy volunteers. Data generated from this trial will be analyzed alongside the Phase 2 IH data to support clinical development of both narcolepsy and IH programs. Zevra completed a Phase 1 clinical trial of KP1077N. This study provided additional information for optimizing the dosing regimen of KP1077 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.
The efficacy and safety of arimoclomol in NPC was evaluated in a 12-month, double blind placebo-controlled trial in 50 patients with NPC. Treatment with arimoclomol demonstrated a clinically meaningful and statistically significant treatment effect on change from baseline in NPC Clinical Severity Score.
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.
NOW ENROLLING COL3A1 positive VEDS patients (15 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.