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.
Orally-delivered, first-in-class investigational product candidate for NPC.
Arimoclomol NDA resubmission to FDA as early as Q3 2023; potential Zevra commercial candidate
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 MoreLead prodrug candidate for idiopathic IH and narcolepsy type I & II
KP1077 IH - Phase 2 trial in IH initiated Dec. 2022; Interim Phase 2 IH efficacy and safety data expected as early as Q3 2023
KP1077 Narcolepsy - IND filing expected Q2 2023; Phase 3 trial in narcolepsy to initiate following IH Phase 2 trial results
IH is a rare neurological sleep disorder that can exhibit symptomatic narcolepsy.
KP1077 was granted Orphan Drug Designation by the FDA and may be eligible for fast-track and breakthrough therapy designation.
Based on anticipated clinical differentiators and possible combination use with sodium oxybate-based products, KP1077 may have the potential to capture a large share of the IH market.
KP1077 is being investigated for the potential to be administered daily to potentially address sleep inertia and daytime brain fog associated with IH. KP1077’s dosing flexibility may improve compliance and effectiveness in IH patients.
There is potential to advance KP1077 directly into phase 3 studies in narcolepsy based on previous phase 1 studies of serdexmethylphenidate, the sole pharmaceutical agent in KP1077, and learnings from the phase 2 study in IH.
Learn MoreArimoclomol 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 clinicaltrials.gov.
Corium Inc.
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.