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Science & Pipeline

Our expertise, elegant solutions, and transparent data narratives have produced a late-stage rare disease clinical pipeline and two partnered commercial products.

Product candidate
Indication
Phase 1 Phase 2 Phase 3 NDA Submission
Milestones

Niemann-Pick Disease Type C (NPC) Program

Arimoclomol

Orally-delivered, first-in-class investigational product candidate for NPC.

Niemann-Pick Disease Type C (NPC)

NDA SUBMISSION

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.

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Rare Sleep Disorders Program

KP1077

Lead prodrug candidate for idiopathic IH and narcolepsy type I & II

Idiopathic Hypersomnia (IH)

Phase 2

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.

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A photo of documents laid out on a desk with a stethoscope and pair of glasses nearby.

Clinical Trials

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.

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Phase 2 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 clinicaltrials.gov.

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Commercial Product

Product Name
Parent Drug
Commercial Partner

Approved product

AZSTARYS®

methylphenidate

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.

Publications and Presentations

Publications

Journal of Inherited Metabolic Disease

- 08/21/2021

Efficacy and safety of arimoclomol in Niemann-Pick disease type C: Results from a double-blind, randomised, placebo-controlled, multinational phase 2/3 trial of a novel treatment

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Drug Development & Delivery

- 03/01/2019

Prodrugs for ADHD Treatments: Opportunities & Potential to Fill Unmet Medical Needs

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Pain Medicine

- 10/28/2017

Pharmacokinetics and Abuse Potential of Benzhydrocodone, a Novel Prodrug of Hydrocodone, After Intranasal Administration in Recreational Drug Users

Read More

Pain Medicine

- 08/25/2017

Relative Bioavailability, Intranasal Abuse Potential, and Safety of Benzhydrocodone/Acetaminophen Compared with Hydrocodone Bitartrate/Acetaminophen in Recreational Drug Abusers

Read More

Pharmacoepidemiology & Drug Safety

- 08/03/2017

Patterns of abuse and routes of administration for immediate-release hydrocodone combination products

Read More

Poster Presentations

The 19th Annual WORLDSymposiumTM

- February 22 – 26, 2023

Association Between NPC Severity Score Domains and Corresponding Items of the Performance-based Scale for the Assessment and Rating of Ataxia (SARA)

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The 19th Annual WORLDSymposiumTM

- February 22 – 26, 2023

Evaluation of the Long-Term Effect of Arimoclomol in NPC

Read More

Academy of Managed Care Pharmacy (AMCP)

- March 29 – April 1, 2022

Serdexmethylphenidate/d-Methylphenidate Capsules for Children With ADHD: Effects on SKAMP-C Evaluated Over 13 Hours in a Randomized, Double-blind, Placebo-controlled Laboratory Classroom Study

Read More

The American Professional Society of ADHD and Related Disorders (APSARD) 2022 Annual Conference

- January 14-16, 2022

Mass Balance and Metabolic Pathway Following Oral Administration of [14C]-Serdexmethylphenidate, a Novel Prodrug of d-Methylphenidate

Read More Listen

The American Professional Society of ADHD and Related Disorders (APSARD) 2022 Annual Conference

- January 14-16, 2022

Dose Proportionality and Effects of Food on the Pharmacokinetics of Single-Entity Serdexmethylphenidate

Read More Listen

Children and Adults with Attention-Deficit/Hyperactiviity Disorder

- November 4–6, 2021

Serdexmethylphenidate/d-Methylphenidate Capsules for Children With ADHD: Effects on SKAMP-C Evaluated Over 13 Hours in a Randomized, Double-blind, Placebo-controlled Laboratory Classroom Study

Read More

Psych Congress

- October 29–November 1, 2021

Steady-State Pharmacokinetics and Relative Bioavailability of Serdexmethylphenidate/d-Methylphenidate, a Treatment for Attention Deficit Hyperactivity Disorder Containing a Novel Prodrug of d-Methylphenidate

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American Academy of Child & Adolescent Psychiatry

- October 18-30 2021

Single-dose Pharmacokinetics of Serdexmethylphenidate/d-Methylphenidate Capsules in Children and Adolescents With ADHD and Healthy Adults: An Evaluation of Age and Body Weight

Read More

American Psychiatric Nurses Association

- October 13-16, 2021

Serdexmethylphenidate/d-Methylphenidate Capsules for Children With ADHD: Effects on SKAMP-C and WREMB-R as Evaluated in a Randomized, Double-blind, Placebo-controlled Laboratory Classroom Study

Read More

American Academy of Child and Adolescent Psychiatry

- October 12-24, 2020

Patterns of Abuse of Prescription Stimulant Products from the National Addictions Vigilance Intervention Program (NAVIPPRO): 2010-2018

Read More

American Academy of Child and Adolescent Psychiatry

- October 14-19, 2019

Effect of Food and Alternative Dosing Methods on the Pharmacokinetics of KP415, an Investigational ADHD Product Containing the Prodrug, Serdexmethylphenidate (SDX)

Read More

The American Professional Society of ADHD and Related Disorders

- January 18-20, 2019

Single-dose Pharmacokinetics of KP415, an Investigational Product Containing the Prodrug Serdexmethylphenidate (SDX), in Children and Adolescents with ADHD

Read More

The American Professional Society of ADHD and Related Disorders

- January 18-20, 2019

Dose-Proportionality and Steady-State Pharmacokinetics of KP415, an Investigational ADHD Product Containing Serdexmethylphenidate (SDX), a Novel Prodrug of d-Methylphenidate

Read More

The American Professional Society of ADHD and Related Disorders

- January 18-20, 2019

Human Abuse Potential of Oral Serdexmethylphenidate (SDX), a Novel Prodrug of d-Methylphenidate, Compared to Focalin® XR and Phentermine in Recreational Stimulant Abusers

Read More

The American Professional Society of ADHD and Related Disorders

- January 18-20, 2019

Human Abuse Potential of Intranasal Serdexmethylphenidate (SDX), a Novel Prodrug of d-Methylphenidate, in Recreational Stimulant Abusers

Read More

American College of Neuropsychopharmacology

- December 9-13, 2018

Assessment of the Intravenous Abuse Potential Of Serdexmethylphenidate (SDX), a Novel, Investigational Prodrug of D-Methylphenidate: Evidence from Nonclinical and Clinical Studies

Read More

American Academy of Child & Adolescent Psychiatry

- October 22-27, 2018

Human Abuse Potential of Intravenous Serdexmethylphenidate (SDX), a Novel Prodrug of d-Methylphenidate, in Recreational Stimulant Abusers

Read More

American Academy of Child & Adolescent Psychiatry

- October 22-27, 2018

Dose-finding Study of Abuse-Related Effects of Intranasal d-Methylphenidate in Recreational Stimulant Abusers

Read More

American Professional Society for ADHD and Related Disorders

- January 12-14, 2018

Single- and Multiple-dose Pharmacokinetics of KP415, a Novel d-Methylphenidate Product Containing a Prodrug of d-Methylphenidate, in Healthy Volunteers

Read More

2017 International Conference on Opioids (ICOO)

- June 11-3, 2016

Pharmacokinetics and Abuse Potential of KP511, a Novel Prodrug of Hydromorphone, after Intranasal Administration in Recreation Drug Users

Read More

American Academy of Pain Medicine (AAPM) Annual Meeting

- March 15-19, 2017

Oral pharmacokinetics of KP511, a prodrug of hydromorphone, relative to hydromorphone in human volunteers

Read More

American Academy of Pain Medicine (AAPM) Annual Meeting

- March 15-19, 2017

Pharmacokinetics and Abuse Potential of Benzhydrocodone, A Novel Prodrug of Hydrocodone, After Intranasal Administration in Recreational Drug Users

Read More

2016 International Conference on Opioids (ICOO)

- June 5-7, 2016

Pharmacokinetics and Abuse Potential of Benzhydrocodone, a Novel Prodrug of Hydrocodone, After Intranasal Administration in Recreational Drug Users

Read More

2016 International Conference on Opioids (ICOO)

- June 5-7, 2016

Relative Bioavailability, Intranasal Abuse Potential, and Safety of Benzhydrocodone/Acetaminophen, a Novel Immediate-Release Hydrocodone Prodrug Combination, Compared With Hydrocodone Bitartrate/Acetaminophen in Recreational Drug Abusers

Read More

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