Developing and Delivering Meaningful Therapies for Patients

Our biopharmaceuticals, in development or available, are designed to address a variety of rare diseases that can have serious consequences for patients and their caregivers. Explore this page to learn more about each.

Proof of Concept (PoC)

Pre-Clinical

Phase I

Phase II

Phase III

Regulatory Submission

Approved

ORGN001 (fosdenopterin) – For MoCD Type A

ORGN001 (fosdenopterin) – For MoCD Type A

Proof of Concept (PoC)

Pre-Clinical

Phase I

Phase II

Phase III

Regulatory Submission

Approved

Stage: Approved

About MoCD Type A

MoCD Type A is an ultra-rare and progressive condition. It is known to impact fewer than 150 patients globally and has a median survival of four years. MoCD Type A presents shortly after birth, often with severe encephalopathy and intractable seizures.

ORGN001 (fosdenopterin)

ORGN001 (fosdenopterin) is a cyclic pyranopterin monophosphate (cPMP) for patients with MoCD Type A.

CUTX-101 (copper histidinate) – For Menkes Disease

CUTX-101 (copper histidinate) – For Menkes Disease

Proof of Concept (PoC)

Pre-Clinical

Phase I

Phase II

Phase III

Regulatory Submission

Approved

Stage: Regulatory Submission

About Menkes Disease

Menkes disease is a rare X-linked pediatric disease caused by gene mutations of the copper transporter ATP7A. The condition is characterized by sparse, depigmented, kinky hair; failure to thrive; low/poor muscle tone (hypotonia); connective tissue disorders; and severe neurological symptoms such as seizures. If untreated, mortality for Menkes disease is high, with many patients dying before the age of 3. Currently, there is no FDA-approved treatment for Menkes disease and related disorders in copper metabolism.

To learn more about Menkes disease, please visit aboutmenkes.com.

CUTX-101 (Copper Histidinate)

Sentynl Therapeutics acquired from Cyprium Therapeutics, Inc. worldwide rights to CUTX-101 (copper histidinate), an investigational medicine to restore copper levels and maintain copper homeostasis in patients with Menkes disease and related disorders.

CUTX-101 has been granted Orphan Drug Designation by the FDA. The FDA previously granted Breakthrough Therapy, Fast Track, and Rare Pediatric Disease Designations to CUTX-101. In July 2020, the European Medicines Agency (EMA) Committee for Orphan Medicinal Products issued a positive opinion on the Orphan Medicinal Product Designation for CUTX-101.

In October 2021, Cyprium announced positive results from an integrated safety and efficacy data analysis  from two completed pivotal studies in patients with Menkes disease treated with CUTX-101. These data were presented as a virtual poster at the 2021 American Academy of Pediatrics National Conference & Exhibition.

In December 2021, Cyprium initiated a rolling submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for CUTX-101 for the treatment of Menkes disease.  In December 2023, Sentynl assumed responsibility for completing the NDA submission.

Also in December 2023, Sentynl assumed responsibility for managing an Expanded Access Protocol to obtain further data on the safety and efficacy of CUTX-101 that may be used to support approval of the product and subsequent wider accessibility to Menkes disease patients.  For more information, please visit this link.

Learn more about Menkes disease at the National Institutes of Health (NIH) and National Organization for Rare Disorders (NORD).

STI-103 — For Spasticity

STI-103 — For Spasticity

Proof of Concept (PoC)

Pre-Clinical

Phase I

Phase II

Phase III

Regulatory Submission

Approved

Stage: Phase I

STI-105 — For Lennox-Gastaut Syndrome

STI-105 — For Lennox-Gastaut Syndrome

Proof of Concept (PoC)

Pre-Clinical

Phase I

Phase II

Phase III

Regulatory Submission

Approved

Stage: Proof of Concept (PoC)

*CUTX-101 designations: Fast Track, Orphan Drug, Rare Pediatric Disease, Breakthrough Therapy.
(Sentynl acquired exclusive rights to CUTX-101 and is supporting its development.)