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

Lonafarnib — For Hutchinson-Gilford Progeria Syndrome and Processing-Deficient Progeroid Laminopathies

Lonafarnib — For Hutchinson-Gilford Progeria Syndrome and Processing-Deficient Progeroid Laminopathies

Proof of Concept (PoC)

Pre-Clinical

Phase I

Phase II

Phase III

Regulatory Submission

Approved

Stage: Approved

About Progeria and Processing-Deficient Progeroid Laminopathy

Progeria, also known as Hutchinson-Gilford Progeria Syndrome (HGPS), and processing-deficient Progeroid Laminopathies (PDPL) are ultra-rare, fatal, genetic, premature aging diseases that accelerate mortality in young patients. In the United States, the prevalence of HGPS is 1 in 20 million, and the prevalence of progeroid laminopathies, which include both processing-deficient and processing-proficient subtypes, is 1 in 36.4 million.

HGPS is caused by a point mutation in the LMNA gene, yielding the farnesylated aberrant protein progerin. PDPL is caused by a heterozygous LMNA mutation with progerin-like protein accumulation or homozygous or compound heterozygous ZMPSTE24 mutations. Both HGPS and PDPL result in premature, extensive cardiovascular disease, which is the primary cause of death in nearly all patients.

For more information about HGPS and PDPL, please visit progeriaresearch.org.

Lonafarnib

Lonafarnib is a farnesyltransferase inhibitor indicated for the treatment of HGPS and processing-deficient Progeroid Laminopathies with either a heterozygous LMNA mutation with progerin-like protein accumulation or a homozygous or compound heterozygous ZMPSTE24 mutation in patients 12 months and older with a body surface area of 0.39 m2 and above.

Fosdenopterin — For MoCD Type A

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 4 years. MoCD Type A presents shortly after birth, often with severe encephalopathy and intractable seizures.

For more information, visit aboutmocdtypea.com/hcp.

Fosdenopterin

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

Copper histidinate* — For Menkes Disease

Copper histidinate US-only expanded access

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/hcp.

CUTX-101 (Copper Histidinate)

CUTX-101 (copper histidinate) is in development to restore copper homeostasis and maintain copper levels in patients with Menkes disease and related disorders. CUTX-101 is a subcutaneous injectable formulation of copper histidinate manufactured under current Good Manufacturing Practice (cGMP) that is intended to improve tolerability due to its physiological pH, and to bypass the oral absorption of copper, which is impaired in patients with Menkes disease. In the Phase 1/2 clinical study conducted at National Institute of Child Health and Human Development (NICHD), early treatment of Menkes patients with CUTX-101 led to an improvement in neurodevelopmental outcomes and survival.

Cyprium Therapeutics, Inc., with support from Sentynl, is developing CUTX-101 to restore copper homeostasis and maintain copper levels in patients with Menkes disease and related disorders.

In October 2021, Cyprium announced positive results from a safety and efficacy analysis of data integrated 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 to the U.S. Food and Drug Administration (FDA) for CUTX-101 for the treatment of Menkes disease.

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

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

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

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