CUTX-101 has potential to be the first FDA-approved treatment for Menkes disease; rolling submission of New Drug Application to FDA is ongoing and expected to be completed in 2024
Dec. 6, 2023
Solana Beach, CA and Ahmedabad, India – December 6, 2023 — Sentynl Therapeutics, Inc. (Sentynl), a U.S.-based biopharmaceutical company wholly-owned by Zydus Lifesciences, Ltd. (Zydus Group), today announced the execution of an Assignment and Assumption Agreement with Cyprium Therapeutics, Inc. (Cyprium), a Fortress Biotech, Inc. (Nasdaq: FBIO) (Fortress) subsidiary company. Under the agreement, Cyprium completed the transfer of its worldwide proprietary rights and U.S. FDA documents pertaining to CUTX-101, the copper histidinate product candidate for the treatment of Menkes disease, to Sentynl. Sentynl now assumes full responsibility for the development and commercialization of CUTX-101. In 2021, Sentynl and Cyprium reported 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. A rolling submission of the CUTX-101 New Drug Application (NDA) to the FDA is ongoing, with expected completion in 2024. Speaking on the development, Dr. Sharvil Patel, Managing Director, Zydus Lifesciences said, “We have been committed to providing access to path-breaking discoveries that can bridge unmet healthcare needs, globally. The rights for CUTX-101 is a significant milestone towards our vision to transform lives and meaningfully impact patients, healthcare providers and the rare disease community at large. This novel, breakthrough therapy could unlock possibilities for the treatment of the life threatening Menkes disease.” “Menkes disease has a devastating impact on patients and their caregivers. With no current approved treatments, death usually occurs between 6 months and 3 years old,” said Matt Heck, President & Chief Executive Officer of Sentynl. “We are committed to advancing CUTX-101, which has the potential to not only become the first FDA-approved treatment for Menkes disease, but also positively impact the lives of the patients and their caregivers affected by this rare, fatal condition.” “We know first-hand the difficult journey that patients and families of Menkes disease face,” commented Drew and Jamie Eckman, Founders of the Menkes Foundation and parents of Wesley, who was diagnosed with Menkes disease at 8.5 months old and passed away three months later. “The development of CUTX-101 represents incredible progress in the fight against Menkes disease. We hold a strong amount of hope for a time when there is an option available to treat this condition, providing a path forward for caregivers and their children.”
About Menkes Disease
Menkes disease is a rare X-linked recessive pediatric disease caused by gene mutations of the copper transporter ATP7A. The minimum birth prevalence for Menkes disease is believed to be 1 in 34,810 live male births, and potentially as high as 1 in 8,664 live male births, based on recent genome-based ascertainment (Kaler SG, Ferreira CR, Yam LS. Estimated birth prevalence of Menkes disease and ATP7Arelated disorders based on the Genome Aggregation Database (gnomAD). Molecular Genetics and Metabolism Reports 2020 June 5;24:100602). The condition is characterized by distinctive clinical features, including sparse and depigmented hair (“kinky hair”), connective tissue problems, and severe neurological symptoms such as seizures, hypotonia, failure to thrive, and neurodevelopmental delays. Mortality is high in untreated Menkes disease, with many patients dying before the age of three years old. Milder versions of ATP7A mutations are associated with other conditions, including Occipital Horn Syndrome and ATP7A-related Distal Motor Neuropathy. Currently, there is no FDA-approved treatment for Menkes disease and its variants.
Sentynl Therapeutics, Inc. is collaborating with researchers at a world-leading academic medical center to develop a laboratory test that could potentially diagnose Menkes disease at birth. The end goal is to use the results of this study to support adding Menkes disease to every state’s Newborn Screening Program.
To conduct the study, a dried blood spot card for your child from your state’s Newborn Screening lab is needed, and, if your child is not currently receiving copper replacement therapy, a very small sample of blood and (optionally) urine would also need to be collected.
If you are interested in participating, please see the official letter at this link which provides further details.
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