Data on CUTX-101, copper histidinate, will be presented at the American College of Medical Genetics Annual Meeting in 2022

Cyprium Therapeutics, Inc. (“Cyprium”), a Fortress Biotech, Inc. (Nasdaq: FBIO) (“Fortress”) subsidiary, today announced positive data on CUTX-101, copper histidinate (CuHis), in patients with Menkes disease. Sentynl Therapeutics, Inc. (“Sentynl”) is a wholly owned subsidiary of Zydus Lifesciences Ltd (formerly known as Cadila Healthcare Ltd). The information will be presented as a “Top-Rated Abstract” and Poster at the 2022 Annual Clinical Genetics Conference of the American College of Medical Genetics and Genomics (“ACMG”), which will be held online and in Music City Center in Nashville, Tennessee, from March 22–26, 2022. Results from an effectiveness and safety analysis of data merged from two completed pivotal studies in patients with Menkes disease treated with CUTX-101 are what were previously published.

The promising results that will be presented at the 2022 ACMG Annual Clinical Genetics meeting show the effectiveness and safety of CUTX-101, as well as its potential to become the first medication authorized by the FDA for Menkes disease patients.
Lung S. Yam, M.D., Ph.D., President and Chief Executive Officer of Cyprium, stated, “We continue to make progress with our rolling submission of a new drug application (“NDA”) for CUTX-101, which we anticipate will be finished in the middle of this year. We are excited about the chance to inform medical geneticists, who frequently assist in the diagnosis and care of the uncommon, deadly pediatric condition Menkes, of the positive efficacy and safety results of CUTX-101.

To bring CUTX-101 to market in 2021, Cyprium collaborated with Sentynl Therapeutics, Inc., a specialized pharmaceutical business with headquarters in the United States and owned by the Zydus Group. Sentynl will be in charge of commercializing CUTX-101 and advancing newborn screening efforts, while Cyprium will continue to be in charge of CUTX-101 development through the FDA’s approval of the NDA. Sentynl Therapeutics, a completely owned subsidiary of Zydus, is committed to empowering patients with rare diseases.

A step in the right direction has been made with the CUTX-101 copper histidinate (CuHis) positive findings. To lower the risk of mortality in patients with molybdenum cofactor deficiency (MoCD) Type A, an extremely uncommon, life-threatening juvenile genetic condition, the group firm has entered into an asset purchase agreement for the purchase of BridgeBio’s NULIBRYTM (Fosdenopterin) for Injection.

About Menkes Disease

Children who have Menkes disease have a rare X-linked recessive condition brought on by mutations in the copper transporter ATP7A gene. According to a recent genome-based ascertainment (Kaler SG, Ferreira CR, Yam LS. Estimated birth prevalence of Menkes disease and ATP7A-related disorders based on the Genome Aggregation Database), the birth prevalence for Menkes disease is thought to be at least 1 in 34,810 live male births and may even reach 1 in 8,664 live male births (gnomAD).
(200605;24:100602) Molecular Genetics and Metabolism Reports. Sparse and depigmented hair (often known as “kinky hair”), connective tissue issues, and severe neurological symptoms like seizures, hypotonia, failure to grow, and developmental delays are some of the condition’s unique clinical hallmarks.

Untreated Menkes illness has a significant mortality rate, and many patients pass away before they turn three. Other diseases including Occipital Horn Syndrome and ATP7A-related Distal Motor Neuropathy are linked to milder ATP7A mutations. For Menkes disease and its variations, there is currently no FDA-approved therapy.

About CUTX-101 (Copper Histidinate)

The drug CUTX-101 is being tested in clinical settings to treat Menkes disease. The Copper Histidinate subcutaneous injectable formulation CUTX-101 is created using physiological pH and current good manufacturing practice (“cGMP”). Patients with Menkes disease were treated with CUTX-101 early on in a Phase 1/2 clinical trial run by Stephen G. Kaler, M.D., M.P.H., at the National Institutes of Health (“NIH”), and this improved their neurodevelopmental outcomes and survival.

Cyprium announced positive topline clinical efficacy results for CUTX-101 in August 2020. These results showed a statistically significant improvement in overall survival for Menkes disease patients who received early treatment (ET) with CUTX-101, compared to an untreated historical control cohort, with a nearly 80% decrease in the risk of death.

FDA Breakthrough Therapy, Fast Track, Rare Pediatric Disease, and FDA Orphan Drug Designations have all been given to CUTX-101. The European Medicines Agency also designated CUTX-101 as an orphan drug. Many U.S. hospitals are currently participating in a Cyprium-sponsored expanded access policy for patients with Menkes disease.

About Zydus

Zydus Cadila is a global pharmaceutical company based in India, founded in 1952. The company is engaged in the research, development, and manufacturing of pharmaceutical products, including generics, branded drugs, biosimilars, vaccines, and other healthcare products.

Zydus has a strong presence in India and has expanded globally, with operations in over 50 countries. The company’s research and development efforts are focused on various therapeutic areas, including cardiovascular, oncology, respiratory, neuroscience, and gastroenterology.

In recent years, Zydus has made significant strides in the development of COVID-19 treatments and vaccines. In August 2021, the company received emergency use authorization for its COVID-19 vaccine, ZyCoV-D, which is the world’s first DNA-based vaccine for COVID-19.

Zydus Cadila is committed to making healthcare accessible and affordable to people around the world, and has a strong track record of delivering innovative and high-quality products.

About Sentynl Therapeutics

Sentynl Therapeutics is a pharmaceutical company based in the United States that specializes in the development and commercialization of products for pain management and other therapeutic areas. The company was founded in 2016 and is headquartered in Solana Beach, California.

Sentynl’s portfolio includes both branded and generic products, with a focus on abuse-deterrent formulations and other innovative delivery technologies. The company’s products are used to treat a range of conditions, including chronic pain, postoperative pain, and opioid dependence.

In addition to its commercial products, Sentynl is also engaged in research and development efforts to expand its pipeline of new products. The company has partnerships with other pharmaceutical companies and academic institutions to support these efforts.

Sentynl is committed to providing safe and effective products that improve the lives of patients suffering from pain and other conditions. The company’s products are distributed throughout the United States and in select international markets.

About Cyprium Therapeutics

Cyprium Therapeutics is a clinical-stage biopharmaceutical company focused on developing therapies for rare and serious genetic disorders. The company was founded in 2017 and is headquartered in Bedminster, New Jersey.

Cyprium’s pipeline is focused on copper metabolism disorders, which are a group of rare diseases caused by genetic mutations that impair the body’s ability to maintain normal copper levels. These disorders can lead to a range of symptoms and complications, including neurological problems, liver damage, and connective tissue disorders.

The company’s lead product candidate, CUTX-101, is a subcutaneous injectable form of copper histidine being developed for the treatment of Menkes disease, a rare and fatal genetic disorder that affects copper transport in the body. CUTX-101 has received both orphan drug designation and rare pediatric disease designation from the US FDA.

Cyprium is committed to developing innovative therapies for rare diseases with significant unmet medical needs. The company works closely with patient advocacy groups, academic researchers, and regulatory agencies to advance its pipeline and bring new treatments to patients as quickly as possible.

About Fortress Biotech

Fortress Biotech is a biopharmaceutical company based in the United States that specializes in the development and commercialization of novel therapies for rare and underserved diseases. The company was founded in 2006 and is headquartered in New York City.

Fortress Biotech’s business model is based on acquiring and developing promising technologies and drug candidates from academic institutions, small biotechnology companies, and other sources. The company operates through a network of subsidiary companies, each focused on a specific area of research or development.

The company’s pipeline includes a range of products in various stages of development, from early-stage research to late-stage clinical trials. These products are focused on a range of therapeutic areas, including oncology, rare genetic disorders, and autoimmune diseases.

Fortress Biotech is committed to advancing innovative therapies for patients with significant unmet medical needs. The company’s collaborative approach to drug development, combined with its experienced management team and extensive network of academic and industry partners, positions it for continued growth and success in the biopharmaceutical industry.

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