Provides new potential treatment option for Menkes disease and establishes Engrail’s rare/orphan disease development portfolio

SAN DIEGO--(BUSINESS WIRE)--Engrail Therapeutics™ (Engrail) (the Company), a neuroscience company focused on the acquisition, development and commercialization of patient-centric therapies and The Texas A&M University System (TAMUS), today announced that they have entered into a license agreement which grants Engrail worldwide exclusive rights to intellectual property around the use of elesclomol for disorders of mitochondrial copper metabolism.

“Mitochondrial copper metabolism disorders, such as ultra-orphan Menkes disease, are devastating for patients and families,” said Vikram Sudarsan, Ph.D., CEO and President of Engrail Therapeutics. “There are no FDA-approved treatments for Menkes disease, so we look forward to working closely with the team of researchers, led by Dr. Vishal M. Gohil, Associate Professor in the Department of Biochemistry and Biophysics at Texas A&M University, to rapidly advance elesclomol with copper as a potential treatment for these patients in need. Our organization has always been highly patient-focused and now, armed with the intellectual property of TAMUS, we will explore the potential of elesclomol-copper to significantly alter the course of mortality and morbidity for this disease. We are also excited to begin building an orphan disease portfolio with elesclomol-copper as our foundation. Engrail will continue to work closely with academic institutions and industry partnerships to expand our pipeline of therapeutics for patients with significant unmet needs in the neurosciences.”

Under the terms of the agreement, Engrail will pay licensing fees to TAMUS that include upfront consideration as well as additional payments upon the achievement of certain development, regulatory and commercial milestones. In addition, further nonclinical work will be completed through a sponsored research agreement with Texas A&M AgriLife Research.

“Our studies demonstrated that administration of a complex of elesclomol and copper to a murine model of severe Menkes disease prevents detrimental neurodegenerative changes and increases survival of these mice from a mere two weeks to over 250 days. With these unprecedented findings, we believe elesclomol, which helps carry copper into mitochondria, holds significant promise for the treatment of Menkes and associated disorders of hereditary copper deficiency,” said Dr. Gohil. “This partnership with Engrail will enable the further development of elesclomol-copper for patients and we look forward to closely collaborating with them every step of the way.”

About Menkes Disease (MD)

Menkes disease (MD) is an inherited, fatal pediatric disease caused by impaired absorption and distribution of dietary copper in the body. MD primarily affects the nervous system and typically manifests during the first few months of life, rapidly progressing over time. Symptoms include sparse and kinky hair, slow growth (failure to thrive), and seizures. Additional features may include low muscle tone (hypotonia), sagging facial features, and developmental and intellectual disability. Most children with MD have severe symptoms that lead to death at an early age. The underlying cause of the disorder is a mutation in the ATP7A gene, resulting in poor distribution of copper to the brain and other tissues. MD is an ultra-rare disease that affects approximately 150 newborns worldwide per year, although much higher number of cases is suspected. There are currently no approved treatments for MD.

About Engrail Therapeutics

Founded in 2019, Engrail is forging a new direction to reduce the enormous burden of diseases that impact the nervous system. We unite biological insights with clinically meaningful solutions to build and catalyze a diversified portfolio of transformative medicines. Harnessing our rigorous scientific approach to identify the most promising therapies, we leverage our flexible transaction model to advance assets with validated mechanisms and efficiently move them through development to commercialization. Engrail is supported by a long-term capital commitment from Nan Fung Life Sciences. For more information, visit www.engrail.com.

About The Texas A&M University System and Texas A&M AgriLife Research

The Texas A&M University System is one of the largest systems of higher education in the nation, with a budget of $9.6 billion. Through a statewide network of 11 universities, a comprehensive health science center, eight state agencies, and the RELLIS Campus, the Texas A&M System educates more than 153,000 students and makes more than 22 million additional educational contacts through service and outreach programs each year. System-wide, research and development expenditures exceed $1 billion and help drive the state’s economy. Texas A&M AgriLife Research, a State of Texas research agency and member of The Texas A&M University System, is the leading research and technology development agency in Texas for agriculture, natural resources, and the life sciences. To learn more, visit www.tamus.edu or agriliferesearch.tamu.edu/.

Originally published on businesswire.com.

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