Alexion Pharmaceuticals agreed to partner with Prothelia and the University of Nevada, Reno, to develop a protein replacement therapy for a life-threatening, ultra-rare disease caused by a genetic deficiency of the laminin-211 protein. The value of the collaboration was not disclosed.
The agreement gives Alexion an exclusive option to acquire privately held Prothelia and license the preclinical therapy, laminin-111 (PRT-01), directly from the University of Nevada, Reno upon the achievement of undisclosed research and development milestones.
Laminin-111 is a form of laminin found in embryonic skeletal muscle that has shown promise in early animal studies as a substitute for the loss of laminin-211. The partners agreed to develop the therapy for merosin-deficient congenital muscular dystrophy (MDC1A), a form of MD linked to hypotonia in children at birth, as well as reduced movements and tightness of the hips, knees, and elbows.
Laminin-111 has received orphan drug status from the FDA for MDC1A as well as for Duchenne muscular dystrophy. The compound is also being developed as a therapy for a third form of MD, dystroglycanopathy (LGMD21).
In addition, the University of Nevada, Reno and Alexion have entered into a sponsored research agreement designed to accelerate further study of the investigational therapy, conditioned on the outcome of development research to be performed by Alexion. The company specializes in developing drugs for severe and life-threatening ultra-rare diseases.
Laminin-111 is based on research by Dean J. Burkin, Ph.D., director of the Cellular and Molecular Pharmacology and Physiology Graduate Program at the University of Nevada School of Medicine. Laminin-111 was licensed to Prothelia in 2008. In a 2012 study published in The American Journal of Pathology, Dr. Burkin and colleagues showed that when laminin-111 was systemically delivered to the muscles of laminin-α2-deficient mice, the protein replacement therapy prevented muscle pathology, improved muscle strength, and dramatically increased life expectancy.
"Dr. Burkin has devoted much of his research to the treatment of muscular dystrophy, and this partnership and continued investigational research builds on his work," Mridul Gautam, the university's vp of research and innovation, said in a statement.
Prothelia was founded in 2007 by Bradley Hodges, Ph.D., who serves as the company’s president and CSO. Since then, Prothelia has received support from the NIH and patient advocacy groups, including Cure CMD, Struggle Against Muscular Dystrophy (SAM), Parent Project Muscular Dystrophy, and Hope for Gus.