NEW ALBANY, Ohio--(BUSINESS WIRE)--Myonexus Therapeutics, a clinical-stage gene therapy company developing first-ever corrective gene therapies for Limb-girdle muscular dystrophies (LGMD), announced today Sarepta will exercise its option to purchase Myonexus for $165 million. The agreement follows positive, preliminary results from the Myonexus Limb-girdle muscular dystrophy (LGMD) 2E program in which the first three patients dosed demonstrated robust and widespread expression of the protein beta-sarcoglycan, properly localized to the muscle sarcolemma, as measured by immunohistochemistry. The lack of beta-sarcoglycan is the root cause of LGMD2E.
In May 2018, Myonexus and Sarepta entered into an exclusive partnership to develop Myonexus’ five LGMD gene therapy candidates that target the most severe and common forms of the disease, three of which are in clinical development and two are in pre-clinical development. As part of the agreement announced last year, Sarepta maintained an option to purchase Myonexus at any time at a pre-determined purchase price with sales-related contingent payments. Under the terms of the agreement, Sarepta made an upfront payment of $60 million in addition to payments for achieving certain development milestones.
“From the beginning, the focus of Myonexus has been to identify and advance outstanding science to make a difference in the lives of patients. Our partnership with Sarepta last year and Sarepta’s early acquisition of Myonexus serves to accelerate what we set out to accomplish,” said Michael Triplett, Myonexus’ president and chief executive officer. “The promising, preliminary data validate the potential of our approach to bringing treatments forward for patients with LGMD and our greatest hopes will be realized when patients benefit from approved treatments.”
LGMD2E is a rare, degenerative and universally fatal form of muscular dystrophy caused by a genetic mutation. Progressive muscle fiber loss, inflammation and scarring in these patients causes degeneration of muscle strength and function. One of the leading causes of early mortality is cardiomyopathy caused by damage to the heart tissue.
The Myonexus gene therapy platform, which uses the AAVrh.74 vector system, was evaluated by a research team including Louise Rodino-Klapac, Ph.D. and Jerry R. Mendell, M.D., and colleagues at Nationwide Children’s Hospital for more than 12 years before being licensed to Myonexus, an independent company formed in 2017.
In addition to the MYO-101 for LGMD2E program, Sarepta will acquire MYO-102 for LGMD2D, MYO-103 for LGMD2C, MYO-201 for LGMD2B, and MYO-301 for LGMD2L. All five programs use the same AAVrh.74 vector, which is designed to systemically and robustly deliver treatment to skeletal, diaphragm and cardiac muscle without promiscuously crossing the blood-brain barrier.
About Limb-Girdle Muscular Dystrophy (LGMD)
Limb-girdle muscular dystrophies are genetic diseases that cause progressive, debilitating weakness and wasting that begins in muscles around the hips and shoulders before progressing to muscles in the arms and legs. LGMD2E affects between 1-in-200,000 to 1-in-1,000,000 newborns without regard to sex, race, or national origin. The disease usually presents before age 10 with difficulty running, jumping and climbing stairs. It progresses to loss of ambulation in the teen years, and often leads to death before age 30. There is currently no treatment or cure for LGMD type 2E.
About Myonexus Therapeutics
Myonexus Therapeutics is a clinical stage, rare disease gene therapy company developing first ever treatments for limb girdle muscular dystrophies (LGMDs) based on research at Nationwide Children’s Hospital, a leader in neuromuscular gene therapy discovery and translational research. Founded in 2017, Myonexus is headquartered in New Albany, Ohio. More information is available at myonexustx.com.
William Blair served as financial advisers and Thompson Hine LLP and Fenwick & West LLP served as legal advisers to Myonexus.