Companies to Exercise Purchase Option Agreement Following Positive, Preliminary Data in Limb-Girdle Muscular Dystrophy Type 2E Program.
Myonexus Therapeutics, a clinical-stage gene therapy company developing first-ever corrective gene therapies for Limb-girdle muscular dystrophies (LGMD), announced 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. 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,
said Michael Triplett,
Myonexus’ president and chief executive officer.
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.