Myonexus Therapeutics Announces Accelerated Acquisition by Sarepta Therapeutics

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

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

William Blair served as financial advisers and Thompson Hine LLP and
Fenwick & West LLP served as legal advisers to Myonexus.

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