Myonexus Therapeutics Receives FDA Rare Pediatric Drug Designation for Pioneering Treatment of Limb Girdle Muscular Dystrophy Type 2E

Rare Pediatric Disease Designation for MYO-101 Program Reflects
Compelling Data and Enables Priority Review Voucher Eligibility

NEW ALBANY, Ohio–(BUSINESS WIRE)–#GeneTherapyMyonexus
, a clinical-stage gene therapy company developing first
ever corrective gene therapies for limb girdle muscular dystrophies, and
Nationwide Children’s Hospital announced today that the U.S. Food and
Drug Administration (FDA) has granted Rare Pediatric Disease Designation
for the MYO-101 program, an AAV-based gene therapy for the treatment of
limb girdle muscular dystrophy (LGMD) type 2E. In February 2018, the
MYO-101 program was granted Orphan Drug Designation (ODD) by FDA.

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. In LGMD type 2E, disease usually occurs before age 10,
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.

“The FDA’s Rare Pediatric Disease designation for MYO-101 reflects the
compelling data underlying the MYO-101 program and the potential to
provide a first-ever treatment option for LGMD type 2E,” said Michael
Triplett, CEO of Myonexus Therapeutics. “We look forward to initiating
an i.v. systemic Phase 1/2a trial of MYO-101 later this year,
said Bruce Halpryn, COO of Myonexus Therapeutics.

The novel gene therapy is one of five licensed by Myonexus and pioneered
within the lab of Louise Rodino-Klapac, Ph.D. and under clinical
guidance by Jerry Mendell, M.D., at Nationwide Children’s Hospital
Center for Gene Therapy.

Recently, Myonexus and Sarepta Therapeutics, Inc., a commercial-stage
biopharmaceutical company focused on the discovery and development of
precision genetic medicine to treat rare neuromuscular diseases,
announced an exclusive partnership to develop multiple gene therapies to
treat five distinct forms of limb girdle muscular dystrophies (LGMDs),
including LGMD type 2E.

The rare pediatric disease designation indicates that the FDA may give
the company a priority review voucher, if the drug is approved for the
rare pediatric indication. A priority review mandates that the FDA will
review a BLA drug submission within 6 months instead of the standard 10
months. A priority review designation is only given to a drug candidate
that has demonstrated the potential to be a significant improvement in
safety and effectiveness for a serious, unmet disease condition. The
priority review voucher may be used by the sponsor or transferred to
accelerate the review timeline of another drug candidate.

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. Myonexus Therapeutics’ pipeline includes three clinical stage
gene therapy programs (LGMD2E, LGMD2D, and LGMD2B) and two preclinical
gene therapy programs (LGMD2C and LGMD2L). Founded in 2017, Myonexus is
headquartered in New Albany, Ohio. More information is available at

About Nationwide Children’s Hospital

Named to the Top 10 Honor Roll on U.S. News & World Report’s 2017-18
list of “America’s Best Children’s Hospitals,” Nationwide Children’s
Hospital is one of America’s largest not-for-profit freestanding
pediatric healthcare systems providing wellness, preventive, diagnostic,
treatment and rehabilitative care for infants, children and adolescents,
as well as adult patients with congenital disease. Nationwide Children’s
has a staff of nearly 13,000 providing state-of-the-art pediatric care
during more than 1.4 million patient visits annually. As home to the
Department of Pediatrics of The Ohio State University College of
Medicine, Nationwide Children’s physicians train the next generation of
pediatricians and pediatric specialists. The Research Institute at
Nationwide Children’s Hospital is one of the Top 10 National Institutes
of Health-funded freestanding pediatric research facilities. More
information is available at


Nationwide Children’s Hospital:
Gina Bericchia, 614-355-0495

Peg Rusconi, 617-910-6217