Market Overview

Myonexus Therapeutics Receives Orphan Drug Designation for MYO-102, an Investigational Gene Therapy for Alpha-sarcoglycanopathy (LGMD2D)


Myonexus Therapeutics, announced today that the United States Food and
Drug Administration (FDA) has granted Orphan Drug Designation for
MYO-102, a novel gene therapy candidate for alpha-sarcoglycanopathy,
also known as Limb Girdle Muscular Dystrophy Type 2D (LGMD2D). Myonexus
Therapeutics is a clinical-stage gene therapy company developing
first-ever corrective gene therapies for limb girdle muscular
dystrophies (LGMD) and has licensed the technology from Nationwide
Children's. Nationwide Children's holds the Investigational New Drug
(IND) application for MYO-102.

LGMD2D is a severe, debilitating condition caused by a defect in the
gene that produces the alpha-sarcoglycan protein. Lack of function of
this protein causes inflammation and progressive loss of muscle fiber,
which is replaced with fat and fibrotic scars. LGMD2D is believed to be
the most common sarcoglycanopathy, with conservative estimates
indicating it affects approximately three per million people, regardless
of gender, race, or ethnicity. There is no treatment or cure for LGMD2D.
Experimental gene therapy treatment aims to deliver alpha-sarcoglycan
genes to permanently restore protein expression, which could
significantly improve symptoms and functional ability for patients.

"The Orphan Drug Designation from the FDA is an important milestone in
the development pathway for MYO-102 and reflects its potential to
address a considerable unmet medical need in treating LGMD2D," said
Michael Triplett, Ph.D., President and Chief Executive Officer of
Myonexus. "As we plan for our first systemic human clinical trial, we
aim to build on the promising results of our research to date and offer
hope that our gene therapy candidates may one day be able to transform
the lives of patients and the families who care for them."

The FDA's designation is granted to applications for new therapies that
offer potentially significant benefit over current options for rare
diseases. As part of the development process, this designation provides
for incentives for research in rare disease, including eligibility for a
period of market exclusivity in the U.S. following product approval.

"With ongoing research, we have increasing evidence demonstrating the
potential for gene therapy. We believe this technology holds promise to
address a critical unmet need in treating LGMD," said Dr. Jerry Mendell,
Curran-Peters Chair of Pediatric Research at Nationwide Children's
Hospital. "The FDA's orphan status represents an appreciation of the
need and the potential of this technology to introduce the first major
advance for the LGMD community."

Early clinical studies of MYO-102 have demonstrated safety and
expression of alpha-sarcoglycan protein using the gene therapy
candidate. After treatment with MYO-102, alpha-sarcoglycan expression
increased versus baseline and the protein was present in muscles, as
shown in biopsies six months after treatment.

As part of an ongoing collaboration with Sarepta Therapeutics (see announcement),
Myonexus is developing five gene therapy candidates, all using the same
AAVrh.74 vector, designed to offer the first-ever corrective treatment
for five distinct forms of LGMD. In addition to the clinical research
underway on MYO-101 (see announcement),
and this designation for MYO-102, the other candidates include MYO-103
for LGMD2C, MYO-201 for LGMD2B, and MYO-301 for LGMD2L. For more
information on the MYO-101 trial, please visit
(study identifier NCT03492346).

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 The Research Institute at Nationwide Children's Hospital

Named to the Top 10 Honor Roll on U.S. News & World Report's
2018-19 list of "Best Children's Hospitals," Nationwide Children's
Hospital is one of America's largest not-for-profit freestanding
pediatric health care systems providing wellness, preventive,
diagnostic, treatment and rehabilitative care for infants, children and
adolescents, as well as adult patients with congenital disease. As home
to the Department of Pediatrics of The Ohio State University College of
Medicine, Nationwide Children's faculty train the next generation of
pediatricians, scientists and pediatric specialists. The Research
Institute at Nationwide Children's Hospital is one of the Top 10
National Institutes of Health-funded free-standing pediatric research
facilities in the U.S., supporting basic, clinical, translational and
behavioral health research. The institute houses a Good Manufacturing
Practices (GMP) facility for producing gene- and cell-based therapies;
one of the largest biorepositories in North America; and comprehensive
genome sequencing and analysis capabilities. More information is
available at

View Comments and Join the Discussion!
Don't Miss Any Updates!
News Directly in Your Inbox
Subscribe to:
Benzinga Premarket Activity
Get pre-market outlook, mid-day update and after-market roundup emails in your inbox.
Market in 5 Minutes
Everything you need to know about the market - quick & easy.
Fintech Focus
A daily collection of all things fintech, interesting developments and market updates.
Thank You

Thank you for subscribing! If you have any questions feel free to call us at 1-877-440-ZING or email us at