Orphan Drug Designation Granted to Investigational Gene Therapy for Rare Form of Muscular Dystrophy

January 2, 2019

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.

“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.”

“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.

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.

As part of an ongoing collaboration with Sarepta Therapeutics, 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 www.clinicaltrials.gov (study identifier NCT03492346).


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