Sarepta Therapeutics Announces Partnership with Myonexus Therapeutics for the Advancement of Multiple Gene Therapy Programs Aimed at Treating Distinct Forms of Limb-Girdle Muscular Dystrophies

-- Sarepta strengthens position as a leader in gene therapy; expands rare disease franchise --

-- The program is designed, if successful, to offer first-ever corrective treatments for five distinct forms of Limb-girdle muscular dystrophy (LGMD) -- 

-- Sarepta is granted exclusive option to acquire Myonexus --

CAMBRIDGE, MA, USA I May 03, 2018 I Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a commercial-stage biopharmaceutical company focused on the discovery and development of precision genetic medicine to treat rare neuromuscular diseases, announced today that it has entered into an exclusive partnership with Myonexus Therapeutics, Inc., a clinical-stage biotechnology company developing transformative gene therapies for various forms of Limb-girdle muscular dystrophies (LGMDs).  Myonexus’ five LGMD gene therapy candidates target the most severe and common forms of the disease and include three clinical and two pre-clinical stage programs.

The most advanced of Myonexus’ programs, MYO-101, aims to treat LGMD2E, also known as beta-sarcoglycanopathy, a severe and debilitating form of LGMD characterized by progressive muscle fiber loss, inflammation and muscle fiber replacement with fat and fibrotic tissue. MYO-101 is designed to transfect a gene that codes for and restores beta-sarcoglycan protein with the goal of restoring the dystroglycan complex.  MYO-101 has generated strong pre-clinical safety and efficacy data utilizing the AAVrh.74 vector system, the same vector used in the micro-dystrophin gene therapy program Sarepta is developing with Nationwide Children’s Hospital. A Phase 1/2a study of MYO-101 is scheduled to begin in mid-2018. The companies plan to report on 60-day biopsy data in late-2018 or early 2019. Additionally, Myonexus is advancing MYO-102 for LGMD2D, MYO-103 for LGMD2C, MYO-201 for LGMD2B, and MYO-301 for LGMD2L.  Like MYO-101, all programs rely upon transfecting a restorative gene utilizing the AAVrh.74 vector. 

Louise Rodino-Klapac, Ph.D., the principal investigator for the Center for Gene Therapy at Nationwide Children’s Hospital and associate professor in the Department of Pediatrics at Ohio State University, is the inventor of the Myonexus portfolio of LGMD candidates and the chief scientific officer and co-founder of Myonexus, a spinout of Nationwide Children’s Hospital, Center for Gene Therapy. Sarepta and Nationwide Children’s are also developing and currently dosing patients in a micro-dystrophin gene therapy for the treatment of Duchenne muscular dystrophy (DMD). Dr. Rodino-Klapac is the co-inventor and serves as one of the principal investigators of the micro-dystrophin program.

Under the terms of the agreement, Sarepta will make an upfront payment of $60 million and additional development-related milestone payments to purchase an exclusive option to acquire Myonexus at a pre-negotiated, fixed price with sales-related contingent payments. If all development-related milestone payments are met, Sarepta will make payments of up to $45 million over an approximately two-year evaluation period. Sarepta has the option to purchase Myonexus at any time, including upon review of proof-of-concept data.

"We are very excited to enter a partnership with an option to acquire Myonexus. Myonexus and its focus on gene therapy using the AAVrh.74 vector to treat forms of LGMD aligns brilliantly with our vision to emerge as one of the most meaningful global precision genetic medicine companies by focusing on the use of genetic medicine to improve the lives of those with rare fatal diseases," stated Doug Ingram, Sarepta’s president and chief executive officer. “We are also delighted to extend our collaboration with Dr. Rodino-Klapac, a renowned thought leader in the field of gene therapy. We share her dedication to rapidly bring life-changing medicines to children suffering and dying from genetic disease.”

Mr. Ingram continued, “With this partnership, we continue to expand our genetic medicine platform of   development compounds from 16 to 21, across RNA and gene therapy development and gene editing research. Further, consistent with the goal we expressed at the January 2018 J.P. Morgan Healthcare Conference, this partnership allows us to leverage our genetic medicine expertise beyond DMD and into new rare diseases in need of therapeutic options.”

Mike Triplett, Myonexus’ president and chief executive officer added, “Our partnership with Sarepta reflects the teams’ mutual commitment to the commercialization of our LGMD product candidates and treating this debilitating and fatal set of diseases.”

Dr. Rodino-Klapac stated, “The culmination of this partnership with Sarepta is important to Myonexus’ mission of rapidly advancing our LGMD pipeline. As the inventor of Myonexus’ LGMD approach and co-inventor of the DMD gene therapy approach, both of which utilize the AAVrh.74 vector system, we are excited to leverage this knowledge from our work in DMD and now apply it to our LGMD portfolio. We are particularly excited to be collaborating again with Sarepta on the development of potentially transformative therapies to treat muscular dystrophies, which represent vast areas of unmet medical need.”

Dr. Rodino-Klapac added, “To date, we are encouraged by the data we have generated in our lead MYO-101 program and I look forward to presenting data from the entire program at Sarepta’s upcoming R&D day.  We intend to initiate our clinical trial by mid-year and soon thereafter generate biopsy data to inform what could be the first-ever corrective treatment for Limb-girdle muscular dystrophy.”

William Blair acted as the exclusive financial advisor and Thompson Hine acted as legal counsel to Myonexus for the transaction.

About Sarepta Therapeutics

Sarepta Therapeutics is a commercial-stage biopharmaceutical company focused on the discovery and development of precision genetic medicine to treat rare neuromuscular diseases. The Company is primarily focused on rapidly advancing the development of its potentially disease-modifying Duchenne muscular dystrophy (DMD) drug candidates. For more information, please visit

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

SOURCE: Sarepta Therapeutics

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