Sarepta Therapeutics Acquires Myonexus Therapeutics for $165M to Advance Gene Therapies for Limb-Girdle Muscular Dystrophy

VCBeat (WeChat Official Account: vcbeat) has learned that on February 28, 2019, biotechnology company Sarepta Therapeutics (NASDAQ: SRPT) announced the acquisition of Myonexus Therapeutics for $165 million. Last May, Sarepta Therapeutics expanded its business into limb-girdle muscular dystrophy, simultaneously spending $60 million to secure licenses for five gene therapies from Myonexus Therapeutics and an option to acquire the company.

This acquisition agreement aims to expand Myonexus Therapeutics’ pipeline of five gene therapy programs. The gene therapy platform utilized in these five research programs is licensed from Nationwide Children's Hospital and is designed to deliver comprehensive and robust therapeutic effects to the heart and skeletal muscles (including the diaphragm), while avoiding interaction with the blood-brain barrier.

All five gene therapy candidates are designed to provide corrective treatments for five distinct types of limb-girdle muscular dystrophy (LGMD): LGMD2B, LGMD2C, LGMD2D, LGMD2E, and LGMD2L. Three of these five projects are still in the clinical stage, with MYM-101, a drug for treating LGMD2E, showing the most advanced progress. MYM-101 is a gene therapy candidate aimed at delivering a gene that encodes and restores the β-sarcoglycan protein, with the goal of reconstituting the associated protein complex.

The second fastest-moving project is MYO-102, a therapy targeting LGMD2D or α-sarcoglycanopathy. The FDA granted orphan drug designation to MYO-102 in January this year and required Phase I studies via intramuscular (IM) injection and Phase I/IIa studies using isolated limb perfusion (ILP) to evaluate its safety.

Doug Ingram, CEO of Sarepta Therapeutics, stated in a press release: “The five LGMD gene therapies we are currently developing hold significant potential to save the lives of more patients with rare genetic diseases. Our confidence in these programs stems from the substantial commonalities between our micro-dystrophin gene therapy and the projects at Myonexus Therapeutics, including the use of a platform invented by Nationwide Children’s Hospital, the shared AAVrh74 vector, and similar preclinical safety data to date.”

Michael Triplett, Ph.D., President and Chief Executive Officer of Myonexus, added, “Less than a year ago, we entered into a collaboration with Sarepta, driven by our shared goal of developing therapies for LGMD patients suffering from debilitating and life-threatening conditions. This acquisition agreement reinforces our commitment to rapidly advancing new therapies on behalf of patients who currently have no treatment options.”

Sarepta Therapeutics is a biotechnology company, formerly known as AVI BioPharma. Sarepta Therapeutics is dedicated to developing best-in-class, ribonucleic acid (RNA)-based therapies to treat patients with severe rare diseases and infectious diseases. The R&D team at Sarepta Therapeutics has developed multiple RNA-based therapies, including their lead clinical candidate, eteplirsen, for the treatment of Duchenne muscular dystrophy (a debilitating, life-threatening genetic disorder).

Myonexus Therapeutics is a biotechnology company focused on gene therapies for rare diseases in the clinical stage. Building upon the pioneering gene therapy technologies for muscular dystrophy developed by Nationwide Children’s Hospital, Myonexus Therapeutics has pioneered therapeutic candidates for Limb-Girdle Muscular Dystrophy (LGMD) types 2D, 2B, 2E, 2L, and 2C.

(Compiled by Ning Chen)