Date: 2018-05-03
Type of information: Development agreement
Compound: MYO-101
Company: Sarepta Therapeutics (USA - MA) Myonexus Therapeutics (USA - MA)
Therapeutic area: Rare diseases - Genetic diseases - Neuromuscular diseases
Type agreement: development
Action mechanism: gene therapy.
Disease: Limb-girdle muscular dystrophy (LGMD)
Details:
- • On May 3, 2018, Sarepta Therapeutics announced that it has entered into an exclusive partnership with Myonexus Therapeutics, 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.
Financial terms:
- 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.
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