Rare Daily Staff

Sarepta Therapeutics said it has entered into an exclusive partnership worth up to $105 million with Myonexus Therapeutics to develop gene therapies for various forms of limb-girdle muscular dystrophies, a group of rare and fatal muscle-wasting diseases.

Under the terms of the agreement Sarepta will pay Myonexus $60 million in an upfront payment and up to an additional $45 million if all development-related milestones are met over a two-year evaluation period. Sarepta has an exclusive option to acquire Myonexus at a pre-negotiated, fixed price with sales-related contingent payments.

Myonexus has five LGMD gene therapy candidates that it says targets the most severe and common forms of the disease. Three of the programs are in clinical development and the other two are pre-clinical.

The most advanced of Myonexus’ programs, MYO-101, is being developed 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. 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.

“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,” said Doug Ingram, Sarepta’s president and chief executive officer. “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.”

May 4, 2018
Photo: Doug Ingram, Sarepta’s president and chief executive officer

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