Portfolio Company Myonexus Therapeutics Acquired By Sarepta Therapeutics

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Acquisition follows promising preliminary clinical trials results

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Myonexus Therapeutics, a clinical-stage gene therapy company developing first-ever corrective gene therapies for Limb-girdle muscular dystrophies (LGMD), announced today Sarepta will exercise its option to purchase Myonexus for $165 million. The agreement follows positive, preliminary results from the first three patients dosed in the Myonexus Limb-girdle muscular dystrophy (LGMD) 2E program.

  • Read the full release from Myonexus here.
  • Read the full release from Sarepta here.

The acquisition comes less than a year after Myonexus and Sarepta entered into an exclusive partnership last May to develop Myonexus’ five gene therapy candidates for treating different forms of limb-girdle muscular dystrophy (LGMD), a group of rare, degenerative disorders. As part of the agreement announced last year, Sarepta maintained an option to purchase Myonexus at any time at a pre- determined purchase price with sales-related contingent payments. Under the terms of the agreement, Sarepta made an upfront payment of $60 million in addition to payments for achieving certain development milestones.

“From the beginning, the focus of Myonexus has been to identify and advance outstanding science to make a difference in the lives of patients. Our partnership with Sarepta last year and Sarepta’s early acquisition of Myonexus serves to accelerate what we set out to accomplish,” said Michael Triplett, Myonexus’ president and chief executive officer. “The promising, preliminary data validate the potential of our approach to bringing treatments forward for patients with LGMD and our greatest hopes will be realized when patients benefit from approved treatments.”

In late 2017, CincyTech led the seed financing for Myonexus, which is based on pioneering research at Nationwide Children’s Hospital Center for Gene Therapy in Columbus.  The $2.5 million dollar seed round included Rev1 Ventures, The Jain Foundation, GFB ONLUS and initial investors from the LGMD community. This financing enabled Myonexus to initiate systemic Phase 1/2a clinical trials for its initial gene therapy candidate, MYO-101.

“We are gratified by the excellent outcome from this transaction as it again validates our ability to source, finance, monetize breakthrough therapies from our local and regional institutional partners,” said John Rice, CincyTech’s Director of Life Sciences and Board Chair of Myonexus.  “This business success should not overshadow the stunning initial clinical results presented this morning showing successful gene therapy in pediatric patients. This transaction will accelerate the benefit to patients.”

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