Pharma Focus Asia

Sarepta Exercises Option to Acquire Myonexus Therapeutics

Thursday, February 28, 2019

Sarepta Therapeutics, Inc., the leader in precision genetic medicine for rare diseases, announced today that it has exercised its option to acquire Myonexus Therapeutics, a clinical-stage biotechnology company developing transformative gene therapies for five LGMDs: LGMD2E, LGMD2D, LGMD2B, LGMD2C and LGMD2L. Subject to satisfaction of closing conditions, Sarepta will pay the Myonexus shareholders $165 million.

LGMDs represent a group of distinct genetic neuromuscular diseases with a generally common set of symptoms, including progressive, debilitating weakness and wasting that begins in muscles around the hips and shoulders before progressing to muscles in the arms and legs. Many LGMD sub-types, including the five programs progressing with Myonexus, are seriously life-limiting and often life-ending diseases.   

In May 2018, Sarepta and Myonexus entered into an exclusive partnership to develop Myonexus’ five LGMD gene therapy candidates, which target the most severe and common forms of the disease. Three of the programs are in clinical development and two are in the pre-clinical stage and ready to progress into the clinic. As part of the agreement, Sarepta had an exclusive option to acquire Myonexus.

As previously announced, Sarepta will host a webcast and conference call at 8:00 am ET today, February 27, 2019, during which the Company will present results from the first 3-patient cohort of the MYO-101 study in patients with LGMD2E. Details to participate in the call are below.

“The five LGMD gene therapies being developed fit brilliantly with Sarepta’s mission to develop therapies with the potential to rescue the lives of patients with serious life-limiting rare genetic diseases,” said Doug Ingram, president and chief executive officer, Sarepta. “Our confidence in these programs has come from the fact that our micro-dystrophin gene therapy and the Myonexus programs have much in common, including inventors from Nationwide Children’s Hospital, a shared vector in AAVrh74 and, to date, similar pre-clinical safety data. We are excited to acquire Myonexus, which will allow us to move rapidly to find solutions for LGMD patients and continue to build out and validate our gene therapy engine.”  

Mr. Ingram continued, “We would also like to take this opportunity to thank Myonexus and Nationwide Children’s Hospital for their contributions, as it was their dedication and tireless efforts that advanced these programs to where they are today.”

Like Sarepta’s micro-dystrophin program, all five Myonexus LGMD sub-type programs employ the AAVrh74 vector, designed to systemically and robustly deliver treatment to cardiac and skeletal muscle, including the diaphragm, without promiscuously crossing the blood brain barrier, making it an ideal candidate to treat muscle disease. The MHCK7 promoter used in MYO-101, which is also used in the micro-dystrophin program, was chosen for 3 of the 5 LGMD programs because it is generally more productive in muscle than other MCK promoters and it robustly expresses in the heart, which is critically important for patients with LGMD2E, LGMD2B, and LGMD2C, many of whom die from pulmonary or cardiac complications.

"We partnered with Sarepta less than a year ago, as we shared the mutual goal of developing LGMD therapies on behalf of patients with debilitating and fatal disease,” said Michael Triplett, Ph.D., president and chief executive officer, Myonexus. “This acquisition solidifies a commitment to rapidly advance therapies on behalf of patients who currently don’t have treatment options.”

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