Milo Biotechnology's Follistatin Gene Therapy Increases Function in Becker Muscular Dystrophy Patients

Oct 20, 2014, 11:07 ET from Milo Biotechnology

CLEVELAND and COLUMBUS, Ohio, Oct. 20, 2014 /PRNewswire-USNewswire/ -- Data published today in the journal Molecular Therapy demonstrates initial clinical proof of principle of a gene therapy to increase muscle strength and function in Becker muscular dystrophy patients.  The paper, titled "A Phase I/IIa Follistatin Gene Therapy Trial for Becker Muscular Dystrophy" describes results of the dose ascending trial to assess safety and efficacy in six patients with Becker muscular dystrophy. 

All six of the patients were followed at least six months and three of the patients were followed for one year with no drug-related adverse events reported.  On average, the patients showed statistically significant improvement in six minute walk distance and in stair climbing tests.  Additionally, muscle biopsy histology demonstrated reductions in muscle fibrosis and central nucleation, and improved muscle fiber regeneration.  Muscle satellite cells were not reduced.    

Jerry Mendell, M.D., Professor at Nationwide Children's Hospital and lead author on the trial and manuscript stated, "This is the first gene therapy clinical trial to demonstrate functional improvement in any form of muscular dystrophy, and a major advance for those suffering with muscle disease."  Brian Kaspar, Ph.D., Associate Professor at Nationwide Children's Hospital, author and compensated advisor and scientific founder of Milo said, "Upon evaluating the top line data in this trial, we are particularly impressed with the effects of our gene therapeutic to express follistatin long term, not only for the functional improvement, but also in improving the state of muscle health in the patients analyzed."

The therapy, developed at Nationwide Children's Hospital by Dr. Mendell and Dr. Brian Kaspar, is based on adeno-associated virus delivery of follistatin 344 to increase muscle strength and prevent muscle wasting.  It could have broad ranging applications in muscular dystrophies, cancer treatment induced muscle wasting and age related muscle wasting. The clinical study, conducted at Nationwide Children's Hospital and funded by the foundation Parent Project MD, is also evaluating safety and efficacy in nine patients with inclusion body myositis.  Results of the trial in inclusion body myositis will be published in mid 2015.

The underlying intellectual property was exclusively licensed in 2012 from Nationwide Children's Hospital to Ohio-based start-up Milo Biotechnology.  Milo Biotechnology has received FDA orphan designation for Becker muscular dystrophy and for Duchenne muscular dystrophy.

Milo Biotechnology CEO Al Hawkins stated "This significant and long lasting clinical effect provides the basis for planning pivotal clinical studies beginning in 2015; we want to speed delivery of this important therapy to patients with debilitating muscular dystrophies."

About Milo Biotechnology
Milo Biotechnology is a clinical stage startup company developing therapies to strengthen muscle and improve the lives of patients with neuromuscular diseases.  Its lead program, AAV1-FS344, leads to the local expression of follistatin, a potent myostatin inhibitor. Milo's core technology was developed at and is exclusively licensed from Nationwide Children's Hospital in Columbus, Ohio; the company is based in Cleveland, Ohio, and has received funding from JumpStart Inc., the Cuyahoga County North Coast Opportunities Fund and from NIAMS Rare Disease SBIR program. Visit for more information


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SOURCE Milo Biotechnology