Milo Biotechnology is developing therapies for the treatment of muscular dystrophy that increase muscle strength or decrease the rate of muscle atrophy. The Company's therapy is based on adeno-associated virus delivery of follistatin, a protein that increases muscle strength and prevents atrophy. Follistatin functions primarily by blocking myostatin, a protein that reduces muscle strength. In nature, reductions in myostatin produce dramatic increases in strength, as shown by myostatin deficient whippets, myostatin-deficient Belgian blue cows and even some humans with documented lower myostatin levels. By blocking myostatin, follistatin increases muscle cell size and also improves recovery after muscle injury. Milo Biotechnologys novel therapy has been successfully tested in mice and macaques and has cleared investigational new drug application (IND)-enabling toxicity studies. Under an FDA-sanctioned IND, the therapy currently is being evaluated in a Phase I/II clinical trial for patients with Becker muscular dystrophy and inclusion body myositis. This initial trial, funded by the foundation Parent Project Muscular Dystrophy, is testing for safety and initial efficacy of follistatin gene therapy delivered locally via quadriceps injections. Milo Biotechnology is concurrently in large animal studies for an additional program to deliver the therapy regionally to multiple muscle groups.
