AAV1-FS344 is a gene therapy technology being developed by Milo Biotechnology for the treatment of Duchenne Muscular Dystrophy as well as other muscle diseases including Becker Muscular Dystrophy and Inclusion Body Myositis. Successful AAV1-FS344 delivery leads to the production of follistatin, a TGF-β inhibitor.
Duchenne Muscular Dystrophy: Phase I/II
Duchenne muscular dystrophy is an X-linked recessive genetic disorder, primarily affecting the muscular system. The disease affects about one in 5,000 males and typically manifests in juveniles prior to the age of 4. It progresses rapidly, leaving most patients unable to walk by age 12 and with an average life expectancy of 26 years.
Milo Biotechnology is engaged in an ongoing Phase I and II clinical trial of AAV1-FS344 in the treatment of Duchenne muscular dystrophy. Final results are expected in early 2017. There is currently no cure for Duchenne, but several competitors are working toward various therapies.
Becker Muscular Dystrophy: Phase I
Becker muscular dystrophy is an X-linked recessive genetic disorder related to Duchenne muscular dystrophy, but much less common. It is a more slowly-progressing muscular dystrophy and affects 1 to 6 per 100,000 births, with symptoms typically manifesting between 8 and 25 years.
Milo Biotechnology released positive results from a Phase I clinical trial of AAV1-FS344 treating Becker Muscular Dystrophy in 2015. The trial is ongoing and final results are expected to be publishedin 2017. There is currently no cure for Becker muscular dystrophy, although treatment through physical therapy and administration of immunosuppresant steroids may slow the progression of the disease.
Inclusion Body Myositis: Phase I
Inclusion body myositis (IBM) is an age-related inflammatory muscle disease. It results in slow wasting and weakness of muscles, primarily in the arms and legs. There are two subtypes of IBM: sporadic and hereditary. Sporadic is the more common, and the one currently being addressed by in clinical trials. sIBM typically manifests itself in patients 50 years or older, and is positively correlated with age. Although the disease is not fatal, most patients require assistive devices within 5 to 10 years of onset, and are at high risk of injury and death due to falls.
Mile Biotechnology published positive results from a Phase I clinical trial of AAV1-Follistatin treating Inclusion Body Myositis were published in 2016. The trial is ongoing and final results are expected in 2017. There is currently no cure for inclusion body myositis, although Novartis is investigating the use of BYM338 to treat the disease.
Managed Care Segment
The managed care segment for AAV1-Follistatin is 2B.
Selected Sample of Epidemiology Sources Used in This Analysis
Alcántara MA, Villarreal MT, Del castillo V, et al. High frequency of de novo deletions in Mexican Duchenne and Becker muscular dystrophy patients. Implications for genetic counseling. Clin Genet. 1999;55(5):376-80.
Romitti et al. Prevalence of Duchenne and Becker muscular dystrophies in the United States. Pediatrics. 2015;135(3):513-521. Pediatrics. 2015;135(5):945.
(See full report for complete source list)
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