“BioEnterprise was critical to the whole incubation of Milo Biotechnology. It is where we envisioned and launched the company, and BioEnterprise supported us from the very beginning.”
Al Hawkins is Founder and CEO of Milo Biotechnology, a clinical-stage start-up developing therapies to strengthen muscle and improve the lives of patients with neuromuscular diseases.
Milo has successfully treated nearly twenty patients, one of the largest totals for any gene therapy, anywhere – by leveraging resources from Cleveland and Columbus seed funds, economic development organizations, disease foundations and Nationwide Children’s Hospital.
Al Hawkins founded Milo Biotechnology in 2011 when he served as a CEO-in-Residence at BioEnterprise. Given the lack of venture capital funding in recessionary times, he was interested in exploring a rare disease space where there were no viable therapies and developing a company that would be primarily foundation-funded and bootstrapped.
“The idea was to take a muscular dystrophy therapy and bring it from animal proof of concept to human proof of concept in a few years with not a lot of funding, without having to bring in institutional money,” said Hawkins.
He attended muscular dystrophy conferences all over the globe in order to learn about the sector and eventually found promising research nearly in his own backyard – at Nationwide Children’s Hospital in Columbus.
Hawkins was impressed with the follistatin program at Nationwide’s Center for Gene Therapy, conducted by Brian Kaspar, Ph.D. and the Center Director Jerry Mendell, M.D. “They had studied muscular dystrophy therapy in mice, had amazing results in non-human primates, and they were ready to start clinical studies,” said Hawkins.
Follistatin is a naturally expressed protein that increases muscle function and prevents fibrosis primarily by blocking other proteins that activate signaling pathways leading to reduced muscle mass and strength. Researchers discovered if you give follistatin to mice you get a stronger mouse, literally “a mighty mouse.”
Al Hawkins and Brian Kaspar founded Milo Biotechnology in late 2011, naming the company after Milo of Croton, a 6th Century B.C. Greek wrestler famed for his strength and athletic prowess. In January 2012, they signed an exclusive license for the technology and clinical trials began at this same time.
Milo Biotechnology’s therapy for multiple muscular dystrophies is based on adeno-associated virus (AAV) delivery of follistatin. AAVs are small, non-pathogenic carriers that can be tailored to deliver long-lasting administration of follistatin via a single dose.
Initial clinical results of the therapy in Becker Muscular Therapy were reported in January 2016 and indicate that the therapy is safe and effective. Milo published new clinical data on Sporadic Inclusion Body Mytosis (SIBM) in March 2017. The company expects to complete Phase l and ll clinical studies in both Becker Muscular Dystrophy and SIBM by end of 2017.
Milo received funding from Cuyahoga County’s North Coast Opportunity Fund, JumpStart, Rev1 Ventures, SBIR and multiple disease foundations. The company was also supported by BioEnterprise and leverages critical clinical, drug manufacturing and regulatory resources from Nationwide Children’s Hospital.