, a BioEnterprise
startup created to find therapies for neuromuscular diseases, received FDA orphan drug designation for its AAV1-FS344, a drug that increases muscle strength.
The drug is a myostatin inhibitor that produces the protein follistatin, which increases muscle strength. Milo is focused on using the drug for treatment of Becker and Duchenne muscular dystrophy. In both types of the disease, patients have progressive muscle weakness and cardiac and respiratory degeneration. The drug also has potential uses in muscle degeneration in AIDS and cancer patients, but Milo's initial focus is on muscular dystrophy.
Orphan drug classification is given to therapies that treat diseases that affect less than 200,000 people nationwide. “Orphan says two things: One, it says this compound looks like it’s effective in some model of whatever disease it’s treating,” explains Al Hawkins, Milo CEO. “Second, it means that the target population is under 200,000 patients." The designation gives expedited regulatory review, seven years of post-market exclusivity and it qualifies for an FDA grant program in clinical trials.
Milo was founded about a year ago, after receiving a $250,000 investment from JumpStart. The company also received funding from the North Coast Opportunities Technology fund
Milo's drug is being used in clinical trials at Nationwide Children’s Hospital in Columbus with patients who have Becker muscular dystrophy and inclusion body myositis. Hawkins says the designation will expedite development of the therapy.
“There are no approved drugs for muscular dystrophy, but there are a lot of promising therapies in development,” says Hawkins. “The community has banded together in supporting this, but we are still years away from getting to market.”
Source: Al Hawkins
Writer: Karin Connelly