San Francisco Business Times by Ron Leuty, Reporter
Orphan drug developer Ultragenyx Pharmaceutical Inc. has gathered $15.1 million toward a goal of raising $30.1 million, according to a Securities and Exchange Commission filing Monday.
The Novato company, started by former BioMarin Pharmaceutical Inc. Chief Medical Officer Emil Kakkis, has won orphan drug designation for two drugs from the Food and Drug Administration as well as from European drug regulators.
In the United States, the designation cuts FDA fees and allows tax credits for companies that develop drugs which treat diseases with 200,000 or fewer patients in the United States.
Ultragenyx said earlier this month that it started a mid-stage study of UX-001, its extended-release oral tablet for hereditary inclusion body myopathy. The rare neuromuscular disease is caused by a deficiency of sialic acid.
The company's other drug, UX-003, is designed as an enzyme-replacement therapy to treat a metabolic disorder called MPS7, also known as Sly disease or mucopolysaccharidosis type 7.
Ultragenyx's board includes representatives of venture capital firms TPG Biotech, HealthCap, Fidelity Biosciences and Pappas Ventures.