Rare disease biotech nails down $15.1M in VC

Ultragenyx Pharmaceuticals has reloaded its coffers after the startup developer of rare disease drugs advanced its lead candidate for hereditary inclusion body myopathy into a Phase II study. According to an SEC filing, the Novato, CA-based biotech has raised a fresh $15.1 million.

Hereditary inclusion body myopathy is an uncommon, genetic neuromuscular disorder that robs patients of a functional enzyme needed for sialic acid biosynthesis for modification of fats and proteins, according to Ultragenyx. And the sialic acid deficiency weakens the body, often requiring patients with the disease to get around in a wheelchair. The company's lead drug, UX001, is an extended-release tablet of sialic acid for patients with the condition, for which there are no approved drugs.  

The San Francisco Business Times was the first to report the latest financing at Ultragenyx.

The startup, whose founder and CEO Emil Kakkis was previously medical chief at rare disease drug specialist BioMarin Pharmaceuticals ($BMRN), has so far found strong support from its venture investors. Last summer the biotech announced that TPG Biotech, Fidelity Biosciences, HealthCap and Pappas Ventures contributed to a Series A round of $45 million. Funding has come to the startup amid a major push around the pharma industry into the rare disease business, which offers the ability to charge big sums for drugs that treat small groups of patients. 

Clinical trials for rare disease drugs can often be completed without enrolling hundreds of patients, potentially saving time and money during development. Ultragenyx, for instance, aims to have data from its recently announced Phase II study of UX001 in 2013. The study is expected to enroll 45 patients with the neuromuscular ailment in the U.S. and Israel, with the main goals of measuring safety of the drug and improvements in sialylation biochemistry of muscle over a 24-week period, the company said in early July. 

- here's the SEC filing
- see the SF Business Times article

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