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Ultragenyx, Led by BioMarin Vet, Snags $75M for Ultra-Rare Diseases

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Japan-based Nobel Pharma in 2011, filed an application to start clinical trials in the U.S., and has already completed a Phase I clinical trial of the drug, Sharp says. That trial suggested the drug was safe at about a 6,000 milligram daily dose, which came in the form of four pills taken three times a day, Sharp says.

That may sound like a heavy pill burden for patients, but Sharp notes that the tablets are “coated, vitamin-size pills,” and that the company didn’t get any complaints from patients in the trial. Ultragenyx has also completed enrollment in a mid-stage clinical trial which will give it a sense of how well the drug works. The 45-patient trial, which is randomly assigning patients to the new drug or a placebo, is designed to answer whether the drug is working as desired at both the molecular and clinical level. Researchers are taking biopsy samples to see if the drug is acting the way it’s supposed to in the muscles, and physicians will be testing patients on real-life outcomes that matter to patients, like muscle strength in 10 different muscle groups, and walking ability.

Ultragenyx has a couple of more drug candidates it has in-licensed to build up an internal pipeline. One is an enzyme replacement therapy for mucopolysaccharidosis type 7 (MPS-7), in-licensed from Saint Louis University, slated to enter clinical trials in 2013. The company also has a third compound in its pipeline, but it isn’t yet saying where it’s from, or what it’s for, Sharp says.

With the new money in hand, and a growing slate of clinical development work, Ultragenyx plans to do some hiring, Sharp says. The company currently has 27 employees.

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  • Jimbo

    looks like a real winner in the future. hard to beleive you can get this much money in a series B with only 1 drug in the pipeline