NOVATO -- A former BioMarin Pharmaceutical Inc. drug developer has started a new company to create therapies for rare diseases.
Emil Kakkis, recently retired from the position of chief medical officer at BioMarin, launched Ultragenyx Pharmaceutical late last year and is in the process of closing a Series A funding round of more than $10 million.
Dr. Kakkis was a co-developer of an enzyme therapy that BioMarin and Genzyme eventually took to market as Aldurazyme for the treatment of mucopolysaccharidosis, a rare genetic disease.
He went on to develop Naglazyme and Kuvan, treatments for the rare metabolic disease phenylketonuria.
“When you see patients (with rare conditions) treated for the first time, you don’t forget that. You want to do it over and over again,” said Dr. Kakkis.
Orphan drugs, the term for those developed for an extremely small segment of the world’s population, are given a fast pass by the FDA to get them into patients’ hands quickly. The approval process can take less than five years compared to 10 to 12 years for drugs with a wide patient population.
“We’re going to show how fast things can get done with planning and execution,” said Dr. Kakkis, who since retirement two years ago has run Kakkis EveryLife Foundation in Novato with the aim of further streamlining the regulatory process for rare diseases.
Ultragenyx is already at work on a drug for the treatment of a biochemical disorder in skeletal muscle. It has partnered with a small Japanese pharmaceutical to manufacture the drug and do preclinical testing.
Dr. Kakkis hopes to start the clinical tests by July of this year and complete the proof of concept stage before 2013. There are several treatments in the pipeline, according to John Ditton, vice president of commercial development for Ultragenix.
“We’re not quite ready to discuss our plans yet, nor name our funders, but we expect to grow fairly rapidly,” he said.
The company is hiring now for an initial staff of 10 to 12. Within three years if things go according to plan Ultragenyx could employ 100, he said.
“We’re negotiating a lease with room to expand here in Novato and have no plans to locate the company elsewhere.”
BioMarin is located in Novato, as is Raptor Pharmaceuticals, another orphan drug developer founded by former BioMarin employees.