Startup Ultragenyx Snags PiII Program for Growing Rare-Disease Pipeline

Flush from raising more than $100 million, Novato, CA-based startup Ultragenyx Pharmaceutical has stepped up with a new pact to partner on a rare bone-disease drug from Japan’s Kyowa Hakko Kirin.

Under the guiding hand of BioMarin veteran Emil Kakkis, Ultragenyx will step in to co-develop KRN23, an antibody designed to treat X-linked hypophosphatemia which is now wrapping a Phase I/II study at KHK. Ultragenyx will now take over development responsibilities, while sharing costs with the Japanese Pharma as they take aim at the U.S., Canadian and European markets.

“The collaboration combines KHK’s broad antibody-based discovery, manufacturing and development capabilities with our expertise in the clinical development of novel therapeutics for rare genetic diseases,” says Kakkis in a statement. “We look forward to working closely with the KHK team, investigators, and patients on continuing development of KRN23, a much-needed new therapy for XLH patients.”

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