NOVATO -- Ultragenyx Pharmaceutical Inc. today said it raised $75 million in Series B financing for development of the company's lead clinical testing--stage drugs to treat rare genetic diseases.

Leading the financing was Adage Capital Partners, LP, followed by new investors advised by T. Rowe Price Associates, Inc., Jennison Associates LLC, funds and accounts managed by subsidiaries of BlackRock, Inc, Sanofi-Genzyme, BioVentures and Shire plc. Existing investors TPG Biotech, Fidelity Biosciences, HealthCap and Pappas Ventures also participated in the transaction, according to Ultragenyx, which focuses on development of treatments for rare and ultra-rare genetic disorders.

“We deeply appreciate the support of all our investors, new and existing, and their confidence in our ability to find and efficiently develop compelling new treatments for devastating rare genetic disorders,” said Emil D. Kakkis, M.D., Ph.D., chief executive officer and founder of Ultragenyx.  “This financing transaction is critical to expanding our efforts to deliver profound novel therapies that benefit even more rare disease patients.”   

The new funding helps development of flagship drugs UX001 and UX003 and other undisclosed programs. UX001 is a potential replacement therapy for hereditary inclusion body myopathy, which is a severe muscle disease caused by a deficiency of an enzyme that processes proteins and fats.  Patients typically show progressive muscle weaknesses around 20 to 30 years old. UX001 is currently being investigated in a fully enrolled and randomized phase two clinical study, with results expected sometime in 2013.

The drug  UX003 is an enzyme replacement therapy designed for the treatment of mucopolysaccharidosis (MPS) type 7, an extremely rare form of MPS originally described in 1973 and also called Sly syndrome. MPS 7 is a progressive lysosomal storage disorder that affects many of the body's systems and shares many features common to other MPS diseases, specifically MPS 1 and MPS 2. UX003 is set to enter phase 1/2 clinical study in MPS 7 patients in 2013.

The company said it intends to expand its pipeline through the potential in-licensing of additional products.

“We welcome the participation of such highly regarded public market and strategic investors in this pivotal financing,” said Eran Nadav, PhD, managing director at TPG Biotech and chairman of the board of Ultragenyx, in a statement. “Ultragenyx, led by a distinguished team experienced in rare disease drug development, is building a world-class orphan drug company with a rich pipeline of transformative therapeutics.”

Ultragenyx (415-483-8800, has raised $130 million in two rounds. In June 2011, it raised $45 million in Series A financing.

Cowen and Company served as financial advisor on the latest round of financing. Ropes & Gray LLP served as legal advisor.