Dimension Therapeutics Announces FDA Acceptance Of Investigational New Drug Application And Orphan Drug Designation For Lead Candidate DTX101 In Patients With Hemophilia B

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Dimension Therapeutics, Inc. (“Dimension” or the “Company”), a leading gene therapy platform company focused on discovering and developing new therapeutic products for people living with rare diseases associated with the liver, today announced that the U.S. Food and Drug Administration (FDA) accepted Dimension’s investigational new drug (IND) application and granted orphan drug designation for the Company’s lead product candidate, DTX101, for the treatment of hemophilia B. Dimension expects to initiate a multi-center phase 1/2 study in adults to evaluate DTX101 in adult patients with moderate/severe to severe hemophilia B by the end of 2015.

“We are pleased to achieve these important milestones for our DTX101 program, enabling us to bring our most advanced product candidate into the clinic later this year, as planned”

DTX101 is designed to deliver blood clotting Factor IX (FIX) gene expression in patients with hemophilia B. Hemophilia B is a rare genetic bleeding disorder resulting from a deficiency in FIX. The current standard of care for patients with hemophilia B involves chronic replacement of FIX protein through intravenous infusion. In 2013, the World Federation of Hemophilia estimated there were more than 28,000 hemophilia B patients worldwide, including 4,000 patients in the United States.

The FDA Office of Orphan Products Development grants orphan designation to drugs and biologics intended for treatment of rare diseases or conditions that affect fewer than 200,000 people in the United States. Orphan drug designation provides certain benefits to sponsors planning to test a product for use in a rare disease or condition. These benefits include tax credits for qualified clinical trials costs, and exemption from FDA user fees, and upon approval, the product may quality for seven years of market exclusivity.

“We are pleased to achieve these important milestones for our DTX101 program, enabling us to bring our most advanced product candidate into the clinic later this year, as planned,” said Annalisa Jenkins, MBBS, MRCP, Dimension’s Chief Executive Officer. “We have a broad-based commitment to advancing gene therapy products in hemophilia, a lifelong condition with substantial need for new, improved therapies, and in other rare diseases associated with the liver, including OTC deficiency, GSDIa and hemophilia A.”

About DTX101

Dimension is developing our lead gene therapy product DTX101, which is expected to enter clinical development in 2015, for the treatment of moderate/severe to severe hemophilia B. DTX101 is designed to deliver Factor IX, or FIX, gene expression in a durable fashion, preventing the long-term complications of hemophilia B. Preclinical studies completed to date indicate DTX101 has the potential to be a well-tolerated, effective therapy for hemophilia B.

About Dimension Therapeutics

Dimension Therapeutics, Inc. is a rare disease company focused on discovering and developing new therapeutic products for people living with devastating rare diseases associated with the liver and based on the most advanced adeno-associated virus (AAV) delivery technology. The company is advancing multiple programs toward clinical development, including: programs addressing unmet needs for patients suffering from OTC deficiency and GSDIa; a collaboration with Bayer HealthCare in hemophilia A, and a wholly owned program in hemophilia B, which is expected to enter clinical testing by the end of 2015. Dimension has preferred access to multiple AAV vectors from REGENXBIO. The Dimension team and senior advisors include biotech industry veterans and renowned thought leaders in gene therapy and rare diseases.

For more information please visit www.dimensiontx.com