Dimension Therapeutics Announces Initiation of Phase 1/2 Study for DTX101 for the Treatment of Hemophilia B
Receives Positive Opinion on European Orphan Drug Designation
CAMBRIDGE, Mass., Jan. 07, 2016 (GLOBE NEWSWIRE) — Dimension Therapeutics, Inc. (NASDAQ:DMTX), a leading gene therapy company advancing novel, liver-directed treatments for diverse rare diseases, today announced the initiation of a phase 1/2 study of DTX101, the company’s lead therapeutic candidate, for the treatment of patients with hemophilia B. DTX101 is designed to deliver stable expression of blood clotting Factor IX (FIX) in patients with hemophilia B, a rare genetic bleeding disorder resulting from a deficiency in FIX. The current standard of care, chronic replacement of FIX protein through routine intravenous infusion, often does not achieve sustained, durable FIX expression, potentially putting patients at risk for spontaneous bleeds and long term joint destruction. 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.
Dimension also received a positive opinion from the European Commission, acting on the positive recommendation from the European Medicines Agency Committee for Orphan Medicinal Products, designating DTX101 (adeno-associated viral vector serotype rh10 containing the gene for human factor IX) as an orphan medicinal product for the treatment of hemophilia B. Dimension received orphan drug designation and fast track status from the U.S. Food and Drug Administration for DTX101 in hemophilia B last year.
“The progression of the Dimension pipeline into the clinic represents an important milestone for the company,” said Annalisa Jenkins, MBBS, MRCP, Chief Executive Officer of Dimension. “We are pleased with the progress of DTX101, and believe it holds substantial potential to address the need for new, improved therapies for hemophilia B patients who are still struggling to manage the ongoing challenges, including joint destruction and recurrent episodes of bleeding.”
Dimension’s phase 1/2 clinical trial of DTX101 is a single arm, open-label, multi-center study, designed to evaluate the safety, dose and early efficacy of DTX101 in adult patients with moderate/severe to severe hemophilia B. Additional information about Dimension’s Phase 1/2 study of DTX101 may be found at ClinicalTrials.gov, using Identifier NCT: NCT02618915.
Orphan drug designation by the European Commission provides regulatory and financial incentives for companies to develop and market therapies that treat a life-threatening or chronically debilitating condition affecting no more than five in 10,000 persons in the European Union (EU), and where no satisfactory treatment is available. In addition to a 10-year period of marketing exclusivity in the EU after product approval, orphan drug designation provides incentives for companies seeking protocol assistance from the EMA during the product development phase, and direct access to the centralized authorization procedure.
About DTX101
Dimension is developing its lead gene therapy product DTX101 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, Inc.
Dimension Therapeutics, Inc. (NASDAQ:DMTX) is a leading gene therapy 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, mammalian adeno-associated virus (AAV) delivery technology. The company is rapidly progressing its clinical-stage development pipeline, including programs addressing unmet needs for patients suffering from OTC deficiency and GSDIa, a collaboration with Bayer in hemophilia A, and a wholly owned clinical program in hemophilia B. The company targets diseases with readily identifiable patient populations, highly predictive preclinical models, and well-described, and often clinically validated, biomarkers. Founded in 2013, Dimension maintains headquarters in Cambridge, Massachusetts.
For more information, please visit www.dimensiontx.com.
Cautionary Note Regarding Forward-Looking Statements
This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding the progress of Dimension’s portfolio and lead programs. All such forward-looking statements are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include the risks that Dimension’s product candidates, including its lead candidate, DTX101, will not successfully be developed or commercialized; and the risks described under the caption “Risk Factors” in Dimension Therapeutics’ Quarterly Report on Form 10-Q for the quarter ended September 30, 2015 (File No. 001-37601), which is on file with the Securities and Exchange Commission, as well as other risks detailed in Dimension Therapeutics’ subsequent filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and Dimension Therapeutics undertakes no duty to update this information unless required by law.
Burns McClellan, on behalf of Dimension Therapeutics, Inc.
Justin Jackson, 212-213-0006, ext. 327