×

Dimension Therapeutics Announces Orphan Drug Designation of DTX301 for the Treatment of Ornithine Transcarbamylase (OTC) Deficiency

CAMBRIDGE, Mass., Jan. 06, 2016 (GLOBE NEWSWIRE) -- Dimension Therapeutics, Inc. (NASDAQ:DMTX), a leading gene therapy company advancing novel, liver-directed treatments for diverse rare diseases, today announced that the U.S. Food and Drug Administration (FDA) granted orphan drug designation for the Company’s product candidate, DTX301, the first AAV gene therapy to enter IND enabling studies for the treatment of Ornithine Transcarbamylase (OTC) Deficiency. Designed to address the underlying genetic defect, DTX301 delivers stable expression and activity of OTC. DTX301 has been shown in preclinical studies to normalize levels of urinary orotic acid, a marker of plasma ammonia, which if uncontrolled can cause serious neurological and metabolic deficiencies in patients. Dimension has completed candidate selection in its DTX301 program and plans to submit an investigational new drug (IND) application with the FDA in the second half of 2016.

“We are pleased to have received U.S. orphan designation for two of our lead candidates, DTX101 for hemophilia B and now DTX301, recognizing the substantial need for new approaches to hemophilia and inborn errors of metabolism,” said Annalisa Jenkins, MBBS, MRCP, Chief Executive Officer of Dimension. “For OTC deficiency, approved therapies unfortunately do not eliminate the risk of metabolic crises from elevated ammonia, and the only curative treatment is liver transplantation, which is associated with significant risks. We look forward to advancing a treatment alternative that we believe holds great potential for addressing unmet needs for patients.”

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 qualify for seven years of market exclusivity.

Background on OTC Deficiency
OTC deficiency, the most common urea cycle disorder, is caused by a genetic defect in a liver enzyme responsible for detoxification of ammonia. Individuals with OTC deficiency can build-up excessive levels of ammonia in their blood, potentially resulting in neurological deficits and other toxicities. Neonatal onset disease in males is severe and can be fatal at an early age. The greatest percentage of patients experience late-onset disease, representing a clinical spectrum of disease severity. It is estimated that more than 10,000 patients are affected by OTC deficiency worldwide, of which approximately 80% are classified as late-onset, Dimension’s target population. Approved therapies, which must be taken multiple times a day for the patient's entire life, do not eliminate the risk of future metabolic crises. Currently, the only curative approach is liver transplantation.

About Dimension Therapeutics
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 DTX301, 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.

CONTACT: Burns McClellan, on behalf of Dimension Therapeutics Media: Justin Jackson 212-213-0006, ext.327 jjackson@burnsmc.com

Source:Dimension Therapeutics