CAMBRIDGE, Mass., May 27, 2016 (GLOBE NEWSWIRE) -- Dimension Therapeutics, Inc. (NASDAQ:DMTX), a biopharmaceutical company advancing novel, adeno-associated virus (AAV) gene therapies targeting the liver, a key organ for human metabolism, today announced poster presentations that highlight the progress of Dimension’s development candidate to treat Ornithine Transcarbamylase (OTC) Deficiency, DTX301, during the 8th European Conference on Rare Diseases & Orphan Products (ECRD), which is taking place in Edinburgh, Scotland, May 26-28, 2016. Designed to address the underlying genetic defect in patients, DTX301 delivers stable expression and activity of OTC. In preclinical studies, DTX301 has been shown to normalize levels of urinary orotic acid, a marker of ammonia metabolism, which if elevated, can cause serious neurological deficiencies in patients. Dimension has completed candidate selection in its DTX301 program and plans to submit an investigational new drug (IND) application with the U.S. Food & Drug Administration and initiate patient dosing in the second half of 2016.
“Dimension’s program is designed to address the substantial unmet needs of patients living with OTC deficiency, for whom the only curative treatment is liver transplantation. Approved therapies unfortunately do not eliminate the risk of metabolic crises due to elevated ammonia,” said Dr. Annalisa Jenkins, MBBS, FRCP, Chief Executive Officer of Dimension. “Dimension’s presentations at the ECRD conference this week provide important preclinical validation for DTX301, demonstrating promising activity in in vivo preclinical models of OTC deficiency. These findings result from our highly productive collaboration with the lab of James Wilson, MD, PhD, professor of Medicine and Pediatrics at the Perelman School of Medicine at the University of Pennsylvania (“PENN”). We are encouraged by our progress and look forward to continued advancement of DTX301 toward clinical testing.”
The poster presentation details are as follows:
Title: Long-term Rescue of a Neonatal Lethal Form of OTC Deficiency by Multiple Treatments With AAV Vectors of Different Serotypes (Poster #19)
Session Title: Game Changers in Research
Session Viewing Times Include: Friday, May 27, 9:45 a.m. - 10:15 a.m. and 1:00 p.m. - 2:00 p.m. GMT, and Saturday, May 28, 10:30 a.m. - 11:00 a.m. and 12:30 - 2:00 p.m. GMT
Location: Cromdale Hall, Edinburgh International Conference Centre
Authors: Lili Wang1, Hiroki Morizono2, Peter Bell1, Jianping Lin1, Marcus G. Davey3, Deirdre McMenamin1, Zhenning He1, Hongwei Yu1, Elena Pumbo2, Mark L. Batshaw2, and James M. Wilson1 1Gene Therapy Program, Department of Medicine, University of Pennsylvania, Philadelphia, PA; 2Center for Genetic Medicine Research, Children’s National Medical Research Institute, Washington, DC; 3The Center for Fetal Research at the Children's Hospital of Philadelphia, Philadelphia, PA
Title: AAV Gene Therapy Corrects OTC Deficiency and Prevents the Development of Liver Fibrosis in Aged OTC-KO Heterozygotes Mice (Poster #20)
Session Title: Game Changers in Research
Session Viewing Times Include: Friday, May 27, 9:45 a.m. - 10:15 a.m. and 1:00 p.m. - 2:00 p.m. GMT, and Saturday, May 28, 10:30 a.m. - 11:00 a.m. and 12:30 - 2:00 p.m. GMT
Location: Cromdale Hall, Edinburgh International Conference Centre
Authors: Hiroki Morizono2, Lili Wang1, Peter Bell1, Zhenning He1, Elena Pumbo2, Hongwei Yu1, John White1, Mark L. Batshaw2, and James M. Wilson1 1Gene Therapy Program, Department of Medicine, University of Pennsylvania, Philadelphia, PA; 2Center for Genetic Medicine Research, Children’s National Medical Research Institute, Washington, DC
PENN and Dr. James M. Wilson are each holders of Dimension stock, and Wilson also holds stock in REGENXBIO, Inc. Dimension holds exclusive sublicenses from REGENXBIO, Inc. for certain PENN-owned AAV technologies related to this development program.
About DTX301
Dimension is developing its AAV gene therapy product DTX301 for the treatment of individuals with OTC deficiency. DTX301 is designed to deliver Ornithine Transcarbamylase gene expression in a durable fashion, preventing the complications associated with OTC deficiency. Preclinical studies completed to date indicate DTX301 has the potential to be a well-tolerated, effective therapy for OTC deficiency.
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. 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. The greatest percentage of patients, including males and females, experience late-onset disease, representing a clinical spectrum of disease severity. Neonatal onset disease occurs in males, presents as severe disease, and can be fatal at an early age. 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, Inc.
Dimension Therapeutics, Inc. (NASDAQ:DMTX) is the leader in discovering and developing new therapeutic products for people living with devastating rare diseases associated with the liver, based on the most advanced, mammalian adeno-associated virus (AAV) gene delivery technology. Dimension is actively progressing its broad pipeline, which features programs addressing unmet needs for patients suffering from inherited metabolic diseases, including OTC deficiency, GSDIa, citrullinemia type 1, PKU, Wilson disease, 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 continued progress of Dimension's portfolio and 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 in the times indicated or at all; and the risks described under the caption "Risk Factors" in Dimension Therapeutics’ Quarterly Report on Form 10-Q for the quarter ended March 31, 2016, which is on file with the Securities and Exchange Commission, as well as other risks detailed in Dimension Therapeutics’ additional 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.