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Dimension Therapeutics Announces Presentations at the Upcoming 21st Congress of the European Hematology Association (EHA)

CAMBRIDGE, Mass., May 19, 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 will highlight the progress of two of Dimension’s lead development candidates in hemophilia B and A, DTX101 and DTX201, respectively, during the 21st EHA Congress, which will take place in Copenhagen, Denmark, June 9-12, 2016. DTX101 is designed to deliver Factor IX, or FIX, gene expression, in adult patients with moderate/severe to severe hemophilia B. An ongoing Phase 1/2 clinical trial of lead candidate DTX101 is anticipated to report initial data in the second half of 2016. DTX201, currently in IND-enabling studies, is designed to deliver Factor VIII, or FVIII, gene expression in adult patients with hemophilia A.

“Presentations at leading peer-reviewed conferences such as EHA are vitally important to advancing the research agenda in gene therapy and to fostering our scientific and medical collaborations with the community of physicians, patients, and regulatory agencies,” said Dr. Annalisa Jenkins, MBBS, FRCP, Chief Executive Officer of Dimension. “The data being reported at EHA result from our highly productive collaboration with the lab of Jim Wilson, MD, PhD, professor of Medicine and Pediatrics at the Perelman School of Medicine at the University of Pennsylvania. We are grateful that our partners at PENN share our focus and deep commitment to innovation and addressing the compelling needs of patients with rare diseases associated with the liver.”

The presentation details are as follows:

Title: A Dose-Escalating Preclinical Study to Determine the Safety, Efficacy, and Minimum Effective Dose of a Clinical Candidate Vector in a Mouse Model of Hemophilia B (Abstract No. EHA-2910)
Session Title: Gene therapy, cellular immunotherapy and vaccination
Session Date & Time: Friday, June 10, 5:15 - 6:45 p.m. CEST
Location: Poster area (Hall H), The Bella Center
Authors: Lili Wang1, John White1, Zhenning He1, Tamara Goode1, Deirdre McMenamin1, Christine Draper1, Cassandra Brown1, Lisa Kattenhorn2, Gwen Wilmes2, K. Reed Clark2, Samuel Wadsworth2, James M. Wilson1 1Gene Therapy Program, Department of Medicine, University of Pennsylvania, Philadelphia, PA, 2Dimension Therapeutics, Cambridge, MA

  • Preclinical research conducted with Dimension’s collaborators at the University of Pennsylvania (“PENN”) demonstrated dose-dependent expression of FIX levels and stability of FIX expression after single dose administration across the majority of doses during the 90-day study period.
  • The study also found there were no apparent safety concerns related to the anticipated clinical dosing.

Title: Optimized AAV-Mediated Human Factor VIII Gene Therapy in Hemophilia A Mice and Cynomolgus Macaques (Abstract No. EHA-2892)
Session Title: Gene therapy, cellular immunotherapy and vaccination
Date & Time: Friday, June 10, 5:15 - 6:45 p.m. CEST
Location: Poster area (Hall H), The Bella Center
Authors: Jenny A. Greig1, Qiang Wang1, Amanda L. Reicherter1, Erin Bote1, Deirdre McMenamin1, Christine Draper1, Shu-Jen Chen1, Alexandra L. Hanlon2, Tamara Goode1, K. Reed Clark3, Samuel Wadsworth3, Lili Wang1, James M. Wilson1 1Gene Therapy Program, Department of Medicine, University of Pennsylvania, Philadelphia, PA, 2School of Nursing, University of Pennsylvania, Philadelphia, PA,3Dimension Therapeutics, Cambridge, MA

  • Preclinical research conducted with Dimension’s collaborators at PENN demonstrated that the selection and combination of specific product components – including the capsid, enhancer, and promoter – further optimized product performance, including long-term expression of Factor VIII (FVIII), the protein missing or deficient in hemophilia A that DTX201 is designed to provide.
  • The FVIII expression levels, achieved during the 30-week course of the study, supported advancement of DTX201 into IND-enabling studies for the potential treatment of patients with hemophilia A.

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. A Phase 1/2 study commenced in January 2016, and the company anticipates reporting interim clinical data in the second half of 2016.

About DTX201

Dimension is developing its gene therapy product DTX201 in partnership with Bayer for the treatment of hemophilia A. DTX201 is designed to deliver Factor VIII, or FVIII, gene expression in a durable fashion, potentially obviating the need for long-term routine intravenous injections of hemophilia A patients. Preclinical studies completed to date indicate DTX201 has the potential to be a well-tolerated, effective therapy for hemophilia A. DTX201 is currently in IND-enabling studies.

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 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 continued progress of Dimension's portfolio and lead programs in hemophilia, including the ongoing Phase 1/2 trial of DTX101 in patients with hemophilia B, and the applicability of preclinical data in hemophilia A to the potential success of Dimension’s partnered program with Bayer for DTX201. 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 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.

Jean Franchi Chief Financial Officer Dimension Therapeutics 617-714-0709 jean.franchi@dimensiontx.com Burns McClellan, on behalf of Dimension Therapeutics Media: Justin Jackson 212-213-0006, ext.327 jjackson@burnsmc.com

Source:Dimension Therapeutics