Dimension Therapeutics Receives Positive Opinion for European Orphan Drug Designation of DTX401 for the Treatment of Glycogen...
November 29 2016 - 8:00AM
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 that the European Medicines Agency (EMA) Committee
for Orphan Medicinal Products (COMP) has issued a positive opinion
recommending DTX401 for designation as an orphan medicinal product
for the treatment of Glycogen Storage Disease Type Ia (GSDIa).
The positive opinion for DTX401 is a key milestone for
Dimension’s leading and unique program advancing the opportunity
for AAV gene therapy for patients with limited treatment options to
manage their disease. Designed to address the underlying genetic
defect, DTX401 has been shown in multiple in vivo preclinical
models to deliver stable expression and activity of the enzyme
glucose-6-phosphatase-α (G6Pase-α). DTX401 has also been shown to
improve G6Pase-α activity and reduce hepatic glycogen levels, a
well-described biomarker of disease progression. Dimension has
completed candidate selection for its DTX401 program
and is currently in IND-enabling studies.
“We are pleased with the progress of
our DTX401 program, which has followed a
research pathway that includes studies in highly
predictive preclinical in vivo models, application
of well-described biomarkers, and the development of a proprietary
manufacturing platform,” said Dr. Annalisa Jenkins,
MBBS, FRCP, Chief Executive Officer of Dimension. “In our
current IND-enabling studies, we are replicating the
groundbreaking work of leading researchers, particularly
through our cooperative research and development
agreement, or CRADA, with Janice Chou, Ph.D., at the Eunice Kennedy
Shriver National Institute of Child Health and Human Development
(NICHD) at the National Institutes of Health (NIH).”
David A. Weinstein, MD, who is joining Connecticut’s UConn
School of Medicine and Connecticut Children’s Medical Center in
early 2017 and is the chair of Dimension’s GSDIa clinical advisory
board, added, “The COMP positive opinion in the EU is an important
milestone as it represents a key step forward for a potential
treatment for GSDIa. Coupled with recent orphan drug designation
for DTX401 in the US, the opinion affirms the substantial need for
patients with GSDIa, who currently have limited therapeutic
options. Affected individuals are at high risk for hypoglycemia
(low blood sugar), coma, and potentially death, and are required to
adhere to burdensome dietary regimens in order to maintain blood
glucose at an appropriate level. Gene therapy is designed to
address these challenges and offer a new option to patients.”
The COMP issues an opinion on the granting of orphan drug
designation, after which the opinion is submitted to the European
Commission (EC) for endorsement of the opinion. Orphan drug
designation by the EC 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.
Background on GSDIa
GSDIa, the most common genetically inherited glycogen storage
disease, has significant unmet needs. Patients have a defective
gene for the enzyme glucose-6-phosphatase-α, resulting in the
inability to regulate blood sugar (glucose). Hypoglycemia in GSDIa
patients can be life-threatening, while the accumulation of the
complex sugar glycogen in certain organs and tissues can impair the
ability of these tissues to function normally. If chronically
untreated, patients develop severe lactic acidosis, can progress to
renal failure, and die in infancy or childhood. There are no
approved pharmacological therapies, and no ongoing clinical
development beyond dietary approaches. An estimated 6,000 or more
patients are affected by GSDIa worldwide.
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 and metabolic 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
clinical and developmental progress of Dimension's portfolio and
lead programs, including DTX401, the timing, scope, or likelihood
of regulatory filings and approvals, the progress and results of
preclinical studies, and our ability to develop and advance product
candidates into, and successfully complete, clinical studies and
the continued development of our manufacturing platform. 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
candidate, DTX401, 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
September 30, 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.
Contacts:
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
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