Dimension Therapeutics Announces Orphan Drug Designation of DTX401 for the Treatment of Glycogen Storage Disease Type Ia (GSD...
October 04 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 U.S. Food and Drug Administration (FDA)
granted orphan drug designation for the Company’s product
candidate, DTX401, the first AAV gene therapy to enter IND-enabling
studies for the treatment of GSDIa. Designed to address the
underlying genetic defect, DTX401 delivers stable expression and
activity of the enzyme glucose-6-phosphatase (G6Pase). DTX401 has
been shown to improve G6Pase activity and reduce hepatic glycogen
levels, a well-described biomarker of disease progression, in
multiple in vivo preclinical models.
“We are pleased to receive U.S. orphan designation for our
second inherited metabolic disease candidate, DTX401, recognizing
the substantial need for a new approach to treating individuals
with GSDIa,” said Dr. Annalisa Jenkins, MBBS, FRCP, Chief Executive
Officer of Dimension. “Affected individuals are unable to tolerate
a fast and are at high risk for hypoglycemia, or low blood sugar,
coma and potentially death. Today, there are limited options,
requiring patients to adhere to burdensome dietary regimens in
order to maintain blood glucose at an appropriate level. DTX401
continues to advance in part through a cooperative research and
development agreement, or CRADA, with Janice Chou, Ph.D., and the
Eunice Kennedy Shriver National Institute of Child Health and Human
Development (NICHD) and we look forward to developing 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 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, 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 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
http://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,
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. 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 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 June 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.
CONTACT:
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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