Dimension Therapeutics Announces FDA Acceptance of Investigational New Drug Application for DTX301 to Treat OTC Deficiency
December 13 2016 - 08:00AM
On Track to Initiate Phase 1/2 Global,
Multi-Center Clinical Trial by Year End 2016
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)
accepted Dimension’s investigational new drug (IND) application for
DTX301 for the treatment of Ornithine Transcarbamylase (OTC)
Deficiency. Dimension anticipates commencing a global, multi-center
Phase 1/2 clinical trial to evaluate DTX301 in patients with OTC
Deficiency by the end of 2016.
DTX301 is designed to deliver stable expression and
activity of OTC following a single intravenous infusion and has
been shown in academic preclinical studies in relevant mouse models
to normalize levels of urinary orotic acid, a marker of ammonia
metabolism. In the late onset form of the disease, elevated ammonia
can lead to significant medical issues for patients who are in need
of better disease-modifying therapies.
“FDA’s acceptance of DTX301’s IND for the treatment
of OTC Deficiency is a key pipeline milestone for Dimension. We are
pleased to continue the momentum and advancement of our leading
pipeline that addresses important inherited metabolic disorders of
the liver,” said Dr. Annalisa Jenkins, MBBS, FRCP, Chief Executive
Officer of Dimension. ”For individuals with OTC deficiency, current
medical options are not curative and unfortunately do not eliminate
the risk of metabolic crises. We believe DTX301 holds great promise
as an AAV-based approach for patients, and we look forward to
partnering with leading physician thought leaders and the patient
community across multiple global clinical sites.”
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 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 development, preclinical results, and the continued
progress of Dimension's portfolio and programs, including the
initiation, timing, scope, or likelihood of regulatory filings and
approvals, 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 its
candidate, 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
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.
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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