Twelve trial sites recruiting patients with OTC
deficiency in the United States, United Kingdom, Spain, and
Canada
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 the initiation of patient dosing in the company’s
global, multi-center Phase 1/2 clinical trial to evaluate DTX301,
the only AAV gene therapy in clinical testing for the treatment of
patients with Ornithine Transcarbamylase (OTC) Deficiency. 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.
“OTC deficiency is an inherited metabolic disease (IMD) for
which approved therapies are unable to eliminate the risk of
metabolic crises from elevated ammonia. We believe that DTX301,
based on our differentiated AAV8 platform in IMD, holds great
promise for addressing the unmet need among patients, and we look
forward to the continued advancement of the trial,” said Annalisa
Jenkins, MBBS, FRCP, Chief Executive Officer of Dimension. Based on
the current progress, Dimension anticipates reporting initial
clinical data from the Phase 1/2 DTX301 clinical trial by late this
year.
Dimension’s phase 1/2 clinical trial is an open-label,
dose-finding safety study of single ascending doses of DTX301 in
adults with late-onset OTC Deficiency. To evaluate therapeutic
response of DTX301, the trial plans to measure ammonia levels and
other biomarkers, including 13C-acetate, which are established
measures of OTC deficiency disease status and hepatocyte (liver)
ureagenesis capacity, respectively. Additional information about
Dimension’s Phase 1/2 study of DTX301 may be found at
ClinicalTrials.gov, using Identifier NCT: NCT02991144.
“Inherited metabolic diseases can have severe effects on patient
health and quality of life, and in many of these urea cycle
diseases, including OTC deficiency, the only curative option is
liver transplantation, which is often associated with significant
morbidity and mortality,” stated Mark L. Batshaw, M.D., Executive
Vice President, Physician-in-Chief & Chief Academic Officer of
Children’s National Medical Center and head of Dimension’s Clinical
Advisory Board (CAB) for urea cycle disorders.
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 or reducing the occurrence of
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. DTX301 was
granted Orphan Drug Designation in the United States and Europe in
January and March 2016, respectively.
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 a 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 and GSDIa, and a collaboration
with Bayer in hemophilia A. Dimension has initiated a phase 1/2
clinical trial with DTX301 for the treatment of OTC deficiency. 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.
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 timing
and likelihood of achievement of our upcoming development
milestones, including timing of disclosure of data, the expected
progress of or portfolio and programs, and our ability to
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
achieve development milestones, including patient enrollment,
dosing of patients, release of initial data, or regulatory filings;
and the other risks described under the caption "Risk Factors" in
Dimension Therapeutics’ Quarterly Report on Form 10-Q for the
period ended June 30, 2017, 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.
Additional Information about the Proposed Transaction
and Where to Find It
This communication does not constitute an offer to sell or the
solicitation of an offer to buy any securities or a solicitation of
any vote or approval. This document relates to a proposed
transaction between Dimension Therapeutics, Inc. (the “Dimension”)
and REGENXBIO Inc. (“REGENXBIO”), which will become the subject of
a proxy statement/prospectus to be filed with the SEC by Dimension,
and may be deemed to be solicitation material in respect of the
proposed transaction. This document is not a substitute for the
proxy statement/prospectus that Dimension will file with the SEC or
any other documents that Dimension may file with the SEC or send to
stockholders in connection with the proposed transaction. Before
making any voting decision, investors and security holders are
urged to carefully read the proxy statement/prospectus and all
other relevant documents filed or that will be filed with the SEC
in connection with the proposed transaction as they become
available because they will contain important information about
Dimension, REGENXBIO, the proposed transaction and related
matters.
Investors and security holders will be able to obtain free
copies of the proxy statement/prospectus and all other relevant
documents filed or that will be filed with the SEC by Dimension
through the website maintained by the SEC at www.sec.gov.
In addition, investors and security holders will be able to
obtain free copies of the proxy statement/prospectus, once it is
filed, from Dimension by accessing Dimension’s website at
www.dimensiontx.com or upon written request to Dimension
Therapeutics, Inc., 840 Memorial Drive, Cambridge, Massachusetts
02139.
Participants in Solicitation
REGENXBIO, Dimension and their respective directors and
executive officers may be deemed to be participants in the
solicitation of proxies from Dimension’s stockholders in connection
with the proposed transaction. Information regarding Dimension’s
directors and executive officers is contained in the proxy
statement for Dimension’s 2017 Annual Meeting of Stockholders,
which was filed with the SEC on April 14, 2017. You can obtain a
free copy of this document at the SEC’s website at www.sec.gov or
by accessing Dimension’s website at www.dimensiontx.com. Additional
information regarding the interests of those persons and other
persons who may be deemed participants in the proposed transaction
may be obtained by reading the proxy statement/prospectus regarding
the proposed transaction when it becomes available. You may obtain
free copies of this document as described in the preceding
paragraph.
Contacts:
Mary Thistle
Chief Operating Officer
Dimension Therapeutics
617-714-0659
mary.thistle@dimensiontx.com
Burns McClellan, on behalf of Dimension Therapeutics
Media: Justin Jackson
212-213-0006, ext.327
jjackson@burnsmc.com
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