Entrada Therapeutics Announces First Participant Dosed in its Phase 1 Clinical Trial of ENTR-601-44 for the Potential Treatment of Duchenne Muscular Dystrophy
21 Setembro 2023 - 8:00AM
Entrada Therapeutics, Inc. (Nasdaq: TRDA), a biopharmaceutical
company aiming to transform the lives of patients by establishing
intracellular Endosomal Escape Vehicle (EEV™)-therapeutics as a new
class of medicines, today announced that the first participant has
been dosed in its Phase 1 clinical trial evaluating ENTR-601-44 for
the potential treatment of individuals with Duchenne muscular
dystrophy who are exon 44 skipping amenable.
“The initiation of our Phase 1 trial for
ENTR-601-44 is an important step forward in addressing this
relentlessly progressive neurodegenerative disease,” said Dipal
Doshi, President and Chief Executive Officer of Entrada
Therapeutics. “Today’s announcement marks Entrada’s transition into
a clinical company. As we enter our next phase of growth, Entrada
is well-positioned to advance this initial program, as well as our
broader Duchenne franchise, and we expect to report data from the
Phase 1 trial in the second half of 2024.”
The primary objective of Entrada’s Phase 1
clinical trial, which is being conducted in the United Kingdom, is
to evaluate the safety and tolerability of a single dose of
ENTR-601-44 in healthy male volunteers, with a target enrollment of
approximately 40 participants. The trial will also evaluate
pharmacokinetics and target engagement, as measured by exon
skipping in the skeletal muscle.
About ENTR-601-44 ENTR-601-44, a
proprietary Endosomal Escape Vehicle (EEV™)-conjugated
phosphorodiamidate morpholino oligomer (PMO), is the lead product
candidate within its Duchenne franchise from Entrada’s growing
pipeline of EEV-therapeutics. Each EEV-PMO therapeutic candidate
has an oligonucleotide sequence designed and optimized for the
specific subpopulation of interest. ENTR-601-44 is designed to
address the underlying cause of Duchenne muscular dystrophy due to
mutated or missing exons in the DMD gene. ENTR-601-44, an
investigational therapy for the potential treatment of people
living with Duchenne who are exon 44 skipping amenable, has the
potential to restore the mRNA reading frame and allow for the
translation of dystrophin protein that is slightly shortened but
still functional.
About Duchenne Muscular Dystrophy
Duchenne muscular dystrophy is a rare genetic disease that causes
progressive muscle degeneration and weakness throughout the body.
Duchenne is caused by mutations in the DMD gene, which leads to
inadequate production of dystrophin, a protein essential to
maintaining the structural integrity and function of muscle cells.
Duchenne causes progressive loss of muscle function throughout the
body, which limits mobility and causes heart and respiratory
complications in the later stages of the disease. Currently
approved therapies for Duchenne seek to improve dystrophin
production, but to date, the clinical benefits of these products
have not been confirmed.
About Entrada Therapeutics
Entrada Therapeutics is a biopharmaceutical company aiming to
transform the lives of patients by establishing a new class of
medicines, Endosomal Escape Vehicle (EEV™)-therapeutics, to engage
intracellular targets that have long been considered inaccessible
and undruggable. The Company’s EEV therapeutics are designed to
enable the efficient intracellular delivery of a wide range of
therapeutics into a variety of organs and tissues, resulting in an
improved therapeutic index. Through its proprietary, highly
versatile and modular EEV platform, Entrada is building a robust
development portfolio of RNA-, antibody- and enzyme-based programs
for the potential treatment of neuromuscular, immunological, ocular
and metabolic diseases, among others. The Company’s lead
oligonucleotide programs include ENTR-601-44 and ENTR-601-45 for
the potential treatment of people living with Duchenne who are exon
44 and 45 skipping amenable, respectively, as well as our partnered
candidate ENTR-701 targeting myotonic dystrophy type 1 (DM1).
For more information about Entrada, please visit
our website, www.entradatx.com, and follow us on LinkedIn.
Forward-Looking Statements This
press release contains forward-looking statements that involve
substantial risks and uncertainties. All statements, other than
statements of historical facts, contained in this press release,
including statements regarding Entrada’s strategy, future
operations, prospects and plans, objectives of management, ability
to recruit for and complete a healthy volunteer trial for
ENTR-601-44 in the United Kingdom with first subject dosed in
September 2023, expectations regarding the timing of data from its
Phase 1 trial for ENTR-601-44 in the second half of 2024,
expectations regarding the safety and therapeutic benefits of
ENTR-601-44, the potential of its EEV product candidates and EEV
platform, and the continued development and advancement of
ENTR-601-44 and ENTR-601-45 for the treatment of Duchenne,
constitute forward-looking statements within the meaning of The
Private Securities Litigation Reform Act of 1995. The words
“anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,”
“intend,” “may,” “might,” “objective,” “ongoing,” “plan,”
“predict,” “project,” “potential,” “should,” or “would,” or the
negative of these terms, or other comparable terminology are
intended to identify forward-looking statements, although not all
forward-looking statements contain these identifying words. Entrada
may not actually achieve the plans, intentions or expectations
disclosed in these forward-looking statements, and you should not
place undue reliance on these forward-looking statements. Actual
results or events could differ materially from the plans,
intentions and expectations disclosed in these forward-looking
statements as a result of various important factors, including:
uncertainties inherent in the identification and development of
product candidates, including the conduct of research activities
and the initiation and completion of preclinical studies and
clinical trials; uncertainties as to the availability and timing of
results from preclinical and clinical studies; the timing of and
Entrada’s ability to submit and obtain regulatory clearance for IND
applications and initiate clinical trials; whether results from
preclinical studies will be predictive of the results of later
preclinical studies and clinical trials; whether Entrada’s cash
resources will be sufficient to fund the Company’s foreseeable and
unforeseeable operating expenses and capital expenditure
requirements; as well as the risks and uncertainties identified in
Entrada’s filings with the Securities and Exchange Commission
(SEC), including the Company’s most recent Form 10-K and in
subsequent filings Entrada may make with the SEC. In addition, the
forward-looking statements included in this press release represent
Entrada’s views as of the date of this press release. Entrada
anticipates that subsequent events and developments will cause its
views to change. However, while Entrada may elect to update these
forward-looking statements at some point in the future, it
specifically disclaims any obligation to do so. These
forward-looking statements should not be relied upon as
representing Entrada’s views as of any date subsequent to the date
of this press release.
Investor and Media Contact Karla
MacDonald Chief Corporate Affairs Officer
kmacdonald@entradatx.com
Entrada Therapeutics (NASDAQ:TRDA)
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