Entrada Therapeutics Receives Authorization in the United Kingdom to Initiate Phase 1 Clinical Trial of ENTR-601-44 for the Potential Treatment of Duchenne Muscular Dystrophy
August 01 2023 - 7:00AM
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 it has received
authorization from the United Kingdom Medicines and Healthcare
Products Regulatory Agency (MHRA) and Research Ethics Committee
(REC) for its CTIMP (Clinical Trial of an Investigational Medicinal
Product) for a Phase 1 clinical trial in healthy volunteers for
ENTR-601-44. ENTR-601-44 is Entrada’s lead product candidate within
its Duchenne franchise and is being developed for the potential
treatment of individuals with Duchenne who are exon 44 skipping
amenable.
“We are looking forward to this important next step in advancing
ENTR-601-44 for the potential treatment of people with exon 44
skipping amenable Duchenne muscular dystrophy, where there exists a
profound unmet medical need,” said Dipal Doshi, President and Chief
Executive Officer of Entrada Therapeutics. “This milestone, coupled
with the extension of our cash runway through the end of 2025,
positions Entrada to advance our Duchenne franchise while
broadening the potential of our intracellular therapeutics across
serious diseases.”
The Phase 1 trial’s primary objective is to evaluate the safety
of a single dose of ENTR-601-44 in healthy volunteers, with a
target enrollment of approximately 40 participants. The trial will
also evaluate tolerability, pharmacokinetics and target engagement
as measured by exon skipping in the skeletal muscle. The first
participant is expected to be dosed in September of this year with
data anticipated in the second half of 2024.
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 initiate and recruit for 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, the continued development and advancement of ENTR-601-44
and ENTR-601-45 for the treatment of Duchenne, and the sufficiency
of its cash resources through 2025, 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.
Entrada Investor/Media Contact Karla
MacDonald Chief Corporate Affairs
Officer kmacdonald@entradatx.com
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