Entrada Therapeutics, Inc. (Nasdaq: TRDA) is a clinical-stage
biopharmaceutical company aiming to transform the lives of patients
by establishing a new class of medicines that engage intracellular
targets long considered inaccessible. The Company today announced
positive preliminary data from its Phase 1 clinical trial,
ENTR-601-44-101. Data will be featured in a presentation at the
29th Annual Congress of the World Muscle Society, taking place in
Prague, Czechia from October 8-12, 2024.
“We are excited to present the first clinical data from our
Duchenne franchise, led by ENTR-601-44. ENTR-601-44 was well
tolerated in healthy volunteers and we are pleased to see
significant plasma concentration, muscle concentration and exon
skipping. We achieved the goals of the ENTR-601-44-101 trial,
including the identification of a clinically relevant starting dose
for the planned Phase 2 global patient study. Based on the
cumulative data to date, we expect to see a significant
accumulation of exon skipping and dystrophin production in
patients, which we believe will lead to an improvement in
functional outcomes after multiple doses,” said Dipal Doshi, Chief
Executive Officer at Entrada Therapeutics.
He continued, “Patients are at the core of our mission at
Entrada. We believe that the flexibility of our EEV-based approach
will allow the therapeutic to be tailored to meet the changing
needs of growing pediatric and young adult patients via dosing and
other important parameters. Today’s update represents a clear
validation and differentiation of Entrada’s approach and brings us
one step closer to providing a potential treatment for this
relentlessly progressive neuromuscular disease.”
The primary objective of Entrada’s Phase 1 clinical trial was to
evaluate the safety and tolerability of a single dose of
ENTR-601-44. ENTR-601-44-101 also evaluated pharmacokinetics and
target engagement, as measured by exon skipping in the skeletal
muscle. The study included a total of 32 healthy male volunteers
across four cohorts, with each cohort consisting of six
participants receiving ENTR-601-44 and two participants receiving a
placebo control. The doses administered across the cohorts were
0.75 mg/kg, 1.5 mg/kg, 3 mg/kg and 6 mg/kg.
There were no serious adverse events, no drug-related adverse
events and no clinically significant changes or trends noted in
vital signs, ECGs, physical exams or laboratory assessments
observed in the trial. The study demonstrated target engagement as
measured by exon skipping on a ng/g of tissue adjusted basis
supporting the importance of endosomal escape to therapeutic index
optimization. Muscle concentration was detected in all six subjects
in the 6 mg/kg dose cohort (mean of 53.8 ng/g, range 40 ng/g-73.5
ng/g) and mean target engagement as measured by exon skipping was
0.44% (range 0.3-0.65%). Exon skipping was statistically
significant compared to the placebo control (p<0.005) in the 6
mg/kg dose cohort. These results are based upon data collected to
date and are aggregated based upon the placebo and study drug
groups.
“I am encouraged to see the preliminary results of Entrada’s
Phase 1 clinical trial of ENTR-601-44. These data in healthy
volunteers represent a potentially transformative treatment option
for boys and young men living with Duchenne who are exon 44
skipping amenable, a population for which there are currently no
exon skipping options,” said Francesco Muntoni, MD, Professor of
Paediatric Neurology. “The data on safety generated so far are very
encouraging. This, coupled with the possibility for Entrada’s
EEV-therapeutics to allow for dosing intervals of at least six
weeks, would significantly reduce the burden of the administration
of this novel therapeutic compound and I am sure this information
will be positively received by the patient community.”
Based on the positive preliminary data from the Phase 1 clinical
trial, the Company is on track to submit regulatory applications in
the fourth quarter of 2024 to initiate separate global Phase 2
clinical trials for ENTR-601-44 in patients with Duchenne who are
exon 44 skipping amenable and for ENTR-601-45 in patients with
Duchenne who are exon 45 skipping amenable. In addition, the
Company plans to submit regulatory applications in 2025 to initiate
a global Phase 2 clinical trial for its third Duchenne candidate,
ENTR-601-50, in patients who are exon 50 skipping amenable.
About ENTR-601-44ENTR-601-44, a proprietary
Endosomal Escape Vehicle (EEV™)-conjugated phosphorodiamidate
morpholino oligomer (PMO), is the lead product candidate within
Entrada’s Duchenne muscular dystrophy franchise from its 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 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, is being evaluated for its 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 (DMD)Duchenne
muscular dystrophy is a rare, genetic disease that causes
progressive muscle degeneration and weakness throughout the body.
DMD 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.
DMD 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 DMD seek to improve dystrophin production,
but to date, the clinical benefits of these products have not been
confirmed.
About Entrada TherapeuticsEntrada Therapeutics
is a clinical-stage biopharmaceutical company aiming to transform
the lives of patients by establishing a new class of medicines that
engage intracellular targets that have long been considered
inaccessible. The Company’s Endosomal Escape Vehicle
(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 this proprietary, versatile and modular approach,
Entrada is advancing a robust development portfolio of RNA-,
antibody- and enzyme-based programs for the potential treatment of
neuromuscular, ocular, metabolic and immunological diseases, among
others. The Company’s lead oligonucleotide programs are in
development for the potential treatment of people living with
Duchenne who are exon 44, 45 and 50 skipping amenable. Entrada has
partnered to develop a clinical-stage program, VX-670, for myotonic
dystrophy type 1.
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, the validation and
differentiation of Entrada’s approach and its ability to provide a
potential treatment for patients, the translatability of the
preliminary ENTR-601-44-101 data to the complete data set,
expectations regarding the starting dose for Entrada’s planned
Phase 2 clinical trial for ENTR-601-44, expectations regarding
significant accumulation of exon skipping and dystrophin production
in patients, expectations regarding improvement in functional
outcomes for patients after multiple doses of ENTR-601-44,
expectations regarding the importance of endosomal escape to
therapeutic index optimization, expectations regarding the timing
of regulatory filings for the planned Phase 2 clinical trials for
ENTR-601-44 and ENTR-601-45 in the fourth quarter of 2024, and
ENTR-601-50 in 2025, the ability to recruit for and complete a
global Phase 2 trial for ENTR-601-44, ENTR-601-45 and ENTR-601-50,
the potential of Entrada’s EEV product candidates, including the
potential for ENTR-601-44 to be a transformative treatment option,
and EEV platform, and the continued development and advancement of
ENTR-601-44, ENTR-601-45 and ENTR-601-50 for the treatment of
Duchenne and the partnered product VX-670 for the treatment of
myotonic dystrophy type 1, 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 and initiate clinical trials; whether results
from preclinical studies will be predictive of the results of later
preclinical studies and clinical trials; whether preliminary
clinical data will be predictive of final clinical data; 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 ContactCaileigh
DoughertyHead of Investor Relations & Corporate
Communicationscdougherty@entradatx.com
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