Entrada Therapeutics Announces Updates on ENTR-601-44 in Duchenne Muscular Dystrophy
22 Novembro 2023 - 9:00AM
Entrada Therapeutics, Inc. (Nasdaq: TRDA), a clinical-stage
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
the completion of dosing for the first and second cohorts of its
Phase 1 clinical trial, ENTR-601-44-101. Entrada plans to announce
data from ENTR-601-44-101 in the second half of 2024.
“Our strategy has always been to run a single Phase 1 clinical
trial for ENTR-601-44 and, notably, that trial is progressing in
the United Kingdom. We are pleased to have completed dosing of the
first and second cohorts of participants,” said Dipal Doshi,
President and Chief Executive Officer at Entrada Therapeutics. “In
parallel with the Phase 1 clinical trial, we continue to plan for
the global development of ENTR-601-44 which will include clinical
trials in patients with Duchenne who are exon 44 skipping
amenable.”
The primary objective of Entrada’s double-blind, single
ascending dose Phase 1 clinical trial, which is expected to enroll
approximately 40 participants, is to evaluate the safety and
tolerability of a single dose of ENTR-601-44 in healthy male
volunteers. ENTR-601-44-101 will also evaluate pharmacokinetics and
target engagement, as measured by exon skipping in the skeletal
muscle.
Update on ENTR-601-44 Investigational New Drug (IND)
ApplicationEntrada was previously notified by the FDA that
the IND for the Phase 1 clinical trial of ENTR-601-44 had been
placed on clinical hold. Despite providing additional information
to the FDA, the Company was informed that the Agency declined to
lift the clinical hold. Importantly, the information that was
submitted to the FDA supported the initiation of the Phase 1
clinical trial in the United Kingdom in September 2023.
“We are disappointed that the U.S. clinical hold has not been
lifted, especially given the strength of the data package submitted
to the FDA. It’s important to emphasize that the ongoing
ENTR-601-44 development program continues to progress, with
ENTR-601-44-101 clinical data expected in the second half of 2024.
We will re-engage the FDA to discuss next steps in due course,”
concluded Mr. Doshi.
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 Entrada TherapeuticsEntrada Therapeutics
is a clinical-stage 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, ENTR-601-45 and
ENTR-601-50 for the potential treatment of people living with
Duchenne who are exon 44, 45 and 50 skipping amenable,
respectively, as well as our partnered candidate ENTR-701 for
myotonic dystrophy type 1.
For more information about Entrada, please visit our
website, www.entradatx.com, and follow us on LinkedIn.
Forward-Looking StatementsThis news release
contains forward-looking statements that involve substantial risks
and uncertainties. All statements, other than statements of
historical facts, contained in this news release, including
statements regarding Entrada’s strategy, future operations,
prospects and plans, objectives of management, Entrada’s ability to
continue to recruit for and complete its ENTR-601-44-101 Phase 1
clinical trial in the United Kingdom, expectations regarding the
announcement of data from its ENTR-601-44-101 clinical trial in the
second half of 2024, express or implied statements regarding the
clinical hold on the IND for ENTR-601-44, Entrada’s ability to
address the FDA’s concerns regarding ENTR-601-44, Entrada’s ability
to initiate and recruit for potential global clinical trials for
ENTR-601-44 in patients with Duchenne muscular dystrophy (DMD) who
are exon 44 skipping amenable, expectations regarding the safety
and therapeutic benefits of ENTR-601-44, the continued development
and advancement of ENTR-601-44, ENTR-601-45 and ENTR-601-50 for the
treatment of DMD, and ENTR-701 for the treatment of myotonic
dystrophy type 1 (DM1), the ability to develop additional
therapeutic programs, including further exon skipping programs, the
potential therapeutic benefits of its EEV candidates, 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 studies and clinical trials; the timing of
and Entrada’s ability to submit and obtain regulatory clearance for
INDs or equivalent foreign applications and initiate or complete
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 news
release represent Entrada’s views as of the date of this news
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 news release.
Investor and Media ContactKarla MacDonaldChief
Corporate Affairs Officerkmacdonald@entradatx.com
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