Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage muscle
disease company focused on advancing innovative life-transforming
therapeutics for people living with genetically driven diseases,
today announced two oral presentations at the Muscular Dystrophy
Association (MDA) Clinical & Scientific Conference being held
March 3-6, 2024, in Orlando, Florida and virtually.
Both presentations will highlight the initial clinical data from
the ACHIEVE and DELIVER trials reported by Dyne in January 2024.
The initial clinical data from the ACHIEVE trial of DYNE-101 in
patients with myotonic dystrophy type 1 (DM1) includes 6-month data
from the 1.8 mg/kg (approximate ASO dose) cohort (n=16) and 3-month
data from the 3.4 mg/kg Q4W cohort (n=16). Initial 6-month clinical
data from the 5 mg/kg (approximate PMO dose) cohort (n=6) of the
DELIVER trial of DYNE-251 in patients with Duchenne muscular
dystrophy (DMD) who are amenable to exon 51 skipping will also be
presented. Additionally, both presentations will include safety
data from multiple cohorts in ACHIEVE and DELIVER trials which was
shared in January.
“We’re excited to present to the rare muscle disease community
initial clinical data from our co-lead programs in DM1 and DMD,
where there remains an urgent and significant need for new and
better treatment options,” said Wildon Farwell, M.D., MPH, chief
medical officer of Dyne. “These data reinforce the potential of
FORCE to deliver for patients, and we look forward to sharing
additional clinical data from our ACHIEVE and DELIVER trials in the
second half of this year.”
Oral Presentations:
Abstract Title: Initial Data from the ACHIEVE Trial of DYNE-101
in Adults with Myotonic Dystrophy Type 1 (DM1)Date and Time:
Wednesday, March 6, 2024, at 10:15 a.m. ETPresenter: Valeria
Sansone, M.D., Ph.D., Clinical and Scientific Director, Clinical
Center NeMO, Milan; Professor of Neurology, University of Milan and
a Principal Investigator for the ACHIEVE Trial
Abstract Title: Initial Data from the DELIVER Trial of DYNE-251
in Males with DMD Mutations Amenable to Exon 51 Skipping Date and
Time: Wednesday, March 6, 2024, at 11:30 a.m.
ETPresenter: Kevin Flanigan M.D., Director, Center for Gene
Therapy, Abigail Wexner Research Institute of Nationwide Children’s
Hospital in Columbus, Ohio and a Principal Investigator for the
DELIVER Trial
The presentations will be available in the Scientific
Publications & Presentations section of Dyne’s
website following the session.
About ACHIEVEACHIEVE is a Phase 1/2 global
clinical trial evaluating DYNE-101, consisting of a 24-week
multiple ascending dose (MAD) randomized placebo-controlled period,
a 24-week open-label extension and a 96-week long-term extension.
The trial, which is designed to be registrational, is enrolling
adult patients with myotonic dystrophy type 1 (DM1) who are 18 to
49 years of age. The primary endpoints are safety and tolerability,
with secondary endpoints of pharmacokinetics and pharmacodynamics,
including change from baseline in splicing, as well as measures of
muscle strength and function. For more information on the ACHIEVE
trial,
visit https://www.clinicaltrials.gov/ (NCT05481879).
About DYNE-101DYNE-101 is an investigational
therapeutic being evaluated in the Phase 1/2 global ACHIEVE
clinical trial for people living with DM1. DYNE-101 consists of an
antisense oligonucleotide (ASO) conjugated to a fragment antibody
(Fab) that binds to the transferrin receptor 1 (TfR1) which is
highly expressed on muscle. It is designed to enable targeted
muscle tissue delivery with the goal of reducing
toxic DMPK RNA in the nucleus, releasing splicing
proteins, allowing normal mRNA processing and translation of normal
proteins, and potentially stopping or reversing the disease
progression. DYNE-101 has been granted orphan drug designation by
the European Medicines Agency and the U.S. Food and Drug
Administration for the treatment of DM1.
About Myotonic Dystrophy Type 1 (DM1)DM1 is a
rare, progressive, genetic disease that affects skeletal, cardiac
and smooth muscle. It is a monogenic, autosomal dominant disease
caused by an abnormal trinucleotide expansion in a region of
the DMPK gene. This expansion of CTG repeats causes toxic
RNA to cluster in the nucleus, forming nuclear foci and altering
the splicing of multiple proteins essential for normal cellular
function. This altered splicing, or spliceopathy, results in a wide
range of symptoms. People living with DM1 typically experience
myotonia and progressive weakness of major muscle groups, which can
affect mobility, breathing, heart function, speech, digestion and
vision as well as cognition. DM1 is estimated to affect more than
40,000 people in the United States and over 74,000 people in
Europe, but there are currently no approved disease-modifying
therapies.
About the DELIVER TrialDELIVER is a Phase 1/2
global clinical trial evaluating DYNE-251, consisting of a 24-week
multiple ascending dose (MAD) randomized placebo-controlled period,
a 24-week open-label extension and a 96-week long-term extension.
The trial, which is designed to be registrational, is enrolling
ambulant and non-ambulant males with Duchenne muscular dystrophy
who are ages 4 to 16 and have mutations amenable to exon 51
skipping. The primary endpoints are safety, tolerability and change
from baseline in dystrophin levels as measured by Western blot.
Secondary endpoints include measures of muscle function, exon
skipping and pharmacokinetics. For more information on the DELIVER
trial,
visit https://www.clinicaltrials.gov/ (NCT05524883).
About DYNE-251DYNE-251 is an investigational
therapeutic being evaluated in the Phase 1/2 global DELIVER
clinical trial for people living with DMD who are amenable to exon
51 skipping. DYNE-251 consists of a phosphorodiamidate morpholino
oligomer (PMO) conjugated to a fragment antibody (Fab) that binds
to the transferrin receptor 1 (TfR1) which is highly expressed on
muscle. It is designed to enable targeted muscle tissue delivery
and promote exon skipping in the nucleus, allowing muscle cells to
create a truncated, functional dystrophin protein, with the goal of
stopping or reversing disease progression. DYNE-251 has been
granted fast track, orphan drug and rare pediatric disease
designations by the U.S. Food and Drug Administration for the
treatment of DMD mutations amenable to exon 51 skipping.
In addition to DYNE-251, Dyne is building a global DMD franchise
and has preclinical programs targeting other exons, including 53,
45 and 44.
About Duchenne Muscular Dystrophy (DMD)DMD is a
rare disease caused by mutations in the gene that encodes for
dystrophin, a protein critical for the normal function of muscle
cells. These mutations, the majority of which are deletions, result
in the lack of dystrophin protein and progressive loss of muscle
function. DMD occurs primarily in males and affects an estimated
12,000 to 15,000 individuals in the U.S. and 25,000 in Europe. Loss
of strength and function typically first appears in pre-school age
boys and worsens as they age. As the disease progresses, the
severity of damage to skeletal and cardiac muscle often results in
patients experiencing total loss of ambulation by their early
teenage years and includes worsening cardiac and respiratory
symptoms and loss of upper body function by the later teens. There
is no cure for DMD and currently approved therapies provide limited
benefit.
About Dyne TherapeuticsDyne Therapeutics is a
clinical-stage muscle disease company focused on advancing
innovative life-transforming therapeutics for people living with
genetically driven diseases. With its proprietary FORCE™ platform,
Dyne is developing modern oligonucleotide therapeutics that are
designed to overcome limitations in delivery to muscle tissue. Dyne
has a broad pipeline for serious muscle diseases, including
clinical programs for myotonic dystrophy type 1 (DM1) and Duchenne
muscular dystrophy (DMD) and a preclinical program for
facioscapulohumeral muscular dystrophy (FSHD). For more
information, please visit https://www.dyne-tx.com, and follow
us on X, LinkedIn and Facebook.
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 Dyne’s strategy, future operations,
prospects and plans, objectives of management, the potential of the
FORCE platform, and the anticipated timelines for reporting data
from the DYNE-101 and DYNE-251 clinical trials, 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. Dyne
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 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 Dyne’s ability to initiate and
enroll patients in clinical trials; whether results from
preclinical studies and initial data from early clinical trials
will be predictive of the final results of the clinical trials or
future trials; whether Dyne’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 Dyne’s filings with the Securities and
Exchange Commission (SEC), including the Company’s most recent Form
10-Q and in subsequent filings Dyne may make with the SEC. In
addition, the forward-looking statements included in this press
release represent Dyne’s views as of the date of this press
release. Dyne anticipates that subsequent events and developments
will cause its views to change. However, while Dyne 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 Dyne’s views as of any date subsequent to the date of
this press release.
Contacts:InvestorsAmy Reillyareilly@dyne-tx.com
857-341-1203
Media Stacy Nartkersnartker@dyne-tx.com 781-317-1938
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