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 that the European Medicines Agency (EMA) has
granted orphan drug designation for DYNE-101. DYNE-101 is being
evaluated in the Phase 1/2 ACHIEVE global clinical trial in adults
with myotonic dystrophy type 1 (DM1).
“We are pleased to receive orphan drug designation from the EMA
for DYNE-101, further supporting our efforts to develop a
potentially transformative therapy for DM1,” said Wildon Farwell,
M.D., MPH, chief medical officer of Dyne. “The DM1 community has
waited far too long for a therapy that addresses the underlying
cause of this devastating rare muscle disease. We are committed to
advancing DYNE-101 as quickly as possible and anticipate our first
clinical data readout from our ACHIEVE trial later this year.”
The EMA grants orphan drug designation to drugs and biologics
intended for the treatment, diagnosis or prevention of rare,
life-threatening or chronically debilitating diseases or conditions
that affect fewer than five in 10,000 people in the European Union.
Orphan designation allows companies certain benefits, including
reduced regulatory fees, clinical protocol assistance, research
grants and up to 10 years of market exclusivity in the European
Union if approved.
About DYNE-101 and the ACHIEVE Trial
DYNE-101 is an investigational therapeutic being evaluated in
the Phase 1/2 global ACHIEVE trial, for people living with myotonic
dystrophy type 1 (DM1). The ACHIEVE clinical trial consists 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
expected to enroll approximately 72 adult patients with 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. Dyne anticipates
reporting initial data from the MAD placebo-controlled portion of
the ACHIEVE trial on safety, tolerability and splicing in the
second half of 2023.
DYNE-101 consists of an antigen-binding fragment antibody (Fab)
conjugated to an antisense oligonucleotide (ASO) and 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. Dyne
has generated comprehensive preclinical data supporting its DM1
program, including reduction of nuclear foci and correction of
splicing in patient cells, robust knockdown of toxic human
nuclear DMPK RNA and correction of splicing in a
novel in vivo model developed by Dyne, and reversal of
myotonia in a disease model. In non-human primates, DYNE-101
demonstrated a favorable safety profile and achieved enhanced
muscle distribution as evidenced by significant reduction in
wild-type DMPK RNA.
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
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 Dyne Therapeutics
Dyne 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 Twitter, 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, the anticipated timelines for
reporting data from the DYNE-101 clinical trial, the trial design
of the DYNE-101 clinical trial, and the potential benefits of
orphan drug designation , 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 will be predictive of the results
of later preclinical studies and clinical trials; whether Dyne will
benefit from the orphan drug designation referred to above; 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:InvestorsDyne
TherapeuticsAmy Reillyareilly@dyne-tx.com 857-341-1203
MediaDyne TherapeuticsStacy
Nartkersnartker@dyne-tx.com781-317-1938
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