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 U.S. Food and Drug Administration (FDA)
has granted orphan drug designation for DYNE-101 for the treatment
of myotonic dystrophy type 1 (DM1). DYNE-101 is being evaluated in
the Phase 1/2 global ACHIEVE clinical trial with initial data on
safety, tolerability and splicing from the multiple ascending dose,
placebo-controlled portion of the trial anticipated in the second
half of 2023.
“Receiving FDA orphan drug designation for DYNE-101 underscores
the importance of bringing new treatment options to people living
with DM1, a rare, devastating disease with no approved therapies.
We believe DYNE-101 has the potential to be a transformative
therapy which is why we designed the ACHIEVE trial to be
registrational,” said Wildon Farwell, M.D., MPH, chief medical
officer of Dyne. “We recognize the sense of urgency within the
DM1 community and look forward to sharing initial data from the
ACHIEVE trial.”
Orphan drug designation is granted by the FDA to drugs or
biologics intended for treatment, prevention or diagnosis of a rare
disease or condition that affects fewer than 200,000 people in the
United States. Under the Orphan Drug Act, orphan drug designation
qualifies a company for incentives, including tax credits,
exemptions from certain FDA fees for clinical trials, and the
potential for seven years of market exclusivity following drug
approval.
About DYNE-101
DYNE-101 is an investigational therapeutic being evaluated in
the Phase 1/2 global ACHIEVE clinical trial for people living with
myotonic dystrophy type 1 (DM1). 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. DYNE-101 was also granted orphan drug
designation by the European Medicines Agency for the treatment of
DM1.
About ACHIEVE
ACHIEVE 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 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. For more
information on the ACHIEVE trial, visit
https://www.clinicaltrials.gov/ (NCT05481879).
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 seen with other
approaches. 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 any potential benefit from
receiving 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,” “on track,”, “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; 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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