Dyne Therapeutics, Inc. (Nasdaq: DYN), a muscle disease
company focused on advancing innovative life-transforming
therapeutics for people living with genetically driven diseases, is
presenting new preclinical data from its myotonic dystrophy type 1
(DM1) program during the American Society of Gene & Cell
Therapy (ASGCT) 24th Annual Meeting today, including results
demonstrating sustained knockdown of toxic human nuclear DMPK RNA,
the genetic basis of the disease.
“We are excited to present these data at ASGCT, which continue
to validate our FORCE™ platform and our approach to developing a
potential therapy for people living with DM1. In particular, we are
seeing impressive reductions in toxic human nuclear DMPK RNA with
twice the duration and at half the dose compared to the data we
reported in January of this year in the same model,” said Romesh
Subramanian, Ph.D., chief scientific officer of Dyne. “This
reinforces the advantage of the FORCE platform and its potential to
enable targeted delivery of therapeutic oligonucleotides to muscle
and supports our goal of offering monthly or less frequent dosing.
We believe the preclinical hTfR1/DMSXL model that we developed
establishes a new standard to evaluate pharmacodynamics in DM1 and
has the potential for translation to human disease.”
Dyne’s lead DM1 candidate consists of an antigen-binding
fragment antibody (Fab) conjugated to an antisense oligonucleotide
(ASO) to enable targeted muscle tissue delivery to reduce
accumulation of toxic DMPK RNA in the nucleus, release
splicing proteins, allow normal mRNA processing and translation of
normal proteins, and potentially stop or reverse the disease. To
assess the ability of its lead DM1 candidate to reduce toxic human
nuclear DMPK RNA, Dyne developed an innovative
hTfR1/DMSXL mouse model that expresses the human TfR1 and carries a
human DMPK gene that represents a severe DM1 phenotype
with more than 1,000 CTG repeats. In January 2021, Dyne reported
data showing that two doses (2 x 10 mg/kg) of its lead DM1
candidate resulted in significant toxic human
nuclear DMPK knockdown at 14 days. New data being
presented at ASGCT are consistent with these findings, with the
candidate demonstrating an approximately 40 percent reduction in
DMPK heart foci at 14 days.
Dyne expanded its analysis in the hTfR1/DMSXL model to evaluate
the administration of a single, low 10 mg/kg dose of its lead DM1
candidate after 4 weeks. These new data show sustained DMPK
knockdown at 4 weeks: 51 percent in the diaphragm, 46 percent in
both the heart and tibialis anterior, and 42 percent in the
gastrocnemius. Dyne’s candidate was well tolerated in the
hTfR1/DMSXL studies.
Additionally, Dyne is reporting during ASGCT new in vitro
findings from DM1 patient cells with approximately 380 and 2,600
CTG repeats, where its candidate showed a robust, dose-dependent
reduction in DMPK RNA, nuclear foci and correction of splicing
defects as measured by BIN1 exon 11 inclusion. The results in the
cell line with approximately 2,600 CTG repeats are particularly
notable given the severity of DM1 disease represented.
“At Dyne we are focused on delivering disease-modification for
patients, and the DMPK knockdown we are observing in our
hTfR1/DMSXL model is consistent with the range that genetic studies
suggest can be clinically meaningful,” said Joshua Brumm, president
and chief executive officer of Dyne. “These latest findings further
strengthen the dataset we’ve already assembled, showing reduction
in nuclear foci and splicing correction in patient cells, as well
as splicing correction and reversal of myotonia in the
well-validated HSALR in vivo model. We believe we are well
positioned as we continue to advance our DM1 program toward the
clinic.”
Data from Dyne’s DM1 program are being featured during the
following presentations at ASGCT today and will be made available
in the Scientific Publications & Presentations section of
Dyne’s website following the meeting:Presentation:
Splice Correction and Reduction of Toxic DMPK RNA In Vitro and In
Vivo Utilizing Novel Antibody Targeted Antisense Oligonucleotides
Scientific Symposium: Hot Topics and Remaining
Challenges in RNAi and Oligonucleotide Therapy for
2021Time: 10:26 a.m. ET
Oral Presentation: The FORCE™ Platform
Achieves Robust Knock Down of Toxic Human
Nuclear DMPK RNA and Foci Reduction in DM1 Cells and in
Newly Developed hTfR1/DMSXL Mouse Model (Abstract
#247)Session: Oligonucleotide
TherapeuticsTime: 1:15 p.m. ETDM1 Program
Webcast
Dyne will host a live webcast event today at 4:00 p.m. ET to
review the company’s DM1 program and preclinical data, and the
importance of targeting the genetic basis of the disease. Joining
management on the webcast will be Charles Thornton, M.D., the
Saunders Distinguished Professor of Neuromuscular Research at the
University of Rochester. Dr. Thornton has been engaged in bench and
clinical research on myotonic dystrophy for 30 years.
To access the event, please visit the Investors & Media
section of Dyne’s website at least 10 minutes before the start time
in order to register:
https://investors.dyne-tx.com/events/event-details/dm1-program-webcast.
The replay of the webcast will be made available shortly after the
event and remain accessible for 90 days. The corresponding slide
presentation will also be available at the time of the event.
About Myotonic Dystrophy Type 1 (DM1)
DM1 is a rare, progressive, genetic disease that affects
skeletal, cardiac and smooth muscles. It is a monogenic, autosomal
dominant disease caused by an abnormal expansion in a region of
the DMPK gene. The expansion in the number of CTG triplet
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 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 the FORCE™ Platform
The proprietary FORCE™ platform drives Dyne’s efforts to
develop targeted, modern oligonucleotide therapeutics with the
potential to be life-transforming for patients with serious muscle
diseases. Dyne designed the FORCE platform using its deep knowledge
of muscle biology and oligonucleotide therapeutics to overcome the
current limitations in delivery to muscle tissue with the goal of
stopping or reversing disease progression. The FORCE platform
leverages the importance of transferrin 1 receptor, TfR1, in
muscle biology as the foundation for its novel approach. TfR1,
which is highly expressed on the surface of muscle cells, is
required for iron transport into muscle cells. Dyne links
therapeutic payloads to its TfR1-binding fragment antibody (Fab) to
develop targeted therapeutics for muscle diseases.
About Dyne Therapeutics
Dyne Therapeutics is building a leading muscle disease company
dedicated to 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’s broad portfolio of therapeutic candidates
for serious muscle diseases includes programs for
myotonic dystrophy type 1 (DM1), Duchenne muscular dystrophy
(DMD) and 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, 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 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; the
timing of and Dyne’s ability to submit and obtain regulatory
clearance for investigational new drug applications; whether
results from preclinical studies will be predictive of the results
of later preclinical studies and clinical trials; whether Dyne’s
cash resources will be sufficient to fund the Company’s foreseeable
and unforeseeable operating expenses and capital expenditure
requirements; uncertainties associated with the impact of the
COVID-19 pandemic on Dyne’s business and operations; 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.
Contact:
Dyne TherapeuticsAmy Reillyareilly@dyne-tx.com857-341-1203
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