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- FORCE™ platform enables targeted muscle delivery with lead FSHD program candidate demonstrating potent suppression of DUX4 biomarkers in patient cell line -
WALTHAM, Mass., June 25, 2021 (GLOBE NEWSWIRE) -- 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 today preclinical data from its facioscapulohumeral muscular dystrophy (FSHD) program during the 28th Annual FSHD Society International Research Congress. Data from in vitro studies in an FSHD patient cell line being presented highlight that Dyne's proprietary FORCE™ platform enabled targeted muscle delivery with its lead FSHD candidate demonstrating potent suppression of DUX4 transcriptome markers.
FSHD is caused by aberrant activation of the double homeobox 4 (DUX4) transcription factor in muscle cells, leading to skeletal muscle loss, progressive muscle weakness and wasting. Dyne's approach is designed to address the genetic basis of the disease by reducing DUX4 mRNA expression. The company's lead FSHD candidate consists of a transferrin 1 receptor-binding fragment antibody (Fab) conjugated to a phosphorodiamidate morpholino oligomer (PMO) targeting DUX4 mRNA.
"It is critically important that we continue to advance the science and new therapeutic candidates for FSHD, a highly debilitating, progressive genetic disease with no approved treatments," said Oxana Beskrovnaya, Ph.D., chief scientific officer of Dyne. "We are excited to share these data, which build upon our prior in vitro studies and support our approach to targeting the genetic basis of FSHD. These results demonstrate that our lead FSHD candidate achieved potent suppression of key DUX4 biomarkers, reinforcing the potential of the FORCE platform to enable targeted delivery to muscle tissue. We look forward to advancing our FSHD program toward the clinic as one of the three IND submissions we have planned between the fourth quarter of 2021 and the fourth quarter of 2022."
The in vitro proof of concept studies were conducted using an immortalized FSHD patient cell line. Three well-known DUX4 transcriptome markers MBD3L2, TRIM43, and ZSCAN4 were tracked and a significant increase in expression was observed in these markers once cells were differentiated to myotubes. Dyne's lead FSHD candidate was subsequently evaluated in vitro and showed potent suppression of these DUX4 transcriptome markers with IC50 in the low nanomolar range and also demonstrated superior reduction of these same markers compared to naked (unconjugated) PMO.
Today's presentation entitled, "FORCE™ platform enables muscle-targeted delivery of antisense oligonucleotide and silencing of DUX4 activity in an FSHD cell line" will be available in the Scientific Publications & Presentations section of Dyne's website following the meeting at https://www.dyne-tx.com/our-forcetm-publications/.
About Facioscapulohumeral Muscular Dystrophy (FSHD)
FSHD is a rare, progressive, genetic disease caused by a mutation in the DUX4 gene, leading to skeletal muscle loss, muscle weakness and wasting. In healthy individuals, DUX4-driven gene expression is active for only a short time in early embryonic development. In individuals with FSHD, the DUX4 gene remains "on" long after it is supposed to be silenced. This genetic mutation leads to surplus production of the DUX4 protein, which causes the gradual destruction of muscle cells throughout the body. People living with FSHD experience weakness in all major muscle groups including the face, joint and spinal abnormalities, and often limited mobility. An estimated 16,000-38,000 individuals in the United States and approximately 35,000 in Europe are affected by FSHD, but there are currently no approved disease-modifying treatments.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 estimates of patient populations will accurately reflect the addressable patient populations of our candidates; 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 Therapeutics
Amy Reilly
[email protected]
857-341-1203
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- Co-founder Romesh Subramanian, Ph.D., stepping down as chief scientific officer and will serve as an advisor after leading successful R&D and pipeline-building efforts -
- Oxana Beskrovnaya, Ph.D., appointed CSO -
WALTHAM, Mass., June 02, 2021 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (NASDAQ:DYN), a muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases, today announced that Romesh Subramanian, Ph.D. has chosen to step down as chief scientific officer for personal reasons and will continue to serve as an advisor to Dyne. Concurrently, Oxana Beskrovnaya, Ph.D., who has served as Dyne's senior vice president, head of research since January 2020, has been appointed CSO.
"In co-founding Dyne, Romesh laid the foundation of our efforts to advance modern oligonucleotide therapies that can be effectively delivered to muscle and ultimately transform the lives of people living with serious genetically driven diseases. We are grateful for his many contributions that have helped us build our pipeline, now on the cusp of entering the clinic, and are pleased that he will remain a part of the Dyne family as an advisor," said Joshua Brumm, Dyne's president and chief executive officer. "I'm very excited to have Oxana step into her new role and build on the impressive work she's led since joining us early last year. She has been instrumental in enhancing our scientific expertise, attracting top talent to our research team and progressing our programs for patients in need."
"It has been a great privilege to be part of Dyne and to have accomplished significant milestones – from designing our innovative FORCE™ platform, to generating validating preclinical data, and to now be driving three promising programs toward the clinic," said Dr. Subramanian. "For my family and me, this is the right time to step down as CSO of Dyne, and I am pleased that Oxana is well-positioned to take our programs forward. Dyne has an exciting platform and a very strong team in place. I look forward to seeing our continued progress and supporting the company in any way that I can."
Prior to joining Dyne, Dr. Beskrovnaya served as head of musculoskeletal and renal research in Sanofi's rare disease and neurological unit, advancing a pipeline of drug candidates using multiple therapeutic modalities, including nucleic acids, proteins and small molecules. She is the author of numerous patents, invited reviews, editorials, book chapters and original research articles in major scientific journals. Dr. Beskrovnaya received her Ph.D. in genetics from Moscow Genetics Institute, followed by postdoctoral fellowship training in neuromuscular diseases at the Howard Hughes Medical Institute at the University of Iowa.
"I joined Dyne because of the tremendous opportunity we have to build the world's leading muscle disease company. I'm honored to lead this team and continue the important work ahead of us to deliver new therapeutic options for patients," said Dr. Beskrovnaya.
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, plans, objectives of management, the submission of investigational new drug applications and commencement of clinical development and the sufficiency of its cash resources, 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 and clinical trials; the timing of and Dyne's ability to submit investigational new drug applications; whether results from preclinical studies will be predictive of the results of later preclinical studies and clinical trials; uncertainties related to Dyne's ability to obtain sufficient cash resources to fund the Company's foreseeable and unforeseeable operating expenses and capital expenditure requirements for the anticipated periods; 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 Therapeutics
Amy Reilly
[email protected]
857-341-1203 -
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WALTHAM, Mass., May 26, 2021 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (NASDAQ:DYN), a muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases, today announced that management is scheduled to participate in a fireside chat at the Jefferies Virtual Healthcare Conference on Thursday, June 3, 2021 at 9:00 a.m. ET.
A live webcast will be available in the Investors & Media section of Dyne's website at https://investors.dyne-tx.com/investors-and-media and a replay will be accessible for 90 days following the presentation.
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.
Contact:
Dyne Therapeutics
Amy Reilly
[email protected]
857-341-1203 -
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- Robust Reduction in DMPK RNA in Multiple Muscles at Four Weeks in Novel In Vivo Model Developed by Dyne; Additional In Vitro Data Support Advancement of Lead DM1 Candidate -
- DM1 Program One of Three IND Submissions Planned Between Q4 2021 and Q4 2022 -
- Company to host webcast today at 4:00 p.m. ET -
WALTHAM, Mass., May 14, 2021 (GLOBE NEWSWIRE) -- 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 2021
Time: 10:26 a.m. ETOral 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 Therapeutics
Time: 1:15 p.m. ET
DM1 Program WebcastDyne 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 Therapeutics
Amy Reilly
[email protected]
857-341-1203 -
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- On Track to Submit INDs for DM1, DMD and FSHD Programs Between the Fourth Quarter of 2021 and the Fourth Quarter of 2022 -
- Well-Resourced with Cash Runway Expected into the Second Half of 2024 -
WALTHAM, Mass., May 06, 2021 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (NASDAQ:DYN), a muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases, today reported financial results for the first quarter 2021 and business highlights.
"We continued to make significant progress to start 2021, including generating new preclinical data in our DM1 program that will be featured during the upcoming ASGCT Annual Meeting, further strengthening our leadership team with key appointments and enhancing our financial foundation with cash runway expected into the second half of 2024," said Joshua Brumm, president and chief executive officer of Dyne. "We are focused on executing across all aspects of the business as we drive our three programs to the clinic, advance our efforts to deliver life-transforming therapies to patients and pursue our goal of building the world's leading muscle disease company."
Upcoming Events & Presentations
- New preclinical data from Dyne's myotonic dystrophy type 1 (DM1) program will be featured in presentations during the American Society of Gene & Cell Therapy (ASGCT) 24th Annual Meeting, to be held virtually May 11-14, 2021. The presentations will include new data, expanding upon initial findings reported in January 2021 utilizing an innovative hTfR1/DMSXL mouse model developed by Dyne, as well as results from in vitro studies.
- Romesh Subramanian, Ph.D., chief scientific officer, will present on Splice Correction and Reduction of Toxic DMPK RNA In Vitro and In Vivo Utilizing Novel Antibody Targeted Antisense Oligonucleotides during the "Hot Topics and Remaining Challenges in RNAi and Oligonucleotide Therapy for 2021" scientific symposium, on Friday, May 14, 2021, at 10:26 a.m. ET.
- Stefano Zanotti, Ph.D., director, mechanistic biology, is scheduled to deliver an oral presentation on Friday, May 14, 2021, at 1:15 p.m. ET, entitled 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.
- Following the ASGCT presentations, Dyne plans to host a live webcast event on May 14, 2021 at 4:00 p.m. ET to review the company's DM1 program and preclinical data, and to provide an overview of the disease and treatment challenges. Joining management on the call will be Charles Thornton, M.D., the Saunders Distinguished Professor of Neuromuscular Research at the University of Rochester, and a leading expert in DM1. The live event and replay will be available in the Investors & Media section of Dyne's website at https://investors.dyne-tx.com/investors-and-media.
- Management is scheduled to participate in a fireside chat during Jefferies Virtual Healthcare Conference being held June 1-4, 2021.
- An abstract featuring data from Dyne's FSHD preclinical program has been accepted for an oral presentation during the virtual 28th Annual FSHD Society International Research Congress being held June 24-25, 2021.
First Quarter 2021 Financial Results
Cash position: Cash, cash equivalents and marketable securities were $483.1 million as of March 31, 2021.
Research and development (R&D) expenses: R&D expenses were $18.6 million for the first quarter of 2021 compared to $6.1 million for the first quarter of 2020.
General and administrative (G&A) expenses: G&A expenses were $6.5 million during the first quarter of 2021 compared to $1.8 million for the first quarter of 2020.
Net loss: Net loss was $25.0 million or $0.50 per common share for the first quarter of 2021 compared to $7.9 million, or $0.90 per common share for the first quarter of 2020.
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, plans, objectives of management, the expected timeline for submitting investigational new drug applications and achieving proof-of-concept data readouts and the sufficiency of its cash resources, 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 and clinical trials; the timing of and Dyne's ability to submit investigational new drug applications; whether results from preclinical studies will be predictive of the results of later preclinical studies and clinical trials; uncertainties related to Dyne's ability to obtain sufficient cash resources to fund the Company's foreseeable and unforeseeable operating expenses and capital expenditure requirements for the anticipated periods; 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 Therapeutics
Amy Reilly
[email protected]
857-341-1203Dyne Therapeutics, Inc. Condensed Consolidated Statement of Operations (Unaudited) (in thousands, except share and per share data) Three Months Ended March 31, 2021 2020 Operating expenses: Research and development $ 18,625 $ 6,089 General and administrative 6,509 1,764 Total operating expenses 25,134 7,853 Loss from operations (25,134 ) (7,853 ) Other (expense) income, net 166 (33 ) Net loss $ (24,968 ) $ (7,886 ) Net loss per share—basic and diluted $ (0.50 ) $ (0.90 ) Weighted-average common shares outstanding used in net loss per share—basic and diluted 49,472,497 8,756,513 Dyne Therapeutics, Inc. Condensed Consolidated Balance Sheet Data (Unaudited) (in thousands) March 31, December 31, 2021 2020 Assets Cash, cash equivalents and marketable securities $ 483,085 $ 345,314 Other assets 8,625 8,020 Total assets $ 491,710 $ 353,334 Liabilities and Stockholders' Equity Liabilities 13,450 10,967 Stockholders' equity 478,260 342,367 Total liabilities and stockholders' equity $ 491,710 $ 353,334 - New preclinical data from Dyne's myotonic dystrophy type 1 (DM1) program will be featured in presentations during the American Society of Gene & Cell Therapy (ASGCT) 24th Annual Meeting, to be held virtually May 11-14, 2021. The presentations will include new data, expanding upon initial findings reported in January 2021 utilizing an innovative hTfR1/DMSXL mouse model developed by Dyne, as well as results from in vitro studies.