DYN Dyne Therapeutics Inc.
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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.
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WALTHAM, Mass., April 27, 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 new preclinical data from its 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. ASGCT abstracts are available on the meeting website, and Dyne's presentations are noted below.
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
Date/Time: Friday, May 14, 2021 at 10:26 a.m. ET
Presenter: Romesh Subramanian, Ph.D., Chief Scientific OfficerOral Presentation: The FORCETM 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
Date/Time: Friday, May 14, 2021 at 1:15 p.m. ET
Presenter: Stefano Zanotti, Ph.D., Director, Mechanistic BiologyThe presentations will include new data, expanding upon initial findings reported in January 2021 utilizing Dyne's innovative hTfR1/DMSXL mouse model expressing human TfR1 and carrying a human DMPK gene that represents a severe DM1 phenotype with more than 1,000 CTG repeats, as well as results from in vitro studies.
Dyne's 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. Dyne's lead DM1 candidate consists of a Fab conjugated to an antisense oligonucleotide (ASO) to enable targeted delivery to muscle tissue 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 disease progression.
DM1 Program Webcast
Following the presentations on May 14, 2021, Dyne plans to host a live webcast event 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 webcast will be Charles Thornton, M.D., the Saunders Distinguished Professor of Neuromuscular Research at the University of Rochester. Dr. Thornton directs the Muscular Dystrophy Cooperative Research Center in Rochester and the Myotonic Dystrophy Clinical Research Network, a multicenter consortium for clinical research and therapeutic trials. He has been engaged in bench and clinical research on myotonic dystrophy for 30 years.
The live and archived webcast can be accessed in the Investors & Media section of Dyne's website at: https://investors.dyne-tx.com/news-and-events/events-and-presentations. The archived webcast will be made available shortly after the event and accessible for 90 days.
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 approval 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; 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 Annual Report on Form 10-K for the year ended December 31, 2020 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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- Preclinical Data Further Validate FORCE™ Platform; Driving Toward IND Submissions for DM1, DMD and FSHD Programs Between Q4'21 and Q4'22 -
- Recent Appointments to Experienced Leadership Team Strengthen Company's Clinical Development Capabilities -
- Successful Financings Expected to Provide Cash Runway into the Second Half of 2024 -
WALTHAM, Mass., March 04, 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 fourth quarter and full year 2020 and recent business highlights.
"2020 was a transformational year for Dyne. We delivered validating data for our FORCE™ platform and programs, further strengthened our exceptional team with the addition of highly experienced leaders and completed our IPO. Together with earlier financings in 2020 and the follow-on public offering completed in January 2021, we have raised more than $550 million, which we expect to support us into the second half of 2024. We begin 2021 focused on driving our DM1, DMD and FSHD programs to the clinic as we pursue our goal of delivering potentially life-transforming therapies to people living with serious muscle diseases," said Joshua Brumm, president and chief executive officer of Dyne.
Recent Highlights
- In January 2021, Dyne announced new preclinical data from its myotonic dystrophy type 1 (DM1) program demonstrating robust RNA knockdown of toxic human nuclear DMPK, the genetic basis of 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 (hTfR1) and carries a human DMPK gene that represents a severe DM1 phenotype with more than 1,000 CTG repeats. In this model, two doses (2 x 10 mg/kg) of Dyne's candidate resulted in significant toxic human nuclear DMPK knockdown at 14 days: 60 percent in the heart; 56 percent in the diaphragm; 54 percent in the tibialis anterior and 39 percent in the gastrocnemius. In the study, Dyne's candidate was well tolerated. Dyne expects to share data from the hTfR1/DMSXL model at a scientific meeting during 2021.
- The Company further strengthened its leadership team with the appointment of Wildon Farwell, M.D., MPH, as chief medical officer, announced separately today, and Ashish Dugar, Ph.D., MBA, as senior vice president, global head of medical affairs, in February 2021.
- In January 2021, Dyne completed its follow-on public offering of 6,000,000 shares of its common stock at a public offering price of $28.00 per share. Gross proceeds of the offering before deducting the underwriting discount and commissions and offering expenses were approximately $168 million. Dyne expects its cash, cash equivalents and marketable securities as of December 31, 2020, together with the net proceeds from the January 2021 public offering, will be sufficient to fund its operating expenses and capital expenditure requirements into the second half of 2024.
Upcoming Scientific & Investor Conference Presentations
- Dyne is scheduled to participate in the 2021 MDA Virtual Clinical & Scientific Conference. Romesh Subramanian, Ph.D., chief scientific officer, will present as part of the panel "Therapeutic Considerations for Dominant Neuromuscular Diseases," and Oxana Beskrovnaya, Ph.D., senior vice president, head of research, will participate in the "Non-viral Delivery in Neuromuscular Disease" session. Both presentations will take place on March 15, 2021 and will be available to registered attendees at: https://mdavirtualconference.org/.
- Dr. Beskrovnaya also intends to present on Dyne's FORCE platform during the virtual 7th Cold Spring Harbor Laboratory meeting on Nucleic Acid Therapies on March 24, 2021, which is accessible to registered participants at: https://meetings.cshl.edu/meetings.aspx?meet=NAT&year=21.
- Mr. Brumm and Dr. Subramanian are scheduled to participate in a virtual fireside chat during Stifel's 3rd Annual CNS Day on April 1, 2021 at 10:30 am 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.
Fourth Quarter and Full Year 2020 Financial Results
Cash position: Cash, cash equivalents and marketable securities were $345.3 million as of December 31, 2020. Additionally, in January 2021, Dyne completed a public offering of common stock, with gross proceeds totaling approximately $168 million.
Research and development (R&D) expenses: R&D expenses were $22.1 million and $4.3 million for the quarters ended December 31, 2020 and 2019, respectively. R&D expenses were $45.2 million and $11.0 million for the years ended December 31, 2020 and 2019, respectively.
General and administrative (G&A) expenses: G&A expenses were $6.5 million and $1.2 million for the quarters ended December 31, 2020 and 2019, respectively. G&A expenses were $13.4 million and $2.8 million for the years ended December 31, 2020 and 2019, respectively.
Net loss: Net loss for the quarter ended December 31, 2020 was $28.6 million, or $0.64 per basic and diluted share. This compares with a net loss of $5.4 million, or $2.10 per basic and diluted share, for the quarter ended December 31, 2019. Net loss for the year ended December 31, 2020 was $59.4 million, or $4.13 per basic and diluted share. This compares with a net loss of $14.9 million, or $6.08 per basic and diluted share, for the year ended December 31, 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 Annual Report on Form 10-K for the year ended December 31, 2020 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 (in thousands, except share and per share data) Three Months Ended December 31, Year Ended December 31, 2020 2019 2020 2019 Operating expenses: Research and development $ 22,098 $ 4,259 $ 45,200 $ 11,040 General and administrative 6,502 1,211 13,447 2,786 Total operating expenses 28,600 5,470 58,647 13,826 Loss from operations (28,600 ) (5,470 ) (58,647 ) (13,826 ) Other (expense) income (49 ) 67 (790 ) (1,033 ) Net loss $ (28,649 ) $ (5,403 ) $ (59,437 ) $ (14,859 ) Net loss per share—basic and diluted $ (0.64 ) $ (2.10 ) $ (4.13 ) $ (6.08 ) Weighted-average common shares outstanding used in net loss per share—basic and diluted 45,058,494 2,567,007 14,395,955 2,442,872 Dyne Therapeutics, Inc. Condensed Consolidated Balance Sheet Data (in thousands) December 31, December 31, 2020 2019 Assets Cash, cash equivalents and marketable securities $ 345,314 $ 14,632 Other assets 8,020 1,804 Total assets $ 353,334 $ 16,436 Liabilities and Stockholders' Equity Liabilities 10,967 2,400 Stockholders' equity 342,367 14,036 Total liabilities and stockholders' equity $ 353,334 $ 16,436 - In January 2021, Dyne announced new preclinical data from its myotonic dystrophy type 1 (DM1) program demonstrating robust RNA knockdown of toxic human nuclear DMPK, the genetic basis of 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 (hTfR1) and carries a human DMPK gene that represents a severe DM1 phenotype with more than 1,000 CTG repeats. In this model, two doses (2 x 10 mg/kg) of Dyne's candidate resulted in significant toxic human nuclear DMPK knockdown at 14 days: 60 percent in the heart; 56 percent in the diaphragm; 54 percent in the tibialis anterior and 39 percent in the gastrocnemius. In the study, Dyne's candidate was well tolerated. Dyne expects to share data from the hTfR1/DMSXL model at a scientific meeting during 2021.
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Seasoned Clinical Leader Brings Deep Experience in Drug Development, Including Approval of an Oligonucleotide Therapy in Neuromuscular Disease
WALTHAM, Mass., March 04, 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 the appointment of Wildon Farwell, M.D., MPH, as chief medical officer. Dr. Farwell brings expertise in clinical development and medical affairs in neuromuscular diseases and oligonucleotide therapies.
"Wildon brings tremendous clinical experience and has played a leading role in the development of multiple oligonucleotide therapies, including one approved for a rare neuromuscular disease," said Joshua Brumm, president and chief executive officer of Dyne. "He will be instrumental in engaging with global regulatory agencies, key clinicians, investigators and patient communities. Wildon's appointment further strengthens Dyne's clinical and medical expertise as we advance our three programs toward the clinic and exemplifies our commitment to building the world's leading muscle disease company."
Dr. Farwell joins Dyne from Biogen, Inc., where he was vice president, global head of neuromuscular diseases, medical affairs. During his 10 years at Biogen, Dr. Farwell led the development and life cycle management of SPINRAZA® (nusinersen), an oligonucleotide and the first therapy approved for the treatment of spinal muscular atrophy. He also led the late-stage development of tofersen, an investigational oligonucleotide therapy for amyotrophic lateral sclerosis, oversaw clinical and biomarker development for Biogen's neuromuscular disease portfolio, and began his tenure at the company leading pharmacovigilance for multiple product candidates. Previously, Dr. Farwell was an Assistant Professor in Medicine at Harvard Medical School and a physician at Brigham and Women's Hospital and the VA Boston Healthcare System. He received his M.D. from the University of Missouri School of Medicine and an MPH in clinical effectiveness from Harvard University School of Public Health.
"I've seen firsthand the profound impact that new therapies for diseases with few or no treatment options can have on patients, and Dyne has an exciting opportunity to make a difference for people living with rare muscle diseases," said Dr. Farwell. "I'm thrilled to be a part of a company leading a differentiated approach to delivering therapeutics to muscle and to join at an important time when we are laser focused on progressing our DM1, DMD and FSHD programs to clinical trials."
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 and objectives 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; whether Dyne's cash resources will be sufficient 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 Annual Report on Form 10-K for the year ended December 31, 2020 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