Dyne Therapeutics Presents Data at World Muscle Society Congress Highlighting Promise of FORCE™ Platform to Address Underlying Causes of Neuromuscular Diseases

Rhea-AI Summary

Dyne Therapeutics (Nasdaq: DYN) is presenting data at the 29th Annual Congress of the World Muscle Society, showcasing the potential of its FORCE™ platform for neuromuscular diseases. Key highlights include:

1. DYNE-251 for DMD: Demonstrated dose-dependent exon skipping, dystrophin expression, and functional improvements. The 20 mg/kg cohort showed 3.7% mean absolute dystrophin expression (8.7% adjusted for muscle content).

2. DYNE-101 for DM1: Exhibited robust muscle delivery, dose-dependent splicing correction, and improvements in myotonia, muscle strength, and patient-reported outcomes.

3. DYNE-302 for FSHD: Preclinical data showed durable DUX4 suppression and functional benefits in a mouse model.

4. Pompe disease: Preclinical data demonstrated potential for enzyme replacement therapy delivery to muscle and CNS.

Both DYNE-251 and DYNE-101 have shown favorable safety profiles in clinical trials.

Positive

• DYNE-251 showed dose-dependent exon skipping and dystrophin expression in DMD patients
• DYNE-251 demonstrated meaningful improvements in Stride Velocity 95th Centile (SV95C) at 6 months
• DYNE-101 exhibited robust muscle delivery and dose-dependent splicing correction in DM1 patients
• DYNE-101 showed improvements in myotonia, muscle strength, and patient-reported outcomes
• Preclinical data for DYNE-302 demonstrated durable DUX4 suppression in an FSHD mouse model
• Preclinical data in Pompe disease showed potential for enzyme replacement therapy delivery to muscle and CNS

Negative

• None.

Insights

Medical Research Analyst

The data presented at the World Muscle Society Congress showcases promising results for Dyne Therapeutics' FORCE™ platform in treating various neuromuscular diseases. Key highlights include:

• DYNE-251 for DMD: Demonstrated dose-dependent exon skipping and dystrophin expression, with 3.7% mean absolute dystrophin expression (unadjusted) and 8.7% when adjusted for muscle content at 20 mg/kg. Importantly, it showed meaningful improvements in Stride Velocity 95th Centile (SV95C), meeting the clinically important difference threshold.
• DYNE-101 for DM1: Exhibited robust muscle delivery and dose-dependent splicing correction, with a 27% mean splicing correction at 5.4 mg/kg Q8W. Improvements were observed in myotonia, muscle strength and patient-reported outcomes.
• Preclinical data for DYNE-302 in FSHD and the FORCE platform in Pompe disease also showed promising results, demonstrating the platform's potential versatility.

These results suggest that Dyne's FORCE platform could potentially address multiple neuromuscular diseases by delivering targeted therapeutics to key tissues, including skeletal, cardiac, smooth muscle and the CNS. This broad applicability could significantly impact the company's future prospects and market potential.

Financial Analyst

From a financial perspective, the positive clinical data presented by Dyne Therapeutics is significant for several reasons:

• Pipeline Validation: The promising results across multiple programs (DMD, DM1, FSHD and Pompe disease) validate the potential of the FORCE™ platform, potentially de-risking the company's technology and increasing investor confidence.
• Market Opportunity: Neuromuscular diseases represent a substantial market. For instance, the global DMD market is projected to reach $4.11 billion by 2023. Positive clinical data positions Dyne to potentially capture a significant share of this market.
• Partnership Potential: Strong clinical data could attract potential partnerships or licensing deals, providing additional revenue streams and validating the technology further.
• Funding Opportunities: Positive results may improve Dyne's ability to secure additional funding or favorable terms in future capital raises, which is important for a clinical-stage biotech company with a $3.37 billion market cap.

While it's important to note that these are early-stage results and further studies are needed, the data presents a positive outlook for Dyne's future prospects and potential long-term value creation for investors.

- Presentations Will Feature Recent Clinical Data From DYNE-251 and DYNE-101 as Well as Preclinical Data in FSHD and Pompe Disease -

WALTHAM, Mass., Oct. 09, 2024 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases, today announced that previously reported clinical and preclinical data across its pipeline will be featured in poster presentations at the 29^th Annual Congress of the World Muscle Society, held virtually and in Prague, Czech Republic, October 8-12, 2024. The presentations highlight the promise of the FORCE™ platform to deliver targeted therapeutics to address neuromuscular diseases.

“The full breadth and versatility of our FORCE platform is on display at this year’s World Muscle Society Congress, with presentations covering both of our co-lead clinical programs in DM1 and DMD, along with preclinical work in FSHD and Pompe disease,” said John Cox, president and chief executive officer of Dyne. “We’ve observed targeted delivery with the FORCE platform showing broad distribution to key tissues like skeletal, cardiac, smooth muscle and the CNS – which has the potential to transform the lives of individuals living with these diseases.”

Poster Highlights

• The Phase 1/2 DELIVER trial evaluating DYNE-251 in males with Duchenne muscular dystrophy (DMD) mutations amenable to exon 51 skipping includes 6-month biomarker and functional data from patients enrolled in the 20 mg/kg (approximate PMO dose) cohort and 12-month functional data from the 10 mg/kg cohort. DYNE-251 demonstrated dose dependent exon skipping and dystrophin expression and improvement in multiple functional endpoints in both cohorts. Patients treated with 20 mg/kg of DYNE-251 Q4W had a mean absolute dystrophin expression of 3.7% of normal (unadjusted for muscle content) and when adjusting for muscle content, it reached 8.7%. Importantly, treatment with DYNE-251 resulted in meaningful improvements in Stride Velocity 95th Centile (SV95C), a digital objective outcome measure of ambulatory performance in a patient’s normal daily environment. The change from baseline in SV95C met the published minimal clinically important difference as defined by the European Medicines Agency (0.1 m/sec) at 6 months for both the 10 and 20 mg/kg cohorts (approximately 0.2 m/sec change from baseline for both cohorts).
• The Phase 1/2 ACHIEVE trial evaluating DYNE-101 in adult participants with myotonic dystrophy type 1 (DM1) includes 12-month data from the 1.8 mg/kg Q4W (approximate ASO dose) cohort, 6-month data from the 3.4 mg/kg Q4W cohort, and 3-month data from the 5.4 mg/kg Q8W cohort. DYNE-101 demonstrated robust muscle delivery and dose-dependent, consistent splicing correction while also showing improvement in myotonia, muscle strength, and timed function tests and in the Myotonic Dystrophy Type 1 Activity and Participation Scale (DM1-ACTIV^c) and the Myotonic Dystrophy Health Index (MDHI) patient reported outcomes. Patients in the 5.4 mg/kg Q8W cohort had a 27% mean splicing correction from baseline across a broad, 22-gene panel at 3 months, with all participants demonstrating splicing correction.
• Both DYNE-251 and DYNE-101 have demonstrated favorable safety profiles. ^{1 2}
• Preclinical data for DYNE-302 in facioscapulohumeral muscular dystrophy (FSHD), demonstrated robust and durable DUX4 suppression and functional benefit in an innovative hTfR1/iFLExD mouse model developed by Dyne. In hTfR1/iFLExD mice, a single intravenous dose of DYNE-302 resulted in dose-dependent and robust reduction of the DUX4 transcriptome (D4T) that lasted up to three months, with benefit on muscle structure and function.
• Preclinical data in a Pompe disease model demonstrated the potential of the FORCE platform to deliver enzyme replacement therapy to cardiac and skeletal muscle and the central nervous system (CNS), potentially expanding the modularity of the platform beyond oligonucleotides.
  

Presentation Information

Title: Initial Data from the DELIVER Trial of DYNE-251 in Males with DMD Mutations Amenable to Exon 51 Skipping
Date/Time: October 9, 2024, 2:30 p.m. CEST/8:30 a.m. ET
Presenter: Liesbeth De Waele, M.D., Ph.D., Pediatric Neurologist at University Hospital Leuven, Belgium

Title: Initial Data from the ACHIEVE Trial of DYNE-101 in Adults with Myotonic Dystrophy Type 1 (DM1)
Date/Time: October 9, 2024, 2:30 p.m. CEST/8:30 a.m. ET
Presenter: Joost Kools, M.D., Neuromuscular Department at Radboud University Medical Center, Netherlands

Title: The FORCE™ Platform Achieves Robust and Durable DUX4 Suppression and Improves Muscle Function in Facioscapulohumeral Muscular Dystrophy Mouse Model
Date/Time: October 9, 2024, 5:15 p.m. CEST/11:15 a.m. ET
Presenter: Tyler Picariello, Ph.D., Director, Neuromuscular Research, Dyne Therapeutics

Title: The FORCE™ Platform Enables Tfr1-mediated Delivery of Enzyme Replacement Therapy to Muscle and Central Nervous System, Resolving Pompe Pathology in Mice
Date/Time: October 11, 2024, 3:45 p.m. CEST/ 9:45 a.m. ET
Presenter: Tyler Picariello, Ph.D., Director, Neuromuscular Research, Dyne Therapeutics

The presentations will be available in the Scientific Publications & Presentations section of Dyne’s website following the session.

Additionally, a symposium titled “Potential of the FORCE™ Platform to Address Unmet Needs in Rare Neuromuscular Diseases” will be held on October 10 at 8:15 a.m. CEST/ 2:15 a.m. ET.

About the FORCE™ Platform

The proprietary FORCE™ platform drives Dyne’s efforts to develop targeted, modern oligonucleotide innovative 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 receptor 1 (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 a clinical-stage muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases. With its proprietary FORCE™ platform, Dyne is developing modern oligonucleotide therapeutics that are designed to overcome limitations in delivery to muscle tissue. Dyne has a broad pipeline for serious muscle diseases, including clinical programs for myotonic dystrophy type 1 (DM1) and Duchenne muscular dystrophy (DMD) and preclinical programs for facioscapulohumeral muscular dystrophy (FSHD). For more information, please visit https://www.dyne-tx.com/, and follow us on X, LinkedIn and Facebook.

Forward-Looking Statements

This press release contains forward-looking statements that involve substantial risks and uncertainties. All statements, other than statements of historical facts, contained in this press release, including statements regarding Dyne’s strategy, future operations, prospects and plans, objectives of management, the potential of the FORCE platform, the potential of DYNE-101, DYNE-251 and DYNE-302, expectations regarding the timing and outcome of interactions with global regulatory authorities, and plans to provide future updates on pipeline programs, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “might,” “objective,” “ongoing,” “plan,” “predict,” “project,” “potential,” “should,” or “would,” or the negative of these terms, or other comparable terminology are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Dyne may not actually achieve the plans, intentions or expectations disclosed in these forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in these forward-looking statements as a result of various important factors, including: uncertainties inherent in the identification and development of product candidates, including the initiation and completion of preclinical studies and clinical trials; uncertainties as to the availability and timing of results from preclinical studies and clinical trials; the timing of and Dyne’s ability to enroll patients in clinical trials; whether results from preclinical studies and initial data from early clinical trials will be predictive of the final results of the clinical trials or future trials; uncertainties as to the FDA’s and other regulatory authorities’ interpretation of the data from Dyne's clinical trials and acceptance of Dyne's clinical programs and the regulatory approval process; whether Dyne’s cash resources will be sufficient to fund its foreseeable and unforeseeable operating expenses and capital expenditure requirements; as well as the risks and uncertainties identified in Dyne’s filings with the Securities and Exchange Commission (SEC), including the Company’s most recent Form 10-Q and in subsequent filings Dyne may make with the SEC. In addition, the forward-looking statements included in this press release represent Dyne’s views as of the date of this press release. Dyne anticipates that subsequent events and developments will cause its views to change. However, while Dyne may elect to update these forward-looking statements at some point in the future, it specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing Dyne’s views as of any date subsequent to the date of this press release.

1. Safety data as of August 21, 2024
2. Safety data as of August 20, 2024

Contacts:

Investors
Amy Reilly
areilly@dyne-tx.com
857-341-1203

Media
Stacy Nartker
snartker@dyne-tx.com
781-317-1938

FAQ

What were the key findings for DYNE-251 in the DMD clinical trial?

DYNE-251 demonstrated dose-dependent exon skipping and dystrophin expression in DMD patients. The 20 mg/kg cohort showed a mean absolute dystrophin expression of 3.7% of normal (8.7% when adjusted for muscle content). Additionally, there were meaningful improvements in Stride Velocity 95th Centile (SV95C) at 6 months for both 10 and 20 mg/kg cohorts.

How did DYNE-101 perform in the DM1 clinical trial?

DYNE-101 showed robust muscle delivery and dose-dependent splicing correction in DM1 patients. The 5.4 mg/kg Q8W cohort had a 27% mean splicing correction from baseline across a 22-gene panel at 3 months. Improvements were observed in myotonia, muscle strength, timed function tests, and patient-reported outcomes.

What were the preclinical results for DYNE-302 in FSHD?

Preclinical data for DYNE-302 in FSHD demonstrated robust and durable DUX4 suppression in a mouse model. A single intravenous dose resulted in dose-dependent reduction of the DUX4 transcriptome lasting up to three months, with benefits on muscle structure and function.

What did the preclinical data show for Dyne's approach to Pompe disease?

Preclinical data in a Pompe disease model demonstrated the potential of the FORCE platform to deliver enzyme replacement therapy to cardiac and skeletal muscle and the central nervous system, potentially expanding the platform's modularity beyond oligonucleotides.