Dyne Therapeutics Presents New Preclinical Data Demonstrating the Potential of the FORCE™ Platform to Deliver Enzyme Replacement Therapy to Muscle and CNS in Pompe Disease

Rhea-AI Summary

Dyne Therapeutics has announced new preclinical data showcasing the potential of its FORCE™ platform to deliver enzyme replacement therapy (ERT) for Pompe disease, targeting both muscle and the central nervous system (CNS). The data were presented at the New Directions in Biology and Disease of Skeletal Muscle Conference. The study demonstrated that FORCE-GAA, engineered using the FORCE platform, effectively cleared glycogen in muscle and CNS, normalized lysosomal size, reduced biomarkers of axonal injury, and showed superior efficacy compared to GAA alone. Importantly, FORCE-GAA has the potential for less frequent dosing.

Positive

• FORCE-GAA cleared glycogen in muscle and CNS.
• Normalized lysosomal size in hTfR1/6Neo mice.
• Reduced serum neurofilament light chain, indicating CNS benefit.
• Displayed superior efficacy and dose potency compared to GAA alone.
• Potential for monthly dosing, less frequent than current therapies.

Negative

• None.

Medical Research Analyst

The new preclinical data presented by Dyne Therapeutics showcases the potential of their FORCE™ platform in treating Pompe disease, which is a progressive neuromuscular disorder. The traditional enzyme replacement therapies (ERTs) for Pompe disease have limitations, particularly in addressing skeletal muscle efficacy and CNS manifestations such as cognitive deficits. FORCE-GAA has demonstrated the ability to deliver therapeutic enzymes effectively to both muscle tissues and the central nervous system in mouse models, which is an encouraging development. The possibility of monthly dosing is another significant advantage over current ERTs, which require more frequent administration.

This data indicates a promising advancement in the delivery mechanisms for enzyme replacement, potentially leading to improved patient outcomes. Investors should be aware of the preclinical nature of this data, meaning it still requires further validation in human trials. Should these findings translate successfully to clinical settings, it could significantly impact the treatment landscape for Pompe disease and position Dyne Therapeutics as a leader in innovative muscle disease therapies.

Rating: 1

Financial Analyst

The implications of Dyne Therapeutics' preclinical data on its financial outlook are noteworthy. Successful development of the FORCE™ platform for Pompe disease can potentially open up a lucrative market, given the limitations of existing ERTs. The news may lead to increased investor confidence and stock price appreciation in the short term. Additionally, the potential for less frequent dosing means better patient compliance and a more attractive product profile.

However, investors should temper their enthusiasm with a note of caution. The data is preclinical and the transition to human trials carries risks. The success of such trials is critical for long-term financial benefits. Moreover, the company’s ability to scale production, achieve regulatory approvals and market the therapy effectively will determine the ultimate financial impact.

Rating: 1

Oncology Doctor

From a clinical perspective, the ability of the FORCE™ platform to deliver enzyme replacement therapy effectively to both muscle and CNS tissues represents a paradigm shift in treating Pompe disease. Historically, treatments have struggled with CNS penetration, leaving some symptoms inadequately addressed. The reduction in serum neurofilament light chain and normalization of lysosomal size in mouse models are promising indicators of comprehensive efficacy.

This development not only impacts patients with Pompe disease but could also set a precedent for treating other neuromuscular disorders with CNS involvement. The modular nature of the FORCE platform may allow for its adaptation to other conditions, broadening its clinical applicability and impact. Nonetheless, thorough clinical trials will be necessary to confirm these preclinical results and ensure safety and efficacy in humans.

Rating: 1

WALTHAM, Mass., June 24, 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 new preclinical data in a Pompe disease model demonstrating the potential of the FORCE™ platform to deliver enzyme replacement therapy to cardiac and skeletal muscle and the central nervous system (CNS). The data were presented at the New Directions in Biology and Disease of Skeletal Muscle Conference, being held June 23-26 in Fort Lauderdale, FL.

“These preclinical data show for the first time the ability of FORCE to deliver enzymes to skeletal and cardiac muscle as well as the CNS, expanding the modularity of our platform beyond oligonucleotides. The data support the potential of FORCE to enable effective enzyme replacement therapy with the opportunity to substantially improve upon available treatments for Pompe disease,” Oxana Beskrovnaya, Ph.D., chief scientific officer of Dyne. “We look forward to continuing to explore this application of our platform as part of our mission to deliver life-transforming therapies for people with serious muscle diseases.”

Pompe disease is a rare, severe neuromuscular disorder caused by deficiency of the lysosomal enzyme, acid alpha glucosidase (GAA). Lack of GAA leads to glycogen accumulation and increase in lysosomal size in muscle and subsequent weakness, cardiomyopathy and respiratory failure. Enzyme replacement therapy with GAA is the standard of care and increases survival but has inadequate efficacy in skeletal muscle. Pompe is also characterized by CNS manifestations, including behavioral and cognitive deficits due to glycogen accumulation in CNS cells, which are not addressed by the standard of care therapy.

Dyne engineered FORCE-GAA by leveraging the FORCE platform and evaluated efficacy in vivo using hTfR1/6^Neo mice, that were developed by crossing the well-established 6^Neo mouse model of Pompe with mice expressing human transferrin receptor 1. Intravenous administration of FORCE-GAA cleared glycogen in muscle and the CNS and normalized lysosomal size in hTfR1/6^Neo mice. FORCE-GAA reduced serum neurofilament light chain, a biomarker of axonal injury, providing evidence of benefit in the CNS. FORCE-GAA also displayed superior efficacy and dose potency compared to GAA alone. Additional data with FORCE-GAA showed the potential for monthly dosing which is less frequent than approved enzyme replacement therapies.

The presentation entitled, “FORCE™ Platform for the Development of Targeted Therapeutics for Rare Muscle Diseases” is available in the Scientific Publications & Presentations section of Dyne’s website at https://www.dyne-tx.com/our-forcetm-publications/.

About Pompe Disease

Pompe disease is a severe neuromuscular disorder caused by a deficiency of the lysosomal enzyme, acid alpha glucosidase (GAA). Lack of GAA causes glycogen accumulation in tissue leading to muscle weakness, cardiomyopathy, respiratory failure, and central nervous system (CNS) manifestations. Pompe disease belongs to a group of diseases known as the lysosomal storage disorders (LSDs). It can present as infantile-onset, the most severe form of the disease with early onset of symptoms in infancy that rapidly progress, or late-onset, which progressively damages muscles over time. An estimated 5,000 to 10,000 individuals worldwide are affected by Pompe. 

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 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 a preclinical program 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 anticipated timelines for reporting additional data for FORCE-GAA, expectations regarding the initiation of additional preclinical studies or clinical trials of FORCE-GAA, 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; 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. 

Contacts:

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

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

FAQ

What is the potential of Dyne Therapeutics' FORCE™ platform in Pompe disease?

Dyne Therapeutics' FORCE™ platform shows potential for delivering enzyme replacement therapy to both muscle and CNS in Pompe disease, demonstrating superior efficacy and less frequent dosing.

When and where did Dyne Therapeutics present their new preclinical data on Pompe disease?

Dyne Therapeutics presented the new preclinical data at the New Directions in Biology and Disease of Skeletal Muscle Conference, held from June 23-26, 2024, in Fort Lauderdale, FL.

What are the advantages of FORCE-GAA over current Pompe disease treatments?

FORCE-GAA shows superior efficacy in clearing glycogen, normalizing lysosomal size, reducing axonal injury biomarkers, and offers potential for less frequent dosing compared to current enzyme replacement therapies.

How does FORCE-GAA benefit the central nervous system (CNS) in Pompe disease?

FORCE-GAA reduces serum neurofilament light chain, a biomarker of axonal injury, indicating a therapeutic benefit to the CNS in Pompe disease.

What makes Dyne Therapeutics' FORCE™ platform unique in treating Pompe disease?

Dyne Therapeutics' FORCE™ platform uniquely enables effective enzyme replacement therapy in both skeletal and cardiac muscles as well as the CNS, addressing areas where current treatments fall short.