Satellos Demonstrates Treatment of DMD Canines with SAT-3247 Improved Measures of Strength to Near Normal Levels

Rhea-AI Summary

Satellos Bioscience Inc. (TSX: MSCL, OTCQB: MSCLF) announced promising results from a pilot study of SAT-3247, their oral small molecule treatment for Duchenne muscular dystrophy (DMD), in a canine model. After four months of daily treatment:

- Muscle function returned to near-normal levels compared to healthy animals
- Increased Regenerative Index (RI) in diaphragm, calf, and quadriceps muscles
- No adverse events or significant changes in hematology or clinical chemistry
- Trends towards lower creatine kinase levels, potentially indicating disease modification

The data will be presented at the 29th Annual Congress of the World Muscle Society in October 2024. Satellos believes these results are groundbreaking, especially given the severity of the canine DMD model compared to mouse models.

Positive

• SAT-3247 treatment improved muscle function to near-normal levels in canine DMD model
• Increased Regenerative Index observed in multiple muscle groups
• No adverse events or significant changes in hematology or clinical chemistry after 4 months of treatment
• Potential disease-modifying effect indicated by trends in lower creatine kinase levels

Negative

• None.

- Analysis showed improvement in muscle morphology and increased levels of regeneration in muscle groups, including the diaphragm, quadriceps, and calf

- No adverse events and no significant changes in hematology or clinical chemistry were observed after four months of daily, oral treatment with SAT-3247, Satellos’ proprietary small molecule

- Data to be presented at the 2024 World Muscle Society Annual Congress

TORONTO--(BUSINESS WIRE)-- Satellos Bioscience Inc. (“Satellos” or the “Company”) (TSX: MSCL, OTCQB: MSCLF), a public biotech company developing new small molecule therapeutic approaches to improve the treatment of muscle diseases and disorders, today announced a presentation of data at the 29th Annual Congress of the World Muscle Society taking place October 8-12, 2024, in Prague. The presentation will provide an overview of key data collected during the open-label pilot study of SAT-3247 in a canine model of Duchenne muscular dystrophy (“Duchenne” or “DMD”).

An initial summary of the data is presented below. After four months of treatment with SAT-3247:

• Treated animals showed a return to muscle function near healthy, non-diseased, age-matched animal levels when evaluated against historical comparator data.
• The animals showed increases in Regenerative Index (RI) in diaphragm, gastrocnemius medialis (calf), and vastus lateralis (quadriceps). This is in addition to previously reported improvements in RI demonstrated in the bicep femoris.
• There were no adverse events and no significant changes in hematology or clinical chemistry observed.
• Trends to lower creatine kinase levels were noted, a finding that could be consistent with a Duchenne disease-modifying treatment.

“We believe the improvements observed in regeneration and muscle force to close to healthy levels within four months of treatment, when compared to published natural history data and other therapeutic interventions in the canine model of DMD, are groundbreaking,” said Frank Gleeson, Co-founder and CEO of Satellos Bioscience. “The canine model is considered more severe than mouse models, and potentially represents a greater test of a treatment’s utility to translate to humans. We are ecstatic that treatment with our oral small molecule, SAT-3247, showed such dramatic improvements. These highly promising results reinforce our belief in the potential of SAT-3247 to offer a meaningful disease-modifying medicine to help patients living with Duchenne.”

The canine model of muscular dystrophy represents a more severe clinical phenotype and reflects the disease progression observed in people with DMD. In this pilot study, each animal (n=2) was treated for four months with a daily oral dose of SAT-3247. This multiparameter pilot study measured clinical chemistry, hematology, muscle function, and included a large-scale muscle histology workup. From the muscle histology, a calculation was made of the Regenerative Index (RI), a measure of the number of newly regenerated muscle fibers over the number of damaged and dying muscle fibers.

The data from this study will be presented in a poster entitled, “SAT-3247: An Oral Small Molecule Inhibitor Targeting AAK1, a Critical Effector of Skeletal Muscle Regeneration.” The poster is available on the Events & Presentations page of the Satellos website located at ir.satellos.com.

About SAT-3247

SAT-3247 is designed as a once-daily, oral small molecule drug that targets the root cause of muscle loss in degenerative diseases, initially targeting Duchenne. SAT-3247 presents a novel mechanism of action to restore impaired muscle regeneration caused by the absence of functional dystrophin.

About Duchenne Muscular Dystrophy

Duchenne muscular dystrophy is an inherited disease caused by mutations in the dystrophin gene that no longer allow the dystrophin protein to function properly. Consequently, as discovered by Satellos, muscle repair and regeneration are impaired. Satellos designed SAT-3247 to restore the process of muscle repair and regeneration by regulating a dystrophin-independent pathway with the goal of increasing muscle function. SAT-3247 is intended to work as a standalone therapeutic without regard to a patient’s genetic mutation or ambulatory status. Our approach also has the potential to complement approaches designed to restore dystrophin production.

About Satellos Bioscience Inc.

Satellos is a publicly traded biotechnology company dedicated to developing life-improving medicines to treat degenerative muscle diseases. Satellos has incorporated breakthrough research in muscle stem cell polarity into a proprietary discovery platform, called MyoReGenX™, to identify degenerative muscle diseases where deficits in this process affect muscle regeneration and are amenable to therapeutic intervention. With this platform, Satellos is building a pipeline of novel therapeutics to correct muscle stem cell polarity and promote the body’s innate muscle repair and regeneration process. The Company’s lead program is an oral, small molecule drug candidate in development as a potential disease-modifying treatment for Duchenne muscular dystrophy. Satellos is headquartered in Toronto, Ontario. For more information, visit www.satellos.com.

Notice on Forward-Looking Statements

This press release includes forward-looking information or forward-looking statements within the meaning of applicable securities laws regarding Satellos and its business, which may include, but are not limited to, statements regarding the value of our DMD program; the advancement of our lead drug candidate into clinical trials; the expected structure and progress of our clinical trials and any potential insights or results that may be obtained from them; the implications of results of our clinical and pre-clinical trials (including the relevance of pre-clinical results to utility of our lead drug candidate in humans); the general benefits of modulating stem cell polarity by administering small molecule drugs, including our lead drug candidate; its/their prospective impact on Duchenne patients, patients with other degenerative muscle disease or muscle injury or trauma, and on muscle regeneration generally; the utility of our lead drug candidate and of regenerating muscle by modulating polarity generally; adoption of Satellos’ approach by the medical community; and Satellos’ technologies and drug development plans. All statements that are, or information which is, not historical facts, including without limitation, statements regarding future estimates, plans, programs, forecasts, projections, objectives, assumptions, expectations or beliefs of future performance, occurrences or developments, are “forward-looking information or statements.” Often but not always, forward-looking information or statements can be identified by the use of words such as “shall”, “intends”, “anticipate”, “believe”, “plan”, “expect”, “intend”, “estimate”, “anticipate”, “potential”, “prospective” , “assert” or any variations (including negative or plural variations) of such words and phrases, or state that certain actions, events or results “may”, “might”, “can”, “could”, “would” or “will” be taken, occur, lead to, result in, or, be achieved. Such statements are based on the current expectations and views of future events of the management of the Company. They are based on assumptions and subject to risks and uncertainties. Although management believes that the assumptions underlying these statements are reasonable, they may prove to be incorrect. The forward-looking events and circumstances discussed in this release, may not occur and could differ materially as a result of known and unknown risk factors and uncertainties affecting the Company, including, without limitation, risks relating to the pharmaceutical and bioscience industry (including the risks associated with preclinical and clinical trials and regulatory approvals), and the research and development of therapeutics, the results of preclinical and clinical trials, general market conditions and equity markets, economic factors and management’s ability to manage and to operate the business of the Company generally, including inflation and the costs of operating a biopharma business, and those risks listed in the “Risk Factors” section of Satellos’ Annual Information Form dated March 26, 2024 (which is located on Satellos’ profile at www.sedarplus.ca). Although Satellos has attempted to identify important factors that could cause actual actions, events or results to differ materially from those described in forward-looking statements, there may be other factors that cause actions, events or results to differ from those anticipated, estimated or intended. Accordingly, readers should not place undue reliance on any forward-looking statements or information. No forward- looking statement can be guaranteed. Except as required by applicable securities laws, forward-looking statements speak only as of the date on which they are made and Satellos does not undertake any obligation to publicly update or revise any forward-looking statement, whether resulting from new information, future events, or otherwise.

View source version on businesswire.com: https://www.businesswire.com/news/home/20241001367017/en/

Investors: Liz Williams, ir@satellos.com

Media: Jessica Yingling, Ph.D., jessica@litldog.com, +1.858.344.8091

Source: Satellos Bioscience Inc.

FAQ

What are the key results of Satellos' SAT-3247 treatment for DMD in canines?

After four months of treatment, SAT-3247 improved muscle function to near-normal levels, increased the Regenerative Index in multiple muscle groups, showed no adverse events, and demonstrated potential disease-modifying effects through lower creatine kinase levels.

When and where will Satellos (MSCLF) present the SAT-3247 study results?

Satellos will present the SAT-3247 study results at the 29th Annual Congress of the World Muscle Society, taking place from October 8-12, 2024, in Prague.

How does the canine model of DMD compare to other animal models?

The canine model of DMD is considered more severe than mouse models and potentially represents a greater test of a treatment's utility to translate to humans, as it better reflects the disease progression observed in people with DMD.

What is the Regenerative Index (RI) measured in Satellos' SAT-3247 study?

The Regenerative Index (RI) is a measure of the number of newly regenerated muscle fibers over the number of damaged and dying muscle fibers, used to assess the effectiveness of SAT-3247 in treating DMD.