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Satellos Announces Formation of Clinical Advisory Board to Support Advancing SAT-3247 in Clinical Trial Development for Duchenne Muscular Dystrophy

May 28, 2024

Satellos expects to begin first-in-human clinical trials mid-year for SAT-3247, an oral small molecule drug candidate in development as a novel regenerative medicine approach to treating DMD

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, announced today the formation of a Clinical Advisory Board comprised of distinguished clinical research leaders and experts in drug development in genetic muscle disorders, including Duchenne muscular dystrophy (DMD or Duchenne).

“The formation of this Clinical Advisory Board marks a major development step for Satellos as we continue our evolution in becoming a clinical stage drug development company,” said Frank Gleeson, Co-founder and CEO of Satellos. “We are proud and excited to bring together leading clinicians and scientists from across the world who are dedicated to, and have decades of experience in, the clinical development of novel therapeutics for degenerative muscle disorders. We believe this will help support Satellos in advancing our lead drug candidate, SAT-3247, as we work to optimize its potential to transform the treatment of Duchenne and serious muscle diseases.”

The members of the Clinical Advisory Board (CAB) are as follows:

Jordan Dubow, M.D., Chair of the CAB and Chief Medical Advisor, Satellos, has extensive experience in the biopharmaceutical industry, leading all aspects of clinical and regulatory development in Chief Medical Officer (CMO) capacities for both public and private companies, playing pivotal roles on 16 new drug applications (NDAs), including in DMD.

Ronald Cohn, M.D., Ph.D., President and CEO, Hospital for Sick Children, is an outstanding clinician scientist devoted to discovering new diagnostic and treatment options for pediatric disease and an accomplished scientist dedicated to muscular dystrophies. Research in his laboratory focuses on utilizing genome editing technologies like CRISPR for the development of therapeutic approaches for neurogenetic disorders.

Richard Finkel, M.D., Director, Center for Experimental Neurotherapeutics, St. Jude Children's Research Hospital, is an experienced translational researcher and international leader in organizing key clinical trials for neuromuscular diseases, including DMD and spinal muscular atrophy, for which he played a vital role in developing the first successful therapy.

Nicholas Johnson, M.D., MSCI, FAAN, Director of the Center for Inherited Myology Research, George Bliley Research Chair, and Professor and Vice Chair of Research in Neurology, conducts therapeutic trials in inherited nerve and muscle disorders and also dedicates significant time to laboratory research as part of a team at VCU Health working to advance the treatment of genetic muscle disorders, with a special emphasis on muscular dystrophies.

Hanns Lochmüller, M.D., Ph.D., Senior Scientist, Children’s Hospital of Eastern Ontario Research Institute, is a neurologist and clinical academic focused on clinical research and care of patients with rare genetic neuromuscular disorders. His research includes molecular therapies of neuromuscular disorders and molecular pathogenesis of muscle and neuromuscular junction disorders.

Francesco Muntoni, M.D., Director, Dubowitz Neuromuscular Centre, Great Ormond Street UCL Institute of Child Health, is focused on advancing novel therapeutics in translational and clinical research for pediatric neuromuscular disorders, especially Duchenne muscular dystrophy. His efforts in the last 20 years led to two FDA-approved therapies for DMD (eteplirsen and golodirsen).

Perry Shieh, M.D., Ph.D., FAAN, Professor of Neurology, UCLA David Geffen School of Medicine. Dr. Shieh’s principal clinical interests include Duchenne muscular dystrophy (DMD), facioscapulohumeral muscular dystrophy (FSHD), and myotonic dystrophy. He has served as an investigator in numerous clinical trials for neuromuscular conditions.

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About SAT-3247

SAT-3247 is an oral small molecule drug designed to target the root cause of muscle loss in degenerative diseases initially in 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 is 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 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

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 anticipated benefits to patients from a small molecule treatment for Duchenne; the advancement of our lead drug candidate into clinical trials; our belief that our Clinical Advisory Board will help support Satellos in advancing our lead drug candidate, the pharmacodynamic properties and mechanism-of-action of our lead drug candidate; the potential of our approach in other degenerative muscle diseases or in muscle injury or trauma; the general benefits of modulating stem cell polarity by administering small molecule drugs; 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 regenerating muscle by modulating polarity; 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 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.

Investors: Liz Williams,

Business Development: Ryan Mitchell, Ph.D.,

Media: Jessica Yingling, Ph.D.,, +1.858.344.8091

Source: Satellos Bioscience Inc.

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