- Phase 1a data shows SAT-3247 was safe and well tolerated in 72 healthy volunteers after both single and repeated administration
- Phase 1a data shows favorable pharmacokinetic (PK) profile of SAT-3247, reinforcing its potential as a first-of-its-kind oral therapy for Duchenne Muscular Dystrophy (DMD)
- Enrollment and treatment are ongoing in the Phase 1b trial in adults with DMD
TORONTO, March 19, 2025--(BUSINESS WIRE)--Satellos Bioscience Inc. (TSX: MSCL, OTCQB: MSCLF) ("Satellos" or the "Company"), a biotech company developing new small molecule therapeutic approaches to improve the treatment of muscle diseases and disorders, today announced initial Phase 1 data in an oral presentation at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference in Dallas, Texas.
"We are very pleased by these early clinical data reinforcing the translation of SAT-3247’s preclinical pharmacokinetic and safety profile," said Frank Gleeson, Satellos Co-Founder and CEO. "We believe these clinical results support the potential for SAT-3247 to have a desirable therapeutic profile as a first-of-its-kind oral therapy for people living with DMD."
SAT-3247 is designed to address progressive muscle loss in DMD patients by aiming to restore regeneration in response to damage. A Phase 1 trial with SAT-3247 is being conducted in healthy volunteers and adults with DMD (Phase 1a and Phase 1b, respectively).
In the Phase 1a, designed to assess the safety and tolerability of SAT-3247, 72 healthy volunteers were randomized across five single ascending dose (SAD) cohorts (including one food effect cohort) with single oral doses of up to 400 mg, and four MAD cohorts with daily oral doses up to 240 mg/day for 7 days. As of a Feb. 20, 2025, data cut-off:
Phase 1a data showed that SAT-3247 was safe and well tolerated across all healthy volunteer cohorts. At predicted human efficacious dose levels, SAT-3247 did not display adverse clinical findings on any parameter measured, including clinical labs, vital signs, ECG, and physical exam. No moderate or greater drug-related adverse events were reported at any dose studied.
Phase 1a PK data demonstrated consistency with results from the Company’s preclinical studies. These PK results confirm post-dose plasma concentrations of SAT-3247 are sustained at levels and time courses, which findings suggest are most likely to yield a therapeutic effect on muscle regeneration and strength.
Satellos expects to report full Phase 1a and Phase 1b data in Q2 2025.
Oral Presentation: Title: First-in-human Phase 1 study of orally administered SAT-3247 in healthy volunteers and adult participants with Duchenne Muscular Dystrophy (DMD) Date: Wednesday, March 19, 2025 Time: 08:00 AM CT Location/Room: Coronado ABCD at the Hilton Anatole, Dallas Presenter: Phil Lambert, Ph.D., Chief Scientific Officer, Satellos
About Phase 1 DMD Trial The Phase 1 clinical trial is comprised of two components. In the first component, Phase 1a, 72 healthy volunteers have been enrolled in a blinded, randomized, placebo-controlled, staggered, parallel design study to assess the safety and pharmacokinetic properties of SAT-3247. Participants were randomized across five SAD cohorts, four MAD cohorts, and one food effect dose cohort. The second component, the Phase 1b portion of the trial, is currently ongoing. Up to 10 adult volunteers with genetically confirmed DMD will be enrolled in a 28-day, open-label, single dose cohort to assess safety and pharmacokinetic properties in patients and explore potential pharmacodynamic markers.
About SAT-3247 SAT-3247 is a proprietary, oral small molecule drug being developed by Satellos as a novel treatment to regenerate skeletal muscle that is lost in Duchenne muscular dystrophy (DMD or Duchenne) and other degenerative or injury conditions. Satellos is advancing SAT-3247 as a potential treatment for DMD, independent of dystrophin and regardless of exon mutation status.
About Satellos Bioscience Inc. Satellos is a clinical-stage drug development company dedicated to developing life-improving medicines to treat degenerative muscle diseases. Satellos has invented SAT-3247 as a first-of-its-kind, orally administered small molecule drug designed to restore skeletal muscle regeneration in degenerative or injury conditions by correcting muscle stem cell polarity. Satellos has generated a body of preclinical evidence with SAT-3247 to support its discovery that correcting muscle stem cell polarity has the potential to restore skeletal muscle regeneration to repair and strengthen muscle that has degenerated or been damaged. SAT-3247 is currently in clinical development as a potential disease-modifying treatment initially for DMD. Additionally, Satellos is leveraging its breakthrough research and proprietary discovery platform MyoReGenX™, to identify additional degenerative muscle diseases or injury conditions where deficits in muscle regeneration occur that are amenable to therapeutic intervention for future clinical development. 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, expected timing for Phase 1a and Phase 1b data; statements regarding the potential for SAT-3247 to represent a disease modifying approach to the therapeutic treatment of people living with Duchenne; anticipated benefits to patients from a small molecule treatment for Duchenne; the advancement SAT-3247 through clinical trials; the pharmacodynamic properties and mechanism-of-action of SAT-3247; the potential of our approach in other degenerative muscle diseases; 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; 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.
