Satellos Presents Initial Data from the Phase 1 Trial of SAT-3247 at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference
Satellos Bioscience (OTCQB: MSCLF) has presented initial Phase 1 data for SAT-3247, their oral therapy candidate for Duchenne Muscular Dystrophy (DMD), at the 2025 MDA Clinical & Scientific Conference.
The Phase 1a trial involved 72 healthy volunteers across five single ascending dose cohorts (up to 400 mg) and four multiple ascending dose cohorts (up to 240 mg/day for 7 days). Key findings show that SAT-3247 was safe and well-tolerated, with no moderate or greater drug-related adverse events reported. The pharmacokinetic profile demonstrated consistency with preclinical studies, maintaining plasma concentrations at levels expected to benefit muscle regeneration and strength.
The drug is designed to address progressive muscle loss in DMD patients by targeting muscle regeneration. A Phase 1b trial in adults with DMD is currently ongoing, with full Phase 1a and 1b data expected in Q2 2025.
Satellos Bioscience (OTCQB: MSCLF) ha presentato i dati iniziali della Fase 1 per SAT-3247, il loro candidato per una terapia orale per la Distrofia Muscolare di Duchenne (DMD), durante la Conferenza Clinica e Scientifica MDA 2025.
Lo studio della Fase 1a ha coinvolto 72 volontari sani suddivisi in cinque coorti a dose singola crescente (fino a 400 mg) e quattro coorti a dose multipla crescente (fino a 240 mg/giorno per 7 giorni). I risultati chiave mostrano che SAT-3247 è stato sicuro e ben tollerato, senza eventi avversi moderati o gravi correlati al farmaco segnalati. Il profilo farmacocinetico ha dimostrato coerenza con gli studi preclinici, mantenendo le concentrazioni plasmatiche a livelli attesi per favorire la rigenerazione e la forza muscolare.
Il farmaco è progettato per affrontare la perdita muscolare progressiva nei pazienti con DMD, mirando alla rigenerazione muscolare. Un trial di Fase 1b negli adulti con DMD è attualmente in corso, con i dati completi delle Fasi 1a e 1b attesi nel secondo trimestre del 2025.
Satellos Bioscience (OTCQB: MSCLF) ha presentado datos iniciales de la Fase 1 para SAT-3247, su candidato a terapia oral para la Distrofia Muscular de Duchenne (DMD), en la Conferencia Clínica y Científica de la MDA 2025.
El ensayo de la Fase 1a involucró a 72 voluntarios sanos en cinco cohortes de dosis única ascendente (hasta 400 mg) y cuatro cohortes de dosis múltiple ascendente (hasta 240 mg/día durante 7 días). Los hallazgos clave muestran que SAT-3247 fue seguro y bien tolerado, sin eventos adversos moderados o graves relacionados con el fármaco reportados. El perfil farmacocinético demostró consistencia con los estudios preclínicos, manteniendo las concentraciones plasmáticas en niveles esperados para beneficiar la regeneración y la fuerza muscular.
El fármaco está diseñado para abordar la pérdida muscular progresiva en pacientes con DMD, enfocándose en la regeneración muscular. Un ensayo de Fase 1b en adultos con DMD está actualmente en curso, con datos completos de las Fases 1a y 1b esperados para el segundo trimestre de 2025.
Satellos Bioscience (OTCQB: MSCLF)는 2025년 MDA 임상 및 과학 회의에서 SAT-3247에 대한 초기 1상 데이터를 발표했습니다. 이는 뒤셴 근육 디스트로피(DMD)를 위한 경구 치료 후보입니다.
1상 a 시험에는 72명의 건강한 자원봉사자가 포함되었으며, 5개의 단일 용량 상승 코호트(최대 400mg)와 4개의 다중 용량 상승 코호트(최대 240mg/일, 7일 동안)로 나누어 진행되었습니다. 주요 결과는 SAT-3247이 안전하고 잘 견딜 수 있었으며, 중간 또는 그 이상의 약물 관련 부작용이 보고되지 않았음을 보여줍니다. 약리학적 프로필은 전임상 연구와 일관성을 보였으며, 근육 재생과 힘에 도움이 될 것으로 예상되는 수준에서 혈장 농도를 유지했습니다.
이 약물은 DMD 환자에서 진행성 근육 손실을 해결하기 위해 근육 재생을 목표로 설계되었습니다. DMD 성인을 대상으로 한 1상 b 시험이 현재 진행 중이며, 1상 a 및 1b의 전체 데이터는 2025년 2분기에 예상됩니다.
Satellos Bioscience (OTCQB: MSCLF) a présenté des données initiales de la Phase 1 pour SAT-3247, leur candidat thérapie orale pour la dystrophie musculaire de Duchenne (DMD), lors de la Conférence Clinique et Scientifique de la MDA 2025.
L'essai de la Phase 1a a impliqué 72 volontaires en bonne santé répartis en cinq cohortes à dose unique croissante (jusqu'à 400 mg) et quatre cohortes à dose multiple croissante (jusqu'à 240 mg/jour pendant 7 jours). Les résultats clés montrent que SAT-3247 était sûr et bien toléré, sans événements indésirables modérés ou graves liés au médicament signalés. Le profil pharmacocinétique a montré une cohérence avec les études précliniques, maintenant les concentrations plasmatiques à des niveaux attendus pour bénéficier à la régénération et à la force musculaire.
Le médicament est conçu pour traiter la perte musculaire progressive chez les patients atteints de DMD en ciblant la régénération musculaire. Un essai de Phase 1b chez des adultes atteints de DMD est actuellement en cours, avec des données complètes des phases 1a et 1b attendues au deuxième trimestre de 2025.
Satellos Bioscience (OTCQB: MSCLF) hat erste Daten der Phase 1 für SAT-3247, ihren oralen Therapie-Kandidaten für Duchenne-Muskeldystrophie (DMD), auf der MDA Klinischen und Wissenschaftlichen Konferenz 2025 präsentiert.
Die Phase 1a-Studie umfasste 72 gesunde Freiwillige, die in fünf Gruppen mit steigenden Einzel-Dosen (bis zu 400 mg) und vier Gruppen mit steigenden Mehrfach-Dosen (bis zu 240 mg/Tag über 7 Tage) eingeteilt wurden. Wichtige Ergebnisse zeigen, dass SAT-3247 sicher und gut verträglich war, ohne gemeldete moderate oder schwerwiegende medikamentenbezogene unerwünschte Ereignisse. Das pharmakokinetische Profil zeigte eine Konsistenz mit den präklinischen Studien und hielt die Plasmakonzentrationen auf einem Niveau, das voraussichtlich die Muskelregeneration und -kraft begünstigt.
Das Medikament ist darauf ausgelegt, den progressiven Muskelverlust bei DMD-Patienten anzugehen, indem es die Muskelregeneration fördert. Eine Phase 1b-Studie bei Erwachsenen mit DMD ist derzeit im Gange, und vollständige Daten der Phasen 1a und 1b werden im 2. Quartal 2025 erwartet.
- Phase 1a trial demonstrated safety and tolerability with no moderate or greater drug-related adverse events
- Pharmacokinetic data aligned with preclinical studies, showing sustained therapeutic levels
- Successfully completed testing across multiple dosing cohorts with 72 volunteers
- Phase 1b trial in DMD patients still ongoing with no efficacy data available yet
- 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
“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,
Presenter: Phil Lambert, Ph.D., Chief Scientific Officer, Satellos
A copy of the presentation will be available after the session on the Events & Presentations page located at: https://ir.satellos.com/events-and-presentations/default.aspx.
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.
View source version on businesswire.com: https://www.businesswire.com/news/home/20250319756033/en/
Investors: Liz Williams, CFO, ir@satellos.com
Media: Jessica Yingling, Ph.D., jessica@litldog.com
Clinical Trial Info: medicalinfo@satellos.com
Source: Satellos Bioscience Inc.
FAQ
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