Satellos Announces First Participant with Duchenne Muscular Dystrophy Dosed in the Phase 1b Clinical Trial of SAT-3247
Satellos Bioscience (OTCQB: MSCLF) has reached a significant milestone in its Phase 1b clinical trial of SAT-3247, with the first Duchenne muscular dystrophy (DMD) participant receiving treatment. The company has successfully completed enrollment of all five single ascending dose (SAD) cohorts and the first two multiple ascending dose (MAD) cohorts in healthy volunteers, with no reported adverse effects.
The Phase 1b trial aims to enroll up to 10 adult participants with genetically confirmed DMD in a 28-day, open-label, single dose study to assess safety and pharmacokinetics. Initial results show SAT-3247 was well-tolerated, with no drug-related adverse events, abnormal clinical findings, or safety concerns. The pharmacokinetic profile successfully translated from pre-clinical models to human subjects. Clinical data is expected to be presented at a major medical conference in Q1 2025.
Satellos Bioscience (OTCQB: MSCLF) ha raggiunto un traguardo significativo nel suo trial clinico di Fase 1b per SAT-3247, con il primo partecipante affetto da distrofia muscolare di Duchenne (DMD) che ha ricevuto il trattamento. L'azienda ha completato con successo l'arruolamento di tutti e cinque i gruppi di dosi ascendenti singole (SAD) e dei primi due gruppi di dosi ascendenti multiple (MAD) in volontari sani, senza effetti avversi riportati.
Il trial di Fase 1b mira a arruolare fino a 10 partecipanti adulti con DMD confermata geneticamente in uno studio a dose singola, aperto e della durata di 28 giorni, per valutare la sicurezza e la farmacocinetica. I risultati iniziali mostrano che SAT-3247 è stato ben tollerato, senza eventi avversi correlati al farmaco, risultati clinici anormali o preoccupazioni per la sicurezza. Il profilo farmacocinetico si è tradotto con successo dai modelli preclinici agli esseri umani. I dati clinici dovrebbero essere presentati in una importante conferenza medica nel primo trimestre del 2025.
Satellos Bioscience (OTCQB: MSCLF) ha alcanzado un hito significativo en su ensayo clínico de Fase 1b de SAT-3247, con el primer participante con distrofia muscular de Duchenne (DMD) recibiendo tratamiento. La compañía ha completado con éxito la inscripción de los cinco grupos de dosis ascendentes únicas (SAD) y los primeros dos grupos de dosis ascendentes múltiples (MAD) en voluntarios sanos, sin efectos adversos reportados.
El ensayo de Fase 1b tiene como objetivo inscribir hasta 10 participantes adultos con DMD confirmada genéticamente en un estudio de dosis única, abierto, y de 28 días para evaluar la seguridad y la farmacocinética. Los resultados iniciales muestran que SAT-3247 fue bien tolerado, sin eventos adversos relacionados con el fármaco, hallazgos clínicos anormales o preocupaciones de seguridad. El perfil farmacocinético se tradujo con éxito de modelos preclínicos a sujetos humanos. Se espera presentar los datos clínicos en una importante conferencia médica en el primer trimestre de 2025.
Satellos Bioscience (OTCQB: MSCLF)는 SAT-3247의 1b상 임상 시험에서 중요한 이정표에 도달했으며, 첫 번째 뒤셴 근육 이영양증(DMD) 참여자가 치료를 받았습니다. 회사는 모든 다섯 개의 단일 용량 상승(SAD) 집단과 첫 두 개의 다중 용량 상승(MAD) 집단에 대한 건강한 자원봉사자의 등록을 성공적으로 완료했으며, 보고된 부작용은 없습니다.
1b상 시험은 유전적으로 확인된 DMD를 가진 최대 10명의 성인 참여자를 등록하는 것을 목표로 하며, 28일 동안의 개방 라벨 단일 용량 연구를 통해 안전성과 약물 동태를 평가합니다. 초기 결과는 SAT-3247이 잘 견딜 수 있었으며, 약물 관련 부작용, 비정상적인 임상 결과 또는 안전성 우려 사항이 없다는 것을 보여줍니다. 약물 동태 프로필은 전임상 모델에서 인간 대상에게 성공적으로 전이되었습니다. 임상 데이터는 2025년 1분기에 열리는 주요 의학 회의에서 발표될 것으로 예상됩니다.
Satellos Bioscience (OTCQB: MSCLF) a atteint une étape significative dans son essai clinique de phase 1b pour SAT-3247, avec le premier participant atteint de dystrophie musculaire de Duchenne (DMD) recevant un traitement. L'entreprise a réussi à compléter l'inscription de tous les cinq groupes de doses uniques ascendantes (SAD) et des deux premiers groupes de doses multiples ascendantes (MAD) chez des volontaires sains, sans effets indésirables rapportés.
L'essai de phase 1b vise à recruter jusqu'à 10 participants adultes atteints de DMD confirmée génétiquement dans une étude de dose unique à étiquette ouverte de 28 jours afin d'évaluer la sécurité et la pharmacocinétique. Les résultats initiaux montrent que SAT-3247 a été bien toléré, sans événements indésirables liés au médicament, ni résultats cliniques anormaux, ni préoccupations en matière de sécurité. Le profil pharmacocinétique s'est traduit avec succès des modèles précliniques aux sujets humains. Les données cliniques devraient être présentées lors d'une importante conférence médicale au premier trimestre 2025.
Satellos Bioscience (OTCQB: MSCLF) hat einen bedeutenden Meilenstein in seiner Phase 1b-Studie zu SAT-3247 erreicht, wobei der erste Teilnehmer mit Duchenne-Muskeldystrophie (DMD) behandelt wurde. Das Unternehmen hat erfolgreich die Einschreibung aller fünf Gruppen für einmalige, steigende Dosen (SAD) sowie der ersten beiden Gruppen für mehrere, steigende Dosen (MAD) bei gesunden Probanden abgeschlossen, wobei keine unerwünschten Nebenwirkungen gemeldet wurden.
Die Phase 1b-Studie zielt darauf ab, bis zu 10 erwachsene Teilnehmer mit genetisch bestätigter DMD in einer 28-tägigen, offenen, Einzeldosisstudie zu rekrutieren, um Sicherheit und Pharmakokinetik zu bewerten. Erste Ergebnisse zeigen, dass SAT-3247 gut vertragen wurde, ohne medikamentenbedingte unerwünschte Ereignisse, abnormale klinische Befunde oder Sicherheitsbedenken. Das pharmakokinetische Profil wurde erfolgreich von präklinischen Modellen auf menschliche Probanden übertragen. Klinische Daten werden voraussichtlich auf einer wichtigen medizinischen Konferenz im ersten Quartal 2025 präsentiert.
- Successful completion of SAD cohorts and first two MAD cohorts with no adverse effects
- Clean safety profile with no drug-related adverse events or abnormal findings
- Successful translation of pharmacokinetic profile from pre-clinical to human studies
- Advancement to Phase 1b trial in DMD patients
- None.
- Marks successful completion of full enrollment of SAD cohorts and the first two MAD cohorts in the healthy volunteer study
- Up to 10 adult participants with genetically confirmed Duchenne Muscular Dystrophy (“DMD”) to be enrolled in the now ongoing Phase 1b trial in DMD patients
- Phase 1 data to be presented at major medical conference in Q1 2025
“The dosing of our first DMD participant is a major milestone in evaluating SAT-3247’s potential benefit for DMD patients,” said Frank Gleeson, Satellos Co-founder and CEO. “We are very pleased to have safely completed the full enrollment and dosing of all five cohorts in the single ascending dose (“SAD”) study and the first two multiple ascending dose (“MAD”) cohorts. This reinforces our view that SAT-3247 has potential to be a safe and tolerable, once-daily, oral treatment option for DMD patients. We look forward to continuing to advance SAT-3247 as a potential disease modifying therapy for degenerative muscle conditions.”
Satellos expects to enroll up to 10 adult participants with genetically confirmed DMD in a 28-day, open-label, single dose cohort to assess safety and PK properties in patients and explore potential pharmacodynamic markers.
Clinical data results to date:
Healthy volunteers dosed with SAT-3247 in the SAD cohorts of the study and the first cohort of the MAD did not experience any adverse side effects and the drug was well tolerated. Safety findings include:
- No drug-related adverse events reported
- No abnormal findings on clinical labs
- No abnormal findings on vitals
- No abnormal findings on ECG
- No abnormal findings on physical exams
In addition, the PK profile of SAT-3247 in healthy volunteers translated effectively from pre-clinical models into human subjects.
About the Phase 1 DMD Trial
The Phase 1 clinical trial is comprised of two components. In the first component, 72 healthy adult volunteers are being enrolled in a blinded, randomized, placebo-controlled study to assess the safety and pharmacokinetic properties of SAT-3247. Volunteers are 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 and up to 10 adult participants 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 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 initially in Duchenne muscular dystrophy (DMD). Satellos has generated a significant body of preclinical evidence in DMD to support its discovery that correcting muscle stem cell polarity with SAT-3247 has the potential to restore skeletal muscle regeneration to repair and strengthen muscle that has been damaged. The Company’s lead drug candidate, SAT-3247, is currently in clinical development as a potential disease-modifying treatment for DMD. Additionally, Satellos is leveraging its breakthrough research and proprietary discovery platform MyoReGenX™, to identify degenerative muscle diseases 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, statements regarding the expected timing for the presentation of Phase 1 data; the expectations regarding enrollment in the Company's trial; 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 into clinical trials; the pharmacodynamic properties and mechanism-of-action of SAT-3247; 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; 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/20241211960957/en/
Investors: Liz Williams, CFO, ir@satellos.com
Media: Jessica Yingling, Ph.D., jessica@litldog.com, +1.858.344.8091
Clinical Trial Info: medicalinfo@satellos.com
Source: Satellos Bioscience Inc.
FAQ
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