SAT-3247 / Satellos Biosci - LARVOL DELTA

Satellos expects to report…data from the LT-001 study in adults with DMD in Q1 2026. (PharmiWeb)

P2 data • Duchenne Muscular Dystrophy

Satellos Receives Clearance by U.S. FDA and Global Regulators to Initiate Pediatric Phase 2 Study of SAT-3247 for Duchenne Muscular Dystrophy (PharmiWeb) - "...the United Kingdom’s Medicine and Healthcare products Regulatory Agency (MHRA) granted authorization of the company’s Clinical Trial Application (CTA); Australia’s Human Research Ethics Committee (HREC) accepted the Therapeutic Goods Administration’s (TGA’s) Clinical Trial Notification (CTN) scheme for regulatory authorization; and the Medicines and Medical Devices Agency of Serbia (ALIMS) approved the company’s CTA. The clinical trial application is still under review in the European Union and Canada in accordance with established timelines and procedures...The first participant is expected to be enrolled into the study by the end of 2025...Satellos expects to report initial interim data from BASECAMP in Q2 2026..."

IND • New P2 trial • Duchenne Muscular Dystrophy

SAT-3247 CLINICAL PROGRESS (Satellos Press Release) - "In September 2025, Satellos announced the submission of an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) to initiate a Phase 2 clinical trial of SAT-3247 in ambulatory children with Duchenne muscular dystrophy (Duchenne or DMD). Parallel global regulatory filings were submitted in the United Kingdom, Europe, Serbia and Australia, with reviews progressing as expected. On November 13, 2025, Satellos submitted a regulatory filing to Health Canada. The Company continues to expect to enroll the first patient into the Phase 2 study by the end of calendar 2025."

Filing • IND • New P2 trial • Duchenne Muscular Dystrophy

Satellos plans to enroll additional patients beyond those returning from the previously reported study and such plans include expansion of LT-001 into the United States. (Satellos Press Release)

Trial status • Duchenne Muscular Dystrophy

Satellos presented new data at the 30th Annual Congress of the World Muscle Society that demonstrated tolerability and initial efficacy of SAT-3247 in adults with DMD. (Satellos Press Release) - "Phase 1b participants demonstrated a 118.6% mean improvement in maximum grip strength in the dominant hand and 97.9% in the non-dominant hand; Phase 1b participants also exhibited a 5.8% mean improvement in respiratory function as measured by predicted forced vital capacity (FVC)...The first patient was dosed in the long-term follow-up, open-label study (LT-001) of SAT-3247 in adult males with DMD...Initial results from patients returning from the Phase 1b trial are anticipated following a further two months of drug treatment for a total of three months. Satellos currently expects to report these data and findings during calendar Q1 2026."

P1 data • Trial status • Duchenne Muscular Dystrophy

A Long-term Follow-up Study for Participants That Completed the SAT-3247-CL-101 Study (clinicaltrials.gov) - P2 | N=10 | Enrolling by invitation | Sponsor: Satellos Bioscience, Inc. | Not yet recruiting ➔ Enrolling by invitation

Enrollment open • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

First in Human SAD/MAD Safety and PK Study With Adult DMD Safety and PK Cohort (clinicaltrials.gov) - P1 | N=77 | Completed | Sponsor: Satellos Bioscience, Inc. | Recruiting ➔ Completed

Trial completion • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Satellos Presents Initial Data from the Phase 1 Trial of SAT-3247 at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference (Businesswire) - P1 | N=82 | NCT06565208 | Sponosr: Satellos Bioscience, Inc. | "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."

P1 data • Duchenne Muscular Dystrophy

A Long-term Follow-up Study for Participants That Completed the SAT-3247-CL-101 Study (clinicaltrials.gov) - P2 | N=10 | Not yet recruiting | Sponsor: Satellos Bioscience, Inc.

New P2 trial • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Satellos Announces Upcoming Presentation at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference (Businesswire) - "'The oral presentation at this year’s MDA conference is an exciting opportunity and significant development milestone for Satellos to showcase our first-ever Phase 1 data for SAT-3247.'"

P1 data • PK/PD data • Duchenne Muscular Dystrophy

Satellos Announces Completed Enrollment of all Four Multiple-Ascending Dose Cohorts for the Phase 1 Clinical Trial of SAT-3247 in Healthy Volunteers (Businesswire) - "Company remains on track to report Phase 1a data from both the Single- and Multiple-Ascending Dose (SAD and MAD) cohorts in the healthy volunteer portion of the study in 1Q 2025 at an upcoming major medical meeting; The Phase 1b portion of the trial, in DMD patients, is underway with the intention of enrolling up to 10 adult volunteers with genetically confirmed DMD during 1Q 2025; Phase 2 IND filing on track to be submitted by end of 1Q 2025...Satellos Bioscience Inc...today announced it has completed enrollment of all four multiple-ascending dose (MAD) cohorts for the Phase 1 clinical trial of SAT-3247 in healthy volunteers."

Enrollment closed • IND • P1 data • Duchenne Muscular Dystrophy

First in Human SAD/MAD Safety and PK Study With Adult DMD Safety and PK Cohort (clinicaltrials.gov) - P1 | N=82 | Recruiting | Sponsor: Satellos Bioscience, Inc.

New P1 trial • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy