ATA-200 / Atamyo Therap - LARVOL DELTA

Early Results from an Interventional Phase 1 Pediatric Study of ATA‑200 Gene Therapy in LGMDR5 Demonstrate Favorable Safety and Encouraging Pharmacodynamic Activity (ESGCT 2026) - No abstract available

Clinical • Gene therapy • P1 data • PK/PD data • Gene Therapies • Pediatrics

Early Results From an Interventional Phase 1 Pediatric Study of ATA‑200 Gene Therapy in LGMD-R5 Demonstrate Favorable Safety and Pharmacodynamic Activity at One Year (ESGCT 2026) - No abstract available

Clinical • Gene therapy • P1 data • PK/PD data • Gene Therapies • Pediatrics

GLP-compliant six-month toxicity and biodistribution study following single intravenous administration of ATA-200 for LGMDR5 treatment in Sprague-Dawley rats (ASGCT 2026) - "Based on the absence of adverse findings at any dose level, the No-Observed-Adverse-Effect Level (NOAEL) was considered to be 20xXMED under the experimental conditions of the study, supporting progression to clinical development. Based on the rarity of the disease, the high unmet medical need, as well as the good safety profile of ATA-200 six months after administration in the pivotal GLP compliant toxicity rat study with large safety margin of seven compared to the expected maximal clinical dose and considering the potential therapeutic benefit of restoring SGCG expression in muscle and thus improving muscular function, the benefit-risk ratio for ATA-200 can be reasonably predicted to be favorable."

Preclinical • Gene Therapies • Hematological Disorders • Muscular Dystrophy • Ophthalmology • FABP3

Gene therapy for LGMDR5: Preliminary safety data from a Phase 1 study of ATA-200 (ASGCT 2026) - P1 | "All patients received corticosteroids (1mg/kg/day for one month followed by progressive tapering) and sirolimus (2 mg/day for 3 months followed by 1mg for an additional 3 months) starting at the day of infusion. The 6-month muscle biopsy confirmed target engagement with widespread expression of gamma-sarcoglycan, with very high proportion of SGCG-positive fibers. Conclusion Systemic delivery of ATA-200 at 1.0E+14 vg/kg was well tolerated in the initial LGMDR5 cohort and showed early evidence of transgene expression and functional benefit, supporting continued clinical development."

Clinical • Gene therapy • P1 data • CNS Disorders • Duchenne Muscular Dystrophy • Gene Therapies • Muscular Dystrophy

ATA-200 Gene Therapy Trial in Patients With LGMDR5 (clinicaltrials.gov) - P1 | N=4 | Active, not recruiting | Sponsor: Atamyo Therapeutics | Recruiting ➔ Active, not recruiting | Phase classification: P1/2 ➔ P1

Enrollment closed • Phase classification • Gene Therapies

Impact of Aortic Valve Calcification on Functional Valve Area and Cardiac Structure and Function in a Phase 2 Trial of Ataciguat (AHA 2025) - P2 | "ATA may have other favorable effects on ventricular, valvular, and/or vascular pathophysiology relevant to patients with CAVS.Hypothesis:We hypothesized that slowing the rate of AVC deposition is associated with improvements in valvular compliance and in measures of cardiac structure and function.A phase 2 study randomized patients with moderate CAVS 1:1 to receive ATA 200 mg/day or placebo for up to 12 months (NCT02481258). These data suggest that slowing the rate of AVC deposition with ATA may result in improvements in CO through improved myocardial function and valvular compliance. Larger controlled trials are needed to assess such favorable myocardial/valvular effects that may help to preserve functional capacity and slow progression to HF."

P2 data • Cardiovascular • Congestive Heart Failure • Heart Failure

ATA-200 Dose-escalation Gene Therapy Trial in Patients With LGMDR5 (clinicaltrials.gov) - P1/2 | N=6 | Recruiting | Sponsor: Atamyo Therapeutics | Trial completion date: Jan 2032 ➔ Jan 2031 | Trial primary completion date: Jul 2026 ➔ Oct 2026

Trial completion date • Trial primary completion date • Gene Therapies

ATA-200 Dose-escalation Gene Therapy Trial in Patients with LGMDR5 (clinicaltrials.gov) - P1/2 | N=6 | Recruiting | Sponsor: Atamyo Therapeutics | Not yet recruiting ➔ Recruiting

Enrollment open • Gene Therapies

ATA-200 Dose-escalation Gene Therapy Trial in Patients With LGMDR5 (clinicaltrials.gov) - P1/2 | N=6 | Not yet recruiting | Sponsor: Atamyo Therapeutics | Trial completion date: Jun 2030 ➔ Dec 2031 | Trial primary completion date: Dec 2025 ➔ Jun 2026

Gene therapy • Trial completion date • Trial primary completion date • Viral vector • Gene Therapies

ATA-200 Dose-escalation Gene Therapy Trial in Patients With LGMDR5 (clinicaltrials.gov) - P1/2 | N=6 | Not yet recruiting | Sponsor: Atamyo Therapeutics | Initiation date: Jan 2024 ➔ May 2024

Gene therapy • Trial initiation date • Viral vector • Gene Therapies

ATA-200 Dose-escalation Gene Therapy Trial in Patients With LGMDR5 (clinicaltrials.gov) - P1/2 | N=6 | Not yet recruiting | Sponsor: Atamyo Therapeutics

Gene therapy • New P1/2 trial • Viral vector • Gene Therapies • Muscular Dystrophy