zocaglusagene nuzaparvovec (AT845) / Astellas - LARVOL DELTA

FORTIS: Gene Transfer Study in Patients With Late Onset Pompe Disease (clinicaltrials.gov) - P1/2 | N=11 | Active, not recruiting | Sponsor: Astellas Gene Therapies | Recruiting ➔ Active, not recruiting | N=18 ➔ 11 | Trial completion date: Nov 2029 ➔ Feb 2030 | Trial primary completion date: Nov 2029 ➔ Feb 2030

Enrollment change • Enrollment closed • Trial completion date • Trial primary completion date • Gene Therapies • Pompe Disease

FORTIS: Gene Transfer Study in Patients With Late Onset Pompe Disease (clinicaltrials.gov) - P1/2 | N=18 | Recruiting | Sponsor: Astellas Gene Therapies | N=12 ➔ 18

Enrollment change • Gene Therapies • Pompe Disease

FORTIS: Gene Transfer Study in Patients With Late Onset Pompe Disease (clinicaltrials.gov) - P1/2 | N=12 | Recruiting | Sponsor: Astellas Gene Therapies | Trial completion date: Apr 2029 ➔ Aug 2029 | Trial primary completion date: Apr 2029 ➔ Aug 2029

Trial completion date • Trial primary completion date • Gene Therapies • Pompe Disease

FORTIS: Gene Transfer Study in Patients With Late Onset Pompe Disease (clinicaltrials.gov) - P1/2 | N=12 | Recruiting | Sponsor: Astellas Gene Therapies | Trial primary completion date: May 2025 ➔ Apr 2029

Trial primary completion date • Gene Therapies • Pompe Disease

AT845 gene replacement therapy for Late Onset Pompe disease: preliminary muscle biomarkers and histopathology results from FORTIS, a phase 1/2 open-label clinical study (SSIEM 2023) - P1/2 | "The muscle biomarker and histopathology results following AT845 gene therapy administration appear promising. Additional analyses will be conducted to correlate these data with functional outcomes as the trial continues."

Biomarker • Clinical • P1/2 data • Gene Therapies • Lysosomal Storage Diseases • Metabolic Disorders • Pompe Disease • Rare Diseases

FORTIS: Gene Transfer Study in Patients With Late Onset Pompe Disease (clinicaltrials.gov) - P1/2 | N=12 | Recruiting | Sponsor: Astellas Gene Therapies | Trial completion date: Nov 2028 ➔ May 2029 | Trial primary completion date: Dec 2024 ➔ May 2025

Trial completion date • Trial primary completion date • Gene Therapies • Pompe Disease

FORTIS: Gene Transfer Study in Patients With Late Onset Pompe Disease (clinicaltrials.gov) - P1/2 | N=12 | Recruiting | Sponsor: Astellas Gene Therapies | Active, not recruiting ➔ Recruiting | Trial completion date: Jan 2027 ➔ Nov 2028 | Trial primary completion date: Dec 2022 ➔ Dec 2024

Enrollment open • Trial completion date • Trial primary completion date • Gene Therapies • Pompe Disease

"FDA delivers good news to @AstellasUS, relaxing a clinical hold on its #genetherapy AT845 for late-onset #Pompedisease https://t.co/4wLvD3OXkK" (@pharmaphorum)

Clinical • FDA event • Gene therapy • Gene Therapies • Pompe Disease

FORTIS: Gene Transfer Study in Patients With Late Onset Pompe Disease (clinicaltrials.gov) - P1/2 | N=12 | Active, not recruiting | Sponsor: Astellas Gene Therapies | Recruiting ➔ Active, not recruiting

Enrollment closed • Gene Therapies • Pompe Disease

"Another setback for @AstellasGeneTx' #genetherapy programmes, as #Pompe disease candidate AT845 is placed on an FDA clinical hold due to an adverse event" (@pharmaphorum)

Adverse events • Clinical • FDA event • Gene Therapies • Pompe Disease

Muscle-directed gene therapy corrects Pompe disease and uncovers species-specific GAA immunogenicity. (PubMed, EMBO Mol Med) - "AT845 is an adeno-associated viral vector designed to express human GAA specifically in skeletal muscle and heart...Western blot analysis showed abnormal processing of human GAA in cynomolgus muscle, adding to the species-specific effects of enzyme expression. Overall, these studies show that AAV-mediated GAA delivery to muscle is efficacious in Gaa mice and highlight limitations in predicting the toxicity of AAV vectors encoding human proteins in non-human species."

Journal • Gene Therapies • Immunology • Inflammation • Lysosomal Storage Diseases • Metabolic Disorders • Pompe Disease • Rare Diseases

Phase 1/2 Trial of Gene Therapy AT845 Recruiting Patients With Late-onset Pompe (Pompe Disease News) - "A Phase 1/2 clinical trial is recruiting adults with late-onset Pompe disease to test Audentes Therapeutics‘ investigational gene therapy AT845, the company has announced. The trial (NCT04174105) will be conducted at clinical sites in the U.S., Germany, and the U.K. It aims to recruit up to eight participants, ages 18 to 80, who have received Lumizyme (alglucosidase alfa, marketed as Myozyme in Europe), an enzyme replacement therapy, for at least two years and as a stable dose for six months prior to enrollment."

Enrollment status • Genetic Disorders • Pompe Disease