fordadistrogene movaparvovec (PF-06939926) / Pfizer - LARVOL DELTA

A Study to Understand the Long-term Safety and Effects of an Experimental Gene Therapy for Duchenne Muscular Dystrophy. (clinicaltrials.gov) - P3 | N=6 | Active, not recruiting | Sponsor: Pfizer | Trial completion date: May 2039 ➔ Aug 2025 | Trial primary completion date: May 2039 ➔ Aug 2025

Trial completion date • Trial primary completion date • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Muscular Dystrophy • TNNI3

Study of Fordadistrogene Movaparvovec in Early Stage Duchenne Muscular Dystrophy (clinicaltrials.gov) - P2 | N=10 | Active, not recruiting | Sponsor: Pfizer | Trial completion date: Jan 2029 ➔ Jun 2025

Trial completion date • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy • TNNI3

AAV9 Neutralizing Antibody Seroconversion in Household Contacts Of Participants With Duchenne Muscular Dystrophy Receiving Fordadistrogene Movaparvovec (ASGCT 2025) - No abstract available

Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Study of Fordadistrogene Movaparvovec in Early Stage Duchenne Muscular Dystrophy (clinicaltrials.gov) - P2 | N=10 | Active, not recruiting | Sponsor: Pfizer | Trial primary completion date: Dec 2024 ➔ May 2025

Trial primary completion date • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy • TNNI3

A Study to Evaluate the Safety and Tolerability of PF-06939926 Gene Therapy in Duchenne Muscular Dystrophy (clinicaltrials.gov) - P1 | N=23 | Active, not recruiting | Sponsor: Pfizer | Trial completion date: Mar 2026 ➔ May 2025

Trial completion date • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Muscular Dystrophy • CRP

A Study to Understand the Long-term Safety and Effects of an Experimental Gene Therapy for Duchenne Muscular Dystrophy. (clinicaltrials.gov) - P3 | N=5 | Active, not recruiting | Sponsor: Pfizer | Recruiting ➔ Active, not recruiting | N=250 ➔ 5

Enrollment change • Enrollment closed • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Muscular Dystrophy • TNNI3

Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy (clinicaltrials.gov) - P3 | N=122 | Active, not recruiting | Sponsor: Pfizer | Trial completion date: Apr 2029 ➔ Apr 2039

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

What Are the Key Themes and Sentiments Captured Through Social Listening in Response to Recent Changes in the Duchenne Muscular Dystrophy Treatment Landscape? (ISPOR-EU 2024) - " Using a social media listening platform (Brandwatch), we assessed the difference in stakeholder sentiment before and after the fordadistrogene movaparvovec announcement (June 12 th 2024), and after the announcement of the delandistrogene moxeparvovec-rokl approval (June 20 th 2024). The variability in sentiment in the DMD population over a relatively short time period confirms the ability of social media listening to capture patient perspectives quickly. However, this variability in sentiment over a 10 day time-period also points to a need for methodological scrutiny to reduce bias in social media listening data collection."

Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

A Study to Evaluate the Safety and Tolerability of PF-06939926 Gene Therapy in Duchenne Muscular Dystrophy (clinicaltrials.gov) - P1 | N=22 | Active, not recruiting | Sponsor: Pfizer | Phase classification: P1b ➔ P1

Gene therapy • Phase classification • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Muscular Dystrophy • CRP

Two year muscle MRI observations from a phase 1b trial of fordadistrogene movaparvovec (PF‐06939926) for Duchenne muscular dystrophy (DMD) (ESGCT 2023) - P1b, P2 | "An external reference (ER) cohort was derived from DMD participants (n = 48) with ≥1 yr of randomized domagrozumab treatment (active→placebo, placebo→active, or active→active) from trial NCT02310763 and met eligibility criteria for this trial. Thigh MRI findings are further supported by evaluating upper limb MRI measure. After dosing with FM, quantitative muscle MRI measurements show favorable changes up to 2-yrs that correlate with functional outcomes."

P1 data • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Study of Fordadistrogene Movaparvovec in Early Stage Duchenne Muscular Dystrophy (clinicaltrials.gov) - P2 | N=10 | Active, not recruiting | Sponsor: Pfizer | Trial completion date: Jun 2028 ➔ Jan 2029 | Trial primary completion date: Jul 2024 ➔ Dec 2024

Trial completion date • Trial primary completion date • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy • TNNI3

Fordadistrogene movaparvovec for Duchenne muscular dystrophy: 3-year functional outcomes and changes in thigh and upper-limb muscle volume (MDA 2024) - "Prior to enrollment, 50% of participants were using daily deflazacort and 50% daily prednisone/prednisolone. At 3y, a clinically meaningful preservation of motor function was observed in participants with DMD aged 6 to <8y receiving FM vs an external control. Sustained increases in upper-limb muscle volume were observed. CFB in NSAA was moderately strongly correlated with %CFB in thigh muscle volume."

Late-breaking abstract • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

CIFFREO, a phase 3, randomized, double-blind, placebo-controlled study of fordadistrogene movaparvovec in Duchenne muscular dystrophy (DMD) (MDA 2024) - P3 | "CIFFREO aims to compare the safety and efficacy of FM with placebo in participants with DMD. ClinicalTrials.gov: NCT04281485"

Clinical • Late-breaking abstract • P3 data • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Muscular Dystrophy • Pediatrics

DAYLIGHT, a phase 2 study of fordadistrogene movaparvovec in participants with early-stage Duchenne muscular dystrophy (MDA 2024) - P2 | "Exclusion criteria include any mutation affecting any exon between 9-13 inclusive, a deletion that affects exons 29 and 30, or a deletion affecting any exon between 56-71 inclusive, known contraindication to eculizumab, cardiac pathology eg myocarditis, prior gene therapy treatment, prior or current glucocorticoid treatment, and positive test for neutralizing antibodies to AAV9. DAYLIGHT aims to assess the safety and mini-dystrophin expression of FM in participants with DMD aged ≥2 to <4 years, prior to the development of significant muscle damage. As FM is designed to provide a functional form of dystrophin, it is hypothesized to have clinically meaningful benefits in participants with early DMD."

Late-breaking abstract • P2 data • Cardiovascular • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Inflammation • Muscular Dystrophy • TNNI3

Cardiac safety of fordadistrogene movaparvovec in Duchenne muscular dystrophy after 3 years follow-up from a phase 1b trial (MDA 2024) - "Natural history studies suggest LVEF declines in DMD patients at a rate of ~1.6% per year. Study participants who received a single infusion of fordadistrogene movaparvovec showed LVEF declines consistent with natural history. Appearance of new LGE signal is expected during typical DMD progression and our findings appear consistent with this natural course."

Clinical • P1 data • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Longitudinal changes of Anti-AAV9 and Anti-AAVrh74 antibodies in treated and untreated patients (MDA 2024) - "Subjects received SGT-001 (N=7), Zolgensma (N=7), PF-06939926 (N=2), AT132 (N=1), LYS-SAF302 (N=1), PGTC PD-AAV004 (N=2), all AAV9 products... 169 untreated subjects (115 male) with 1 plasma/serum sample for analysis were included. Median age was 13 (3-37). Of these, 92 (54%) were seropositive for Anti-AAV9 IgG at initial collection."

Clinical • CNS Disorders • Gene Therapies • Immunology

Disease progression modeling of NSAA total score in Duchenne Muscular Dystrophy and treatment effects of fordadistrogene movaparvovec (MDA 2024) - P=N/A, P1b, P2, P3 | "Median simulated CFB at 1 year was greater following FM administration compared with natural disease progression, and higher for younger (6 - < 8 years old) than older (≥ 8 years old) baseline ages. Conclusion The indirect response model described the data well, predicting slower disease progression following FM treatment particularly for younger participants."

Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Fordadistrogene movaparvovec for Duchenne muscular dystrophy: 3-year functional outcomes and changes in thigh and upper-limb muscle volume (MDA 2024) - No abstract available

Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Evaluation of an AAV9-mini-dystrophin gene therapy candidate in a rat model of Duchenne muscular dystrophy. (PubMed, Mol Ther Methods Clin Dev) - "The effective dose was then tested in older DMD rats with a more dystrophic phenotype similar to the pathology observed in older patients with DMD. Except for a less complete rescue of muscle function in the oldest cohort, fordadistrogene movaparvovec was also found to be therapeutically effective in older DMD rats, suggesting that this product may be appropriate for evaluation in patients with DMD at all stages of disease."

Gene therapy • Journal • Preclinical • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Muscular Dystrophy

Two-year muscle MRI observations from a phase 1b trial of fordadistrogene movaparvovec (PF 06939926) for Duchenne muscular dystrophy (DMD) (WMS 2023) - No abstract available

P1 data • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Two-year clinical outcomes with fordadistrogene movaparvovec (FM) for Duchenne muscular dystrophy (DMD) and contextualization with external controls (WMS 2023) - No abstract available

Clinical • Clinical data • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Study of Fordadistrogene Movaparvovec in Early Stage Duchenne Muscular Dystrophy (clinicaltrials.gov) - P2 | N=10 | Active, not recruiting | Sponsor: Pfizer | Recruiting ➔ Active, not recruiting

Enrollment closed • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy • TNNI3

A Study to Understand the Long-term Safety and Effects of an Experimental Gene Therapy for Duchenne Muscular Dystrophy. (clinicaltrials.gov) - P3 | N=250 | Recruiting | Sponsor: Pfizer | Not yet recruiting ➔ Recruiting

Enrollment open • Gene therapy • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Muscular Dystrophy • TNNI3

Two-Year Clinical Outcomes with Fordadistrogene Movaparvovec for Duchenne Muscular Dystrophy (DMD) and Contextualization with External Controls (ASGCT 2023) - P1b | "A second external control group with similar baseline characteristics was constructed using 2-year data from patients with at least 1 year of treatment randomized to active→placebo, placebo→active, or active→active in a null phase 3 domagrozumab trial. At 2 years, fordadistrogene movaparvovec was associated with clinically meaningful preservation of motor function in subjects with DMD when compared with selected external controls with similar pre treatment characteristics. As this was a comparison with nonrandomized groups or pooled clinical trial data there is the potential for confounding bias. Continued assessment of fordadistrogene movaparvovec is warranted in randomized, placebo-controlled trials."

Clinical • Clinical data • Late-breaking abstract • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy (clinicaltrials.gov) - P3 | N=82 | Active, not recruiting | Sponsor: Pfizer | Recruiting ➔ Active, not recruiting

Enrollment closed • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy