Safety, efficacy, and pharmacodynamic results show good tolerance of GNT0004 associated with transient immunological prophylactic treatment, as well as efficacy data in terms of microdystrophin expression, CPK reduction, and clinical criteria (NSAA, timed tests). Patients treated at the effective dose show prolonged improvement or stabilization of motor functions and significant persistent reduction in creatine kinase (CPK) levels, a key marker of muscle damage.
Key Results of GNT0004 Clinical Trial
- GNT0004 product confirms its clinical efficacy with stabilization of motor functions in patients treated at the effective dose for up to 2 years.
- Sustained significant reduction in levels of creatine phosphokinase (CPK), a biomarker of muscle damage, with an average decrease of over 75% at 18 months in the 3 patients treated at the effective dose.
- Pivotal phase planned for mid-2025 in Europe and the United States.
One-year post-treatment, the comparison of the three patients treated at the effective dose with a group of 34 untreated patients, matched by age and followed in the same centers and by the same practitioners, shows a difference of +4.7 points in the score obtained using the internationally recognized clinical evaluation scale NSAA between treated and untreated patients.
At 24 months post-treatment, key observations include:
- For the 2 patients who reached 2 years post-treatment out of the 3 treated at the effective dose, the trial shows stabilization of motor functions measured by the NSAA scale , while untreated patients from the parallel natural history study showed a continuous and significant average decline in NSAA. For one treated patient, the observed improvement allowed reaching the maximum score of 34 at 12 months, confirmed at 24 months post-treatment.
- Stabilization of CPK reduction between 50% and 87% on average: >75% at 18 months post-treatment (data from the 3 patients treated at the effective dose), and persistent (up to 24 months follow-up for the first two patients treated at this dose).
- The reassuring safety profile of the gene therapy drug is confirmed two years after injection, without the occurrence of serious adverse effects at the selected dose, which is notably lower than that used for other gene therapy products under development for Duchenne Muscular Dystrophy.
Learn More: Everything You Should Know About Genethon’s Gene Therapy GNT0004
