EDG-5506 (Edgewise Therapeutics) is a small molecule which limits the recruitment of fast muscle fibres during muscular effort, protecting the muscle from possible mechanical damage. This molecule is being evaluated in several trials, some of the results of which have been shared in recent months:
- A phase I trial in participants aged between 18 and 55, including 97 healthy volunteers and 7 patients with walking Becker muscular dystrophy, showed that EDG-5506 administered for 14 days (several doses compared with placebo) was well tolerated, with no serious side-effects, the most frequent being dizziness and drowsiness. In BMD patients, creatine kinase (CK) levels fell by an average of 70%, fast fibre troponin I by 98% and myoglobin by 45%.
- In the ARCH trial, two years of treatment with EDG-5506 in 12 walking patients (aged 18 to 55) enabled NSAA scores to be maintained at significantly higher levels compared with data from external natural history controls (+2.3 points after 1 year of treatment, +3.2 points at 18 months and +3.1 points at two years).
- The CANYON trial in 40 adults (aged 18 to 50) and 29 adolescents (aged 12 to 18) with BMD confirmed the good safety profile of EDG-5506. In the adult group, 12 months’ treatment with EDG-5506 reduced the level of CK enzymes in the blood by 28%, and resulted in relatively stable NSAA functional scores throughout treatment (63% of patients treated were stable or improved) and higher results (+1.12 points on average) compared with the placebo group, as well as a faster speed for climbing 4 steps (-0.12 seconds).
The GRAND CANYON trial currently underway, particularly in France, will provide results on a larger scale. These are expected at the end of 2025.
