Wave Life Sciences Announce the Start of the FORWARD-53 Clinical Study
On the 15th of December 2023, Wave Life Sciences announced the initiation of their phase 2 FORWARD-53 study. The potential registrational study is now fully enrolled and aims to evaluate WVE-N531 in 10 boys with Duchenne muscular dystrophy that are amenable to exon 53 skipping treatment.
The primary endpoint of this study is functional dystrophin protein levels which will be measured following 24 and 48 weeks of every other week dosing at 10 mg/kg. The trial is also evaluating safety and tolerability, digital and functional endpoints. Wave Life Sciences expects to deliver data, including dystrophin expression from muscle biopsies, in 2024.
What is WVE-NE53?
WVE-NE53 is an exon skipping therapy that has shown good potential in both the preclinical trials, and the proof-of-concept clinical trial in boys with Duchenne. The treatment has been demonstrated to increase the level of dystrophin in the skeletal muscle as well as the heart and diaphragm. WVE-NE53 was shown to be generally safe and tolerable in these trials.
What is Exon-53 skipping?
Exon skipping is believed to be applicable to 80% of Duchenne patients, but the treatments are not ‘one size fits all’ and precise exon targeting is required. The WVE-NE53 targets Duchenne where the causal mutation has occurred in exon 53, which accounts for approximately 10% of the Duchenne community. As the cellular machinery reads the DNA to transcribe for the protein Dystrophin, this treatment causes the cellular machinery to skip the mutated exon 53, allowing for the production of a truncated (or shortened) functional dystrophin protein.
Read more about FORWARD-53 and WVE-NE53
More information on science behind WaveLife’s therapeutic research
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