PYC, page-25

If we have to wait until approval of Sarepta’s SRP-5051 to light up market interest in PYC then we’re sunk!

The second part of Sarepta's (hoped for) pivotal trial commenced last September and estimated study completion date on the clinical trial website is August 2024. Hence, if the trial is successful, any approval may not come before late 2025.

Still, Sarepta has already shown the promise of its PPMO approach in its Part A trial results which were presented at a muscular dystrophy scientific conference this week.

Compared with Sarepta’s FDA-approved Exondys 51 (which has no conjugated peptide), SRP-5051 has demonstrated 18 x exon-skipping and 8 x dystrophin production using monthly rather than weekly dosing.