PRO044

Prosensa’s second product in development, PRO044, also highly sequence-specific, specifically induces exon 44 skipping in the DMD gene. This is applicable to around 6% of all DMD patients, including patients with deletions of exon 43, exon 45, exons 38-43, exons 40-43, exons 42-43, and exons 45-54. Similar to drisapersen (PRO051/GSK2402968), PRO044 is extensively tested in vitro in series of cultured muscle cells from patients with different relevant mutations, and in the hDMD mouse model.

The underlying chemistry and mechanism are similar to drisapersen (PRO051/GSK2402968). A Phase I/IIa dose-escalating safety study is currently on-going. Each trial is posted at the website www.clinicaltrials.gov as soon as the study design is finalized and all the required approvals of authorities and ethics committees are granted.

PRO044 has obtained an orphan drug designation in the EU and the US.