PRO044
| Indication | Compound | Discovery | Pre-clinical | Phase I / II | Phase III |
|---|---|---|---|---|---|
| Duchenne Muscular Dystrophy (DMD) | PRO044 | 100% finished | 100% finished | 70% finished |
Prosensa’s second product in development, PRO044, also highly sequence-specific, specifically induces exon 44 skipping in the DMD gene. This is applicable to 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 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 PRO051/GSK2402968. Prosensa requested authorization from European national authorities for a phase I/IIa dose-escalating safety study. This study has been initiated in the last quarter 2009 and first patients have been enrolled.
PRO044 has obtained an orphan drug designation in the EU and the US.
