Prosensa has completed their Phase II systemic delivery. AVI’s dose escalation trials are still in progress in the UK. Both companies are expected to discuss progress to date during the annual World Muscle Society meeting in Geneva, Switzerland, September 9-12, 2009. Both companies have targeted skipping exon 51 and trials were done to prove safety and to determine therapeutic dose. In both cases biopsies were performed before and following delivery of the exon 51 skipping chemistry.
Next steps include multi-centered trials. Prosensa is in discussion with regulatory agencies and has plans to open trials skipping exon 51 and exon 44. These multi-centered trials are expected to be Phase IIb trials. AVI has announced plans for trials skipping exons 51 and 50. The multi-centered trials will concentrate on efficacy, paving the way for exon skipping as a potential therapy for Duchenne.
Many of the Duchenne specific foundations are providing financial support to academic laboratories collaborating with Industry partners and/or to AVI or Prosensa – Action Duchenne (UK), AFM, Charley’s Fund, Cure Duchenne, DPP (Italy, the Netherlands), MDA, PPMD (USA) and UPPMD.
As always, we will keep you up-to-date as information becomes available.