I understand completely about the urgency/need/anxiety around the loss of ambulation and while the discussion today suggested that muscle quality presents a significant factor with regard to uptake. But please remember that, when you are thinking about independence, it is actually the upper body strength that is essential. At this time, there are no validated measures for upper body strength. We will be getting together with our international partners to discuss this urgent need and do whatever it takes to move forward. As we think about outcome measures - stabilization of disease (no further progression) would be remarkable for instance. OR turning over in bed which improves the life of both the individual as well as his caregivers. The important point here is that the goal is to EndDuchenne, stop the progression for every boy.
About the dystrophin expression the answer was “unequivocal, wide spread, robust response in terms of dystrophin positive fibers” and “results highly competitive with Prosensa”. Very important, no immune response was detected! They cannot provide any data at this time, the results will be published.
Important, about PPMO, only the work funded by Charley’s Fund for skipping exon 50 using PPMOs was mentioned. One such trial is possible to start in 2010 in the US. Other than that, skipping exon 51 in US could start in 2009 using PMO (not PPMO!) after the clinical hold from FDA is lifted. Unfortunately, all the info coming from Eric Hoffman’s group about starting trials using PPMOs for other exons like 51 etc. in 2009 doesn’t seem to hold. The only trial planned using PPMOs is for skipping exon 50, work funded by Charley's Fund.
The earliest they could fill for market approval (if everything works fine) would be 2012.
Of course, they would like to find a partner with deep pockets, which would allow for conducting parallel trials for several exons.
My understanding from the CC was that the hold is until the mouse toxicology work is completed. Now I am not sure how they were able to start the trial in UK w/o complete toxicology data (?). Are the FDA requirements a lot more strict than the EMEA's?
Of course, the hope is that the first results from the UK systemic trial will be convincing and FDA will lift the hold. I think he mentioned that the results for the first cohort of patients in that trial could be available Q2-Q3 2009.
The conference call mentioned that the IND status in the US for this drug is on clinical hold by the FDA. Does anyone know why?