In yesterday's presentation during the Q&A Chris laid out the strategy for Accelerated Approval based on the phase II data.
After listening to the strategy, I think there is a reasonable chance of it happening.
If it does, phase III has the drugs paid for by the insurance companies.
Accelerated Approval defines the use of surrogate endpoints.
In this case, it is dystrophin production:
In a placebo controlled trial of 12 patients - 100% - 8 out of 8 of the patients dosed at 30 mg/kg and 50 mg/kg showed increased dystrophin. The 4 placebo patients were then randomized to 30 mg/kg or 50 mg/kg. At the end of 24 weeks of treatment those 4 patients showed dystrophin production.
There will be bunch of numbers to show how much dystrophin and a bunch of slides, all good stuff.
But the great stuff is: 100% of the patients at 30 mg/kg and 50 mg /kg showed dystrophin production.
At 52 weeks of treatment the 8 originally treated patients had no treatment related adverse events. The 4 placebo/delayed treatment patients had no treatment related adverse events at 36 weeks.
In a previous 12 week open label study 8 of 8 patients treated with 10 mg/kg or 20 mg/kg showed dystrophin production.
At 1, 2, and 4 mg/kg 3 of 9 boys showed significant dystrophin production.
In these 19 boys there were no treatment related adverse events.
At this point - The drug is shown efficacious, if adequate doses are administered in terms of the surrogate endpoint and safe.
This data alone MIGHT be enough to approve the drug with a requirement for a confirmatory study.
BUT THERE IS MORE!
The stabilization of the boys in the placebo controlled trial treated at 50 mg/kg on the 6 MWT as compared to the decline in the placebo boys at 26 weeks. A clear demonstration of clinical benefit p<=.012. Hopefully that benefit is still in place in the 48 week data. Comparisons will be more tricky, since the placebo boys are on treatment for 24 weeks at that point. It may be that at 48 weeks, the 30 mg/kg data will also show a benefit.
Then there is the story of at least 1 boy running and jumping. There well may be more of such, but these are icing on the cake.
The statistics and the demonstration of meeting the surrogate endpoint in 100% of the patients and safety in 100% of the patients.
*** I think the chances are pretty good for approval, this assumes the data stacks up like the last data. And the package is well done. Redplate has pointed out the weakness may be in the manufacturing plan.