ann: data from final cohort of patients, page-44

"But the only thing im not sure about is the sample size they are using in their trials... how many people exactly?"

Only 12 patients as is often the case with rare diseases the pool is not exactly large to enrol from in addition they are also the individuals that have failed on every other available treatment option - PAT-SM6 has in effect been a last chance for these individuals grabbing from an even smaller pool but as has been show in the small number of patients the result has come against the odds where all others have failed. Of course trials forward will be larger patient numbers and it will be very interesting to see the structure of the combination trial and enrolment target. The small number to date though hasn't been insignificant in that the both EU & US have been convinced enough to grant the Orphan Designation & Amgen to move in with funding the next phase - the results today from the 4th Cohort with the dosage doubled have only strengthened the resolve of the above parties on decisions made prior.


I personally don't care what others do but I think anyone selling here is only going to regret that decision in the very near future.

Good luck.