Trust is a problem, and it will be until something really positive that cannot be disputed occurs. I believe they do have technology that works, but the proof can only come from properly designed trials. Clearly the failed trial wasn't well designed, but believe that was KERX's responsibility. In the Phase II the overwhelming number of patients were Stage 3, yet Stage 4 was all that were used in the Phase III. In my view the mistake was using the data from to small a sample to attempt a trial that would be dramatically faster and cheaper to run. Had they either run the Phase II with more Stage 4 patients to learn more about the drug in that mix, they'd either not have tried in that group, or they'd have determined a protocol that would have worked in that patient group. More importantly, if they'd gone with Stage 3 patients, I believe they'd have had success. Certainly these patients live longer, so the trial would have taken much longer, it therefore would have been far more expensive, but in the end it would have much more likely been successful.
AEZS is taking a better approach with AEZS-108. Without the companion diagnostic they've chosen a cancer where 80% of the patients have the LHRH indicator, I believe that's it's designation, but am not certain. While other cancers they could have tried have shorter life spans, they don't have 80% with the right indicator. Had they had the companion diagnostic, they might very well have chosen a cancer where 30% or less have the right identifier, the key was getting the drug to just that 30%. As is, they have a trial where 80% could see benefits, and if benefits are seen by substantially less, the drug should still garner approval, the key is people seeing benefits that otherwise wouldn't be seen. I believe it should happen, and it should happen with Perifosine in MM as well. As always, the key is what should happen actually happening.
Gary |
