Biotech Values

[jq1234]

1) The trial actually closed enrollment after enrolling multiple cancer types and allowing co-administered drugs of many types for about 2 years. (6 cancer types and thus 6 potential co-drugs)

It was exploratory trial, trying different drug combinations for different types of cancer.

2) AEZS Investor status gives status on PR/CR/ORR in Aug 2009 for 35 randomized patients per +/- cap.

3) In Sept 2009 it re-opens the trial to "enrollment by invitation" of only CRC patients

But the recently published data doesn't meaningfully expand the number of patients - what happened to the invited patients, or was it only 3 invitees (the latest paper, pointed to by genisi, had 38 patients)?

You can't depend on Clinicaltrial.gov for accurate timeline. More often than not, update time lag is signficant. Updated in Sept 2009 doesn't mean the evet actually happened in Sept 2009.

AEZS's 35 patients are the same as 38 patients in final analysis, the difference AEZS didn't include 3 patients couldn't be assessed for response.

I think the more important question is how number of 38 patients was decided, and what assumption, ie power calculation etc was made. These are important information companies should disclose.

Perifosine in phase I with different dosing regimens as single agent, found no response in CRC. Thus this could be the drug finally found the right combination for right disease setting, or the drug worked in a trial for patients that have certain hidden characteristics/inbalance that cannot be repeated in large trial. I don't think anyone knows at this point.