Biotech Values

[poorgradstudent]

ONXX:

1. Since the trial was open label, it is possible ONXX sensed it might not achieve original PFS primary endpoint either due to small sized trial (80 patients) or other reason, thus amend the trial with larger sample size (300 patients) and OS as primary endpoint.

2. ONXX is uneasy with the recent tough talk of accelerated approval out of ODAC and FDA, thus want to play safe by amending the trial to achieve two possible goals: one is in case NDA for accelerated approval is rejected, this study can step in; two is in case FDA insists confirmatory trial to be substantial underway before granting accelerated approval to carfilzomib, this study can be used as confirmatory trial.

I lean toward possibility number 2.

They already have the ASPIRE trial running since last year, so I'm not sure that your point #2 is the most likely concern.

I lean towards #1. The company has money and a good shot at carfilzomib approval in the US (and, therefore, more income). In that respect I don't see any reason to be cheap / foolish in running an 80 patient trial. This comes off as a conservative move to me.