In today's Lazard CC Scott Cormack said, about 20 minutes in, that because the randomized P2 trial for 427 in metastatic bladder cancer will enroll 180 patients and has a survival endpoint it's "set up as a baby phase 3" and "if you were to hit the statistical endpoint you would go ahead and submit this for approval; at a minimum it would set up to be basically a supportive trial and then you'd only have to do one phase 3 on the back end of that, so it's really set up for a pretty big swing and mainly because of survival as the primary endpoint and the way it's set up." He goes on to say they expect to complete enrollment in late 2013 and expect data in 2014.
This strikes me as pretty big news. Cormack has occasionally hinted in the past that if this trial were successful there's a chance they'd file for approval but I think this is the first time he's said that's what they plan to do. That would mean that within a year of getting results on SYNERGY they could potentially file for approval for a different first-line cancer indication for 427. Granted, since the trial is smaller than SYNERGY there's less statistical power so it will be harder to achieve statistical significance, and they don't have an SPA so it's possible the FDA will insist on a P3. But there have been lots of rumblings recently that the FDA is moving towards allowing approval based on smaller, earlier-stage trials for diseases for which there is no effective treatment -- and bladder cancer has fewer approved treatments than, for example, prostate -- so that's probably behind Cormack's new stance.
Agree. Today's PR says "If either Borealis trial shows a survival advantage, OncoGenex plans to initiate conversations with the Food and Drug Administration about the possibility of a Phase 3 study of OGX-427 in bladder cancer as part of the ORCA program. " Cormack has gone back and forth on whether if the other P2 bladder trial (the first-line trial now called "Borealis-1") succeeds they'd go on to a P3 or just try to file for approval. Obviously it all depends on what the FDA will say, and Cormack has to be careful not to presume they'll give their okay to skipping P3. But this new Boralis-2 trial is a randomized 200-patient trial with participation by Sloan Kettering and Dana Farber so I have to think if the results are positive OGXI's goal will be to file.
And maybe best of all, unlike Borealis-1 it seems to be fully sponsored (by the Hoosier Oncology Group), so it shouldn't cost OGXI much or change their projection that they have enough cash to last into 2015. (I think that projection assumes they'll launch additional P2 trials for 427 in lung and pancreatic.)
Definitely a nice, unexpected development that should take the focus away from l'affaire Burris. As Dr. K has said, it's the clinical program and its success that will drive OGXI's pps.