I was disappointed to learn today that the FORTIS-M trial, a study of 720 patients with previously treated advanced NSCLC randomized to receive talactoferrin-alpha (TLF) or placebo, was reported as being negative in a press release late yesterday.   The primary endpoint was overall survival, and not only was the study quite negative for any improvement in survival (median 7.5 months with talactoferrin vs. 7.7 months on placebo arm), it also demonstrated no improvement in progression-free survival.

We haven't received any additional details, and perhaps there will be a subset identified for further study, but officials from the company indicated that they're likely to be shutting down research on it and cutting their losses.  The stock for Agennix, the manufacturers of TLF, dropped nearly 80% over the course of the last day, so this may well shut down the company.

Of course, I'm disappointed on several levels.  Not only was I truly very hopeful about this agent, partly because of its efficacy in phase II trials and partly because of its extremely favorable tolerability (it actually seems to reduce side effects when paired with chemo, compared with chemo alone).  Although I'd hope to see results of a trial of chemo + TLF vs. chemo + placebo, it seems that's unlikely to happen.

What went wrong?  The best answer is that I don't know, and I don't think anyone does.  It may be that this is just one of too many agents that looked very promising in early work, but once the larger trials more carefully tested it and held it up to higher scrutiny, it didn't live up to that promise.  I must admit that TLF got a fair test in the form of a 720 patient randomized trial that appears to not show any hint of benefit (as far as we know from a press release; the full data should be presented at a meeting in the next couple of months).  

I'm quite saddened that an agent I was pretty hopeful about fail to emerge as a new effective treatment for lung cancer, but there are others out there.