Replies to post #130020 on Biotech Values

[jq1234]

given the FDA response, why did EXEL choose to focus first on the 306 study instead of putting their energy and $ into the larger, more expensive survival study that they knew they would have to do anyway? Resources are now tight for EXEL, and to run even 307 to completion is likely to need more money which would be painful at the current share price.

That's a reasonable question/suggestion.

The cynical answer is: Smaller biotechs are more conscious about catalysts, thus more willing to run smaller trials to fill the void. 307 trial is going to take awhile to start, mid 2012, the trial hasn't been fully designed yet, waiting on more data.

Personally I don't mind EXEL start 306 trial with current design now. The trial size is small, 246 patients - if it were 500 patients, I would have different opinion - it is not much bigger than one cohort of their RDT trial. Given cabo's unique data to date, it is not a bad idea IMO to run a RANDOMIZED trial this size to explore the relationship among pain, bone scan, survival. Their existing data while compelling are post hoc, and the relationship among pain/bone scan/survival is extrapolation of post hoc data. I still don't know how to interpret the bone scan data. 306 trial will answer many if not most questions. If it meets all endpoints spectacularly, including survival (80% powered for 50% increase in survival), take the data to FDA for approval. IMO FDA would be much more willing to approve with very compelling data than giving SPA endorsement for something they don't have enough data to endorse/interpret. If it just meets endpoint, they'll have to wait to file along with 307 if 307 result is positive. XL184 will get label including bone pain relief. During the wait, if XL184 is approved in MTC, it might get some off label use. All in all, I don't see it is a bad idea to run this 246 patients exploratory registration trial. EXEL shouldn't have made this high risk 306 trial SPA as all or nothing for them.

[NP1986]

For me the interesting question that I would like to see explained more to my satisfaction is, given the FDA response, why did EXEL choose to focus first on the 306 study instead of putting their energy and $ into the larger, more expensive survival study that they knew they would have to do anyway? Resources are now tight for EXEL, and to run even 307 to completion is likely to need more money which would be painful at the current share price.

I agree, I think EXEL would be better off just focusing resources on a trial with an OS endpoint. I've mentioned before that I don't think the FDA will approve cabozantinib unless they see OS data, and I think a rejection of the SPA for '306 affirms that view.