Replies to post #130008 on Biotech Values

[tinkershaw]

Pain is a valid clinical target for an NDA submission. It may be harder to hit, and impossible to get an SPA for, but that does not mean it can not be a basis for an NDA.

The commercial value of this might on it's own be questionable, but as you say they already could be on the market so the broader indication could be of more value than a stand alone pain NDA.

Correct me if I am wrong, but it is my understanding that if a drug has published efficacy, say here from the 306 trial, and it is already approved for MTC, that insurance may very well cover it, if used off label consistent with the published data.

If so, 306, even if not registrational, can serve a similar purpose (albeit muted from what it would be if atually registered), to get the drug reimbursed for the pain indication as well.

Frankly, given what is already suspected about its survival benefit, it certainly beats just giving a pateint some narcotic pain killer that will do nothing to help ameliorate the disease.

But hey, down 40%, world is ending, stock and drug will of course never recover, so what the heck...306 is a worthless target without an SPA...or is it? If nothing else it does act as the second clinical trial that the FDA so appreciates, and yes, if approved for MTC (which appears very likely), it will give ammunition to obtain reimbursement when used in the pain indication as well as long as the 306 trial procedures and succeeds.

In the end, again, it is the drug...procedure is important, timeline important, but it is the drug that coutns as long as the rest is reasonable and manageable that will determine the share price in the end.

Tinker

[p3analyze]

>>How do you know 306 is not potentially registrational? <<

It's not clear to me whether 306 is sufficient for a standalone NDA. What if 306 were to show benefit in pain and bone mets, but neither 306 nor 307 show a survival advantage?

So I maintain that's most likely registrational as part of an NDA based on a positive 307 trial (which yeilds final result at the same time) to gain a pain response label statement for CRPC with bone mets to differentiate from other drugs in the CRPC arena.

There was a typo in my previous post, I really meant to say without the positive thyroid data which increased confidence in the anti-tumor activities, EXEL would be more reluctant to invest in 306.