Not bad, but not the sort of thing that would make Azixa first choice over Avastin for second-line GBM treatment. One long-term survivor out of 31 patients treated. I think they would have called out hemorrhage from the tumors, so we'll presume none of those. I've said it before: killing a patient tears you up inside, but no hemorrhages probably means under-treatment.
Comparable to Avastin at a third of the cost would be a business proposition, but not a good one. I expect some investigators to approach the company with the intent of sponsoring their own phase 2s.
I don't understand your rationale for assuming Azixa wouldn't be able to compete with Avastin in a 2nd line setting. First, you have to look at this indication and know that even with Avastin, an $8B a yr. drug, the overall survival in GBM has not gotten any better in 30 or so yrs. I'm not familiar with Genentech's trials for GBM, but I read somewhere that there was a case of a complete response, mostly partial responses. There really aren't many options and those that fail treatment on Avastin and Temozolomide will look to any alternative, making 2nd and 3rd line therapies even more valuable. Case in point, Avastin as a 2nd line therapy.
Assuming Azixa makes it to an NDA as a front line therapy, the market potential is at least a billion. I suspect the cases of GBM to grow along with the baby boomer population over the next few decades as they've already hit retirement age.
Celldex is supposedly starting a PIII trial in GBM at the end of this year, but I'm not sure they have had any complete responses. Pfizer backed out of their deal with CLDX because they said it no longer fit in with their strategic future or something like that. Do you really think they would have backed out of the deal if there were several complete responses in the PII trials?
I think people are underestimating Azixa and the potential of the current PIIb trial in first-line therapy using the Raino Criteria. What if there's more than 1 complete response? That would seem to confirm that this drug is crossing the blood brain barrier and actually killing the tumors. Even partial responses from patients that failed on Avastin is pretty remarkable, imo.
So are you saying that maybe, just maybe, it is not worth MYRX spending hundreds of millions of dollars on this drug, because it will never recoup the investment? And perhaps the money could be better spent some other way, perhaps selling the pipeline and liquidating via a special dividend? Just a thought.
What I'm saying is that A business is in the bag, but to develop a WORTHWHILE business, they have to do testing to broaden the eligible patient population. The reported results would probably be enough to take much of the GBM salvage treatment market away from Avastin, but they aren't dramatic enough to get fast-track approval.
One sincerely hopes that there will be an investigator-sponsored phase 2 combining Azixa with Avastin.
You still ought to be in index funds, or closed-ends at the riskiest; not a little drug developer.
Nice activity snapshot on the NAMPT inhibitor. But it doesn't look like that companion diagnostic they talk about will be needed. Once you hit effective concentration, going higher doesn't seem to make much difference.