Avid Bioservices Inc (CDMO)

[bimerkjaere]

What statistical significance means in Phase II trials. One needs to put some perspective on the rhetoric by all the 'so-called' analysts that without a s.s. p-value any trial is a failure. In reality, Phase II trials are not powered to prove a 5% chance of random failure (p<0.05), but instead are designed to give the developer a good signal that the therapy has the potential for success and has no significant safety concerns.

Peregrine's Phase II 2nd-NSCLC has proven exactly that. The study shows a p-value <0.217 on a 60% improvement in OS from 11.7mo to 7.3mo for control. To put this in perspective, I will quote authors Ratain and Sergent's article, "Optimising the design of phase II oncology trials: The importance of randomisation" that appeared in the European Journal of Oncology in 2009:

"We and others (Rubinstein et al.) propose that a one-sided test of the null hypothesis that the true primary outcome is no different between treatment and control with a false-positive rate of 0.20 (type I error) is appropriate. In theory, if every phase III
trials were required to be justified by a randomised phase II trial with p < 0.20, this would lead to a phase III success rate of 80%, a significant improvement from the status quo."

Although we are not quite at the p<0.2, the point is that there is enough here to warrant further investigation and we should see a major pharma challenging this in Ph III once FDA signs off on the request.

Yes the trial has discpreancies, but randomization was still maintained despite the incorrect drug given, which should strengthen the results. Additionally, a strong 1st-line NSCLC number of 40% or more increase in survival would further validate bavi's use in this indication, and potential partners will take notice.

IMO, major pharma needs new drugs, especially high priced, lucrative oncology drugs. Pipelines are unusually bare in Ph III across multiple major pharmas that have an oncology platform, and one of these companies will take a chance on bavi.

Linked to article: http://old.ecco-org.eu/binarydata.aspx?type=doc/6-Optimising-the-design-of-phase-II-oncology-trials-The-importance-of-randomisation.pdf