June 26, 2012

Pivotal MM Phase 3 Trial Design: Study Size Dramatically Reduced

Tucked away inside Dr. Agarwala's presentation was a slide on the planned MM Phase 3 trial.

It appears no details of the design of this pivotal trial remain to be hammered out.

I found it very notable that the study size figure dropped dramatically from prior guidance in past presentations. Now180 patients; down from 300. Why the dramatic reduction of almost 40%? The FDA wanted Provectus to have a higher effect size (i.e., a lower hazard ratio), which drives a lower study size. Most studies have much larger patient numbers because their effect sizes are lower (i.e., higher hazard ratios), which means these studies have very incremental improvements. PV-10's improvement is staggeringly large.

While the the physician (and patient) have a choice between DTIC and TMZ, the selection of comparator (or standard of care) is essentially the same, as one is the metabolized form of the other.

The response assessment period of 12 weeks is a time point to start tracking progression and allow crossover to the active PV-10 arm.

Takeaway: The dramatic reduction in the agreed upon study size is a very clear and very meaningful example of the FDA helping Provectus to get PV-10 approved and in the hands of doctors and bloodstreams of patients.

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