December 2, 2011

Trial Design: Primary Endpoint

Dr. Jen noted a modified PFS, instead of time to progression, could potentially be the primary endpoint since it is appropriate for demonstrating treatment efficacy.

Remember, from the previous blog post, I believe Dr. Jen's equity research note essentially telegraphs the trial design for the MM RCT Phase 3 SPA trial.

Recall the PFS results by disease stage from the MM Phase 2 trial:

DTIC: 1.6 months (BRIM 3 trial), 2.6 months (ipi trial)
PLX4032-a: 6.7 months (BRIM2 trial; 60% of subjects were Stage IV M1c)
PLX4032-b: 5.3 months (BRIM3 trial; 65% were Stage IV M1c, with 5% Stage III, and most of the
remainder being Stage IV M1b)
ipi+DTIC: 2.8 months (BRIM3 trial; 65% were Stage IV M1c and 25% Stage IV M1b, with only 3% Stage III)

Same questions:
  • Do you see PV-10's PFS above for the target population of the Phase 2 trial?
  • What would you hazard the likely PFS to be for the Phase 3 trial?
  • Do you think the study will be stopped early due to the clear superiority of the PV-10 arm?
  • How will the likely dramatic difference in PFS influence valuation?
  • What is the likelihood of clinical and regulatory milestones payments being made?
Again, the hallmark of modern science is reproducibility. I know what my answers are to these questions. What are yours?

Note: All lesions would be treated for Provectus' Phase 3 study. In the Phase 2 trial, 1-10 target lesions and up to 1-10 non-target lesions were treated. How would that affect the eventual PFS?

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