Innovation Pharmaceuticals Inc (IPIX)

[TOB]

Preliminary signs of efficacy do not exist so far as I know. Unless we mean for mice -TIAB

We've heard of preliminary signs of efficacy in the ongoing human Phase 1 clinical trial.

Stable disease. Depending on the cancer type, stage, duration of SD and how it was progressing prior to starting Kevetrin. Note that Progression Free Survival (PFS) is emerging as an FDA endpoint.

Near Complete Response (CR) for an Ovarian Carcinoma Spleen Metastatic lesion.

Reduction in associated peritoneal fluid (ascites).

Increased levels of P21, a secondary outcome measure suggesting activated P53 and predicted by the pre-clinical studies.

These would be more correctly termed data points or case studies. Once the trial is complete, preliminary signs of efficacy would be a more appropriate term. I agree with that. I am making the assumption that what's been reported to date is accurate.

We have not been shown any dose response curves from p21 and that would be only biomarker data, and we do not even have that. -TIAB

I'd expect that at the end of the trial, as an outcome measure. Unless data to date is released prior to that. Preliminary indications are that this biomarker is showing the expected response, dose dependent as predicted by the pre-clinical studies. (Not yet published, but I'm willing to trust what the company has said so far)

One patient with some response but increase in CA125 is suggestive but not enough to count without additional info on additional patients -TIAB

If it appears in the bottom line data, it certainly will qualify as a preliminary indication of efficacy. "Suggestive" and "preliminary indication" I would consider the same concept. Those are exactly the sort of indications we will be looking for, a Complete Response for an Ovarian Cancer spleen metastatic lesion is especially compelling. Keeping in mind of course;

1. At this point these are data points.

2. The small sample size will make all but the most compelling data "preliminary", awaiting confirmation with larger sample sizes in later stage trials.

3. The limited number of patients of each cancer type, and the even more limited number at the higher dosage levels.

There could be a 30% complete response rate for a certain type of cancer proven in later stage trials across a larger population group. But if only one or a few patients of that cancer type are dosed at the therapeutic level, it is hit or miss in the current trial.* Especially if the therapeutic dose requires multiples doses per week. So any preliminary indications/suggestions of efficacy at this early stage are compelling.

* Say a 30% response. As you well know, that doesn't mean 1 out of 3 patients will respond in a sample size of 3. It may take 100 to 1,000 patients to determine the accurate response rate. In any random, small group there could be zero responses. So a few positive data points across the various cancer types and variable dosages will indeed be preliminary indications of efficacy, or suggestive of efficacy if you prefer that term.