Biotech Values

[DaveAu]

The results seen in the ph ii you are referring to (42% PR) are absolutely nothing to write home about since they appear to be entirely consistent with the PR expected for treatment with Pac alone in platin resistant head and neck. See here for instance:

The company is claiming the patients were platinum “refractory” rather than platinum “resistant” and claiming that the refractory patients have a much shorter expected survival.

Even if, against all indications, the ph iii were stat sig it would be only one trial. Very unlikely to result in approval because the supporting trials are not very supporting.

They do have a SPA. It may depend on how strong the results are.

A ton of trials that can be predicted in advance to neither confirm nor deny efficacy - and no meaningful trials until 8 years after ph i. This likely requires concious planning

The initial trial demonstrated safety of a single treatment by intratumoral injection. When this produced only mediocre results, the company went back to the clinic.

Later trials were needed to demonstrate safety of intravenous administration, longer term usage, and usage in combination with radiation and various chemo drugs. The investigators and the regulatory agencies were very cautious on this product initially - understandable since it’s a high dose live infectious agent being administered to immune compromised patients.

It was also necessary to work on manufacturing issues as a lot more product was needed for larger trials using intravenous administration than could be provided using the initial laboratory techniques.

In this light, the 8 years it took is understandable but I’ll grant that the CEO should have been more open about those early challenges.

The poster for the ph ii actually makes note of the fact that the PR patients lived statistically longer than the Progressive Disease patients. Well, duh?! (Mentioning of stupid statistics always makes me suspicious.) Not clear whether this was intentional or just idiocy - but either way it is not a promising indicator.

The poster was produced by independent investigators so I doubt it was part of a stock fraud. I believe the point was that time to progression could be used as a proxy for overall survival and this is how the registration trial is designed – with an initial look at time to progression (overall survival is the primary end point, not time to progression) in the first 80 patients in order to power the trial.