BOT 1503 Study Analysis

Analysis of BTX1503 Phase 2 Randomised Controlled Trial

Firstly this is a high-quality multi-centre RCT which had the primary endpoint of measuring reduction in inflammatory acne lesions in 12-50 year olds who had moderate-to-severe acne. There were several secondary endpoints being measured which were of lesser importance but could still yield interesting findings. Here is the summary from the US Trial Registry:


At a basic level, the study failed its primary endpoint.

There was no statistically significant difference found between the treatment group and the placebo (vehicle) for the primary endpoint of inflammatory acne reduction.

When we look at the secondary endpoints (which are not the primary purpose of this trial but to guide us for future trial design) we can see that BTX1503 may show some signs of opportunity in selective cases (such as non-inflammatory acne), however, this is not of primary concern at this point of time.

Interestingly, when we delve into a sub-group analysis, it was noted that the performance of the US vs Australian placebo were quite different. Once again, this is a point of interest and may guide some future study design but has no bearing on the interpretation of the results from this study. It does not invalidate or negate the primary outcome of this study.

Firstly congratulations to the dermatologists and researchers involved in this study for completion of such a high-quality, multicentre trial and commiserations to the Botanix team on the failure of their investigational product BTX1503.

I think its time to go back to the chemistry bench and redesign/ redose the product and return for a future drug discovery attempt. I think its close and hopefully with a small number of tweaks, the product can be re-engineered to be successful next time.