Hi all, wanted to chime in here and breakdown the probability of the success of positive phase 2 trial results for acne. Due Q3 CY2019 (approx. September).
I'd like to start off by saying, these are my thoughts on the process and hence why I am invested in the company. I have a background in the field, but by no means should what I say to be taken as gospel. It merely is a set of somewhat logical thoughts on the matter at hand.
SOME MAJOR RISKS ASSOCIATED WITH PRELIM-TO-PHASE 2 DATA REPRODUCTION: Let me begin by flagging the issue with MOST biotech companies transitioning from preliminary phase1a/1b results into successful phase 2 results. It is in most cases it is the models of choice. Scientists, researchers, practitioners open themselves up to huge amounts of genetic variability and physiological differences when transitioning between the likes of animal models and human models. Reading through recent competitive ASX listed companies you find a huge discrepancy in preliminary models, most selecting for mice models due to their ease of breeding and genetic manipulation (also cost). In cases of testing neurological based drugs for example, this presents huge physiological and genetic barriers when looking into phase 2 trial development with > ~95% of drugs, in this case, will fail.
It is flagged that only ~ 39% of drugs will make it phase 3 trials, compared to ~ 67% which make it to phase 2.
Another major issue is the disease pathology. Without going into the science (never-ending), I have shown below the % breakdown of phase success rates based on pathology. Being a dermatological disorder, acne, I would put it under the category "other". It is, however, comorbidity with some endocrine disorders.
ref: Michael Hay et.al., 2014. Clinical development success rates forinvestigational drugs. Nature biotechnology ....
WHY IS $BOT DIFFERENT?
As flagged, it is quite well known that the phase 2 success rate is a hurdle for most bio/pharma companies. The major key-point that can be taken out of the announcements so far is that Matt & Vince have derisked the company moving into phase 2 results by already conducting trials on
human patients. This already alleviates the pressure of transitioning between animal to human models. This is a huge derisk for the company IMO.
This has been done for psoriasis, where they have highlighted in human patients that BTX 1308 targets p38 MAP Kinase pathways and IL-6 indicated in psoriasis and down-regulated their processes unique to psoriasis patients, leading to reduced skin cell growth and inflammation.
Additionally, we are targetting a topical superficial area of the body. You aren't dealing with potential issues regarding uptake efficacy, blood-brain-barrier perfusion etc. And once again, they've addressed this in their ann regarding the efficiency of Permetex in targeting the derma layers.
HOW DOES THIS DERISK ACNE PHASE 2 RESULTS?
What we can take out of this (aside for the photos showing an improvement in efficacy) is that BTX reduces inflammation in human patients for psoriasis (shown above). It also (as we have found out) has anti-microbial effects. What hasn't been shown yet is its ability to inhibit keratinocyte proliferation, however, it is a fair extrapolation based on previous data.
PREDICTED OUTCOME OF PHASE 2 ANCE RESULTS? Now knowing that it decreases inflammatory effects, looking at the endpoints for their phase 2 data,
you'd expect to see a significant % change in INFLAMMATORY lesions from baseline after 12 weeks varying with dosages/topical application. Given the success rate of phase 2 results, and that it is higher in dermatological disorders by ~ 100%, you can see an increase in success rate off the pathological differences alone.
The major key point here is the overlap between dermatological disorders, and the human trials already conducted.
Post-phase2 trials the chances of success are greater which is really why most are holding this. It further de-risks the company.
But to recap my initial statement: most go wrong but not testing the drug preliminary in humans. We have had the privlidge of doing so.
Now don't get me started on the management .... the costs they've spent on R&D .... their scientific board etc etc.
Best of luck all.
Cheers,
Michael (
@zabba198).
(20min delay)