Paradigmers, a special report tonight...yes the interview with the friend of a friend who has worked closely with the FDA. This interview resulted in more material than I originally anticipated thus I will break it up into two parts.
THE INTERVIEW - PART 1
INTRODUCTION
When someone tells me they have a hot tip for a company that is relatively new...relatively small...I'm always a little cautious....the odds of small companies actually making it ain't great. In fact on my path to finding PAR I was given some ten tips over 6 months by a broker .
8 of them went backwards, 1 was quite good and resulted in a 3X and I have now long since sold this. The last one out of the ten recommendations I sit here writing to you about today, PAR, and has already covered the losses on all of the 8 losers...
So what helps me to figure out if this stock is the one? If this stock has better potential than others? Well research for a start. Yes of course there is stuff on the internet, there is the glossy prospectus and annual reports...but the real insight, the real research is done in the quiet of your study...it's done by observation and sheer brute force of simple thinking and gathering evidence. Out of all the books...out of all the articles, you get just a handful that give you a clue over the years. There are very few advisors that will give you true unbiased opinions and knowledge...so how does one make a compelling case? What filters do we use?
In my mind there are two broad categories...FA and TA....Fundamental Analysis is how the company is doing in terms of revenue, profits, debt...and their future growth profile...do they have a good product? Technical Analysis are the charts, what has happened, what is happening and where does it look like it is going? Some investors disregard this TA field totally but I have found that it can and does add weight at times....Both of these fields are large and would need a series of posts to even give a high overview of some of the ins and outs.
Ok so back to the story and my point. My point is that I want evidence that is NOT easy to get....I want to dig into things that most people can't be stuffed and is just too tedious to do. I want to go back to older articles and studies and find out just how compelling what we have actually is! It's that little study conducted way back in 2010 that hardly has 125 views on their page...it is the little conversation I just had at the back of the room with a director at last year's AGM. I can read his statement in a mag...but when I see how he looks at me with his eyes and he speaks of his own experience and he his genuine...this is the clue I want. I do not do this for all companies, I only do it for ones that have the potential and where I have a vested interest for who can give up so much bandwidth in the pursuit of greatness? There are many opportunities out there, we are selecting the best...it is the icing on the donuts that I am after...I don't want just one filter...I'm not just decanting the wine once.....multiple times through multiple grades of filters. This means sourcing info from different areas...
So tonight we cover off one really different source, someone that works closely with the FDA...think of this as getting insight from across a divide rather than always just relying on what we get fed using our own common pastures.
PRE MEETING THOUGHTS
I thought that this friend of a friend we would be chatting to would have some knowledge and would be a good source to ask some general trial and FDA related questions. This contact works for a Clinical Technology company. Typically companies like our PAR would engage such companies as this to assist in putting their trials together in a prescribed format and assisting with data gathering and to some extent trial design. There are many such companies and they all have their specialty, some have a lot of Inflammatory experience, others could be strong in the Oncology segment, some might be localised to the Aussie industry etc.
I only found out just a few minutes before the interview that he has had a wealth of experience. This guy works for a company that had involvement in some 400 trials and that's just in the last 12 months! Throughout the interview he quoted me drugs and trial experiences off the top of his head that he has worked with in the past all from memory and on the fly and he gave us examples at exactly the right time like quotes in a book. He was indeed a wealth of practical knowledge with not just a few years of experience but some 17 years.......I thought I'd be happy to get 20 mins of his time, he gave us three sessions for a total of about 1 hr and 30 mins all up....bliss.
You've heard of 60 minutes? Let's start a new channel 90 minutes! Full of awesome detail about our Company and where it might go next?!
NOTE
Some of the original order of these questions has changed between my first post in this thread. Some extra questions were also discussed as below and in Part 2. See also additional notes in Part 2 for some unplanned discussions that also took place.To keep him anonymous, though there wasn't anything terribly sensitive that he mentioned, but as this entire forum here is anonymous let's stick with this theme, we will call him Mr X.
THE INTERVIEW
Ok let's tackle the set questions first and then I can add some thoughts and some of his comments at the end. Please enjoy, I certainly did, it was quite an insight.
QUESTION 1 Our trial size for Phase 3 OA is 2 trials (750 plus 400 for confirmatory trial), total of 1150, is this low in respect of the fact of addressable market of OA being so great?
No, In X's opinion the numbers weren't necessarily too small. He did mention how the costs can easily blow out and the FDA would be aware that this is not a large International Pharmaceutical that is conducting the study and so they also want to be at least somewhat accommodating by not making the numbers unnecessarily onerous but still having a suitable data set to obtain meaningful results from. The fact that it is an OA pain trial it has some similarities to a neuroscience indications (that's another of his expertise area) and X said that our sample size equates pretty much to how many would be in a typical neuroscience related trial such as Schizophrenia or Alzheimer's for example. He also thought 1150 was not bad. (ie not a bad size).
QUESTION 2 What is the typical/average time between Pre-IND and IND meetings? (in terms of normal pathway and also in the case of an Orphan labelled drug).
X seemed to think about a year is not out of the realms as substantial data must be submitted to the FDA IND. [Mozz's thoughts -In light of this the fact that we had the Pre Ind back in April and we might have the IND for OA in December (possibly Jan) this is a good outcome for us].
QUESTION 3 In order to declare a trial 'open', what are some of the criteria? Ie if a trial typically would go for 18 months, what would be some of the indicators/reasons to stop the trial early in order to get the drug out quicker? Is this a higher chance of occurring if the drug delivers very good efficacy without any safety concerns/issues in the case of an Orphan type indication/trial?
Endpoints are determined prior to the trial starting, If there are to be any intermediate analysis points and any stages during the trial if statistical analysis will occur these are usually pre determined if applicable, and seem to be more common in the case of rare diseases. The Chief investigator will have some criteria to measure against which boils down to stats. If there are x number of patients and the results are better than a benchmark of y then this could trigger an earlier halt to the trial.
In the case of an Orphan (Rare) pathway, it is different to a normal (regular) pathway in that if a regular pathway is shown to be spectacular in terms of efficacy, benefit and safety profile and the underlying disease is deemed to be of a critical nature and is 'serious' there is more impetus to stop the trial earlier as there is a moral obligation to the patients that have the same disease but are on placebo. This is generally not the case in an Orphan pathway as there is usually no Placebo, so if a drug is working and giving good results, well there is less incentive to stop the trial early! In this case there is a way to register patients and keep them going on the drug at the sponsor's cost and the trial is stopped early and approval may be given. The patients on the trial can report back in case any subsequent AE's show up but the drug is effectively approved earlier than anticipated. The FDA in this case it takes the view that it is also good to keep the patients on the drug under an Orphan type designation as the drugs are usually very expensive and provides the patient with good results.
X stated it succinctly by saying that the pressures to stop a trial in 'Rare' is not the same as the 'Regular' pathway.
QUESTION 4 Apart for Orphan designation for MPS (which in itself is a fast track type designation), we don’t have any fast track or priority or other designation, if eligible, can this be awarded more typically at IND or after IND meeting?
Basically X said you need to have a breakthrough therapy/type result or you are addressing some huge un-met need. It needs to be addressing a serious medical condition. It could still could occur especially after some data comes back after the trial commences, he suggested you shouldn't rule it out but the fact that the FDA have requested 2 Phase 3 trials for OA, at this stage, the Fast Track designation would prob not occur in his view. at least in the short term.
In PART 2 of the interview we will cover other questions such as the relationship between the FDA and EMA, a surprise as far as the amount of communication that goes on between FDA and the sponsor during a typical trial and much more of an insight on the FDA and the trial itself!
PAR Price at posting:
99.0¢ Sentiment: Buy Disclosure: Held
(20min delay)