AF - Those models seem happy and delighted
without being PAR shareholders? At least I'm assuming they aren't yet share owners? Might need to convince them over a bevvie or three? Your shout or mine?
Pool_Governor, PAR party sounds good! But which Phase three are you talking about? OA OR MPS?
MPS will be a lot sooner than OA now ....
Explanation, I think I need to clarify this one as even I was a little confused:
There are 3 points here....(taken from the last PAR presentation slides)
1) Mid 2020 we are submitting to both agencies the MPS trial application. This process is due to start any day now with preliminary phone chats etc. Official submission will hopefully be on time (mid this year)
2) Late 2020 we actually commence the combined Phase 2/3 for the Orphan condition of MPS. Now this is extra good for us in my mind...why? Because effectively we have to wait longer for OA now (read out at this stage is pushed back to Q3 2022, ages away)...there is only
little chance it will be much earlier than this as we have discussed as the FDA want to see the durability over 12 months...so there is little to no chance of cutting at least these 12 months time down, get me? So why is this extra good? Because MPS is the next cab off the rank...rather than having to wait even longer, with nothing in between, at least there should be some revenue before the big OA read out...this of course is coupled with the hopeful timing of the TGA process and subsequent first real revenue...next year should be a very good year for us.
3) Q3 CY 2020 Par to commence Phase 2 trial, this was the confusing bit (see below) and my understanding is that this is actually a dedicated and focused trial to tackle specifically MPS 1. This is a new thing in my mind... I was not aware of the fact that they would effectively split MPS in this fashion (Note, there are "Seven distinct clinical types [of MPS]" and there are "numerous subtypes of the mucopolysaccharidoses have been identified"
1Par-community...this is great news for us..multiple trials to really sort out what we do...what we have...what we are capable of? I'm all for it...as I have mentioned, the Phase 2/3 trial is a combined trial..it's for an Orphan disease and thus we are effectively skipping a step...ie combining Phase two and three together. The trial size numbers will be much lower (instead of 1100 or so like OA , it might only be 50?) and the other good thing is that our drug takes an average of 4 or so weeks to start working in terms of decreasing pain levels...that's a very quick turnaround specially when you consider that this could be the primary endpoint for the MPS study along with reduction of GAGS? Although this might be true it's important to note that the first Phase 1 trial was conducted in two tandem 12 week batches...ie 24 weeks altogether, I have no idea if the more comprehensive 2/3 trial will require something similar (just with greater numbers [4 patients in trial 1, maybe round 50 in trial 2/3?]). There is always a small chance the trial could be deemed open sooner..at any rate even if it is 24 weeks..that could mean some sort of read out by mid to end next year?
As I said, bring next year on.
DYOR - speculative time tables being mentioned here, who knows the future...it's still a "buy" in my kind. My views.
References
1)
https://www.ninds.nih.gov/Disorders/Patient-Caregiver-Education/Fact-Sheets/Mucopolysaccharidoses-Fact-Sheet2)
https://www.ncbi.nlm.nih.gov/pubmed/27590017