I would
so like to spend a good hour and answer this properly and thoroughly.
It's an hour I don't think I have, dag nab it.
All you need to do is read every announcement in the last 4 years...it'll take some time but how about some highlights to make it easier for you and any others that are also wondering that:
1) We got harmonised!!!!
What?
No no, less this:
...and more this:
2) We got a great chart and table of the additional SAS patient's data in early 2020, here is a quick snapshot:
Yes that is a decrease of some 64.6 incredible percentage points for "sleep at night"
We also got my fodder to tease Anchor Man with 91% deriving some benefit (He says it's 83%, we know who is right...though he has been right quite a lot of times too!)....
3) Later in 2021 we got RRV and for the first time we heard about CTX-II. It wasn't for a few years after that, that I would understand what a reduction of CTX II actually means and stumble across Dr V Kraus and the Biomarker Consortium coming up with CTX II as one of THE most prognostic of all OA biomarkers. This is so us.
It was also in that same announcement we saw this line:
"At Day 81, 61.5% of participants in the iPPS group showed near remission in comparison to 14.3% in the placebo group".'Near remission' is a good one.
4) Some say PAR aren't working hard enough and things are too delayed and taking too long. 30,000 pages is what it took to formulate the IND, this occurred in April 2026. We learnt a lot from the Pre IND. It was also here that the FDA said that we could only use the bene product on the DMF. No other.
5) MPS VI study was kicked off in 2021.
6) 2021 also saw a surprise of another indication out of the blue for the vigilant holders amongst us.
Read the two bullet points below, SS everywhere.
- PPS treatment demonstrated statistically significant reductions in the levels of
complement 3a, a marker of tissue damage in the lung, and in serum levels of
inflammatory biomarkers IL-6, INF-gamma, IL-12p70 and CCL2 in influenza infected
mice compared to vehicle treated controls in the acute phase of infection.
- PPS at the post-acute phase of infection (21-days post infection) demonstrated a
statistically significant reduction in pulmonary fibrosis compared to vehicle treated
controls based on histological staining of collagen.
7) Point 7 is a big one, the kicking off and the revision of 008. The greatest study that was never powered to show SS in the multiple areas it did....
This was the study that covered it ALL...Pain, function...but Wet Chemical Biomarkers too....and as if that wasn't enough...STRUCTURAL ramifications?
Did someone also say Rescue Medication? No wonder they changed it from 6 to 12 months....as a direct result of this study...well we are set up for the next few years and potentially for life.
(Spec statement of course)
8) The end of Nov 2021 PAR revealed a study they had done that was 'blinded' in terms of possible pricing, with some 50% estimated patient use and even higher if DM was on the label, this was a clue for us of the potential, if we didn't already know it.
9) Positive interim results from the ongoing MPS I program were also released in 2021:
10) The big year of 2021 concluded with another surprise...another indication. Half the world's heart failures
cannot be treated by ANY drug that's out there, HELLO iPPS. Read that second bullet point below:
Who wants me to keep going? That's just up to end 2021.
(20min delay)
