JT, I agree with you here, about the sweet spot...
TOO EARLY and we risk undercutting ourselves...selling out wayyy too early, not realising much value, or as much value...
TOO LATE and we wait and wait and we possibly go lower and need a more dilutive cash raise?
Its a mix between the two....BUT there are things that can strike that can help shift this sweet spot...
Not saying we can rely heavily on these milestones...but at the same time we need to factor some chance of these happening in the future.
What helps us then?
Data data data
Yes I know we have had some really good read outs so far...some have surprised even the most pessimistic of us...
We need more though...why? Because our n was small.,..but it was very illuminating...it was pivotal..and it was actually unexpected.
No one thought we would get Statistical Significance with such a small number...no one expected us to get such incredible structural observations in a crazy short time...this wasn't supposed to happen...those BMLS CAN regress on their own...but not in such a short time on average....this is another Mozz post on this topic alone, one day...
I know a fair chunk of you will say Mozz, we now the sci works...we want the COMMERCIAL side to work...
Yes I agree, this is not only important but its vital...we need to get this right...I believe we do have the skills in place, I believe PAR do have guidance in place...yes they will take their time to get it right...they will use their consultants and those all important World leading KOLs to guide them. Don't forget each of those KOL's in our list have worked v closely with the authorities.
I just came cross another paper by T Schnitzer...they are really SME. (Subject Matter Experts)
Now just because we have taken x years to get HERE, doesn't necessarily mean it will take another same x years to progress from here...
Just take a look at what is coming on the back of our results so far....
MPS Program...This is the best example I can give you guys.
IMAGINE we didn't have a chance of not having to do a P3....(Let me rephrase)..Imagine we had to do a P3 in Brazil as well:
We will get the MPS VI read out by the end of the year Then we need a couple of months to package it and to sit with our consultants and work out the way forward, design the next Phase Then we go to the Authorities and get their seal of approval....Ethics as well... Then we go out and start recruiting and screening and finally enrolment... Where are we u to by now? Prob 3rd Q next year if it all works steadily...call it end of the year then.
Then we run the trial for a full year..last patient dosed when? Mid 2025? Prob a little later, but let me be aggressive... Thats mid 2026 trial done...yay a P3 out of the way...2 months for analysis and compilation.... Another 1 month for read out... 2 months for Marketing and labelling and partnering (yeah ok lets make that 4 months)...and then we start selling...
THIS IS THE PATHWAY if we need a P3...it's LONG!
But PAR are telling us, there is a CHANCE that we don't need a P3 in all regions and one of the regions identified by Paul is Brazil, the simple fact here is that in terms of Lysosomal diseases, this is one of the most densely populated cohorts for these indications and also in terms of absolute numbers. This is the perfect place to start.
It's populated...quite densely populated. Brazil's overall population is 216 million! 1
Getting a 'Provisional' GO here will cut all that time. This is potential progress. How many people understand this? It's easy to say we are delayed...and I agree, maybe we are here and there, but the programs are progressing...perhaps not at a pace we are all comfortable with...but it is getting closer.
That's my point. This program starting could actually trigger some events that a lot of us aren't even aware of us.
Some examples:
1) Validation
Others will finally know we are real....the drug works, others have done their DD and an authority has accepted us....we also have an early GO (potentially)
2) Cross Over
Yes there is some pathologies that are common to MPS and OA....Think Biomarkers, pain...muscleklosketal ramifications . I can see at least some other parties being very happy that they have at least some sort of proving ground, some sort of test they can apply and observe with MPS that can have this positive cross over into OA.
3) Buying pressure
Yeah we need some of this and soon....we need our SP to get out of this rut and start moving...HOW do we do this? Well through a program like MPS..once those other shareholders and potential shareholders see us moving and bringing forward some of these timetables (see above)...that's going to help...not saying we are going to double o'night...I am saying it will help...maybe not all up front.,.but as we further progress.
4) Value Add
A partner here (MPS) will be looking very closely with what they are about to get....you get an early GO....they are going to MOVE.....for a few reasons...
A) They don't want competition grabbing us first B) They understand that the data is showing a whole heap of points, some include: i - Pain relief ii - Function improvement (ROM) iii - Reduction of GAGS in urine (as PR mentioned)
There will be real merit in first mover advantage...snapping us up early will also not be too expensive for them, it will help us but it will be the start of other triggers in my view.
When new holders see that we are finally presenting data as well as some non data related milestones...things will change...maybe not too soon, maybe still in the background for now...but wait and watch (my views).
(20min delay)