Hmm some good questions there, lets tackle them, though remember none of this is advice, this is only my thoughts. Timetable? Well, they fluctuate, we know about that right? It's all a guide...things could move out, there could be hurdles, these twists and turns can actually play out. I certainly can't pretend to even know a small % of what might actually eventuate, so DYOR works here.
Northeasttrader
It's has been said that despite MPS being a niche and rare disease, the figures still can stack up. "Company maker in its own right" is another saying I've heard.
MPS has a number of subdivisions, around 7 main strains and more subdivisions under those...there are some 40 lysosomal diseases that have been identified....the main ones we are currently tackling are MPS I and MPS VI.
Now this condition is rare...I've heard 3.5 births per 100,000 or so for MPS and about 1 in 7,700 for the lysosomal. The estimates that I have also read in the past are for around 11,000 to 13,000 potential patients in the US. Each country has certainly varying numbers, some more 'hotspots' than others and one such example is Brazil. No surprise that we have chosen that as our main clinical trial recruitment country for MPS VI (Phase 2).
Umm so Mozz...$2000 x lets say we treat 50% of them....= $2000 x 50% x 11,000 in USA = $11 million USD ??
While thats great mate, its not going to put us in the Aussie Company Hall of Fame.
True, and those numbers are wrong....its not $2000 that we may charge, its more like $50,000....
How can we charge so much? Because it is intense, it is weekly, there are many costs involved upfront and the administration has to be carefully monitored needless to say a myriad of other conditions, the dosing must be tailor made and would possibly involve a drip not to mention how it is to be administered along with ERT. Add to that the efficacy of the drug and what it can do, think multi actionary. A reminder here that some current ERT program treatments cost close to $750,000 per annum...thats not a typo.
So multiply it out and all of a sudden we are in the $300 Million per annum space and that could just be for the USA...add a few more countries and you start to see why it has been estimated that our addressable market in revenue could be close to the blockbuster $1 Billion....yes thats per annum.
Timeframes? Well this is a bit harder to work out, better to go conservative in light of all we have experienced so far.
So at the moment we have our MPS I trial in Adelaide, we have recruited 3 patients, we should get a few more over the months, this is a year long trial for each patient...so Im guessing we will be at least another full 1.5/2 years away before we have a read out? At the same time we are about to kick off Brazil at some stage (MPS VI). The good news with MPS VI trial is that it is half the time, 24 weeks. We are looking for about 10 patients here so I'm thinking by the time we recruit, dose and compile the data, its prob a good year/year and a half away from now, subject to Covid and any other hurdles. This will basically end up with the two trial data readouts coming out by lets say mid to end 2023? We also have to remember that these are both Orphan designations so in theory the FDA should be quite quick in terms of communication and the like?
Can you imagine how complex some of their Gantt charts would get? All we can do is guess at a very high level....
Now before you think, man that is so far away....don forget that there is a chance of a partnership being struck before the final P2 readout? Sure the chances prob. increase after the readout...but lets see. After that is P3....again because we are an Orphan designation, there could be a faster pathway if the data is stellar and in my humble opinion it's quite probable that a partner in this area will help us to get a P3 ready in terms of process work and funding etc.
HRDWRK
Good question, the data seems like it is supportive of us going it alone, and of course we need the larger numbers of a P2 in a couple of the MPS strains to add to this. Data coming from rats and dogs is a great start, you really need the efficiencies shown at some scale in human trials to really go to the market...
So despite some potential here, it still makes sense for us to partner up with an ERT specialist. ERT is a good std. of care and would be fairly complementary to us at least with what data and evidence we have to date. No doubt we are a small company, we certainly are in no way positioned to be able to go out globally in any distributional sense on our own. It would take years to establish such networks and acquire such skills...think warehousing, logistics, marketing...sales....no way.
So from that point of view it does make a whole heap of sense to get a big brother/sister that already has the infrastructure set up, they already have the MPS Docs on their books. Don't forget its an unwritten code that the Docs that treat MPS patients have a different level of care for their patients. This relationship has been established and fostered and plays a big role in the care of the patient. I think it makes a lot of sense to partner up and offer a suite of care to the MPS patient with someone that already has the knowledge, experience and relationships in place. This is a lifelong deal here....a perpetual requirement from the patient's point of view. As I have posted before, there are a few ERT providers out there, once they know of us and what we can do...it will be nice to negotiate a good deal.
Hope that helps
Mozz
PS: Robbie1 - Hearty rich soup? Good analogy...good way of eating those natural veggies we all need too...
(20min delay)