PAR & MPS

Paradigmers, after a rather quiet week and perhaps a few more quieter days to come, tonight let me present to you MPS.

I’m sure most of you are by now aware of this condition but let me give you some of my thoughts and research to add value and analysis to what you may already know.The more I research this amazing company the more intriguing and compelling it becomes. I understand we need to temper our excitement, I also know that we must be patient as these things will take time. I also understand that there will be unforeseen hurdles that may crop up along our journey. But Paradigmers, do we know the full extent of what we own? Read below and enjoy this unfolding journey.


PAR, a magical opening of a bright future?


Definition of MPS

MPS a rare condition that affects many parts of the body. A lack of a key enzyme results in large sugar molecules called Glycosaminglycans - GAG's that then accumulate in certain cells called Lysomes. This then results in the Lysomes increasing in size causing tissues and organs to enlarge in size¹. There are 7 different types of MPS and some 40 different type of Lysosomal Storage Diseases²


Symtoms?

Unfortunately for patients, there are many:

Enlarged head, lips, cheeks, tongue, and nose
Enlarged vocal cords, resulting in a deep voice
Frequent upper respiratory infections
Sleep apnea
Hydrocephalus
Hepatosplenomegaly (enlarged liver and spleen)
Umbilical herniaInguinal hernia
Hearing loss
Recurrent ear infections
Corneal clouding
Carpal tunnel syndrome
Narrowing of the spinal canal (spinal stenosis)
Heart valve abnormalities, which can lead to heart failure
Short stature
Joint deformities (contractures)
Dysostosis multiplex (generalized thickening of most long bones, particularly the ribs)
Developmental delays and regression


Treatment

There are two treatment options for MPS I, HSCT (Hematopoietic stem cell treatment) and ERT (enzyme replacement treatment).
Timing is important and its better to start this off in patients that are under the age of 2.


Numbers and potential revenue

Before coming across PAR that one fine day back in 2015..I knew nothing about Orphan diseases. I in no way knew how lucrative they could be. How can an unfortunate condition be rare and yet amount to an amazing amount of revenue (and of course just as importantly, health benefits) as some of the other bigger blockbuster drugs? Paradigmers, I give you just one statement that blew me a way:


“MPS has the potential to be just as big for paradigm as BME /KOA”³

Imagine a condition, such as OA that prob affects a few hundred million patients, is actually comparable in revenues to something that affects just 1 or 2 births per 100,000?Being conservative, the numbers in my opinion gives us something like this:

With some 65,000 patients in the US, lets say 60% of them have access to hospitals and care and then 40% of these can afford the treatment or have access to insurance that will cover it. That's revenue at a conservative $50 K per patient equating to some $780 Million. There are other markets such a UK, Europe and Aus/NZ that could push this to $1 + Billion.Now don’t forget that a company such as CUV which we have covered in the past (https://hotcopper.com.au/threads/par-v-cuv.4747202/)
has a target market of only 10,000 patients and they're still waiting the result of the phase three trial in the US (due October this year) yet already has a SP of $32 and in the year 2018 revenue of just 25 Million AUD ⁴...don't forget we could possibly achieve that sort of revenue in our first year of OA and MPS sales!

Dont believe me that our PPS could ever sell for so much ($50K)? Take a look at the following Cut-out from Express Scripts<sup>5.


The 70K figure above is PER MONTH!</sup>

More testing in the MPS realm through a clinical trial needs to be done but initial results show potentially three big benefits:
1-inhibit gag synthesis
2-enhance gag degradation
3-directly affect lysome storage function³

In addition, a decrease in urine concentrations was evident with the tests done so far and combining with ERT (see cut out below for definition of ERT) may provide additional benefits such as pain relief and the fact that it could be applied to all 7 strains of this nasty disease.

Definition of ERT ⁶

In regards to an Orphan designation, It’s also potentially extra rewarding for us as there is a chance “to catch the entire value chain”³, we may not have to share this revenue sandpit with anyone.


A sandpit in amongst green pastures that we may not have to share with anyone else (ie Big Phrama partner)

Why is the ramp up in potential revenue from MPS so much quicker than the potential ramp up compare to osteoarthritis?
Answer- most patients live close to hospitals due to the acute and constant treatments required. Word will spread quickly once this treatment is released.As it is an orphan drug only one trial (combined Phase 2/3) should be required and there is a good chance it will be fast tracked.Once this process starts it will be quick. Perhaps the trial itself may take some time but once approved just watch the fun.

My fellow Paradigmers, these are my views, please take that into consideration. Hey, Im excited but I also understand we have a ways to go. Do your own research, dont rely on anything I've said, I'm not qualified. But remember the potential here, in my opinion is great. Lets stick with this one!


References
1 https://rarediseases.info.nih.gov/diseases/10335/mucopolysaccharidosis-type-i
2 https://www.gaucherdisease.org/about-gaucher-disease/what-is/lysosomal-storage-disorders/
3 Lodge Partners 23 Jan 2019
4 https://www.globenewswire.com/news-release/2018/08/29/1558116/0/en/CLINUVEL-delivers-second-positive-financial-result.html
5 https://www.google.com/urlsa=i&source=images&cd=&cad=rja&uact=8&ved=0ahUKEwjz07675ObjAhVZX30KHarHAZ4QMwhBKAAwAA&url=https%3A%2F%2Fkhn.org%2Fnews%2Fdrugmakers-manipulate-orphan-drug-rules-to-create-prized-monopolies%2F&psig=AOvVaw1htsJfmGRrM0anO6ipMY5K&ust=1564924573447038&ictx=3&uact=3
6 https://livertox.nih.gov/EnzymeReplacementTherapy.htm