“Because of pain I'm hardly focused on anything. I can't pay attention.”
MPS Patient1
urrently with so much human suffering at the moment, its topical we cover a subset of our market that we will one day cater for. In fact, it may just be the very first cab off the proverbial rank when it comes to getting registration and that final Golden ticket to sales at scale...
Tonight we look at MPS and what patients want?
MPS?
Most of us know what this is, but a few new to us may be wondering about this and may be much more familiar with our grand daddy/mummy of indications, OA. Do not discount the wonderful action of iPPS on MPS and what a leading result this might turn out to be before the main show of OA.
MPS is a rare lysosomal storage disease which results in inherited mutations. These mutations lead to the progression of accumulation of partially degraded glycosaminoglycans in organs and tissues. This causes cell damage and this in turn manifests into many symptoms that are diverse as they are debilitating.
Disfiguring, swelling, teeth deformations, stunted growth, pain and lack of flexion are just some of those symptoms and manifestations.
IN THE BACKGROUND
Now Paradigmers, its very easy to feel nothing is happening but delays. Its very easy for some of us to give up all hope when we see a total disconnect in our share price and immediately come to the conclusion that our company is terrible and is being run by people that could do better. I'm not just blindly defending them. Please do not feel that I turn a blind ear and a deaf eye...(err... you know what I mean)....but at the very same time I have to state the facts.
This company is doing stuff that YOU and I BOTH do not realise in the quieter times. They are building a cathedral, its just that we are at the foundational stage so all YOU see is a BIG bl**dy pit and you think we are going to be buried in it.
A large pit, hurry up...when will our building rise?
In that pit goes MASSIVE foundations...what do I mean?
This entire post is structure around a study of patients....MPS patients, and what their needs are, it was a study conducted to get a sense of what the patients and their respective carers actually wanted, what are the problems they face...this study was sponsored by PAR. Its peer reviewed.
Yes those two work for us.
HOW CAN WE ASSIST, WHATS OUR SCOPE?
Yeah but Mozz, Isn't this a rare disease, rare means not very often, not many people. Leave aside the good moral-compass type addressing, how is this even worth pursuing ?
Ahh dear reader, don't just think its only the BIG indications (Cancer, Diabetes, Cholesterol etc) that can make money. Yes regarding OA we might be able to charge in the order of $2500 USD per patient and multiples of this if DMOAD is proven a number of years later...BUT the charge for MPS is totally different, we may well be up around the $50 K mark plus, per patient and you know what, that's per annum!. There are an estimated 13,000 patients in the USA with a lysosomal disease and let me tell you (and Morgan's) that the uptake one day is going to be more than 10% penetration (My views).
The MPS set is a close knit community, once a few patients in this field get whiff that there is a:
NEW
SAFE
EFFECTIVE
...solution for joint pain relief and a number of other attributes that we will soon cover, its going to spread fast.
Let me say this, once we eventually hit even 10% market penetration in this MPS domain, our share price IS going to be somewhat north of $1.20.
WHAT DO THEY WANT?
To be honest, there isn't a lot of research out there as to what paediatric patients in MPS VI want addressed as a priority and how they go about their lives and the impact of this terrible condition.There is one study I came across which forms the basis of this post.
Back in 2019, some 9 patients along with their 9 caregivers were individually interview over the course of a day, all were between 4 and 18 years of age. Now all of these guys were on ERT however, residual symptoms exist and they do impact on their quality of life.
What were the key findings?Well look at the most bothersome/challenging functional aspects:
MOST BOTHERSOME/CHALLENGING:
Mobility (33%) Independence in daily activities (33%) Sleep (22%) and Pain (11%),
And as a secondary?
The second most bothersome/challenging aspect nominated was predominantly pain (67%).
Paradigmers, read trough those aspects above again...now compare that with what exactly iPPS can do...It addresses mobility, it certainly allows the patient to have a better night's sleep and it reduces pain. Enzyme Replacement Therapy does not do this.
Lets just tackle this one category in isolation before we continue...
LET ME SLEEP
Most patients indicated that pain affected their ability to fall and/or stay asleep at night.
Sleep deprivation does not just lead to a blurry and slow day the next day..it can have a number of longer term repunctuations 2:
How does iPPS help? Where is the proof?
It was THE SINGLE BEST LEADING INDICATOR IN WOMAC TO DATE 3.
I don't see this kinda evidence in the newspapers. I don't see interviews on channel 9 and on the ABC...but this will come one day (well, after P3 I hope, I don't want it to influence Placebo)....Look at the raw evidence...this is the reason WHY I am NOT personally worried about the share price disconnect. I know my patients (yours too, you are owners of the company as well remember)....are sleeping much better, they are healing much better, they are happier, they are healthier, their bodies are getting better. they are in less pain.
How do I know this?
If you are new, read this table in its entirety. Also check out the references below. I ain't making this sh*t up.
An impressive 64.6% drop in pain at night? There are huge positive ramifications both physically and psychologically of being able to sleep better at night, allow the body to heal!
Pain reduction allows for independence in activities, We have heard of patients that simply cannot bend to put on socks, to get out of the car, to pick up things from the floor due to crushing and debilitating OA in joints.And finally, the sheer pain...
"Pain was identified by one caregiver as the most challenging symptom, and by 6 caregivers as the second most challenging symptom. Pain was present in hands, wrists, shoulders, knees, hips, and back. Most caregivers reported that pain impacted their child’s sleep and ability to participate in sports and recreational activities and complete schoolwork."
Mozz, great in theory that we know what THEY want...but what does OUR drug do to address this?
So glad you asked that.
Mate, you don't need to be Einstein here to understand that those above columns are DECREASING, and these charts span not over DAYS...they span WEEKS...in fact 25 weeks...It doesn't take an analyst working for Merrill Lynch to understand that its trending down AND this has NEVER happened before and it has never happened SAFELY....
I knew I was right about Afterpay....you wont believe the feeling I'm getting with PAR. (Spec remark, must DYOR, be aware of risks).
To address these highly wanted categories SAEFLY will mean a world of difference.
Sure we can bring down pain...but it has to be safely...what's the point of having a meaningful reduction in the number 1 or 2 category AND not being safe, causing more harm and destruction, opioids have a limited life....I can see a future where they are drastically reduced...
PROMIS
PROMIS stands for Patient-Reported Outcomes Measurement Information System and has "demonstrated faster administration, lower burden of data capture and reduced floor and ceiling effects compared to traditional Patient Reported Outcomes Measurements." 5
There is a negative strong correlation between PI (Pain interference) and PF (Physical function). "The predictive relationship of function from pain scores supports the PROMIS PI being used as an important adjunct measure of physical function in patients with spinal pain." 6
Above we have the theory, below we have a quote from the carers in this super real world patient study:
“They'll cry, they'll get frustrated that everybody ran from this area of the playground to the other and they couldn't keep up and they're yelling at them to wait for them and it's frustrating for them, but they still do it”.
Well, after iPPS, that run to the playground might just be a few percentage points easier, how did iPPS make a difference in this area of PROMIS for our drug? Lets have a look:
Look at those trends up....its so crystal clear and with just three patients.
I love the consistency of the the drug, from batch to FDA random audited batch...But I REALLY love the consistency of the results even more...and you know what, one day the FDA may just agree.
Speaking of which, the FDA are becoming more aware and sympathetic while striving to make a difference in the lives of children with rare diseases. Once our data set becomes more prolific in statistical meaningful numbers, we need to push to get a better deal...not just with a Big Pharma...but someone that is a good fit and has previous track records with the FDA and EMA.
A matter of months, maybe a year for a deal? The additional benefits of iPPS to an ERT program is coming...one day.
We single handily represent a new force in a field that hasn't had much of a forefront revolution. We need to capitalise on this and sell our wares to the highest but classiest outfit. Our product is not only superior...not only durable...but safe. (Subject to further trial determination).
"Over the past 15 years, we have evolved from a view that we must protect children from research to a view that we must protect children through research."
FDA - Finding a Path Forward Feb 28, 2012.4
We are valuable (You just cant tell that yet from our share price)
As our share price has fallen I admit I have watched it...but I have not acted, quite the contrary, I have added bites to my PAR holding...my determination to uncover more and deeper research is resolute. I find my enthusiasm all the more fired up as we ascribe even more acute and sheer value at these incredulous share price levels.
For things that are going to make a global impact, they take time...think Lithium batteries...think Green Power...its not over months...its over years....a hydrogen fork-lift was unthinkable just 15 years ago....that we could have electrical cars buzzing around that has the acceleration that rivals sports cars was unthinkable just 10 years ago...the meaningful reduction of joint pain isn't too far away...a few more years that's all we need...we are getting there.
Green energy, lithium plays, electric cars and trucks, yes they are coming to fruition, but how long has it taken? Bio Pharma plays take time...specially from scratch, specially ones with Global reach? Patience Grasshopper.
Paradigmers, the evidence is crystal clear, the data is coming ...
What indeed do patients want? A safe effective drug that reduces pain in joints and can have meaningful improvements in other aspects such as pain at night, increased mobility and an improved chance to be able to catch up a bit more to those kids in the park!
A basic source of joy? A once terribly painful experience....
Yes yes just three patients so far in MPS VI, but by year's end? We may have a handful from MPS 1 and possibly as many as 9 or more from MPS VI..the data set is building...our time is surely coming...it might be months, it might be a year...but every week that goes by.....we draw closer to making a real difference,..and becoming a truly valuable global entity.
urrently with so much human suffering at the moment, its topical we cover a subset of our market that we will one day cater for. In fact, it may just be the very first cab off the proverbial rank when it comes to getting registration and that final Golden ticket to sales at scale...
NEW
SAFE
EFFECTIVE
(20min delay)