Mozzarc
Emerged
Being held in San Diego, California. Dr Roberto Giugliani will be presenting a poster session titled "A double-blind placebo-controlled phase 2 study to evaluate the safety and tolerability of pentosan polysulfate sodium in subjects with mucopolysaccharidosis type VI (MPS VI)". I believe we will have our own section where he will be presenting from, complete with the Paradigm Logos in the background.
Dr Giugliano's resume is impressive. He is a MD and PhD and leading Geneticist, he is also our Principal Investigator for the MPS VI program in Brazil. This is the largest study of its kind in the world. Dr Giugliani has published some 450 scientific papers and has worked in a number of different roles in different parts of the world (NSW - Australia, Italy and France are some examples, here is link to his resume(Think it is in Portuguese but you'll get a drift).
Here is just another quality example of the talent we have working for us, and this is in an area that is simply not garnering enough attention. Its been said that the MPS program for us is a company maker in it's own right. Estimated sales in this area could hit $1 Billion per annum for us. I have a feeling that a tie up here could quite conceivably happen before OA. It would indeed be one heck of a stepping stone to the big OA global scene in terms of legitimacy and proving ourselves.
PAR said they would put out an update/announcement further on the conference which could happen in the next few days. Not sure about any more data release but would be nice to get an update on the conference or a snippet on what was presented.
The Lysosomal Market is bigger than just the MPS strain subset, there is further potential here and there is currently NO available longer term, safe, joint pain solution out there. It would indeed be a very complimentary addition to a suite of products that could be offered to a given patient in this space. Some Lysosomal storage diseases you may have come across in addition to MPS include:
- Fabry disease
- Pompe disease
- Gaucher disease
- Lysosomal acid lipase deficiency
NOT JUST PAIN
Sure our main thrust is joint pain relief in this area, but there are other possibilities when we are dealing with the addressing of inflammation.
The above image relates to the neurologic symptoms, this reminds me of the MPS society's forum where there was evidence uncovered that iPPS has the ability to potentially traverse THROUGH the Blood Brain Barrier, something that wasn't thought possible as our molecule is too big (around 5000 Daltons). The researchers amazingly observed a decrease in one of the inflammatory biomarkers that's prevalent in the brain under such conditions (IL-8), and further witnessed some 16% reduction in a study.
Exciting stuff with some amazing future ramifications. Much more to be studied here.PERSPECTIVE
The FDA are acutely aware of the severity of such diseases and gives sponsors like PAR the ability to have their clinical trial fees waived, shorter time frames (Defaulted Fast Track under the Orphan designation) and a specialised consulting team in terms of communications during trials. PGIC becomes a much more important factor and the Caring Guardians/Family and specialised Doctor's view are taken into consideration to try and improve the overall quality of life.
PAR will make a difference here and we should be proud as shareholders to be involved in this effort. We, are after, all owners of the company.
PLAYERS
There are a number of key players in the ERT area some names include:
- Shire Plc; Sanofi S.A.
- Biomarin Pharmaceutical Inc.
- AbbVie
- Alexion Pharmaceuticals Inc.
- Allergan plc
- Horizon Pharma Public Limited Company
- Actelion (Janssen)
- Recordati Rare Diseases
- Protalix Biotherapeutics
- Amicus Therapeutics, Inc.
- Actelion,
- Pfizer
- Digestive Care
- Leadiant Biosciences
Who knows, some of these guys above might just be talking to the PAR folks right now? I reckon that last MPS Poster would be an exciting one for them to read. (See Appendix below)
MARKET
In 2020 the ERT market was some $10.6 Billion and with a CAGR of some 14% between 2021 and 2027, its going to equate into some big biccies. Of course we will only be able to attract a proportion of that, but lucrative it will be for us all the same.
I reckon its exciting for us when we have a number of other indications that we are working on, but a deal here could be as nice surprising stepping stone and possibly propel us onto a much larger stage as our data continues to roll in from both MPS I and MPS VI programs...
Watch this space!
- Mozz
FURTHER READING
Interested in more info on ERT?
See this reference:
An Overview of Enzyme Replacement Therapy for Lysosomal Storage Diseases
APPENDIX
Here is the link for the ICIEM POSTER, well worth a scan if you have not seen this:
https://paradigmbiopharma.com/wp-content/uploads/2021/11/PARA_MPSI_001-ICIEM-2021-poster2.pdf
REFERENCES
1]https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6128977/
2]https://www.grandviewresearch.com/industry-analysis/enzyme-replacement-therapy-market
3]https://www.marketwatch.com/press-r...ominant-sectors-and-countries-data-2021-12-07
4]https://pedclerk.bsd.uchicago.edu/page/degenerative-cns-disease-lysosomal-storage-disorders
5]https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3383490/
