Yeah a bit of a marathon here, I've been building this one up over the last few months...I kinda also wanted to wait for the 6 month data result before posting this marathon....its been a lot of fun and while I have tried to cover it from a number of angles, there is a lot of material here if you dig deep. A lot of the past AA filings have predominantly been in the Oncology space, but you know what? That makes it even more exciting and lucrative for us! Wouldn't it be great to crack open this field of OA with our super drug and blow away some of the longer timetables that most holders out there (outside of HC of course) expect?
Wouldn't it be lovely to have first time revenue much closer than we all think? Of course its mere speculation as to when this might play out...but as I have said, IF that data keeps coming, the safety profile is intact AND we are indeed as consistent and as statistically material as we have been with increased numbers to come....surely an AA ratchets up on the probability scale?
The landscape for an AA is more complex than we know...the new act passed in 2016 called the 21st Century Cures Act has a lot in it. How about this for a statement:
"But under the new Act, newly approved drugs may never have to produce a randomized study to establish long-term safety. Instead, real world evidence like insurance claims might be deemed sufficient to allow the drug to stay on the market". 13Another future change may involve the entire placebo arm:
"And so even if the FDA might still prefer the classic randomized trial against placebo, there are other considerations besides their time and cost. For example, the FDA is trying to reconcile with the fact that many trial participants do not want to potentially receive a placebo. So one key new initiative is to ensure that future trials are designed with input from the affected patient community, which the FDA hopes can help avoid high trial dropout rates and reduce trial costs". 13
Victory? Well actually Virigina...
I present to you ...Dr Virginia Kraus,...forget that she is a world renowned OA expert with some 40 years of pure research under her belt, I have one better....ready for this new peoples to us?
SHE WORKS FOR US!Yes that's true, she is a consultant. PAR have not only employed some talent, they have employed some of the world's BEST. I'll go you even one better...a commoner......but a trusty holder that we all know - has met her.
Yes yours truly. Greatest moment of my recent life?
You bet!
As a quick recap, here are her amazing credentials:
While researching for this AA I came across some good slides from a presentation by Dr Kraus and a Dr M Karsdal.
14
Don't forget, Dr Virginia Kraus et al. also wrote an entire paper on Accelerated and what sponsors need to incorporate. The rationale of the Accelerated program, that reference is 15 in the reference list at the end of this part 4 post.
Single left click to enlarge image aboveThe thing about the above table from the paper above that I like, is the fact that at the time, they wouldn't have had much idea about iPPS and certainly very scant of any details on
DMOAD aspects., they assumed some future drug would always have something lacking, ie it would demonstrate "statistical difference in structure (less worsening compared to placebo)" but it would fail to show symptomatic benefits.
In fact, each and every "Description of Profile" column above has some compensation for the drug lacking in some area...
HELLLLLOOOOOOOOOOOOOO - our data shows efficacy in BOTH AREAS...structurally AND symptomatically. I'm literally crying tears of joy when I read PAR's announcement ...Par said in their 008 6 month read out...they got structural marked benefits BUT they also get crazy a88sed pain reliefas well as function improvement.
This ONE drug (of ours) is doing literally both. Structural and symptomatic. Guys, I'm no darned soothsayer...but I see the queues of patients lining up one day (Spec statements).
OH It's easy for us in the heat of the moment to forget about all that is happening under the covers. I do clearly remember at the time mid way through the 008 program we got word that it has been markedly delayed. There were many posters all concerned about the status of the 008 program...
Perhaps there was some problem
Perhaps the data was so poor that PAR decided to pull it?
Blast PR...he has delayed us again!?
Yes we didn't get word from PAR for quite some time, they were busy re-organising this entire program and changed it in three distinct ways.
1) Extra 6 months tacked on to the end
2) Second Site
3) Third dosing arm added
This one study has made a PIVITOAL difference to our distinct chances of AA...in my view.
It has shown us connotations of what exactly is happening in the joint form a modification point of view. There are a few that argued that WE WOULD NOT SEE ANY STRUCUTRAL CHANGES SO EARLY INTO THIS STUDY.
There are others that said that PAR would NOT GET STATSTICAL SIGNIFICANCE ON SUCH LOW NUMBERS
Fellow holders, today we have this data and it is in fact pivotal as well as WORLD FIRST..
Just imagine those upcoming Authority discussions....now THAT will be amazing. The smile on PAR's faces when they simply hand over the dossier. Cant express to you what that means to them...what that means to YOU ...what that means to ME.It is this data that will get us into the room with the authorities to potentially begin a route down a pathway that may lead us to AA one day.
Its always fun to dream and we should consider the positives, its one of the main reasons we all invest. Yes we all have different timing's and goals. If we manage to file for AA and we are successful, this will not only finally move the needle, it will keep it there...the sheer scope of our markets and the wonder of our drug will become acutely clear at this juncture. Why would the market start to value us at different levels on filing? A number of reasons but the two biggest:
1) Imminent sales. The AA allows sponsors to go to market. Yes we wouldn't be covered by insurance, but there will still be a number of patients that are in line for surgery on the wait lists that want to try
any alternative before going under the knife. There are also a heap in acute and chronic pain...they will also want to try a new drug that has a great safety profile and actually has a chance of working!
2) AA will be the ultimate validation. If we don't have a Big Pharma OA USA/European partner by this point, it won't be long before we would. I can clearly see a frenetic silent auction occurring within a few weeks of this news. Add a sense of DMOAD and it will be the spraying of a thin vapour of a misty fuel that will result in a positive explosion of a share price, My opinions and not advice.
It will be a very different company IF they file for AA and IF they are granted this designation.
Look, I see great data, I see safe data, I also see sheer consistent data. I hear what patients are saying, I look at the odds, how many % are reaping benefit...I see that even for a good swipe of the population so far (SAS for instance) even the lower stratums of pain relief there is still some efficacy..some good percentages of people...AND for those that it unfortunately DOESN'T work, there are no serious AE's...why wouldn't you at least test it out?
Now add ALL the other potential benefits...multiple joints simultaneously, not intrusive....lasting duration and the chance of other amazing symptomatic and possible disease alternation -think blood pressure, maybe sugar levels, heart conditions...this is something incredible that could actually strike one day.
There is good evidence for companies that make it to a successful AA, the rewards are there...immediate share price ramifications, expediated time to market and of course the increased chance of a meaningful Global Distribution Deal. This is the one of a handful milestones that will actually result in a sustained and material lift in one's share price, specially when we are at a very reduced base level. My thoughts.
A lottery ticket? Lottery tickets are full of random chance...the consistent and empirical data we have seen so far over the years that could very well be formulating for a possible Accelerated Application is not random.
The potential here is incredible.
My views
- Mozz
.MAIN REFERENCE
1]
https://www.fda.gov/media/86377/downloadOTHER REFERENCES USED
1.5] https://jamanetwork.com/journals/jamanetworkopen/fullarticle/2798005#:~:text=Importance%20The%20US%20Food%20and,Fast%20Track%2C%20and%20Priority%20Review.
2]
https://reader.elsevier.com/reader/sd/pii/S1063458423000201?token=D6A85053314394EC0A7DB4E99881EC1438D21FDD96C050CE6E628DCB55173A23B6D84A24B326AC169439BEA0B581D3F7&originRegion=us-east-1&originCreation=202305180019113] https://www.sydneyknee.com.au/knee-info/knee-anatomy/#:~:text=The%20main%20weightbearing%20part%20of,side%20is%20lateral%20tibiofemoral%20compartment.
4]
https://medlineplus.gov/ency/imagepages/8844.htm5]
https://app.sharelinktechnologies.com/announcement/asx/63a249bdb0b4e5e1dc93c8ee2644f3a26]
https://app.sharelinktechnologies.com/announcement/asx/32c48c1b6e394b137d0897c29353e00c7]
https://paradigmbiopharma.com/wp-content/uploads/2023/03/PAR_OARSI_Theatre_Symposium_Mar_2023.pdf7.5]
https://www.pfizer.com/news/articles/speeding_up_the_drug_approval_process_and_what_that_means_for_patients8]
https://paradigmbiopharma.com/wp-content/uploads/2023/03/Paradigm-NWR-Presentation-Mar-2023.pdf9]
https://app.sharelinktechnologies.com/announcement/asx/1801a407c6bff2b2200d2ab1a15c725910]
https://app.sharelinktechnologies.com/announcement/asx/fa31694b6b5919adc469e18a683a3c8311]
https://www.biopharmadive.com/news/jpm-2023-califf-fda-startups-funding-cliff-editas/640107/12]
https://www.pfizer.com/news/articles/speeding_up_the_drug_approval_process_and_what_that_means_for_patients13]
https://www.optum.com/business/insights/pharmacy-care-services/page.hub.drug-approval-process.html14]
https://www.slideshare.net/OARSI/understanding-the-accelerated-pathway15]
https://www.oarsijournal.com/action/showPdf?pii=S1063-4584%2818%2931527-916]
https://everylifefoundation.org/accelerated-approval/
(20min delay)
