1 - Cantor, SI - COVID ARDS should not have any impact on OPEX in short term.... will provide more granuality on that in the short term. CLBP we are in the process of formulating the power for this program, so no plans that this would impact OPEX in short term.
2, Answer timing on GvHD commercialization and lessons learnt from Japan - worked with FDA to understand precisely what FDA want. We understood far better than a year ago the mechanism of action, and how they are more effective in treating inflammation. We are putting that together in a detailed package for resubmission... the resubmission is a process ( including inspection of the manufacturing site ), and that may drive the timeline for approval, with the maximum time being 6 months. We are working for an approval second half this year.
*** That means submission first half.
Si - Think within 3 years penetration would be 40% of addressable market... and they would expand into the adult market as soon as the children is approved...... and then inflammatory bowel disease.
** 40% of Pediatric GvHD is 200+ patients per year by year 3....... approx 50 million per annum in sales
CMO - on IBD No question if the cell therapy is effective, it would be preferred over destructive therapy ( the current therapies available )
CLBP - 2 trials so far, and we had pain reduction across the entire population for both trials. We are looking at 50% pain reduction where the FDA standard is only 30% reduction, over a 12 month duration, function a secondary end point. Based on past trials we think it's achievable.
3. You will submit a BLA , or submit data for checking and then BLA for GvHD in kds? Because we have open dialogue, we are in a position to submit the BLA and then they can check the data. Crohns - The size for a Cronhs phase 3 won't need to be anywhere near the size of the trial the biologics since we are only treating non responders and the patients have no other option. The objective was to see if we have a signal of safety and efficacy, to have a view to how to structure a well controlled study, and we may not need to go to all 48 before seeing enough information required to create the next trail.
4. Remestemcel Lifecycle. Number 1 priority is Pediatric GvHD where there is no other approved treatment... Laser focused. Then we would expand to adults and will discuss this with the FDA as we go for approval for Pediatrics. ARDS is important and will be completed as will Crohns which is a huge opportunity if the signals are there. CLBP Pain reduction of 35 points from baseline...... which is a 50% reduction in pain with CLBP( A large proportion of our patients had complete remission or over 50% of pain reduction.
CHF - in the process of providing the revised analysis as guided by the FDA for CHF to the FDA. MACE was originally the primary end point for the trial, which was changed by our partner at the time to a Frailty model. The FDA has asked us to pick a patient population that would maximize public health benefit, which we think we are drilling down to. It's a very large population that could have significant benefit. .
We were quite staggered by the effect size in the CHF study. When we did the analysis that the FDA asked for, the group with the highest risk was ischemic diabetic. 87% higher chance of MACE than the controls, and cells had the greatest benefit in this same group. Rather than pre-empt the discussions that we are having with the FDA on future trials, we are in the process of a formal submission of our documents, and will update the market shortly.
My take on the Q & A is,
The company is following all and every instruction from the FDA in order to get Pediatric GvHD approved this year - this simply has to happen.
BLA resubmission will happen Q1 or Q2 2022 with approval expected in Q3 or Q4.
Commitment for an adult study will also be made at the approval meeting in Q3 or Q4, and if the past is anything to go by they wanted to do a study vs best available standard of care available for adults.
I think** the COVID ARDS trial will not be announced, until GvHD is approved in Q3 / Q4 because the potency assays need to be accepted first.
Crohns I think they will stop the trial early.... probably at 24 patients, or maybe even if the 6 placebo get treated and then they all go into remission.... well that's a 100 % success rate, fairly certain they know what to do next in the follow on trial.
The Back pain scores, I was not aware that a reduction of 35 points, represented a pain reduction of 50%, but there you go. MSB had pain reduction in all treated patients over both trials they ran, and importantly the majority had over 50 % pain reduction or
complete remission when treated within the first 5- 6 years. The end point of a 50% pain reduction in the next trial should do the trick as it is substantially ( roughly Double ) above and beyond the FDA current requirements.
CHF remains the dark horse here. Si did not want to talk about it at all.... that means Something is brewing..... maybe something Huge as a few of us on here have suspected. They are still in the process of formally submitting documents........ and will advise the market shortly on the outcome................... Did I hear someone think accelerated approval?????
This has to be every shorts' worst nightmare, we know the CHF announcement is coming, we don't know when, it is a game changer for the company regardless of weather they get accelerated approval or not....... but if they do !!!!!. The results were fantastic, best in class, and the FDA knows it.
I would not at all be surprised if the formal submission is of a type for accelerated approval, and MSB are playing their cards close to their chest this time.
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