From the 14th/Jun/22 Late Stage Advancements in Heart Failure
Apologies - much is paraphrased as they were speaking too fast for me.
Question:
Where do you see extending life most effectively for your stemcells.
SI:
We are targetting the core mechanism of action that drives heart failure which is inflammation. Dream read out is the largest and the longest stem cell by far. We were amazed at the reduction in Mortality and 3 point mace . Endothelial dysfunction causes the heart attacks and vascular complications. Over a 50% reduction in 3 point MACE in the inflamed patients. The big question is - how do you work within the FDA, and target the patient populations that have the highest risk, and demonstrate the greatest patient benefit.
30% of LVAD patients generically have hospitalization due to GI bleeding. We observed a 65% reduction in GI bleeding. We have 2 very large datasets that are confirmatory for each other in inflammatory reduction, and reduction in 3 point MACE. We have an RAMT, and were asked to come back to the FDA with another dataset, and we will have a discussion with the FDA to talk about both datasets; being complimentary datasets for the reduction of inflammation.
The sorts of reduction big pharma get is 10 – 12% over placebo, and we have over 50%.
The real question now is how to accelerate the approval process. IL6 and CRP being the mediators in both these indications…………. Questioner - We will come back to that question, I hae more on that one.
Later on:
Question - The benefit your saw in your trials were in sub groups, which can cause problems in the medical community. Do you have results from the same sub groups accross the trials, such as in the CLBP trial that back up the results?
Si
" If you know what you're looking for on day 1, there is no need to do any trials"
"We are at the cutting edge. The reason we did the 500 person trial was to discover who the treatment is best for, how the MOW works, and what we have found is that a very simple blodd test, that all doctors will do who are treating CHF, that measures CRP, is able to predict for us patient response. The CRP risk factor, is a well established, well observed risk factor that has been identified in many studies, and is related to poor patient outcomes.
We found, that the worst outcomes were from patients with underlying inflammation, and that the same group had by far the greatest response to our treatment. Not only improvement in 3 point Mace and GI bleeding, but after 12 months we observed improvment in the ejection fractions.
This allows us to potentially use it as a surrogate market for accelerated approval. We allready have RMAT and orphan drug designations.
In fact - in every indication we have looked at, now we can predict treatment response based on patienet biomarkers
You are going to see over time, that kind of precision medicine so that the optimal treatment is given becoming more regular.
We are working very closely with the FDA, and taking their guidance on how to target the appropriate patients for the best treatment effects"
Question
What catalysts do you have coming up over the next 6 - 12 months.
"
Very busy 6 months coming up. Most important is refiling BLA for GVHD targeting for this quarter. Have a rolled out plan for commercialisation with inventory and MSLs. 2nd we expect to commence confirmatory phase 3 trial for CLBP as a 12 month pain reduction, to start by end of the year. CHF we have RMAT designation, and will be meeting with the FDA to talk about the totality about the two trials, and expect to be updating the market shortly about the pathway to approval. Also we are having discussion with Several major partners, and potentially one for back pain as well asCHF.
"
Hmmmmmmmmmmmm Accelerated approval with a phase 4 confirmatory, delivered to high CRP level CHF patients anyone?
I think that is what they are looking for !
Any day now...... the BOMB will drop !
(20min delay)