@Wilba32Celyad IP
https://www.fsma.be/sites/default/files/prospectus/files/2024/REGN-20241502-EN-DOCREG-2024-000026-03.pdf
“Back to square one about the catheter.”
Au contraire Madame
@Wilba32 Au contraire.. love the hair by the way.
As far as I am concerned , Mesoblast has covered its bases. The Dream CHF did indeed use the NOGA cardiac mapping system but shrewdly gained access to superior needle IP, post the Chart 1 trial when Celyad had to jettison research into this area post trial results. As you can see from the needle exhibit it has 7 side holes and a curved tip to reduce the potential risk for perforations during the delivery procedure. There is an argument that reducing the number of injections might improve patients outcomes . The Chart 1 trial certainly provided Mesoblast with some clinical evidence that less injections might mean more in that respect. Chart also was a stark reminder of the superiority of allogenic versus autologous. The mechanism of action for CHF is multi factorial with the resident macrophages playing a major role but the THI must believe that there is merit in delivering the cells directly to the margin of damaged cardiac tissue. I believe Mesoblast had concerns that they could not rely on NOGA CMS or that it could be superseded so having control of all parts of the process was important .
On a separate note . I would suggest that everyone takes a deep breath. I believe the markets in small to mid size companies is totally dysfunctional over recent years , particularly for loss making businesses. There is no reason why this share should be any particular price, at any time of the day or month…but if approved , the explosion of revenues (assuming previously guided pricing points) will I believe shock you all. Lets be honest , despite me correcting Dr McStuffins months ago (who said further trials would be necessary before approval for CHF for end stage CHF with LVAD ) many of you still thought we required another drawn out process. The wording used in Mesoblasts latest webinar stated quite clearly that no further trials will be required. What we will need to complete for heart as opposed to sr aGVHD is a series of pre approval harvesting and processing runs for Rexlemestrocel similar to the exercise for Remestemcel if we want the clinical data for our accelerated approval applications for Rex . I believe this normally takes just under two weeks to undertake the ten ? runs required for comparability. There is obviously some time required to consider the results. When Mesoblast wants to press the button they only have to wait 30 days to convene the Pre BLA meeting with the FDA for end stage heart failure as the have an existing RMAT for this indication
Mesoblast might want to court the FDAs opinion on when would be a suitable time for manufacturing upgrades to xeno free culture mediums, etc., I suspect that discussion might be a lot easier if the first product Ryoncil is already approved . Did anyone notice that in the last slide presentation Mesoblast used had taken out the word “LVAD” and just used the phrase , “end stage heart disease “. I am sure the latter change will be a subject for a deeper discussion with the regulator (so a little early to draw conclusions from semantics) but the shares should in my opinion have trebled when the FDA instructed Mesoblast to prepare its accelerated approval submission .
Like many of you I am frustrated at the time taken to submit this application. We are fortunate to have a formidable regulatory expert in Philip Krause coordinating our resubmission and if Silviu has any sense he has probably deferred to his opinion as to when it will be fit for purpose. Also the new clinical data being generated for the FDA for the resubmission had to be collated and checked by outside consultants, whose own bandwidth to speed up their contractural performance obligations is unknown to us.
So the boring part is more or less coming to an end. I believe there is a good chance this will be categorised as a Class 1 resubmission with a PDUFA date 2 months from submission. If it is designated a Class II the PDUFA will say up to six months in reality i think we are probably talking three to four months.
Paediatric steroid refractory acute GVHD is a small population subset ..but the obvious off label use for Ruxolitinib Refractory is approx two to three times the size . The point most people miss is that pricing on a third line refractory option for an orphan disease should be far more attractive as insurers may wish to exhaust the more affordable option first. Then of course is the market for Chronic GVHD . Now with have the data for long term mortality from GVHD001/2 and the EAP programme we are able to show how Ryoncil / Remestemcel has activity, as a prophylaxis or otherwise complete remission ? from both forms of the disease for the majority of survivors . I believe i read that 80% of CGVHD cases occur in the first three years of a BMT so the data we already have generated should be pretty compelling in this regard. A further clinical trial for CGVHD will be required of course, but prepare to see a whole series of investigator led smaller trials spring up all over the place for many inflammatory illnesses as people try to fast track this incredible therapy. Good luck to all. OP
Please do not rely on the facts or opinions contained in the above post when making an investment decision.
(20min delay)