“Additionally we've generated data from the expanded access program, EAP 275 that followed 241 children over more than 10 years, who -- where Remestemcel was used as salvage therapy. And in that program, the existing potency assay targeting T cells demonstrating how Remestemcel targets T cell activation has shown that changes in manufacturing, which resulted in enhanced bio activity of the product could be predicted by the assay as could improvements in outcomes, notably survival.
So these new data will all be part of the BLA that's submitted, resubmitted to the FDA. And we expect to completethat by the end of this quarter”
@col69
My take, for what its worth, is that Mesoblast had a subtle ambiguity in their last official earnings release, compared to the wording used in Silviu’s results commentary (as taken from the transcript of the last conference call).. Firstly, they guided that the final data sets from the EAP required for inclusion in the submission (which had been delayed by procurement issues from the sourcing of testing kits) would be available right at the end of the quarter and then implied that they could immediately incorporate all that data without so much a weeks delay in time for submission. I think they will most likely file a few weeks late…but in taking full legal advantage of forward guidance rules, they might only seek to reassure us, when the quarter has actually ended.
The irony is that the Company has never been so close to triggering a whole host of transformative milestones and yet some shareholders are now so beaten up, some might throw the towel in , rather than consider waiting for possibly a few more weeks. Whilst i think many delays were not of the company’s making they have been slow at revising timescales…something that has personally cost me a small fortune in longer dated options. In their defence, how were they to know that their Singapore manufacturing faculty would be Covid quarantined until the Spring ? or that in March they would be able to secure the services of the remarkable Dr Philip Krause, whose invaluable counsel, has unquestionably resulted in a much strengthened BLA submission document… but no doubt at a cost of further months being added to the process. Can you imagine how stupid Mesoblast would have looked if they did not give Krause, one of the FDA’s former top experts, the opportunity to review the data and come back with amendments and an appropriate filing strategy ?
No folks , I am going to have to suck it up.
Regarding the heart submission . I believe the Company will continue to deploy the same old tactics of past years. Wait until a major cardiology conference, then surprise surprise, a peer reviewed paper suddenly appears just before the conference starts …so that a buzz can greet “breaking news” sharing of new ad hoc data. Many Pharma companies do the same thing, as it is a sensible marketing strategy to reach the target professional audience on a global stage and still take a bow for some incredible work . For over two years the Company has not released proper weaning data from the ischemic sub set of the LVAD trial, but they have referred to a positive outcome, which was doubtless obscured from reaching statistical significance by problems using the faulty LVADS chosen by the NIH, which have now been withdrawn from the market. The efficacy of the LVAD trial results must have been further diluted by having to include data from including the larger non ischemic cohort.
Mesoblast, must realise by now , that if it is to convince the FDA that Rexlemestrocel is worthy of an accelerated approval designation, they must first show a continuum of clinical response data in the varies stages of the ischemic/disease disease from late stage 2 to end stage LVAD heart failure. If they can do the latter I think everyone is going to be very happy . OP.
Please do not rely on the facts or opinions expressed in the above post when making an investment decision.
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“BLA resubmission for remestemcel-L in children with SR-aGVHD expected to be filed this quarter, with potential US approval Q1 CY2023”
(20min delay)