@bedgerInvestors may wish to focus on the “finish” line. Mesoblast has a vastly superior new potency assay but in order to get
paediatric approval it needs to complete further validation of
existing inventory by using the
old assay used in the
original trial . I believe they need approx three months to collate assay data from the same inventory source (used during 2015-18comparable period) to ensure potency, consistency and purity,
comparable to the cells used in GVHD001. …just as they proved they were able to do with 241 EAP patients. Once this task is completed, the Company should expect a 30 day turnaround from the FDA (this does not constitute a new BLA submission) to determine if these meets their requirements. Let’s remember that this is a Company that has already passed its manufacturing inspection and now used its cryopreserved cells in over 1300 patients. In my view , there is nothing to suggest this is anything other than a procedural requirement for a comparability study . It is difficult not to suffer conspiracy theories after so many delays and reading CRL’s …because few of us are familiar with FDA procedure…but the pathway to paediatric approval (full or accelerated) could be only 6 months away. My only slight disappointment is that the adult trial protocol could be agreed in the next few months but if we want to use the same old potency assay in adult, we will have to wait 4-5 months for the FDA to sign off on the old assay. If we want the approved assay for adult to be the same one validated for paediatric…the delay of a few months is as very
small price to pay.
In the absence, of further information on working capital , the bears will no doubt try and work the price down in anticipation of a further raise …which is somewhat paradoxical …as we are probably closer to approval than we have ever been. As always , place your bets… In my opinion , the involvement of Philip Krause overseeing the paediatric submission combined with the implied tacit support of the MAGIC Consortium in agreeing to run the adult trial, clearly establishes the credibility of the Mesoblasts approach.
Even if shareholders had to suffer a 30% dilution to ensure the Company was adequately funded , post a successful fund raise ,the shares will be extremely attractive ….how on earth can the FDA refuse a treatment refractory , third line therapy population in adults for sr aGVHD. It is an unmet need with approx 20-30% mortality rates. I think we might double survival rates, so we may only need a low cost trial involving 70-80 patients to adequately power the results.
There is also
nothing to stop the back pain trial commencing other than moola..CLBP has a different potency assay ,so assuming additional funding,our largest indication should be able to commence a 12 month endpoint pivotal Phase 3. I would like to see the Mesoblast share price movements as that prospective trial concludes …as it will seek just to replicate the successful results in pain achieved in the first Phase 3.
The market capitalisation of the Company is ridiculous..but until it addresses
short term funding requirements , it is unlikely to get much better. If a white knight emerges, people may well kick themselves black and blue that they did not take advantage of the uncertainty . Insurers will pay large amounts, maybe as high as US$700,000 for an individual treatment that works on a refractory population…paediatric and treatment refractory sr aGVHD is most likely a $US 1bn market with a 80-90% gross margin . CLBP is an entirely separate indication which is worth multiples more. Try not to blink …i know it’s difficult to believe right now but
approval for paediatric should be only 5-6 months way . OP
Please do not rely on the facts or opinions contained in the above post when making an investment decision .
(20min delay)