@Armyne I believe you are absolutely correct. The OTAT meeting represents the “logjam”. Once the rules of the game are formally established at that meeting re “in vitro potency” assays we should be back on track…this point was also made in the latest Bell Potter note. . After a year of total failure by the regulatory authorities to approve a desperately needed therapy for paediatric sr GVHD , Mesoblast has had to remain “Schtum”. Napoleon Bauer et al , are clearly way out of their depth. Their comments about cytoskeletons of MSCs after passage 7 or finding ridiculous examples of less efficacious therapies for use as comparators against Remestemcel for the ODAC meeting, exposed them. I believe they have spent the last 12 months getting comfortable with how they want to regulate the regenerative medicine industry. The FDA was tasked with finding ways of accelerating approval processes for promising therapies for orphan indications. They have clearly failed…and Mesoblast look as though they will be rescued by a dispute resolution process instead. What is the point of having a “rolling BLA” ,where each applicant is supposed to register individual modules early for submission after extensive guidance from the FDA, when the FDA take the opportunity to then
ambush the applicant at the subsequent ODAC hearing. Their insistence on a randomised controlled trial for children where there was no proper standard of care for patients and mortality rates were up to 90% was abhorrent to Kurtzberg as it was to many on the ODAC Committee . Takeda are no doubt in the same position, trying to get Alofisel approved in the US, even though it gained European Authorisation back in Dec 2017 ! Put quite simply, the FDA is reasonably relaxed about autologous therapies because no one is likely to sue them for approving them…but are still running scared of approving far more promising allogenic donor options. This is despite the fact that people who often require the most help, have compromised immune systems, so taking their own defective cells and replicating them outside the body as practised by autologous donors, is often doomed to failure (as Emerson Perin has pointed out from past clinical trials). I am not sure that outside of a few blood products there has been any successful BLA applications for an allogenic therapy to date.
https://www.ema.europa.eu/en/news/meeting-highlights-committee-medicinal-products-human-use-chmp-11-14-december-2017As for our wonderful contributor
@The Yankee. , I feel your pain personally .both financially and equally from frustration with management in failing to explain the timelines involved. Shareholders have been served up rose tinted and sometimes incomplete information as the Company realises that any implied criticism of the regulator for delaying proceedings , might hurt their approval chances. For a long time now I have advocated capital markets day presentations, semi annually, to update shareholders properly ….as opposed to the stale format of 30 minute post results meetings which rarely have time to debate matters of substance or scratch the surface on the technological advances . Mesoblast has to compete for capital and potential shareholders need to be able to do proper due diligence on the Company. Silviu lurched from one mini funding crisis to another over the years ,as the cash burn continues to attract the shorting fraternity. It appears pretty obvious that being a public company at moments like this leaves shareholders exposed to considerable market manipulation…which makes your point about good communication even more important. Despite
AMAZING clinical data in CLBP, ARDS and CHF, which promises major breakthroughs ahead and an imminent OTAT meeting which should finally see the FDA getting off the fence, the market cap of the Company is a disgrace. Just based on the CHF results from the Revascor trial we deserve to have a market cap of at least US $3-5 bn in my opinion. The Revascor RCT, over 5 years, tracking over 500 patients, reducing heart attacks and strokes by an unprecedented 60% tells me all I need to know. Furthermore the opinion of the ODAC Committee matters. So does the IP/due diligence reviews of Messrs JCR, Tasly, NovaQuest , Grunenthal and Novartis. Two of the latter companies were collectively prepared to commit billions in milestone payments, part of which will be paid before a single cent is sold…. so long as we can show a satisfactory regulatory pathway for each indication. The NIH has enough respect for the potential of our therapy as first in class that they have financed trials for both Covid ARDS and LVAD therapies…and I would not be surprised if they offer further assistance in the near future.
The intial tests by the Joanne Kurtzberg , on the first few sr Chronic GVHD Patients resulted in three patients (2 children and 1 adult) showing clinically significant results and were in remission within weeks or our cells being administered. The scale of this achievement will doubtless be reflected when results are revealed in the larger cohort receiving Remestemcel . Some patients are still on Ruxolitinib for the same indication THREE YEARS later. Similarly, something special is clearly going on at the Cleveland Clinic with regards to our steroid refractory Chrohns Colitis therapy where we are seeking to put patients into remission within 4 weeks and avoid the need for elective surgery.
Silviu is a Doctor by training. He may not the complete deal as the CEO but no one ever is. I have huge respect for his capabilities. I know he works incredibly long hours and is just as frustrated as us by the hold ups. Mesoblast is paying the regulatory price for failing to meet its primary endpoints in subsequent phase 3 clinical trials…but NO THERAPY has ever got close in any major indication to matching our potential. It’s not as if ANY of our competitors has managed to achieve more over the last 20 years. talk of removing Silviu at this crucial stage is total madness. He needs support from all of us right now ….and in return we deserve to have our hands held …because loyalty works both ways. It saddens me when I see a tremendous supporter like The Yankee lashing out in frustration. All i can say is that the best investment opportunities often occur at moments like this. As for the NEJM article, it is
obvious from the Texas Heart Institute video that one will be published relatively soon …so everyone keep calm and carry on . I increased my holdings today. Within six to twelve months I expect to be handsomely rewarded.. . But I sure wish Novartis would confirm our joint venture…because I want those shorters to feel real pain ……sadly it will not be as excruciating, as the poor kids that we are trying to help with GVHD . OP
Please do not rely on any facts or opinions in the above post when making an investment decision .
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