Today Silviu has had to slightly reset his timelines for commercialisation. It has become a bi annual event . Whilst this is a little frustrating ....your vitriol should be saved for the FDA. There is currently no treatment in the market to treat children under the age of 12 with acute SR GVHD.
The FDA is more concerned about totally uniform batch consistency and reproducibility rather than the 70-90% mortality rates for a treatment for an unmet need ,which their review team has accepted, has no safety concerns. To quote Philip Hoffman, Chairman of the ODAC committee (page 194 of the transcripts) i voted yes (to Remestemcel) because i found “the clinical evidence compelling”. Many of the ODAC committee pointed out the horrendous toxicity profiles of competing treatments that showed no real efficacy. The FDA has 309 case studies of children showing Mesoblast can deliver a consistent product. They are happy for Mesoblast to supply these cells for free under an EAP...so why not give accelerated approval ?. The Japanese use of Temcell continues to make the FDA look like Neanderthals when “fast tracking” therapies for orphan indications. Fast tracking my ass ! Just like the FDA cocked up in approving dexamethasone for Covid ARDS... until the UK showed meta data which proved the US trial data obsolete, they will continue to drag feet and prevaricate. After all, they cant afford to let their original review committee lose face ! Let’s try to kick this approval down the road for a few more months so as not to embarrass themselves. Anyone following the farce over Biogen’s Alzheimer’s treatment (aducanumab) which has the review committees support despite a negative Advisory Committee vote 0-7 can see how ridiculous the current state of affairs is . Peter Marks please grow some. The whole point of “conditional approvals” is to allow some common sense to prevail. As to our dear
@Pledge , some of your barbed comments I agree with (at least with the sentiment behind them)...but try to cut me some slack. I have caveated my comments on several occasions by saying they only opinion that mattered was the FDA’s. Page 33 of the ODAC transcripts confirms the 2014 meeting with the FDA to agree a trial protocol for our open label 001/2 sr GVHD trial. The review committee later moved the goalposts... that is clear ...not Mesoblast. Some members of the ODAC Committee could not see sense in the FDAs approval of less efficacious and more toxic alternative treatments and said so publicly at the hearing. The delay in approving Ryoncil is truly outrageous in my opinion...but Pledge suggests I was busy “upramping”even though I believe sincerely in the efficacy of the treatment and my support for this Company has personally cost me millions.
Mesoblast can make about 1000 doses from one donor batch pool (which is enough to treat an entire annual patient population with this condition) surely the FDA can undertake representative sampling for potency ? Did they ask Incyte to explain the greater percentage of non responders when treated with Jakafi ? Surely, the mechanism of action was inconsistent and shows statistical inferiority to Ryoncil in all but mild skin cases? The ODAC Committee of specialists agree 9-1 with my view
@Pledge ...so get a life ! I may not have successfully predicted the outcome but i think I have tired to present reasonably cogent and logical explanations for my opinions. My recent view was that the FDA would probably allow a resubmitted BLA application for Ryoncil ...so i have been right on the money with that assessment .
Moving on . Many of you might feel a little disillusioned by headlines ...but i think we are currently tip toeing over delicate egoes to the approval finish line with sr GVHD. Hopefully the potency assays can be agreed over the next few months and the resubmission of our application for Ryoncil will have overcome most obstacles in advance of the formal submission.
Novartis will be making a decision shortly. I think no more than 1-2 months will be sufficient to review the data. I expect the shares to recover substantially if their involvement is confirmed . The Covid ARDS data for under 65’s has been spectacular...why wouldn’t they want to proceed with the j/v ?
Lastly, I would implore Mesoblast to get its IR act together. The almost farcical way it releases earnings with no warning (although I notice that the favoured analysts are all ready by their phones ) is not in step with best practice. I have never before dealt with a Company who would prefer to start an investor presentation without the press release even being available in advance for scrutiny . I felt the FD was evasive and unhelpful today in providing guidance on costs . This has to stop. If there are exceptional items of expenditure provide clarity and stop being so defensive.
A respected poster on this website recently commented that the shares could be worth $20 each if the heart treatment alone is successful.. I would concur ...I believe Mesoblast has shown clinical data which is truly jaw dropping...even if it is another RCT away from having first in class approvals.. Someone opined to me today, that if Mesoblast was a private Company, they thought it might be valued in excess of US $3-4bn . I agree,pre revenue listed companies are often targeted by shorters .... but we are where we are ! By the end of this calendar year our faith in Silviu should be rewarded in several indications. Good luck to all holders.
Please do not rely on the facts or opinions expressed in this post when making an investment decision. OP
(20min delay)