I suggest we are approximately four weeks away from an Adcom meeting for Ryoncil. It is normally held as a public hearing. Members of the public share invited to view the application materials a few days beforehand, note the FDA COMMENTS and listen to some of the world’s foremost experts opine on the efficacy of Ryonicil live on a webcast. Mesoblast had the presence of mind to circumvent much of the panel due diligence by asking the MAGIC consortium to audit the patient control data against a contemporaneous database for accuracy...this verified the accuracy of the data submitted as part of the BLA application . The absence of virtually any major adverse events in almost 1000 patients which have participated in clinical trials and expanded access programmes means i cannot see any major concerns being aired.
https://www.globenewswire.com/news-release/2020/05/25/2038051/0/en/Clinical-Outcomes-Using-RYONCIL-remestemcel-L-in-Children-and-Adults-With-Severe-Inflammatory-Graft-Versus-Host-Disease-Published-in-Three-Articles-in-Biology-of-Blood-and-Marrow-T.html “In the accompanying editorial, Dr Jacques Galipeau, Professor and Assistant Dean of Medicine at the Stem Cell & Regenerative Medicine Center at the University of Wisconsin–Madison and Chair of the International Society of Cell and Gene Therapy (ISCT) MSC Committee, concluded that after more than a decade of clinical study involving three distinct advanced trials, it appears that remestemcel-L might well have finally met the regulatory requirements for marketing approval in the United States for steroid refractory acute GVHD in children, and it is to be determined whether this industrial MSC product will find utility for adults afflicted by acute GVHD or other indications”
Seriously, even Jacques Galipeau, whose “fresh is best mantra”, (which left him at loggerheads with Silviu’s cryopreservation protocols), has seen the light, and is happy to now seemingly endorse the therapy.
The panel will vote...I believe the decision will be unanimous. It is virtually unheard of for a positive decision of the panel to not be accepted by the FDA.
If the Panel vote as i predict, we can then look forward to the adult label extension opportunity. However, with all the data from Japan, plus the excellent results recorded in the US EAP which included adults, I suspect off label sales for adult sr aGVHD will be very encouraging whilst any 50-70 patient ? confirmatory trial is likely to be undertaken over the next six to twelve months to formally allow adult use. I would remind you that at the AGM Silviu was quoted by
@col69 as referencing a $700m peak sales opportunity for sr acute GVHD . I could foresee sales of of paediatric sr aGVHD in the US and Europe achieving $200m per annum within two years and adult sales adding a further $300m within 2 years as well. In my opinion, Ryonicil’s superiority over Jakafi (Incyte NASDAQ) in grade 3/4 GI,Liver and multi organ segments is pretty clear cut, especially when you look at side effects.
By my estimation that makes Ryoncil worth at least a valuation of US$1.5bn within the next 12-18 months ( 3x sales mulitple)....before I allow a 20% chance of success for chronic GVHD which would add a further $150M of risked value. I acknowledge that data from three compassionate use patients of Dr Joanne Kurtzberg is hardly conclusive, but the chronic results were truly exceptional (curing all 3 patients in less than 28 days ) and we will probably only need a small patient open label trial of 50-60 patients to get a label extension for this market segment.
If you are minded to agree with the above analysis you might want to discount this valuation by a further 25% until the FDA formally approve at the PDUFA date of 30th September . Fair enough...that leaves a valuation of US$1.23 bn for sr aGVHD alone !!! I believe this is plainly ludicrous so i was not surprised to be offered an almost 10% fee by one of my brokers if I would allow my shares to be used in a stock lending programme. When i looked up the short interest on MESO (Nasdaq) last week , it was over 12% ! Meanwhile over in Australia , SHORTMAN website shows just under 6% short interest in the MSB ASX listing.
Someone is heading for an early bath in the next few weeks and I am pretty sure it is not me.
I estimate we are 22-25 days away from a 90 patient interim analysis for Covid 19. The recent Lancet peer review of Covid patients receiving Adipose MSCs in Spain as a salvage therapy when steroids and best available treatments failed....is a mirror image of our own Mount Sinai results. Whilst we still need confirmation from a proper RCT, I believe an informed observer would conclude that we have a very strong chance of achieving overwhelming efficacy in our first interim analysis. If my hopes are confirmed, the shares would probably rise to over $20 in Australia, before people begin tho realise the opportunity to use this therapy to combat bacterial and viral sepsis, influenza A, COPD, ...and a whole host of other inflammatory conditions such as emphysema and idiopathic pulmonary fibrosis. The fact that we have a list of such prestigious US hospitals participating in our trial plus the sponsorship of the NIH should tell you our chances are excellent.
I also think the comments made in the video by Amy Lightner (posted earlier by others) bode extremely well for our Phase 3 readout for Biologic refractory Crohn's Disease....which may well add a further significant amount to valuations when analysts get hold of all the published data.
Indeed all around the world MSCs are being used by medical institutions for a whole new range of conditions...the University of Birmingham are currently conducting the MERLIN clinical trial to treat 56 patients with either Primary Sclerosing Cholangitis or Autoimmune Hepatitis...the University of Sydney is targetting preeclampsia and Mesoblast has already referenced HIE as one of our near term label extension opportunities.
VIDEO Lastly, I cant be bothered to argue with the siren voices spamming/downramping/trolling this website .. i think Mesoblast has consciously prioritised the BLA sr aGVHD application and Covid 19, over all other opportunities. It is a logical decision. I am sure Silviu will reach agreement with the FDA over what cell harvesting protocols he will be permitted to use to scale up production, he will then be able to give far more accurate short term production and margin guidance to analysts . The Lonza websites press release yesterday , in conjunction with Silviu Itescu’s podcast appearance, suggests a US manufacturing based solution is waiting to be unravelled. After securing such a promising partnership deal with Grunenthal for CLBP in Europe and Latin America, I have no doubt the global pharma cos will be salivating over the Revascor opportunity in CHF. Will a partnership be “data dependent” on our phase 3 read outs ... or will the urgency to scale up manufacturing to treat victims of a global pandemic, mean that we see a deal in the next few weeks. Time will tell...shorting this stock now is the nearest thing to insanity i can think of ! I added appreciably to my own shareholding today . OP
Please do not rely on the accuracy of facts or opinions expressed on the above post when making an investment decision .
(20min delay)