Having just listened to the conference call , I would make the following comments :
1) As ecool2 has already alluded to, we were treated to a ringing endorsement from Dr Joanne Kurtzberg, Director of the Pediatric Blood and Marrow Transplant Programme at Duke University Medical Center . She stated without any hesitation that she would “definitely” use Remestemcel-L (after approval) as first line therapy for children after being unresponsive to steroids. As a physician she would only be likely to try Jakafi, if Remestemcel-L failed to get a response. Prof S I compared the public trial data showing that patients treated with grades C/D aGVHD Mesoblast achieved 89% response rate compared to 41-43% for Jakafi. Dr Kurtzberg then proceeded to mention that Remestemcel-L was also preferred because it did not suppress the immune system and there were no problems with toxicity.
2) If priority review is given by the FDA for Remestemcel-L , (which as an orphan drug is highly likely in my opinion), we should receive a decision on approval within 6 months of the final module being filed by Mesoblast. In my view we should hear the decision around the end of March/April 2020. Very encouraging that confirmatory trials for adult and chronic aGVHD will be pursued as soon as possible assuming a successful outcome. SI also added “other indications” to his slide for label extension opportunities in 2020...other than the EB opportunity, this is something I do not remember hearing before....
3) AT LAST THE GENIE IS OUT OF THE BAG... 85% of MACE events have already occurred in the 566 patient Revascor trial. S I referenced the fact that many of phase 3 clinical trial patients were now in their 3rd and 4th year post enrolment...so he notes that there is now “an increase in MACE rate and an accelerating rate accrual. He let slip right at the end the Revascor trial is likely to complete “sooner than we had anticipated...see how the next few months will go”.
By my estimation, a continuance of a typical j curve of mortality risk and hospitalisation plotted against the enrolled patient profiles leads me to conclude that the REVASCOR TRIAL WILL CONCLUDE IN ABOUT 9-11 weeks. This will not come as a surprise to anyone who has read my posts before. Just consider the ramifications of the last statement. Revascor is aimed at 15-20% out of 8m patients suffering with CHF market that has failed to respond to all other treatments. It also could be fast tracked to give unique support to those 5,000 patients who annually undergo LVAD implants. If you want to be prudent and risk a successful Revascor trial at say 30%, you would still get a share price many many multiples of what it is today. Expect some real interest to develop in the shares in the next 2 months !
4) Interesting that in CLBP, over 50% of the 404 enrolled patients in the Phase 3, 3 arm study have already completed their 24 month study. S.I. said that “further disclosure” from the Phase 2 results would be made available “in due course” but there would be no public interim read outs of the current trials. He did mention, however, that there have been no “adverse events to date during the trial”. With a 50% improvement in pain score and 15% improvement in function, set as the hurdle rates to be achieved from the phase 3 trials ( simi lar to outcomes achieved in phase 2 trials ) we will have to wait until April 2020 for the trial to conclude .
In summary, many slides were familiar to us all from previous presentations. Mesoblast is shifting to a commercial footing. R&D costs will fall as phase 3 trials reach their conclusion whilst manufacturing facilities will correspondingly rise to facilitate production.
We already knew that Remestemcel-L achieved vastly superior clinical results to Jakafi in clinical trials...but it was good to hear, from a world renowned expert on the subject of aGVHD, state that MSB should be “first line” after steroids. Looking at the valuation of Incyte we should be very pleased at the thought of label extensions being sought in 2020 to allow Mesoblast entry into the adult and chronic market for aGVHD.
Lastly, the Revascor trials which first started back in 2014 is probably less than 3 months away from reaching its targeted 540 Major Cardiac Events...you have been warned ! This is the key takeaway from todays presentation !!!
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