“And are now applying for accelerated approval.”
@DocMcstuffinsDoc, the resubmission for paediatric sr aGVHD is for a FULL APPROVAL. When you get the basics wrong..the rest becomes hypothesis generating (sic)
Please do us all a favour..write to the Company and ask for clarity. Alternatively, continue to mislead people if you prefer ? You will be found out within approx six weeks, when the resubmission is due to be processed… so you might as well make a clean breast of it now.
Another point you might like to ask yourself is how useful is a new clinical trial based against a surrogate endpoint designed to show durability of effect when the FDA already have spectacular four year actual mortality data for Ryoncil from GVHD001 . In contrast the only approved therapy for over 12 years old,Ruxolitinib, has shown in Reach 2, that it doesn’t not have durability of effect in severe grade disease relative to controls based on longer than 12 month endpoints. Based on this evidence, why go head to head with a therapy using surrogate endpoints which are not fit for purpose? . Furthermore, the majority of severe grade Ruxolitinib patients , which represents approx 45% of total patients treated, become Ruxolitinib refractory which is quite shocking. If Ryoncil wants to apply as a second line therapy for adult sr aGVHD they will have to do a head to head with approx 200-300 patients. They will achieve
better pricing and probably get a quick approval based on a small investigator lead trial if they stick to a Ruxolitinib
refractory population. In my opinion, most physicians will use Ryoncil as a second line therapy for severe grade adult patients post paediatric approval as further RWE emerges .
In contrast , Mesoblast appears to be free to apply for accelerated approval for its CHF treatment, without a further trial being required until
after approval.
How you want to spin that I will leave to you. A further trial in ischemic CHF is being considered to fulfil that post trial requirement that will likely take a 2-3 years if based on traditional MACE endpoints. Post any Accelerated Approval for Destination Therapy LVAD ischemics I would expect an explosion of off label use in ischemic patients because the DREAM Revascor Phase 3 showed an unprecedented efficacy in strokes and heart attacks. The numbers are pretty mind boggling.
Lastly CLBP, based on a conservatively risked chance of success for the imminent enrolment of the Phase 3, could easily be valued right now in the billions of dollars…but i believe it will take a first approval in GVHD before investors are totally convinced on this. If you look at the Grunenthal royalty deal it clearly validates my opinion based on the size of the milestone payments and subsequent royalties. With an adjusted patient cohort of CLBP less than 68 months and just a 12 month pain reduction endpoint, the efficacy required does not look demanding at all, when referenced against the p values achieved in the previous phase 3 subset population. OP
Please do your own research and do not rely on any facts or opinions contained in the above post when making an investment decison.
(20min delay)