@rvajnaTo recap. The FDA has reportedly told the Company to make submissions for three different indications…namely
1) resubmit BLA for Steroid Refractory acute GVHD for paediatric use.
2) apply under Accelerated approval pathway for end stage heart disease using LVAD
3) apply under Accelerated approval pathway for HLHS
EACH one of these indications, if authorised, would generate in excess of a US $1bn tag …I reference below a US$2bn tag for Kadmon in 2021 which only had initial use as a third line treatment in adult cGVHD. If pricing for Ryoncil reflects the long term the mortality data for four years , the duration of response is so superior we might see pricing upwards of US$800k per course and off label use in refractory grade 3/4 adults looks inevitable post approval…which might be much higher as it is priced based on dosage per kilogram.
The current pivotal trial for CLBP offers at least a US$3 bn sales opportunity on reasonably conservative pricing . All the Company has to do is REPEAT the results of its previous Phase 3 trial in the pain endpoint for the trial currently recruiting Even if you risked the chances of success at 20% …the market cap could rise to US$3-4 bn on that opportunity alone and nearer US$ 6-10b on meeting the primary endpoint. Just remember the size of the milestone payments for the Grunenthal deal which excluded the US if you do not believe me.
As for ischemic Heart Failure, it is the number one cause of mortality worldwide so off label use there would be substantial sales opportunity …in my opinion…not to mention ARDS, refractory Crohn’s , etc.
As a first in class therapy for a whole host of inflammatory conditions with clinical data going back the best part of 20 years with the IP to match, your whole argument hinges on authorisation…which is a fair point. We shall see. For those interested in following the format of this authorisation process for the Ryoncil resubmission ..i believe resubmissions fall under SOPP8405 , which then reverts to SOPP8401 according to item H on page 7.
https://www.fda.gov/media/84417/downloadIf Ryoncil was a totally new submission , i might have some sympathy with your argument , but we have passed an ADCOM and the manufacturing PLI , which means by my calculations, we should be entering into labelling discussions around the next week or so . I could be wrong …I have been before ..but we have far more boxes already ticked and a request from the FDA to file…which would make a further CRL highly improbable. Apparently the FDA have already seen the new top level data for potency and asked them to refile. I guess it’s down to whether the new data stands up to interrogation. As Philip Krause has packaged it altogether, I am not expecting a negative review.
https://www.sanofi.com/en/media-room/press-releases/2021/2021-09-08-05-00-00-2293017
Lastly, the IP portfolio Mesoblast has in CMC and composition of matter is substantial…enough to make Takeda pay royalties on Alofisel for peri anal fistula.
You only have to look at the huge number of clinical trials underway using MSCs to know that our IP will be very valuable on widespread commercialisation !Please do not rely on the accuracy of any facts or opinions expressed in the above post when making an investment decision. OP
(20min delay)