Ann: Mesoblast to File BLA for Ryoncil FDA Approval Next Week, page-124

Post #: 44794678 Second post on the entire banter thread

“Primary endpoint” is a sensitive term at Mesoblast’s Collins Street HQ after a 159-patient trial of Revascor for end-stage patients using left ventricle assist devices (LVADs or heart pumps) came a cropper last year.
Well, in the eyes of the market it did, because the shares tumbled 28 percent.
In short, the trial - carried out by independent investigators at New York’s Mt Sinai School of Medicine and funded by the US Government National Institutes of Health - did not meet its primary endpoint of temporarily weaning patients from the LVADs.
But Prof Itescu stresses the “academic” endpoint was set by the investigators - not Mesoblast - and was never viewed by the FDA as clinically relevant.
“That [weaning] was not something of any interest to us,” he says. “What we were interested in, based on FDA guidance in writing,was reducing the major clinically meaningful problem of recurrent hospitalizations from major gastrointestinal bleeding.”
“And we did. We reduced bleeding rates by 76 percent and hospitalization by 65 percent and these numbers were identical to an earlier pilot trial.”


Reg