In a very revealing interview which was published in...

In a very revealing interview which was published in Biopharmadive in May 2021, the new emerging CEBR position on Biologic therapies was revealed .(link below).

"Just like manufacturers like consistency, FDA likes consistency in product," said Peter Marks, head of the regulator's biologic drugs division, at the World Medical Innovation Forum's virtual meeting.

Consistency is more challenging with cell and gene therapies — which are highly engineered products that can involve a patient's own cells — than with pharmaceuticals or even the antibody drugs that are now commonplace across the industry.

According to Marks, cell and gene therapy developers haven't always done a good job constructing tests early on that allow them to consistently measure their products as they move from early testing into larger clinical trials and, eventually, to the FDA.

"It sounds almost sing-songy," he added. "But many times developers get very excited about the fact that their product produces an important effect that they don't worry as much about reproducibly making that product."

Because cell and gene therapies rely on biological material, such as patient cells or viruses, scaling up production isn't as straightforward as linearly increasing a product's component parts. Several developers, including Pfizer, SareptaTherapeutics and Bluebirdbio, have had to work through disagreements with the FDA on how they are comparing commercial-scale production to earlier clinical manufacturing, for example.

In addition, as many of the cell and gene therapies now in clinical testing are among the firstoftheirtype developed, it's not always clear from the beginning what characteristics or attributes are most important for reliably assessing clinicaleffect.

But, according to Marks, developers still need to try. "Picksomething. Pick some quality of the cell. Pick something that you think might correlate and measure that," he said. "We'll take any offers that are reasonable."
https://www.biopharmadive.com/news/fda-marks-gene-therapy-consistency/600445/


Funny word that “reasonable” . That was precisely the same words used bytheFDA to describe our potency assays in Mesoblast’s latest summary of the FDA’sminutes of the meeting ...which I believe were lifted almost verbatim and published in the full year results …because the Company did not want to be accused of misleading any investor about their content.

I quote from one of the main bullet points found in the latest “ OperationalHighlights” of the full year results .
“FDA indicated that the potencyassayscurrentlyunderdevelopment appeared to be reasonable based on the in vitro results in the briefing document” and that the “in vitro activity of the product” appears relatively well established.

Was this a coincidence ? I think not ! It certainly suggests to me that Mesoblast is about to jump successfully through the outstanding regulators hoops at the upcoming OTAT meeting. If I am right they will most likely file immediately afterwards for a new BLA. The logjam will be removed and the share price will rebound substantially . By allowing Mesoblast to fast track its Covid ARDS treatment by cross referencing manufacturing data from its sr aGVHD results the FDA appears to want to accelerate the approval process which is great news. Will Novartis want to finalise their deal, before or after OTAT ? I believe the former . If they wait until after and the outcome is positive their leverage will be gone and they will not look as attractive a partner in other indications.
Very few people talk currently mention Chronic Heart Failure right now but with such outstanding clinical data from the Revascor Phase 3 trial, it cannot remain off centre stage for long ! The preliminary findings of the Cleveland Clinics P1/2 trial into Chrohn’s & Ulcerative Colitis using Mesoblasts MSCs are also due shortly. It looks like being a very exciting couples of months . OP



Please do not rely on any facts or opinions contained in this post when making an investment decision.