Ann: Investor Webinar Presentation, page-5

The wisdom is not the RCT alone, but a combination of having the Osiris/MSB knowledge of past trials combined with commentary of industry specialists such as Galipeau and Murata and the ability to develop a potency assay based on our own early-stage trials conducted vs the acquisition of a late-stage platform with a history of P3 trials conducted that missed primary endpoints.

How they have used that wisdome is yet to be seen however. It is still too early to tell given that we only have data of 15 patients in SR-aGvHD and are about to conduct a trial in HR-aGvHD patients. But - given the nature of the therapy here - collecting enough data during early stages of development to support an assay matrix approach as a measure of potency is definitely a big advantage.
If you are in late stage development and you discover that your previous potency assay contained errors, it is a big task measuring potency of an active product that was used years ago, especially when the study has been undertaken in a small sub-group which then has to be further reduced to patients that received a product that is in line with the new potency assay. Whilst the announcements along the way sounded promising, it turned out that whilst the potency approach appears to be reasonable, there simply wasn't enough data available to support that. The FDA wasn't stopping MSB to conduct another trial in adults once they believed to have fixed the issues as outlined in the first CRL before resubmitting the BLA.

This document may be a little outdated, but is still relevant:







https://www.fda.gov/files/vaccines,%20blood%20%26%20biologics/published/Final-Guidance-for-Industry--Potency-Tests-for-Cellular-and-Gene-Therapy-Products.pdf

The above is something that @JB1975 correctly predicted based on the very limited information published in the public domain by MSB.


https://hotcopper.com.au/threads/ann-trading-halt.7506871/page-181?post_id=69115569

There are many posts like the above that showed the reasoning behind this prediction and is definitely worth reading if you haven't done so already.

With that in mind, I will definitely re-visit some of JB's critical commentary on CYP's manufacturing approach and claims over the next few weeks and follow up on that with our new CEO.