FDA ODAC Meeting Material discussion analysis, page-170

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Stellowe

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12/08/20

13:18

Post #: 46482668
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Originally posted by stockrock: ↑

I think the FDA is asking for guidance as there are two Osiris trials using the same cells that didn’t meet their primary outcomes. MSB have stated their case on this, and in simple terms one of the studies was for early stage aGVHD and those patients are a lot more responsive to steroids in that stage, so there was not clinically significant difference using Ryoncil. So really they are not comparing apples with apples- as we are going for pediatric SR aGVHD approval... so kids who did not respond to steroid treatment. MSB did some data mining and found a positive impact on using Ryincil on a population of 14 ... so not a large population but it supports MSB’s argument that in the target population for this phase 3 trial, it appears the old trials support it.

at the end of the day the FDA need to make a decision... do we withhold this treatment that has shown to be highly effective in saving children’s lives who have SR-aGVHD so we can run another trial.

Or do we approve it because there are no safety concerns and request a post approval trial to alleviate any concerns the FDA may have on manufacturing.

at a very high level, MSB needs to satisfy the FDA on three levels... first is safety, second is efficacy and third is manufacturing.

the two most important in my view are efficacy, followed by safety... no issues with safety whatsoever... and while efficacy is questioned I.e single arm trial, and what about the two older trials that failed , I am confident MsB and the ODAC can easily address it.

manufacuring is in my view the final hurdle and being to demonstrate quality control and potency from batches is the key issue raised by the FDA. This is important, and the biomarkers used by MsB are different to the FDA, but that doesn’t make them wrong. In fact I believe this is where the ODAC process will shine and actually help MSB’s case.

but even if they don’t, I don’t feel that in itself will stop the FDA from approving Ryoncil. There is a clear clinical benefit in using Ryoncil, and its extremely safe. What they are concerned about is whether MsB can ensure they can produce Ryoncil with the same potency from batch the batch. They can mull on it, but another trial won’t really give them that much comfort in my view. I’d provably take more comfort that these same cells have been used in Japan for 3 years and the clinicians seems to be endorsing it by using it more each year... I don’t know if MsB will be able to share the data on that, I hope they can.

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Do the drafted am discussion questions about potency and quality control hold any relevance to the final vote? These relate to manufacturing and mechanism of action concerns.

pm questions appear more relevant as they relate to study flaws and relevance of prior studies

safety is not mentioned so is obviously no concern

vote - does data support efficacy in rem- l for p sr agvhd

Appears they are using allot of the discussion to gather some insight into possible future study designs, markers for quality control. And the fda raised a number of concerns in their briefing but how relevant is it to the voting question? Can we assume a positive vote regarding efficacy in the trial is all we really need to worry about given the correlation to fda approval.

MSB Price at posting: $3.18 Sentiment: Hold Disclosure: Held
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FDA ODAC Meeting Material discussion analysis, page-170

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