Ann: Submits New Information to FDA IND File for SR-aGVHD, page-121

The problem with MSB announcements is that they fail to properly contextualise the information presented.

Take the MAGIC study. This was presented to investors as a great success. MAGIC had provided an “unbiased and independent” confirmation of the P3 single arm control group estimate. On the surface you may have imagined that things were just getting better and better.

The reality was that MSB were in the deep doo doo and MAGIC was a last ditch effort to try and save the day. Because unbeknown to investors the FDA had rejected the P3 control group estimate. Arguably the announcement was very misleading because MSB failed to properly contextualize the reason MAGIC was being conducted.

Once bitten – twice shy.

Why are MSB providing "further evidence of the ability of rem-l's ability to save lives rather than simply resubmitting their application? Answer because they are stymied.

So the question becomes - will this new information cause the FDA to rethink their request that MSB conduct another trial; which then enables MSB to resubmit?

It's a long shot. The FDA have already indicated that they are not interested in survival outcomes here. From the Ad Com briefing:

“In addition, as described in the 2018 FDA guidance document for clinical trial endpoints, time-to-event measures such as overall survival are difficult to interpret in single-1 arm trials, and Study 001 was not designed to detect differences in survival. And therefore, survival endpoints will not be discussed further by FDA.”

So what next?

If this new survival information doesn’t cause the FDA to rethink their request for an additional trial MSB will simply try something else, and this will go on ad infinium. So basically a black hole.