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Ok... I am an amateur with respect to regulatory affairs, but did pay my dues at the periphery supporting drug development from a program management standpoint. Question for the clinical/regulatory experts.

Data Point #1:
IF – per AGIO press release the following is true – “ AGIO announced that the FDA has granted Breakthrough Therapy designation…This prestigious tag to Tibsovo is based on data from the initial 12 patients in the MDS arm of Agios’ phase I study….Agios re-opened the MDS arm of the study in October 2019 and is focused on generating data to submit a potential regulatory filing for this indication. It will enroll up to 25 total patients in this cohort.”

THEN … doesn’t GERN have more than 25 patients already?
And if Data Point #1 is true ~ then does it mean that GERN is /should effectively argue that GERN is in filing prep mode, armed with more than 25 patients?

I don't think its a matter of number of patients but in Agios case a defined molecular target IDH1, something present in <4% of MDS patients. The FDA seems to like targeted molecular end points. bp

The issue isn't number of patients, it's statistical power. If you get a high percentage of CRs in your 25 patients, that's a lot better than unclear results on a larger number.

That's how effective drugs get conditional approval straight out of Phase 1 and 2.