The sponsor is blinded to efficacy data but not dropout numbers from DSMB. So CM knew about the 50mg dropout issue back in 2022 and then decided to pool both cohorts together in the final SPA, and subsequently changed the titration protocol in OLE. Both were wise moves. It also explained why CM floated the idea of using OLE as a confirmatory P3 trial in 2023 since p2b/3 was not able to confirm dose dependency due to titration issues.
However, the question now is why the company no longer prioritizes the OLE (e.g. did not announce the June trial completion, only mentioning the interim report by year-end). The wording (if you compare the May 10Q and Aug 10Q) has changed to "up to 96 weeks" from "additional 96 weeks", indicating dosing patients not staying through 96 weeks again in OLE.
It's still not clear to me whether the FDA guideline changing to one endpoint can be retroactive or only applies to further studies. Otherwise, all the companies with previous trials hitting one endpoint will come back to apply for NDA.
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