The FDA doesn’t suggest endpoints for a phase 2 with a novel treatment for an unmet need. They suggest relevant endpoints that relate to the unmet need, in order to find which types of efficacy the drug with show, not to establish what is necessary for approval.
Even if endpoints are not met in a phase 2 trial, a P3 can be designed based on the data and agreed with the FDA. For that the PDD P2 readout we have seen is great.
No approval until P3 trial endpoints are met and no meaningful SP improvement from the PDD P2 data to date.
SP might increase with the analysis and release of the full PDD P2 data or from Rett readouts. Otherwise will be late 2022 or into 2023 for next hope of getting rich.
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