All clinical_trials

PHOENIX failed primary; Relyvrio voluntarily withdrawn April 2024. Triggered post-hoc reflection on Phase 2 effect-size inflation.

Failed primary endpoint despite Mendelian-randomization support for urate→PD; classic example of MR-justified target failing in trial.

Halted March 2021 — exploratory analyses suggested potential harm from non-allele-specific HTT lowering. Reframed the field around earlier intervention + allele-selective targeting.

Failed primary on cUHDRS; subgroup analyses suggested benefit in patients not on anti-dopaminergic background therapy.

First systemic AAV gene therapy for a PD subtype; targets the most common genetic risk factor (GBA1 LoF).

Failed primary in VALOR; FDA accelerated approval April 2023 based on plasma neurofilament-light reduction. Surrogate-endpoint case study.

35% slowing on iADRS in low/medium tau population; 2nd FDA-approved disease-modifier (Kisunla, July 2024).

Anti-amyloid monoclonal that achieved 27% slowing on CDR-SB; led to traditional FDA approval July 2023. Defined the post-amyloid AD-disease-modifying era.