Pridopidine

Pridopidine, tested in multiple HD trials, has consistently failed to meet primary endpoints, despite Prilenia's mixed messaging. The drug's intended use has shifted from movement control to disease modification, with inconsistent results. Prilenia's recent press releases about regulatory submission should be interpreted cautiously, as acceptance for review is a standard step in the approval process. HDBuzz emphasizes the importance of accurate reporting and the distinction between hope and hype.

EMA to review Prilenia's pridopidine for Huntington's disease, based on clinical data showing potential to slow progression. Pridopidine, with orphan drug status in EU and US, aims to offer a new therapeutic approach. Phase 2 trial showed efficacy, while Phase 3 trial results were mixed, with potential benefits in specific subgroups.

The ALS drug development pipeline is rich with promising therapies targeting TDP-43 pathology, including Biogen’s BIIB-105, AbbVie/Calico’s ABBV-CLS-7262, Denali’s DNL-343, and QurAlis’ QRL-201. Trials range from Phase 1 to Phase 3, with completion dates extending to 2026. These therapies aim to slow ALS progression by addressing TDP-43 mislocalization and aggregation, a hallmark of ALS pathology. The article also highlights the importance of clinical trials in advancing ALS treatment and the potential for FDA approval of these therapies.