Pridopidine

Prilenia Therapeutics and Ferrer have signed a collaboration and license agreement worth up to €500 million for pridopidine, a selective sigma-1 receptor agonist targeting Huntington's disease and ALS.

Pridopidine demonstrated significant benefits on clinical progression, cognition, and motor function in Huntington's disease patients not taking antidopaminergic medications during the PROOF-HD phase 3 trial.

Pridopidine demonstrated safety and potential efficacy in secondary endpoints for ALS patients, particularly improving motor speech performance, despite not meeting the primary endpoint in the Healey Platform Trial.

The HEALEY ALS Platform Trial is pioneering a new approach to accelerate the development of effective treatments for amyotrophic lateral sclerosis (ALS).

Several companies are advancing potential Huntington's disease treatments, including Wave Life Sciences with WVE-003 and uniQure with AMT-130.