ACT-02

ACT-02 is a first-in-class, orally active small molecule drug candidate being developed by Accure Therapeutics for the treatment of Parkinson's disease. It acts as a selective inhibitor of prolyl endopeptidase (PREP), also known as prolyl oligopeptidase, a target involved in the regulation of protein aggregation and inflammation. ACT-02 possesses disease-modifying potential through a dual mechanism of action that interferes with key pathological pathways, including the reduction of α-synuclein aggregation, mitigation of neuroinflammation, and improvement of mitochondrial function. The compound is brain-penetrant and has demonstrated neuroprotective effects in dopaminergic neurons along with improved motor and cognitive performance in preclinical models. As of early 2024, ACT-02 is in advanced preclinical development with an Investigational New Drug (IND) application expected in 2026.