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Table 2 Drug-target pairs for neurodegenerative diseases associated with misfolded proteins

From: Protein misfolding in neurodegenerative diseases: implications and strategies

Compound name Company Disease indication Mechanism of action Status
TRx0237 TauRx Therapeutics Alzheimer’s disease Tau aggregation inhibitor Phase II clinical trials completed
AADvac1 Axon Neuroscience SE Alzheimer’s disease Active tau based immunotherapy Phase I clinical trials completed
ACI-35 AC Immune AG Alzheimer’s disease Phospho-tau vaccine Phase I trial active
Arimoclomol Orphazyme ApS Amyotrophic Lateral Sclerosis HSP activation Phase II/III active
Nuedexta Avanir Pharmaceuticals Amyotrophic Lateral Sclerosis – PBA symptom treatment Unknown for PBA treatment; NMDA receptor antagonist FDA approved
Deferiprone Generic Parkinson’s disease Iron chelator Phase II recruiting
Istradefylline Kyowa Hakko Kirin Parkinson’s disease Adenosine A2A receptor antagonist Approved in Japan; no FDA approval