Protein misfolding in neurodegenerative diseases: implications and strategies

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

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

ISSN: 2047-9158