Table 2. List of compounds targeting either directly or indirectly α-synuclein which are in the pipeline but not yet translated to clinical trials.
Compounds listed here are still in preclinical development. For clarity purpose, we excluded the two compounds that are already in clinical development: PD01 and PRX002. Targets, mechanism of action, PCT numbers are indicated when information was available. α-syn: α-synuclein; GCase: glucocerebrosidase; GAIM: general amyloid interaction motif; IgG: immunoglobulin G; PTC: patent cooperation treaty.
| Drug Name | Sponsor | Target Name | Mechanisms of action | PCT or priority patent |
|---|---|---|---|---|
| AT-3375 |
Amicus Therapeutics
(Cranbury, USA) |
GCase | Pharmacological chaperone that increases GCase enzyme activity |
WO2007137237, WO2007150064, WO2010118282 |
| PD-0805 |
BioArctic Neuroscience
(Stockholm, Sweden) |
α-syn | Antibody against “toxic” forms of α-syn | WO2009133521 |
| EDP-32 |
BioLineRx
(Jerusalem, Israel) |
α-syn | Aggregation inhibitor | WO2010103515 |
| NI-202 |
Neurimmune Therapeutics
(Schlieren, Switzerland) |
α-syn | Antibody | WO2010069603 |
| NPT-088 |
NeuroPhage
(Cambridge, USA) |
α-syn, Tau, Aß | Second generation of NPT-001; human IgG1 fusion protein containing GAIM |
|
| NPT-001 |
NeuroPhage
(Cambridge, USA) |
α-syn, Tau, Aß | Bacteriophage M13 capsid with GAIM recognizing and remodeling misfolded protein aggregates |
|
| NLF-1233 |
nLife Therapeutics
(Granada, Spain) |
α-syn | RNAi | WO2014064257, WO2011131693 |
| PBT-434 |
Prana Biotechnology
(Parkville, Australia) |
Unspecified | Metal (iron) attenuating compound | |
| USP14 inhibitors |
Proteostasis Therapeutics
(Cambridge, USA) |
Ubiquitin specific peptidase 14 |
Proteasome activity enhancer | |
| ReS9-S7 |
reMYND
(Leuven, Belgium) |
Unspecified | Facilitates α-syn degradation | |
| ReS12-S |
reMYND
(Leuven, Belgium) |
α-syn | Chemical, synthetic | WO2009019295 |
| SIG-1012 |
Signum Biosciences
(Monmouth Junction, USA) |
Protein phosphatase 2 (formerly 2A) |
α-syn phosphorylation enhancer | |
| G2 PD |
TauRx Pharmaceuticals
(Singapore, Republic of Singapore) |
Unspecified | Compound targeting misfolded α-syn |