PBT434 inhibits alpha- synuclein in the brain., page-19

Good luck kpax. Here is some preclinical animal model work:
[ Here we report the preclinical characterization of PBT434, a novel quinazolinone compound bearing a moderate affinity metal-binding motif, which is in development for Parkinsonian conditions. In vitro, PBT434 was far less potent than deferiprone or deferoxamine at lowering cellular iron levels, yet was found to inhibit iron-mediated redox activity and iron-mediated aggregation of α-synuclein, a protein that aggregates in the neuropathology. In vivo, PBT434 did not deplete tissue iron stores in normal rodents, yet prevented loss of substantia nigra pars compacta neurons (SNpc), lowered nigral α-synuclein accumulation, and rescued motor performance in mice exposed to the Parkinsonian toxins 6-OHDA and MPTP, and in a transgenic animal model (hA53T α-synuclein) of PD. These improvements were associated with reduced markers of oxidative damage, and increased levels of ferroportin (an iron exporter) and DJ-1. We conclude that compounds designed to target a pool of pathological iron that is not held in high-affinity complexes in the tissue can maintain the survival of SNpc neurons and could be disease-modifying in PD.] note they did give a preclinical A-Syn type.
https://actaneurocomms.biomedcentral.com/articles/10.1186/s40478-017-0456-2
Now if PBT434 has inhibited A-Syn in the human trial, that is strong evidence the drug has made that important jump by reproducing an effect documented in animals to humans.
This is Fairpark2: https://cordis.europa.eu/project/rcn/193184/reporting/en
This from the MSA conference before the trial :
[The new experimental data demonstrate that in an animal model of multiple system atrophy, PBT434 prevents α-synuclein accumulation, preserves neurons and decreases the number of glial cell inclusions in the brains of treated animals. Glial cell inclusions are the key pathological finding in MSA and contain abundant aggregated α-synuclein that is associated with neurodegeneration. Importantly, these benefits led to improved motor function in treated animals. Alpha-synuclein is of great interest because aggregated forms of the protein are considered a pathological hallmark of Parkinsonian conditions and are a recognised therapeutic target by basic and clinical neuroscientists. “Multiple system atrophy, or MSA, is a devastating disease with limited treatment options. The data from this animal model of MSA are robust and indicate that PBT434 has excellent potential to treat this progressive neurodegenerative disease.]
https://alteritytherapeutics.com/investor-centre/news/2018/03/06/pbt434-poster-presented-6th-international-multiple-system-atrophy-conference/