Table 2.
Triage of 842 compounds through validation counter-screens (SNCA-luc, CMV-luc, Cytotox) and orthogonal assays (ELISA)
| Triage step | Criteriona | No. of Compounds |
|---|---|---|
| Total evaluated in follow-ups | 1 | 842 |
| Confirmed in the SNCA-luc assay (of 842) | 2 | 446 |
| Passing CMV-luc and Cytotox counter-screens (of 446) | 3 | 33 |
| Lowering SNCA in 3XSNCA fibroblasts by ELISA (of 33) | 4 | 5 |
Criteria for advancing compounds: (1) Negative curve class in primary screen. (2) Confirmed negative curve class, >30% inhibition for SNCA-luc assay. (3) ΔAUC (single metric of potency and efficacy) of >40 comparing SNCA-luc versus both CMV-luc and cytotox assays. (4) >50% reduction for SNCA at 1 μM.