Preclinically, our compounds produce significant and durable increases in synaptic density within several days. In patients, the emergence of new synapses should lead to measurable improvements in clinical symptoms. This synaptic-targeting mechanism should therefore allow clinical trial durations of three to six months and enrollment of patients across a broad range of disease severities.
We completed a successful Phase 1 study in June 2019. Our lead compound, RDN-929, was found to be safe and well-tolerated at all dosage levels, with favorable pharmacokinetic and pharmacodynamic properties. We will soon move into a Phase 1b study in patients. This trial will continue to assess safety, tolerability and PK over a longer dosing period, as well as exploratory measures including fluid and neuroimaging biomarkers measuring synaptic density.
Phase 1b data will help inform our plans for conducting multiple Phase 2 trials in synaptopathies with high unmet need. Each Phase 2 trial will enroll well-characterized patients who will be assessed via multiple outcomes: cognition, function, behavior, neuroimaging, digital endpoints and fluid biomarkers.