First in class neuroprotective small molecule for ischemic stroke

Small molecule that stabilizes the retromer complex, showing strong neuroprotective effects in highly excitotoxic conditions and maintaining key neuronal survival proteins like KIDINS220 in validated in‑vitro models.

Market need: Ischemic stroke represents one of the greatest global health burdens: it is the second leading cause of death and the third leading cause of death and disability combined worldwide. Globally, its prevalence exceeds 68 million cases, driven by aging populations along with the rising incidence of hypertension, diabetes, obesity, and other major risk factors. Despite this vast disease burden, current treatments remain limited: no effective neuroprotective therapies exist to prevent irreversible brain damage during the acute phase of ischemia, and millions of patients consequently suffer long‑term disability.

Proposed solution: R55 is a first‑in‑class small molecule that stabilizes the retromer complex inside cells, demonstrating robust neuroprotective activity in highly excitotoxic environments and preserving levels of neuronal pro-survival molecules such as KIDINS220, in validated in vitro excitotoxicity models. We have further conducted preclinical assays in a mouse model of ischemic stroke, administering these small molecules to show that they also confer strong in vivo neuroprotection against excitotoxicity. Taken together, these results position R55 as a highly promising therapeutic candidate for neurological disorders with significant unmet medical need.

Competitive advantages

First‑in‑class small molecule for unmet medical need.
Strong neuroprotective effect, preventing excitotoxic neuronal death and brain damage caused by ischemic stroke.
Significantly reduces cerebral infarct volume.