ABSTRACT NUMBER: ESOC2026OT73 BB-031 IN ACUTE TREATMENT: SAFETY, TOLERABILITY, PHARMACOKINETICS (PK) AND PHARMACODYNAMICS (PD) IN ACUTE ISCHEMIC STROKE – THE RAISE TRIAL

Abstract Background and aims BB-031, an RNA aptamer, potently and rapidly inhibits von Willebrand Factor (vWF) binding to its platelet receptor, is developed to impede and resolve thrombus formation and embolization. A Phase1 study in healthy volunteers demonstrated its safety and dose-dependent inhibition of vWF-binding and thrombosis. Methods The Recanalization in Acute Ischemic StrokE (RAISE) trial is an ongoing Phase 2, international, randomized, placebo-controlled, double-blind study evaluating BB-031’s safety, tolerability, pharmacokinetics, and pharmacodynamics in acute ischemic stroke (AIS) patients (NCT06226805). Results Part A assessed single ascending doses (0.5–4 mg/kg) in 48 patients (3:1 randomization BB-031:placebo). Following unblinded DSMC review, Part B will enroll ~180 patients, starting with 2 mg/kg and escalating to 6 mg/kg after DSMC approval. Eligible patients must be enrolled within 24 hours of symptom onset, with confirmed anterior circulation occlusion and no prior/planned thrombolysis. Conclusions The primary endpoint is incidence of symptomatic intracranial hemorrhage (ICH) at 24 hours. Secondary endpoints include any ICH, pharmacokinetic parameters, and vWF activity modulation. Exploratory clinical outcomes are recanalization rates at 24-hours, NIHSS, and mRS at 90 days. Conflict of interest