ABSTRACT NUMBER: ESOC2026A1168 HEALTH-RELATED QUALITY OF LIFE OUTCOMES IN PATIENTS WITH ACUTE ISCHAEMIC STROKE (AIS) TREATED WITH APTOLL 0.2 MG/KG IN THE APRIL PHASE 1B/2A STUDY

Abstract Background and aims ApTOLL, a TLR4-antagonist single-stranded DNA aptamer, was assessed in patients with AIS treated with endovascular thrombectomy in the APRIL Phase 1b/2a randomised, double-blind trial (NCT04734548). Primary results, previously reported, show favourable safety and early clinical/imaging signals for the 0.2 mg/kg dosage. Here we report exploratory Day-90 health-related quality of life (HRQoL) outcomes. Methods In APRIL, 139 patients were randomised to two ApTOLL doses or placebo. HRQoL was assessed using EQ-5D-5L and EQ-VAS; data were available for 42 patients receiving ApTOLL 0.2 mg/kg and 50 patients receiving placebo. Analyses were descriptive and examined the 0.2 mg/kg dose and placebo arms. Results Baseline severity indicated moderate-to-severe stroke (pre-randomisation NIHSS medians: 17.0 [12–21] versus 18.0 [13–20]; pre-stroke mRS medians 0 [IQR:0–0] in both arms). At Day 90, a higher proportion of patients receiving ApTOLL reported ‘no problems’ across all EQ-5D-5L dimensions versus placebo (Figure 1). The largest absolute between-group differences were in anxiety/depression (‘no problems’ 27% more with ApTOLL) and usual activities (18%), with smaller differences (9–14%) in mobility, self-care and pain/discomfort. Patient-reported health status favoured ApTOLL (EQ-VAS medians were 70.0 versus 53.5), suggesting better overall health (Figure 2). Conclusions ApTOLL 0.2 mg/kg was associated with numerically more favourable HRQoL scores versus placebo, particularly in mood and daily activities, with higher overall health status. These HRQoL findings align with previously reported favourable safety and early clinical/imaging signals in APRIL, supporting continued evaluation of ApTOLL in AIS. Conflict of interest Conflict of interest: Dr Ribo is a consultant to Merck KGaA, CMO of AptaTargets, Cerenovus, Medtronic, Philips, Rapid Pulse, Stryker Corporation, and Vesalio; has stock options in Anaconda Biomed, Methinks, and Nora; and received compensation from Sensome for data and safety monitoring services. Dr Hernández-Jiménez is a Scientific Advisor to aptaTargets S.L. Caroline Petit is an employee of Merck Santé S.A.S., Lyon, France, an affiliate of Merck KGaA. Daniela Piani Meier is an employee of Ares Trading SA, Eysins, Switzerland, an affiliate of Merck KGaA, Darmstadt, Germany. Achille Dunne is an employee of Merck Serono Ltd., Feltham, UK, an affiliate of Merck KGaA, Darmstadt, Germany. Silvia Zaragoza Domingo is an external consultant, Merck KGaA, Darmstadt, Germany. Danielle Harlow is an employee of EMD Serono. Namita Tundia is an employee of EMD Serono. Figure 1 - belongs to Results Figure 2 - belongs to Results