aptaTargets’ Phase 1b clinical data for ApTOLL published in ‘Molecular Therapy: Nucleic Acids’ journal

12 April, 2022

aptaTargets Molecular Therapy journal
aptaTargets’ Phase 1b clinical trial data for ApTOLL-FIH-01 has been published in the American Society of Gene & Cell Therapy’s Molecular Therapy: Nucleic Acids journal and it shows that the drug ApTOLL has an excellent safety and pharmacokinetic profile.

ApTOLL is an aptamer (single-stranded DNA molecule) that has been selected to block the Toll-like receptor 4 (TLR4) with high specificity and, therefore, block the inflammatory response produced after acute ischemic stroke or acute myocardial infarction.

The randomised, placebo-controlled ApTOLL-FIH-01 clinical trial was performed in 46 healthy adult male volunteers to whom a 30-minute intravenous infusion of ApTOLL and placebo was administered. The study was divided into two parts: part A, which included seven single ascending dose levels, and part B, where three doses were administered in a period of 24 h. No adverse effects related to the drug of the study or biochemical alterations were detected in any ApTOLL administration pattern.

“These results, together with the demonstrated efficacy of ApTOLL in non-clinical studies, support the clinical development of ApTOLL. In this regard, we started the APRIL Phase 1b/2a international clinical trial in acute ischemic stroke patients at 16 hospitals in Spain, Germany and France” explains Dr Macarena Hernández, Chief Scientific Officer of aptaTargets.

Dr Macarena Hernández and Dr Marc Ribó, Chief Medical Officer of aptaTargets and Assistant Professor in the Stroke Unit at the Vall d’Hebron Hospital in Barcelona, presented the results of the Phase 1b study at the International Stroke Conference 2022 last February.


Hernández-Jiménez M, Martín-Vílchez S, Ochoa D, Mejía-Abril G, Roman M, Camargo-Mamani P, Luquero-Bueno S, Jilma B, Moro MA, Fernández G, Piñeiro D, Ribó M, Gonzalez VM, Lizasoain I, Abad-Santos F. First-in-Human Phase I Clinical Trial of a TLR4-binding DNA aptamer, ApTOLL: safety and pharmacokinetics in healthy volunteers. Molecular Therapy Nucleic Acids (2022). DOI: https://doi.org/10.1016/j.omtn.2022.03.005