A4
ICR-001: First-in-class dual action oral therapy for secondary stroke prevention
Dr Szandor Simmons, Charité - Universitätsmedizin Berlin; Prof. Wolfgang Kübler, Charité - Universitätsmedizin Berlin; Erik Asmus, Charité - Universitätsmedizin Berlin; Prof. Robert Szulcek, Charité - Universitätsmedizin Berlin; Prof. Robert Preissner, Charité - Universitätsmedizin Berlin
Ascenion GmbH
Challenge
Stroke remains a leading cause of cardiovascular morbidity and mortality worldwide. Patients who have experienced an initial ischemic stroke are at a significantly increased risk of recurrent events. Secondary strokes are often associated with greater neurological impairment, permanent disability, reduced quality of life, and an increased risk of mortality. Although antiplatelet therapies, such as aspirin and clopidogrel, reduce the risk of recurrence, a significant proportion of patients cannot be adequately treated due to bleeding risk, contraindications, or an insufficient response related to pharmacogenetic variability. Therefore, there is an evident unmet medical need for safer and more universally effective antiplatelet therapeutics to prevent secondary strokes.
Technology
Icoris addresses this unmet need with its lead candidate, ICR-001, a first-in-class, orally administered medicinal product developed for the secondary prevention of stroke. ICR-001 repurposes an approved drug with a robust long-term clinical safety record, enabling a differentiated antiplatelet approach with a low inherent risk of ischemia and bleeding. ICR-001 is a dual-acting drug that targets the cystic fibrosis transmembrane conductance regulator (CFTR), a protein that has recently been recognized as a key regulator of both platelet activity and blood vessel integrity. ICR-001 modulates CFTR to attenuate platelet activation and stabilize the vascular wall. This addresses two fundamental processes that drive recurrent ischemic events and secondary stroke risk. Ex vivo studies using blood from healthy donors and high-risk patient populations, including those with COVID-19, demonstrated a significant decrease in platelet activation, adhesion, and aggregation. Importantly, real-world outcome analyses further support the therapeutic potential of CFTR modulation by ICR-001. Patients treated with the active compound for its intended use had significantly lower rates of major thromboembolic events, including ischemic stroke, myocardial infarction, arterial thrombosis, and pulmonary embolism, than matched control groups. In vivo testing in a relevant animal model demonstrated antiplatelet efficacy with a favorable bleeding profile compared to current standard-of-care therapies.
ICR-001 is currently reformulated as a once-daily, sustained-release, oral dosage form optimized for stroke patients. It is designed to improve adherence while minimizing peak-related adverse effects. The program has reached an advanced preclinical stage, and the new formulation is being prepared for evaluation in a Phase I clinical study. By combining a differentiated mechanism of action with a repurposing strategy, ICR-001 has the potential to provide a safer alternative to current antiplatelet therapies while significantly reducing development time and cost.
Commercial Opportunity
Available for licensing and cooperation
Development Status
Development of novel formulation for approved drug ongoing which shall be tested in a clinical phase 1 study.
Patent Situation
Patent applications with priority date of 15.3.2022:
European patent application EP23710069
US patent application US18/845,938
Japanese patent application JP2024554770
Canadian patent application CA3245258
Australian patent application AU2023233813
Further Reading
Asmus E, Karle W, Brack MC, Wittig C, Behrens F, Reinshagen L, Pfeiffer M, Schulz S, Mandzimba-Maloko B, Erfinanda L, Perret PL, Michalick L, Smeele PJ, Lim EHT, van den Brom CE, Vonk ABA, Kaiser T, Suttorp N, Hippenstiel S, Sander LE, Kurth F, Rauch U, Landmesser U, Haghikia A, Preissner R, Bogaard HJ, Witzenrath M, Kuebler WM, Szulcek R, Simmons S. Cystic fibrosis transmembrane conductance regulator modulators attenuate platelet activation and aggregation in blood of healthy donors and COVID-19 patients. Eur Respir J. 2023 Mar 23;61(3);2202009.
