Objective
Relapsed or refractory acute myeloid leukaemia (AML) in patients after the state of the art treatment with venetoclax plus azacitidine remains a critical unmet medical need, with median survival often measured in weeks and no approved mutation-agnostic therapeutic options. The compound CI2420B discovered by CasInvent Pharma is a first-in-class, highly potent and selective inhibitor of the kinases CK1α, CK1δ and CK1ε. CI2420B efficiently inhibits both p53-dependent and p53-independent survival pathways including Wnt and other resistance-associated signalling, and demonstrates strong dose-dependent in vivo efficacy in venetoclax-resistant and TP53-altered AML models. This project aims to progress CI2420B from TRL 4 toward TRL 5/6 by validating the technology in relevant preclinical environments and preparing for first-in-human clinical trials. The work plan includes expanding efficacy testing across genetically diverse AML models, completing the PK/PD and biomarker characterisation, developing scalable CMC processes to generate technical API batches and validated analytical methods, and initiating GLP-enabling toxicology. Regulatory readiness will be advanced through EMA scientific advice, orphan designation activities, and development of an IMPD/CTA-aligned data package. Clinical positioning and Target Product Profile will be refined with input from EU and US key opinion leaders. CI2420B targets a clearly defined orphan disease segment with extremely poor outcomes and no effective standards of care. Its differentiated mechanism provides a credible route to clinical benefit both as monotherapy and in rational combinations, supporting potential for rapid adoption in specialised treatment centres. Strong international IP protection (WO2019185631) ensures FTO and partnering potential. Market validation, competitive landscaping, early pricing analogues and focused EU/US go-to-market strategy will reinforce investment readiness and position the project for future EIC-Acc.
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Keywords
Project’s keywords as indicated by the project coordinator. Not to be confused with the EuroSciVoc taxonomy (Fields of science)
Project’s keywords as indicated by the project coordinator. Not to be confused with the EuroSciVoc taxonomy (Fields of science)
Programme(s)
Multi-annual funding programmes that define the EU’s priorities for research and innovation.
Multi-annual funding programmes that define the EU’s priorities for research and innovation.
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HORIZON.4.1 - Widening participation and spreading excellence
MAIN PROGRAMME
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Topic(s)
Calls for proposals are divided into topics. A topic defines a specific subject or area for which applicants can submit proposals. The description of a topic comprises its specific scope and the expected impact of the funded project.
Calls for proposals are divided into topics. A topic defines a specific subject or area for which applicants can submit proposals. The description of a topic comprises its specific scope and the expected impact of the funded project.
Funding Scheme
Funding scheme (or “Type of Action”) inside a programme with common features. It specifies: the scope of what is funded; the reimbursement rate; specific evaluation criteria to qualify for funding; and the use of simplified forms of costs like lump sums.
Funding scheme (or “Type of Action”) inside a programme with common features. It specifies: the scope of what is funded; the reimbursement rate; specific evaluation criteria to qualify for funding; and the use of simplified forms of costs like lump sums.
HORIZON-CSA - HORIZON Coordination and Support Actions
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Call for proposal
Procedure for inviting applicants to submit project proposals, with the aim of receiving EU funding.
Procedure for inviting applicants to submit project proposals, with the aim of receiving EU funding.
(opens in new window) HORIZON-WIDERA-2025-02
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Net EU financial contribution. The sum of money that the participant receives, deducted by the EU contribution to its linked third party. It considers the distribution of the EU financial contribution between direct beneficiaries of the project and other types of participants, like third-party participants.
602 00 Brno
Czechia
The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed.
The total costs incurred by this organisation to participate in the project, including direct and indirect costs. This amount is a subset of the overall project budget.