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Revolutionizing AML Therapy - A First-in-Class CK1 Inhibitor for Relapsed Refractory Patients

Objectif

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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Mots‑clés

Programme(s)

Thème(s)

Régime de financement

HORIZON-CSA - HORIZON Coordination and Support Actions

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Appel à propositions

(s’ouvre dans une nouvelle fenêtre) HORIZON-WIDERA-2025-02

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Coordinateur

CASINVENT PHARMA A.S.
Contribution nette de l'UE
€ 485 034,38
Adresse
KOMENSKEHO NAMESTI 220/2
602 00 Brno
Tchéquie
PME
Oui
Région
Česko Jihovýchod Jihomoravský kraj
Type d’activité
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Liens
Coût total
Aucune donnée