Our lead candidate, AMI463, has received Orphan Drug Designation from the European Medicines Agency (EMA) and from the U.S. Food and Drug Administration (FDA) for the treatment of Soft-Tissue Sarcoma (STS). We are currently advancing its development for Rhabdomyosarcoma (RMS), a rare and challenging cancer predominantly found in soft tissues like skeletal muscle or occasionally in hollow organs, indication for which AMI463 has obtained the Rare Pediatric Disease Designation (RPDD) from the FDA. This development underscores our commitment to addressing areas of significant unmet medical need. Furthermore, we recognize the broader potential of AMI463 in treating various forms of soft-tissue sarcoma and other solid tumors in both pediatric and adult patients.
Our Pipeline
Candidate
Indication
Preclinical
Phase 1
Phase 2/3 (PoC)
NDA/MAA
AMI463
Pediatric RMS – Rhabdomyosarcoma
- Preclinical – Novel CDO inhibitor (in collaboration with Vall d’Hebron Institute of Research) – initiating IND enabling studies 25% 25%
AMI605
Pediatric Glioblastoma and CNS tumors
- Preclinical – Novel RCAN1 inhibitor – Completing in-vivo efficacy studies 22% 22%