On December 15, 2025 Amira Therapeutics ("Amira"), an innovative biotechnology company focused on improving outcomes for pediatric cancer patients, reported that its lead compound, AMI463, demonstrated significant efficacy in a preclinical murine model following oral administration, achieving a 60% reduction in tumor volume compared with untreated controls.
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In addition to the in vivo results, in vitro studies have shown that AMI463 exhibits strong synergistic activity when combined with standard chemotherapy treatments in relapsed rhabdomyosarcoma (RMS), supporting its potential as a novel therapeutic option in difficult-to-treat pediatric cancers.
Soft tissue sarcomas (STS) are a rare and heterogeneous group of malignancies arising from mesenchymal tissues and are often associated with poor prognosis and limited treatment options beyond chemotherapy. Amira is advancing AMI463 primarily for rhabdomyosarcoma, the most common soft tissue sarcoma in children. RMS has an annual incidence of approximately 500 new cases in the United States and 400 pediatric cases in Europe, and it is characterized by aberrant activation of the Hedgehog signaling pathway, primarily affecting muscular tissue and hollow organs.
AMI463 is a first-in-class inhibitor that blocks the cell adhesion molecule (CAM)-related down-regulated by oncogenes (CDON). By selectively blocking CDON, AMI463 has shown strong preclinical efficacy across RMS models, including aggressive subtypes, as well as potential activity in other sarcomas and solid tumors.
The preclinical development of AMI463 has been conducted in close collaboration with the Vall d’Hebron University Hospital Research Institute Foundation (VHIR), with whom Amira has partnered since the inception of the program. This collaboration has resulted in a family of jointly owned patents protecting the use of AMI463 across multiple indications, which have been granted in Europe, the United States, and Japan.
Amira is currently preparing a scientific advice meeting with the Spanish Agency of Medicines and Medical Devices (AEMPS) to define a regulatory pathway toward first-in-human (FIH) clinical studies. The outcome of this meeting is expected to guide the required CMC, toxicology, and non-clinical development activities, enabling the company to accelerate clinical advancement of AMI463.
(Press release, Amira Therapeutics, DEC 15, 2025, View Source [SID1234661417])