Amira Therapeutics’ Lead Compound AMI463 Demonstrates 60% Tumor Reduction in Preclinical Oral Study
- AMI463 has been tested in a murine model showing a tumor reduction of 60% compared with the control group.
- Amira is currently preparing a meeting with the AEMPS to agree a regulatory pathway to reach FIH.
- Amira is associated with the Fundació Mutua General de Catalunya (MGC) and collaborates closely with the Vall d’Hebron University Hospital Research Institute Foundation (VHIR) in the development of this compound.
Amira Therapeutics (“Amira”), an innovative biotechnology company focused on improving outcomes for pediatric cancer patients, today announced 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.
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.
About Amira Therapeutics
Amira Therapeutics is a preclinical-stage biotechnology company developing innovative therapies for pediatric cancer. The company is advancing two lead programs, AMI463 and AMI605, with a focus on addressing high unmet medical needs in rare and aggressive childhood malignancies. A spin-off of the Leitat Technological Center, Amira is headquartered in Barcelona, Spain, and operates under a fast development model through strategic collaborations with leading European research institutions, including VHIR.
Amira Therapeutics is also deeply committed to collaborative efforts with society in general, as exemplified in our partnership with the Fundació Mutua General de Catalunya (MGC), a non-profit foundation that shares our dedication to combating pediatric cancer.
Amira is also committed to societal collaboration, exemplified by its partnership with the Fundació Mutua General de Catalunya (MGC), a non-profit organization that shares our dedication to combating pediatric cancer.
About Leitat:
Founded in 1906, Leitat is one of the reference entities at national and European level in technological management. It has a team of more than 500 professionals, experts in applied research, technical services and management of technological initiatives and innovation. Leitat provides social, industrial, economic and sustainable value, offering comprehensive solutions in multiple sectors and areas: development of new materials, eco-sustainable production, occupational health prevention systems, revaluation of waste and utilization of natural resources, interconnectivity and digitization of the industry, green energy and maximizing energy efficiency. Leitat is recognized by the Spanish Ministry of Economy, Industry and Competitiveness and is one of the main start-up entities in the European Union’s Horizon 2020 program.