Our Science
Pioneering research for Life-Changing SCD Therapy
Imalia’s lead product, IMA001, is an innovative small molecule featuring a novel dual mechanism of action (MoA) targeting the cellular mechanisms responsible for RBC sickling, oxidative stress, and hemolysis.
Our pre-clinical results showed a significant reduction in vaso-occlusive crises (VOCs), prolonging RBC lifespan. IMA001 is positioned as a true disease-modifying therapy, all with no significant side effect reported.
Thanks to a suitable formulation to younger and adult Sickle Cell Disease populations, IMA001 aims to ease the burden of underserved populations.
Preclinical Findings
IMA001’s preclinical development for SCD included 17 studies using the Townes mouse model, recognized as the reference animal model in this indication. By examining effects across juvenile, adult, and senior subjects, the positive results of these studies provide an unparalleled foundation to explain the mechanism of action, validate the compound’s safety and substantiate the efficacy profile.
Key preclinical findings demonstrate that IMA001 significantly reduces RBC sickling, prolongs RBC half-life, and improves anemia markers (hemoglobin, hematocrit, RBC count). It also reduces oxidative stress and regulates stress erythropoiesis. In addition, IMA001 administration to Townes mice exposed to conditions for acute episode of VOC reduced intravascular hemolysis, and attenuated microvascular stasis.