Imalia will present two abstracts at the 66th meeting of the American Society of Hematology:
•⁠ ⁠Title: IMA001 is a novel therapeutic molecule that reduces sickling and hemolytic anemia markers in a sickle cell disease mouse model
Format: Oral presentation
https://doi.org/10.1182/blood-2024-199612

•⁠ ⁠Title: IMA001 is a novel therapeutic molecule that improves hematological parameters during vaso-occlusion in sickle cell disease in vivo
Format: Poster
https://doi.org/10.1182/blood-2024-203501

Completion of the clinical phase of randomized, double blind, placebo controlled pilot study conducted in India to evaluate safety and efficacy of orally administered IMA001 in sickle cell disease patients.

Completion of the clinical phase of the monocentric, prospective, open label pilot study conducted in France of daily dosing of IMA001 in 12 sickle cell disease patients.

Precinical proof-of-concept demonstration of the protective effect of the Administration for 36 days of IMA001 during an acute episode of vaso-occlusion in sickle Townes HbSS mice. IMA001 reduced intravascular hemolysis and inhibited microvascular stasis in animals subjected to an acute hypoxia followed by reoxygenation (8% oxygen for 3 hours and reoxygenation at room air for 1 hour).

Imalia started a multicenter, randomized, double blind, placebo controlled pilot study in India to evaluate safety and efficacy of orally administered IMA001 in sickle cell disease patients. A total of 170 patients will be included in the study. The treatment duration is 90 days. The primary end point is to check the safety and tolerance of the orally administered IMA001. Secondary endpoints are intended to evaluate the efficacy of IMA001, in particular by evaluating hematological parameters associated with anemia and hemolysis and patient functioning and well-being through a questionaire related to physical pain, sleep, and the emotional impact of SCD.

Imalia is conducting in France a second monocentric, prospective, open label pilot study in France of daily dosing of IMA001 in 12 sickle cell disease patients with 3 months of follow-up plus 1 month post dosing.The present study is designed to evaluate, first, the safety and tolerability parameters as well as to measure the plasma and urinary residues of daily oral doses of IMA001. Secondly, the study will evaluate the impact of IMA001 on biological parametersrelated to SCD and on the quality of life of patients.

Imalia reports initial proof-of-concept data for the effect of IMA001,on the progression of markers of sickle cell disease (SCD) in the Townes HbSS experimental mouse model. IMA001administered at 185 mg/kg/day for up to 28 days, improves the critical hematological markers of SCD (e.g. hemoglobin level, hematocrit, Red blood cell (RBC) and reticulocyte counts…), reduces sickling and increases RBC half-life in the circulation. This feature on anemia alleviation has a direct physiological effect in the Townes mice, through mitigation in spleen size indicating a positive impact on stress erythropoiesis.