Apogenix’s APG101 demonstrates efficacy in preclinical MDS study

In preclinical studies Apogenix’s APG101 rescues the production of red blood cells (erythropoiesis) in bone marrow samples from patients with lower-risk myelodysplastic syndromes (MDS).

By inhibiting their apoptosis, APG101 increases the number of erythrocyte precursor cells ex vivo and improves their overall proliferation rate. The observational study shows that CD95 – a receptor that can induce apoptosis of cells when triggered by the CD95 ligand – is overexpressed in two thirds of patients with lower-risk MDS, and that overexpression of CD95 is predictive of a lower response rate to erythropoiesis-stimulating agents. Erythroid response to APG101 could especially be observed in samples from MDS patients with severe impairment of erythropoiesis.

Commenting on the study results, Professor Michaela Fontenay, corresponding author of the publication, from the Institut Cochin in Paris, France, said: “APG101 added to cellular assays efficiently rescued the growth of erythroid progenitors in MDS patients harbouring a profound defect of erythropoiesis, independent of the expression level of CD95 or CD95 ligand. This study provides a rationale for further clinical investigation of this potential new therapeutic option in patients with severely impaired erythropoiesis who are resistant to erythropoiesis-stimulating agents.”

“The results of this study clearly illustrate the potential of APG101 in the treatment of lower-risk MDS patients with severe impairment of erythropoiesis,” said Harald Fricke, M.D., Chief Medical Officer of Apogenix. “Since these patients generally fail to respond to erythropoiesis-stimulating agents, there are currently no treatment options available for them. In a phase I trial in transfusion-dependent low to intermediate I risk MDS patients, Apogenix has evaluated the safety, tolerability, and efficacy of APG101. We expect the results of this clinical trial in the coming months.”