Apogenix Receives FDA Orphan Drug Designation for APG101 to Treat Myelodysplastic Syndromes and Initiates Clinical Phase I Study


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Apogenix GmbH, a biopharmaceutical company developing novel protein therapeutics for the treatment of cancer and inflammatory diseases, announced that its lead compound, APG101 (Apocept), has been granted orphan drug designation from the FDA for the treatment of myelodysplastic syndromes (MDS). 

Dr. Harald Fricke, COO/CMO of Apogenix, commented, “MDS is the second indication for which [APG101] received orphan designation in the United States. With its novel mode of action, [APG101] restores the causal impairment of erythropoiesis in MDS. After the successful proof of concept in a randomized controlled trial in glioblastoma demonstrating excellent efficacy of [APG101] both in prolonging progression-free survival as well as overall survival, we are confident that the success story of [this agent] will continue….”

Phase I Trial

Simultaneously, Apogenix announced the initiation of a clinical phase I trial of APG101 in patients with MDS; the clinical trial is designed as an open-label study and is conducted in clinical centers throughout Germany. Recruitment of subjects began in January 2013. Endpoints of the study include efficacy (improvement of erythropoiesis), safety, and tolerability parameters. Results of the trial are expected by mid-2014.

APG101 binds to the CD95 ligand and blocks the activation of the CD95 receptor. Excessive stimulation of the CD95 receptor on hematopoietic cells present in the bone marrow of patients with MDS inhibits erythropoiesis. As a result, transfusion-dependent anemia develops, which is mostly refractory to erythropoiesis-stimulating agents. Preclinical studies using hematopoietic stem cells obtained from patients MDS show that APG101 dose-dependently stimulates erythropoiesis and thus may help treat anemia. ■



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