The US Food and Drug Administration (FDA) has granted orphan drug designation to Apogenix's lead product, Apocept (APG101) for the treatment of myelodysplastic syndromes (MDS). MDS are clonal hematopoietic stem cell disorders characterized by ineffective hematopoiesis leading to blood cytopenias, especially anaemia.

Simultaneously, Apogenix also initiated a clinical phase I trial with its lead compound Apocept in patients with MDS. The clinical trial is designed as an open-label study and is conducted in clinical centers throughout Germany. Recruitment of MDS patients for the study 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.

Apocept binds to the CD95 ligand (CD95L) and blocks the activation of the CD95 receptor. Excessive stimulation of the CD95 receptor on hematopoietic cells present in the bone marrow of MDS patients inhibits erythropoiesis. As a result, transfusion-dependent anaemia develops, which is mostly refractory to erythropoiesis-stimulating agents. Preclinical studies using hematopoietic stem cells obtained from MDS patients show that Apocept dosedependently stimulates erythropoiesis and thus may help treat anaemia.

Dr Harald Fricke, COO/CMO of Apogenix, said, "MDS is the second indication for which Apocept received orphan designation in the US. With its novel mode of action, Apocept 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 Apocept both in prolonging progression-free survival as well as overall survival, we are confident that the success story of Apocept will continue, with MDS representing the second field of application."

Apogenix, a spin-out from the German Cancer Research Center (DKFZ), is developing novel protein therapeutics for the treatment of cancer and inflammatory diseases based either on the targeted modulation of apoptosis (programmed cell death) or on blocking the growth of tumour cells.