Apogenix myelodysplastic syndrome therapy wins FDA orphan drug designation

Apogenix also announced the commencement of Apocept Phase I trial in MDS patients in Germany.

Endpoints of the open-label study include efficacy (improvement of erythropoiesis), safety, and tolerability parameters and the trial results are expected by mid-2014.

Apogenix COO/CMO Dr. Harald Fricke said Apocept works by restoring 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," Fricke added.

Apocept, which binds to the CD95 ligand and blocks the CD95 receptor activation, was shown to dose-dependently stimulate erythropoiesis in various preclinical studies.