APG101 is a soluble CD95-Fc fusion protein which is in development for the treatment of Glioblastoma multiforme (GBM) and myelodysplastic syndromes (MDS). APG101 blocks the CD95–ligand (CD95L) from binding to the CD95-receptor (CD95). A randomized,double-blind, placebo-controlled phase I study to examine the safety and tolerability of APG101 has shown that it is well tolerated. The efficacy of APG101was tested in a randomized controlled phase II trial with patients suffering from GBM, the most aggressive brain tumor. The study was initiated at the end of 2009 and first results were released in 2012. A total of 83 patients with first or second relapse of GBM were enrolled in the successful trial. The primary goal of doubling the number of patients reaching progression-free survival at six months (PFS6) was substantially exceeded.
APG101 constitutes an innovative approach to treating GBM. The therapeutic aim is to increase both progression-free as well as overall survival of patients with GBM. The therapeutic approach is based on research conducted by the German Cancer Research Center (DKFZ). As demonstrated by Kleber et al.,the binding of the CD95 ligand to its cognate receptor stimulates the invasive growth of glioblastoma cells. Thus, the inhibition of this interaction through APG101 reduces tumor cell growth and migration. An article published in the scientific journal Nature by Prof. Marcus Peter and his team at the University of Chicago provides additional evidence for APG101’s principle of inhibiting the CD95/CD95L system in the treatment of tumors.
APG101 is in a phase I trial to treat patients with MDS. MDS is a disease in which the bone marrow does not make enough healthy blood cells, which leads to blood cytopenias, especially anemia.
APG101 has been granted orphan drug status for the treatment of GBM in the EU and the US and for the treatment of MDS in the US.