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Asunercept ( INN; development codes APG101 and CAN008) [1] is a soluble CD95-Fc fusion protein which is in clinical development for the treatment of glioblastoma multiforme (GBM) and myelodysplastic syndromes (MDS). [2] Asunercept has been granted orphan drug status for the treatment of GBM and MDS in the EU and the US. [3] [4] [5] It has also received PRIME designation by the European Medicines Agency (EMA) for the treatment of GBM. [6]
Asunercept blocks the CD95–ligand ( CD95L) from binding to the CD95-receptor (CD95), which induces apoptosis. In oncology, this blockade is intended to prevent the killing of activated T cells [7] that can be induced by tumor-associated macrophages (TAMs), [8] endothelial cells, [9] or fibroblasts. [10]
In MDS, CD95L-signaling is a negative regulator of erythrocyte production in the bone marrow and its blockade has been shown to rescue erythroid progenitors. [11]
A randomized, double-blind, placebo-controlled phase I study to examine the safety and tolerability of asunercept has shown that it is well tolerated. [12] The efficacy of asunercept was tested in a phase II randomized controlled trial for patients with GBM. A total of 83 patients with first or second relapse of GBM were enrolled in the successful proof-of-concept trial. The primary goal of doubling the number of patients reaching progression-free survival at six months (PFS6) was substantially exceeded. [13]
Asunercept has also been successfully tested in a phase I trial to treat patients with MDS. [14] MDS is a disease in which the bone marrow does not make enough healthy blood cells, which leads to blood cytopenias, especially anemia. [15]
Legal status | |
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Legal status |
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Identifiers | |
CAS Number | |
UNII |
Asunercept ( INN; development codes APG101 and CAN008) [1] is a soluble CD95-Fc fusion protein which is in clinical development for the treatment of glioblastoma multiforme (GBM) and myelodysplastic syndromes (MDS). [2] Asunercept has been granted orphan drug status for the treatment of GBM and MDS in the EU and the US. [3] [4] [5] It has also received PRIME designation by the European Medicines Agency (EMA) for the treatment of GBM. [6]
Asunercept blocks the CD95–ligand ( CD95L) from binding to the CD95-receptor (CD95), which induces apoptosis. In oncology, this blockade is intended to prevent the killing of activated T cells [7] that can be induced by tumor-associated macrophages (TAMs), [8] endothelial cells, [9] or fibroblasts. [10]
In MDS, CD95L-signaling is a negative regulator of erythrocyte production in the bone marrow and its blockade has been shown to rescue erythroid progenitors. [11]
A randomized, double-blind, placebo-controlled phase I study to examine the safety and tolerability of asunercept has shown that it is well tolerated. [12] The efficacy of asunercept was tested in a phase II randomized controlled trial for patients with GBM. A total of 83 patients with first or second relapse of GBM were enrolled in the successful proof-of-concept trial. The primary goal of doubling the number of patients reaching progression-free survival at six months (PFS6) was substantially exceeded. [13]
Asunercept has also been successfully tested in a phase I trial to treat patients with MDS. [14] MDS is a disease in which the bone marrow does not make enough healthy blood cells, which leads to blood cytopenias, especially anemia. [15]