German biopharmaceutical company Apogenix’s asunercept (APG101) has received orphan drug designation from the European Commission (EC) to treat patients with myelodysplastic syndromes (MDS).
MDS is a disorder of the bone marrow characterised by ineffective haematopoiesis (blood cell formation) and can cause severe anaemia.
Patients suffering from the condition have chances of being affected with life-threatening infections, as well as acute myeloid leukaemia.
Apogenix’s lead immuno-oncology candidate, Asunercept, is a fully human fusion protein that comprises the extracellular domain of the CD95-receptor and the Fc domain of an IgG1 antibody.
It binds to the CD95 ligand (CD95L) and blocks the activation of the CD95 receptor, of which the excessive stimulation on haematopoietic precursor cells in the bone marrow of MDS patients inhibits erythropoiesis.
This leads to the development of transfusion-dependent anaemia that is refractory to erythropoiesis-stimulating agents.
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Apogenix chief medical officer Dr Harald Fricke said: “The vast majority of patients suffering from MDS are anaemic and dependent on frequent regular blood transfusions.
“Asunercept prevents premature death of red blood cells in the bone marrow and thus reduces the need of blood transfusions, even making them superfluous in many patients.”
The therapy is currently being developed for the treatment of solid tumours and malignant haematological diseases.
The efficacy of asunercept has been assessed in an open label, single-arm Phase I clinical trial, involving 20 patients affected with a low-to-intermediate risk of MDS.
The trial demonstrated that treatment with the Apogenix’ medicine was well tolerated and resulted in a significant reduction in transfusion frequency.