Myleodysplastic syndromes (MDS) affects haematopoiesis within its sufferers, this leads to severe anaemia. Currently the way that the majority of patients are treated is to receive a blood transfusion.
Asunercept, currently in development as a immuno-oncology for solid tumours and glioblastomas, was seen to interact with the same pathway which inhibits erythropoiesis within MDS patients. Following a phase II trial in glioblastoma the drug underwent a phase I trial in MDS showing a significant reduction in transfusion frequency.
This trial was only carried out in 20 patients with a low-to-intermediate risk of MDS however these results are very positive. The drug was well tolerated up to 20mg/kg of body weight and will be looking to engage in further trials in the future.
German biopharmaceutical company Apogenix’s asunercept (APG101) has received orphan drug designation from the European Commission (EC) to treat patients with myelodysplastic syndromes (MDS).