Orphan designation granted to treatment for bone marrow disorder

The European Commission (EC) has granted orphan designation to Apogenix’s lead product candidate, asunercept (APG101), for the treatment of myelodysplastic syndromes (MDS).

MDS is a bone marrow disorder that is characterised by ineffective haematopoiesis (blood cell formation) and can lead to severe anaemia. Patients suffering from MSD can often suffer from life-threatening infections and are at risk of developing acute myeloid leukaemia.

“The vast majority of patients suffering from MDS are anaemic and dependent on frequent regular blood transfusions,” explained Dr Harald Fricke, chief medical officer of Apogenix. “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. We are highly encouraged by the data from our clinical phase I trial with asunercept in these patients and are currently preparing to initiate a clinical phase II proof-of-concept trial to further evaluate the efficacy of asunercept in MDS.”

The Phase I clinical trial evaluating the therapy was a single-arm, open label study that included 20 patients with low to intermediate risk in MDS. In this study, asunercept was well tolerated and led to a significant decrease in transfusion frequency.

Asunercept works by binding to the CD95 ligand (CD95L) and blocking activation of the CD95 receptor. Excessive stimulation of this receptor on haematopoietic precursor cells in the bone marrow of MDS patients inhibits erythropoiesis, which leads to the development of transfusion-dependent anaemia. Therefore, inhibiting the CD95 system addresses this major cause of the disorder.