Indication and market
Glioblastoma Multiforme (GBM)
Glioblastoma multiforme is the most frequent and aggressive brain tumour belonging to the group of gliomas. The tumour cells show a high resistance to radiation and chemotherapy. They spread and infiltrate the neighboring tissue so quickly that eradicative surgery is often rendered impossible.
Due to the diffuse infiltration into brain tissue, recurrence is often experienced within months of the initial treatment. Approximately 28 000 new cases of malignant gliomas are diagnosed in the US and EU each year (Source: US National Cancer Registry). The current standard therapy focuses on surgery, followed by radiotherapy and chemotherapy. The relative survival rate for adults diagnosed with GBM is less than 30% within one year of diagnosis, only 3% of patients live longer than five years after primary diagnosis (Source: Central Brain Tumour Registry of the United States). Therefore, there is a high unmet medical need for new treatment options for glioblastoma patients.
Further information about GBM can be found under emedicine.medscape.com
Myelodysplastic Syndrome (MDS)
Myelodysplastic syndromes (MDS) are clonal hematopoietic stem cell disorders characterised by ineffective hematopoiesis leading to blood cytopenias, especially anemia. The median age for MDS is more than 60 years. The incidence rate for MDS is about 4/10,000 per year; it is increasing to 20-50/10,000 above the age of 70. The disease is often diagnosed during routine check-ups. MDS patients suffer from a deficiency of red blood cells (anemia), feel tired and are prone to infections. In most cases, the anemia is treated with blood transfusions leading to an iron overload which might damage e.g. the liver. In parallel, the number of thrombocytes (blood platelets, responsible for coagulation) and leucocytes (white blood cells, responsible for immune defense) is decreasing. Due to this, MDS patients frequently suffer from infections and bleedings which can prove fatal.
Further information about MDS can be found at www.marrow.org
Acute Graft-versus-Host Disease (aGvHD)
Acute Graft-versus-Host-Disease is a life-threatening complication following hematopoietic stem cell transplantation or allogeneic bone marrow transplantation. In aGvHD, T lymphocytes of the transplant (“graft”) attack cells of the receiving organism (“host”) which the transplant treats as “foreign”. This disease predominantly affects the skin, liver, and gastrointestinal tract of the transplant recipient. The risk of developing an aGvHD after transplantation increases with the age of the patient along with the degree of tissue incompatibility between donor and recipient. Approximately 18,000 patients in the US and EU undergo allogeneic bone marrow transplantation each year (Source: European Bone Marrow Transplantation Survey 2006) of which, on average, every second patient develops aGvHD. Despite prophylactic immunosuppressive therapy with corticosteroids, antimetabolites or antibodies, the mortality rate within the first three years after the transplantation is about 70%. In 2006, Apogenix was granted orphan drug status in the EU for APG101 for the treatment of aGvHD. (“Orphan drug status” means that the drug are developed to treat rare diseases with an incidence of less than 5/10,000 people). This status must not be confounded with marketing approval as there are many orphan drug candidates currently in clinical trials with no marketing approval; however, the status grants longer market exclusivity and also the possibility of an accelerated approval.
