Berlin, June 22, 2010 – The Berlin-based biotechnology company MOLOGEN AG has received approval from the responsible German health authority, the Paul-Ehrlich-Institut, to carry out a phase I/II clinical study with its innovative new MGN1601 cancer medicine. MGN1601 is a cell-based gene therapy for the treatment of advanced renal cancer.

The responsible ethics committee has also agreed to the execution of the study.

It is intended that the study will be carried out at 3 different study centers in Germany and will be led by PD Dr. Steffen Weikert. The participating study centers will be the Charité in Berlin, the University clinic in Bonn and the Hannover Medical School. According to current plans, the study is likely to start in the fourth quarter of the year, when all preparations have been completed.

The purpose of the open, non-randomized, phase I/II clinical study is to investigate the safety and efficacy of MGN1601. It should include a total of 24 patients suffering from advanced renal cancer.

Dr. Matthias Schroff, CEO of MOLOGEN AG, comments: “We are delighted that we will soon be able to start the clinical study with this innovative therapy. We are putting a lot of our hopes into our new approach to the battle with cancer. Following the start of the clinical study with MGN1703, our medication for colorectal cancer, this represents another very significant milestone for us which we have already reached this year."

 

About the clinical study with MGN1601

The purpose of this phase I/II clinical study (ASET study) is to investigate the safety and efficacy of MGN1601. This will be done at several oncological clinics in Germany. 24 patients who are suffering from advanced renal cancer and with whom the standard treatment has proved to be unsuccessful are to be included in this open, single-arm, non-randomized, multi-center, clinical proof-of-principle study. In the course of the first treatment phase of the study, the patients will be treated with MGN1601 several times over a period of 12 weeks. Patients who respond to the treatment will then receive further treatment at specific intervals in an extension to the course of therapy and the effects will be monitored.

The primary aim of the study is to collect safety and tolerability data about the drug. In addition, efficacy data, including clinical, immunological and radiological parameters of the patients, will also be collected.

The safety data and the preliminary efficacy data about MGN1601 will already be available after the first treatment phase is completed. An application for the phase II clinical study required for drug approval is to be submitted once these data have been presented.

Additional data about the safety and efficacy of MGN1601 when administered for a longer period of time are to be obtained during the extended treatment period.

 

About MGN1601

The process developed by MOLOGEN to treat renal cancer with cell-based gene therapy involves therapeutic vaccination to combat advanced tumors and to prevent reoccurrence (recidivation) of them after an operation and medical treatment.

Human renal cancer cells that have been obtained from a renal tumor and are available in a standardized and characterized cell bank (master cell bank) form the basis for the vaccination. MOLOGEN itself has established a unique renal cancer cell line for this purpose. Cells that are derived from a single person, are then established as a cell line and used with other patients are known as allogeneic cells.

The principle by which the cell-based gene therapy works is to induce a cross reaction of the immune system against the patients’ own cancer cells, after the immune system has learned what cancer cells are typically look like, via the reaction against the allogeneic cancer cells. In order to achieve this efficiently, the allogeneic cancer cells are “genetically modified” with additional genetic information and are combined with MOLOGEN’s own DNA immunomodulator dSLIM® as an adjuvant to maximize the effect of the vaccination, before they are finally injected into the patient. The genetic modification is done with the help of the DNA-based MIDGE® vectors that have also been developed by MOLOGEN.

The cell-based gene therapy for the treatment of renal cancer has been granted orphan drug status by the European Medicines Agency (EMA). This enables the product to be marketed exclusively within the European Union for ten years. Additionally, the company receives comprehensive advice and support from the EMA during the approval procedure at reduced cost. The purpose of the European Union’s orphan drug program is to promote the development of medicines for rare and severe illnesses.

 

About renal cancer

Renal cancer is the most frequent malignant tumor of the kidney. About 209 000 new cases are registered all over the world every year (European Society for Medical Oncology, 2009). 15 000 patients suffer from the disease in Germany alone (Robert Koch Institute and Society of the Epidemiological Cancer Registers in Germany, 2008). About 30% of all patients already have remote metastases on initial diagnosis, which reduces the success of treatment substantially.

The tumor is well known for the fact that it does not respond to radiotherapy or chemotherapy. Although the specialized and targeted drugs recently developed and approved for this indication can improve the treatment results, they are not able to cure the disease and are often accompanied by significant side-effects. So there continues to be a tremendous medical need for new and effective drugs for the treatment of renal cancer that minimize the side-effects.