APEIRON Biologics AG ("Apeiron") announced today that it has entered into an exclusive license agreement with Columbia University through their technology transfer office, Columbia Technology Ventures. Under the terms of the agreement, Apeiron obtains worldwide exclusive rights to develop and commercialize active cellular immunotherapies under the licensed patents, which cover the inhibition of Cbl-b in T cells for the treatment of cancer.
Cbl-b is a central checkpoint for limiting immune responses. Inhibition of Cbl-b is a new strategy for checkpoint blockade, designed to activate the immune system against cancer. T cells that are deficient in Cbl-b are more strongly activated, produce enhanced levels of cytokines and proliferate longer. Hence, Cbl-b knockout mice spontaneously reject various tumors. Apeiron's project APN401 is based on the collection, stimulation (by Cbl-b inhibition) and subsequent re-administration of a patient's immune cells. A phase I clinical trial studying safety, tolerability and optimal dose of APN401 is ongoing at the Wake Forest Baptist Medical Center in North Carolina, USA.
Dr. Hans Loibner, Chief Executive Officer of Apeiron Biologics, commented: "This license agreement represents an expansion of our intellectual property portfolio for the transient inhibition of Cbl-b in a patient's immune cells, such as T cells and NK cells, for the treatment of cancer and is an important step forward in the development of a cancer immunotherapy based on the Cbl-b gene."
APN401 is an individual adoptive cell therapy in which human peripheral blood mononuclear cells are silenced ex-vivo for Cbl-b (an E3 ubiquitin ligase) using small interfering ribonucleic acid (siRNA), thereby activating them to fight cancer more efficiently. They are subsequently re-administered to the patient, with the entire procedure taking place on the bedside in one day, avoiding any additional handling and shipment.
About APEIRON Biologics AG (as of February 2016) Apeiron is a private biotech company based in Vienna, Austria, founded by Josef Penninger, and developing immunological therapies against cancer. Its lead project, APN311 (ch14.18/CHO), is a chimeric monoclonal antibody against the GD2 antigen expressed on neuroblastoma and other tumors. The project has recently been submitted for marketing authorization in the EU. Apeiron's project APN301 is an anti-GD2 antibody-IL2 fusion protein (immunocytokine) which is currently in phase II in the US and Canada in neuroblastoma and is also clinically investigated in melanoma. Furthermore, a broad program is pursued to develop therapies to selectively boost the immune system via checkpoint blockade to fight cancer: besides APN411, an early-stage project which aims for development of low molecular weight compounds to boost immune cells via novel checkpoint blockade mechanisms (in collaboration with Evotec and Sanofi), Apeiron pursues APN401, a clinical stage project currently in a phase I trial in the U.S. based on the collection, stimulation (by ex-vivo silencing of the checkpoint gene Cbl-b) and subsequent re-administration of a patient’s immune cells.