VAC2 is an allogeneic (non-patient-specific or “off-the-shelf”) cancer immunotherapy of antigen-presenting dendritic cells that is currently in a Phase I clinical trial in non-small cell lung cancer (“NSCLC”).  The VAC2 trial is being funded and conducted by Cancer Research UK (“CRUK”), the world’s largest independent cancer research charity.

To provide a more effective and targeted cancer treatment, we are developing VAC2 as an allogeneic, or non-patient specific, cancer vaccine candidate designed to stimulate patient immune responses to telomerase which is commonly expressed in cancerous cells but not in normal adult cells. VAC2, which is produced from our pluripotent cell technology using a directed differentiation method, is comprised of a population of mature dendritic cells.  As the most potent type of antigen presenting cell in the body, dendritic cells instruct our body’s immune system to attack and eliminate harmful pathogens and unwanted cells. To target cancerous cells, VAC2 is engineered to express the tumor-selective antigen telomerase, which is found in over 85% of all cancers. Because the tumor antigen is loaded exogenously into the dendritic cells prior to administration, VAC2 is a platform technology that can be modified to carry any antigen, including patient-specific tumor neo-antigens.

Market Opportunity

Cancer, which afflicts millions worldwide, is one the largest unmet clinical needs with current treatment options providing limited efficacy and a wide range of debilitating side effects. The use of pluripotent cells as the starting material for VAC2 production adds several additional advantages to this candidate therapeutic.  In comparison to technologies that rely on the use of a patient’s own blood, our pluripotent cell technology provides a scalable system for production of a large number of vaccine doses in a single lot, lower manufacturing costs, greater product consistency, and off-the-shelf availability to provide broader access to patients. In addition, we believe that as an allogeneic therapy, VAC2 has the potential to stimulate a more robust immune response through an adjuvant effect resulting from the partial immune mismatch between the VAC2 cells and patients receiving the therapy.

Development Status

In September 2014, clinical development of VAC2 was initiated by CRUK in non-small cell lung cancer. Upon completion of the CRUK Phase 1 study, we will have an exclusive first option to acquire the data generated in the trial and conduct further development of VAC2.  Results from the study are expected during 2019 and 2020.