GM-CSF as immunomodulator for cancer vaccine TG01 and novel RAS peptide formulations

The project has progressed according to plan and minor revisions. The obtained results are satisfactory. A production process and analytical methods for manufacturing of GM-CSF to phase III level have been established. A plan for GMP production has been made and is ready for implementation. A clinical phase I study is ongoing. The results have provided a good basis for continuing the development project.

Targovax develops the peptide based anti cancer vaccine TG01 for treatment of cancers with RAS mutations. Such mutations are present in about 25% of all cancers and especially in pancreatic cancer (80%), colorectal cancer (40%) and lung cancer (30%). TG0 1 is designed to induce and enhance immune responses (T-cells) in the body that will recognize and kill cancer cells with mutated RAS. Phase I clinical development for treatment of pancreatic cancer is ongoing and phase II will be initiated immediately af ter phase I has been completed. The peptides of TG01 are little immunogenic by themselves and granulocyte macrophage-colony stimulating factor (GM-CSF) is used as immunomodulator together with TG01 to obtain enhanced immuneresponses to TG01 treatment. Be fore phase III studies can be initiated it is necessary to establish production and documentation of GM-CSF in compliance with EU/US regulatory standards for phase III. The project will also involve development of novel immunogenic formulations of the R AS peptides. It is believed that it might be possible to optimise the treatment of cancer by using such formulation. Novel formulations of RAS peptides (TG02)with a selected immunestimmulating adjuvant will be investigated and tested in clinical phase I i n patients with lung and colorectal cancer. The goal is to create a basis for development of a second generation of RAS peptide immunotheraphy with an improved clinical profile for broader development in RAS positive cancers.