PARIS - Vaxon Biotech has been granted orphan drug designation for its lead compound, Vx-001, a peptide-based vaccine for the treatment of TERT-positive non-small-cell lung cancer, by the Committee for Orphan Medicinal Products of the European Medicines Agency.

Vx-001 contains hTERT572Y, a single optimized cryptic peptide that activates HLA-A2 cytotoxic T lymphocytes to target tumors expressing the telomerase antigen, which is overexpressed in many cancers, including colon, breast, prostate and lung cancer. The product completed a Phase I clinical trial in 2005 in which it was found to induce a strong, broad immune response.

Vaxon Biotech, which was founded in 2004 and operates out of the Genopole biotechnology science and business park at Evry, France, is developing therapeutic vaccines for cancer based on optimized cryptic peptides, with the emphasis on the human leukocyte antigen (HLA). The company aims to develop a pipeline of products covering major HLA super-families to treat most types of cancer.

Both Vx-001 and the company's next most advanced product, Vx-006, are designed to treat solid tumors, but both are restricted to HLA-A2 patients. Vx-001 currently is undergoing Phase II clinical trials in both NSCLC and hepatocellular cancer, while a Phase I/II trial of Vx-006 is just getting under way in the indication of prostate cancer.