NEO-PV-01 is an investigational immunotherapy being developed as a personalized cancer vaccine by Neon Therapeutics. It is currently being investigated as a treatment for melanoma, lung cancer, and bladder cancer.

How NEO-PV-01 works

NEO-PV-01 is a cancer vaccine that is unique to the patient. The vaccine “educates” the immune system to recognize and kill cancer cells.

The immune system can identify and “remember” abnormal cells, such as those infected by a bacteria or virus, or those that are cancerous, based on the presence of substances that are not normally present in the body, called antigens. It produces specialized immune cells, known as T-cells, that can recognize and kill these abnormal cells based on these antigens.

Cancer is often caused by mutations in genes that lead to a slightly different protein being produced by the cells. These mutated proteins have the potential to be recognized and targeted by the immune system as a so-called “neoantigen”.

To create the personalized vaccine, Neon sequences the DNA present in the patient’s tumor and, using their proprietary RECON computational pipeline, identifies mutations that could result in the production of a tumor-specific antigen.

Based on the individual’s genetic information, Neon synthesizes up to 20 custom-designed peptides or small parts of the protein. These are injected into the patient to stimulate an immune response directed against the cancer cells producing these mutated proteins.

NEO-PV-01 is administered alongside Hiltonol (poly-ICLC), an adjuvant or “helper” therapy, to boost the immune response directed against the peptides.

NEO-PV-01 in clinical trials

Neon is currently carrying out an open-label Phase 1b clinical trial (NCT02897765) to assess the safety and efficacy of NEO-PV-01, in combination with Opdivo (nivolumab) in an estimated 90 patients with melanoma, bladder, or lung cancer. The trial also aims to assess whether NEO-PV-01 plus Opdivo will have a greater clinical benefit than Opdivo alone. The trial is currently recruiting participants at sites across the U.S. and is expected to be completed by December 2020.

Neon announced that it also will be initiating a Phase 1b clinical trial in collaboration with Apexigen to assess the safety, tolerability, and efficacy of NEO-PV-01 in combination with Apexigen’s APX005M, a therapy that aims to boost the immune response by increasing the activation of the specialized immune cells called antigen presenting cells (APCs) that are essential to the NEO-PV-01-induced immune response. Researchers hope that taking the two therapies in combination will boost the immune system’s anti-tumor activity.

