Updated
July 15th, 2019

Personalized Cancer Vaccine Targeting Neoantigens Announces Positive Study Results

Neon Therapeutics NEO-PV-01 cancer vaccine candidate may play a role in prolonging clinical benefit in combination with checkpoint inhibition

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A new study targeting neoantigens that are specific to each patient’s tumor is reporting a personal neoantigen vaccine candidate helped direct a patient’s immune system to cancer targets, which produced extended clinical benefit. 

This small study focused on Neon Therapeutics’s NEO-PV-01 personalized cancer vaccine candidate in combination with OPDIVO® (nivolumab) in patients with advanced or metastatic melanoma, smoking-associated non-small cell lung cancer (NSCLC) and bladder cancer. 

Neon’s investigational personal neoantigen vaccine, NEO-PV-01, is custom-designed and manufactured based on the unique mutational fingerprint of each individual patient.   

NEO-PV-01, which is designed to include up to 20 neoantigen-targeting peptides, is intended to generate an anti-tumor immune response directing T cells to target particular neoantigens in the patient’s tumor. 

Across all 3 distinct tumor types, this study’s results demonstrated prolonged and consistent improvements in progression-free survival (PFS) that compared favorably to that observed with checkpoint inhibitor monotherapy, based on historical benchmark data.   

At the 13.4-month median follow-up in 34 patients with metastatic melanoma, median PFS had not yet been reached. In 27 patients with metastatic NSCLC, median PFS was 5.6 months; and in 21 patients with metastatic bladder cancer, median PFS was 5.6 months.   

These top-line data, which come from 82 patients who received at least one dose of OPDIVO in the Phase 1b NT-001 trial (Intention-to-Treat analysis, or ITT), support the further development of NEO-PV-01, including randomized Phase 2 trials of NEO-PV-01 in metastatic disease settings, said the company in a press release. 

Neoantigens, which are tumor-specific antigens, are derived from random somatic mutations in tumor cells and not present in normal cells, reported a 2018 study.   

When compared to non-mutated self-antigens, neoantigens could be recognized as non-self by the host immune system and are thus attractive targets for immunotherapies, with potentially increased specificity, efficacy, and safety. 

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Which means, neoantigen-related immunotherapy is a truly personalized therapy. Therefore, identification of patient-specific immunogenic neoantigens is the first step in developing such personalized vaccines. 

“With these NT-001 results, we are observing consistent prolongation of progression-free survival across all three tumor types compared with historical checkpoint inhibitor monotherapy studies involving patients with similar baseline characteristics,” said Patrick Ott, M.D. Ph.D., Clinical Director, Melanoma Center, Center for Immuno-Oncology at the Dana-Farber Cancer Institute and a lead investigator in the NT-001 trial. 

Previously, Neon announced in April 2019, that it had completed enrollment in NT-002, its Phase 1b clinical trial evaluating NEO-PV-01 in combination with the current standard of care, KEYTRUDA® (pembrolizumab) and chemotherapy, in first-line patients with untreated advanced or metastatic NSCLC.   

Neon expects to report immune monitoring and clinical outcome data from this trial by the end of the third quarter of 2020, which will inform future randomized clinical trials.

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OPDIVO® is a registered trademark of Bristol-Myers Squibb Company. KEYTRUDA® is a registered trademark of Merck Sharp & Dohme Corp., a subsidiary of Merck & Co., Inc., Kenilworth, NJ, USA.

Neon Therapeutics is a clinical-stage immuno-oncology company and a leader in the field of neoantigen-targeted therapies, dedicated to transforming the treatment of cancer by directing the immune system towards neoantigens.