We have assembled a team of world experts in cancer immunotherapy to develop therapies that target abnormal proteins called neoantigens, which are produced by cancer cells. Our programs are designed to leverage multiple facets of the immune response to elicit potent immune attack of tumors, with the aim of providing durable response to patients in need.
At Neon we are committed to advancing the field of cancer immunotherapy by investing in research in neoantigen biology, epitope prediction, cancer bioinformatics, personalized therapies and advanced immune profiling of cancer patients.
Headquartered in Cambridge, MA, Neon Therapeutics is a private company launched in 2015. The company has raised $125 million in venture financing to date from investors Third Rock Ventures, Clal Biotechnology Industries, Access Industries, Partner Fund, Fidelity, Wellington, Inbio Ventures, Nextech Invest, and other investors.
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Using Mono-allelic Mass Spectrometry Data to Improve Epitope Prediction Algorithms Published in Immunity
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Neon Therapeutics Secures $70 Million in Series B Financing
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Neoantigens arise as a result of accumulating somatic mutations and foreign sources such as viruses. These alterations in a patient’s tumor DNA occur during the development and progression of tumors. These neoantigens are inherently non-self, thus are seen as foreign by the immune system. Mounting evidence suggests that immune rejection of tumors, for example that which is seen with checkpoint modulators, may be mediated by recognition of neoantigens.
Neon Therapeutics is building a product engine poised to take full advantage of neoantigen biology. We are developing multiple programs in therapeutic vaccines and T cell modalities, targeting both neoantigens that are specific to individual patients as well as neoantigens that are shared across patients and tumor types. By taking this multimodality approach, Neon Therapeutics hopes to easily integrate with standard-of-care therapies as well as with complementary experimental immunotherapies to dramatically improve the patient response to treatment.
With an aim to generate truly novel immunotherapies, Neon is building end-to-end capabilities that includes such areas as tumor cell DNA and RNA sequencing, advanced algorithms for neoantigen prediction, as well as in vitro and in vivo immune stimulation and advanced immune monitoring approaches.
Neon’s focus is to bring truly personalized medicine to patients. Cutting edge genomic technologies and immune profiling tools are core to Neon’s therapeutic approach. High-throughput Next Generation Sequencing (NGS) and sophisticated bioinformatics approaches to data analysis allow for full characterization of the genetic aberrations and their abundance in an individual patient’s tumor. This is a molecular fingerprint that defines how each patient’s disease is unique. State-of-the-art computational approaches convert the massive datasets from exome sequencing and distill the key pieces of information that support the generation of personalized therapeutics. In effect, computational methods find the ‘needles in the haystack’ that define the therapeutic strategy for each individual.
This paradigm-changing application of NGS and bioinformatic techniques, coupled with an advanced toolbox of immune monitoring and profiling technologies, promotes further understanding of how and why a patient responds to therapy. The insights gleaned from this work can help us to tailor the right therapy for the right patient in addition to providing scientific insights into the fundamentals of tumor biology and tumor immunology.
Our lead program, NEO-PV-01, is a personalized neoantigen vaccine that builds upon initial clinical trials developed collaboratively by the Broad Institute and Dana-Farber Cancer Institute. In 2016, Neon initiated its first clinical study to evaluate NEO-PV-01 combined with Opdivo (nivolumab), a PD-1 immune checkpoint inhibitor from Bristol-Myers Squibb, in patients with measurable metastatic melanoma, non-small cell lung cancer and bladder cancer. Dana-Farber is also currently enrolling two investigator-initiated trials studying the personalized neoantigen vaccine in melanoma and glioblastoma.
NEO-PV-01 utilizes a proprietary epitope computational engine called RECON (Real-time Epitope Computation for Oncology). RECON ensures accurate mutation calling, and uses unique tools and data to ensure the most immunogenic epitopes are selected for ultimate manufacturing. The RECON bioinformatics engine contains machine-learning algorithms which will continue to evolve as Neon Therapeutics generates additional immunological and clinical data from its programs, and utilizes high-throughput mono-allelic mass spectrometry to ensure binding motifs use unbiased datasets.
NEO-PTC-01 leverages much of the personalized pipeline built for NEO-PV-01. As such, much of the tools and know-how developed around tumor immunology, sequencing and RECON bioinformatics. Unlike NEO-PV-01, where the immunogens are used directly for in vivo vaccination, NEO-PTC-01 uses these immunogens in co-culture with T cells and monocyte-derived dendritic cells from patients in order to ex vivo stimulate autologous T cells to respond against neoantigen targets. Key technical advances to this process have been made that will be important for GMP manufacturing. Using neoantigens for T cell therapies may allow this approach to be tumor-specific and minimize toxicities that have been problematic for prior T cell approaches tackling solid tumors.
The vast majority of tumor mutations are unique to the patient. However, a small subset of neoantigens are common or ‘shared’ across patients and tumor types. The Broad Institute and subsequently Neon Therapeutics undertook a systematic, comprehensive analysis of high quality, cross-validated data to identify relevant shared neoantigens that could be utilized in both tumor-specific and pan-tumor indications. Neon Therapeutics is now undergoing a validation process, looking at key biochemical and immunological properties, for the first wave of these putative targets.
Shared neoantigen targets have the potential to be utilized in several product formats such as off-the-shelf vaccines, antibody approaches and TCR-based T cell therapies.