Compugen Ltd. is a clinical-stage therapeutic discovery and development company creating new cancer immunotherapies. The company focuses on identifying new drug targets and biological pathways to expand treatment options for patients who do not benefit from existing therapies.
The Computational Discovery Platform
Compugen developed a proprietary computational discovery platform, Unigen, which uses artificial intelligence and machine learning to identify new drug targets. The system processes a wide variety of biological data, including multi-omics and single-cell RNA sequencing. The platform covers the entire cycle from hypothesis and data collection to target discovery and clinical validation.
By computationally analyzing diverse biological data across numerous tumor types, Unigen builds proprietary datasets. This process allows for the predictive identification of previously unknown immune regulatory proteins that can serve as targets for new therapies.
A feature of the platform is its ability to learn and improve from the company’s own research. Outcomes from preclinical and clinical studies are fed back into the system’s knowledge base. This enrichment of the database provides new insights for future hypotheses and refines the platform’s predictive accuracy.
Compugen has collaborated with Ultima Genomics, using high-throughput single-cell sequencing to gain insights into gene structure and regulation within tumors. By developing algorithms to study gene splicing at the single-cell level, Compugen is building proprietary single-cell atlases.
Key Therapeutic Pipeline
The company’s lead assets from the Unigen platform include COM701, an antibody targeting the immune checkpoint PVRIG, and COM902, an antibody targeting TIGIT. PVRIG and TIGIT are proteins on immune cells that act as “off switches,” preventing the immune system from attacking cancer. By blocking these proteins, COM701 and COM902 are designed to reactivate the immune response against tumors.
COM701 is a potential first-in-class antibody, as PVRIG was a previously unknown immune checkpoint discovered by Compugen’s platform. The development of antibodies against both PVRIG and TIGIT is based on the observation that they are part of a network of inhibitory pathways that tumors exploit. This network often includes the PD-1 pathway, a target of many existing immunotherapy drugs.
Compugen is exploring dual and triple blockade strategies, investigating the combination of COM701 with COM902 and a PD-1 inhibitor. The hypothesis is that blocking all three pathways (PVRIG, TIGIT, and PD-1) could produce a more robust anti-tumor immune response. Another candidate is rilvegostomig, a bispecific antibody targeting both PD-1 and TIGIT.
The company also has an antibody, COM503, that targets the IL-18 binding protein (IL-18BP). Blocking IL-18BP is another strategy to enhance the immune system’s activity against cancer. The pipeline also includes undisclosed programs in earlier stages of development.
Strategic Collaborations and Licensing
Compugen forms partnerships with large pharmaceutical companies to advance its pipeline. These collaborations provide financial resources and leverage the development expertise of its partners. This model allows Compugen to focus on computational discovery while mitigating the costs and risks of late-stage clinical development.
A partnership with AstraZeneca focuses on the development of rilvegostomig, the bispecific PD-1/TIGIT antibody. The agreement has a potential value of $200 million, with Compugen eligible for milestone payments and royalties on sales.
The company also has a collaboration with Gilead Sciences focused on a different set of targets. This partnership has a potential value of up to $850 million, including upfront payments, milestone opportunities, and tiered royalties. These agreements provide external validation for the targets identified by the Unigen platform.
As part of its clinical development strategy, Compugen is leading a Phase 1 trial with Gilead for a candidate designated GS-0321, focused on solid tumors. Such co-development structures allow Compugen to retain involvement in the clinical progress of its discoveries.
Clinical Trial Progress and Data
Compugen’s therapeutic candidates are being evaluated in multiple clinical trials. The company’s proprietary candidates, COM701 and COM902, are in Phase 1 development for solid tumors. These early-stage trials determine appropriate dosage and look for initial signs of anti-cancer activity, both as single agents and in combination.
A focus of the clinical strategy is combining COM701 with other checkpoint inhibitors. Studies are evaluating COM701 with a PD-1 inhibitor and in a triple-blockade regimen with a PD-1 inhibitor and COM902. The COM701 program is targeting indications like ovarian cancer, with interim data anticipated in 2026 to validate the PVRIG pathway’s role in overcoming resistance.
Partnered programs are also in clinical trials. The TIGIT program with AstraZeneca, rilvegostomig, has progressed to Phase 3 trials for lung and gastrointestinal cancers, with early data expected in 2025. The collaboration with Gilead has led to a Phase 1 trial for GS-0321 in solid tumors, with plans to expand based on initial results.
Compugen presents findings from its research at scientific conferences. The company has shared computational research on predicting immune evasion and resistance in specific cancer subtypes. These presentations highlight the work to identify biomarkers that could help select patients most likely to respond to its therapies.