Compugen NASDAQ: CGEN outlined progress across its immuno-oncology pipeline and partnerships at the Needham & Company Healthcare Conference, emphasizing the company’s AI-driven target discovery platform and multiple programs now in or approaching clinical readouts.
Gil Blum, a senior analyst at Needham & Company, hosted the session with remarks and a Q&A with Compugen CEO Eran Ophir, who described the company’s strategy of using its computational discovery engine to identify new immune targets and then advance select assets into clinical development while partnering others with large pharmaceutical companies.
From computational discovery to clinical pipeline
Ophir said Compugen was founded in the 1990s and later shifted toward software and pharmaceutical collaborations, ultimately deciding about a decade ago to develop drugs internally based on its computational discoveries. He framed the company’s current approach around its “validated computational engine,” Unigen, which he said has produced targets that have advanced into clinical testing and attracted partnerships with AstraZeneca and Gilead.
Compugen’s focus is immune oncology—aiming to activate the immune system against cancer—where Ophir noted that PD-1 blockers such as Keytruda have not been effective for all patient populations, increasing interest in new mechanisms and combination approaches.
Key programs: COM701, rilvegostomig collaboration, and GS-0321
Ophir highlighted two wholly owned clinical programs:
- COM701, a “potential first-in-class” antibody targeting PVRIG.
- COM902, described as a “potential best-in-class” anti-TIGIT antibody, which is also used by AstraZeneca as part of a PD-1/TIGIT bispecific antibody, rilvegostomig.
On COM701, Ophir said PVRIG was “not known to the scientific community” when Compugen identified it computationally. He described the biology of PVRIG as distinct from TIGIT and other checkpoints, with potential relevance in “less inflamed” tumors that typically respond poorly to checkpoint therapies. He pointed specifically to ovarian cancer, stating that the pathway is “very high” in ovarian cancer.
Ophir said Compugen previously treated “last-line platinum-resistant ovarian cancer patients” and saw clinical results with COM701 both as monotherapy and in combination, with “very durable responses with excellent safety” among responders. Building from those signals, the company is now focusing on a platinum-sensitive maintenance setting in the MAIA-ovarian trial. He described subtrial one as a randomized study of COM701 monotherapy versus placebo in 60 patients who responded to chemotherapy, with a readout expected in Q1 2027. Ophir said there is “no standard of care for this patient population” and that the goal is to prolong progression-free survival by extending the response period after chemotherapy.
For AstraZeneca’s rilvegostomig, Ophir said the program is advancing across 11 phase III trials and 14 phase I or II trials in multiple indications and combinations. He argued that the bispecific format—binding PD-1 with one arm and TIGIT with the other—may be a differentiator versus prior TIGIT efforts. Citing AstraZeneca translational ex vivo work using patient tumors, he said AstraZeneca has shown cooperative binding and higher activity than PD-1 alone, and “even more active than” a PD-1/TIGIT two-drug combination in that system. He also pointed to what he characterized as a comprehensive clinical strategy, including combinations with antibody-drug conjugates such as Enhertu and Dato-DXd, and trial designs that he said appear informed by “mistakes of others.”
Compugen also discussed its IL-18 binding protein program, previously known as COM503 and now referred to as GS-0321 following the licensing agreement with Gilead. Ophir described the approach as “first-in-class,” seeking to address cytokine development challenges by “inhibiting the inhibitor” rather than administering recombinant cytokines. He said the company’s strategy leverages naturally high IL-18 in the tumor microenvironment that is blocked by IL-18 binding protein, with the goal of modulating activity “only the tumor and not the periphery,” which he said could improve the therapeutic window based on preclinical data. He said the phase I study began with the first patient dosed at the beginning of 2025, and that Compugen is running the ongoing phase I trial while Gilead will take the program into later-stage trials.
Partnership economics and cash position
Ophir detailed financial terms and positioning. For the AstraZeneca collaboration, he said Compugen has received $95 million to date and is eligible for an additional $195 million in milestones. He added that AstraZeneca had “previously predicted more than $5 billion” in peak sales for rilvegostomig and that Compugen could be eligible for “up to mid-single digits” royalties.
For the Gilead deal, Ophir said Compugen has received $90 million to date for the IL-18 binding protein program licensed at the preclinical stage, and is eligible for an additional $758 million in milestones and “up to low double digits” royalties.
Ophir also said Compugen ended 2025 with “more than $145 million” in cash and that this balance should allow the company to continue operations “without any further potential milestones” through 2029.
Q&A: IO biology, trial design, and bispecific rationale
In the Q&A, Blum asked why PD-1 inhibitors have achieved broader activity than many newer IO agents. Ophir said PD-1 responders often have baseline inflammation and T cells in the tumor microenvironment, and that patients whose tumors are not “prone to respond to IO” often do not benefit even from combinations. He noted TIGIT showed activity in randomized studies but not enough to succeed in phase III, while LAG-3 achieved approval in melanoma with a “properly designed trial.”
Blum also asked whether TIGIT programs may have failed due to study design. Ophir pointed to “Fc-active TIGIT” antibodies as a factor, arguing that safety issues and discontinuation rates could erode modest efficacy. He said factors such as drug format, combinations, and trial design are “critically important,” and noted that some AstraZeneca phase III trials include head-to-head comparisons versus Keytruda and selection by PD-L1 status.
On Compugen’s decision to study COM701 in platinum-sensitive maintenance rather than the more common path from platinum-resistant disease, Ophir said the company wanted to move earlier to patients with a less compromised immune system and lower tumor burden after chemotherapy, describing it as giving COM701 “the opportunity” to extend responses given its safety profile.
Blum asked why AstraZeneca pursued a bispecific rather than combining separate PD-1 and TIGIT agents. Ophir said cooperative binding may improve activity versus two-drug combinations and also can simplify clinical strategy, adding that safety “until now seems quite similar to PD-1 blockers alone.” He cited trial examples where rilvegostomig is used as an IO backbone in combinations, including comparisons of rilvegostomig plus an ADC versus Keytruda.
Ophir closed by reiterating Compugen’s intent to use its computational platform and financial resources to expand its pipeline while its partnered programs advance toward potential later-stage outcomes.
About Compugen NASDAQ: CGEN
Compugen Ltd. NASDAQ: CGEN is a clinical-stage therapeutic discovery company that leverages proprietary computational discovery platforms to identify novel immuno-oncology targets and biomarkers. The company combines large-scale biological datasets with machine learning algorithms to generate and validate new therapeutic and diagnostic candidates. Founded in 1993 and headquartered in Tel Aviv, Israel, Compugen also maintains a presence in the United States to support its clinical development and commercial collaborations.
Compugen's predictive discovery engine scans complex biological systems in silico to reveal previously unrecognized pathways and immune checkpoints involved in cancer progression.
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