Compugen (NASDAQ:CGEN) is focusing its near-term clinical strategy on a randomized ovarian cancer study of COM701 while continuing to rely on partnerships with Gilead Sciences and AstraZeneca for broader pipeline leverage, Chief Executive Officer Eran Ophir said during a Stifel investor discussion moderated by Stephen Willey, senior biotech analyst at Stifel.
Ophir described Compugen as a “pioneer in AI-based computational discovery,” saying the company has used its platform to identify immuno-oncology drug targets, including internal assets and partnered programs. He said the platform has been validated through both internal development and collaborations with Gilead and AstraZeneca.
COM701 readout expected in Q1 2027
The trial is enrolling patients without liver metastases and with low tumor burden. Ophir said the company chose this population after observing clinical benefit in later-line ovarian cancer patients, including durability and a favorable safety profile. He said Compugen believes moving earlier in treatment could improve the probability of response because patients may have a less exhausted immune system.
The randomized, blinded trial will include 40 patients receiving COM701 monotherapy and 20 receiving placebo. Ophir said the primary readout will be progression-free survival, with data expected in the first quarter of 2027. He said the trial also includes a futility analysis, and that Compugen is looking for a “clearly positive” monotherapy signal rather than a borderline result.
For the placebo arm, Ophir said Compugen expects median progression-free survival to fall in a range of 3.8 to 5.8 months based on prior studies in similar ovarian cancer settings. He estimated that excluding patients with liver metastases would likely screen out “not much more than 10%” of patients in this earlier-line population.
If the study is positive, Ophir said Compugen would first discuss a registrational strategy with the FDA. He also said the adaptive design could allow the company to explore combinations, including with antibody-drug conjugates, PARP inhibitors, bevacizumab or other agents.
Company outlines ovarian cancer market opportunity
Ophir estimated the initial target population for COM701 in second- and third-line platinum-sensitive ovarian cancer patients who have received, or are not eligible for, bevacizumab or PARP inhibitors at roughly 8,000 to 12,000 patients annually. He said the opportunity could expand to around 30,000 patients if combinations such as bevacizumab are added and the company moves into a broader second- or third-line population.
Gilead-partnered GS-0321 advances in Phase 1
Ophir also discussed GS-0321, a Gilead-partnered program currently in Phase 1 dose escalation in solid tumors. He described the program as a first-in-class approach using an antibody to harness cytokine biology for cancer treatment, rather than relying on engineered cytokines.
Compugen is leading the Phase 1 study, although the asset has been licensed to Gilead. Ophir said the trial includes dose escalation in solid tumors as monotherapy and in combination with Gilead’s PD-1 agent, along with backfill cohorts and a later dose-expansion portion in selected indications. Under the collaboration, Gilead is expected to take over Phase 2 and later development after Phase 1.
Ophir said Compugen has received $90 million to date from Gilead, including a $60 million upfront payment and $30 million after IND clearance. He said the company is eligible for an additional $758 million and low double-digit tiered royalties, though he did not provide specific details on milestone timing.
AstraZeneca’s rilvegostomig program remains a key external catalyst
Willey noted that AstraZeneca’s rilvegostomig, a TIGIT/PD-1 bispecific antibody, is now in 11 Phase 3 studies and that AstraZeneca has attached more than $5 billion in peak sales guidance to the drug. Ophir said he was not speaking on AstraZeneca’s behalf but believes the drug’s bispecific format, combination strategy and clinical trial design differentiate it from prior TIGIT programs that have disappointed in late-stage testing.
Ophir said AstraZeneca’s approach may allow rilvegostomig to function as an immuno-oncology backbone in some studies and to combine with other drugs, including antibody-drug conjugates. He said upcoming data at ASCO from studies including combinations with ENHERTU and chemotherapy could be informative for safety, combinability and efficacy compared with historical benchmarks.
Compugen recently monetized a portion of its royalties from AstraZeneca. Ophir said the company retained eligibility for mid-single-digit royalties and received $65 million upfront, with an additional $25 million tied to the next milestones and up to $195 million in total potential additional payments. He said the transaction extended Compugen’s cash runway into 2029 and allows the company to keep investing in its internal and early-stage pipeline.
Computational platform remains central to strategy
Ophir said Compugen continues to invest heavily in early discovery, with the company’s largest team working on new assets from its computational engine. He said the company’s improved financial position gives it more flexibility to decide whether to license future assets early or retain them longer to generate additional value, such as through Phase 1 data.
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.
