Compass Therapeutics (CMPX) 3rd Annual Guggenheim Healthcare Innovation Conference summary

Strategy and funding

• Four clinical-stage drugs; funded into 2028 for all development programs, with priorities centered on advancing programs to clinical-data inflection points.

Tovecimig in biliary tract cancer

• COMPANION-002 showed more than 3x the overall response rate and a PFS hazard ratio of 0.44; data were presented at ESMO and published online in the Journal of Clinical Oncology on Oct. 24.

• More than half of control-arm patients crossed over and 85% of patients received combination therapy; intent-to-treat OS was not different, while crossover-adjusted Two-Stage Estimation showed an HR of 0.85.

• RPSFT analysis could not be performed because its statistical assumptions were not met; the Two-Stage analysis was disclosed after the summer FDA submission.

• Safety was described as similar to VEGF-targeted agents, with somewhat more hypertension and less proteinuria, clotting, and bleeding.

• FDA specifically requested paclitaxel as the control; in the U.S., second-line BTC has varied regimens and no labeled therapy outside actionable mutations.

Regulatory path and clinical studies

• Summer FDA feedback was written only; the agency recommended a study demonstrating an OS advantage, and re-engagement is underway to seek alignment on next steps.

• Potential paths include accelerated approval based on ORR/PFS with a confirmatory study, including a possible randomized OS trial of tovecimig plus paclitaxel versus FOLFOX; cooperative-group discussions are ongoing.

• A 10,000-patient BTC meta-analysis published at the end of 2025 found PFS HR <0.61 predictive of OS benefit; FDA could also determine the data do not support approval.

• An update on the U.S. regulatory path is expected in the next several months, with a goal of providing it during calendar 2026 if possible.

• Investigator-sponsored studies include frontline BTC adding tovecimig to GemCis and durvalumab, plus gastric, colorectal, and glioblastoma studies; early frontline BTC data may inform a study, potentially with a partner.