Summary: development and regulatory status as of September 2026

Tovecimig (CTX-009, ABL001, NOV1501, TR009) is investigational and holds no marketing authorization in any jurisdiction. The registrational study in second-line advanced biliary tract cancer is COMPANION-002 (NCT05506943), a randomized, open-label phase 2/3 trial of tovecimig plus paclitaxel versus paclitaxel alone that enrolled 168 participants at 34 sites in the United States and is listed as active, not recruiting, with an estimated primary completion date of 31 August 2027. The trial met its primary endpoint of objective response rate, reported in April 2025 as 17.1% versus 5.3% (p=0.031) and revised in the final analysis to 18.0% versus 5.3% (p=0.0228); median progression-free survival was 4.7 months versus 2.6 months (hazard ratio 0.44, p<0.0001), and the overall survival endpoint was not met.

The FDA granted Fast Track designation to CTX-009 in combination with paclitaxel in April 2024 for previously treated metastatic or locally advanced biliary tract cancer, and orphan drug designation for biliary tract cancer in April 2026. In its report for the second quarter of 2026, filed 6 August 2026, Compass Therapeutics stated that it expected FDA feedback on the COMPANION-002 data during the third quarter of 2026 before a biologics license application filing later in 2026. The July 2026 corporate presentation places the FDA meeting in the third quarter of 2026, the start of the biologics license application filing in late 2026, and potential approval and launch in 2027, with approval anticipated in the second half of 2027. No submission outside the United States has been announced. The complete COMPANION-002 dataset was selected for oral presentation at the 2026 European Society for Medical Oncology Congress in October 2026.

Earlier development was conducted in South Korea: a phase 1 monotherapy dose escalation and expansion study (NCT03292783), a phase 1b combination study, and a single-arm phase 2 study of tovecimig plus paclitaxel in biliary tract cancer. Investigator-sponsored trials are ongoing in first-line biliary tract cancer with gemcitabine, cisplatin and durvalumab (NCT06548412), in second-line colorectal cancer with FOLFIRI (NCT07662031), and in second-line glioblastoma with CTX-471 (NCT07392957). A company-sponsored phase 2 basket study in DLL4-positive tumors was planned for initiation in mid-2026.

Regulatory submission: BLA planned for the fourth quarter of 2026

“The Company expects feedback from the FDA this quarter regarding the COMPANION-002 Phase 2/3 data for tovecimig (DLL4 x VEGF-A bispecific antibody) in patients with biliary tract cancer (BTC), prior to a potential BLA filing later this year.” (opens the source at this quote in a new tab)
“In April 2026, the Company announced positive data from its Phase 2/3 study of tovecimig, which it plans to include in a BLA submission later this year. The company expects to receive feedback from the FDA on these data in Q3 2026.” (opens the source at this quote in a new tab)

“Q3 26: Meet with FDA Q4 26: Submit BLA” (opens the source at this quote in a new tab)
“Expected FDA meeting in Q3 2026” (opens the source at this quote in a new tab)
“BLA filing initiating in late 2026” (opens the source at this quote in a new tab)

Launch: Anticipated in 2027

“2027 potential approval and launch” (opens the source at this quote in a new tab)
“Planning to file BLA based on data with potential approval in 2H 2027” (opens the source at this quote in a new tab)
“Highly focused US BTC market; targeted launch prep underway” (opens the source at this quote in a new tab)

Current development phase: randomized phase 2/3 COMPANION-002 trial

“Tovecimig (CTX-009), our bispecific antibody targeting DLL4 and VEGF-A, is currently being evaluated in a randomized Phase 2/3 trial in the United States in combination with paclitaxel in patients with biliary tract cancer (“BTC”) who received one prior treatment regimen.” (opens the source at this quote in a new tab)

Field	Quoted record
Official title	“A Phase 2/3 Randomized, Controlled Study of CTX-009 in Combination With Paclitaxel Versus Paclitaxel Alone in Adult Patients With Unresectable Advanced, Metastatic or Recurrent Biliary Tract Cancers Who Have Received One Prior Systemic Chemotherapy Regimen”
Brief title	“A Study of CTX-009 in Combination With Paclitaxel in Adult Patients With Unresectable Advanced, Metastatic or Recurrent Biliary Tract Cancers (COMPANION-002)”
Lead sponsor	“Compass Therapeutics”
Brief summary	“This is a multi-center, open-label, randomized, phase 2/3 trial of the bispecific antibody CTX-009 plus paclitaxel versus paclitaxel in patients with previously treated, unresectable advanced or metastatic biliary tract cancers.”
Estimated primary completion	“2027-08-31”

United States: Fast Track designation

“today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation (FTD) to CTX-009, the Company’s bispecific DLL4/VEGF-A antibody, in combination with paclitaxel for the treatment of patients with metastatic or locally advanced BTC that have been previously treated.” (opens the source at this quote in a new tab)

“This orphan drug designation follows a prior regulatory decision by the FDA to grant fast track designation to tovecimig in April 2024 for the treatment of metastatic or locally advanced biliary tract cancers in the second-line setting.” (opens the source at this quote in a new tab)

United States: orphan drug designation

“ABL Bio announced Wednesday that Tovecimig (CTX-009/ABL001), an innovative bispecific antibody cancer treatment, has been granted Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA). The drug, currently in development by global partner Compass Therapeutics, targets bile duct cancer.” (opens the source at this quote in a new tab)
“Upon designation as an orphan drug, the company receives a period of market exclusivity, typically seven years, under U.S. regulations. It is also eligible for benefits such as tax credits, partial waivers of FDA review fees, clinical trial grants, and regulatory support for development.” (opens the source at this quote in a new tab)

“Tovecimig received Orphan Drug Designation in April and the Company intends to meet with the FDA in advance of its planned BLA submission later this year.” (opens the source at this quote in a new tab)

Prior clinical development completed in South Korea

“Previously, tovecimig underwent a Phase 1 dose escalation and dose expansion monotherapy trial in patients with solid tumors, a Phase 1b trial in combination with chemotherapy, and a Phase 2 trial in combination with chemotherapy in patients with advanced biliary tract cancer, all of which were completed in South Korea.” (opens the source at this quote in a new tab)
“Based on the efficacy data and following discussions with the FDA, while the trial had met the criteria to advance to Part 2, FDA recommended and we agreed to forego the second stage of the study and advance tovecimig directly to a randomized Phase 2/3 study in patients with BTC treated in the second-line setting.” (opens the source at this quote in a new tab)

Planned development beyond second-line biliary tract cancer

“Beyond BTC, we intend to expand the development of tovecimig to additional solid tumor indications with significant unmet need and a mechanistic rationale for using an angiogenic inhibitor, such as colorectal cancer (“CRC”), ovarian cancer, gastric cancer, renal cell carcinoma, and hepatocellular carcinoma.” (opens the source at this quote in a new tab)
“We are preparing for a Phase 2 basket study of tovecimig in a broader set of patients with DLL4+ cancers, potentially including gastric, ovarian, renal, hepatocellular, and colorectal cancers. We expect to initiate the study mid-2026 following a comprehensive analysis of the complete data set from the COMPANION-002 BTC trial.” (opens the source at this quote in a new tab)

Investigator-sponsored trials in progress

“The investigator sponsored trial (IST) of tovecimig in combination with the current first-line, standard-of-care regimen of gemcitabine, cisplatin, and durvalumab in patients with BTC (NCT06548412) is ongoing with expansion to additional sites expected.” (opens the source at this quote in a new tab)
“Additional ISTs of tovecimig have been initiated, including a study of tovecimig plus FOLFIRI in patients with colorectal cancer in the second line setting (NCT07662031); and a novel-novel combination study of tovecimig plus CTX-471 in patients with glioblastoma in the second line setting (NCT07392957).” (opens the source at this quote in a new tab)

Presentation of the complete dataset

“Tovecimig data from this study has also been selected for an oral presentation at the 2026 European Society for Medical Oncology (ESMO) Congress in October.” (opens the source at this quote in a new tab)

Patent term

“Patents in these patent families are generally expected to start to expire in 2033, subject to possible extension.” (opens the source at this quote in a new tab)