FDA has granted fast track designation to varsetatug masetecan (Varseta-M), CytomX Therapeutics' investigational antibody-drug conjugate (ADC), for the treatment of relapsed/refractory metastatic colorectal cancer, the company announced August 27, 2026.¹ According to the company, Varseta-M is a potential first-in-class ADC directed toward epithelial cell adhesion molecule (EpCAM), armed with a topoisomerase-1 inhibitor payload.¹
Key facts
- Drug: Varsetatug masetecan (Varseta-M; CX-2051; CytomX Therapeutics)
- Class: EpCAM-directed antibody-drug conjugate (PROBODY platform)
- Indication: Relapsed/refractory metastatic colorectal cancer
- Regulatory status: FDA fast track designation, August 27, 2026
- Key trial: CTMX-2051-101 (NCT06265688); phase 1
- Key data: 32% ORR (10 mg/kg), 20% ORR (8.6 mg/kg), 88% disease control rate
- Safety: Diarrhea most common AE; 10% grade 3 rate with prophylaxis
- Discovery partner: ImmunoGen (now part of AbbVie)
- Next milestone: Potential registrational trial planned for 1H 2027
- Geography: US development program
"Varseta-M was intentionally designed for patients with colorectal cancer based on the high expression of EpCAM in this cancer type," said Sean McCarthy, DPhil, chairman and CEO of CytomX Therapeutics, in a company press release.1 "Receiving [f]ast [t]rack [d]esignation is an important milestone for the program and reflects its potential to address a highly unmet medical need in patients with relapsed/refractory metastatic colorectal cancer where we continue to work towards a planned first registrational trial in the first half of 2027."
What clinical data support the designation?
Fast track designation follows earlier phase 1 data from the ongoing CTMX-2051-101 trial (NCT06265688).¹,² As of a January 16, 2026 data cutoff, 93 patients with late-line metastatic colorectal cancer had been enrolled, including 56 evaluable for efficacy in the expansion cohort. According to the company in a March 2026 announcement, Varseta-M produced a confirmed objective response rate of 32% at the 10 mg/kg dose and 20% at the 8.6 mg/kg dose, with an overall disease control rate of 88% across the expansion cohort.³ Estimated progression-free survival was 7.1 months at the 10 mg/kg dose and 6.8 months at 8.6 mg/kg.³ The most common treatment-related adverse event was diarrhea, with a grade 3 diarrhea rate of 10% among patients receiving optimized doses and prophylactic management.³
"Patients with late-stage metastatic CRC [colorectal cancer] face a poor prognosis and have very limited treatment options," said Kimmie Ng, MD, MPH, associate chief of the division of gastrointestinal oncology at Dana-Farber Cancer Institute, in the March 2026 company announcement.3 "These exciting clinical data demonstrate that Varseta-M can drive consistent and durable responses with a manageable tolerability profile in patients with heavily pretreated CRC."
How does Varseta-M work?
Varseta-M uses CytomX's PROBODY platform, in which the antibody is masked with a peptide that prevents binding to healthy tissue until it reaches the tumor microenvironment. Once activated, the antibody binds EpCAM-expressing tumor cells and releases its cytotoxic topoisomerase-1 inhibitor payload, masetecan.³ EpCAM is highly expressed in colorectal and other epithelial cancers but has historically been difficult to target directly because of its expression on normal tissue as well.¹