Confirmed Objective Response Rates (ORR)
Strong antitumor activity with confirmed ORR of 32% at 10 mg/kg and 20% at 8.6 mg/kg in late-line metastatic CRC expansion cohorts.
Improved Progression-Free Survival (PFS)
Preliminary median PFS improved from 5.8 months (May 2025) to a range of 6.8–7.1 months (6.8 months at 8.6 mg/kg and 7.1 months at 10 mg/kg), representing ~17%–22% increase versus prior estimate.
Enrollment Growth and Trial Momentum
Total Phase I enrollment increased from 73 to 93 patients (an increase of 20 patients, ~27% growth) to support dose optimization and registrational planning.
Target Validation — High EpCAM Expression
EpCAM IHC consistently high across evaluable tumors (all H-scores >200, majority ≥250), supporting target selection and broad applicability in CRC.
Safety: Absence of Historical EpCAM and TOPOI ADC Toxicities
No evidence observed of prior EpCAM-associated severe toxicities (pancreatitis, severe liver toxicity) or interstitial lung disease seen with some TOP1 ADCs; no dose-limiting toxicities (DLTs) reported.
Meaningful Reduction in Severe Diarrhea with Dose Optimization
Implementation of adjusted ideal body-weight (AIBW) dosing plus mandatory dual prophylaxis (loperamide + budesonide) reduced grade 3 diarrhea from 29% (escalation/expansion) to 10% in early dose-optimization cohorts — a 19 percentage-point absolute reduction (~65% relative decrease versus 29%).
Durable Disease Control and Ongoing Treatment Duration
Durable responses observed with median follow-up >8 months; 16 patients remained on therapy at data cut with several on treatment >11 months.
Favorable PK Characteristics and Low Free Payload Exposure
Varseta-M PK is dose-proportional with mean half-life ~6–8 days; Varseta circulates primarily masked and unconjugated CAMP59 is low (~1%–3% of total), supporting therapeutic window and masked-PROBODY approach.
Clear Development Path and Near-Term Milestones
Company plans rapid advancement toward registrational studies (target start H1 2027), additional dose-optimization data and pivotal design details planned in H2 2026, and combination/expansion studies (bevacizumab combos, other tumor types) underway.