GDFATHER-NEO Reports Bladder Cancer Response Findings

The exploratory GDFATHER-NEO bladder cancer study recorded higher response proportions with visugromab plus nivolumab than with placebo plus nivolumab, according to the presentation account. Results varied between local and blinded central assessments, and the study design did not include formal hypothesis testing.

GDFATHER-NEO (NCT06059547) evaluated treatment before surgery in a phase 2a study. Participants had newly diagnosed muscle-invasive bladder cancer, had not previously received immune checkpoint inhibitors, and either could not receive cisplatin-based chemotherapy or declined it. Stratified sequential allocation assigned 16 participants to the visugromab combination and 15 to the placebo combination.

Local assessment recorded pathological complete response in 33.3% of evaluable participants receiving visugromab, or five of 15, compared with 7.7%, or one of 13, receiving placebo. The report defined that outcome as a bladder specimen without residual tumor and no lymph-node involvement. Blinded central assessment recorded the same outcome in seven of 15 participants (46.7%) in the visugromab group and two of 12 (16.7%) in the placebo group.

For major pathological response, defined as disease at stage ypT1 or below without involved nodes, local assessment recorded nine of 15 participants (60.0%) in the visugromab group and three of 13 (23.1%) in the placebo group.

Imaging assessments also differed by reviewer. Local review recorded objective responses in nine of 15 participants (60.0%) receiving visugromab and two of 13 (15.4%) receiving placebo; complete radiological responses numbered seven and zero, respectively. Central reviewers recorded objective radiological responses in eight of 16 participants (50.0%) in the visugromab group and four of 12 (33.3%) in the placebo group.

The analysis used a July 15, 2026, data cutoff, with median follow-up of 15.7 months. According to the presentation account, no recurrence had been observed during that follow-up among participants with pathological complete response. Four such participants receiving visugromab retained their bladders following repeat transurethral resection alone. The account stated that the small number of patients and limited observation period did not establish whether omitting cystectomy was safe over the long term.

Treatment comprised three cycles at four-week intervals, with intravenous nivolumab at 480 mg in both groups and either visugromab at 20 mg/kg or placebo. CT restaging followed treatment. Radical cystectomy or repeat transurethral resection of the bladder tumor was scheduled for four to eight weeks after the final dose.

According to investigators, there were no new safety signals, and severe treatment-related and immune-related adverse events were similar across the groups. The presentation account supplied no numerical rates for those events.

Andrea Necchi presented the updated findings on October 24, 2026, at the ESMO Congress in Madrid. Nature Medicine published the study online that day, according to an OncoDaily account relaying the trial investigator’s publication post.

Visugromab is a monoclonal antibody that neutralizes growth differentiation factor 15, or GDF-15. CatalYm describes that protein as a factor produced by tumors that participates in local immune suppression.

* This article is based on publicly available information at the time of writing.