Precision Oncology Advances with New Clinical Data from GT Biopharma, IDEAYA, Zymeworks, and Regeneron

Biomarker-focused drug development is accelerating in oncology, with several companies reporting progress in targeted and immune-based therapies, according to PR Newswire.

GT Biopharma, Inc. has advanced its Phase 1 trial of GTB-3650, an investigational therapy for relapsed or refractory CD33-expressing blood cancers such as acute myeloid leukemia and myelodysplastic syndrome. The company moved into its third dosing cohort after safety reviews showed no issues, with early data indicating increased natural killer cell activation. GT Biopharma also plans to begin human testing of GTB-5550, a B7H3-targeting drug for solid tumors, later this year.

IDEAYA Biosciences reported positive Phase 1/2 results for its MAT2A inhibitor IDE397 in combination with Gilead’s Trodelvy for MTAP-deleted urothelial cancer. The treatment achieved a 57% overall response rate at the higher dose level with a manageable safety profile. IDEAYA expects to finalize its Phase 2 dosing plan by the end of 2025.

Zymeworks will present preliminary Phase 1 data for ZW191, an antibody-drug conjugate targeting folate receptor-α, at the AACR-NCI-EORTC Conference in October 2025. The therapy is designed to treat high-grade serous ovarian and lung adenocarcinomas.

Regeneron Pharmaceuticals received FDA approval for Libtayo (cemiplimab-rwlc) as the first immunotherapy for adjuvant treatment of cutaneous squamous cell carcinoma at high risk of recurrence following surgery and radiation. The approval was based on Phase 3 trial data showing a 68% reduction in recurrence or death compared with placebo.