Genmab Receives FDA Breakthrough Therapy Designation for Rinatabart Sesutecan (Rina-S) in Advanced Endometrial Cancer.

Genmab has received FDA Breakthrough Therapy Designation for Rinatabart Sesutecan (Rina-S) in advanced endometrial cancer. The designation was granted based on data from the Phase 1/2 RAINFOL-01 trial, which showed encouraging responses in heavily pretreated endometrial cancer patients. Rina-S is being evaluated as a single-agent in the ongoing Phase 1/2 RAINFOL-01 trial and will be further evaluated in a planned Phase 3 trial.

Genmab A/S (Nasdaq: GMAB) has received a significant milestone in its quest to develop innovative cancer treatments, as the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) to Rinatabart Sesutecan (Rina-S) for the treatment of adult patients with recurrent or progressive endometrial cancer (EC) who have disease progression on or following prior treatment with a platinum-containing regimen and a PD-(L)1 therapy. The designation aims to expedite the development and review of investigational medicines for serious or life-threatening diseases, given preliminary clinical evidence of substantial improvements over existing therapies.

The Breakthrough Therapy Designation was supported by data from the Phase 1/2 RAINFOL™-01 trial, which demonstrated encouraging responses in heavily pretreated endometrial cancer patients. The trial enrolled 64 patients with advanced or recurrent EC whose disease had progressed on or after anti-PD-(L)1 and platinum-based chemotherapy. The results were presented at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting.

Rina-S is an investigational folate receptor alpha (FRα)-directed, TOPO1-inhibitor antibody-drug conjugate (ADC). It is composed of a novel human monoclonal antibody directed at folate receptor α (FRα), a novel hydrophilic protease-cleavable linker, and exatecan, a topoisomerase I inhibitor payload. The clinical trial program for Rina-S continues to expand, including trials in ovarian, endometrial, and other cancers of unmet need.

The designation underscores the potential of Rina-S as a treatment option for women diagnosed with advanced endometrial cancer, who often face a poor prognosis after progressing on standard of care treatment. Genmab remains committed to driving a strong clinical development program to redefine the treatment landscape for gynecologic cancers.

References:
[1] https://www.businesswire.com/news/home/20250825239262/en/Genmab-Receives-FDA-Breakthrough-Therapy-Designation-for-Rinatabart-Sesutecan-Rina-S-in-Advanced-Endometrial-Cancer-EC
[2] https://www.stocktitan.net/news/GMAB/genmab-receives-fda-breakthrough-therapy-designation-for-rinatabart-t9nmztar07wt.html