China’s NMPA grants Breakthrough Therapy Designation for anti-CTLA-4 antibody candidate Gotistobart (BNT316/ONC-392)
Tuesday, October 28, 2025
OncoC4 announced that China’s National Medical Products Administration (NMPA) has granted Breakthrough Therapy Designation (BTD) for gotistobart (BNT316/ONC-392) to treat patients with squamous non-small cell lung cancer (sqNSCLC) who have progressed following prior immuno-oncology therapies. Gotistobart is a next-generation anti-CTLA-4 antibody being jointly developed by BioNTech and OncoC4.
Squamous non-small cell lung cancer accounts for around 25–30% of all NSCLC cases and is known for its aggressive nature. The five-year relative survival rate for sqNSCLC remains about 15%. Despite improvements with first-line therapies combining immune checkpoint inhibitors and chemotherapy, patients who progress after these treatments continue to face limited options, highlighting a significant unmet medical need.
Gotistobart is an advanced, acid pH-sensitive anti-CTLA-4 monoclonal antibody designed to enhance the therapeutic index by preserving CTLA-4 on regulatory T cells. Preclinical studies have shown encouraging results, supporting its clinical development as a potential new approach to CTLA-4-targeted immunotherapy.
The antibody is currently under evaluation in a two-stage, randomised Phase 3 trial named PRESERVE-003 (NCT05671510). The study compares gotistobart monotherapy with standard-of-care chemotherapy (docetaxel) in patients with metastatic sqNSCLC who have previously received PD-(L)1 inhibitor therapy. The Phase 3 trial follows positive findings from an earlier Phase 1/2 study (NCT04140526) investigating gotistobart alone and in combination with pembrolizumab in advanced solid tumours.
Gotistobart previously received Fast Track Designation from the U.S. Food and Drug Administration (FDA) in 2022. The NMPA’s Breakthrough Therapy Designation is based on preliminary data from the Stage 1 portion of the PRESERVE-003 trial, which demonstrated favourable safety and efficacy outcomes. Detailed results are expected to be presented at a medical conference in December 2025.
With this designation, the development of gotistobart will benefit from closer communication with the NMPA to support accelerated development and potential priority review upon meeting the required criteria.
Source: oncoc4.com
