OncoC4, a late-stage biopharmaceutical company, announced that China’s National Medical Products Administration (NMPA) has granted Breakthrough Therapy Designation (BTD) to gotistobart (BNT316/ONC-392), a next-generation anti-CTLA-4 antibody, for the treatment of squamous non-small cell lung cancer (sqNSCLC) in patients who have progressed on prior immuno-oncology therapies. Developed in collaboration with BioNTech, gotistobart aims to address the urgent need for effective treatments for this aggressive lung cancer subtype, which accounts for 25-30% of all non-small cell lung cancer cases and has a five-year survival rate of just 15%.
The BTD, which expedites development and regulatory review for therapies targeting serious conditions, was based on promising safety and efficacy data from the first stage of the ongoing Phase 3 PRESERVE-003 trial. This global, two-stage, randomized trial compares gotistobart as a monotherapy against standard-of-care chemotherapy (docetaxel) in patients with metastatic sqNSCLC who have progressed after PD-(L)1 inhibitor treatment. The trial, which plans to enroll around 600 patients across 12 countries, including China and the United States, evaluates overall survival as its primary endpoint, with secondary measures including response rate, progression-free survival, and safety.
Gotistobart’s innovative design as an acid pH-sensitive monoclonal antibody aims to enhance the therapeutic index compared to existing anti-CTLA-4 therapies by preserving regulatory T-cell function, as shown in preclinical studies. The drug is also being investigated in multiple clinical trials for various tumor types, both as a standalone treatment and in combination with other agents. The program previously received Fast Track Designation from the U.S. FDA in 2022.
OncoC4, based in Rockville, Maryland, focuses on developing novel biologics for cancer and immunological diseases. Its pipeline includes other promising candidates, such as AI-081, a bispecific antibody targeting PD-1 and VEGF, and ONC-841, a first-in-class anti-SIGLEC10 antibody. Detailed data from the PRESERVE-003 trial will be presented at a medical conference in December 2025. The BTD is expected to facilitate closer collaboration with the NMPA, potentially accelerating the drug’s path to market in China.
