China's NMPA Grants Breakthrough Therapy Designation for Anti-CTLA-4 Antibody Candidate Gotistobart (BNT316/ONC-392)
Designation granted for gotistobart (BNT316/ONC392) for the treatment of patients with squamous non-small cell lung cancer (sqNSCLC), an aggressive subtype of lung cancer
Breakthrough Therapy designation will allow for an expedited development and regulatory review of gotistobart in China
Global pivotal PRESERVE-003 Phase 3 clinical trial in squamous non-small cell lung cancer Is Ongoing
ROCKVILLE, Md., Oct. 27, 2025 /PRNewswire/ -- OncoC4 announced today that China's National Medical Product Administration (NMPA) has granted Breakthrough Therapy Designation (BTD) for gotistobart (BNT316/ONC392) for the treatment of patients with squamous non-small cell lung cancer (sqNSCLC) who have progressed on prior standard immuno-oncology therapies (IO). Gotistobart is a next-generation anti-CTLA-4 antibody candidate which is being jointly developed by BioNTech and OncoC4.
Squamous non-small cell lung cancer (sqNSCLC) is an aggressive subtype of lung cancer accounting for approximately 25-30% of all NSCLC cases, the most common type of lung cancer1. The 5-year relative survival rate for patients with sqNSCLC is 15%2. While novel first line treatments combining immune-checkpoint inhibition and chemotherapy have improved outcomes of patients with NSCLC, the medical need remains high for novel treatments options, especially for patients with lung cancer that have progressed on first-line therapy and have limited treatment options3.
"For the past 5 years, limited advances in treatment outcomes have been reported for EGFR wild-type NSCLC patients who progressed on the first-line IO and chemotherapies. We believe gotistobart has the potential to broaden the reach of CTLA-4-targeting immunotherapy and to improve outcomes for patients living with sqNSCLC." said Pan Zheng, MD, PhD, Co-founder and Chief Medical Officer of OncoC4.
"Gotistobart is a next-gen acid pH-sensitive anti-CTLA4 mAb that conferred an improved therapeutic index compared with approved anti-CTLA-4 antibodies by preserving CTLA-4 on regulatory T cells as demonstrated in preclinical models. The emerging data support clinical proof of concept for this new approach", added Yang Liu, PhD, Co-Founder, Chief Executive Officer, and Chief Scientific Officer of OncoC4.
Gotistobart is currently being evaluated in a registrational two-stage randomized Phase 3 trial, PRESERVE-003 (NCT05671510). The clinical trial assesses the efficacy and safety of gotistobart as monotherapy compared to the standard-of-care chemotherapy (docetaxel) in patients with metastatic sqNSCLC that progressed under previous PD-(L)1-inhibitor treatment. The initiation of the Phase 3 trial was based on positive safety and efficacy data from an ongoing Phase 1/2 trial (NCT04140526) with gotistobart alone and in combination with pembrolizumab in patients with advanced solid tumors. The program received Fast Track Designation from the U.S. Food and Drug Administration (FDA) in 2022 and represents an innovative approach which aims to leverage the full potential of CTLA-4-targeting therapies.
Breakthrough Therapy Designation is an NMPA program designed to expedite the development and regulatory review of investigational therapies that are designed to address serious or life-threatening conditions. To receive the designation, the candidate needs to demonstrate preliminary clinical evidence that indicates that it may offer substantial improvement over existing therapies on one or more clinically significant endpoints. The NMPA's BTD designation is based on the Stage 1 safety and efficacy data of the PRESERVE-003 trial. The detailed data will be reported in an upcoming medical conference in December 2025. With the Breakthrough Therapy designation, the development of gotistobart may benefit from more frequent engagement with the NMPA, which will support the collection of appropriate data needed to accelerate development and may also allow for priority review if the relevant criteria are met.
About PRESERVE-003
The two-stage, open-label, randomized Phase 3 trial, PRESERVE-003 (NCT05671510), will assess the efficacy and safety of gotistobart (BNT316/ONC-392) as monotherapy compared to the standard-of-care chemotherapy (docetaxel) in patients with metastatic NSCLC that progressed under previous PD-(L)1-inhibitor treatment. Stage 1, the non-pivotal dose-confirmation stage, assessed the efficacy and safety of two gotistobart dosing regimens in comparison to docetaxel in patients with squamous and non-squamous non-small cell lung cancer (NSCLC). Stage 2 is the pivotal stage of the trial and will assess the safety and efficacy of gotistobart in squamous cell NSCLC. The primary endpoint measure is overall survival. Secondary endpoints include overall response rate, progression-free survival and adverse event profile. Approximately 600 patients are planned to be enrolled at clinical sites in 12 countries and regions globally, including China, Germany, Italy, South Korea, Türkiye, the United Kingdom, and the United States.
About gotistobart (BNT316/ONC392)
Gotistobart is licensed to BioNTech for commercialization and jointly developed clinically by OncoC4 and BioNTech for oncology indications. There are several ongoing clinical trials in different tumor types, investigating gotistobart either as monotherapy or in combination with other therapeutic agents.
About OncoC4
Based in Rockville, Maryland, OncoC4 is a privately held, late-stage biopharmaceutical company that is actively engaged in the discovery and development of novel biologicals for the treatment of cancer and immunological diseases. OncoC4's pipeline features assets with first-in-class and best-in-class potential targeting both novel and well validated targets across oncology and immunological diseases. Among them, AI-081 is a fully owned and potentially best-in-class bispecific antibody candidate targeting PD-1 and VEGF. AI-081 is currently enrolling patients in a Phase 2 trial that could enable two or more pivotal studies. ONC-841 is a first-in-class anti-SIGLEC10 antibody currently in a Phase 2 trial for oncology indications and being explored for neurodegenerative diseases. OncoC4 has a strategic collaboration with BioNTech to co-develop gotistobart (BNT316/ONC-392), a next-generation anti-CTLA-4 antibody candidate, in multiple solid tumor indications, including an ongoing pivotal clinical trial in squamous non-small cell lung cancer.
More information: www.oncoc4.com.
Investor Contact:
Ryan Cui, Executive Director of Investor Relations
[email protected]
1 Houston KA, Henley SJ, Li J, White MC and Richards TB, Lung Cancer, 86:22-28, 2014.
2 Hu S, Zhang W, Guo Q, Ye J, Zhang D, Zhang Y, Zeng W, Yu D, Peng J, Wei Y, Xu J, International Journal of General Medicine, 14:9567-9588.
3 Lau SCM, Pan Y, Velcheti V and Wong K, Cancer Cell, 40:1279-1293, 2022.
SOURCE OncoC4
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