China Authorizes Gotistobart for Breakthrough Therapy against Aggressive Lung Cancer

China Authorizes Gotistobart for Breakthrough Therapy Against Aggressive Lung Cancer

In a significant advancement for cancer treatment, China's National Medical Products Administration (NMPA) has granted Breakthrough Therapy Designation to Gotistobart (BNT316/ONC-392). This groundbreaking authorization targets squamous non-small cell lung cancer (sqNSCLC), a particularly aggressive type of lung cancer that poses significant challenges in treatment.

With approximately 25-30% of all non-small cell lung cancer cases classified as sqNSCLC, the need for effective therapies is paramount. Current treatments often fall short, especially for patients who have already undergone standard immuno-oncology therapies. The survival rate for sqNSCLC patients remains distressingly low, at about 15% over five years.

OncoC4, the company behind Gotistobart, expressed optimism about the new therapy’s potential. "For the past five years, limited advances have been made for EGFR wild-type NSCLC patients who have progressed on initial treatments. We believe Gotistobart can significantly improve outcomes for these individuals," said Pan Zheng, MD, PhD, Co-founder and Chief Medical Officer at OncoC4.

Gotistobart is a next-generation anti-CTLA-4 antibody that boasts an enhanced therapeutic index compared to existing treatments. According to Yang Liu, PhD, Co-Founder and CEO of OncoC4, the therapy preserves CTLA-4 on regulatory T cells, which could represent a breakthrough in targeted cancer therapy. Emerging data indicates this innovative approach shows clinical proof of concept that could greatly benefit patients.

Currently, Gotistobart is undergoing the registrational two-stage randomized Phase 3 trial, PRESERVE-003 (NCT05671510). This trial is assessing its efficacy and safety compared to standard chemotherapy—docetaxel—specifically for patients who have seen progression following prior PD-(L)1-inhibitor treatment. The initiation of this pivotal trial was encouraged by promising early results from ongoing Phase 1/2 studies demonstrating safety and efficacy of Gotistobart both alone and in combination with Pembrolizumab.

The Breakthrough Therapy designation is part of NMPA’s program tailored to expedite the development and regulatory review process for therapies addressing serious conditions. To achieve this designation, Gotistobart has demonstrated preliminary clinical evidence indicating a substantial improvement over existing therapies on significant clinical endpoints.

Following the initial results from Stage 1 of the PRESERVE-003 trial, which confirming safety and efficacy, detailed findings will be disclosed at an upcoming medical conference in December 2025. The approval allows for greater interaction with NMPA during the development phase, aiming to facilitate the accelerated collection of necessary supportive data. This might even allow for priority review if certain criteria are met.

The two-stage Phase 3 study will involve around 600 participants from clinical sites located across 12 countries, including China, Germany, Italy, South Korea, the UK, and the USA. This extensive research underscores the global effort to address the critical needs of sqNSCLC patients.

Gotistobart, which is licensed to BioNTech for commercialization, reflects ongoing collaboration between OncoC4 and BioNTech. The partnership is jointly developing the treatment to tackle various oncology indications. Furthermore, OncoC4 is a privately held biopharmaceutical firm based in Rockville, Maryland, focused on pioneering biological treatments for cancer and immunological diseases. Their R&D pipeline emphasizes both first-in-class and best-in-class candidates targeting established cancer pathways.

As the medical community eagerly anticipates the results from these trials, Gotistobart stands poised as a beacon of hope for individuals battling the challenges of sqNSCLC, shining a light on the future of cancer treatment.