ACT001， a novel anti-tumor drug candidate, invented by a research team led by Professor Chen Yue from the College of Chemistry and the State Key Laboratory of Medicinal Chemical Biology at Nankai University, has been approved by China’s National Medical Products Administration (NMPA) to enter the Phase III clinical trial, based on the promising results from its Phase IIb clinical study.

ACT001, a novel anti-tumor drug independently developed by Professor Chen’s team over 15 years, is capable of crossing the blood-brain barrier and selectively targeting cancer stem cells，NF-κB and STAT3 pathways. It has been tested in more than 14 clinical trials worldwide.

According to the study team, data from recently released Phase IIb trial demonstrated that ACT001 exhibits significant synergistic effects when combined with immune checkpoint inhibitors, radiotherapy and chemotherapy. The intracranial response rate nearly doubled compared with radiotherapy alone. Notably, for patients with brain metastases from small cell lung cancer (SCLC), the median survival increased by 53% (9.5 months vs. 6.2 months). When further combined with immune checkpoint inhibitors, median overall survival was extended by 75% in SCLC patients (14.7 months vs. 8.4 months), and doubled in those with brain metastases from non-small cell lung cancer (NSCLC) (11.4 months vs. 4.7 months).

“Data from several studies have confirmed the significant advantages of ACT001 in radiosensitization, toxicity reduction and prolonging survival in advanced cancer patients. Regardless of the tumor types such as high grade gliomas or brain metastases, as a sensitizer of chemoradiotherapy, ACT001 has the potential to become the first drug capable of enhancing efficacy of both chemoradiotherapy and immunotherapy, offering cancer patients a safer and more effective treatment option and promoting innovation and breakthroughs in radiotherapy,” said Chen Yue.

To date, ACT001 has received five major regulatory designations from authorities in US and Europe, including orphan drug status, rare pediatric disease designation, and fast track designation. Notably, the Rare Pediatric Disease Designation awarded by the FDA represents the first such recognition for a drug developed in China. In January 2025, ACT001 was also included in China’s Breakthrough Therapy list by the Center for Drug Evaluation (CDE) of the NMPA.

The upcoming Phase III trial is expected to involve 50 leading hospitals across China, including Cancer Hospital, Chinese Academy of Medical Sciences, Sun Yat-sen University Cancer Center, Peking Cancer Hospital, Tianjin Medical University Cancer Institute & Hospital, Shanghai Chest Hospital, Shanghai Pulmonary Hospital, Peking Union Medical College Hospital, West China Hospital of Sichuan University, Henan Cancer Hospital, and Hunan Cancer Hospital. Accendatech R&D center in the Tiankai Higher Education Innovation Park will provide critical technical support and ensure drug production quality control for the trial.

（Edited and translated by Nankai News Team.)