Shanghai Securities News China SecuritiesNet News (Reporter Zhang Xue) The reporter learned from InnoCare Pharma that at the 2025 International Society of Pediatric Oncology (SIOP) Annual Meeting held recently, Professor Zhang Yizhuo, Director of the Department of Pediatric Oncology at the Cancer Prevention and Treatment Center of Sun Yat-sen University (as the main researcher), and team member Professor Wang Juan (representing a multi-center researcher) orally reported the Phase I/II clinical data of China's independently developed new generation pan-TRK inhibitor zolecletinib in children and adolescents with advanced solid tumors, which attracted the attention of the global academic community.

Studies have shown that zolecatinib exhibits controllable safety and good anti-tumor activity in children/adolescents with NTRK gene fusion-positive solid tumors, indicating that it is a potential treatment for NTRK gene-driven malignant tumors, especially in overcoming the resistance of first-generation TRK inhibitors, showing good effectiveness, bringing new treatment directions for this type of tumor.

The report notes that while NTRK gene fusion-positive tumors are rare overall, they occur more frequently in children than in adults and are commonly seen in specific types of tumors, such as infantile fibrosarcoma, secretory breast cancer, and glioma. These tumors are driven by abnormal fusions of the NTRK gene with other genes. Traditional chemotherapy and radiotherapy are limited in effectiveness and have significant toxicity. The advent of TRK inhibitors marks a fundamental shift in cancer treatment from a "site-based" approach to a "driver gene-based" approach.

"As long as NTRK gene fusion is detected, the same targeted drug can be used to treat the tumor, regardless of whether it occurs in the brain, soft tissue, or other organs. This is the concept of 'treating different diseases with the same drug,'" Zhang Yijue explained. "Zolecatetinib, a second-generation TRK inhibitor originally developed in my country, is not only effective for treatment-naive patients but also for those who are resistant to first-generation TRK inhibitors."

According to the study results, the objective response rate (ORR) for enrolled children with NTRK fusion-positive tumors reached 90%. In terms of safety, no dose-limiting toxicities were observed. The report emphasizes that this data demonstrates the potential of zoledronic acid (zoledronic acid) in treating patients with NTRK/ROS1 gene fusion-positive solid tumors, particularly in overcoming drug resistance, providing new hope for survival for children who have developed resistance and are otherwise refractory to other treatments.

Notably, zolecletinib was recently included in the "Pilot Program for Encouraging Anti-tumor Drug R&D (Starlight Program)" by the Center for Drug Evaluation (CDE) of the National Medical Products Administration. Zhang Yiju believes this not only demonstrates the country's commitment to pediatric cancer patients but will also significantly accelerate the drug's review and market launch. "This means more children will have the opportunity to use this highly effective and safe targeted drug sooner rather than later, potentially even changing treatment outcomes for some."

Source：https://finance.eastmoney.com/a/202510243543410637.html