InnoCare's Zurletrectinib Shows 90% Response Rate in Pediatric NTRK-Fusion Solid Tumors
核心洞察
InnoCare's next-generation pan-TRK (搜索) inhibitor zurletrectinib achieved a 90% objective response rate in pediatric and adolescent patients with NTRK fusion-positive solid tumors (搜索) in a phase I/II trial.
All patients resistant to first-generation TRK (搜索) inhibitors who completed full efficacy evaluations achieved partial responses, demonstrating the drug's ability to overcome resistance mechanisms.
The treatment showed a favorable safety profile with no dose-limiting toxicities observed and treatment-related adverse events predominantly grade 1-2.
InnoCare Pharma (搜索) announced promising results from its phase I/II clinical trial of zurletrectinib (ICP-723), a next-generation pan-TRK (搜索) inhibitor, showing remarkable efficacy in pediatric and adolescent patients with advanced solid tumors. The data, presented at the Congress of International Society of Paediatric Oncology (SIOP) 2025, demonstrated a 90% objective response rate in patients with NTRK (搜索) fusion-positive tumors as of July 31, 2025.
Strong Efficacy Against Treatment-Resistant Tumors
The multicenter trial, led by Professor Yizhuo Zhang from the Department of Pediatric Oncology at Sun Yat-sen University Cancer Center (搜索), enrolled patients with histopathologically confirmed, surgically unresectable, locally advanced or metastatic solid tumors. The predominant tumor types included NTRK (搜索)-rearranged spindle cell tumors (搜索) and primary central nervous system tumors (搜索).
Notably, all patients who were resistant to first-generation TRK (搜索) inhibitors and completed full efficacy evaluations achieved partial responses, highlighting zurletrectinib's potential to overcome resistance mechanisms that limit current therapies. This represents a significant advancement for patients who have exhausted first-line TRK inhibitor options.
Favorable Safety Profile and Dosing
The trial established recommended phase 2 doses of 7.2 mg/m² for pediatric patients and 8 mg for adolescents. Pharmacokinetic analysis revealed comparable exposure levels between pediatric/adolescent and adult patients at these doses, supporting consistent drug exposure across age groups.
Safety data showed no dose-limiting toxicities were observed throughout the study. Treatment-related adverse events were predominantly grade 1-2, indicating a well-tolerated safety profile suitable for the pediatric population.
Regulatory Progress and Clinical Context
The drug's development has gained regulatory momentum, with the Centre for Drug Evaluation accepting zurletrectinib's new drug application for NTRK (搜索) gene fusion-positive tumors and granting priority review status in May 2025. Previous interim findings presented at the 2025 ASCO Annual Meeting showed that median duration of response and progression-free survival were not reached as of November 2024, with 12-month rates of 92.0% and 90.5%, respectively.
Among three patients with brain metastases (搜索), two achieved intracerebral responses, suggesting potential efficacy in central nervous system involvement.
Company Expansion Strategy
"Zurletrectinib has demonstrated outstanding efficacy and safety in adult, adolescent, and pediatric patients with tumors harboring NTRK (搜索) fusion genes, bringing better treatment options for patients with solid tumors," said Jasmine Cui, co-founder, chairwoman, and chief executive officer of InnoCare. The company is expanding its solid tumor pipeline through targeted therapies, immune-oncology approaches, and antibody drug conjugate technology.
The phase I/II trial enrolled patients 12 years and older with measurable lesions per RECIST v1.1 criteria, or for primary central nervous system tumors (搜索), per Response Assessment in Neuro-Oncology or International Neuroblastomas Response Criteria. Primary endpoints focused on safety, tolerability, and maximum tolerated dose, while secondary endpoints included pharmacokinetics and objective response rate.