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Ref Type Abstract
PMID
Authors Anwen Xiong, Xiangjiao Meng, Yongzhong Luo, Baogang Liu, Jian Fang, Qitao Yu, Liping Tan, Zhangzhou Huang, Qiming Wang, Li Yang, Yanmin Wu, Yanru Qin, Yongsheng Wang, Longhua Sun, Yinghui Sun, Zhihua Liu, Bo Feng, Anna Wang, Qiang Cai, and Caicun Zhou
Title Clinical efficacy and tolerability of the selective RET inhibitor soxataltinib (SY-5007) in advanced RET fusion–positive non–small cell lung cancer (NSCLC): Primary findings from a confirmatory phase III trial.
Journal Vol Issue Date Pages Journal Short Title
Journal of Clinical Oncology 44 16_Suppl May 27, 2026
URL https://ascopubs.org/doi/10.1200/JCO.2026.44.16_suppl.8639
Abstract Text Background: Oncogenic RET gene fusions represent a clinically validated molecular driver occurring in approximately 1–2% of all NSCLC cases, establishing a critical need for targeted therapeutic strategies. Soxataltinib is a novel, orally bioavailable, and highly selective small-molecule inhibitor of RET kinase. Its anti-tumor potency and safety have been previously reported in a phase I/II study. Here we confirmed its clinical value in a pivotal phase III study. Methods: This multicenter, single-arm Phase III clinical trial was designed to evaluate the efficacy and safety of Soxataltinib in patients with RET fusion–positive NSCLC who were previously untreated for advanced disease. The primary endpoint was the confirmed Objective Response Rate (ORR), as determined by Blinded Independent Central Review (BICR) per RECIST v1.1. The secondary endpoints included investigator-assessed ORR, Disease Control Rate (DCR), Duration of Response (DOR), Progression-Free Survival (PFS), Overall Survival (OS), and safety. Results: At the data cutoff (April 10, 2025), the Per-Protocol Population (PPP) comprised 95 patients, 61 of whom were Key efficacy population (KEP) for statistical hypothesis. Soxataltinib demonstrated profound anti-tumor efficacy. The primary endpoint was met. The BICR-confirmed ORR was 90.0% (95%CI: 79.5, 96.2) for KEP and 87.4% (95%CI: 79.0, 93.3) for PPP. The overall DCR was 96.7% (95%CI: 88.5, 99.6) for KEP and 93.7% (95%CI: 86.8, 97.6) for PPP. The median PFS and DOR had not yet been reached, with an estimated 15-month PFS rate of 68.9% (95%CI: 54.3, 79.7) and 65.0% (95%CI: 51.5, 75.6) and 12-month DOR rate of 73.8% (95%CI: 58.2, 84.4) and 69.1% (95%CI: 53.8, 80.1), respectively for KEP and PPP. The OS data remained immature. The safety analysis population comprised 96 patients who received at least one dose of Soxataltinib. The most common Grade ≥3 treatment-emergent adverse events (TEAEs) were hypertension (22.9%), diarrhea (16.7%), Aspartate aminotransferase increased (6.3%), Alanine aminotransferase increased (5.2%), and hyponatraemia (5.2%). These events were predominantly manageable. None of the patients permanently discontinued treatment due to a treatment-related adverse event. No patient died due to TEAE that was definitely related, probably related, or possibly related to Soxataltinib. Conclusions: The primary analysis of this Phase III trial confirmed Soxataltinib as a highly effective and well-tolerated therapeutic agent for patients with RET fusion–positive NSCLC in the first-line setting. This observed high response rates and durable disease control benefit underscored its potential as a best-in-class RET inhibitor. The adverse event profile was predictable and manageable, supporting its feasibility for long-term administration. Clinical trial information: NCT06031558.

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Molecular Profile Treatment Approach
Gene Name Source Synonyms Protein Domains Gene Description Gene Role
Therapy Name Drugs Efficacy Evidence Clinical Trials
Drug Name Trade Name Synonyms Drug Classes Drug Description
Gene Variant Impact Protein Effect Variant Description Associated with drug Resistance
Molecular Profile Indication/Tumor Type Response Type Therapy Name Approval Status Evidence Type Efficacy Evidence References
RET fusion lung non-small cell carcinoma sensitive SY-5007 Phase III Actionable In a Phase III trial, first-line treatment with SY-5007 in patients with non-small cell lung cancer harboring RET fusions resulted in a BICR-confirmed object response rate (ORR) of 90%, disease control rate (DCR) of 96.7%, and estimated 15-month progression-free survival (PFS) rate of 68.9% for the key efficacy population (n=61) and an 87.4% ORR, 93.7% DCR, and 65.0% estimated 15-month PFS for the per-protocol population (n=95) (J Clin Oncol 44: 16_Suppl, 8639; NCT06031558). detail...