Preclinical evaluation and phase 1 study of the PI3Kα/δ inhibitor TQ-B3525 in Chinese patients with advanced cancers

Cancer. 2024 Nov 1;130(21):3686-3698. doi: 10.1002/cncr.35453. Epub 2024 Jun 26.

Abstract

Background: Phosphatidylinositol 3-kinase (PI3K) inhibitors transformed management of various malignancies. This study preclinically characterized TQ-B3525 (dual PI3Kα/δ inhibitor) and assessed the recommended phase 2 dose (RP2D), safety, efficacy, and pharmacokinetics in relapsed or refractory (R/R) lymphoma or advanced solid tumors (STs).

Methods: Oral TQ-B3525 was given at eight dose levels on a 28-day cycle. Primary end points were dose-limiting toxicity (DLT), maximum tolerated dose (MTD), and safety.

Results: TQ-B3525 showed high selectivity and suppressed tumor growth. Between June 12, 2018, and November 18, 2020, 80 patients were enrolled (63 in dose-escalation cohort; 17 in dose-expansion cohort). Two DLTs occurred in two (two of 63, 3.2%) DLT-evaluable patients; MTD was not identified. TQ-B3525 at 20 mg once daily was selected as RP2D. Grade 3 or worse treatment-related adverse events mainly included hyperglycemia (16.3%), neutrophil count decreased (15.0%), and diarrhea (10.0%). Two (2.5%) treatment-related deaths were reported. Sixty patients with R/R lymphoma and 11 advanced STs demonstrated objective response rates of 68.3% and 9.1%, disease control rates of 91.7% and 54.6%, median progression-free survivals of 12.1 and 1.1 months; median overall survivals were not reached.

Conclusion: TQ-B3525 exhibited rapid absorption and a nearly proportional increase in exposure. Acceptable safety and promising efficacy support further investigation of TQ-B3525 (20 mg once daily) for R/R lymphoma.

Keywords: PI3K inhibitor; TQ‐B3525; advanced solid tumors; phase 1 study; relapsed or refractory lymphoma.

Publication types

  • Clinical Trial, Phase I

MeSH terms

  • Adult
  • Aged
  • Class I Phosphatidylinositol 3-Kinases / antagonists & inhibitors
  • Class I Phosphatidylinositol 3-Kinases / genetics
  • Dose-Response Relationship, Drug
  • East Asian People
  • Female
  • Humans
  • Lymphoma / drug therapy
  • Lymphoma / pathology
  • Male
  • Maximum Tolerated Dose*
  • Middle Aged
  • Neoplasms* / drug therapy
  • Neoplasms* / pathology
  • Phosphoinositide-3 Kinase Inhibitors* / administration & dosage
  • Phosphoinositide-3 Kinase Inhibitors* / therapeutic use
  • Young Adult

Substances

  • Phosphoinositide-3 Kinase Inhibitors
  • Class I Phosphatidylinositol 3-Kinases
  • PIK3CD protein, human
  • PIK3CA protein, human