HPB-092 tablet

HPB-092 effectively inhibits Fms-like tyrosine kinase 3 (FLT3) mutants with comparable or superior potency to approved FLT3 inhibitors and demonstrates improved selectivity, potentially reducing toxicity. Its highly selective and potent inhibition of Interleukin-1 Receptor-Associated Kinase 4 (IRAK4) may provide additional therapeutic benefits that could enhance treatment efficacy and durability for patients with relapsed or refractory acute myeloid leukemia (RR-AML), further improving clinical outcomes in this population. HPB-092 also has a favorable safety profile, with no major risks identified in preclinical studies. Phas 1 Study Outline: 1. This is a multicenter, open-label, phase 1 study to evaluate the safety and efficacy of oral HPB-092 as monotherapy in patients with RR-AML. 2. The study aims to assess safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary efficacy. 3. It consists of two parts: Part A for dose escalation and Part B for dose expansion, involving single or multiple doses. 4. Patients must be diagnosed with morphologically documented RR-AML according to World Health Organization (WHO) 2022 criteria. 5. Baseline assessments will include RR-AML with FLT3 mutations, spliceosome mutations in SF3B1 and U2AF1, as well as other biomarkers, which will be monitored throughout the study.