Chipscreen Biosciences Announced NMPA Approval of IND for CS43158, an AML Candidate Discovered via the AI — enabled Chemical Genomics Platform

, Ltd. (“Chipscreen Biosciences” or the “Company”)(SSE: 688321) announced that the China National Medical Products Administration (NMPA) has approved the Investigational New Drug (IND) application for CS43158 tablets, a new molecular entity for the treatment of acute myeloid leukemia (AML), discovered through the company’s AI-enabled Chemical Genomics Platform. AML is the most common type of acute leukemia in adults. It’s a highly heterogeneous disease with limited treatment options and high relapse risks.

FLT3 mutations are key oncogenic drivers and occur in approximately 35%—45% of patients with relapsed/refractory (R/R) AML. The efficacy of currently approved FLT3 inhibitors as monotherapy remains limited, with a 30-40% objective response rate (ORR), a median progression-free survival (mPFS) of 2-6 months; approximately 60-80% of patients develop resistance within 6—12 months due to activation of bypass pathways, secondary mutations, and clonal evolution. Therefore, developing next-generation drugs that simultaneously inhibit FLT3 driver mutations and multiple bypass resistance pathways is considered a key strategy for overcoming current treatment limitations.

CS43158 is a multikinase inhibitor with global patent protection, independently developed by Chipscreen Biosciences leveraging its AI-ChemGen Platform. It employs a distinctively synergistic multi-target inhibition strategy that not only broadly suppresses FLT3 signaling but also precisely targets bypass resistance pathways. CS43158 shows potent inhibitory activity against clinically relevant FLT3 alterations, including wild-type FLT3 overexpression, FLT3-ITD, and FLT3-TKD mutations.

Meanwhile, it also suppresses aberrant activation of cytokine- and growth factor-associated signaling pathways within the bone marrow microenvironment, thereby broadly blocking non-FLT3-dependent tumor cell survival and proliferation pathways. Preclinical studies indicate that CS43158 has favorable pharmacokinetics and a favorable safety profile, and exhibits superior antitumor activity over reference compound across multiple models. In resistant models of existing therapies, sequential treatment with CS43158 significantly suppresses tumor progression, demonstrating its robust potential to overcome clinical resistance.

Furthermore, CS43158 demonstrates clear synergy with BCL-2 inhibitors, providing strong preclinical rationale for future combination regimens in AML. CS43158 has the potential to pair the precision of targeted therapy with durable control of drug resistance. This approach may further enhance antitumor activity, overcome or delay resistance to FLT3 inhibitors, and offer AML patients a differentiated therapeutic option for durable clinical benefit. About the AI-enabled Chemical Genomics Platform True to its name, Chipscreen Biosciences has been building a chemical genomics-based platform since its founding in 2001.

Based on gene expression profiling analysis through microarray technology, this platform systematically evaluates efficacy- and safety-related biological activities, thereby supporting screening and evaluation in early-stage small-molecule drug discovery. To convert the biological effects of drug molecules into computable data, Chipscreen Biosciences has established its AI-ChemGen Platform by integrating internal and external expertise over the past two decades.

Furthermore, to solve the verifiable data bottleneck, the Company has been building a structured and traceable proprietary database leveraging interdisciplinary approaches in cheminformatics and bioinformatics to bridge the closed loop of “data-model-wet lab-clinical trial”. Several molecules based on AI-ChemGen Platform have entered the global clinical development. com)