2025/12/09
Polaris Group (TWSE: 6550) is pleased to announce that its lead drug pegargiminase (ADI-PEG 20) which is already under FDA review for a new drug application for the treatment of malignant pleural mesothelioma is also showing remarkable progress in the treatment of acute myeloid leukemia (AML). The latest encouraging data from its Phase 1 clinical trial were officially presented on December 6, 2025, at the annual meeting of the American Society of Hematology.
Patients with previously treated AML, as well as newly diagnosed AML patients who carry high- risk features (such as p53 mutations, complex monosomal karyotype (CMK), PTPN11 or RAS mutations, etc.), have very limited treatment options and generally respond poorly to standard therapies.
This Phase 1 clinical trial focused on newly diagnosed AML patients with such high-risk characteristics, or those who are older or unfit for intensive chemotherapy, to evaluate the efficacy and safety of combining pegargiminase with the standard treatments venetoclax and azacitidine.
Of the 25 patients enrolled, 16 (64%) had high risk mutations, and 9 others (36%) had secondary type mutations. Of the 23 patients evaluable, 16 (69.6%) had a complete response, and the total composite objective response rate was 21/23 (91.3%) with durable responses. Median overall survival was 19.1 months, and 14/23 (60.9%) were censored for overall survival (still alive).
In extremely high-risk groups such as those with TP53 mutations or CMK, the tumor response rate achieved by combining ADI-PEG 20 with AZA-VEN has surpassed all historical results from chemotherapy alone, demonstrating a significant advantage.
In terms of treatment safety, pegargiminase was well tolerated, with no dose limiting toxicity observed, and the overall safety profile was consistent with expectations.
Steve Hsu, Chairman of Polaris Group, stated: “The high rate of responses durability of these responses, and overall survival was better than expected in this very-high risk AML population. We are encouraged by these results and are considering next steps in our development and clinical strategy.”