Aslan Pharmaceuticals presents positive data from Phase 2a leukemia study at ASH

ASLAN Pharmaceuticals announced the presentation of new positive data from the ongoing phase 2a study of ASLAN003 for the treatment of acute myeloid leukaemia at the 60th American Society of Hematology Annual Meeting. ASLAN003 is an orally active, potent inhibitor of dihydroorotate dehydrogenase that has the potential to be first-in-class in AML. As of 16 November 2018, 14 patients with AML ineligible for standard treatment had been enrolled in the multicentre dose optimisation study to evaluate ASLAN003 monotherapy administered as a 28-day cycle. Eight patients had received at least one post-treatment assessment at the cut-off date and were evaluable for efficacy. Of the 8 evaluable patients, 4 patients showed clinical signs of efficacy: 2 patients exhibited evidence of myeloid differentiation and 1 patient in the 100mg QD cohort developed suspected differentiation syndrome. Overall, 4 patients had stable disease for more than 3 months. ASLAN003 has been well tolerated in patients treated to date. The most commonly occurring related adverse events were leukocytosis, nausea and rash, with grade 3/4 leukocytosis in 1 patient. The study contains 4 cohorts for the optimum dose determination, and an additional expansion cohort with the selected optimum dose. The primary outcome of the phase 2a study is to determine the optimum monotherapy dose of ASLAN003 and provide a preliminary estimate of efficacy evaluated by overall complete remission rate. A phase 1 trial showed that ASLAN003 demonstrated dose proportional pharmacokinetics and was safe and well tolerated in healthy volunteers compared to the side effect profiles of existing AML induction and maintenance chemotherapies. ASLAN003 has demonstrated potent inhibition of DHODH, lack of toxicities associated with first generation inhibitors and other novel AML therapies, and the potential to induce differentiation in blast cells and applicability in a broad range of AML patients. The US Food and Drug Administration has granted ASLAN003 Orphan Drug Designation as a treatment for AML. ASLAN will also present new data from a preclinical study evaluating the effects of ASLAN003 on cell growth, differentiation, apoptosis, and gene expression changes in AML cell lines and primary bone marrow cells from patients with AML.
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