On May 4, 2022 Aptose Biosciences Inc. ("Aptose" or the "Company") (NASDAQ: APTO, TSX: APS), a clinical-stage precision oncology company developing highly differentiated oral kinase inhibitors to treat hematologic malignancies, reported that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to HM43239, an oral, myeloid kinome inhibitor, for the treatment of patients with relapsed or refractory (R/R) acute myeloid leukemia (AML) with FLT3 mutation (Press release, Aptose Biosciences, MAY 4, 2022, View Source [SID1234613551]). Currently, an international Phase 1/2 clinical trial is ongoing for HM43239 in the R/R AML patient population. HM43239 received orphan drug designation from the FDA for treatment of acute myeloid leukemia in 2018.
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"Fast Track status acknowledges HM43239’s potential to fill an unmet need for AML patient populations and supports our efforts as we advance it towards a potential registration study," said William G. Rice, Ph.D., Chairman, President and Chief Executive Officer. "HM43239, which potently inhibits all tested forms of FLT3 and the SYK and JAK driven pathways, already has delivered complete remissions in a broad diversity of relapsed or refractory AML patients in an ongoing Phase 1/2 clinical trial, including patients with prior failure of other FLT3 inhibitor agents. Fast Track designation will help facilitate the drug’s development."
About Fast Track Designation
Fast Track is a process designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need. The purpose is to get important new drugs to the patient earlier. Once a drug receives Fast Track designation, early and frequent communication between the FDA and the sponsor is encouraged throughout the entire drug development and review process. The frequency of communication assures that questions and issues are resolved quickly, often leading to earlier drug approval and access by patients. Visit the FDA website for more information.
About HM43239
HM43239 is a potent oral genotype-agnostic small molecule inhibitor of a constellation of kinases operative in myeloid malignancies and known to be involved in tumor proliferation, resistance to therapy, and differentiation. Currently being developed for the treatment of patients with acute myeloid leukemia (AML), HM43239 is a potent inhibitor of FLT3, SYK, mutant forms of cKIT, JAK1/2, and other kinases. Regarding FLT3, HM43239 is highly active in vivo against FLT3 internal tandem duplication (ITD), as well as resistance-conferring D835 and gatekeeper (F691) tyrosine kinase domain (TKD) mutations. In vivo murine xenograft models suggest superior antitumor activity and favorable tolerability relative to established kinase inhibitor in AML, including gilteritinib (FLT3 inhibitor) and entospletinib (SYK inhibitor). Additionally, in vivo xenograft models suggest synergy with inhibitors of DNMT, BCL-2, and other key therapeutic targets, highlighting the combinatorial optionality of HM43239 in AML. In an ongoing international Phase 1/2 clinical trial, HM43239 has delivered complete remissions in a diversity of relapsed or refractory AML patients and continues to be a well-tolerated oral agent.