The Best Target & The Best Chemical


PHI-101-AML is a treatment drug for refractory/recurrent acute myelogenous leukemia (AML). It is a ”Best-in-Class” next-generation FLT3 inhibitor with high safety and compared to global competitors and differentiated efficacy against resistance of existing FLT3 inhibitors. Currently, we are conducting phase 1 global clinical trials in Australia and Korea.

Unmet Needs
  • FLT3-ITD mutation was found in 25% to 35% of all AML patients

  • Development of next-generation FLT3 inhibitor capable of treating FLT resistant mutations is required

  • Superior efficacy about Drug resistant FLT3 Mutation (D835 or F691)
  • Effect of reducing malignant cells in the bone marrow by up to 98% in 1 month in patients who are non-adaptive to competitive drugs
  • Selection of candidate by prediction of Chemiverse ADMET (Confirmed by clinical trial results)
  • Less adverse events and better hematological recovery compared to competing
  • Suppression of malignant prognostic factor (DNMT3A, IDH1, TET2) in AML
  • Conditional marketing authorization and US orphan drug designation
  • Number of patients About 14,000people
  • Market size KRW 570.4B (2023) Target Market
    • FLT3 Mutation positive patients

Sources : DRG, 2019

  • ASP2215, Gilteritinib
    (Astellas Pharma.)

    • FLT3 & Axl double inhibitor, response rate in phase 3 clinical trial was 34%.
    • 30.5% recurrence (96% within 4 weeks) in phase 3 clinical trial. Major cause of recurrence is FLT3 F691 resistance mutation.
    • Japanese and USA FDA approved only for FLT3-ITD and D835 mutations.
  • Midostaurin

    • Efficacy in AML patients with FLT3-ITD. It is very likely to lead to another adverse effects due to non-selectivity to kinases
      (clinical dose; 40mg).
    • Low efficacy in clinical trials when administered alone.
    • US FDA approval for combined administration, not a single treatment.
  • Quzartinib(AC-220)
    (Ambit Biosciences)

    PHASE 3
    • Licensing agreement with Astellas after phase 1 clinical trial (390 mUSD).
    • Decreased efficacy for AML patients with TKD(D835V,D835Y) and Gatekeeper(F691L) mutations among point mutations other than FLT3-ITD.
    • US FDA notified of NDA rejection due to concerns for the data reliability and adverse effects compared to drug efficacy.
  • Crenolanib
    (AROG Pharma.)

    PHASE 3
    • As an inhibitor with high selectivity and activity against FLT3, it also shows high activity against D835V, a mutant resistant to quizartinib.
    • No activity is reported against the FLT3-ITD F691L mutation.
    • Clinical trials are progressing of taking TID.
  • Life-Span
    Animal test results

    Excellent life extension
    effect in the FLT3-ITD
    mutant animal model

  • Bioluminescence
    Animal test results

    Strong efficacy
    compared to competing
    drugs in FLT3-ITD/F691L+D835Y

  • AML patient
    Sample test result

    Stronger activity compared to
    competitors in AML patient

  • GLP
    Toxicity test result

    2.5 folds higher tolerability
    than competing drugs in GLP