The AML Hub uses cookies on this website. They help us give you the best online experience. By continuing to use our website without changing your cookie settings, you agree to our use of cookies in accordance with our updated Cookie Policy

FDA clears IO-202, a novel LILRB4 antibody, for investigation as a new drug in AML

Jun 2, 2020

On May 29, 2020, the United States Food & Drug Administration (FDA) cleared IO-202, an antibody targeting leukocyte immunoglobulin-like receptor B4 (LILRB4), for investigation as a new drug treatment for acute myeloid leukemia (AML) in patients with monocytic differentiation and chronic myelomonocytic leukemia (CMML). 1

Key features 1

IO-202:

  • A first-in-class monoclonal antibody that blocks the signaling of LILRB4, an immune inhibitory receptor
  • The first T cell activator for AML
  • Known to activate T cell cytotoxicity against leukemia cells in preclinical studies and block tumor infiltration

Study Design 1,2

  • Phase I, multicenter, open-label, dose-escalation and dose expansion study, to determine the optimal dose of IO-202 in adult patients with AML with monocytic differentiation and in those with CMML
  • Will assess IO-202 as a monotherapy or in combination with azacitidine
    • For the dose escalation phase, dose cohorts will be treated with intravenous (IV) IO-202 monotherapy, in ascending doses every 2 weeks, followed by IO-202 every 2 weeks at the same dose in combination with IV or subcutaneous azacitidine, 75 mg/m 2for 7 days every 28 days
    • For the expansion phase, IV IO-202 monotherapy will be given at the recommended phase II dose and frequency; combination therapy will consist of IV IO-202 every 2 weeks at the recommended phase II dose and frequency with IV or subcutaneous azacitidine, 75 mg/m 2for 7 days every 28 days
  • Primary outcomes will evaluate the safety and tolerability of IO-202
  • Secondary outcomes will evaluate pharmacokinetics, pharmacodynamics, and clinical activity of IO-202

Biomarkers assessment will also enable a mechanistic understanding of the clinical data and inform future trials.

  1. Immune-Onc Therapeutics. Immune-Onc Therapeutics IND application for first-in-human trial of IO-202, a novel antibody for the treatment of acute myeloid leukemia, receives FDA clearance. https://www.immune-onc.com/news/2020-05-29-io-202-ind-clearance. Published May 29, 2020. Accessed June 1, 2020.
  2. Clinicaltrials.gov. IO-202 as monotherapy and in combination with azacitidine in AML and CMML. https://www.clinicaltrials.gov/ct2/show/study/NCT04372433?term=IO-202&draw=2&rank=1. Updated May 4, 2020. Accessed June 1, 2020.