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Novel Combination Therapy to Treat Neutropenia

The combination of RAR agonist Am80 and G-CSF will provide therapeutic benefit to patients suffering from neutropenia beyond G-CSF alone

Published: 2nd October 2019
Novel Combination Therapy to Treat Neutropenia
Please note, header image is purely illustrative. Source: Roberto Galindo, Wikimedia Commons, CC-BY-SA-3.0


For over 20 years, G-CSF has been an effective treatment for increasing numbers of neutrophils and white blood cell count. However, despite this therapy, chemotherapy induced neutropenia continues to be a problem with infections, febrile neutropenia and associated deaths.

Technology Overview

The combination of Am80 (Tamibarotene) and G-CSF will provide therapeutic benefit to patients suffering from neutropenia beyond G-CSF alone. Am80, an oral retinoic acid derivative, promotes neutrophil maturation significantly better than G-CSF and effectively differentiates a GCSF-induced immature granulocytes into mature neutrophils. Unlike G-CSF, Am80 promotes the production of differentiated neutrophils that closely mimic fully mature neutrophils. Am80-induced neutrophils exhibit cellular morphology and functional reactive oxygen species (ROS) production characteristic of mature neutrophils. Together, Am80 and G-CSF work synergistically to provide sufficient numbers of mature neutrophils to combat infection and promote survival. 

Further Details

  • Blood. 2013 Feb 7;121(6):996-1007 Retinoid agonist Am80-enhanced neutrophil bactericidal activity arising from granulopoiesis in vitro and in a neutropenic mouse model
  • EMBO 2016 Mol Med 8(11): 1340-1359  Am80‐GCSF synergizes myeloid expansion and differentiation to generate functional neutrophils that reduce neutropenia‐associated infection and mortality
  • Pharmacology & Therapeutics Available online 27 August 2019, Strategies to generate functionally normal neutrophils to reduce infection and infection-related mortality in cancer chemotherapy


  • Drug repurposing allows shortened development timeline, API and Drug Master File available 
  • Shortened duration of cancer chemotherapy induced neutropenia (CCIN)
  • Lowers risk of both fungal and bacterial infection in patients suffering from CCIN 


  • Am80 used in combination with G‑CSF biosimilar for CCIN serves to differentiate from other G‑CSF biosimilars on the market
  • Breast cancer and AML patients with CCIN identified as target patient populations for clinical proof of concept


Looking for a licensee and development partner, preferably with an approved biosimilar G‑CSF or one in clinical development.

  • PCT/US13/44828 / Methods for Treating Neutropenia Using Retinoid Agonists
  • PCT/US15/16447 / Compositions and Methods for Treating Neutropenia
IP Status
  • Patented
  • Licensing
  • Development partner