Summary


Elacytarabine key points

  • No product approved in 30 years for relapsed or refractory AML
  • Pivotal Phase III study in progress worldwide
  • Phase II combination study with idarubicine ongoing
  • Phase II results:
    • Long median survival (5.3 months vs. 1.5 months in historical control)
    • Complete remission rate was significantly higher (18% vs. 2.5% in historical control)
  • Mechanism of action addresses major unmet need
    • Elacytarabine enables long leukaemia cell exposure to the active metabolite ara-CTP
    • Low hENT1 expression/level is a major cause of resistance to cytarabine
    • Ongoing biomarker programme for hENT1 in AML
  • Opportunity to broaden value in haematology
    • Expansion to front-line AML and high risk MDS
  • Orphan drug designation in the US and EU (product protection of 7 and 10 years, respectively)
  • FDA fast track designation 

LVT elacytarabine