Therapy Trends KOL Insight: Acute Myeloid Leukaemia [2019]

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Publication Date:
February 2019
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How has the advent of novel targeted therapies impacted AML?

For decades, there were no approved treatments for acute myeloid leukaemia (AML) other than chemotherapies. However the past two years has witnessed the approval of eight new therapies for AML: Novartis’ Rydapt, Celgene/Agios’ Idhifa, Jazz Pharma’s Vyxeos, AbbVie/Roche’s venetoclax, Agios’ Tibsovo, Astellas’ Xospata, as well as Pfizer’s Mylotarg and Daurismo. How are these agents faring on the market so far and what’s expected to happen to uptake in the future? The FLT3-inhibitors and IDH inhibitors are paving the way for more personalised therapy, but which drugs stand out to key opinion leaders (KOLs)?

Learn how KOLs see the market evolving, and how they expect developers to differentiate their therapies in KOL Insight: Acute Myeloid Leukaemia. Twelve US and European KOLs give their insight on eight marketed products and eight pipeline programmes.

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Top takeaways

  • How has the arrival of targeted therapies impacted the treatment of AML? Eight new targeted therapies are now approved for AML; how has this impacted AML practice? Find out KOLs’ views on these agents and how the landscape will continue to evolve.
  • How will the three next-generation FLT3 inhibitors stack up against the established product Rydapt? What do KOLs consider to be the differences between Xospata, quizartinib and crenolanib? Find out how KOLs see usage of these next-generation drugs evolving.
  • How do KOLs perceive the IDH inhibitors Idhifa and Tibsovo?  How do the experts view the response rates obtained with this class of molecularly-targeted agents andwhere do they envisage these niche agents fitting in long-term?
  • Will AbbVie/Roche’s venetoclax transform AML treatment practice? The drug has been hyped as a ‘game-changer’, do KOLs consider this warranted?
  • Is there a place for early-stage antibody-based approaches in AML? The early-stage pipeline for AML includes bispecific antibodies and antibody-drug conjugates. Do KOLs consider any promising?
  • More personalised approaches on the horizon for AML? Do KOLs believe AML has reached a key turning point where toxic chemotherapy regimens will be replaced by targeted drugs?
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“We will have more progress with rapid testing to understand what mutations there are, and then we can establish the correct treatment for each patient.”
EU Key Opinion Leader

“You’re going to see a lot more multi-drug therapy and multiple combinations. Combinations of everything plus venetoclax.” 
US Key Opinion Leader

Sample of therapies covered

Marketed Therapies

  • Vyxeos (CPX-351; Jazz Pharmaceuticals)
  • Mylotarg (gemtuzumab ozogamicin; Pfizer)
  • Rydapt (midostaurin; Novartis)
  • Idhifa (enasidenib; Agios/Celgene)
  • Tibsovo (ivosidenib; Agios)
  • Daurismo (glasdegib; Pfizer)
  • Xospata (gilteritinib; Astellas)
  • Venclexta/Venclyxto (venetoclax; Roche/ AbbVie)

Pipeline Therapies

  • quizartinib (Daiichi Sankyo)
  • crenolanib (Arog)
  • pracinostat (Helsinn/MEI Pharma)
  • idasanutlin (Roche)
  • pevonedistat (Takeda)
  • Iomab-B (Actinium)
  • guadecitabine (Otsuka/Astex)
  • uproleselan (Glycomimetics)

KOLs interviewed

KOLs from North America

  • Dr. Jorge Cortes, MD, Professor in the Department of Leukemia, MD Anderson Cancer Center, Houston, TX
  • Dr. Elihu Estey, MD, Professor of Medicine in the Division of Hematology at University of Washington School of Medicine, Seattle, WA
  • Dr. Ivana Gojo MD, Co-Director of the Leukemia Drug Development Program and Associate Professor of Oncology at Johns Hopkins Medicine Hospital, Baltimore, MD
  • Dr. Farhad Ravandi MD, Professor and Chief in the Department of Leukemia at MD Anderson, Houston, TX
  • Dr. Eytan Stein MD,Assistant Professor in the Leukemia Service at the Memorial Sloan Kettering Cancer Center, New York
  • Dr. Roland B. Walter MD PhD MS, Associate Professor of Medicine in the Division of Hematology at the University of Washington School of Medicine, Seattle, WA

KOLs from Europe

  • Professor Alan Burnett, MD, PhD, Professor and Head of the Department of Haematology at the University of Wales College of Medicine, Cardiff, UK
  • Dr. Richard Dillon MB BS PhD MRCP FRCPath, Consultant Haematologist at Guy's and St Thomas' Hospitals, London, UK
  • Professor Xavier Thomas, MD, Department of Haematology, Edouard Herriot Hospital, Hospices Civils de Lyon, Lyon, France
  • Professor Norbert Vey, MD, PhD, Professor of Hematology at the University of Lille and Hospital Claude-Huriez – CHRU Lille, France
  • Professor Gert Ossenkoppele, MD, PhD, Professor in Translational Hematology at the VU University Medical Center, Amsterdam, Netherlands
  • Anonymous German KOL, MD, PhD, Professor at the Department of Haematology and Oncology at a leading university hospital, Germany

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