Acute Myeloid Leukaemia: KOL Insight [2018]

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Publication Date:
February 2018
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How will four newly approved therapies transform treatment of AML?

For decades, there were no approved treatments for acute myeloid leukaemia (AML) other than chemotherapies. The past year has witnessed the approval of four new therapies for AML; Novartis’ Rydapt, Celgene/Agios’ Idhifa, Jazz Pharma’s Vyxeos and Pfizer’s Mylotarg. How are these agents faring on the market so far and will more personalised treatments follow their lead? AbbVie/Roche’s Venclexta/Venclyxto (venetoclax), Astellas’ gilteritinib and Agios’ ivosidenib all form part of a rich pipeline, 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 pipeline therapies in KOL Insight: Acute Myeloid Leukaemia. Twelve US and European KOLs give their insight on six marketed products and 13 pipeline programmes. KOLs also provide their candid views on the potential for novel chemotherapies and early-stage pipeline programmes.

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Top takeaways

  • Chemotherapies dominated the landscape for 40 years, but how has this all changed in the last year? Four therapies have been approved for AML in the past year [Novartis’ Rydapt, Celgene/Agios’ Idhifa, Jazz Pharma’s Vyxeos and Pfizer’s Mylotarg]. Find out how KOLs perceive these agents and how the landscape will continue to evolve.
  • Three next-generation FLT3 inhibitors are in the pipeline but how can they successfully differentiate themselves? Novartis’ Rydapt is currently in the lead with next-generation FLT3 agents hot on its heels. Find out how KOLs weigh up these next-generation drugs against Rydapt.
  • How do KOLs perceive the IDH inhibitors Idhifa and ivosidenib?  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?
  • Which of the pipeline drugs hold the most promise in AML? KOLs weigh up the data so far with AbbVie/Roche’s Venclexta/Venclyxto (venetoclax), Helsinn/MEI Pharma’s pracinostat and Roche’s idasanutlin and reveal which drug they view as most promising in AML.
  • Will novel chemotherapies have a chance to improve upon older agents and if so, what challenges do they face? Novel chemotherapies such as Otsuka/Astex’ guadecitabine are hoping to compete with conventional standard-of-care regimens. Find out what KOLs think about this.
  • What other early-stage programmes are KOLs particularly excited about? The early-stage pipeline for AML encompasses PD-1 inhibitors, CAR-T therapies and other bispecific monoclonal antibody approaches. Which ones do KOLs hold high hopes for?
  • 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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“It's going in the right direction. AML is being unfolded as multiple diseases which are defined by single mutations. It's getting more into the targeted therapies. Standard chemotherapy will probably disappear from the scene in a decade.”
EU Key Opinion Leader

“The FLT3 inhibitors [are the most promising]. If I had to put another one, I would put venetoclax.”
US Key Opinion Leader

Sample of therapies covered

Marketed Therapies

  • Rydapt (midostaurin; Novartis)
  • Vyxeos (CPX-351; Jazz Pharmaceuticals)
  • Mylotarg (gemtuzumab ozogamicin; Pfizer)
  • Dacogen (decitabine; Otsuka/Janssen)
  • Vidaza (azacitidine; Celgene)

Pipeline Therapies

  • quizartinib (Daiichi Sankyo)
  • gilteritinib (Astellas)
  • crenolanib (Arog Pharmaceuticals)
  • ivosidenib (Agios Pharmaceuticals)
  • Venclexta/Venclyxto (venetoclax; AbbVie/Roche)
  • pracinostat (Helsinn/MEI Pharma)
  • idasanutlin (Roche)
  • Opdivo (nivolumab; Bristol-Myers Squibb)
  • pevonedistat (Takeda)
  • Iomab-B (Actinium Pharmaceuticals)
  • vadastuximab talirine (Seattle Genetics)
  • guadecitabine (Otsuka/Astex Pharmaceuticals)
  • CC-486 (Celgene)

KOLs interviewed

KOLs from North America

  • Dr. Mark Levis, MD PhD, Associate Professor of Oncology and Medicine in the Division of Hematologic Malignancies at the John Hopkins School of Medicine, Maryland, Baltimore
  • Dr. Farhad Ravandi MD, Professor and Chief in the Department of Leukemia at MD Anderson, Houston, Texas
  • Dr. Roland B. Walter MD PhD MS, Associate Professor of Medicine, Division of Hematology, University of Washington School of Medicine, Seattle.Dr Farhad Ravandi, MD, Professor and Chief in the Department of Leukaemia at MD Anderson, Houston, Texas
  • Dr. Daniel Pollyea MD MS, Assistant Professor of Medicine and Clinical Director of Leukemia Services in the Division of Hematology, School of Medicine at the University of Colorado, Colorado
  • Dr. James K. Mangan MD PhD, Assistant Professor of Clinical Medicine at the Abramson Cancer Center, Perelman Center for Advanced Medicine, University of Pennsylvania (Penn Medicine), Philadelphia, Pennsylvania
  • Dr. Ran Reshef MD, Director of Translational Research, Blood and Marrow Transplantation in the Division of Hematology/Oncology at the Columbia University Medical Center, New York

KOLs from Europe

  • Professor Bruno Quesnel MD PhD, Professor of Hematology at the University of Lille and Hospital Claude-Huriez - CHRU Lille, France
  • Dr. Donal McLornan MB BCH (Honours) PhD MRCP FRCPath, Consultant Haematologist at King’s College Hospital London, London, UK
  • Dr. Richard Dillon MB BS PhD MRCP FRCPath, Consultant Haematologist at Guy's and St Thomas' Hospitals, London, UK
  • Dr. Nicolas Blin MD, Consultant Physician of Hematology at Nantes University Hospital, France
  • Anonymous German KOL MD PhD, Professor of Haematology and Oncology at a leading University Hospital, Germany
  • Anonymous German KOL MD PhD, Professor at the Department of Haematology and Oncology at a leading University Hospital, Germany

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