Treating Refractory Hematological Malignancies – Myelodysplastic Syndromes (MDS) and Acute Myeloid Leukemia (AML): Emerging Therapeutics

Published: August 2013
No. of Pages: 0

Targeting unmet needs in the treatment of cancer through innovative drug development strategies have witnessed favorable outcomes recently.  New targets have been validated in this process where kinases lead the way.  Approval of Kyprolis (carfilzomib, Onyx), Pomalyst (pomalidomide, Celgene) for the Relapsed and Refractory Multiple Myeloma (RRMM) and their successful commercial launch validate the strategy deployed by the companies.  We expect a similar scenario to unfold in the treatment of recurrent/ refractory MDS (RR-MDS, High-Risk MDS), and AML (relapsed/ refractory AML, R/R AML).  Innovative approach to treating unmet need has been handsomely rewarded in the past through in-licensing or outright acquisition of the mature/nascent product/innovator company at a premium.  The successful IPOs’ of Ambit and Onconova are indicators of the optimism prevailing amongst the investors, Patients and Physicians community.

The report provides an overview of the approved therapies for AML, MDS, unmet need and limitations of the current SOC for relapsed and refractory pts.  The report highlights the competition and commercial opportunity in pursuing this therapy area – Comprehensive list of the early and late stage drugs in the clinic, their MoA and the companies developing them.  Key M&A activities that have taken place in this area in the last 5 years and a list of products in early /late stages of development available for in-licensing are highlighted in the report.

Key Points Discussed in the Report –

  • Overview of the disease and Unmet need
  • Drugs in the pipeline – Mechanism of Action (MoA) and Clinical Stage of development
  • Myelodysplastic Syndromes –  Lower-risk, Higher-risk, and recurrent / refractory MDS
  • Acute Myeloid Leukemia – Untreated/ newly diagnosed and relapsed / refractory AML
  • Key milestones
  • Launch Timeline and Commercial Opportunity of Late-stage pipeline (RR MDS and RR AML)
  • M&A and Licensing deals in the last 5 years
  • In-licensing and/or M&A Opportunity
  • Detailed Company analysis includes Clinical data of drugs , Milestones, and Valuation
    • Ambit Biosciences (AMBI)
    • Onconova Therapeutics (ONTX)

Published By: MP Advisors
Product Code: MP Advisors10

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