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

Published: August 2013
No. of Pages:

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)

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

Table of Contents

1. Executive Summary

2. Investment Drivers of The Select Companies Covered

3. Myelodysplastic syndrome (MDS)
3.1 Disease Overview
3.2 Unmet Need
3.3 Current Standard of Care
3.3.1 Supportive Care
3.3.2 Low-Intensity Therapy
3.3.3 High-Intensity Therapy
3.3.4 Recurrent/ Refractory or relapsed MDS

4. Acute Myelogenous Leukemia (AML)
4.1 Disease Overview
4.2 Current Standard of Care
4.2.1 AML pts younger than 60 years
a Induction Therapy
b Post-remission/ consolidation therapy
4.2.2 AML pts Older than 60 years
a Current treatment options – Hypomethylating agents and SCT
4.3 Unmet Need
4.4 Refractory or relapsed AML

5. Competitive Landscape: RR/ High-Risk MDS and RR AML

6. Competitive Landscape: Untreated/ Newly Diagnosed MDS and AML

7. Key Milestones

8. Launch Timeline and Commercial Opportunity of Late-stage Pipeline (RR MDS and RR AML)

9. Drivers of M&A / Licensing Deals in MDS/ AML
9.1 Select M&A Deals in Last 5 Years – 2007 to 2012
9.2 Select Licensing Deals in Last 5 Years – 2007 to 2012
9.3 M&A and Licensing Deals Opportunity

10. Detailed Company Analysis (incl. Clinical Data of Drugs, Milestones, and Valuation)
10.1 Ambit Biosciences (AMBI)
10.1.1 Investment Drivers
10.1.2 Other Assets – Not Fully valued in the Price
10.1.3 Key Milestones
10.1.4 NPV Valuation – Scenario Analysis (Early Approval)
10.1.5 Background about AMBI’s products
a. Quizaritinib – Key profile, Clinical Data, IP protection
b. Other assets – CEP-32496, AC410, & AC708
c. Competitive Landscape – Late- and Mid-stage AML drugs
d. Select JAK 2/1 Inhibitor: Late- and Mid-stage Pipeline
10.2 Onconova Therapeutics (ONTX)
10.2.1 Investment Drivers
10.2.2 Upside from Early-stage pipeline
10.2.3 Strong IP position
10.2.4 Key Milestones
10.2.5 NPV Valuation
10.2.6 Background about ONTX’s products
a. Estybon (rigosertib) – Key Profile, Primary Indications
b. Rigosertib IV – 2nd-line Higher-Risk MDS (clinical data and competition landscape)
c. Rigosertib Oral – 1st-line Lower-Risk MDS (clinical data and competition landscape)
d. Estybon (rigosertib) – Label Expansions
Metastatic Pancreatic cancer
Head and Neck cancer
e. Other Early-stage drugs – ON 013105, Recilisib
f. Preclinical Programs – ON 1231320, ON 123300, ON 108600, ON 044580
g. Patents
h. Collaboration/ Partnership Agreements

Published By: MP Advisors
Product Code: MP Advisors10

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