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

August 2013 | 37 pages | ID: T6B5F5BF5F8EN
MP Advisors

Abstracts

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.

Detailed financial and competitive analysis of the companies leading in the race to launch new therapies for this indication – Ambit Biosciences, Onconova Therapeutics, SymBio Pharma, and Celgene are available as a separate or with the full report. 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. This report is built using primary and secondary research data and in-house proprietary database.

Contents

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 Detailed Company Analysis (incl. Clinical Data of Drugs, Milestones, and Valuation)

10. AMBIT BIOSCIENCES (AMBI)

10.1 Investment Drivers
  10.1.1 Other Assets – Not Fully valued in the Price
  10.1.2 Key Milestones
  10.1.3 NPV Valuation – Scenario Analysis (Early Approval)
  10.1.4 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

About

Targeting unmet needs in the treatment of cancer through innovative drugdevelopment strategies have witnessed favorable outcomes recently. New targetshave been validated in this process where kinases lead the way. The success hasboosted the innovative biotech sector and is reflected in the performance of the IPOs'since 2010 till date. Positive clinical data/ regulatory approval, lucrative partnership/acquisition announcement have increased the confidence of investors on innovationand whetted their appetite for risk associated with innovation.

Several companies that have recently made their debut in public (NASDAQ) areaddressing unmet needs/ niche indications with novel technology platforms/targets.Earlier, we had highlighted the investment opportunity and drugs being developed forRelapsed and Refractory Multiple Myeloma (RRMM). Approval of Kvprolis(carfilzomib, Onyx), Ponialyst (pomalidomide, Celgene) for this indication and theirsuccessful commercial launch validate the strategy deployed by the companies. Weexpect 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) and in thisreport highlight the new drugs and companies developing these drugs.

Innovative approach to treating unmet need has been handsomely rewarded in thepast through in-licensing or outright acquisition of the mature/nascentproduct/innovator company at a premium. The successful IPOs' of Ambit andOnconova are indicators of the optimism prevailing amongst the investors, Patientsand Physicians community. In this report, details of some of these deals are includedwhere acquirers like Celgene, Takeda, Astellas, Roche etc are reporting robust salesand progress in pipeline from these acquisitions. This once again reiterates the thesisthat R&D focus is essential and worth the risk for a pharma company. While some ofthe compounds are still in the clinic or even returned to the innovators on ground ofstrategic decisions taken by the management, it has not discouraged new companiesfrom pursuing new targets and compounds leaving room for in-licensing and/or M&Aopportunity drugs in the early stages of development. The report highlights some ofthose compounds available for partnering- “tapping them young”.