While targeting unmet needs in the treatment of hematological malignancies/ cancer through innovative drug development strategies have witnessed favorable outcomes, the specific choice of the therapies is dependent on the identification of important genomic alterations in cancerous cells that allow for the tumor’s sub classification.  Over the past decade, Proteasome inhibitor and the immunomodulatory drugs have become the cornerstone of treatment for pts with Multiple Myeloma (MM) resulting in improved survival.  However, eventually all pts relapse and use of modern diagnosis will enable risk stratification to help distinguish pts along the spectrum of the condition.  This can aid in the selection of new treatment options for Relapsed/ Refractory Multiple Myeloma (RRMM) to further improve survival and quality of life of each patient.  Two new drugs have successfully fulfilled this need – POMALYST/ IMNOVID (pomalidomide – POM, Celgene, approved) and Kyprolis (carfilzomib-CFZ, Amgen/ Ono Pharma – JP, approved).  While novel targets address unmet need, the pertinent Question remains – is there a need for further improvement?  What should be the diagnosis criteria and/or novel imaging / diagnostic tools that can further stratify a pts’ risk profile?

Precision medicine approaches in myeloma require fast, robust, and practicable molecular diagnostic tools, and the current diagnostic standard iFISH (interphase fluorescence in situ hybridization) is unable to fulfill any of these criteria.  Integration of Diagnostics into therapeutic products/ industry has potential to improve trial design, enhance safety profile, enhance therapeutic efficacy, accelerate trial outcome, and increase commercial success.  However, there are few hurdles which are also associated with new model, such as understanding the diagnostic industry, complex trial execution, seeking a ‘right’ diagnostic partner, managing the co-development process, regulatory uncertainty around companion diagnostics and intellectual property issues.

In this report, we highlight the emerging new treatments in RRMM /NDMM, which includes combination of small molecules and biologics, and novel approach of molecular analysis of MM.  This report also provides M&A deals in the diagnostic, cancer area along with growing market opportunities. Furthermore, the competitive landscape of NDMM and RRMM is also highlighted.  The rivalry prevalent in the global onco-diagnostic and therapeutic MM market is quite fierce with numerous local and global players contenting for the market share.  On the global diagnostic front, the key players are Roche, Abbott Diagnostics, and Illumina; and in Tx area, the key players are Celgene, Takeda pharma, Amgen, Novartis, and JNJ”

Companies Mentioned

Affymetrix,  SkylineDx, AgenaBio, Signal Genetics, Cancer Genetics Inc, Illumina, NeoGenomics, Exiqon, Regulus Therapeutics, Rosetta Genomics, Sequenta, Takeda Pharma, Celgene, Amgen, On Pharma, Abbott, BMS, Mundipharma, Novartis,MorphoSys, JNJ, Innate Ph

Table of Contents

1. Executive Summary

2. Disease Overview, Diagnosis, and Current Treatment
2.1 Overview of Diagnostic tools for Multiple Myeloma
2.2 Diagnostic Criteria of Myeloma and Recent Amendment
2.3 Diagnostic Approaches for Multiple Myeloma
2.3.1 Molecular pathogenesis of Myeloma
2.3.2 Unmet Need
2.3.3 New Diagnostics Perspective and Technologies
a ‘Companion’ Diagnostics (CDx)
b Genome-wide array
c RAN based technologies
d Next-Generation Sequencing (NGS) technology

3. Diagnostic Tools for Multiple Myeloma
3.1 Affymetrix GeneChip
3.2 SkylineDx MMprofiler
3.3 AgenaBio iPLEX Genotyping
3.4 Signal Genetics MyPRS
3.5 Cancer Genetics Genomic Products
3.6 Illumina NGS
3.7 NeoGenomics CLIA-certified Cancer Test
3.8 Exiqon microRNA PCR
3.9 Regulus Therapeutics microRNA Marker
3.10 Rosetta Genomics Cancer Origin Test
3.11 Sequenta LymphoSIGHT Platform

4. M&A Deals and Market Opportunity in Diagnostic Space

5. Current Therapies
5.1 Newly Diagnosed Multiple Myeloma (NDMM)
5.1.1 Competitive Landscape for NDMM
5.2 Relapsed and/or Refractory Multiple Myeloma (RRMM)
5.2.1 Competitive Landscape for RRMM
5.3 Approved Therapies
5.3.1 Revlimid
5.3.2 Velcade
5.3.3 Kyprolis
5.3.4 POMALYST
5.4 Data Comparison
5.4.1 Newly Diagnosed Multiple Myeloma (NDMM)
5.4.2 Relapsed and/or Refractory Multiple Myeloma (RRMM)

6. Novel Targets
6.1 Monoclonal Antibodies
6.1.1 Elotuzumab
6.1.2 Situximab
6.1.3 Daratumumab
6.1.4 MOR202
6.1.5 SAR650984
6.1.6 BI-505
6.1.7 BHQ880
6.1.8 CT-011
6.1.9 IPH2101/ Lirilumab
6.1.10 BMS-936564 (ulocuplumab)
6.2 Small Molecules
6.2.1 Panobinostat
6.2.2 Ixazomib (MLN9708)
6.2.3 Aplidin (plitidepsin)
6.2.4 ARRY-520 (filanesib)
6.2.5 Oprozomib
6.2.6 KPT-330 (selinexor)
6.2.7 SNS01-T
6.2.8 Ibrutinib
6.2.9 Ricolinostat
6.2.10 Afuresertib
6.2.11 KW-2478
6.2.12 BT-062

7. Clinical Milestones

8. Launch Timeline and Commercial Opportunity of Late-Stage Pipeline

9. M&A/ Licensing Deals and Unpartnered Product Opportunities

List of Tables

Table 1: Overview of Diagnostic Tools for Mm
Table 2: Diagnostic Criteria of Myeloma and Related Disorders
Table 3:Recent Companion Deals In Onco Area: 1h 2014
Table 4: Competitive Landscape – NDMM
Table 5: Competitive Landscape – RRMM
Table 6: Pfs and Interim Os In Renal Subgroups
Table 7: Maintenance Lenalidomide After Auto-Hct: Summary of Survival Outcomes
Table 8: Data Comparison – Newly Diagnosed Multiple Myeloma (NDMM)
Table 8a : Data Comparison – Newly Diagnosed Multiple Myeloma (NDMM)
Table 9: Data Comparison – Relapsed and Refractory Multiple Myeloma (RRMM)
Table 10: Indirect Treatment Comparison: Reported Coefficients And
Table 11: Patent Expiries of Market Products
Table 12: Launch Timeline and Market Potential
Table 13: Select M&A and Licensing Deals In Last 6 Years – 2007 To 2014
Table 14: Select In-Licensing Opportunities: Small Molecules & MABS

List of Figures

Figure 1: Molecular Pathogenesis of Myeloma
Figure 2: Targets for Novel Targets Incl. MAB In Multiple Myeloma