BCMA Targeted Therapies, 2017-2030

BCMA Targeted Therapies, 2017-2030

  • March 2017 •
  • 143 pages •
  • Report ID: 4819007 •
  • Format: PDF
B-cell maturation antigen (BCMA), an important biomarker of the B-cells, has emerged as a promising therapeutic target for the treatment of multiple myeloma and other hematological malignancies. The antigen is universally expressed on the surface of multiple myeloma cells.

The research focus shifted towards BCMA targeted therapies in 2004, when the role of BCMA was first indicated in the progression of multiple myeloma. It is the second most common type (13%) of all hematological malignancies. The disease proves to be fatal due to serious complications associated with it and the frequent events of re-occurrence of illness. The widespread presence of multiple myeloma and other related B-cell malignancies demands confident diagnosis and treatments. Hence, there is an immediate need for effective therapies for proper medical care.

Currently, researchers are actively involved in developing three major types of immunotherapies (classified by product class) targeting BCMA; these are chimeric antigen receptor T-cells (CAR-T cells), bispecific antibodies and antibody drug conjugates (ADCs). Several biopharmaceutical companies have been active in this area since last few years while others have recently stepped in.

A number of strategic partnerships have also been inked between various stakeholders to advance R&D activities in this domain. Results of preclinical and clinical studies have demonstrated the potential benefits of this class of therapies; the major highlight being their attractive safety profile. As more molecules undergo clinical validation and eventually get commercialized, we believe the overall interest will continue to rise. In fact, our promising outlook is backed by a strong belief that this novel class of therapies is likely to cater to the current unmet need where the existing treatment modalities are not efficient. The upside could be higher; however, it depends on a favorable market environment, reimbursement practices and regulatory regimes.

The “BCMA Targeted Therapies, 2017-2030” report features an extensive study on the current market landscape of B-cell maturation antigen (BCMA) targeted therapies and offers a comprehensive discussion on the future potential of this market. With no commercial products, the market is still in its infancy. However, industry experts have pinned significant hopes on the novel technologies being developed by start-ups / small companies and the research being conducted at academic institutes.

The prime target indication of these novel molecules is B-cell malignancies, specifically multiple myeloma. BCMA-specific therapies are anticipated to emerge as viable treatment options for such indications. Post initial research on such therapies, many players have entered into collaborations with other stakeholders to fund the clinical and commercial development of their products. Some clinical stage products that have emerged out of such collaborations include bb2121 (bluebird bio / Celgene) and BCMA-CART (Novartis / Abramson Cancer Center of the University of Pennsylvania).

The pipeline currently comprises of 23 molecules that are under development for the treatment of a variety of B-cell malignancies. One of the key objectives of this report was to understand the evolution of the current market and to quantify the opportunities laid down by the innovative BCMA targeted programs of both small and big pharma companies.

Among other elements, the report provides information on the following:
- The current state of the market with respect to key players, phase of development of pipeline products (both clinical and preclinical / discovery) and the type of molecules.
- Comprehensive profiles highlighting clinical trial details such as dosage regimens, key preclinical / clinical findings, and future market opportunity for the clinical stage BCMA targeted therapies.
- Comparative analysis of the design of clinical trials being conducted for therapies in clinical stages of development.
- Various investments and grants received by companies focused in this area supporting their R&D activities.
- Partnerships that recently been inked amongst different stakeholders, covering product development / commercialization agreements, research collaborations, license agreements and acquisitions.

The study provides a detailed market forecast and opportunity analysis for the period between 2017 and 2030. The research, analysis and insights presented in this report are backed by deep understanding of key insights gathered from a variety of sources. To account for future uncertainties and add robustness to our model, we have provided three scenarios of our market forecast, namely the conservative, base and optimistic scenarios. All actual figures have been sourced and analyzed from publicly available information forums. All financial figures mentioned in this report are in USD, unless otherwise specified.

For most of our projects, we conduct extensive secondary research as well as interviews with experts in the area (academia, industry, medical practice and other associations) to solicit their opinions on emerging trends in the market. This is primarily useful for us to draw out our own opinion on how the market may evolve across different regions and sub-segments. Wherever possible, the available data has been checked for accuracy from multiple sources of information.

The secondary sources of information include
- Annual reports
- Investor presentations
- SEC filings
- Industry databases
- News releases from company websites
- Government policy documents
- Industry analysts’ views

While the focus has been on forecasting the market over the coming ten years, the report also provides our independent view on various non-commercial trends emerging in the industry. This opinion is solely based on our knowledge, research and understanding of the relevant market gathered from various sources of information.

Chapter 2 provides an executive summary of the insights captured in our research. It offers a high level view on the current state of the BCMA targeted therapies market and its likely evolution.

Chapter 3 provides a general introduction to BCMA, highlighting its expression profile, role in progression of multiple myeloma and mechanism of action. In this section, we have talked about the current research landscape of the human BCMA and its emergence for the treatment of multiple myeloma. Further, we have briefly discussed the molecules (bispecific antibodies, ADCs and CAR-T cells) that are being exploited for the development of BCMA-specific drug candidates.

Chapter 4 provides a comprehensive overview of the current landscape of the BCMA targeted therapies market. It includes information on the drug candidates that are currently in different stages of development (both clinical and preclinical / discovery). It presents a detailed analysis of the pipeline on the basis of current phase of development, type of molecules and key players involved in this space. We have also provided details on additional parameters (such as associated domains and transfection methods) being utilized for the development of T-cell based BCMA-specific therapies. In addition, the chapter includes an overview on the developer landscape based on the size and the activity of the industry / non-industry players and a geographical representation mapping the presence of various organizations involved in the clinical development of candidates in this field.

Chapter 5 presents detailed profiles of the BCMA targeted therapies in clinical phases of development (phase I/II and phase I). Each profile covers information on drug specifications, mechanism of action, dosage regimen, technology overview, manufacturing details, key preclinical / clinical findings and collaborations. In addition, we have also provided a comparative analysis on the design of clinical trials being conducted for several molecules, highlighting the study goals of the trials, primary and secondary endpoints being evaluated and the interim trial results.

Chapter 6 presents details on the investments and grants received by companies working on the development of BCMA targeted therapies. The analysis highlights the growing interest of the venture capital community and other strategic investors in this market.
Chapter 7 features an elaborate discussion on the collaborations and partnerships that have been inked amongst players in this market. We have also discussed the various partnership models that have been implemented, highlighting the most common forms of deals / agreements prevalent in this domain.

Chapter 8 provides a comprehensive market forecast, highlighting the future potential of the market till 2030. It includes future sales projections of the molecules in clinical stages of development (phase I/II and phase I). The sales potential and growth opportunity is based on the target patient populations, existing / likely future competition, likely adoption rates, the type of molecule and likely price points.

Chapter 9 summarizes the overall report and provides a recap of the key takeaways from the study. It also presents our independent opinion on the future of BCMA targeted therapies market based on the research and analysis described in previous chapters.

Chapter 10 is an appendix, which provides tabulated data and numbers for all the figures provided in the report.

Chapter 11 is an appendix, which provides the list of companies and organizations mentioned in the report.

1. BCMA is an important antigen that is expressed during B-cell development in almost 60%-70% of the multiple myeloma cases. During the course of our research, we identified over 20 therapies targeting BCMA that are currently in different stages of development. Of these, 26% are already undergoing clinical validation.
2. CAR-T therapies (constituting 44% of the development pipeline) are the most common anti-BCMA therapies, followed by bispecific antibodies (26%) and ADCs (13%). Multiple myeloma remains the prime focus of drug developers in this space. However, efforts are also being made to develop these therapies for the treatment of other B-cell malignancies such as leukemia and lymphoma.
3. We have provided a comparative analysis of the design of clinical trials being conducted for therapies in clinical stages of development. This primarily includes comparisons on the basis of study goals, primary and secondary endpoints being evaluated and the interim trial results.
4. The current market landscape features contributions from big pharmaceutical companies and small to mid-sized players. Established pharmaceutical players engaged in this space include (in alphabetical order) Amgen, bluebird bio, Boehringer Ingelheim, Celgene, GSK, Juno Therapeutics, Novartis and Pfizer. Small firms / start-ups and mid-sized firms that are actively investing in this market include (in alphabetical order) Affimed, Alexo Therapeutics, Cellectis, Five Prime Therapeutics, Heidelberg Pharma (a subsidiary of WILEX), Kite Pharma, Poseida Therapeutics (spun out of Transposagen Biopharmaceuticals), Sutro Biopharma and Triumvira Immunologics.
5. Prominent academic players that are involved in this space include (in alphabetical order) the Abramson Cancer Center of the University of Pennsylvania, California Institute for Biomedical Research, Memorial Sloan Kettering Cancer Centre (MSKCC), National Cancer Institute (NCI), Southwest Hospital (China) and The Scripps Research Institute.
6. We came across over 25 collaborations covering product development / commercialization agreements, product licensing agreements, technology licensing, acquisitions and research collaborations. Among these, development / commercialization agreements (52%) have been the most popular, followed by licensing agreements (26%).
7. Overall, we expect the field to witness considerable success in the long term. In fact, we expect six such therapies to be made commercially available in different geographies in the coming decade. Post the launch of the first wave of products, we predict the market to grow at an annualized rate of 98% till 2030.


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