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Millions of people around the world suffer from blood disorders today. That’s why this December, blood disease specialists from around the globe are convening in New Orleans to share the latest advancements in the field during the 64th Annual Meeting of the American Society of Hematology (ASH), a professional society devoted to conquering blood diseases.

With presentations of more than 30 abstracts across its hematology portfolio at ASH, Pfizer is doubling down to address unmet needs in blood cancer, sickle cell disease and other hematologic diseases. Pfizer is especially enthusiastic about sharing data from a robust clinical trial program for its investigational multiple myeloma treatment, for which it recently received Breakthrough Therapy designation from the U.S. Food and Drug Administration for the treatment of relapsed or refractory multiple myeloma.

This is Pfizer Oncology’s 12th Breakthrough Therapy designation, which aims to speed up the development and regulatory review of a medicine for serious or life-threatening diseases and when preliminary clinical evidence indicates that it may have substantial improvement over existing therapies on a clinically significant endpoint.

In addition to sharing advances in oncology, Pfizer will be presenting important findings in sickle cell disease, including the latest in a growing body of real-world data. New data will also be presented demonstrating the potential of next-generation therapies to reduce complications and improve red blood cell health.

Through a deep knowledge of rare blood disorders and the recent acquisition of Global Blood Therapeutics, a company founded to address sickle cell disease, Pfizer has a promising pipeline of preclinical and clinical assets that could improve the treatment paradigm for sickle cell disease. Two of those pipeline products have best-in-class potential and Orphan Drug and Rare Pediatric Disease designations from the FDA.

Pfizer will also share hemophilia data at the congress which builds on the company’s extensive legacy in the space that transcends the recombinant era and extends to the ongoing development of gene therapies that are being investigated in Hemophilia A and B.

“At Pfizer, we’re applying decades of scientific expertise and in-depth understanding of blood disorders to identifying new targets, applying novel technologies and accelerating development to address areas of significant unmet need.”

Chris Boshoff, Chief Development Officer of Oncology and Rare Diseases at Pfizer.

Blood cancer is one area of particularly high unmet need that Pfizer is striving to address, through the development of innovative new treatments as well as programs designed to meaningfully improve health equity.

Some blood cancers are treatable, but so far, incurable

Blood cancers often start in the bone marrow, where new blood cells form, and affect the growth and function of white blood cells. While there are more than 100 different blood cancers, most of them are forms of leukemia, lymphoma, and myeloma. Some progress slowly, and others more aggressively. Despite recent medical advances, myeloma is one of the blood cancers that still has no cure.

Despite being first described in the 1840s, people diagnosed with multiple myeloma still have a median life expectancy of just over five years. Multiple myeloma is a devastating disorder of plasma cell proliferation in the bone marrow that can result in skeletal destruction. The clonal malignant plasma cells multiply, usually in more than one location, therefore the reference to ‘multiple myeloma.’ Nearly all patients who survive initial treatment will experience disease relapse at some point. With each relapse, the remission period is shorter and the treatment options narrow.

At the ASH Annual Meeting, Pfizer is sharing data that advances the science of hematology, including on the use of an emerging cancer immunotherapy called bispecific antibodies (BsAbs) to tackle multiple myeloma.

Immune cell engaging BsAbs bind to and engage a tumor cell and an immune cell. One arm binds directly to specific antigens on cancer cells, for instance, B-cell maturation antigen (BCMA), which is highly expressed on the surface of multiple myeloma cells. The other arm binds to T-cells, bringing the cells together to activate an immune response. In this way, these immune engagers enable the patient’s own immune system to kill cancer cells, irrespective of the cytogenetic risk group, as long as BCMA is expressed on the tumor cells.

“Agents targeting BCMA are emerging as the next wave of breakthrough in multiple myeloma,” said Boshoff. “This is an exciting area of innovative research, and we are well-positioned to be competitive in this space.”

Meanwhile, the company has been expanding its portfolio of blood cancer therapies, building on its six FDA-approved blood cancer medicines. Last year, Pfizer purchased Canadian drug developer Trillium Therapeutics, which brought in a suite of promising clinical-stage blood cancer immunotherapy candidates. Trillium’s portfolio includes SIRPα-CD47 biologics that are designed to enhance the ability of patients’ innate immune system to detect and destroy cancer cells.

Seeing health disparities and setting an industry benchmark to measure progress in clinical trial diversity and representation

Achieving health equity is a significant barrier across multiple disease areas. Sickle cell disease, for instance, is a life-long disease affecting mainly those with ancestors from sub-Saharan Africa, and also affecting people of Hispanic, South Asian, Southern European and Middle Eastern descent.

Multiple myeloma is a disease that primarily affects older populations, and also disproportionately affects ethnic and racial minority groups in both incidence and outcome. Yet these groups continue to be underrepresented in cancer clinical trials and studies.

“An important factor to consider when translating research into effective medicines is the diversity of the real-world patient population. Clinical trials often fail to capture that diversity — a failure that needs correcting.”

Dany Habr, MD, Oncology Chief Medical Affairs Officer at Pfizer and co-author of a paper about the importance of diversity of participants in multiple myeloma studies.

In their paper, Habr and co-author Massimo Corsaro cite a recent analysis of 112,293 patients recruited across 230 oncology trials that led to FDA approvals within the last decade. Only 3.1% of participants were Black and 6.1% were Hispanic (compared to 76.3% White). That’s less than 22% and 44% of their expected representation, respectively, based on U.S. cancer incidence.

A separate analysis of 177 multiple myeloma treatment trials found that the median age for participants was 62, while the median age for patients with multiple myeloma overall is 70. Only 5 of these trials reported outcomes for participants over the age of 75.

At ASH, Habr will join Pfizer’s Suneet Varma, Global Oncology and US President, and Sriram Krishnaswami, Vice President and Development Head, Multiple Myeloma, at the industry’s theater symposium entitled “Pursuing Scientific Breakthroughs for Patients with Hematologic Malignancies,” which will feature discussion on Pfizer’s research in blood cancer, its vision for delivering innovation and for addressing healthcare disparities in treating multiple myeloma.

Pfizer has committed to addressing both informational and practical barriers to diverse clinical trial participation and has published a 10-year retrospective analysis of ethnic, racial, age, and gender diversity in its past clinical trials to serve an industry benchmark from which to measure future progress.

Through its Diversity in Clinical Trials Center of Excellence program, the company has been soliciting feedback from patient advocacy groups on the design of its clinical trials, holding clinical trials in areas that represent a diverse pool of potential participants and providing age- and culturally- appropriate educational materials to build trust in clinical research.

Pfizer has also been collaborating with the sickle cell patient and advocacy communities, soliciting input on its drug development programs and better understanding barriers to treatment and care.“Listening to patients’ voices is critical for understanding and addressing disparities in care,” Habr said. “Our partnerships with the blood disorders community are key to this mission, and we look forward to continuing to work together at ASH and beyond.”

To learn more about Pfizer’s commitment to patients, visit us.

Chris Boshoff, Chief Development Officer of Oncology and Rare Diseases at Pfizer.
Dany Habr, MD, Oncology Chief Medical Affairs Officer at Pfizer and co-author of a paper about the importance of diversity of participants in multiple myeloma studies.


1. Facts and Statistics Overview. Leukemia and Lymphoma Society.
2. Facts and Information about Blood Cancer. Blood Cancer UK.
3. Myeloma. Centers for Disease Control.
4. Relapsed/Recurrent Multiple Myeloma. WebMD.