Science Will Win Against Multiple Myeloma

Nearly 200 years after multiple myeloma (MM), an aggressive blood cancer that affects plasma cells made in the bone marrow, was first described, there is still no cure. Yet, I’m struck every day by the resilience and drive among those who are impacted by this disease, including Dr. Freeman Williams, an esteemed educator and community leader living in Delaware.

“Those myeloma cells, they change up the script and they force you to be adaptable,” says Dr. Williams. “It’s not the end. It’s the beginning of a new normal. I’m going to continue to live my life.”

At Pfizer, we focus our efforts on the areas that have the highest unmet needs and where we can bring real value to impact as many lives as possible, as quickly as possible. Over 10 years ago, we set our sights on blood cancer and more recently we have been pursuing a potentially transformative breakthrough in MM, with a promising pathway for treating the disease. Now, that treatment has received accelerated approval by the FDA , offering people with MM a new option when prior treatments have stopped working.

In tackling MM, we rapidly identified the promise of B-cell maturation antigen (BCMA)-directed bispecific antibodies and accelerated the project from discovery in our laboratories in California to approval, with urgency. We went from a first-in-patient trial to approval in under five years, compared to about nine years for the typical innovative new medicine.(1) I’m beyond proud of what we’ve accomplished. Now, we have 15 clinical trials exploring the use of our BCMA-directed bispecific antibody among a diverse set of people with MM around the world.

Addressing Unmet Need

MM is the second most common type of blood cancer, with over 35,000 new cases diagnosed annually in the U.S. and 176,000 globally.(2,3) For those with recurrent and relapsed disease, the use of available treatment options often results in patients enduring a high symptom burden, low response rates, and low overall survival. While disease trajectory varies for each person, relapses are nearly inevitable, and most people become resistant to late-line therapies. There is a high unmet need for safe and effective new treatments, as patients face dwindling therapeutic options when they relapse or become resistant to successive medicines.(4) Today, the median life expectancy following a MM diagnosis is just over five years.(5)

The Rise of Targeted Immunotherapy

Like most cancers, MM was historically treated with the blunt tools of chemotherapy and radiation. Later, the advent of proteasome inhibitors, immunomodulatory agents and monoclonal antibodies presented patients with additional options, which resulted in prolonged life, though their disease often becomes resistant to these treatments after just a few rounds of therapy and re-treating with the same drug classes is common.(6)

The discovery of BCMA ushered in a new era for MM treatment.(7) BCMA is highly expressed on myeloma cells, so it presents a key to unlocking precision medicine, targeting the cancer preferentially.(8) There have been several different approaches to exploiting BCMA in the treatment of MM, including antibody-drug conjugates that deliver targeted chemotherapy and chimeric antigen receptor T-cell therapy (CAR-T) that uses the patient’s own immune cells to fight their cancer.

Nearly a decade ago, Pfizer began investigating a different way to use BCMA to treat MM – bispecific antibodies. Bispecific antibodies are a novel type of cancer immunotherapy that bind to surface proteins on cancer cells and T-cells, bringing the two together and activating the person’s own immune system to kill their cancer cells. Unlike cell-based therapy, bispecific antibodies can be delivered subcutaneously and are available off-the-shelf (ready-to-use). That means they can be administered promptly and in community clinics for ongoing treatment, where the majority of patients receive their care, enabling broad access to treatment. In clinical trials, our BCMA-directed bispecific antibody led to meaningful clinical responses among patients with MM who have relapsed or developed resistance to earlier treatments.

Ongoing Commitment to the MM Community and Beyond

Although today’s approval now provides a much-needed new treatment option for patients with relapsed or refractory MM, we’re not stopping. We’re expanding our ongoing MagnetisMM clinical development program into additional patient populations, including newly diagnosed MM, testing our BCMA-directed bispecific antibody both as a solo treatment and in combination with other drugs. We’re also exploring the use of bispecific antibodies for treating other types of cancer, where off-the-shelf, targeted immunotherapy is sorely needed.

Everything we do at Pfizer is intended to help patients everywhere live longer and healthier lives. That’s why we don’t just work for patients — we work with them, through initiatives like our Multiple Myeloma Patient Steering Committee, which Dr. Williams takes part in. 

“It has opened up a whole new world, sitting down with doctors and practitioners and giving them a different perspective from the vantage point of a patient with myeloma,” says Dr. Williams.

From the earliest stage of drug development to final approval, our purpose is breakthroughs that change patients’ lives. By nurturing an environment where these breakthroughs can thrive, sourcing the best science in the world, partnering with others to improve access to our medicines, and using digital technologies to enhance both patient outcomes and drug discovery/development, every action we take at Pfizer is done for patients, globally.

References

1 Brown DG, Wobst HJ, Kapoor A, Kenna LA, Southall N. Clinical development times for innovative drugs. Nat Rev Drug Discov. 2022;21(11):793-794. doi:10.1038/d41573-021-00190-9

2 American Cancer Society: Key Statistics About Multiple Myeloma. Accessed July 12, 2023. Available at: https://www.cancer.org/cancer/multiple-myeloma/about/key-statistics.html 

3 World Health Organization: Globocan 2020: Multiple Myeloma. Accessed July 12, 2023. Available at: https://gco.iarc.fr/today/data/factsheets/cancers/35-Multiple-myeloma-fact-sheet.pdf 

4 Sonneveld P, Broijl A. Treatment of relapsed and refractory multiple myeloma. Haematologica. 2016 Apr;101(4):396-406. doi: 10.3324/haematol.2015.129189

5 Leukemia and Lymphoma Society. Facts and Statistics Overview. https://www.lls.org/facts-and-statistics/facts-and-statistics-overview

6 Guillaume X, Horchi D, Gomez J, et al. Real-world treatment patterns of triple-class refractory (TCR) multiple myeloma (MM) across the United States (Us), Canada, and Western Europe: a retrospective chart study. American Society of Clinical Oncology (ASCO) 2023, June 2-6, in Chicago, IL. doi: 10.2217/fon-2020-1003

7 Kleber M, Ntanasis-Stathopoulos I, Terpos E. BCMA in multiple myeloma-A promising key to therapy. J Clin Med. 2021;10(18):4088. doi:10.3390/jcm10184088

8 Carpenter RO, Evbuomwan MO, Pittaluga S, et al. B-cell maturation antigen is a promising target for adoptive T-cell therapy of multiple myeloma. Clin Cancer Res 15 April 2013; 19 (8): 2048–2060. doi: 10.1158/1078-0432.CCR-12-2422