On November 2, 2023 Pfizer Inc. (NYSE: PFE) reported that it will present its latest data showcasing advances in the treatment of hemophilia, sickle cell disease, and blood cancers at the 65th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition in San Diego from December 9-12 (Press release, Pfizer, NOV 2, 2023, View Source [SID1234636836]). These data from 39 presentations represent continued innovation and advancement in hemophilia including pivotal findings for Pfizer’s novel anti-tissue factor pathway inhibitor (anti-TFPI) candidate marstacimab and the latest findings on a next-generation investigational treatment for sickle cell disease (SCD) in GBT021601 (GBT601). Pfizer will also present the latest research in blood cancer, including for ELREXFIO (elranatamab-bcmm), a BCMA-directed bispecific antibody recently approved by the U.S. Food and Drug Administration (FDA) for the treatment of adult patients with relapsed or refractory multiple myeloma (RRMM) who have received at least four prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody.
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"Pfizer has been advancing science in hematology for more than 30 years, starting with the introduction of recombinant factor replacement therapy, which became the standard of care for people living with hemophilia. This year at ASH (Free ASH Whitepaper) we will deliver five oral presentations including the latest clinical findings from our hemophilia programs and exciting data from the GBT601 program in sickle cell disease, representing progress in our unrelenting efforts to address the broad spectrum of patient needs," said Sonal Bhatia, M.D., Chief Medical Officer, Rare Disease, Pfizer. "The findings reflect the company’s scientific capabilities and use of translational science to potentially offer improved treatment options to help people living with these rare diseases."
"During ASH (Free ASH Whitepaper), we are pleased to present new clinical and real-world data in multiple myeloma from our broad development program for ELREXFIO, following recent FDA accelerated approval. This includes extended efficacy and safety results from MagnetisMM-3, highlighting sustained clinical efficacy and no new safety signals after 20 months of follow-up," said Chris Boshoff, Chief Oncology Research and Development Officer and Executive Vice President, Pfizer. "These data continue to support the potential of ELREXFIO as the next standard of care for patients with advanced multiple myeloma."
Highlights from company-sponsored abstracts include:
Six presentations from our hemophilia pipeline, including the first presentation of primary results from the non-inhibitor cohort of the pivotal Phase 3 BASIS clinical trial evaluating marstacimab in people living with hemophilia A or B. Additionally, four-year data from Pfizer’s Phase 1/2 study of investigational gene therapy giroctocogene fitelparvovec in adults living with severe hemophilia A will be presented.
Fourteen abstracts focused on the treatment of SCD, including Phase 2a safety, efficacy, and pharmacodynamic data from the ongoing Phase 2/3 clinical trial study of GBT601. In addition, long-term safety and efficacy findings on voxelotor (over four years follow-up) will be presented as well as data illustrating insights into the experience of SCD patients and their caregivers through a social media listening study.
Nineteen abstracts from our blood cancer portfolio, including six which continue to support the use of ELREXFIO, a B-cell maturation antigen (BCMA)-CD3-directed bispecific antibody immunotherapy, in patients with RRMM. This includes extended findings from MagnetisMM-3 cohort A (~20 months median follow-up) and a pooled analysis of efficacy and safety in Black patients with RRMM from MagnetisMM-1, MagnetisMM-3, and MagnetisMM-9, reinforcing Pfizer’s commitment to health equity.
A complete list of Pfizer-sponsored accepted abstracts is available here.
Key Pfizer-sponsored oral presentations at ASH (Free ASH Whitepaper) 2023 include:
Predictive Biomarker Analysis from the GBT021601 Survival Study in Townes Sickle Mice (Abstract #14)
Pochron M
Saturday, December 9, 9:30 – 11:00 AM PST
Presentation Time: 9:45 AM PST
Efficacy and Safety of the Anti-Tissue Factor Pathway Inhibitor Marstacimab in Participants with Severe Hemophilia without Inhibitors: Results from the Phase 3 Basis Trial (Abstract #285)
Matino D
Saturday, December 9, 4:00-5:30 PM PST
Presentation Time: 4:30 PM PST
Preliminary Results from a Multicenter Phase 2/3 Study of Next-Generation HbS Polymerization Inhibitor GBT021601 for the Treatment of Patients with Sickle Cell Disease (Abstract #274)
Saraf S
Saturday, December 9, 4:00 – 5:30 PM PST
Presentation Time: 4:45 PM PST
Understanding the Experiences of Patients with Sickle Cell Disease and their Caregivers by Social Media Listening in the UK (Abstract #1057)
Shastri O
Monday, December 11, 4:30 – 6:00 PM EST
Presentation Time: 4:30 PM PST
Four-Year Follow-up of the Alta Study, a Phase 1/2 Study of Giroctocogene Fitelparvovec (PF-07055480/SB-525) Gene Therapy in Adults with Severe Hemophilia Α (Abstract #1054)
Leavitt A
Monday, December 11, 2023, 4:30 PM – 6:00 PM PST
Presentation Time: 5:15 PM
About Hemophilia
Hemophilia is a family of rare genetic blood diseases caused by a clotting factor deficiency (FVIII in hemophilia A, FIX in hemophilia B) which prevents normal blood clotting. Hemophilia is often diagnosed in early childhood and impacts more than 400,000 people worldwide.1 The inability of the blood to clot properly can increase the risk of painful bleeding inside the joints and other serious or even life-threatening bleeding. People living with hemophilia can suffer permanent joint damage following repeated bleeding episodes.1,2
For decades, the most common treatment approach for hemophilia A and B has been factor replacement therapy, which replaces the missing clotting factors. Factor replacement therapies increase the amount of clotting factor in the body to levels that improve clotting, resulting in less bleeding; however, they must be administered by IV infusion on a regular basis.2,3 Approximately 25-30% of people with hemophilia A and 3-5% of people with hemophilia B are unable to continue taking factor replacement therapies because they develop inhibitors to FVIII and FIX.4,5
About Sickle Cell Disease
Sickle cell disease (SCD) is a lifelong, debilitating inherited blood disorder characterized by hemolytic anemia, which drives vascular inflammation, acute pain crises and progressive end organ damage. Acute pain crisis, or vaso-occlusive crisis (VOC), occurs when sickled red blood cells, white blood cells and platelets stick to the inflamed lining of blood vessels leading to vascular occlusion, tissue ischemia and pain. Complications of SCD begin in early childhood and are associated with shortened life expectancy. Early intervention and treatment of SCD have shown potential to reduce symptoms, events, long-term organ damage, and extend life expectancy. Historically, there has been a high unmet need for therapies that address SCD and its acute and chronic complications. SCD occurs particularly among those whose ancestors are from sub-Saharan Africa, though it also occurs in people of Hispanic, South Asian, Southern European, and Middle Eastern ancestry.
About Multiple Myeloma
Multiple myeloma (MM) is an aggressive and currently incurable blood cancer that affects plasma cells made in the bone marrow. Healthy plasma cells make antibodies that help the body fight infection.6 MM is the second most common type of blood cancer, with over 35,000 new cases of MM diagnosed annually in the U.S. and 176,000 globally.7,8 About half of those diagnosed with MM won’t survive beyond five years, and most will receive four or more lines of therapy due to relapse.9 While disease trajectory varies for each person, relapses are nearly inevitable.10 Real-world evidence shows that people with RRMM often become resistant to the three main classes of treatment – proteasome inhibitors, immunomodulatory agents and anti-CD38 monoclonal antibodies – after just a few rounds of therapy, and re-treating with these classes was common.11 The goal of therapy for people with RRMM is to achieve disease control with acceptable toxicity and improved quality of life.12
Prescribing Information for Pfizer Medicines
Please read full Prescribing Information, including BOXED WARNING, for ELREXFIO.
Please read full Prescribing Information for OXBRYTA.