Last update 21 Jun 2024
Marstacimab
Last update 21 Jun 2024
Overview
Basic Info
Drug Type Monoclonal antibody |
Synonyms Marstacimab (USAN), 马塔西单抗, PF-06741086 + [1] |
Target |
Mechanism TFPI inhibitors(Tissue factor pathway inhibitor inhibitors) |
Therapeutic Areas |
Active Indication |
Inactive Indication |
Originator Organization |
Active Organization |
Inactive Organization- |
Drug Highest PhaseNDA/BLA |
First Approval Date- |
RegulationOrphan Drug (EU) |
Gene Sequence
Sequence Code 13046469L
Source: *****
Sequence Code 13046734H
Source: *****
Related
16
Clinical Trials associated with Marstacimab在有或无抑制物的儿童（＜18 岁）重度（凝血因子活性 <1%）甲型血友病或中重度至重度（凝血因子活性 ≤2%）乙型血友病受试者中比较既往 12 个月的历史标准治疗与 MARSTACIMAB 预防治疗的开放性研究
[Translation] An open-label study comparing 12 months of historical standard of care with MARSTACIMAB prophylaxis in pediatric (<18 years) subjects with severe (coagulation factor activity <1%) hemophilia A or moderately severe to severe (coagulation factor activity ≤2%) hemophilia B with or without inhibitors
本研究的目的是在有或无抑制物的 ≥1 至 <18 岁重度甲型血友病或中重度至重度乙型血友病（分别定义为 FVIII 活性 <1% 或 FIX 活性 ≤2%）的儿童受试者中评估 Marstacimab 预防治疗的安全性、耐受性和疗效。大约 100 例儿科人群受试者将入组这项开放性 III 期研究，并接受 1 年的 Marstacimab 治疗。
与目前的甲型或乙型血友病治疗相比，Marstacimab 治疗预计可展现临床相关优势或对患者治疗的重大贡献，因为它与因子替代药品的作用方式不同，并且其预期疗效不受抑制物存在的影响。每周一次皮下注射给药可在不具备可靠血管通路的情况下提供治疗选择，更加便利，并且可能改善依从性。综合而言，这些特性应该导致出血事件减少
[Translation]
The purpose of this study is to evaluate the safety, tolerability, and efficacy of marstacimab prophylaxis in pediatric subjects aged ≥1 to <18 years with severe hemophilia A or moderate to severe hemophilia B (defined as FVIII activity <1% or FIX activity ≤2%, respectively) with or without inhibitors. Approximately 100 subjects from the pediatric population will be enrolled in this open-label Phase III study and receive 1 year of marstacimab treatment.
Marstacimab treatment is expected to demonstrate clinically relevant advantages or a significant contribution to patient care compared with current treatments for hemophilia A or B because it works in a different way than factor replacement medicines and its efficacy is expected to be unaffected by the presence of inhibitors. Once-weekly subcutaneous administration provides a treatment option in the absence of reliable vascular access, is more convenient, and may improve compliance. Taken together, these features should result in fewer bleeding events
Start Date18 Jul 2023 |
Sponsor / Collaborator |
在有或无抑制物的重度（凝血因子活性<1%）甲型血友病或中重度至重度（凝血因子活性≤2%）乙型血友病的受试者中评估MARSTACIMAB 预防治疗的长期安全性、耐受性和疗效的开放性扩展研究
[Translation] An open-label extension study to evaluate the long-term safety, tolerability, and efficacy of MARSTACIMAB prophylaxis in subjects with severe (coagulation factor activity <1%) hemophilia A or moderate to severe (coagulation factor activity ≤2%) hemophilia B with or without inhibitors
主要目的：在有或无抑制物的12 至<75 岁重度甲型血友病或中重度至重度乙型血友病（分别为FVIII 活性<1% 或FIX 活性≤2%）受试者中评估Marstacimab 长期治疗的安全性和耐受性。
次要目的：1. 在有或无抑制物的12 至<75 岁重度甲型血友病或中重度至重度乙型血友病（分别为FVIII 活性<1% 或FIX 活性≤2%）受试者中评估Marstacimab 治疗的长期疗效。2. 评估 Marstacimab 对HRQoL 的影响。
[Translation]
Primary Objectives: To evaluate the safety and tolerability of long-term treatment with Marstacimab in subjects aged 12 to <75 years with severe hemophilia A or moderate to severe to severe hemophilia B (FVIII activity <1% or FIX activity ≤2%, respectively) with or without inhibitors.
Secondary Objectives: 1. To evaluate the long-term efficacy of Marstacimab treatment in subjects aged 12 to <75 years with severe hemophilia A or moderate to severe to severe hemophilia B (FVIII activity <1% or FIX activity ≤2%, respectively) with or without inhibitors. 2. To evaluate the effect of Marstacimab on HRQoL.
Start Date16 Mar 2023 |
Sponsor / Collaborator |
AN OPEN-LABEL STUDY IN PEDIATRIC (<18 YEARS OF AGE), SEVERE HEMOPHILIA A PARTICIPANTS (COAGULATION FACTOR ACTIVITY <1%) WITH OR WITHOUT INHIBITORS OR MODERATELY SEVERE TO SEVERE HEMOPHILIA B PARTICIPANTS (COAGULATION FACTOR ACTIVITY =2%) WITH OR WITHOUT INHIBITORS COMPARING 12 MONTHS OF HISTORICAL STANDARD TREATMENT TO MARSTACIMAB PROPHYLAXIS
The purpose of this clinical trial is to learn about the safety and effects of the study medicine (called marstacimab) for the potential treatment of hemophilia in pediatric patients.
This study will enroll pediatric participants from ages 1 to 17 years in a sequential manner. The study will open enrollment to adolescent participants aged 12 to 17 years first. Then children aged 6 to 11 years will be permitted to enroll. Lastly, children aged 1 to 5 years will be permitted to enroll.
This study will enroll participants who:
have severe Hemophilia A or moderately severe to severe Hemophilia B (with or without inhibitors)
have accurate historical records documenting all factor VIII, factor IX, or bypass agent infusions and hemophilia bleed events for at least 1 year prior to entering the study
if a non-inhibitor patient, must be on a stable routine prophylaxis regimen with factor VIII or factor IX replacement products for at least 12 months prior to study entry
if an inhibitor patient, must be on an on-demand bypass treatment regimen during the 12 months prior to study entry
All participants in this study will receive marstacimab to use prophylactically. Marstacimab will be given once a week as a subcutaneous (under the skin) shot. The first dose of marstacimab will be given at the study site by the study site staff. During the 12-month treatment period, weekly doses of marstacimab can be given at home, or if preferred, the doses may be given by the study site staff.
To help us determine if the study medicine is safe and effective, we will compare participant experiences when they are taking the study medicine to a historical period when they were not. Researchers want to see if the study medicine works to prevent the bleeding episodes commonly experienced by patients with Hemophilia.
Participants will be in this study for about 14 months (approximately 1 month in a Screening period, 12 months receiving treatment, and 1 month in a follow-up period) during which they will visit the study site at least 10 times. If preferred, and if local regulations allow it, 2 of the study visits can be completed at the participant's home instead of at the study site. There will also be 6 scheduled telephone calls approximately every 2 months.
This study will enroll pediatric participants from ages 1 to 17 years in a sequential manner. The study will open enrollment to adolescent participants aged 12 to 17 years first. Then children aged 6 to 11 years will be permitted to enroll. Lastly, children aged 1 to 5 years will be permitted to enroll.
This study will enroll participants who:
have severe Hemophilia A or moderately severe to severe Hemophilia B (with or without inhibitors)
have accurate historical records documenting all factor VIII, factor IX, or bypass agent infusions and hemophilia bleed events for at least 1 year prior to entering the study
if a non-inhibitor patient, must be on a stable routine prophylaxis regimen with factor VIII or factor IX replacement products for at least 12 months prior to study entry
if an inhibitor patient, must be on an on-demand bypass treatment regimen during the 12 months prior to study entry
All participants in this study will receive marstacimab to use prophylactically. Marstacimab will be given once a week as a subcutaneous (under the skin) shot. The first dose of marstacimab will be given at the study site by the study site staff. During the 12-month treatment period, weekly doses of marstacimab can be given at home, or if preferred, the doses may be given by the study site staff.
To help us determine if the study medicine is safe and effective, we will compare participant experiences when they are taking the study medicine to a historical period when they were not. Researchers want to see if the study medicine works to prevent the bleeding episodes commonly experienced by patients with Hemophilia.
Participants will be in this study for about 14 months (approximately 1 month in a Screening period, 12 months receiving treatment, and 1 month in a follow-up period) during which they will visit the study site at least 10 times. If preferred, and if local regulations allow it, 2 of the study visits can be completed at the participant's home instead of at the study site. There will also be 6 scheduled telephone calls approximately every 2 months.
Start Date09 Dec 2022 |
Sponsor / Collaborator |
100 Clinical Results associated with Marstacimab
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14
Literatures (Medical) associated with Marstacimab01 Sep 2023·Seminars in thrombosis and hemostasis
The Use of Bypassing Treatment Strategies in Hemophilia and Their Effect on Laboratory Testing.
Review
Author: Pruthi, Rajiv K ; Chen, Dong
Factor VIII and IX inhibitors in congenital hemophilia A and B, respectively, neutralize the infused coagulation factor concentrate rendering them ineffective. Bypassing agents (BPAs) that circumvent the block imposed by the inhibitors are used for the prevention and management of bleeding. Activated prothrombin complex concentrate was the original BPA, recombinant activated factor VII was then introduced, and more recently nonfactor agents that target the procoagulant and anticoagulant systems have been developed and are in clinical use (e.g., emicizumab, a bispecific antibody for hemophilia A). Other BPAs are in clinical trials (e.g., fitusiran targets antithrombin, concizumab and marstacimab target tissue factor pathway inhibitor, and SerpinPC targets activated protein C). The BPAs have a varied effect on coagulation assays, and as more patients are exposed to these agents, it is important to be aware of the effects. Herein, we present an overview of the effect of BPAs on routine and specialized coagulation assays including thrombin generation and viscoelastic assays.
23 Aug 2023·The AAPS journal
Evolution of Antidrug Antibody Assays During the Development of Anti-Tissue Factor Pathway Inhibitor Monoclonal Antibody Marstacimab.
Article
Author: Arkin, Steven ; Jani, Darshana ; Xiang, Yuhong ; Donley, Jean ; Gorovits, Boris ; Zhu, Tong
Tissue factor pathway inhibitor (TFPI) is an endogenous inhibitor of the extrinsic coagulation pathway. In patients with hemophilia A or B, inhibition of TFPI is an alternative therapeutic approach that augments the extrinsic coagulation pathway. Marstacimab is an investigational fully human monoclonal antibody that binds and neutralizes TFPI and is being evaluated as a prophylactic treatment to prevent or reduce the frequency of bleeding episodes in patients with severe hemophilia A or B, with or without inhibitors (antibodies against coagulation factors). However, the efficacy, safety, and pharmacokinetics of marstacimab may be affected by the induction of antidrug antibody (ADA) responses. Here, we describe the evolution and validation of three quasi-quantitative electrochemiluminescence-based methods to detect marstacimab ADAs, starting from their use in a first-in-human phase 1 study to their use in phase 2 and 3 clinical studies of patients with severe hemophilia. For all three methods, validation criteria evaluated the performance of the assays in screening and confirmatory cut points, precision, selectivity, drug tolerance, target interference, and stability. Additional criteria for validation were dilution linearity (Methods 1 and 2) and low positive control concentration, prozone effect, plate homogeneity, and robustness (Method 3). The three methods met validation criteria and are a potentially valuable tool in detecting the induction of marstacimab ADAs during treatment in patients with hemophilia.
01 Jul 2023·Haemophilia : the official journal of the World Federation of Hemophilia
Safety, tolerability, pharmacokinetics and pharmacodynamics of a single dose of marstacimab in Chinese participants with severe haemophilia.
Letter
Author: Sun, Xiaohong ; Liu, Wei ; Zhou, Yangmei ; Peng, Ao ; Tong, Xin ; Luo, Binyu ; Kalluru, Hindu ; Sun, Lei ; Yang, Renchi ; Raje, Sangeeta ; Ma, Dongwei ; Li, Jin ; Teeter, John ; Liu, Yuwang
24
News (Medical) associated with Marstacimab26 Apr 2024
A one-time dose of BEQVEZ has reduced bleeds post-treatment compared to standard of care with a median of zero bleeds (range 0 to 19) after up to three years of follow-up, providing sustained bleed protection and potentially avoiding years of treatment burden with prophylaxis for many patients
NEW YORK--(BUSINESS WIRE)-- Pfizer Inc. (NYSE: PFE) announced today that the U.S. Food and Drug Administration (FDA) has approved BEQVEZ™ (fidanacogene elaparvovec-dzkt) for the treatment of adults with moderate to severe hemophilia B who currently use factor IX (FIX) prophylaxis therapy, or have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes, and do not have neutralizing antibodies to adeno-associated virus serotype Rh74var (AAVRh74var) capsid as detected by an FDA-approved test. BEQVEZ is a one-time treatment that is designed to enable people living with hemophilia B to produce FIX themselves rather than the current standard of care, which requires regular intravenous infusions of FIX that are often administered multiple times a week or multiple times a month.1,2
“Many people with hemophilia B struggle with the commitment and lifestyle disruption of regular FIX infusions, as well as spontaneous bleeding episodes, which can lead to painful joint damage and mobility issues,” said Adam Cuker, M.D., M.S., Director, Penn Comprehensive and Hemophilia Thrombosis Program. “A one-time treatment with BEQVEZ has the potential to be transformative for appropriate patients by reducing both the medical and treatment burden over the long term.”
Hemophilia B is a rare genetic bleeding disorder that prevents normal blood clotting because of a deficiency in FIX that causes those with the disease to bleed more frequently and longer than others.3,4 The standard of care for hemophilia B treatment is prophylactic infusions of FIX replacement therapy that temporarily replace or supplement low levels of blood-clotting factor.2,4 Despite prophylaxis and regular intravenous infusions, many people living with moderate to severe hemophilia B are at risk of spontaneous bleeding episodes.5,6,7 The current standard of care also places strain on healthcare systems’ budgets and resource utilization.6,8,9,10 According to the World Federation of Hemophilia, more than 38,000 people worldwide are living with hemophilia B.11
“This milestone is a testament to Pfizer’s continued effort to advance the standard of care for people living with hemophilia, with the delivery of a medicine that has the potential to offer both long-term bleed protection and value to the healthcare system because of its one-time administration,” said Aamir Malik, Chief U.S. Commercial Officer and Executive Vice President, Pfizer. “We are leveraging our expertise that comes with more than 40 years of experience in the hemophilia space, and are proactively working with treatment centers, payers, and the hemophilia community to appropriately help ensure the healthcare system is prepared to readily deliver BEQVEZ to the patients who can benefit from it.”
With BEQVEZ now approved for use, Pfizer is launching an innovative warranty program based on durability of patient response to treatment. The goal of the warranty is to provide greater certainty to payers, maximize access for eligible patients who receive BEQVEZ, and offer financial protection by insuring against the risk of efficacy failure.
“For people living with hemophilia, disease management can interfere with many aspects of their lives. A one-time infusion of BEQVEZ may allow eligible patients more time for the things they love,” said Kim Phelan, Chief Operating Officer, The Coalition for Hemophilia B. “We are excited to have BEQVEZ as a promising treatment option for eligible people living with hemophilia B. We look forward to learning more and celebrating with the community and with Pfizer at our annual conference that is currently taking place.”
BEQVEZ is currently under review with the European Medicines Agency (EMA), and the treatment recently received regulatory approval in Canada. In addition to BEQVEZ, Pfizer currently has two other Phase 3 programs investigating gene therapy in populations where there is a high unmet need: hemophilia A (giroctocogene fitelparvovec) and Duchenne muscular dystrophy (fordadistrogene movaparvovec). Additionally, a Phase 3 trial is investigating marstacimab, a novel, investigational anti-tissue factor pathway inhibitor for the treatment of people with hemophilia A and B with and without inhibitors. A Biologics License Application and European Marketing Authorization Application for marstacimab are currently under review with the FDA and EMA, respectively.
About BEQVEZ (fidanacogene elaparvovec-dzkt)
BEQVEZ is an adeno-associated virus (AAV)-based gene therapy designed to introduce in the transduced cells a functional copy of the FIX gene encoding a high-activity FIX variant. For eligible patients living with hemophilia B, the goal of this gene therapy is to enable them to produce FIX themselves via this one-time treatment rather than having to receive frequent infusions of FIX, as is the current standard of care.1,2,4 It is currently approved in the U.S. for the treatment of adults with moderate to severe hemophilia B (congenital factor IX deficiency) who:
Currently use factor IX prophylaxis therapy, or
Have current or historical life-threatening hemorrhage, or
Have repeated, serious spontaneous bleeding episodes, and,
Do not have neutralizing antibodies to adeno-associated virus serotype Rh74var (AAVRh74var) capsid as detected by an FDA-approved test.
For eligible patients who are prescribed BEQVEZ, Pfizer offers personalized patient support services including financial assistance resources and logistical support through the Pfizer Gene Together program. More information is available on .
In December 2014, Pfizer licensed BEQVEZ from Spark Therapeutics. Under the agreement, Pfizer assumed responsibility for pivotal studies, any regulatory activities, and potential global commercialization of this gene therapy.
About BENEGENE-2
The FDA approval is based on the results from the pivotal BENEGENE-2 study, a Phase 3, open-label, single-arm study to evaluate the efficacy and safety of BEQVEZ in adult male participants (age 18–65) with moderately severe to severe hemophilia B (defined as FIX circulating activity of 2% or less). The main objective of the study is to evaluate the annualized bleeding rate (ABR) for participants treated with gene therapy versus FIX prophylaxis replacement regimen, administered as part of usual care.
The study enrolled 45 participants. Eligible study participants have completed a minimum of six months of routine FIX prophylaxis therapy during the lead-in study (NCT03587116) and received a single intravenous infusion of BEQVEZ at a dose of 5 x 1011 vg/kg of body weight. Clinical trial participants will be followed for up to a total of 15 years, including six years in the BENEGENE-2 study and an additional nine years as part of a separate Phase 3 study (NCT05568719) to learn about the long-term safety and efficacy of BEQVEZ.
BENEGENE-2 met its primary endpoint of non-inferiority in the ABR of total bleeds post-BEQVEZ infusion versus prophylaxis regimen with FIX, administered as part of usual care. A mean ABR of 2.5 was observed among patients who received BEQVEZ in the efficacy evaluation period – defined as between week 12 and data cutoff (median 1.8 years of follow-up) – after receiving the one-time dose compared to a mean ABR of 4.5 during the lead-in pre-treatment period of at least six months (median 1.2 years of follow-up). Bleeds were eliminated in 60% of patients compared to 29% in the prophylaxis arm. A median ABR of zero (range of 0 to 19) was observed during the efficacy evaluation period compared to the prophylaxis arm in which a median ABR of 1.3 (range of 0 to 53.9) was observed.
BEQVEZ was generally well-tolerated in patients who received it. The most common adverse reaction (incidence ≥5%) reported in Phase 3 and 1/2 clinical studies was an increase in transaminases. No deaths, serious adverse events related to treatment or associated with infusion reactions, thrombotic events, or FIX inhibitors were reported. Elevated transaminases were observed in 26 out of 60 patients treated at the recommended dose and 31 out of 60 patients received corticosteroids.
In addition to the Phase 3 trial, BEQVEZ patients have been followed up to six years in a Phase 1/2a study and its corresponding Phase 2a long-term follow-up study. Pfizer is continuing to monitor for long-term treatment durability and safety in its clinical program over the course of 15 years.
About Hemophilia B
Hemophilia is a rare genetic bleeding disorder that prevents normal blood clotting because of a deficiency in one of several blood clotting factors and is predominately found in males.3,4 People with hemophilia are at risk for excessive and recurrent spontaneous and/or post-traumatic bleeding, which can be life-threatening, particularly in those with severe hemophilia.3,4 People with severe hemophilia often bleed spontaneously into their muscles or joints, or rarely into other critical closed spaces such as the intracranial space, where bleeding can be fatal.3,4
According to the World Federation of Hemophilia, more than 38,000 people worldwide are living with hemophilia B.11 People with hemophilia B have a deficiency in clotting FIX, a specific protein in the blood. Hemophilia B is also called congenital FIX deficiency or Christmas disease. The current standard of care requires recurrent intravenous infusions of either plasma-derived or recombinant FIX to control and prevent bleeding episodes.1,2,4
BEQVEZ (fidanacogene elaparvovec-dzkt) U.S. Important Safety Information
What is BEQVEZ?
BEQVEZ is a one-time gene therapy used for the treatment of adults with moderate to severe hemophilia B who are receiving routine prophylaxis, have a current life-threatening bleed or a history of life-threatening bleeds, or have repeated serious spontaneous bleeds.
Before treatment with BEQVEZ, your healthcare professional will conduct a blood test to check for antibodies to the AAVRh74var virus. The results of this testing will help determine if you may receive BEQVEZ.
Before receiving BEQVEZ, tell your healthcare professional about all your medical conditions, including if you:
Have kidney or liver problems, including hepatitis
Have factor IX inhibitors or a history of factor IX inhibitors
Have an active infection
BEQVEZ may cause serious side effects, including:
Increased Liver Enzymes. Most patients treated with BEQVEZ developed elevated liver enzyme levels and most did not experience any symptoms.
Your healthcare professional will monitor liver enzymes and factor IX activity levels before administration of BEQVEZ and frequently following the administration to detect and identify possible elevations in liver enzymes and to monitor your response to BEQVEZ. Your doctor may prescribe a corticosteroid for the treatment of elevated liver enzymes.
Avoid or limit alcohol consumption during the first year following BEQVEZ infusion, as alcohol may reduce the effect of BEQVEZ and may increase liver enzyme levels.
Infusion reactions, including hypersensitivity and severe allergic reactions (anaphylaxis) may occur. Alert your healthcare professional right away if you get any symptoms of hypersensitivity, which may include but are not limited to low blood pressure, fever, heart palpitation, nausea, vomiting, chills, or headache.
BEQVEZ can insert itself into the DNA of cells in the human body. The effect that insertion may have on those cells is unknown but may contribute to a theoretical risk of cancer. There have been no reported cases of cancer caused by treatment with BEQVEZ.
The most common side effect of BEQVEZ is increased liver enzymes. These are not all the possible side effects of BEQVEZ. For more information, ask your healthcare professional.
Talk to your healthcare professional before receiving any vaccinations if you are taking a corticosteroid.
Talk to your doctor about any medications you plan to take including over the counter medications, herbal supplements, and vitamins as certain substances can affect the liver and may reduce the effectiveness of BEQVEZ.
Your healthcare professional will test your factor IX activity levels and for neutralizing factor IX inhibitors.
After receiving BEQVEZ, your doctor will discuss whether and when you are able to stop prophylaxis, if you need to resume prophylaxis, and actions you may need to take for surgeries, procedures, injuries, and bleeding events.
Do not donate blood, organs, tissues, or cells for transplantation following administration of BEQVEZ.
BEQVEZ is not intended for administration to women. Males should not donate sperm and should use a male condom or not have sexual intercourse for up to 6 months after receiving BEQVEZ.
Patients and caregivers should ensure proper handling of any materials that have come into contact with the patient’s urine, feces, saliva, mucus, or semen in the first 6 months after BEQVEZ infusion.
You are encouraged to report negative side effects of prescription drugs to the FDA. Visit or call 1-800-FDA-1088.
The full Prescribing Information can be found here. If it is not currently available via this link, it will be visible as soon as possible as we work to finalize the document. Please check back for the full information shortly.
About Pfizer: Breakthroughs That Change Patients’ Lives
At Pfizer, we apply science and our global resources to bring therapies to people that extend and significantly improve their lives. We strive to set the standard for quality, safety and value in the discovery, development and manufacture of health care products, including innovative medicines and vaccines. Every day, Pfizer colleagues work across developed and emerging markets to advance wellness, prevention, treatments and cures that challenge the most feared diseases of our time. Consistent with our responsibility as one of the world's premier innovative biopharmaceutical companies, we collaborate with health care providers, governments and local communities to support and expand access to reliable, affordable health care around the world. For 175 years, we have worked to make a difference for all who rely on us. We routinely post information that may be important to investors on our website at . In addition, to learn more, please visit us on and follow us on X at @Pfizer and @Pfizer_News, LinkedIn, YouTube and like us on Facebook at .
Category: Prescription Medicines
Disclosure notice
The information contained in this release is as of April 26, 2024. Pfizer assumes no obligation to update forward-looking statements contained in this release as the result of new information or future events or developments.
This release contains forward-looking information about BEQVEZ, a gene therapy, including its potential benefits and an approval in the U.S. of BEQVEZ for the treatment of adult patients with hemophilia B, and Pfizer’s other investigational gene therapy and hemophilia candidates that involves substantial risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements. Risks and uncertainties include, among other things, uncertainties regarding the commercial success of BEQVEZ; the uncertainties inherent in research and development, including the ability to meet anticipated clinical endpoints, commencement and/or completion dates for our clinical trials, regulatory submission dates, regulatory approval dates and/or launch dates, as well as the possibility of unfavorable new clinical data and further analyses of existing clinical data; the risk that clinical trial data are subject to differing interpretations and assessments by regulatory authorities; whether regulatory authorities will be satisfied with the design of and results from our clinical studies; whether and when drug applications may be filed in particular jurisdictions for BEQVEZ or any other product candidates; whether and when any applications that may be pending or filed for BEQVEZ or any other product candidates may be approved by regulatory authorities, which will depend on myriad factors, including making a determination as to whether the product's benefits outweigh its known risks and determination of the product's efficacy and, if approved, whether BEQVEZ or any such other product candidates will be commercially successful; decisions by regulatory authorities impacting labeling, manufacturing processes, safety and/or other matters that could affect the availability or commercial potential of BEQVEZ or any other product candidates; uncertainties regarding the impact of COVID-19 on Pfizer’s business, operations and financial results; and competitive developments.
A further description of risks and uncertainties can be found in Pfizer’s Annual Report on Form 10-K for the fiscal year ended December 31, 2023, and in its subsequent reports on Form 10-Q, including in the sections thereof captioned “Risk Factors” and “Forward-Looking Information and Factors That May Affect Future Results”, as well as in its subsequent reports on Form 8-K, all of which are filed with the U.S. Securities and Exchange Commission and available at and .
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1 Ohmori T, Mizukami H, Ozawa K, et al. New approaches to gene and cell therapy for hemophilia. J Thromb Haemost. 2015;13(Suppl 1): S133-142.
2 Furlan R, Krishnan S, Vietri J. Patient and parent preferences for characteristics of prophylactic treatment in hemophilia. Patient Prefer Adherence. 2015; 9:1687-1694.
3 Mayo Clinic. Hemophilia. August 2023. Available at: . Last accessed: April 2024.
4 Centers for Disease Control and Prevention. What is hemophilia? October 2023. Available at: . Last accessed: April 2024.
5 Srivastava A, Santagostino E, Dougall A, et al. WFH Guidelines for the Management of Hemophilia, 3rd edition. Haemophilia. 2020;26 (Suppl 6):1-158.
6 Burke T, Asghar S, O'Hara J, et al. Clinical, humanistic, and economic burden of severe hemophilia B in the United States: results from the CHESS US and CHESS US+ population surveys. Orphanet J Rare Dis. 2021;16(1):143.
7 van Overbeeke E, Hauber B, Michelsen, et al. Patient preferences for gene therapy in haemophilia: Results from the PAVING threshold technique survey. Haemophilia. 2021;27(6):957-966.
8 Buckner TW, Bocharova I, Hagan K, et al. Health care resource utilization and cost burden of hemophilia B in the United States. Blood Adv. 2021;5(7):1954-1962.
9 Chen CX, Baker JR, Nichol MB. Economic burden of illness among persons with hemophilia B from HUGS Vb: examining the association of severity and treatment regimens with costs and annual bleed rates. Value Health. 2017;20(8):1074-1082.
10 O’Hara J, Hughes D, Camp C, et al. The cost of severe haemophilia in Europe: the CHESS study. Orphanet J Rare Dis. 2017;12(1):106.
11 World Federation of Hemophilia. Report on the annual global survey 2021. October 2022. Available at: . Last accessed: April 2024.
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Clinical ResultPhase 3Phase 2Gene TherapyLicense out/in
01 Apr 2024
The year started well for the biotechnology industry. Steady dealmaking coupled with a rebound in initial public offerings helped lift investor confidence and, with it, drugmaker stocks.While that optimism has moderated somewhat since, a series of decisions by the Food and Drug Administration over the next three months could help the sector sustain its upturn. The agency may expand approvals for two cancer cell therapies and clear a third vaccine for respiratory syncytial virus. Sanofi and Regeneron await a decision on a lucrative new use for their top-selling Dupixent, while Geron could see decades of drug research finally pay off.Here are five FDA decisions to watch in the second quarter.CAR-T therapies for early multiple myeloma treatmentThe FDA this quarter could broaden the use of two cell therapies for multiple myeloma in decisions potentially worth billions of dollars to their respective developers.By April 5, the agency will decide whether to clear Johnson & Johnson and Legend Biotechs Carvykti for use in myeloma patients as early as after one relapse. That decision could be accompanied by a similar OK for Bristol Myers Squibbs Abecma and open up use of the therapies, which are currently only available in the U.S. for people with more advanced disease.European regulators have already endorsed earlier use of Abecma and Carvykti based on trial data showing they help people live longer without their disease progressing. But the FDA has appeared less supportive. The agency delayed a decision on Abecma last year and held a March advisory meeting to more closely scrutinize the data for it and Carvykti. In briefing documents, staff scientists flagged an apparent increase in the risk of death early on in the clinical trials of both therapies.The companies attributed that finding to limitations in the bridging therapies study participants could receive while waiting for CAR-T treatment. Panelists were convinced and backed both, agreeing that those issues likely wouldnt occur as frequently in practice.Nonetheless, the regulatory scrutiny suggests that the FDA might put new safety restrictions on Carvykti and Abecma if it chooses to clear them for earlier use. Jonathan GardnerModernas vaccine for RSVCOVID-19 vaccine makers Pfizer and Moderna have been facing the downside of their pandemic success, as falling sales have brought tough questions from investors.Moderna, for its part, aims to follow its COVID shot with several other messenger RNA-based vaccines, including one for respiratory syncytial virus thats now under FDA review.Respiratory syncytial virus, or RSV, can cause severe illnessin infants, older adults and people with compromised immune systems. Two vaccines, from GSK and Pfizer, are already approved to prevent RSV-related disease in adults over the age of 60. Sales of each have been strong, although GSK has established a clear edge.Moderna argues its vaccine, dubbed mRNA-1345, can compete. The shot is a pre-filled, single-dose vaccine, potentially offering more convenient administration.In Phase 3 testing, the vaccine was 84% effective at preventing infection with two or more symptoms in older adults. That efficacy does appear to wane over time, however.At a recent meeting, Centers for Disease Control and Prevention advisers reviewed known safety risks for GSKs and Pfizers shots, both of which were associated in very rare cases with Guillain-Barr syndrome, or GBS, a neurological condition affecting the nerves. Moderna has not reported any cases of GBS in Phase 3 testing of its shot.An FDA decision on mRNA-1345 is set for May 12. Delilah AlvaradoGerons imetelstat for myelodysplastic syndromesDrug development is a time-consuming, expensive process. Geron, a biotech founded 34 years ago to pursue stem cell research, knows this well.The companys long journey may finally reach an end by June 16, when the FDA will decide whether to approve a bone marrow disease drug Geron has been developing for nearly two decades. Called imetelstat, the drug treats myelodysplastic syndromes and some analysts believe it has blockbuster potential.Over the many years of Gerons research, imetelstat failed studies in multiple cancer types, and the company lost Johnson & Johnson as a partner along the way. But the drug finally succeeded in Phase 3 testing for myelodysplastic syndromes last year. And in March, it secured the endorsement of an FDA advisory panel despite skepticism from agency reviewers.Geron aims to challenge Bristol Myers Squibbs Reblozyl, first with a clearance in people with lower-risk disease and then in more people who have the condition. Stifel analyst Stephen Wiley estimated the drug could generate $2 billion in peak yearly sales, as it could be helpful in people who dont respond well to Reblozyl.Geron could be a buyout target, too, according to Wiley, though the company has said it plans to sell the drug on its own. Ben FidlerSanofi and Regenerons Dupixent for COPDDupixent is one of the pharmaceutical industrys most widely used and lucrative products. Approved for asthma, eczema and three other inflammatory conditions, the drug earned the companies more than $11 billion in sales last year.By June 27, the FDA could add another major indication to Dupixents list of approved uses. The regulator is currently reviewing the injectable medicine as a treatment for a type of chronic obstructive pulmonary disease, or COPD.If cleared by the FDA, Dupixent would be the first biologic drug for people with moderate-to-severe COPD who also have evidence of whats called Type 2 inflammation. A pair of late-stage studies, which enrolled nearly 1,900 people, showed Dupixent could cut the rate of COPD attacks by about one-third. Analysts therefore expect the FDA to issue an approval and forecast billions of dollars in additional yearly sales. Ned PagliaruloPfizers gene therapy for hemophilia BSome time during the second quarter, the FDA will decide whether to clear a gene therapy from Pfizer for hemophilia B, the less common form of the blood clotting disorder.Reaching this point has been a long time coming for Pfizer, which licensed the therapy from Spark Therapeutics a decade ago. In subsequent testing, treatmentlowered bleeding rates and reduced the need for regular blood transfusions powerful benefits meant to last many years with a single infusion.But its unclear whether Pfizer will find a market if the FDA chooses to approve its therapy. A rival genetic medicine, from CSL Behring and UniQure, won U.S. approval for hemophilia B in November 2022. However, few patients have been treated to date, sluggish uptake that could reflect the breadth of effective options available.The story is similar with Roctavian, a gene therapy BioMarin Pharmaceutical sells for the more common A form of hemophilia. Only three patients received Roctavian last year: two in Germany and one in the U.S.Pfizers therapy, which in January won Canadian approval, could provide another data point. But the company seems to be downplaying its potential. In recent presentations for investors, a once-weekly shot called marstacimab has gotten higher billing. Ned Pagliarulo '
Phase 3VaccineDrug ApprovalClinical ResultmRNA
30 Jan 2024
2023 Sets Stage for Future Growth Potential: Completed Seagen Acquisition, Creating World-Class Oncology Organization; Launched Significant Number of New Products and Indications; and Realigned Commercial Organization to Improve Focus, Speed and Execution
Full-Year 2023 Revenues of $58.5 Billion
Expected Decline in Comirnaty(1) and Paxlovid Revenues Drove 41% Operational Decrease in Year-Over-Year Revenues
Excluding Contributions from Comirnaty(1) and Paxlovid, Revenues Grew 7% Operationally, Driven by a Combination of New Product and Indication Launches and In-Line Product Growth
Full-Year 2023 Reported(2) Diluted EPS of $0.37, Down 93% Year-Over-Year, and Adjusted(3) Diluted EPS of $1.84, Down 72% Year-Over-Year, Significantly Impacted by One-Time Events(4)
Fourth-Quarter 2023 Revenues of $14.2 Billion
Expected Decline in Comirnaty(1) and Paxlovid Revenues Drove 42% Operational Decrease in Fourth-Quarter Revenues
Excluding Contributions from Comirnaty(1) and Paxlovid, Revenues Grew 8% Operationally
Fourth-Quarter 2023 Reported(2) Diluted Loss Per Share (LPS) of $(0.60) and Adjusted(3) Diluted EPS of $0.10, Significantly Impacted by One-Time Events(4)
On Track to Deliver at Least $4 Billion in Annual Net Cost Savings by End of 2024 from Previously Announced Cost Realignment Program
Reaffirms Full-Year 2024 Guidance(5) Provided on December 13, 2023, of Revenues of $58.5 to $61.5 Billion and Adjusted(3) Diluted EPS of $2.05 to $2.25
NEW YORK--(BUSINESS WIRE)-- Pfizer Inc. (NYSE: PFE) reported financial results for fourth quarter and full-year 2023 and reaffirmed its 2024 financial guidance(5) provided on December 13, 2023.
The fourth-quarter 2023 earnings presentation and accompanying prepared remarks from management as well as the quarterly update to Pfizer’s R&D pipeline can be found at .
EXECUTIVE COMMENTARY
Dr. Albert Bourla, Chairman and Chief Executive Officer, stated: “We are encouraged by the strong performance of our non-COVID products in the fourth quarter of 2023, including significant contributions from new launches and robust year-over-year growth for several key in-line brands. In 2023, Pfizer received a record number of nine new molecular entity approvals by the U.S. Food and Drug Administration (FDA)—medicines and vaccines that are expected to favorably impact Pfizer’s performance in the coming years.
“In addition, we completed the acquisition of Seagen in December 2023, a critical step toward our goal to achieve world-class Oncology leadership. With the combined strength of Pfizer’s and Seagen’s talent, portfolios and platforms, we believe we have the potential to transform outcomes by delivering cancer medicines that help patients live better and longer lives. We look forward to sharing the vision of the new Pfizer Oncology Division at our announced Oncology Innovation Day on Thursday, February 29.
“We are entering 2024 with a solid foundation. We believe our commitment to execution, maximizing the performance of our new products, and delivering the next wave of pipeline innovation will fuel Pfizer’s growth and make a difference in the lives of patients everywhere.”
David Denton, Chief Financial Officer and Executive Vice President, stated: “We are pleased with the strong 8% operational revenue growth of Pfizer’s non-COVID products in the fourth quarter of 2023, achieving our full-year 2023 non-COVID operational revenue growth target of 6% to 8%. In addition, we are on track to deliver at least $4.0 billion in annual net cost savings by the end of 2024 from our cost realignment program. We are prepared to execute our commercial strategy to drive continued growth from our newly launched and acquired products, and to deliver on our targeted cost savings that we expect will expand our operating margins in 2024 and beyond.”
Results for fourth-quarter and full-year 2023 and 2022(6) are summarized below.
OVERALL RESULTS
($ in millions, except
per share amounts)
Fourth-Quarter
Full-Year
2023
2022
Change
2023
2022
Change
Revenues
$ 14,249
$ 24,290
(41%)
$ 58,496
$ 100,330
(42%)
Reported(2) Net Income/(Loss)
(3,369)
4,995
*
2,119
31,372
(93%)
Reported(2) Diluted EPS/(LPS)
(0.60)
0.87
*
0.37
5.47
(93%)
Adjusted(3) Income
593
6,551
(91%)
10,501
37,717
(72%)
Adjusted(3) Diluted EPS
0.10
1.14
(91%)
1.84
6.58
(72%)
* Indicates calculation not meaningful.
REVENUES
($ in millions)
Fourth-Quarter
Full-Year
2023
2022
% Change
2023
2022
% Change
Total
Oper.
Total
Oper.
Global Biopharmaceuticals Business (Biopharma)
$ 13,867
$ 23,922
(42%)
(42%)
$ 57,186
$ 98,988
(42%)
(41%)
Primary Care
6,986
17,348
(60%)
(60%)
30,589
73,023
(58%)
(57%)
Specialty Care
3,949
3,566
11%
11%
14,970
13,833
8%
11%
Oncology
2,932
3,007
(3%)
(2%)
11,627
12,132
(4%)
(3%)
Business Innovation
$ 382
$ 368
4%
2%
$ 1,310
$ 1,342
(2%)
(2%)
TOTAL REVENUES
$ 14,249
$ 24,290
(41%)
(42%)
$ 58,496
$ 100,330
(42%)
(41%)
In the first quarter of 2023, Pfizer established an operating segment, Business Innovation, that includes Pfizer CentreOne (PC1), the company’s global contract development and manufacturing organization and a leading supplier of specialty active pharmaceutical ingredients; and Pfizer Ignite, an offering that provides strategic guidance and end-to-end R&D services to select innovative biotech companies that align with Pfizer’s R&D focus areas. The prior period has been revised to conform to the current period presentation.
Some amounts in this press release may not add due to rounding. All percentages have been calculated using unrounded amounts. References to operational variances pertain to period-over-period changes that exclude the impact of foreign exchange rates(7).
CAPITAL ALLOCATION
During full-year 2023, Pfizer deployed its capital in a variety of ways, which primarily include the following two categories:
Reinvesting capital into initiatives intended to enhance the future growth prospects of the company, including:
$10.7 billion invested in internal research and development projects, and
Approximately $43.8 billion invested in completed business development transactions, net of cash acquired, including approximately $43 billion for the acquisition of Seagen Inc.
Returning capital directly to shareholders through $9.2 billion of cash dividends, or $1.64 per share of common stock.
No share repurchases were completed in 2023. As of January 30, 2024, Pfizer’s remaining share repurchase authorization is $3.3 billion. Current financial guidance does not anticipate any share repurchases in 2024.
For the fourth quarter of 2023, basic weighted-average shares outstanding of 5,647 million were used to calculate Reported(2) LPS and diluted weighted-average shares outstanding of 5,692 million were used to calculate Adjusted(3) diluted EPS.
2024 FINANCIAL GUIDANCE(5)
Pfizer expects full-year 2024 revenues(2) to be in the range of $58.5 to $61.5 billion, which includes approximately $8 billion in anticipated revenues for Comirnaty(1) and Paxlovid (approximately $5 billion and $3 billion, respectively), approximately $3.1 billion in anticipated revenues from Seagen and approximately $1 billion related to the reclassification of Pfizer’s royalty income from Other (Income)/Deductions into the Revenue line.
Including the contribution from Seagen and excluding revenues from Comirnaty(1) and Paxlovid, Pfizer expects to achieve full-year 2024 operational revenue growth of 8% to 10% compared to 2023 revenues. Excluding revenues from Comirnaty(1) and Paxlovid and the expected contribution from Seagen, Pfizer expects to achieve full-year 2024 operational revenue growth of 3% to 5% compared to 2023 revenues. While the company will begin reporting royalty income in the revenue line in 2024, for growth rate purposes, the company has included royalty income in revenues in both 2023 and 2024. Consequently, there is no operational revenue growth attributable to the reclassification of royalty income.
Including the impact of Seagen, Pfizer anticipates full-year 2024 Adjusted(3) SI&A expenses to be in the range of $13.8 billion to $14.8 billion and full-year 2024 Adjusted(3) R&D expenses to be in the range of $11.0 to $12.0 billion. Consequently, total 2024 Adjusted(3) SI&A and R&D expenses are expected to be in the range of $24.8 to $26.8 billion. This range reflects an anticipated decline of approximately $4 billion by the end of 2024 versus the midpoint of Pfizer’s SI&A and R&D expense guidance provided on August 1, 2023, solely driven by Pfizer’s cost realignment program, partially offset by the impact of Seagen.
2024 Adjusted(3) diluted EPS is anticipated to be in a range of $2.05 to $2.25, which primarily reflects:
Expected operational revenue growth of 8% to 10% compared to 2023 revenues, excluding Comirnaty(1) and Paxlovid and including the impact of Seagen; and
Anticipated operating margin improvement from the company’s cost realignment activities; partially offset by
An expected $0.40 dilutive impact related to the Seagen acquisition, which is predominantly driven by costs to finance the transaction.
Pfizer’s 2024 financial guidance(5), including the impact of certain significant factors, is presented below.
2023
Results
2024 Legacy
Pfizer
Guidance
Anticipated
Impact of Royalty
Income Reclass
Included in 2024
Guidance
Anticipated 2024
Seagen Impact
Included in 2024
Guidance
2024 Financial
Guidance(5)
Revenues ($ in billions)
$58.5
$54.5 to $57.5
$1.0
$3.1
$58.5 to $61.5
Adjusted(3) SI&A Expenses ($ in billions)
$14.4
$13.8 to $14.8
Adjusted(3) R&D Expenses ($ in billions)
$10.6
$11.0 to $12.0
Effective Tax Rate on Adjusted(3) Income
9.0%
~15.0%
Adjusted(3) Diluted EPS
$1.84
$2.45 to $2.65
-
$(0.40)
$2.05 to $2.25
Financial guidance for Adjusted(3) diluted EPS is calculated using approximately 5.75 billion weighted average shares outstanding and assumes no share repurchases in 2024.
QUARTERLY FINANCIAL HIGHLIGHTS (Fourth-Quarter 2023 vs. Fourth-Quarter 2022)
Fourth-quarter 2023 revenues totaled $14.2 billion, a decrease of $10.0 billion, or 41%, compared to the prior-year quarter, reflecting an operational decline of $10.1 billion, or 42%, primarily due to a significant decrease in Comirnaty(1) and Paxlovid revenues globally, as well as a de minimis impact of foreign exchange. Excluding contributions from Comirnaty(1) and Paxlovid, company revenues grew $934 million, or 8%, operationally.
Fourth-quarter 2023 Comirnaty(1) revenues declined $6.1 billion, or 54%, operationally compared with the prior-year quarter, largely driven by lower U.S. government contracted deliveries following the transition to traditional U.S. commercial market sales, which began in September 2023, and by lower contracted deliveries and demand in international markets.
Fourth-quarter 2023 Paxlovid revenues declined $5.0 billion, to $(3.1) billion, compared with the prior-year quarter, primarily driven by a non-cash revenue reversal of $3.5 billion recorded in the fourth quarter of 2023, of which a portion was associated with sales recorded in 2022, related to the expected return of an estimated 6.5 million treatment courses of Emergency Use Authorization (EUA)-labeled U.S. government inventory; partially offset by sales under traditional commercial markets following transition, primarily in the U.S.
Excluding contributions from Comirnaty(1) and Paxlovid, fourth-quarter 2023 operational revenue growth was primarily driven by:
Abrysvo, which contributed $515 million in global revenues, driven primarily by launch of the older adult indication in the U.S. in July 2023;
Vyndaqel family (Vyndaqel, Vyndamax, Vynmac) globally, up 39% operationally, driven largely by continued strong uptake of the transthyretin amyloid cardiomyopathy (ATTR-CM) indication, primarily in the U.S. and developed Europe; and
Eliquis globally, up 9% operationally, driven primarily by continued oral anti-coagulant adoption and market share gains in the non-valvular atrial fibrillation indication in the U.S. and certain markets in Europe, partially offset by declines due to loss of exclusivity and generic competition in certain international markets;
partially offset primarily by lower revenues for:
Ibrance globally, down 13% operationally, driven primarily by lower demand globally due to competitive pressure and lower clinical trial purchases in certain international markets; and
Prevnar family (Prevnar 13 & 20) globally, down 7% operationally, driven primarily by the pediatric indication in emerging markets due to lower demand and unfavorable timing of customer orders.
GAAP Reported(2) Statement of Operations Highlights
SELECTED REPORTED(2) COSTS AND EXPENSES
($ in millions)
Fourth-Quarter
Full-Year
2023
2022
% Change
2023
2022
% Change
Total
Oper.
Total
Oper.
Cost of Sales(2)
$ 7,562
$ 9,648
(22%)
(24%)
$ 24,954
$ 34,344
(27%)
(29%)
Percent of Revenues
53.1%
39.7%
N/A
N/A
42.7%
34.2%
N/A
N/A
SI&A Expenses(2)
4,575
4,644
(1%)
(2%)
14,771
13,677
8%
9%
R&D Expenses(2)
2,815
3,615
(22%)
(22%)
10,679
11,428
(7%)
(6%)
Acquired IPR&D Expenses(2)
73
73
—
—
194
953
(80%)
(80%)
Other (Income)/Deductions––net(2)
(480)
(846)
(43%)
(51%)
(835)
217
*
*
Effective Tax Rate on Reported(2) Income/(Loss)
19.2%
4.4%
(105.4%)
9.6%
* Indicates calculation not meaningful.
Fourth-quarter 2023 Cost of Sales(2) as a percentage of revenues increased by 13.4 percentage points compared with the prior-year quarter, driven primarily by the $3.5 billion non-cash Paxlovid revenue reversal; unfavorable changes in sales mix, primarily due to lower sales of Paxlovid and Comirnaty(1); and the unfavorable impact of foreign exchange.
Fourth-quarter 2023 SI&A Expenses(2) decreased 2% operationally compared with the prior-year quarter, driven primarily by a decrease in marketing and promotional expenses for Paxlovid, a lower provision for U.S. healthcare reform fees related to Paxlovid and Comirnaty(1), and lower compensation-related expenses, partially offset by increases in marketing and promotional expenses, including those related to recently launched and acquired products, as well as increased investments to build new internal marketing capabilities.
Fourth-quarter 2023 R&D Expenses(2) decreased 22% operationally compared with the prior-year quarter, driven primarily by lower compensation-related expenses, as well as by lower spending across (i) vaccine programs, (ii) certain acquired assets and (iii) for ongoing rare disease programs.
The unfavorable period-over-period change in Other income—net(2) of $366 million for the fourth quarter of 2023, compared to the fourth quarter of 2022, was driven primarily by higher intangible asset impairment charges, partially offset by an increase in net gains on equity securities.
Pfizer’s positive effective tax rate for the fourth quarter of 2023 reflects a tax benefit on a pre-tax Reported(2) loss and includes changes in the jurisdictional mix of earnings and the resolution of uncertain tax positions in various markets.
Adjusted(3) Statement of Operations Highlights
SELECTED ADJUSTED(3) COSTS AND EXPENSES
($ in millions)
Fourth-Quarter
Full-Year
2023
2022
% Change
2023
2022
% Change
Total
Oper.
Total
Oper.
Adjusted(3) Cost of Sales
$ 7,265
$ 9,475
(23%)
(26%)
$ 23,988
$ 34,096
(30%)
(31%)
Percent of Revenues
51.0%
39.0%
N/A
N/A
41.0%
34.0%
N/A
N/A
Adjusted(3) SI&A Expenses
4,471
4,414
1%
1%
14,446
13,049
11%
12%
Adjusted(3) R&D Expenses
2,770
3,610
(23%)
(24%)
10,568
11,409
(7%)
(7%)
Adjusted(3) Other (Income)/Deductions––net
(815)
(656)
24%
14%
(2,281)
(1,954)
17%
8%
Effective Tax Rate on Adjusted(3) Income/(Loss)
(24.0%)
11.1%
9.0%
11.7%
Reconciliations of certain Reported(2) to non-GAAP Adjusted(3) financial measures and associated footnotes can be found in the financial tables section of the press release located at the hyperlink below.
FULL-YEAR REVENUE SUMMARY (Full-Year 2023 vs. Full-Year 2022)
Full-year 2023 revenues totaled $58.5 billion, a decrease of $41.8 billion, or 42%, compared to full-year 2022, reflecting an operational decline of $40.8 billion, or 41%, and an unfavorable impact of foreign exchange of $1.0 billion, or 1%. Excluding contributions from Comirnaty(1) and Paxlovid, revenues for the full-year grew 7% operationally.
The operational revenue decline compared to the prior year was driven primarily by significantly lower global revenues for Comirnaty(1) and Paxlovid and, to a much lesser extent, lower revenues for Ibrance, partially offset by an increase in revenues from Nurtec ODT/Vydura and Oxbryta, which were acquired in the fourth quarter of 2022; revenues from Abrysvo, primarily driven by the launch of the older adult indication in the U.S.; and continued growth from the Vyndaqel family and Eliquis.
RECENT NOTABLE DEVELOPMENTS (Since October 31, 2023)
Product Developments
Elrexfio (elranatamab-bcmm) – In December 2023, Pfizer announced the European Commission (EC) granted conditional marketing authorization for Elrexfio for the treatment of adult patients with relapsed and refractory multiple myeloma who have received at least three prior therapies, including a proteasome inhibitor, an immunomodulatory agent and an anti-CD38 antibody and have demonstrated disease progression on the last therapy.
Padcev (enfortumab vedotin-ejfv)
In January 2024, Pfizer and Astellas Pharma Inc. (Astellas) announced that the European Medicines Agency (EMA) validated for review a Type II variation application for Padcev, an antibody-drug conjugate (ADC), with Keytruda(8) (pembrolizumab, a PD-1 inhibitor) as a combination therapy for the first-line treatment of adult patients with previously untreated locally advanced or metastatic urothelial cancer (la/mUC). The EMA’s Committee for Medicinal Products for Human Use (CHMP), and subsequently the EC, are expected to share their opinions and decisions on the Type II variation application in calendar year 2024.
In December 2023, Pfizer and Astellas announced that the FDA approved Padcev with Keytruda(8) for the treatment of adult patients with la/mUC based on the results from the Phase 3 EV-302 clinical trial. This combination is the first approved to offer an alternative to platinum-containing chemotherapy, the current standard of care in first-line la/mUC.
Prevnar 20 (20-valent pneumococcal conjugate vaccine) – In January 2024, Pfizer announced the CHMP of the EMA adopted a positive opinion, recommending the granting of a marketing authorization for its 20-valent pneumococcal conjugate vaccine candidate for active immunization for the prevention of invasive disease, pneumonia and acute otitis media caused by Streptococcus pneumoniae in infants, children and adolescents from 6 weeks to less than 18 years of age. The CHMP’s positive opinion will now be reviewed by the EC to decide whether to approve the vaccine. This decision is expected in the coming weeks and will apply to all 27 European Union (EU) member states plus Iceland, Liechtenstein and Norway.
Talzenna (talazoparib) – In January 2024, Pfizer announced that the EC approved Talzenna, an oral poly ADP-ribose polymerase (PARP) inhibitor, in combination with Xtandi (enzalutamide), for the treatment of adult patients with metastatic castration-resistant prostate cancer (mCRPC) in whom chemotherapy is not clinically indicated. With this approval, Talzenna is now the first and only PARP inhibitor licensed in the EU for use with Xtandi for patients with mCRPC, with or without gene mutations. The approval is valid in all 27 EU member states plus Iceland, Liechtenstein and Norway.
Tivdak (tisotumab vedotin-tftv) – In January 2024, Pfizer and Genmab A/S announced the FDA accepted the supplemental Biologics License Application (sBLA) seeking to convert the accelerated approval of Tivdak, an ADC, to full approval, for the treatment of patients with recurrent or metastatic cervical cancer with disease progression on or after first-line therapy. The sBLA is supported by efficacy and safety data from the global, randomized, Phase 3 innovaTV 301 trial. The application was granted Priority Review with a Prescription Drug User Fee Act (PDUFA) goal date of May 9, 2024.
Velsipity (etrasimod) – In December 2023, Pfizer announced the EMA’s CHMP adopted a positive opinion for Velsipity, an oral, once-daily, selective sphingosine-1-phosphate (S1P) receptor modulator for the treatment of patients 16 years of age and older with moderately to severely active ulcerative colitis (UC) who have had an inadequate response, lost response, or were intolerant to either conventional therapy or a biological agent. The EC will review the CHMP recommendation and is expected to make a final decision in the coming months.
Xtandi (enzalutamide) – In November 2023, Astellas and Pfizer announced the FDA approved a supplemental New Drug Application (sNDA) for Xtandi following FDA expedited development and review programs (Priority Review designation, Fast Track designation, Real-time Oncology Review), based on results from the Phase 3 EMBARK trial. With this approval, Xtandi becomes the first and only androgen receptor signaling inhibitor approved by the FDA for the treatment of patients with nonmetastatic castration-sensitive prostate cancer (nmCSPC) with biochemical recurrence at high risk for metastasis (high-risk BCR). Patients with nmCSPC with high-risk BCR may be treated with Xtandi with or without a gonadotropin-releasing hormone (GnRH) analog therapy.
Pipeline Developments
A comprehensive update of Pfizer’s development pipeline was published today and is now available at . It includes an overview of Pfizer’s research and a list of compounds in development with targeted indication and phase of development, as well as mechanism of action for some candidates in Phase 1 and all candidates from Phase 2 through registration.
Danuglipron – In December 2023, Pfizer announced topline data from the Phase 2b clinical trial investigating its oral glucagon-like peptide-1 receptor agonist (GLP-1RA) candidate danuglipron in adults with obesity and without type 2 diabetes. The study met its primary endpoint demonstrating statistically significant change in body weight from baseline. While the most common adverse events were mild and gastrointestinal in nature consistent with the mechanism, high rates were observed. High discontinuation rates were seen across all doses compared to placebo. No new safety signals were reported, and treatment with danuglipron was not associated with increased incidence of liver enzyme elevation compared to placebo. Future development of danuglipron will be focused on a once-daily formulation, with pharmacokinetic data anticipated in the first half of 2024, which will help to inform a potential path forward.
Marstacimab
In December 2023, Pfizer announced the FDA had accepted the company’s Biologics License Application (BLA) for its anti-tissue factor pathway inhibitor (anti-TFPI) candidate marstacimab for individuals living with hemophilia A or hemophilia B without inhibitors to Factor VIII (FVIII) or Factor IX (FIX). A marketing authorization application for marstacimab is also currently under review by the EMA. The FDA has set a PDUFA action date in the fourth quarter of 2024, and a decision from the EC is anticipated by the first quarter of 2025.
In December 2023, Pfizer presented results from the pivotal Phase 3 BASIS clinical trial evaluating marstacimab for the treatment of people with severe hemophilia A and moderately severe to severe hemophilia B without inhibitors to FVIII or FIX. Results from the BASIS trial, which were presented at the 2023 American Society of Hematology Annual Meeting and Exposition, demonstrated a statistically significant and clinically meaningful effect on annualized bleeding rate. The safety pro marstacimab was consistent with Phase 1/2 results, and treatment was generally well-tolerated in the study. No deaths were reported, and there have been no thromboembolic events or events of consumptive coagulopathy recorded in hemophilia patients in clinical trials investigating marstacimab.
Vepdegestrant (ARV-471) – In December 2023, Arvinas, Inc. and Pfizer presented interim results from the Phase 1b trial evaluating vepdegestrant, a novel oral PROteolysis TArgeting Chimera (PROTAC) estrogen receptor (ER) degrader, in combination with palbociclib (Ibrance). Study data from the combination cohort demonstrated encouraging clinical activity in heavily pre-treated patients with a median of four lines of therapy across disease settings with locally advanced or metastatic ER positive/human epidermal growth factor 2 (HER2) negative (ER+/HER2-) breast cancer. These data were presented in a spotlight presentation at the 2023 San Antonio Breast Cancer Symposium.
VLA15 (Lyme Disease Vaccine Candidate) – In December 2023, Pfizer and Valneva SE announced the completion of recruitment for the Phase 3 VALOR trial evaluating VLA15, an investigational multivalent protein subunit vaccine for Lyme disease. The trial builds on previous positive Phase 1 and 2 trial results and includes both adult and pediatric participants, with the aim to confirm the efficacy, safety, lot consistency and immunogenicity of VLA15. The VALOR trial is expected to be concluded by the end of 2025.
Corporate Developments
In December 2023, Pfizer announced the completion of its acquisition of Seagen for $229 per share, in cash. The total fair value of the consideration transferred was $44 billion ($43 billion, net of cash acquired). To address U.S. Federal Trade Commission concerns, Pfizer has chosen to irrevocably donate the rights of royalties from sales of Bavencio(9) (avelumab) in the U.S. to the American Association for Cancer Research. With the addition of Seagen’s four in-line medicines, Adcetris (brentuximab vedotin), Padcev (enfortumab vedotin), Tivdak (tisotumab vedotin) and Tukysa (tucatinib), Pfizer’s industry-leading Oncology portfolio now includes over 25 approved medicines and biosimilars across more than 40 indications, including nine medicines that are either blockbuster or have the potential to be blockbuster.
In December 2023, Pfizer announced changes in its commercial organization to incorporate Seagen and improve focus, speed and quality of execution, effective January 1, 2024. Three new organizations have been created, each led by a member of Pfizer’s executive leadership team reporting to Dr. Albert Bourla, Chairman and Chief Executive Officer: (1) the Pfizer Oncology Division, which brings together U.S. oncology commercial operations from both Pfizer and Seagen and is led by Chris Boshoff, M.D., Ph.D., Chief Oncology Officer, Executive Vice President, who also leads Pfizer’s newly combined global oncology R&D operations; (2) the Pfizer U.S. Commercial Division, led by Aamir Malik, Chief U.S. Commercial Officer, Executive Vice President; and (3) the Pfizer International Commercial Division, led by Alexandre de Germay, who has joined Pfizer as Chief International Commercial Officer, Executive Vice President.
Please find Pfizer’s press release and associated financial tables, including reconciliations of certain GAAP reported to non-GAAP adjusted information, at the following hyperlink:
(Note: If clicking on the above link does not open a new webpage, you may need to cut and paste the above URL into your browser's address bar.)
For additional details, see the financial schedules and product revenue tables attached to the press release located at the hyperlink above, and the attached disclosure notice.
(1)
As used in this document, “Comirnaty” refers to, as applicable, and as authorized or approved, the Pfizer-BioNTech COVID-19 Vaccine, the Pfizer-BioNTech COVID-19 Vaccine, Bivalent (Original and Omicron BA.4/BA.5), Comirnaty (COVID-19 Vaccine, mRNA, 2023-2024 Formula), the Pfizer-BioNTech COVID-19 Vaccine (2023-2024 Formula), Comirnaty Original/Omicron BA.1, Comirnaty Original/Omicron BA.4/BA.5 and Comirnaty XBB.1.5. “Comirnaty” includes direct sales and alliance revenues related to sales of the above-mentioned vaccines, which are recorded within Pfizer’s Primary Care customer group. It does not include revenues for certain Comirnaty-related manufacturing activities performed on behalf of BioNTech, which are included in the Pfizer CentreOne contract development and manufacturing organization. Revenues related to these manufacturing activities totaled $33 million for full-year 2023 and $188 million for full-year 2022.
(2)
Revenues is defined as revenues in accordance with U.S. generally accepted accounting principles (GAAP). Reported net income/(loss) and its components are defined as net income/(loss) attributable to Pfizer Inc. common shareholders and its components in accordance with U.S. GAAP. Reported diluted earnings per share (EPS) and reported diluted loss per share (LPS) are defined as diluted EPS or LPS attributable to Pfizer Inc. common shareholders in accordance with U.S. GAAP.
(3)
Adjusted income and Adjusted diluted EPS are defined as U.S. GAAP net income attributable to Pfizer Inc. common shareholders and Reported diluted EPS attributable to Pfizer Inc. common shareholders before the impact of amortization of intangible assets, certain acquisition-related items, discontinued operations and certain significant items. See the accompanying reconciliations of certain GAAP Reported to Non-GAAP Adjusted information for the fourth quarter and full-year 2023 and 2022. Adjusted income and its components and Adjusted diluted EPS measures are not, and should not be viewed as, substitutes for U.S. GAAP net income/(loss) and its components and diluted EPS/(LPS)(2). See the Non-GAAP Financial Measure: Adjusted Income section of Management’s Discussion and Analysis of Financial Condition and Results of Operations in Pfizer’s 2022 Annual Report on Form 10-K and the accompanying Non-GAAP Financial Measure: Adjusted Income section of the press release located at the hyperlink above for a definition of each component of Adjusted income as well as other relevant information.
(4)
Significant one-time impacts to Reported(2) and Adjusted(3) diluted EPS/LPS:
Full-year 2023 Reported(2) and Adjusted(3) diluted EPS were significantly impacted by $5.6 billion of non-cash COVID product inventory write-offs and related charges in third-quarter 2023 and by a $3.5 billion non-cash Paxlovid revenue reversal in fourth-quarter 2023, which together unfavorably impacted Reported(2) and Adjusted(3) diluted EPS by $(1.36).
Fourth-quarter 2023 Reported(2) and Adjusted(3) diluted LPS/EPS were significantly impacted by a $3.5 billion non-cash Paxlovid revenue reversal in fourth quarter 2023, which unfavorably impacted Reported(2) and Adjusted(3) diluted LPS/EPS by approximately $(0.54).
(5)
Pfizer does not provide guidance for GAAP Reported financial measures (other than revenues) or a reconciliation of forward-looking non-GAAP financial measures to the most directly comparable GAAP Reported financial measures on a forward-looking basis because it is unable to predict with reasonable certainty the ultimate outcome of unusual gains and losses, certain acquisition-related expenses, gains and losses from equity securities, actuarial gains and losses from pension and postretirement plan remeasurements, potential future asset impairments and pending litigation without unreasonable effort. These items are uncertain, depend on various factors, and could have a material impact on GAAP Reported results for the guidance period.
Financial guidance for full-year 2024 reflects the following:
Does not assume the completion of any business development transactions not completed as of December 31, 2023.
An anticipated immaterial impact in fiscal-year 2024 of recent and expected generic and biosimilar competition for certain products that have recently lost patent protection or that are anticipated to lose patent protection.
Exchange rates assumed are actual rates at mid-November 2023. Financial guidance reflects the anticipated unfavorable impact of approximately $0.3 billion on revenues and approximately $0.01 on Adjusted(3) diluted EPS as a result of changes in foreign exchange rates relative to the U.S. dollar compared to foreign exchange rates from 2023.
Guidance for Adjusted(3) diluted EPS assumes diluted weighted-average shares outstanding of approximately 5.75 billion shares, and assumes no share repurchases in 2024.
(6)
Pfizer’s fiscal year-end for international subsidiaries is November 30 while Pfizer’s fiscal year-end for U.S. subsidiaries is December 31. Therefore, Pfizer’s fourth quarter and full year for U.S. subsidiaries reflects the three and twelve months ended on December 31, 2023 and December 31, 2022, while Pfizer’s fourth quarter and full year for subsidiaries operating outside the U.S. reflects the three and twelve months ended on November 30, 2023 and November 30, 2022.
(7)
References to operational variances in this press release pertain to period-over-period changes that exclude the impact of foreign exchange rates. Although exchange rate changes are part of Pfizer’s business, they are not within Pfizer’s control, and because they can mask positive or negative trends in the business, Pfizer believes presenting operational variances excluding these foreign exchange changes provides useful information to evaluate Pfizer’s results.
(8)
Keytruda is a registered trademark of Merck Sharp & Dohme Corp., a subsidiary of Merck & Co., Inc., Rahway, NJ, USA.
(9)
Bavencio is a registered trademark of Merck KGaA.
DISCLOSURE NOTICE: Except where otherwise noted, the information contained in this earnings release and the related attachments is as of January 30, 2024. We assume no obligation to update any forward-looking statements contained in this earnings release and the related attachments as a result of new information or future events or developments.
This earnings release and the related attachments contain forward-looking statements about, among other topics, our anticipated operating and financial performance, including financial guidance and projections; reorganizations; business plans, strategy, goals and prospects; our Environmental, Social and Governance (ESG) priorities, strategy and goals; expectations for our product pipeline, in-line products and product candidates, including anticipated regulatory submissions, data read-outs, study starts, approvals, launches, clinical trial results and other developing data, revenue contribution and projections, potential pricing and reimbursement, potential market dynamics, including patient demand, market size and utilization rates and growth, performance, timing of exclusivity and potential benefits; strategic reviews; capital allocation objectives; an enterprise-wide cost realignment program, which we launched in October 2023 (including anticipated costs, savings and potential benefits); dividends and share repurchases; plans for and prospects of our acquisitions, dispositions and other business development activities, including our recent acquisition of Seagen, and our ability to successfully capitalize on growth opportunities and prospects; manufacturing and product supply; our ongoing efforts to respond to COVID-19, including our plans and expectations regarding Comirnaty (as defined in this earnings release) and our oral COVID-19 treatment (Paxlovid); and our expectations regarding the impact of COVID-19 on our business, operations and financial results. Given their forward-looking nature, these statements involve substantial risks, uncertainties and potentially inaccurate assumptions and we cannot assure that any outcome expressed in these forward-looking statements will be realized in whole or in part. You can identify these statements by the fact that they use future dates or use words such as “will,” “may,” “could,” “likely,” “ongoing,” “anticipate,” “estimate,” “expect,” “project,” “intend,” “plan,” “believe,” “assume,” “target,” “forecast,” “guidance,” “goal,” “objective,” “aim,” “seek,” “potential,” “hope” and other words and terms of similar meaning. Pfizer’s financial guidance is based on estimates and assumptions that are subject to significant uncertainties.
Among the factors that could cause actual results to differ materially from past results and future plans and projected future results are the following:
Risks Related to Our Business, Industry and Operations, and Business Development:
the outcome of research and development (R&D) activities, including, the ability to meet anticipated pre-clinical or clinical endpoints, commencement and/or completion dates for our pre-clinical or clinical trials, regulatory submission dates, and/or regulatory approval and/or launch dates; the possibility of unfavorable pre-clinical and clinical trial results, including the possibility of unfavorable new pre-clinical or clinical data and further analyses of existing pre-clinical or clinical data; risks associated with preliminary, early stage or interim data; the risk that pre-clinical and clinical trial data are subject to differing interpretations and assessments, including during the peer review/publication process, in the scientific community generally, and by regulatory authorities; and whether and when additional data from our pipeline programs will be published in scientific journal publications and, if so, when and with what modifications and interpretations;
our ability to successfully address comments received from regulatory authorities such as the FDA or the EMA, or obtain approval for new products and indications from regulators on a timely basis or at all; regulatory decisions impacting labeling, including the scope of indicated patient populations, product dosage, manufacturing processes, safety and/or other matters, including decisions relating to emerging developments regarding potential product impurities; uncertainties regarding the ability to obtain, and the scope of, recommendations by technical or advisory committees; and the timing of, and ability to obtain, pricing approvals and product launches, all of which could impact the availability or commercial potential of our products and product candidates;
claims and concerns that may arise regarding the safety or efficacy of in-line products and product candidates, including claims and concerns that may arise from the outcome of post-approval clinical trials, which could impact marketing approval, product labeling, and/or availability or commercial potential;
the success and impact of external business development activities, such as the recent acquisition of Seagen, including the ability to identify and execute on potential business development opportunities; the ability to satisfy the conditions to closing of announced transactions in the anticipated time frame or at all; the ability to realize the anticipated benefits of any such transactions in the anticipated time frame or at all; the potential need for and impact of additional equity or debt financing to pursue these opportunities, which could result in increased leverage and/or a further downgrade of our credit ratings; challenges integrating the businesses and operations; disruption to business and operations relationships; risks related to growing revenues for certain acquired or partnered products; significant transaction costs; and unknown liabilities;
competition, including from new product entrants, in-line branded products, generic products, private label products, biosimilars and product candidates that treat or prevent diseases and conditions similar to those treated or intended to be prevented by our in-line products and product candidates;
the ability to successfully market both new and existing products, including biosimilars;
difficulties or delays in manufacturing, sales or marketing; supply disruptions, shortages or stock-outs at our facilities or third-party facilities that we rely on; and legal or regulatory actions;
the impact of public health outbreaks, epidemics or pandemics (such as COVID-19) on our business, operations and financial condition and results, including impacts on our employees, manufacturing, supply chain, sales and marketing, R&D and clinical trials;
risks and uncertainties related to our efforts to develop and commercialize our COVID-19 products, as well as challenges related to their manufacturing, supply and distribution, including, among others, the risk that as the market for COVID-19 products becomes more endemic and seasonal, demand for any of our COVID-19 products has and may continue to be reduced or not meet expectations, or may no longer exist, which has and may continue to lead to reduced revenues, excess inventory on-hand and/or in the channel which, for Paxlovid and Comirnaty, has resulted in significant inventory write-offs in 2023 and could continue to result in inventory write-offs or other unanticipated charges; challenges related to the transition to the commercial market for our COVID-19 products; uncertainties related to the public’s demand for vaccines, boosters and COVID-19 treatments; risks related to our ability to accurately forecast and achieve our revenue forecasts for Comirnaty and Paxlovid or any potential future COVID-19 vaccines or treatments; uncertainties inherent in R&D, including the ability to meet anticipated clinical endpoints, commencement and/or completion dates for clinical trials, regulatory submission dates, regulatory approval dates and/or launch dates, as well as risks associated with pre-clinical and clinical data (including Phase 1/2/3 or Phase 4 data for Comirnaty or any vaccine candidate in the BNT162 program or Paxlovid or any future COVID-19 treatment) in any of our studies in pediatrics, adolescents or adults or real world evidence, including the possibility of unfavorable new pre-clinical, clinical or safety data and further analyses of existing pre-clinical, clinical or safety data or further information regarding the quality of pre-clinical, clinical or safety data, including by audit or inspection; the ability to produce comparable clinical or other results for Comirnaty, any vaccine candidate or other vaccines that may result from the BNT162 program, Paxlovid or any future COVID-19 treatment or any other COVID-19 program, including the rate of effectiveness and/or efficacy, safety and tolerability pro to date, in additional analyses of the Phase 3 trial for any such products and additional studies, in real-world data studies or in larger, more diverse populations following commercialization; the ability of Comirnaty or any future vaccine to prevent, or Paxlovid or any future COVID-19 treatment to be effective against, COVID-19 caused by emerging virus variants; the risk that use of Comirnaty or Paxlovid will lead to new information about efficacy, safety or other developments, including the risk of additional adverse reactions, some of which may be serious; the risk that pre-clinical and clinical trial data are subject to differing interpretations and assessments, including during the peer review/publication process, in the scientific community generally, and by regulatory authorities; whether and when additional data from the BNT162 program, Paxlovid or other COVID-19 programs will be published in scientific journal publications and, if so, when and with what modifications and interpretations; whether regulatory authorities will be satisfied with the design of and results from existing or future pre-clinical and clinical studies; whether and when submissions to request emergency use or conditional marketing authorizations for Comirnaty or any future vaccines in additional populations, for a potential booster dose for Comirnaty, or any potential future vaccine or vaccine candidates (including potential future annual boosters or re-vaccinations), and/or biologics license and/or EUA applications or amendments to any such applications may be filed in particular jurisdictions for Comirnaty or any other potential vaccine or vaccine candidates, and if obtained, whether or when such EUA or licenses, or existing EUAs, will expire or terminate; whether and when submissions to request emergency use or conditional marketing authorizations for Paxlovid or any future COVID-19 treatment and/or any drug applications and/or EUA applications or amendments to any such applications for any indication for Paxlovid or any future COVID-19 treatment may be filed in particular jurisdictions, and if obtained, whether or when such EUA or licenses, or existing EUAs, will expire or terminate; whether and when any application that may be pending or filed for Comirnaty, any vaccine candidate or other vaccines that may result from the BNT162 program, Paxlovid or any future COVID-19 treatment or any other COVID-19 program may be approved by particular regulatory authorities, which will depend on myriad factors, including making a determination as to whether the vaccine’s or drug’s benefits outweigh its known risks and determination of the vaccine’s or drug’s efficacy and, if approved, whether it will be commercially successful; decisions by regulatory authorities impacting labeling or marketing, manufacturing processes, safety and/or other matters that could affect the availability or commercial potential of any vaccine or drug, including the authorization or approval of products or therapies developed by other companies; disruptions in the relationships between us and our collaboration partners, clinical trial sites or third-party suppliers, including our relationship with BioNTech; the risk that other companies may produce competitive products that may be superior in terms of efficacy, safety, affordability, convenience, or a number of other competitive factors; risks related to the availability or cost of raw materials to manufacture or test any such products; challenges related to our vaccine’s formulation and attendant storage, distribution and administration requirements, including risks related to storage and handling after delivery by us; challenges and risks related to medication errors such as prescribing or dispensing the wrong strength, improper dosing and self-administration errors; the risk that we may not be able to successfully develop other vaccine formulations, booster doses or potential future vaccines, potential combination respiratory vaccines or next generation COVID-19 treatments; the risk that we may not be able to recoup costs associated with our R&D and manufacturing efforts; risks associated with any changes in the way we approach or provide research funding for the BNT162 program, Paxlovid or any other COVID-19 program; challenges and risks associated with the pace of our development programs; the risk that we may not be able to maintain manufacturing capacity or access to logistics or supply channels commensurate with global demand for our COVID-19 products, which would negatively impact our ability to supply our COVID-19 products within the projected time periods; whether and when additional supply or purchase agreements will be reached or existing agreements will be modified; uncertainties regarding the ability to obtain recommendations from vaccine or treatment advisory or technical committees and other public health authorities and uncertainties regarding the commercial impact of any such recommendations; pricing and access challenges for such products; challenges related to public confidence in, or awareness of Comirnaty, Paxlovid or any future COVID-19 product candidates, including challenges driven by misinformation or disinformation, access, concerns about clinical data integrity, or prescriber and pharmacy education; trade restrictions; potential third-party royalties or other claims related to Comirnaty or Paxlovid; and competitive developments;
trends toward managed care and healthcare cost containment, and our ability to obtain or maintain timely or adequate pricing or favorable formulary placement for our products;
interest rate and foreign currency exchange rate fluctuations, including the impact of currency devaluations and monetary policy actions in countries experiencing high inflation or deflation rates;
any significant issues involving our largest wholesale distributors or government customers, which account for a substantial portion of our revenues;
the impact of the increased presence of counterfeit medicines, vaccines or other products in the pharmaceutical supply chain;
any significant issues related to the outsourcing of certain operational and staff functions to third parties;
any significant issues related to our JVs and other third-party business arrangements, including modifications related to supply agreements or other contracts with customers including governments or other payors;
uncertainties related to general economic, political, business, industry, regulatory and market conditions including, without limitation, uncertainties related to the impact on us, our customers, suppliers and lenders and counterparties to our foreign-exchange and interest-rate agreements of challenging global economic conditions, such as inflation or interest rate fluctuations, and recent and possible future changes in global financial markets;
the exposure of our operations globally to possible capital and exchange controls, economic conditions, expropriation, sanctions and/or other restrictive government actions, changes in intellectual property legal protections and remedies, unstable governments and legal systems and inter-governmental disputes;
the impact of disruptions related to climate change and natural disasters, including uncertainties related to the impact of the tornado at our manufacturing facility in Rocky Mount, NC in 2023;
any changes in business, political and economic conditions due to actual or threatened terrorist activity, geopolitical instability, political or civil unrest or military action, including the ongoing conflicts between Russia and Ukraine and in the Middle East and the resulting economic or other consequences;
the impact of product recalls, withdrawals and other unusual items, including uncertainties related to regulator-directed risk evaluations and assessments, including our ongoing evaluation of our product portfolio for the potential presence or formation of nitrosamines;
trade buying patterns;
the risk of an impairment charge related to our intangible assets, goodwill or equity-method investments;
the impact of, and risks and uncertainties related to, restructurings and internal reorganizations, as well as any other corporate strategic initiatives and growth strategies, and cost-reduction and productivity initiatives, each of which requires upfront costs but may fail to yield anticipated benefits and may result in unexpected costs, organizational disruption, adverse effects on employee morale, retention issues or other unintended consequences;
the ability to successfully achieve our climate goals and progress our environmental sustainability and other ESG priorities;
Risks Related to Government Regulation and Legal Proceedings:
the impact of any U.S. healthcare reform or legislation or any significant spending reduction or cost control efforts affecting Medicare, Medicaid or other publicly funded or subsidized health programs, including the Inflation Reduction Act of 2022, or changes in the tax treatment of employer-sponsored health insurance that may be implemented;
U.S. federal or state legislation or regulatory action and/or policy efforts affecting, among other things, pharmaceutical product pricing, intellectual property, reimbursement or access or restrictions on U.S. direct-to-consumer advertising; limitations on interactions with healthcare professionals and other industry stakeholders; as well as pricing pressures for our products as a result of highly competitive biopharmaceutical markets;
legislation or regulatory action in markets outside of the U.S., such as China or Europe, including, without limitation, laws related to pharmaceutical product pricing, intellectual property, medical regulation, environmental protections, reimbursement or access, including, in particular, continued government-mandated reductions in prices and access restrictions for certain biopharmaceutical products to control costs in those markets;
legal defense costs, insurance expenses, settlement costs and contingencies, including without limitation, those related to actual or alleged environmental contamination;
the risk and impact of an adverse decision or settlement and risk related to the adequacy of reserves related to legal proceedings;
the risk and impact of tax related litigation and investigations;
governmental laws and regulations affecting our operations, including, without limitation, the Inflation Reduction Act of 2022, changes in laws and regulations or their interpretation, including, among others, changes in tax laws and regulations internationally and in the U.S., the adoption of global minimum taxation requirements outside the U.S. generally effective in most jurisdictions since January 1, 2024 and potential changes to existing tax law by the current U.S. Presidential administration and Congress, including the proposed “Tax Relief for American Families and Workers Act of 2024”;
Risks Related to Intellectual Property, Technology and Security:
any significant breakdown or interruption of our information technology systems and infrastructure (including cloud services);
any business disruption, theft of confidential or proprietary information, security threats on facilities or infrastructure, extortion or integrity compromise resulting from a cyber-attack or other malfeasance by, but not limited to, nation states, employees, business partners or others;
risks and challenges related to the use of artificial intelligence-based software;
the risk that our currently pending or future patent applications may not be granted on a timely basis or at all, or any patent-term extensions that we seek may not be granted on a timely basis, if at all; and
risks to our products, patents and other intellectual property, such as: (i) claims of invalidity that could result in loss of exclusivity; (ii) claims of patent infringement, including asserted and/or unasserted intellectual property claims; (iii) claims we may assert against intellectual property rights held by third parties; (iv) challenges faced by our collaboration or licensing partners to the validity of their patent rights; or (v) any pressure, or legal or regulatory action by, various stakeholders or governments that could potentially result in us not seeking intellectual property protection or agreeing not to enforce or being restricted from enforcing intellectual property rights related to our products, including Comirnaty and Paxlovid.
Should known or unknown risks or uncertainties materialize or should underlying assumptions prove inaccurate, actual results could vary materially from past results and those anticipated, estimated or projected. Investors are cautioned not to put undue reliance on forward-looking statements. A further list and description of risks, uncertainties and other matters can be found in our Annual Report on Form 10-K for the fiscal year ended December 31, 2022 and in our subsequent reports on Form 10-Q, in each case including in the sections thereof captioned “Forward-Looking Information and Factors That May Affect Future Results” and “Item 1A. Risk Factors,” and in our subsequent reports on Form 8-K.
This earnings release may include discussion of certain clinical studies relating to various in-line products and/or product candidates. These studies typically are part of a larger body of clinical data relating to such products or product candidates, and the discussion herein should be considered in the context of the larger body of data. In addition, clinical trial data are subject to differing interpretations, and, even when we view data as sufficient to support the safety and/or effectiveness of a product candidate or a new indication for an in-line product, regulatory authorities may not share our views and may require additional data or may deny approval altogether.
The information contained on our website or any third-party website is not incorporated by reference into this earnings release. All trademarks mentioned are the property of their owners.
AcquisitionFinancial Statement
100 Deals associated with Marstacimab
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External Link
| KEGG | Wiki | ATC | Drug Bank |
|---|---|---|---|
| D11261 | - | - | - |
R&D Status
10 top R&D records. to view more data
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| Indication | Highest Phase | Country/Location | Organization | Date |
|---|---|---|---|---|
| Hemophilia A | NDA/BLA | US | 11 Dec 2023 | |
| Hemophilia A | NDA/BLA | EU | 11 Dec 2023 | |
| Hemophilia B | NDA/BLA | US | 11 Dec 2023 | |
| Hemophilia B | NDA/BLA | EU | 11 Dec 2023 | |
| Hemorrhage | Phase 1 | CN | 16 Apr 2021 |
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Clinical Result
Clinical Result
Indication
Phase
Evaluation
View All Results
| Study | Phase | Population | Analyzed Enrollment | Group | Results | Evaluation | Publication Date |
|---|
Phase 3 | 128 | Marstacimab (On Demand) (Observational Phase) | oshshqkpkx(xdmamewjas) = hhnvolijui akebixbkez (gkrldgqxeb, 31.03 - 46.54) View more | Positive | 09 Dec 2023 | ||
Marstacimab (Routine Prophylaxis) (Observational Phase) | oshshqkpkx(xdmamewjas) = xegishelnn akebixbkez (gkrldgqxeb, 5.09 - 10.61) View more | ||||||
Phase 1 | 6 | pcvzpgjeza(vsppvtolkj) = pmwztkiczk dwjkxrgtsr (nousvkwxey, erggasegyo - cndqkbcuus) View more | - | 07 Jul 2023 | |||
NCT03363321 (Pubmed) Manual  | Phase 2 | 18 | tjmdobivur(zclvyopdcp) = ntnnlzpikh xyjlnhkrue (hqedbkiiml ) | Positive | 11 Oct 2022 | ||
Phase 2 | 27 | (PF-06741086 300 mg SC QW Non-Inhibitor) | klyfborzcl(sdmqnvqolx) = wiojratltd ltqpbecebn (dybhqfqalr, tyyldytptk - ouorkknfzd) View more | - | 04 Dec 2019 | ||
(PF-06741086 300 mg SC Loading + 150 mg SC QW Non-Inhibitor) | klyfborzcl(sdmqnvqolx) = lxmnurdlux ltqpbecebn (dybhqfqalr, ccqfxazayd - vksmhywwui) View more |
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