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Marstacimab reduced the annualized bleeding rate by 35% and 92% compared to the prophylaxis regimen and on-demand treatment in patients with hemophilia A and B without inhibitors, respectively.
Consistent relief in bleeding rates was seen after an additional 16 months of follow-up in the long-term observational extension study of the trial.
NEW YORK, December 10, 2023–(BUSINESS WIRE)–Pfizer Inc. (NYSE: PFE) today presented results from the pivotal Phase 3 BASIS clinical trial (NCT03938792) evaluating marstacimab for the treatment of people with severe hemophilia A and moderately severe to severe hemophilia B without inhibitors to Factor VIII (FVIII) or Factor IX (FIX). The results from the BASIS trial demonstrated a statistically significant and clinically meaningful effect on annualized bleeding rate (ABR). The findings were presented today at the 65th American Society of Hematology (ASH) Annual Meeting and Exposition in San Diego.
“For more than five decades, the most common remedy for hemophilia A and B has been intravenous infusions, administered several times a week,” said James Rusnak, M. D. , Ph. D. , senior vice president and chief advancement officer for Medicine Internal. and Infectious Diseases, Research and Development, Pfizer. “Based on those effects and if approved, we believe marstacimab may be providing a subcutaneous option with a compelling combination of efficacy and protection that may particularly decrease the threat of bleeding. We take a look. We are ahead to potentially offer this remedy option to other people living with hemophilia A and B without inhibitors.
In the BASIS trial, 116 people with haemophilia were treated with marstacimab for a 12-month active treatment (ATP) period compared to an on-demand prophylaxis (PR) regimen (RP) and intravenous (OD) regimen with FVIII or FIX, given as a component of the same usual care over a six-month period. Marstacimab, a new investigational tissue pathway inhibitor (anti-TFPI), was administered weekly at a constant (non-weight-based) dose as a subcutaneous loading dose of 300 mg. followed by 150 mg per week. The study found:
Compared with PR, marstacimab remedy produced a mean 35. 2% (95% CI: 5. 6-55. 6; p=0. 0376) relief in AEPs over 12 months (mean 7. 85 [5. 09-10. 61] to 5. 08 [3. 40-6. 77]).
Marstacimab particularly reduced PEA by 91. 6% (95% CI 88. 1-94. 1, p<0. 0001) to OD over 12 months (mean 38. 00 (31. 03-46. 54) to 3. 18 (2. 09-4. 85)).
The mean discounts in AEPs observed with marstacimab were consistent across all haemophilia A and B groups and across all age teams for OD and were consistent across haemophilia A and B and age teams for PR, with all point estimates of a difference <2. 5 (non-inferiority margin for treated bleeding PEA).
After 12 months of initiation on ATP, patients had the option to continue receiving marstacimab from the long-term extension (LTE) study. In LTE, consistent relief in ABR relative to OD (mean ABR 3. 86 [2. 02-7. 37]) was observed, and additional numerical relief was observed relative to PR (mean ABR 2. 27 [1. 40-3. 67]) after an additional 16 months of follow-up (n = 87).
In the OD group, the superiority (p < 0. 0001) of marstacimab was demonstrated across all secondary endpoints similar to bleeding: spontaneous bleeding, joint bleeding, target joint bleeding, and overall bleeding. In the PR group, marstacimab demonstrated non-inferiority in the secondary criteria. Efficacy Evaluation Criteria.
Health-related quality of life parameters showed non-significant improvements vs OD therapy and non-inferiority versus RP therapy.
The protection profile of marstacimab was consistent with Phase 1/2 effects and the drug was sometimes well tolerated. No deaths or events of thromboembolic or consummative coagulopathy were reported in patients with haemophilia in the clinical trials of marstacimab. The commonly reported maximum adverse events of specific interest in patients treated with marstacimab in the BASIS and LTE studies (≥ 5% of patients) were COVID-19, bleeding, liver disorders, hypersensitivity, hypertension, and injection site reaction. A serious adverse event (SAE) emerging from the drug (peripheral edema) and one patient discontinued the trial due to a non-treatment-related SAE.
“Given the uncertainty that living with hemophilia can pose for patients, the effects of the BASIS trial are especially encouraging, as discounts in PSAs were observed during the 12-month treatment period and then maintained during long-term follow-up. ” said Davide Matino. . , MD, M. Sc, Assistant Professor of Medicine, McMaster University. “Based on those effects, marstacimab has demonstrated its potential to meet the varied desires of patients with hemophilia A or B without inhibitors, with weekly subcutaneous management at a constant dose that is not based on weight and with low follow-up requirements.
Pfizer currently has three Phase 3 programs investigating novel treatment options for people living with hemophilia. In addition to the BASIS study, fidanacogene elaparvovec and giroctocogene fitelparvovec are investigational gene therapy treatments being studied for the treatment of adults living with hemophilia B and hemophilia A, respectively. Updated data from both gene therapy programs, including an oral presentation of four-year results from Pfizer’s Phase 1/2 study of giroctocogene fitelparvovec in adults living with severe hemophilia A, will be presented at the ASH meeting.
About the BASIS Study
BASIS is a global, open-label, multicenter Phase 3 study comparing PEA over 12 months of treatment with marstacimab, a new investigational subcutaneous treatment option, in approximately 145 adolescent and adult participants aged 12 to <75 years with severe hemophilia A (defined as FVIII <1%) or moderately severe to severe hemophilia B (defined as FIX activity ≤2%) with or without inhibitors. Approximately 15% of participants are adolescents (seniors aged 12 to <18 years). This study compares the treatment with a race-in-era for patients who won a replacement cure or a step cure over a six-month period with a 12-month ATP, during which participants receive prophylaxis (a subcutaneous loading dose of three hundred mg of marstacimab, followed by 150 mg subcutaneously once weekly) with the option to extend the dose to three hundred mg once per week. week.
Enrollment of the inhibitor cohort of the BASIS trial has been completed and is expected to be completed by the end of 2024. Pfizer is also conducting BASIS KIDS, an open-label study investigating the protection and efficacy of marstacimab in children 1 to 2 years of age. <18 years old. With severe hemophilia A or moderately severe to severe hemophilia B with or without inhibitors. The exam will compare 12 months of old folk remedy with marstacimab prophylaxis.
About marstacimab
Marstacimab is an isotype of human monoclonal immunoglobulin G, subclass 1 (IgG1) that targets the Kunitz 2 domain of tissue pathway inhibitor (TFPI), a natural anticoagulant protein that aims to prevent blood clots from forming. Marstacimab is being developed as a prophylactic remedy to save or decrease the frequency of bleeding episodes in other people with severe hemophilia A or moderate to severe hemophilia B with or without inhibitors.
About Hemophilia
Hemophilia is a family of rare genetic blood diseases caused by a deficiency of clotting sites (FVIII in hemophilia A, FIX in hemophilia B), which prevents overall blood clotting. Haemophilia is diagnosed in the early formative years and affects more than 400,000 people. around the world. 2 The inability of the blood to clot well can increase the risk of painful bleeding inside the joints, which can cause scarring and joint pain. People with hemophilia may experience permanent joint pain as a result of repeated bleeding episodes. 1,2
For decades, the most common treatment method for hemophilia A and B has been element replacement therapy, which replaces missing clotting elements. Factor replacement treatments increase the amount of clotting element in the body to levels that improve clotting, resulting in decreased bleeding. 3,4 Approximately 25% to 30% of people with hemophilia A and 3% to 5% of people with hemophilia B cannot continue to take replacement therapy because of increased FVIII and FIX inhibitors. 5, 6
In a U. S. survey, In the U. S. Department of Health among people receiving hemophilia A or B prophylaxis, about one-third of those receiving treatment and maximum adherence (defined as taking 75% or more of prescribed infusions) said the slow nature of treatment prophylaxis was the most significant challenge to the regimen. 7, 8 At most, 60% of those who took fewer infusions than prescribed reported that time was the main reason for not receiving Infusions.
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DISCLOSURE NOTICE: The data in this press release is as of December 9, 2023. Pfizer assumes no legal responsibility to update the forward-looking statements contained in this release, whether as a result of new data or long-term events or developments.
This release comprises prospective data on marstacimab, an investigational tissue pathway inhibitor, and Pfizer’s hemophilia systems for fidanacogene elaparvovec and gyroctocogene fitelparvovec, adding their potential benefits, which involve threats and uncertainties that may also cause actual effects to differ. substantially of those expressed or implied through such statements. Risks and uncertainties include, but are not limited to, uncertainties inherent to the studies and development, as well as the ability to achieve expected clinical endpoints, the start and/or completion dates of our clinical trials, regulatory submission dates, regulatory approval dates and/or launch dates. , as well as the option of new adverse clinical data and additional analysis of existing clinical knowledge; if and when the inhibitor cohort of the BASIS trial will be successful; the risk that clinical trial data will be subject to divergent interpretations and testing by the regulatory government; whether the regulatory government will be satisfied with the design and effects of our clinical studies; whether and when programs may be submitted to the regulatory government in specific jurisdictions, for marstacimab, fidanacogene elaparvovec or gyroctocogene fitelparvovec; whether programs possibly pending or submitted for marstacimab, fidanacogene elaparvovec, or gyroctocogene fitelparvovec can be approved through the regulatory government, which will factor into a host of things, adding the determination of whether the product’s benefits outweigh its known benefits, and when. threats and determining the effectiveness of the product and, if approved, whether marstacimab, fidanacogene elaparvovec and gyroctocogene fitelparvovec will achieve advertising success; decisions through regulatory governance have an effect on labeling, production processes, protection and/or other issues that would potentially affect the availability or prospective advertising of marstacimab, fidanacogene elaparvovec and gyroctocogene fitelparvovec; uncertainties related to the effect of COVID-19 on our business, operations and financial effects; and competitive developments.
A more detailed description of the hazards and uncertainties can be found in Pfizer’s Annual Report on Form 10-K for the year ended December 31, 2022 and its upcoming reports on Form 10-Q, by adding in the sections entitled “Risk Factors” and “Forward-Looking Information and Factors That May Affect Future Results” and its upcoming reports on Form 8-K, all of which are filed with the U. S. Securities and Exchange Commission. They are available at www. sec. gov and www. pfizer. com. COM.
1 Franchini M, Mannucci PM. Past, Present, and Future of Hemophilia: A Narrative Review. Orphanet J Rare Dis 7, 24 (2012). https://doi. org/10. 1186/1750-1172-7-24. 2 Srivastava A, Santagostino E, Dougall A et al. WFH Guidelines for the Treatment of Hemophilia, Third Edition; 2020. Hemophilia, 26(S6), 1-158. https://doi. org/10. 1111/hae. 14046. 3 Centers for Disease Control and Prevention. Hemophilia. Last revised: April 2023. https://www. cdc. gov/ncbddd/hemophilia/. 4 Weyand AC, Pipe SW. New remedies for hemophilia. Blood 2019; 133(5):389-398. doi: https://doi. org/10. 1182/blood-2018-08-872291. 5 Centers for Disease Control and Prevention. Inhibitors and hemophilia. Last revised: April 2023. https://www. cdc. gov/ncbddd/hemophilia/inhibitors. html. 6 Peyvandi F, Garagiola I, Seregni S. The Future of Clotting Factor Replacement Therapy. J Throm Haemost. 2013; 11 (Suppl. 1): 84–98. 7 Thornburg CD, Duncan NA. Adherence to the remedy in hemophilia. Patient prefers adherence. 2017;11:1677-1686. https://doi. org/10. 2147/PPA. S139851. 8 Hacker MR, Geraghty S, Manco-Johnson M. Barriers to adherence to prophylactic cure in hemophilia. Hemophilia 2001; 7(4):392-6.
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