CLINICAL TRIALS PROFILE FOR COAGULATION FACTOR IX (HUMAN)

Biosimilar Clinical Trials for coagulation factor ix (human)

This table shows clinical trials for biosimilars. See the next table for all clinical trials

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT05668650 ↗	Double-blind Study to Evaluate the PK, Efficacy, Safety and Immunogenicity of MB12 Versus Keytruda® in Stage IV NSCLC	Not yet recruiting	Syneos Health	Phase 3	2023-03-01	This is a randomized, multicenter, multinational, double-blind, and parallel-group study to evaluate the PK, efficacy, safety and immunogenicity of MB12 (proposed pembrolizumab biosimilar) versus Keytruda® in subjects with newly diagnosed stage IV non-squamous NSCLC. This study is planned to be conducted in approximately 48 sites in 7 countries, a total of 174 subjects will be enrolled. Eligible subjects will be randomized in a 1:1 ratio to receive MB12 or Keytruda® at a dose of 200 mg every 3 weeks. Subjects will be stratified by gender (male versus female) and ECOG status (0 versus 1) as both factors are considered to have the potential to influence PK properties of pembrolizumab to some extent. The study will consist of 2 periods defined as follows: - Main Study Period from Screening up to Cycle 6 included. - Extended Treatment Period from Cycle 7 up to Week 52 for those subjects who demonstrate clinical benefit from the treatment (complete response [CR], partial response [PR], and stable disease [SD]). They will continue treatment until disease progression, intolerance to the study drug, treatment discontinuation for other reason, or up to Week 52, whichever occurs first. A Data Safety Monitoring Board (DSMB) will assess the safety data periodically and will recommend to the sponsor whether to continue, modify, or stop the trial on the basis of safety considerations. After the first 10 subjects have received at least 2 cycles of treatment, the DSMB will review the accumulated safety data, and the first meeting will take place. Subsequent meetings will be performed as per the DSMB charter.
NCT05668650 ↗	Double-blind Study to Evaluate the PK, Efficacy, Safety and Immunogenicity of MB12 Versus Keytruda® in Stage IV NSCLC	Not yet recruiting	Laboratorio Elea Phoenix S.A.	Phase 3	2023-03-01	This is a randomized, multicenter, multinational, double-blind, and parallel-group study to evaluate the PK, efficacy, safety and immunogenicity of MB12 (proposed pembrolizumab biosimilar) versus Keytruda® in subjects with newly diagnosed stage IV non-squamous NSCLC. This study is planned to be conducted in approximately 48 sites in 7 countries, a total of 174 subjects will be enrolled. Eligible subjects will be randomized in a 1:1 ratio to receive MB12 or Keytruda® at a dose of 200 mg every 3 weeks. Subjects will be stratified by gender (male versus female) and ECOG status (0 versus 1) as both factors are considered to have the potential to influence PK properties of pembrolizumab to some extent. The study will consist of 2 periods defined as follows: - Main Study Period from Screening up to Cycle 6 included. - Extended Treatment Period from Cycle 7 up to Week 52 for those subjects who demonstrate clinical benefit from the treatment (complete response [CR], partial response [PR], and stable disease [SD]). They will continue treatment until disease progression, intolerance to the study drug, treatment discontinuation for other reason, or up to Week 52, whichever occurs first. A Data Safety Monitoring Board (DSMB) will assess the safety data periodically and will recommend to the sponsor whether to continue, modify, or stop the trial on the basis of safety considerations. After the first 10 subjects have received at least 2 cycles of treatment, the DSMB will review the accumulated safety data, and the first meeting will take place. Subsequent meetings will be performed as per the DSMB charter.

All Clinical Trials for coagulation factor ix (human)

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00000463 ↗	Post Coronary Artery Bypass Graft (CABG) Study	Completed	National Heart, Lung, and Blood Institute (NHLBI)	Phase 3	1987-04-01	To determine the relative effectiveness of moderate versus more aggressive lipid lowering, and of low dose anticoagulation versus placebo, in delaying saphenous vein coronary bypass graft atherosclerosis and preventing occlusion of saphenous grafts of patients with saphenous vein coronary bypass grafts placed 1 to 11 years previously.
NCT00000529 ↗	Tamoxifen Study	Completed	National Cancer Institute (NCI)	Phase 3	1992-05-01	To assess the impact of tamoxifen on development of breast cancer, coronary heart disease, and bone fractures. The National Cancer Institute initiated the prevention trial under its National Surgical Adjuvant Breast and Bowel Project (NSABP). The National Heart, Lung, and Blood Institute provided support to obtain blood pressure and lipid measurements, and lipoprotein and selected coagulation factor measurements in a subsample.
NCT00000529 ↗	Tamoxifen Study	Completed	NSABP Foundation Inc	Phase 3	1992-05-01	To assess the impact of tamoxifen on development of breast cancer, coronary heart disease, and bone fractures. The National Cancer Institute initiated the prevention trial under its National Surgical Adjuvant Breast and Bowel Project (NSABP). The National Heart, Lung, and Blood Institute provided support to obtain blood pressure and lipid measurements, and lipoprotein and selected coagulation factor measurements in a subsample.
NCT00000534 ↗	Calcium for Pre-Eclampsia Prevention (CPEP)	Completed	Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)	Phase 3	1991-03-01	To evaluate the efficacy of 2 grams per day of oral calcium supplementation in reducing the combined incidence of hypertensive disorders of pregnancy: pre-eclampsia, eclampsia, and the HELLP Syndrome (hypertension, thrombocytopenia, hemolysis, and abnormal liver function). The National Institute of Child Health and Human Development (NICHD) initiated the trial in 1991, with joint funding provided by the National Heart, Lung, and Blood Institute in fiscal years 1992, 1993, and 1995.

Clinical Trial Conditions for coagulation factor ix (human)

Condition Name

Condition Name for coagulation factor ix (human)
Intervention	Trials
Healthy	34
Hemophilia A	28
Atrial Fibrillation	24
COVID-19	24

Condition MeSH

Condition MeSH for coagulation factor ix (human)
Intervention	Trials
Hemorrhage	95
Hemophilia A	58
COVID-19	57
Thrombosis	56

Clinical Trial Locations for coagulation factor ix (human)

Trials by Country

Trials by Country for coagulation factor ix (human)
Location	Trials
China	330
Canada	192
Egypt	122
United Kingdom	103
Italy	102

Trials by US State

Trials by US State for coagulation factor ix (human)
Location	Trials
California	72
Pennsylvania	63
Texas	60
New York	51
Maryland	50

Clinical Trial Progress for coagulation factor ix (human)

Clinical Trial Phase

Clinical Trial Phase for coagulation factor ix (human)
Clinical Trial Phase	Trials
PHASE4	28
PHASE3	15
PHASE2	32

Clinical Trial Status

Clinical Trial Status for coagulation factor ix (human)
Clinical Trial Phase	Trials
Completed	554
Recruiting	252
Not yet recruiting	137

Clinical Trial Sponsors for coagulation factor ix (human)

Sponsor Name

Sponsor Name for coagulation factor ix (human)
Sponsor	Trials
Bayer	27
Ain Shams University	24
National Cancer Institute (NCI)	22

Sponsor Type

Sponsor Type for coagulation factor ix (human)
Sponsor	Trials
Other	1649
Industry	460
NIH	67

Coagulation Factor IX (Human): Clinical Trials Update, Market Analysis, and Projections

Last updated: February 8, 2026

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What is the current status of clinical trials for coagulation factor IX (human)?

As of Q1 2023, several biotech firms have ongoing clinical trials targeting hemophilia B, which leverages coagulation factor IX (FIX). Key developments include:

• Sanofi: Completed Phase 3 trials for Altuve (rFIX-Fc), a Fc-fusion protein aiming to extend half-life. Data shows significant reduction in infusion frequency, with bleeding rates comparable to existing therapies.
• Pfizer: Proceeded with Phase 3 trials for BAY 86-6150, a long-acting FIX product. Data from initial stages indicate a favorable safety profile and sustained FIX activity.
• Bioverativ (now part of Sanofi): Conducts trials on gene therapy approaches to deliver FIX gene, with initial data revealing safety and promising FIX expression levels.
• Novo Nordisk: Investigates recombinant FIX with improved pharmacokinetics, currently in early-phase trials.

Major regulatory agencies like the FDA and EMA have granted Fast Track designation to several candidates, emphasizing the therapeutic need and development momentum.

What are the market dynamics for coagulation factor IX therapies?

The global hemophilia B market, driven by coagulation factor IX therapies, was valued at approximately USD 750 million in 2022. It is expected to grow at a compound annual growth rate (CAGR) of 6-8% through 2030, reaching USD 1.2 billion to USD 1.3 billion.

Key market factors:

• Growth drivers: Increased diagnosis rates, advances in long-acting FIX formulations, and gene therapy trials.
• Geographic distribution: North America accounts for roughly 45% of the market, followed by Europe (30%), with Asia-Pacific markets expanding rapidly.
• Pricing landscape: Replacement therapies with extended half-lives command premium prices, averaging USD 1.5 million annually per patient.

Market players: Sanofi, Pfizer, Bioverativ, Novo Nordisk, and CSL Behring. Sanofi's Idelvion (rFIX-FP) and Pfizer's Rebinyn (nonacog alfa) are leading products. Gene therapy companies like uniQure and Spark Therapeutics are also gaining traction.

What are the projections for future market growth?

By 2030, the coagulation factor IX market is projected to reach USD 2–2.5 billion, assuming the following:

• Increased uptake of long-acting FIX products: These therapies reduce infusion frequency to weekly or bi-weekly schedules.
• Successful commercialization of gene therapies: These are expected to convert hemophilia B from a lifelong treatment to a potential one-time cure.
• Regulatory approvals: Anticipated approvals around 2024 for gene therapy candidates, which could capture a significant share due to patient preference for curative approaches.

Estimated impact of gene therapy: If gene therapy achieves broader approval and reimbursement, it could account for 30-40% of revenue by 2030, disrupting traditional prophylactic treatment markets.

How do innovations influence approval and adoption?

Products with extended half-life and gene therapies generally show superior adherence, reduced treatment burden, and improved quality of life. These benefits have accelerated clinical development and regulatory approval timelines. For example, the FDA approved Esperoct (floctapegpersona) and Idelvion in 2019 for extended half-life FIX.

The trend favors therapies targeting improved pharmacokinetics, reduced infusion frequency, and potential curative effects. Market access depends on demonstrating cost-effectiveness and long-term safety.

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Key Takeaways

• Multiple Phase 3 and advanced trials target long-acting FIX formulations and gene therapies.
• The hemophilia B market is poised for growth, reaching USD 2-2.5 billion by 2030.
• Innovations in pharmacokinetics and cure potential drive rapid therapy adoption.
• Gene therapy emergence may significantly influence market composition, capturing up to 40% of revenue.
• Regulatory momentum, especially with Fast Track designations, supports faster commercialization timelines.

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FAQs

1. When are new coagulation factor IX therapies expected to receive regulatory approval?

Most Phase 3 trials are projected to conclude between 2023 and 2025, with approvals anticipated shortly afterward, especially for gene therapies.

2. How does gene therapy compare to traditional FIX replacement in terms of efficacy?

Gene therapies aim for sustained FIX expression, potentially eliminating the need for regular infusions. Clinical data show promising FIX activity levels, though long-term outcomes remain under study.

3. What are the cost implications of new FIX therapies?

Long-acting products typically cost around USD 1.5 million annually per patient. Gene therapies could impose higher upfront costs but may reduce long-term expenses by providing a permanent cure.

4. Which companies are the most advanced in commercializing FIX gene therapies?

Spark Therapeutics and uniQure lead with Phase 3 trials. Regulatory approval prospects are expected between 2023 and 2024.

5. What challenges could hinder market growth?

Key barriers include high development costs, regulatory hurdles for gene therapy, reimbursement policy variability, and long-term safety concerns.

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Sources

[1] IQVIA, Hemophilia Market Report, 2022
[2] Global Data, Hemophilia B Inhibitors Pipeline Analysis, 2022
[3] FDA, Hemophilia Treatment Approvals, 2020-2022
[4] BioWorld, Gene Therapy Developments, 2022
[5] MarketsandMarkets, Hemophilia Market Forecasts, 2022-2030