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.
NCT00000534 ↗	Calcium for Pre-Eclampsia Prevention (CPEP)	Completed	National Heart, Lung, and Blood Institute (NHLBI)	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	33
Hemophilia A	28
COVID-19	24
Atrial Fibrillation	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	326
Canada	191
Egypt	120
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	23
PHASE3	12
PHASE2	29

Clinical Trial Status

Clinical Trial Status for coagulation factor ix (human)
Clinical Trial Phase	Trials
Completed	554
Recruiting	250
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	23
National Cancer Institute (NCI)	22

Sponsor Type

Sponsor Type for coagulation factor ix (human)
Sponsor	Trials
Other	1631
Industry	454
NIH	66

Clinical Trials Update, Market Analysis, and Projection for Coagulation Factor IX (Human)

Last updated: November 9, 2025

Introduction

Coagulation Factor IX (human), widely recognized as a pivotal component in hemophilia B treatment, has undergone significant developments over recent years. This article provides a comprehensive update on ongoing and completed clinical trials, analyzes current market dynamics, and projects future growth trajectories, aiming to inform business decision-makers and industry stakeholders.

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Clinical Trials Update

Recent and Ongoing Clinical Trials

Advancements in recombinant and plasma-derived Factor IX therapies have propelled clinical research focused on improving efficacy, safety, and administration convenience.

Key Trials and Their Outcomes:

• Gene Therapy Trials: Multiple gene therapy initiatives, led by biotech companies like BioMarin Pharmaceuticals and Sangamo Therapeutics, aim to provide durable, single-dose treatments. For instance, BioMarin's AAV-mediated gene therapy (valoctocogene roxaparvovec) has shown proficiency in increasing endogenous Factor IX levels, with Phase 3 data indicating sustained benefit over two years (BioMarin, 2022) [1].

• Extended Half-life (EHL) Products: Innovations such as Alnylam's siRNA-based therapies and CSL Behring’s eftrenonacog alfa have completed Phase 3, demonstrating prolonged half-life and reduced infusion frequency. Notably, CSL Behring's Hemgenix—an EHL Factor IX gene therapy—initiated pivotal trials in late 2022, aiming to improve patient adherence.

• Subcutaneous Delivery: Trials exploring subcutaneous administration of Factor IX—with the potential to replace intravenous infusions—are underway. For example, the phase 2 study by Green Cross Corporation (Korea) evaluates subQ formulations for ease of use.

Clinical Trial Trends and Challenges

The trend toward gene therapy and EHL factors reflects a strategic focus on reducing treatment burden and enhancing patient quality of life (QoL). Nonetheless, challenges persist:

• Immunogenicity: A significant concern across gene therapies, with neutralizing antibodies complicating treatment efficacy.

• Long-term Safety: Limited data on durability and adverse events associated with novel therapies necessitate vigilant post-marketing surveillance.

• Regulatory Pathways: Divergent approval processes across jurisdictions impact development timelines.

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Market Analysis

Current Market Landscape

The global coagulation factor IX market was valued at approximately $1.5 billion in 2022, with a compound annual growth rate (CAGR) of roughly 7.4% projected through 2030 [2].

Major Market Players:

• Pfizer (Benefix): Leading plasma-derived product, although sales declining due to recombinant alternatives.
• Pfizer (BeneFIX): Among the early recombinant therapies, experiencing steady demand.
• CSL Behring: Innovator in EHL products with substantial market share.
• BioMarin Pharmaceuticals: Pioneering gene therapy solutions, targeting a premium segment.

Segment Breakdown:

• Recombinant Factor IX Products: Constitute over 65% of the market, driven by safety profile and improved efficacy.
• Plasma-Derived Products: Gradually declining amid safety concerns and technological upgrades.

Market Drivers

• Rising Hemophilia B Prevalence: Estimated at approximately 1 in 25,000 male births, projected to create sustained demand.
• Advancements in Therapy: The shift toward gene therapy and EHL products encourages market expansion.
• Improved Diagnostics: Enhanced screening accelerates diagnosis, boosting treatment initiation rates.

Market Challenges

• High Costs: Treatment prices range from $250,000 to $500,000 annually per patient, imposing access barriers, especially in low-to-middle-income economies.

• Regulatory and Reimbursement Hurdles: Variability across regions hampers widespread adoption of novel therapies.

• Competition from Emerging Biosimilar and Gene Therapies: Patent cliffs and innovation pipelines intensify the competitive landscape.

Future Market Projections

The market is expected to expand at a CAGR of 8% to 10% through 2030. Notably:

• Gene Therapy: Predicted to account for approximately 30% of total sales by 2028, driven by breakthroughs like BioMarin’s valoctocogene roxaparvovec.

• Diversification of Administration: SubQ formulations and oral delivery systems aim to improve adherence, broadening market reach.

• Geographical Expansion: Emerging markets, including Asia-Pacific and Latin America, are poised for rapid growth, contingent upon regulatory approvals and affordability interventions.

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Regulatory and Industry Dynamics

• FDA and EMA Approvals: Recent approvals of gene therapies are setting new standards, influencing regulatory frameworks worldwide.

• Reimbursement Policies: Payers are increasingly scrutinizing cost-effectiveness, prompting negotiations for value-based pricing models.

• Partnerships & Mergers: Consolidations, such as the acquisition of BioMarin by Roche and alliances between biotechs and pharma giants, aim to accelerate innovation and market penetration.

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Projected Clinical and Market Evolution (2023–2030)

• Clinical Progress: Anticipated approvals of next-generation EHL products and continued refinement of gene therapies will improve safety and durability profiles.

• Market Dynamics: Enhanced immunogenicity management and cost reductions will facilitate broader access.

• Emerging Trends: Personalization of therapies, including tailored dosing and combination regimens, will reshape treatment paradigms.

• Economic Impact: Despite high upfront costs, long-term savings through sustained cure potential and reduced bleeding episodes will support investment.

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Key Takeaways

• The coagulation factor IX (human) market is undergoing a paradigm shift from traditional plasma-derived and recombinant therapies toward gene therapy and EHL products, promising significant improvements in patient outcomes and adherence.

• Current clinical trials underscore promising efficacy signals but highlight ongoing challenges around immunogenicity and long-term safety.

• Market growth is forecasted at a CAGR of 8–10% until 2030, propelled by technological innovation, expanding indications, and emerging economies.

• Industry stakeholders should prioritize adaptive regulatory strategies, cost reduction initiatives, and robust post-approval surveillance to capitalize on market opportunities.

• The evolution towards personalized, convenient, and affordable therapies will define competitive advantages in this dynamic landscape.

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FAQs

1. What is the current status of gene therapy for coagulation Factor IX (human)?
   Several gene therapy candidates, including BioMarin’s valoctocogene roxaparvovec and CSL Behring’s Hemgenix, have completed or are nearing approval stages, demonstrating promising durable efficacy in clinical trials.

2. How do extended half-life (EHL) Factor IX products differ from traditional therapies?
   EHL products utilize modifications—such as Fc fusion or PEGylation—to prolong circulation time, reducing infusion frequency from 2–3 times weekly to once every 7–14 days, improving patient adherence and QoL.

3. What are the main challenges facing market growth for coagulation Factor IX therapies?
   High costs, immunogenicity concerns, regulatory variability, and limited access in emerging markets hinder widespread adoption.

4. How might upcoming clinical trial results influence market projections?
   Positive long-term safety and efficacy data could accelerate regulatory approvals and market penetration for gene therapy and EHL products, boosting growth projections.

5. What strategic moves should industry players focus on to remain competitive?
   Investing in innovative delivery modalities, forging strategic partnerships, optimizing manufacturing costs, and engaging with regulatory bodies are critical for sustained success.

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References

[1] BioMarin Pharmaceuticals. (2022). Phase 3 Data on Valoctocogene Roxaparvovec.
[2] Market Research Future. (2023). Global Coagulation Factor IX Market Forecast.