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.
NCT00000690 ↗	Single Dose Pharmacokinetics of Oral Dextran Sulfate (UA001) and Intravenous Dextran Sulfate in Healthy Volunteers	Completed	National Institute of Allergy and Infectious Diseases (NIAID)	Phase 1	1969-12-31	To evaluate how the drug dextran sulfate (DS) is absorbed by the stomach and intestines when taken by mouth. To evaluate its effect on blood coagulation. DS has been reported to have anti-HIV activity. However, it is not known how much of the drug is absorbed into the bloodstream and can be used by the body when DS is taken by mouth.

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

Last updated: May 16, 2026

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

Coagulation Factor IX (Human) market demand is driven by prophylaxis in hemophilia B, with growth tied to subcutaneous delivery, long-acting products, and expanded patient treatment. Competitive intensity is rising as additional recombinant and gene-therapy alternatives progress through late-stage development. For revenue projection, the near-term base case depends on (1) uptake of extended half-life (EHL) IX therapies, (2) conversion from older recombinant IX and plasma-derived IX, and (3) reimbursement and hospital formularies that favor reduced infusion burden.

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What clinical trials are underway for coagulation factor IX (human) in hemophilia B?

Clinical development activity concentrates on:

1. Long-acting recombinant factor IX (EHL) and improved formulations
2. Higher-potency or easier administration platforms that reduce infusion frequency
3. Non-factor approaches adjacent to factor IX use (notably gene therapy and bispecifics) that may change the competitive landscape after adoption

Late-stage and registration-relevant trial themes

Featured search intent for “clinical trials update” in hemophilia B typically breaks down into the following endpoints and trial structures.

1. EHL recombinant factor IX trials

Common design elements:

• prophylaxis cohorts (continuous or intermittent)
• annualized bleeding rate (ABR) endpoints
• pharmacokinetics (PK), half-life, and trough factor activity
• inhibitor screening and inhibitor-free survival

2. Subcutaneous or infusion-sparing factor IX programs

Subcutaneous delivery is a recurring objective where manufacturers aim to:

• extend time-to-dose
• reduce infusion time and healthcare-resource use
• enable home treatment and improved adherence

3. Switching and crossover studies

Trials increasingly track real-world-like transitions from:

• standard half-life recombinant IX to EHL IX
• plasma-derived IX to recombinant IX
• one EHL product to another (to manage tolerability, PK, or access)

What’s the current clinical “shape” of the factor IX pipeline?

A complete, product-level “clinical trials update” requires current trial registries and submission timelines for each sponsor and protocol. Without that dataset in scope, the only accurate statement is that the clinical pipeline is dominated by EHL and improved delivery approaches in hemophilia B, with gene therapies and non-factor modalities increasingly shaping future demand.

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How big is the market for coagulation factor IX (human) in hemophilia B?

Market definition matters. “Coagulation factor IX (human)” is a category that includes:

• recombinant factor IX products (including EHL products)
• plasma-derived factor IX products in some regions
• branded and biosimilar-adjacent offerings where regulatory frameworks permit

Primary demand drivers

• Prophylaxis uptake: more patients treated with prophylaxis rather than episodic on-demand therapy.
• Inhibitor management: demand remains for patients who require sustained factor exposure with inhibitor monitoring.
• Administration convenience: EHL and reduced-burden regimens influence hospital and payer coverage.
• Pediatric expansion: growing treated population in pediatric hemophilia B cohorts increases long-term volume.

Key segmentation used in market models

• By patient population (hemophilia B severity, prophylaxis eligible)
• By product generation (standard half-life vs EHL vs other modalities)
• By route of administration (IV vs SC)
• By region (US, EU5, UK, Canada, Japan, rest of world)

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When does coagulation factor IX (human) lose exclusivity and what does that do to pricing?

Exclusivity timing is product- and jurisdiction-specific, typically governed by:

• biologics licensing and regulatory exclusivity
• patent term and patent-specific expiration schedules
• device and delivery-method IP (for modified dosing regimens)

A defensible exclusivity and generic entry timeline requires exact product identity and the associated Orange Book or EP register and patent family data. Without product-specific mapping, only the directional impact can be stated: when EHL factor IX exclusivity or key patents expire, pricing pressure usually follows through:

• formulary switches to lower-cost alternatives
• discounting strategies by incumbents
• higher switching rates in institutional settings after a competitor secures access

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What patents protect coagulation factor IX (human) therapies and where are they concentrated?

Patent estates in factor replacement products typically cover multiple layers:

1. Molecule and sequence identity
2. Fusion proteins / linkers / Fc or PEGylation components (for EHL architectures)
3. Glycosylation and manufacturing process parameters
4. Formulations (stabilizers, excipients, reconstitution approaches)
5. Methods of use (prophylaxis regimens, dosing strategies, target trough activities)
6. Delivery devices and administration systems (where applicable)

How long do these estates last in practice?

• Early filings can extend into the late-teens to 20-year range for key composition and process claims.
• Method-of-use and formulation claims may add staggered protection windows.
• Litigation and settlement agreements can extend market exclusivity through delayed launch dates even after nominal patent expiry.

A product-level mapping (specific patent numbers, assignees, and expiration dates) requires a defined list of factor IX brands in scope.

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What is the Orange Book status of coagulation factor IX (human) and how does it affect generic entry risk?

Orange Book status is determined per NDA/BLA product and typically includes:

• listed patents covering drug substance, drug product, and method-of-use
• “Orange Book” patent codes and expiration dates

For factor IX products, entry risks depend on:

• whether the candidate is a generic vs a biosimilar vs another regulated pathway
• the strength and remaining life of the “listed” patents
• whether Paragraph IV-style disputes are available under the relevant regulatory framework

A correct Orange Book status summary must be built from product-specific Orange Book records, including listed patent numbers and expiration dates.

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Which companies dominate coagulation factor IX (human) supply and what is the competitive landscape?

The hemophilia B competitive set has two tiers:

1. Factor IX replacement manufacturers (recombinant and EHL)
2. Non-factor and gene therapy developers that can shift treatment paradigms

In market analyses, competition is measured by:

• product share among treated hemophilia B patients
• EHL penetration rate
• contracting outcomes with national and regional payers
• home-care and patient support programs that improve adherence

A quantified competitor ranking and share breakdown requires current market-share and prescription or treatment data not included in the prompt.

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How strong is the patent estate for coagulation factor IX (human) and what litigation affects launch timing?

In biologics and factor replacement categories, litigation and settlements frequently involve:

• composition-of-matter and structural claims
• process and manufacturing method claims
• formulation and device administration patents
• method-of-use dosing regimen patents

Launch timing is influenced when:

• an injunction is sought and granted or threatened
• settlements include “carve-outs” or delayed effective dates
• payers enforce pathway switching only after disputes resolve

A litigation-specific update requires the named products, case captions, and docket timelines.

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What is the biosimilar or “next-gen” entry risk for factor IX therapies?

Biosimilar or next-gen risks in factor IX are shaped by:

• regulatory requirements for comparability (structural and functional)
• clinical equivalence studies and immunogenicity monitoring
• patent landscapes that can delay or condition entry
• interchangeability and payer willingness to switch EHL regimens

In practical market models, biosimilar entry risk is often mitigated by:

• high clinical inertia and switching costs
• inhibitor concerns and patient-level tailoring of dosing
• ongoing rebates and contracting by incumbents

A credible biosimilar risk assessment needs sponsor-specific development pipelines and relevant patent expiration/entry pathways.

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How do gene therapies change the factor IX (human) market projection?

Gene therapies are the key structural variable because they can replace lifelong prophylaxis in a subset of hemophilia B patients. Market projections typically incorporate:

• eligible patient share (tailored by age, liver health, and prior exposure)
• durability of transgene expression
• retreatment risk and long-term safety considerations
• payer strategies that price outcomes over time

Where gene therapy uptake accelerates, factor IX demand can plateau or grow more slowly, even if overall hemophilia B prevalence rises.

A quantitative projection requires:

• gene therapy adoption curve assumptions
• treatment capacity and manufacturing constraints
• regional reimbursement and health-technology assessment timelines

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Revenue projection model for coagulation factor IX (human): base case, bull case, bear case

A correct revenue projection for “coagulation factor IX (human)” must aggregate all relevant products in the category and segment by region and patient treatment modality. Without a defined brand list, a category-only projection risks being structurally inaccurate.

Directional revenue drivers by scenario

• Bull case: faster conversion to EHL prophylaxis, higher home-treatment penetration, slower substitution by non-factor alternatives, durable pricing.
• Base case: steady prophylaxis growth offset partially by therapy substitution and pricing normalization.
• Bear case: accelerated uptake of gene therapy and non-factor options, payer pressure on high-cost prophylaxis, faster switching after competitive entry.

What business leaders should monitor each quarter

• ABR trends from real-world registries
• EHL trough activity adoption patterns and switching volumes
• formulary exclusions or step-therapy policy changes
• payer rebate intensity and net price trends
• gene therapy payer coverage decisions and uptake rate signals

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What filings and regulatory events matter most for coagulation factor IX (human) over the next 12–24 months?

Regulatory catalysts typically include:

• phase 3 readouts and label expansions for EHL or delivery innovations
• CMC approvals for manufacturing capacity and product stability upgrades
• pediatric study plan milestones and extensions to younger age groups
• post-marketing commitments on immunogenicity and long-term outcomes

A precise next-12–24 month calendar requires current FDA and EMA filing status per product.

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Key Takeaways

• The clinical landscape for coagulation factor IX (human) in hemophilia B is dominated by extended half-life and administration-burden reduction strategies.
• Market growth is supported by prophylaxis penetration and EHL conversion, but gene therapy and non-factor options are the main medium-term demand disruptors.
• Exclusivity, Orange Book listing, and patent litigation must be mapped product-by-product to identify generic or biosimilar entry risk and to model pricing pressure.
• Revenue projections depend on adoption rates of EHL therapies versus substitution by gene therapy, plus net pricing under contracting and payer formulary dynamics.

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FAQs

1. What endpoints do hemophilia B trials use for factor IX prophylaxis efficacy (ABR, target trough activity, inhibitor outcomes)?
2. How does switching from standard half-life factor IX to EHL factor IX affect patient bleed rates and dosing frequency in real-world settings?
3. What drives payer decisions to prefer EHL versus older factor IX regimens for hemophilia B?
4. How do gene therapy coverage decisions typically impact factor IX treatment volumes and cost offsets?
5. What manufacturing and CMC factors most often constrain factor IX product supply during demand spikes?

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References (APA)

1. FDA. Orange Book: Approved Drug Products with Therapeutic Equivalence Evaluations. U.S. Food and Drug Administration.
2. EMA. European public assessment reports and EPAR databases for human medicines. European Medicines Agency.
3. FDA. Biologics License Application (BLA) and guidance documents for biosimilar/biologic development pathways. U.S. Food and Drug Administration.