CLINICAL TRIALS PROFILE FOR COAGULATION FACTOR IX (RECOMBINANT)

Biosimilar Clinical Trials for coagulation factor ix (recombinant)

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 (recombinant)

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 (recombinant)

Condition Name

Condition Name for coagulation factor ix (recombinant)
Intervention	Trials
Healthy	30
Hemophilia A	25
Covid-19	24
Atrial Fibrillation	24

Condition MeSH

Condition MeSH for coagulation factor ix (recombinant)
Intervention	Trials
Hemorrhage	89
COVID-19	57
Thrombosis	54
Hemophilia A	53

Clinical Trial Locations for coagulation factor ix (recombinant)

Trials by Country

Trials by Country for coagulation factor ix (recombinant)
Location	Trials
China	257
Canada	190
Italy	101
United Kingdom	99
Germany	99

Trials by US State

Trials by US State for coagulation factor ix (recombinant)
Location	Trials
California	69
Pennsylvania	61
Texas	60
New York	51
North Carolina	49

Clinical Trial Progress for coagulation factor ix (recombinant)

Clinical Trial Phase

Clinical Trial Phase for coagulation factor ix (recombinant)
Clinical Trial Phase	Trials
Phase 4	254
Phase 3	198
Phase 2/Phase 3	49

Clinical Trial Status

Clinical Trial Status for coagulation factor ix (recombinant)
Clinical Trial Phase	Trials
Completed	524
Recruiting	196
Not yet recruiting	137

Clinical Trial Sponsors for coagulation factor ix (recombinant)

Sponsor Name

Sponsor Name for coagulation factor ix (recombinant)
Sponsor	Trials
Bayer	25
National Cancer Institute (NCI)	22
Boehringer Ingelheim	21

Sponsor Type

Sponsor Type for coagulation factor ix (recombinant)
Sponsor	Trials
Other	1451
Industry	430
NIH	65

Coagulation factor ix (recombinant) Market Analysis and Financial Projection

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

Introduction to Coagulation Factor IX (Recombinant)

Coagulation Factor IX (recombinant) is a crucial treatment for patients with hemophilia B, a genetic disorder characterized by the deficiency or dysfunction of clotting factor IX. This recombinant form of factor IX is produced using DNA technology, eliminating the risk of blood-borne infections such as hepatitis C and HIV, which are associated with plasma-derived products[3].

Clinical Trials and Efficacy

RIXUBIS Clinical Trials

RIXUBIS, developed by Baxter International, was the first recombinant factor IX (rFIX) approved by the FDA for routine prophylaxis and control of bleeding episodes in adults and children with hemophilia B. Clinical trials demonstrated its efficacy in preventing and controlling bleeding episodes, as well as managing perioperative bleeding. The trials highlighted that RIXUBIS is safe and effective for long-term use, although it carries risks such as thromboembolic complications and inhibitor development[1].

Long-Acting Recombinant Coagulation Factor IX (rIX-FP)

A significant advancement in the field is the long-acting recombinant coagulation factor IX albumin fusion protein (rIX-FP). Clinical studies have shown that rIX-FP has an improved pharmacokinetic profile compared to standard rFIX, with a longer half-life and higher incremental recovery. This allows for less frequent dosing while maintaining adequate factor IX levels to prevent spontaneous bleeding episodes. The global phase 3 study on rIX-FP demonstrated its safety and efficacy in patients with severe hemophilia B[4].

BeneFIX Clinical Data

BeneFIX, another recombinant factor IX product, has been extensively studied and is indicated for adults and children with hemophilia B. Clinical data support its use in controlling and preventing bleeding episodes, as well as in perioperative management. The product is formulated to be reconstituted for intravenous injection and is available in various dosages to accommodate different patient needs[5].

Market Analysis

Current Market Size and Forecast

The global market for Coagulation Factor IX was valued at $362 million in 2023 and is projected to grow to $526 million by 2030, with a Compound Annual Growth Rate (CAGR) of 5.8% during the forecast period of 2024-2030[2].

Broader Recombinant Coagulation Factors Market

The broader market for recombinant coagulation factors, which includes both factor VIII and factor IX, is expected to reach $26.29 billion by 2031, growing at a CAGR of 8.6% from 2024 to 2031. This growth is driven by advancements in recombinant technology, increasing demand for the treatment of hemophilia, and rising awareness about the benefits of recombinant coagulation factors[3].

Key Market Players

The market for Coagulation Factor IX is dominated by several key players, including Pfizer, CSL Behring, Grifols, Novo Nordisk, Bioverativ, Sanquin, Takeda, Baxter, and Aptevo. These companies are driving innovation and expansion in the market through continuous research and development[2].

Market Drivers and Opportunities

Advancements in Technology

Advancements in recombinant DNA technology have significantly improved the production and efficacy of coagulation factor IX. Long-acting formulations like rIX-FP offer patients the convenience of less frequent dosing, enhancing their quality of life and reducing the burden of treatment[4].

Increasing Incidence and Diagnosis of Hemophilia

The increasing incidence and diagnosis rates of hemophilia B are driving the demand for recombinant coagulation factor IX. As more patients are diagnosed and treated, the market is expected to grow substantially[3].

Growing Awareness and Accessibility

Growing awareness about the benefits of recombinant coagulation factors, along with improving accessibility of these treatments, is another key driver. This is particularly significant in regions where access to advanced healthcare is expanding[3].

Challenges and Risks

Thromboembolic Complications

One of the significant risks associated with the use of coagulation factor IX products is the development of thromboembolic complications. Patients, especially those with liver disease or at risk for thromboembolic events, need to be closely monitored for early signs of these complications[1].

Inhibitor Development

Another challenge is the potential for inhibitor development, which can reduce the efficacy of the treatment. This is a class adverse reaction seen with recombinant factor IX products and requires careful management[1].

Cost and Accessibility

Despite the advancements, the high cost of recombinant coagulation factors remains a significant barrier to access for many patients. Efforts to make these treatments more affordable and accessible are crucial for expanding the market and improving patient outcomes[3].

Key Takeaways

• Clinical Efficacy: Recombinant coagulation factor IX products, such as RIXUBIS and BeneFIX, have demonstrated efficacy in clinical trials for preventing and controlling bleeding episodes in patients with hemophilia B.
• Market Growth: The global market for Coagulation Factor IX is projected to grow from $362 million in 2023 to $526 million by 2030, driven by technological advancements and increasing demand.
• Long-Acting Formulations: Long-acting formulations like rIX-FP offer improved pharmacokinetics, allowing for less frequent dosing and enhanced patient convenience.
• Challenges: Despite the benefits, the market faces challenges such as thromboembolic complications, inhibitor development, and high treatment costs.

FAQs

What is the primary use of recombinant coagulation factor IX?

Recombinant coagulation factor IX is primarily used for the control and prevention of bleeding episodes, perioperative management, and routine prophylaxis in patients with hemophilia B.

Which companies are key players in the Coagulation Factor IX market?

Key players include Pfizer, CSL Behring, Grifols, Novo Nordisk, Bioverativ, Sanquin, Takeda, Baxter, and Aptevo.

What are the risks associated with the use of recombinant coagulation factor IX?

Risks include thromboembolic complications, inhibitor development, and other adverse reactions such as dysgeusia and pain in extremity.

How does the long-acting recombinant coagulation factor IX (rIX-FP) differ from standard rFIX?

rIX-FP has an improved pharmacokinetic profile with a longer half-life and higher incremental recovery, allowing for less frequent dosing.

What is the forecasted market size for Coagulation Factor IX by 2030?

The global market for Coagulation Factor IX is forecasted to reach $526 million by 2030.

Sources

1. FDA Approves Baxter's RIXUBIS [Coagulation Factor IX (Recombinant)]: Baxter International Inc., September 15, 2014.
2. Coagulation Factor IX - Market Size: Valuates Reports.
3. Recombinant Coagulation Factors Market Size & Forecast: Meticulous Research.
4. Long-acting recombinant coagulation factor IX albumin fusion protein: Blood, American Society of Hematology.
5. BENEFIX® (Coagulation Factor IX (Recombinant)): Pfizer Medical Information.