Hemophilia Clinical Trials and Studies 2025: EMA, PDMA, FDA Approvals, Mechanism of Action, ROA, NDA, IND, and Companies | Centessa Pharmaceuticals, Alnylam Pharmaceuticals, ASC Therapeutics, Spark Therapeutics, Freeline Therapeutics, BioMarin Pharmaceuti

DelveInsight's, "Hemophilia Pipeline Insight 2025" report provides comprehensive insights about 80+ companies and 80+ pipeline drugs in Hemophilia pipeline landscape. It covers the Hemophilia pipeline drug profiles, including clinical and nonclinical stage products. It also covers the Hemophilia therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

Stay ahead with the latest insights! Download DelveInsight's comprehensive Hemophilia Pipeline Report to explore emerging therapies, key Hemophilia Companies, and future Hemophilia treatment landscapes @ Hemophilia Pipeline Outlook Report- https://www.delveinsight.com/sample-request/hemophilia-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=ypr

Key Takeaways from the Hemophilia Pipeline Report
• In March 2025, Novo Nordisk A/S announced a study is investigating how Mim8 works compared to other medicines in people with haemophilia A, who either have inhibitors or do not have inhibitors. Mim8 is a new medicine that will be used for prevention of bleeding episodes. Mim8 works by replacing the function of the missing clotting factor VIII (FVIII).
• In March 2025, Pfizer conducted a study to establish baseline prospective efficacy data of current FIX prophylaxis replacement therapy in the usual care setting of hemophilia B subjects, who are negative for nAb to AAV-Spark100, prior to the Phase 3 gene therapy study. To establish baseline prospective efficacy data of current FVIII prophylaxis replacement therapy in the usual care setting of hemophilia A subjects, who are negative for nAb to AAV6, prior to the Phase 3 gene therapy study.
• In January 2025:- Staidson (Beijing) Biopharmaceuticals Co., Ltd:- This study will assess the safety and efficacy of multiple-dose of STSP-0601 for the treatment of bleeding episodes in hemophilia A or B patients with inhibitor.
• In January 2025:- ApcinteX Ltd:- The purpose of the study is to evaluate the efficacy, safety, tolerability and pharmacokinetics of prophylactic SerpinPC administered subcutaneously (SC) to participants with severe hemophilia A (HemA) (with or without inhibitors) or moderately severe to severe hemophilia B (HemB) (without inhibitors) as part of the SerpinPC registrational program.
• In January 2025:- Novo Nordisk A/S- This study will test how well a new medicine called concizumab works in the body of people with haemophilia A or B without inhibitors. The purpose is to show that concizumab can prevent bleeds in the body and is safe to use. Participants who usually only take medicine to treat bleeds (on-demand) will be placed in one of two groups.
• In January 2025:- Pfizer:- To establish baseline prospective efficacy data of current FIX prophylaxis replacement therapy in the usual care setting of hemophilia B subjects, who are negative for nAb to AAV-Spark100, prior to the Phase 3 gene therapy study. To establish baseline prospective efficacy data of current FVIII prophylaxis replacement therapy in the usual care setting of hemophilia A subjects, who are negative for nAb to AAV6, prior to the Phase 3 gene therapy study. The enrollment for hemophilia A participants is completed. At this time participants are only being enrolled for hemophilia B cohort.
• In January 2025:- CSL Behring- This study will investigate the pharmacokinetics (PK), efficacy, and safety of rIX-FP for the routine prophylaxis of bleeding episodes in male Chinese previously treated patients (PTPs) with hemophilia B (FIX activity of ≤ 2%). In addition to the scheduled rIX-FP prophylaxis regimen, subjects may also receive rIX-FP episodic (on-demand) treatment for breakthrough bleeding episodes and rIX-FP for the prophylaxis and treatment of bleeding in emergency surgical procedures.
• In January 2025:- Octapharma:- Female patients with mild to moderate haemophilia A will often need FVIII concentrates to provide haemostatic cover during major surgery. This prospective, open-label, non-controlled, single-arm, multinational, multicentre study aims to evaluate the overall perioperative haemostatic efficacy of Nuwiq in women/girls over 12 with haemophilia A undergoing major surgery requiring FVIII treatment
• DelveInsight's Hemophilia pipeline report depicts a robust space with 80+ active players working to develop 80+ pipeline therapies for Hemophilia treatment.
• The leading Hemophilia Companies such as Centessa Pharmaceuticals, Alnylam Pharmaceuticals, ASC Therapeutics, Spark Therapeutics, Freeline Therapeutics, BioMarin Pharmaceutical, Staidson Beijing BioPharmaceuticals, Pfizer, Sangamo Therapeutics, Amunix, Bioverativ, Novo Nordisk and others.
• Promising Hemophilia Therapies such as ELOCTA, ALPROLIX, Mim8, NNC0365-3769, MG1113, STSP-0601 for Injection, SerpinPC, Fitusiran, Esperoct and others.

Discover how the Hemophilia treatment paradigm is evolving. Access DelveInsight's in-depth Hemophilia Pipeline Analysis for a closer look at promising breakthroughs @ Hemophilia Clinical Trials and Studies- https://www.delveinsight.com/sample-request/hemophilia-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=ypr

Hemophilia Emerging Drugs Profile
• SerpinPC: Centessa Pharmaceuticals
SerpinPC, a specific inhibitor of activated protein C (APC), for the treatment of hemophilia A and hemophilia B. SerpinPC has been observed to be well-tolerated in the clinical setting, associated with promising reductions in bleeding rates, and has PK suitable for infrequent subcutaneous dosing without the need for factor replacement. SerpinPC has human genetic target validation and established proof of concept Phase 2a clinical data.

• Fitusiran: Alnylam Pharmaceuticals
Fitusiran (ALN-AT3) is a subcutaneously administered, investigational RNAi therapeutic targeting antithrombin (AT) in development for the treatment of hemophilia and rare bleeding disorders (RBDs) by their collaborators at Sanofi Genzyme. Currently, the drug is in Phase III stage of Clinical trial evaluation for the treatment of Hemophilia.

• ASC 618: ASC Therapeutics
ASC618 is an AAV8-based gene therapy for the treatment of hemophilia A, affecting approximately 1 of every 5000 live-born males. ASC618 incorporates a novel liver-specific promoter and a bioengineered, codon-optimized B domain-deleted FVIII variant; in preclinical studies, ASC618 exhibits at least a 10-fold increase in the biosynthesis and secretion of FVIII compared with native human FVIII bioengineered gene constructs. ASC618 has the potential to increase durability of clotting factor biosynthesis and secretion by minimizing cellular stress and induction of the unfolded protein response, which may lead to diminished FVIII production from liver cells. ASC Therapeutics will conduct a phase 1/2 clinical trial to evaluate the safety, tolerability, and preliminary efficacy of ASC618. The program received IND clearance from the U.S. Food and Drug Administration.

Get a detailed analysis of the latest innovations in the Hemophilia pipeline. Explore DelveInsight's expert-driven report today! @ Hemophilia Unmet Needs- https://www.delveinsight.com/sample-request/hemophilia-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=ypr

Hemophilia Companies
Centessa Pharmaceuticals, Alnylam Pharmaceuticals, ASC Therapeutics, Spark Therapeutics, Freeline Therapeutics, BioMarin Pharmaceutical, Staidson Beijing BioPharmaceuticals, Pfizer, Sangamo Therapeutics, Amunix, Bioverativ, Novo Nordisk and others.

Hemophilia pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as
• Intra-articular
• Intraocular
• Intrathecal
• Intravenous
• Ophthalmic
• Oral
• Parenteral
• Subcutaneous
• Topical
• Transdermal

Hemophilia Products have been categorized under various Molecule types such as
• Oligonucleotide
• Peptide
• Small molecule

Download DelveInsight's latest report to gain strategic insights into upcoming therapies and key developments @ Hemophilia Market Drivers and Barriers, and Future Perspectives- https://www.delveinsight.com/sample-request/hemophilia-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=ypr

Scope of the Hemophilia Pipeline Report
• Coverage- Global
• Hemophilia Companies- Centessa Pharmaceuticals, Alnylam Pharmaceuticals, ASC Therapeutics, Spark Therapeutics, Freeline Therapeutics, BioMarin Pharmaceutical, Staidson Beijing BioPharmaceuticals, Pfizer, Sangamo Therapeutics, Amunix, Bioverativ, Novo Nordisk and others.
• Hemophilia Therapies- ELOCTA, ALPROLIX, Mim8, NNC0365-3769, MG1113, STSP-0601 for Injection, SerpinPC, Fitusiran, Esperoct andothers.
• Hemophilia Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination
• Hemophilia Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III

Which companies are leading the race in Hemophilia drug development? Find out in DelveInsight's exclusive Hemophilia Pipeline Report-access it now! @ Hemophilia Emerging Drugs and Major Companies- https://www.delveinsight.com/sample-request/hemophilia-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=ypr

Table of Content
1. Introduction
2. Executive Summary
3. Hemophilia: Overview
4. Pipeline Therapeutics
5. Therapeutic Assessment
6. Hemophilia- DelveInsight's Analytical Perspective
7. Late Stage Products (Phase III)
8. Fitusiran: Alnylam Pharmaceuticals
9. Drug profiles in the detailed report.....
10. Mid Stage Products (Phase II)
11. SerpinPC: Centessa Pharmaceuticals
12. Drug profiles in the detailed report.....
13. Early Stage Products (Phase I)
14. Drug name: Company Name
15. Drug profiles in the detailed report.....
16. Preclinical and Discovery Stage Products
17. Drug name: Company Name
18. Drug profiles in the detailed report.....
19. Inactive Products
20. Hemophilia Key Companies
21. Hemophilia Key Products
22. Hemophilia- Unmet Needs
23. Hemophilia- Market Drivers and Barriers
24. Hemophilia- Future Perspectives and Conclusion
25. Hemophilia Analyst Views
26. Hemophilia Key Companies
27. Appendix

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