DelveInsight’s, “Paroxysmal Nocturnal Haemoglobinuria Pipeline Insight 2024” report provides comprehensive insights about 20+ companies and 25+ pipeline drugs in Paroxysmal Nocturnal Haemoglobinuria pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

 

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Key Takeaways from the Paroxysmal Nocturnal Hemoglobinuria Pipeline Report

• In October 2024:- Alexion Pharmaceuticals- A Phase 3, Single-arm, Open-label, Multicenter Study to Assess the Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Ravulizumab in Complement Inhibitor Treatment Naïve Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) in China. The primary objective of this study is to evaluate the efficacy of ravulizumab in adult participants with PNH.
• In October 2024:- Hoffmann-La Roche- A Phase III, Randomized, Open-Label, Active-Controlled, Multicenter Study Evaluating The Safety, Pharmacokinetics, Pharmacodynamic And Efficacy of Crovalimab Versus Eculizumab In Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Complement Inhibitors. A study designed to evaluate the safety of crovalimab with eculizumab in participants with PNH currently treated with complement inhibitors. This study will enroll approximately 190 participants.
• In October 2024:- Kira Pharmacenticals (US), LLC- The purpose of this study is to evaluate the safety, tolerability, immunogenicity, pharmacokinetics, pharmacodynamics, and efficacy of KP104 in complement inhibitor-naïve participants with PNH. The study will be conducted in 2 parts. Part 1 is a dose-selection study to assess escalating doses and varying dose intervals of KP104. Part 2 is a proof-of-concept (POC) study assessing the efficacy of the optimal intravenous (IV) loading dose followed by the optimal maintenance dose and regimen of KP104. Participants who complete the Initial Treatment Period and demonstrate benefit from KP104 will be eligible for a 9-month open-label extension (OLE) treatment period.
• In October 2024:- Regeneron Pharmaceuticals- This study is researching an experimental treatment combination with two experimental drugs called pozelimab and cemdisiran. The study is focused on patients with paroxysmal nocturnal hemoglobinuria (PNH). The aim of this study is to see how safe and effective the pozelimab + cemdisiran combination is for patients with PNH in the long term. The pozelimab + cemdisiran combination may be referred to as “study drugs” in this section.
• In October 2024:- Novartis Pharmaceuticals- The purpose of this phase 3 open-label, single arm, multicenter study is to evaluate the long-term safety, tolerability and efficacy of iptacopan in patients with PNH and to provide access to patients who have completed (without tapering down) Phase 2 and Phase 3 trials and derived benefit from iptacopan treatment.
• DelveInsight’s Paroxysmal Nocturnal Hemoglobinuria pipeline report depicts a robust space with 20+ active players working to develop 25+ pipeline therapies for Paroxysmal Nocturnal Hemoglobinuria treatment.
• The leading Paroxysmal Nocturnal Hemoglobinuria Companies such as Hoffmann-La Roche, Apellis Pharmaceuticals, Regeneron Pharmaceuticals, Biocad, AKARI Therapeutics, Alexion Pharmaceuticals, Novartis Pharmaceuticals, Amgen, BioCryst Pharmaceuticals, MorphoSys, Ra Pharmaceuticals, Alnylam Pharmaceuticals, Arrowhead Pharmaceuticals, Wuhan Createrna Science and Technology, CANbridge, Attune Pharmaceuticals, RallyBio, and others.
• Promising Paroxysmal Nocturnal Hemoglobinuria Therapies such as Eculizumab, BCX9930, Crovalimab, HRS-5965 tablets, Iptacopan, vMY008211A tablets, Pozelimab, Cemdisiran, KP104, Eculizumab, and others.

 

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Paroxysmal Nocturnal Hemoglobinuria Emerging Drugs Profile

• Crovalimab: Hoffman-La-Roche

Crovalimab (RG6107) is a humanised complement inhibitor C5 monoclonal antibody discovered by Chugai using recycling antibody technology. By blocking the cleavage of C5 to C5a and C5b, it is expected to inhibit complement activation, which is the cause of a number of diseases. As the complement system is a key innate immune defense mechanism, we plan to study the potential of this antibody in a broader range of complement-mediated diseases. A phase III clinical trial is evaluating crovalimab for the treatment of paroxysmal nocturnal hemoglobinuria.

• Pozelimab: Regeneron Pharmaceuticals

Pozelimab is an investigational, fully-human monoclonal antibody designed to block complement factor C5 and prevent the destruction of red blood cells (hemolysis) that cause the symptoms of PNH and other diseases mediated by complement pathway activity. It is an IgG4 antibody that binds with high affinity to wild-type and variant human C5 and blocks its activity. Pozelimab was invented using Regeneron’s proprietary VelocImmune technology, which uses a unique genetically-humanized mouse to produce optimized fully-human antibodies. Pozelimab is currently under clinical development, and its safety and efficacy have not been evaluated by any regulatory authority.

 

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Paroxysmal Nocturnal Hemoglobinuria Companies

Hoffmann-La Roche, Apellis Pharmaceuticals, Regeneron Pharmaceuticals, Biocad, AKARI Therapeutics, Alexion Pharmaceuticals, Novartis Pharmaceuticals, Amgen, BioCryst Pharmaceuticals, MorphoSys, Ra Pharmaceuticals, Alnylam Pharmaceuticals, Arrowhead Pharmaceuticals, Wuhan Createrna Science and Technology, CANbridge, Attune Pharmaceuticals, RallyBio, and others.

 

Paroxysmal Nocturnal Haemoglobinuria pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

• Oral
• Parenteral
• Intravitreal
• Subretinal
• Topical
• Molecule Type

 

Paroxysmal Nocturnal Hemoglobinuria Products have been categorized under various Molecule types such as

• Monoclonal Antibody
• Peptides
• Polymer
• Small molecule
• Gene therapy
• Product Type

 

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Scope of the Paroxysmal Nocturnal Hemoglobinuria Pipeline Report

• Coverage- Global
• Paroxysmal Nocturnal Hemoglobinuria Companies- Hoffmann-La Roche, Apellis Pharmaceuticals, Regeneron Pharmaceuticals, Biocad, AKARI Therapeutics, Alexion Pharmaceuticals, Novartis Pharmaceuticals, Amgen, BioCryst Pharmaceuticals, MorphoSys, Ra Pharmaceuticals, Alnylam Pharmaceuticals, Arrowhead Pharmaceuticals, Wuhan Createrna Science and Technology, CANbridge, Attune Pharmaceuticals, RallyBio, and others.
• Paroxysmal Nocturnal Hemoglobinuria Therapies- Eculizumab, BCX9930, Crovalimab, HRS-5965 tablets, Iptacopan, vMY008211A tablets, Pozelimab, Cemdisiran, KP104, Eculizumab, and others.
• Paroxysmal Nocturnal Hemoglobinuria Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination
• Paroxysmal Nocturnal Hemoglobinuria Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III

 

For a detailed overview of our latest research findings and future plans, read the full details of Paroxysmal Nocturnal Hemoglobinuria Pipeline on our website @ Paroxysmal Nocturnal Hemoglobinuria Emerging Drugs and Companies

 

Table of Content

1. Introduction
2. Executive Summary
3. Paroxysmal Nocturnal Haemoglobinuria: Overview
4. Pipeline Therapeutics
5. Therapeutic Assessment
6. Paroxysmal Nocturnal Haemoglobinuria – DelveInsight’s Analytical Perspective
7. In-depth Commercial Assessment
8. Paroxysmal Nocturnal Haemoglobinuria Collaboration Deals
9. Late Stage Products (Phase III)
10. Crovalimab: Hoffman-La-Roche
11. Drug profiles in the detailed report…..
12. Mid Stage Products (Phase II)
13. BCX9930: BioCryst pharmaceuticals
14. Drug profiles in the detailed report…..
15. Eary Stage Products (Phase I)
16. CAN106: CanBridge
17. Drug profiles in the detailed report…..
18. Inactive Products
19. Paroxysmal Nocturnal Haemoglobinuria Key Companies
20. Paroxysmal Nocturnal Haemoglobinuria Key Products
21. Paroxysmal Nocturnal Haemoglobinuria- Unmet Needs
22. Paroxysmal Nocturnal Haemoglobinuria- Market Drivers and Barriers
23. Paroxysmal Nocturnal Haemoglobinuria- Future Perspectives and Conclusion
24. Paroxysmal Nocturnal Haemoglobinuria Analyst Views
25. Paroxysmal Nocturnal Haemoglobinuria Key Companies
26. Appendix

 

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