Paroxysmal Nocturnal Hemoglobinuria Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update

June 2022 | 131 pages | ID: PF65B2FF6789EN
Global Markets Direct

Abstracts

Paroxysmal Nocturnal Hemoglobinuria Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update

SUMMARY

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Paroxysmal Nocturnal Hemoglobinuria – Drugs In Development, 2022, provides an overview of the Paroxysmal Nocturnal Hemoglobinuria (Hematological Disorders) pipeline landscape.

Paroxysmal nocturnal hemoglobinuria is a rare disease in which red blood cells break down earlier than normal. Symptoms include abdominal pain, back pain, dark urine, easy bruising or bleeding, headache and shortness of breath. The predisposing factors include age, obesity and hormone therapy. Treatment includes surgery, chemotherapy and radiation therapy.

REPORT HIGHLIGHTS

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Paroxysmal Nocturnal Hemoglobinuria – Drugs In Development, 2022, provides comprehensive information on the therapeutics under development for Paroxysmal Nocturnal Hemoglobinuria (Hematological Disorders), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The guide covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases.

The Paroxysmal Nocturnal Hemoglobinuria (Hematological Disorders) pipeline guide also reviews of key players involved in therapeutic development for Paroxysmal Nocturnal Hemoglobinuria and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Phase III, Phase II, Phase I, IND/CTA Filed, Preclinical and Discovery stages are 10, 6, 6, 1, 15 and 1 respectively.

Paroxysmal Nocturnal Hemoglobinuria (Hematological Disorders) pipeline guide helps in identifying and tracking emerging players in the market and their portfolios, enhances decision making capabilities and helps to create effective counter strategies to gain competitive advantage. The guide is built using data and information sourced from Global Markets Direct’s proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

Note: Certain content / sections in the pipeline guide may be removed or altered based on the availability and relevance of data.

SCOPE

• The pipeline guide provides a snapshot of the global therapeutic landscape of Paroxysmal Nocturnal Hemoglobinuria (Hematological Disorders).
• The pipeline guide reviews pipeline therapeutics for Paroxysmal Nocturnal Hemoglobinuria (Hematological Disorders) by companies and universities/research institutes based on information derived from company and industry-specific sources.
• The pipeline guide covers pipeline products based on several stages of development ranging from pre-registration till discovery and undisclosed stages.
• The pipeline guide features descriptive drug profiles for the pipeline products which comprise, product description, descriptive licensing and collaboration details, R&D brief, MoA & other developmental activities.
• The pipeline guide reviews key companies involved in Paroxysmal Nocturnal Hemoglobinuria (Hematological Disorders) therapeutics and enlists all their major and minor projects.
• The pipeline guide evaluates Paroxysmal Nocturnal Hemoglobinuria (Hematological Disorders) therapeutics based on mechanism of action (MoA), drug target, route of administration (RoA) and molecule type.
• The pipeline guide encapsulates all the dormant and discontinued pipeline projects.
• The pipeline guide reviews latest news related to pipeline therapeutics for Paroxysmal Nocturnal Hemoglobinuria (Hematological Disorders)

REASONS TO BUY

• Procure strategically important competitor information, analysis, and insights to formulate effective R&D strategies.
• Recognize emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage.
• Find and recognize significant and varied types of therapeutics under development for Paroxysmal Nocturnal Hemoglobinuria (Hematological Disorders).
• Classify potential new clients or partners in the target demographic.
• Develop tactical initiatives by understanding the focus areas of leading companies.
• Plan mergers and acquisitions meritoriously by identifying key players and it’s most promising pipeline therapeutics.
• Formulate corrective measures for pipeline projects by understanding Paroxysmal Nocturnal Hemoglobinuria (Hematological Disorders) pipeline depth and focus of Indication therapeutics.
• Develop and design in-licensing and out-licensing strategies by identifying prospective partners with the most attractive projects to enhance and expand business potential and scope.
• Adjust the therapeutic portfolio by recognizing discontinued projects and understand from the know-how what drove them from pipeline.

Contents

Introduction
Global Markets Direct Report Coverage
Paroxysmal Nocturnal Hemoglobinuria – Overview
Paroxysmal Nocturnal Hemoglobinuria – Therapeutics Development
Pipeline Overview
Pipeline by Companies
Products under Development by Companies
Paroxysmal Nocturnal Hemoglobinuria – Therapeutics Assessment
Assessment by Target
Assessment by Mechanism of Action
Assessment by Route of Administration
Assessment by Molecule Type
Paroxysmal Nocturnal Hemoglobinuria – Companies Involved in Therapeutics Development
Paroxysmal Nocturnal Hemoglobinuria – Drug Profiles
Paroxysmal Nocturnal Hemoglobinuria – Dormant Projects
Paroxysmal Nocturnal Hemoglobinuria – Discontinued Products
Paroxysmal Nocturnal Hemoglobinuria – Product Development Milestones
Featured News & Press Releases
Jun 12, 2022: Samsung Bioepis presents new phase 3 Study of SB12 (Eculizumab), a proposed biosimilar to Soliris, at the European Hematology Association (EHA) Congress 2022
Jun 10, 2022: CANbridge CAN106 phase 1 data presented at the European Hematology Association 2022 Congress
Jun 10, 2022: Apellis and Sobi report new data reinforcing the robust efficacy and safety profile of EMPAVELI (pegcetacoplan) for PNH at EHA 2022 Congress
May 12, 2022: Apellis announces seven abstracts in PNH to be presented at the European Hematology Association Congress
May 12, 2022: CANbridge to present CAN106 phase 1 data at the European Hematology Association 2022 Congress
Apr 08, 2022: BioCryst pauses enrollment in BCX9930 clinical trials
Mar 28, 2022: CANbridge doses first subject in Phase Ib/II PNH treatment trial in China
Feb 07, 2022: CANbridge reports positive top-line CAN106 Phase 1 data
Feb 04, 2022: Novartis Japan developing Iptacopan as priority product, initially targeting 3 indications
Jan 07, 2022: BioCryst begins patient enrollment in REDEEM-1 pivotal trial evaluating BCX9930 as oral monotherapy for patients with PNH
Dec 15, 2021: Apellis and Sobi announce EU approval of Aspaveli (pegcetacoplan) for treatment of PNH
Dec 13, 2021: Apellis and Sobi Report Empaveli (pegcetacoplan) demonstrated sustained normalization of clinical measures in a broad PNH patient population
Dec 11, 2021: Samsung Bioepis’ SB12 Soliris (Eculizumab) Biosimilar Demonstrates PK, PD Bioequivalence in phase 1 study
Nov 29, 2021: BioCryst begins patient enrollment in REDEEM-2 pivotal trial evaluating BCX9930 as oral monotherapy for patients with PNH
Nov 04, 2021: Apellis to present new data reinforcing EMPAVELI (pegcetacoplan) efficacy and safety in patients with PNH at the 2021 ASH Annual Meeting
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer

List Of Tables

LIST OF TABLES

Number of Products under Development for Paroxysmal Nocturnal Hemoglobinuria, 2022
Number of Products under Development by Companies, 2022
Number of Products under Development by Companies, 2022 (Contd..1)
Products under Development by Companies, 2022
Products under Development by Companies, 2022 (Contd..1)
Products under Development by Companies, 2022 (Contd..2)
Number of Products by Stage and Target, 2022
Number of Products by Stage and Mechanism of Action, 2022
Number of Products by Stage and Route of Administration, 2022
Number of Products by Stage and Molecule Type, 2022
Paroxysmal Nocturnal Hemoglobinuria – Pipeline by Aevitas Therapeutics Inc, 2022
Paroxysmal Nocturnal Hemoglobinuria – Pipeline by Akari Therapeutics Plc, 2022
Paroxysmal Nocturnal Hemoglobinuria – Pipeline by Alexion Pharmaceuticals Inc, 2022
Paroxysmal Nocturnal Hemoglobinuria – Pipeline by Alnylam Pharmaceuticals Inc, 2022
Paroxysmal Nocturnal Hemoglobinuria – Pipeline by Amgen Inc, 2022
Paroxysmal Nocturnal Hemoglobinuria – Pipeline by Amyndas Pharmaceuticals LLC, 2022
Paroxysmal Nocturnal Hemoglobinuria – Pipeline by Apellis Pharmaceuticals Inc, 2022
Paroxysmal Nocturnal Hemoglobinuria – Pipeline by Arrowhead Pharmaceuticals Inc, 2022
Paroxysmal Nocturnal Hemoglobinuria – Pipeline by Attune Pharmaceuticals, 2022
Paroxysmal Nocturnal Hemoglobinuria – Pipeline by Biocad, 2022
Paroxysmal Nocturnal Hemoglobinuria – Pipeline by BioCryst Pharmaceuticals Inc, 2022
Paroxysmal Nocturnal Hemoglobinuria – Pipeline by CANbridge Life Sciences Ltd, 2022
Paroxysmal Nocturnal Hemoglobinuria – Pipeline by Chugai Pharmaceutical Co Ltd, 2022
Paroxysmal Nocturnal Hemoglobinuria – Pipeline by Homology Medicines Inc, 2022
Paroxysmal Nocturnal Hemoglobinuria – Pipeline by ImmunAbs Inc, 2022
Paroxysmal Nocturnal Hemoglobinuria – Pipeline by ISU ABXIS Co Ltd, 2022
Paroxysmal Nocturnal Hemoglobinuria – Pipeline by Kira Pharmaceuticals, 2022
Paroxysmal Nocturnal Hemoglobinuria – Pipeline by Novartis AG, 2022
Paroxysmal Nocturnal Hemoglobinuria – Pipeline by NovelMed Therapeutics Inc, 2022
Paroxysmal Nocturnal Hemoglobinuria – Pipeline by Omeros Corp, 2022
Paroxysmal Nocturnal Hemoglobinuria – Pipeline by Prestige BioPharma Ltd, 2022
Paroxysmal Nocturnal Hemoglobinuria – Pipeline by Ra Pharmaceuticals Inc, 2022
Paroxysmal Nocturnal Hemoglobinuria – Pipeline by Rallybio Corp, 2022
Paroxysmal Nocturnal Hemoglobinuria – Pipeline by Regeneron Pharmaceuticals Inc, 2022
Paroxysmal Nocturnal Hemoglobinuria – Pipeline by Samsung Bioepis Co Ltd, 2022
Paroxysmal Nocturnal Hemoglobinuria – Pipeline by Shanghai ComGen Biopharmaceutical Co Ltd, 2022
Paroxysmal Nocturnal Hemoglobinuria – Pipeline by Shanghai Meiyue Biotechnology Development Co Ltd, 2022
Paroxysmal Nocturnal Hemoglobinuria – Pipeline by Tasly Biopharmaceuticals Co Ltd, 2022
Paroxysmal Nocturnal Hemoglobinuria – Pipeline by Turgut Ilaclari AS, 2022
Paroxysmal Nocturnal Hemoglobinuria – Pipeline by Wuhan LL Science and Technology Development Co Ltd, 2022
Paroxysmal Nocturnal Hemoglobinuria – Pipeline by Zydus Lifesciences Ltd, 2022
Paroxysmal Nocturnal Hemoglobinuria – Dormant Projects, 2022
Paroxysmal Nocturnal Hemoglobinuria – Discontinued Products, 2022

List Of Figures

LIST OF FIGURES

Number of Products under Development for Paroxysmal Nocturnal Hemoglobinuria, 2022
Number of Products under Development by Companies, 2022
Number of Products by Targets, 2022
Number of Products by Stage and Targets, 2022
Number of Products by Mechanism of Actions, 2022
Number of Products by Stage and Mechanism of Actions, 2022
Number of Products by Routes of Administration, 2022
Number of Products by Stage and Routes of Administration, 2022
Number of Products by Top 10 Molecule Types, 2022
Number of Products by Stage and Top 10 Molecule Types, 2022