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Cystic Fibrosis (CF) - Pipeline Insight, 2020

  • ID: 4989149
  • Drug Pipelines
  • September 2020
  • Region: Global
  • 102 pages
  • DelveInsight
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FEATURED COMPANIES

  • AbbVie
  • Allergan PLC
  • Chiesi Farmaceutici SpA
  • Gilead Sciences
  • Novartis AG
  • Pharmaxis Ltd
  • MORE
This “Cystic Fibrosis (CF) - Pipeline Insight, 2020,” report provides comprehensive insights about 50+ companies and 50+ pipeline drugs in Cystic Fibrosis 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.

Geography Covered
  • Global coverage
Cystic Fibrosis Understanding

Cystic Fibrosis (CF): Overview

Cystic fibrosis is an inherited disease characterized by the buildup of thick, sticky mucus that can damage many of the body's organs. The disorder's most common signs and symptoms include progressive damage to the respiratory system and chronic digestive system problems. The features of the disorder and their severity varies among affected individuals.

Mucus is a slippery substance that lubricates and protects the linings of the airways, digestive system, reproductive system, and other organs and tissues. In people with cystic fibrosis, the body produces mucus that is abnormally thick and sticky. This abnormal mucus can clog the airways, leading to severe problems with breathing and bacterial infections in the lungs. These infections cause chronic coughing, wheezing, and inflammation. Over time, mucus buildup and infections result in permanent lung damage, including the formation of scar tissue (fibrosis) and cysts in the lungs.

Symptoms

The most common symptoms of Cystic fibrosis are:
  • salty-tasting skin
  • persistent coughing
  • shortness of breath
  • wheezing
  • poor weight gain in spite of excessive appetite
  • greasy, bulky stools
  • nasal polyps, or small, fleshy growths found in the nose
Causes

Mutations in the CFTR gene cause cystic fibrosis. The CFTR gene provides instructions for making a channel that transports negatively charged particles called chloride ions into and out of cells. Chloride is a component of sodium chloride, a common salt found in sweat. Chloride also has important functions in cells; for example, the flow of chloride ions helps control the movement of water in tissues, which is necessary for the production of thin, freely flowing mucus.

Diagnosis

Cystic fibrosis is usually diagnosed through a sweat test. Sweat is collected and the amount of chloride, a component of salt in the sweat, is measured. A high level of chloride is an indication of Cystic fibrosis. Genetic tests can also be carried out by analyzing cheek cells or a blood sample. These tests are mainly used to find out if a person carries the CF gene, but they can also be used to confirm a CF diagnosis following an unclear sweat test result.

Treatment

There is currently no cure for Cystic fibrosis. Treatment can manage the symptoms of the disease, however, and improve quality of life. Symptoms can vary and treatment plans will be individualized. Management includes:
  • Airway clearance: It is crucial for people with Cystic fibrosis to get rid of mucus from their lungs to allow clear breathing and minimize lung infections.
  • Antibiotics are an important part of regular care. These can be taken orally, intravenously, or through inhalation.
  • Other drugs, such as ibuprofen and azithromycin, have been found to preserve and improve lung function, and are now considered to be a part of standard therapy for people with Cystic fibrosis.
Cystic Fibrosis Emerging Drugs Chapters

This segment of the Cystic Fibrosis report encloses its detailed analysis of various drugs in different stages of clinical development, including phase II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.

Cystic Fibrosis Emerging Drugs

MRT5005: Translate Bio

MRT5005 is the first clinical-stage mRNA product candidate designed to address the underlying cause of CF by delivering mRNA encoding fully functional cystic fibrosis transmembrane conductance regulator (CFTR) protein to the lung epithelial cells through nebulization. MRT5005 is being developed to treat all patients with CF, regardless of the underlying genetic mutation, including those with limited or no CFTR protein. This mRNA therapeutic remains on track to report results from its ongoing multiple-ascending dose (MAD) portion of Phase 1/2 clinical trial in the third quarter of 2020.

Cystic Fibrosis: Therapeutic Assessment

This segment of the report provides insights about the different Cystic Fibrosis drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Cystic Fibrosis

There are approx. 50+ key companies which are developing the therapies for Cystic Fibrosis. The companies which have their Cystic Fibrosis drug candidates in the mid to advanced stage, i.e. phase III and Phase II include, SolAeroMed Inc., Eloxx Pharmaceuticals, Inc., Corbus Pharmaceuticals Inc., Laurent Pharmaceuticals Inc. etc.

Phases

This report covers around 50+ products under different phases of clinical development like
  • Mid-stage products (Phase II and Phase I/II)
  • Early-stage products (Phase I/II and Phase I) along with the details of
  • Pre-clinical and Discovery stage candidates
  • Discontinued & Inactive candidates
  • Route of Administration
Cystic Fibrosis pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as
  • Subcutaneous
  • Intravenous
  • Intramuscular
  • Oral
  • Molecule Type
Products have been categorized under various Molecule types such as
  • Small molecules
  • RNA
  • Gene therapies
  • Immunotherapies
  • Vaccines
  • Product Type
The drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

Cystic Fibrosis: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Cystic Fibrosis therapeutic drugs key players involved in developing key drugs.

Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Cystic Fibrosis drugs.

Report Highlights
  • The companies and academics are working to assess challenges and seek opportunities that could influence Cystic Fibrosis R&D. The therapies under development are focused on novel approaches to treat/improve Cystic Fibrosis.
  • In September 2020, the FDA has granted MRT5005 both orphan drug and fast track designation for the treatment of cystic fibrosis
Cystic Fibrosis Report Insights
  • Cystic Fibrosis Pipeline Analysis
  • Therapeutic Assessment
  • Unmet Needs
  • Impact of Drugs
Cystic Fibrosis Report Assessment
  • Pipeline Product Profiles
  • Therapeutic Assessment
  • Pipeline Assessment
  • Inactive drugs assessment
  • Unmet Needs
Key Questions

Current Treatment Scenario and Emerging Therapies:
  • How many companies are developing Cystic Fibrosis drugs?
  • How many Cystic Fibrosis drugs are developed by each company?
  • How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Cystic Fibrosis?
  • What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Cystic Fibrosis therapeutics?
  • What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
  • What are the clinical studies going on for Cystic Fibrosis and their status?
  • What are the key designations that have been granted to the emerging drugs?
Key Players
  • AbbVie
  • Allergan PLC
  • Chiesi Farmaceutici SpA
  • F. Hoffmann-La Roche AG
  • Gilead Sciences
  • Novartis AG
  • Pharmaxis Ltd
  • PTC Therapeutics
  • Vertex Pharmaceuticals Incorporated
  • Novoteris, LLC
  • Neupharma Srl
  • AlgiPharma AS
  • Arrowhead Pharmaceuticals
  • Eloxx Pharmaceuticals, Inc.
Key Products
  • Teicoplanin
  • MRT5005
  • Inhaled AR-501
  • ARO-ENaC
  • OligoG DPI
  • ELX-02
  • POL6014
  • CB-280
  • S-1226
  • ELX/TEZ/IVA
  • Ivacaftor


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Note: Product cover images may vary from those shown
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FEATURED COMPANIES

  • AbbVie
  • Allergan PLC
  • Chiesi Farmaceutici SpA
  • Gilead Sciences
  • Novartis AG
  • Pharmaxis Ltd
  • MORE
Introduction

Executive Summary

Cystic Fibrosis: Overview
  • Causes
  • Mechanism of Action
  • Signs and Symptoms
  • Diagnosis
  • Disease Management
Pipeline Therapeutics
  • Comparative Analysis
Therapeutic Assessment
  • Assessment by Product Type
  • Assessment by Stage and Product Type
  • Assessment by Route of Administration
  • Assessment by Stage and Route of Administration
  • Assessment by Molecule Type
  • Assessment by Stage and Molecule Type
Cystic Fibrosis - Analytical Perspective

In-depth Commercial Assessment
  • Cystic Fibrosis companies’ collaborations, Licensing, Acquisition -Deal Value Trends
Cystic Fibrosis Collaboration Deals
  • Company-Company Collaborations (Licensing / Partnering) Analysis
  • Company-University Collaborations (Licensing / Partnering) Analysis
Late Stage Products (Phase III)
  • Comparative Analysis
ELX/TEZ/IVA: Vertex Pharmaceuticals Incorporated
  • Product Description
  • Research and Development
  • Product Development Activities
Mid Stage Products (Phase II)
  • Comparative Analysis
ELX-02: Eloxx Pharmaceuticals, Inc.
  • Product Description
  • Research and Development
  • Product Development Activities
Mid Stage Products (Phase I/II)

MRT5005: Translate Bio
  • Product Description
  • Research and Development
  • Product Development Activities
Pre-clinical and Discovery Stage Products
  • Comparative Analysis
  • Product Description
  • Research and Development
  • Product Development Activities
Inactive Products
  • Comparative Analysis
Cystic Fibrosis Key Companies

Cystic Fibrosis Key Products

Cystic Fibrosis- Unmet Needs

Cystic Fibrosis- Market Drivers and Barriers

Cystic Fibrosis- Future Perspectives and Conclusion

Cystic Fibrosis Analyst Views

Cystic Fibrosis Key Companies

Appendix

List of Tables
Table 1 Total Products for Cystic Fibrosis
Table 2 Late Stage Products
Table 3 Mid Stage Products
Table 4 Early Stage Products
Table 5 Pre-clinical & Discovery Stage Products
Table 6 Assessment by Product Type
Table 7 Assessment by Stage and Product Type
Table 8 Assessment by Route of Administration
Table 9 Assessment by Stage and Route of Administration
Table 10 Assessment by Molecule Type
Table 11 Assessment by Stage and Molecule Type
Table 12 Inactive Products

List of Figures
Figure 1 Total Products for Cystic Fibrosis
Figure 2 Late Stage Products
Figure 3 Mid Stage Products
Figure 4 Early Stage Products
Figure 5 Preclinical and Discovery Stage Products
Figure 6 Assessment by Product Type
Figure 7 Assessment by Stage and Product Type
Figure 8 Assessment by Route of Administration
Figure 9 Assessment by Stage and Route of Administration
Figure 10 Assessment by Molecule Type
Figure 11 Assessment by Stage and Molecule Type
Figure 12 Inactive Products
Note: Product cover images may vary from those shown
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  • AbbVie
  • Allergan PLC
  • Chiesi Farmaceutici SpA
  • F. Hoffmann-La Roche AG
  • Gilead Sciences
  • Novartis AG
  • Pharmaxis Ltd
  • PTC Therapeutics
  • Vertex Pharmaceuticals Incorporated
  • Novoteris, LLC
  • Neupharma Srl
  • AlgiPharma AS
  • Arrowhead
Note: Product cover images may vary from those shown
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