Focal Segmental Glomerulosclerosis (FSGS) – Pipeline Insight, 2020

September 2020 | 61 pages | ID: F7922959B88FEN
DelveInsight

Abstracts

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DelveInsight’s, “Focal Segmental Glomerulosclerosis (FSGS) – Pipeline Insight, 2020,” report provides comprehensive insights about 15+ companies and 15+ pipeline drugs in Focal Segmental Glomerulosclerosis 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

Focal Segmental Glomerulosclerosis Understanding

Focal Segmental Glomerulosclerosis: Overview

Focal Segmental glomerulosclerosis is a type of glomerular disease and describes scarring (sclerosis) in your kidney. The scarring of FSGS only takes place in small sections of each glomerulus (filter), and only a limited number of glomeruli are damaged at first. Focal Segmental Glomerulosclerosis affects both children and adults. Males are affected slightly more often than females. FSGS is not caused by a single disease. It can have many different causes. The scarring may happen because of an infection, or drug, or a disease that affects the entire body, like diabetes, HIV infection, sickle cell disease or lupus. FSGS can also be caused by another glomerular disease that you had before you got FSGS. FSGS has different types based on the cause.

Below are the types of FSGS:

• Primary FSGS: This type of FSGS means that the disease happened on its own without a known or obvious cause.
• Secondary FSGS: This type is caused by another disease or a drug. Examples include: viruses such as HIV or drugs such as anabolic steroids that some people use to speed up their muscle growth

Symptoms

Early stages may not cause any symptoms. Signs and symptoms of FSGs include:

• Swelling in body parts like your legs, ankles and around your eyes (called edema)
• Weight gain due to extra fluid building in your body
• Foamy urine caused by high protein levels in the urine (called proteinuria)
• High fat levels in the blood (high cholesterol)
• Low levels of protein in the blood

Diagnosis

A blood test, urine test, and a kidney biopsy will determine if one have FSGS.

• Urine test: A urine test will help find protein and blood in urine.
• Blood test: A blood test will help find levels of protein, cholesterol, and wastes in blood.
• Glomerular filtration rate (GFR): A blood test will be done to know how well kidneys are filtering the wastes from body.
• Kidney biopsy: In this test, a tiny piece of kidney is removed with a special needle, and looked at under a microscope.
• Genetic testing: A genetic test may be done to see if the patient was born with genes that caused kidney disease. This information may help doctor decide what type of treatment is best for the patient.

Treatment

The type of treatment depends on the cause. Everyone is different and the doctor will make a treatment plan that is right for the type of FSGS. Usually, treatments for FSGS include:

• Corticosteroids
• Immunosuppressive drugs
• Plasmapheresis
• ACE inhibitors and ARBs
• Diuretics
• Diet change

Focal Segmental Glomerulosclerosis Emerging Drugs Chapters

This segment of the Focal Segmental Glomerulosclerosis 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.

Focal Segmental Glomerulosclerosis Emerging Drugs

• Sparsentan: Retrophin

Sparsentan is a novel small-molecule candidate in phase III development for the treatment of focal segmental glomerulosclerosis (FSGS). Retrophin is examining the ability of sparsentan to slow the decline of kidney function in patients with FSGS and IgAN. It is a single molecule designed to be a dual endothelin receptor type A (ETA) and angiotensin II receptor type 1 (AT1) antagonist. The investigational approach is to attempt to reduce proteinuria, a characteristic marker of kidney injury in both FSGS and IgAN, with sparsentan. Lowering proteinuria levels is associated with better outcomes.

• CXA-10: Complexa

CXA-10 is an oral nitrated fatty acid compound that acts through a constellation of actions including:

• Upregulation of Nrf2 pathways, a key pro-reparative and metabolic pathway
• Inhibition of NF-Kappa B and TLR4, key drivers of inflammatory molecules and pathways
• Inhibition of Xanthine Oxidoreductase, a key intracellular producer of Reactive Oxygen Species add that result in cellular oxidative stress
• Increasing the expression of heat shock proteins, which act as chaperones during cellular stress to restore cellular homeostatic mechanisms
• GFB-887: Goldfinch Bio

GFB-887 is a TRPC5 ion channel inhibitor in development for the treatment of FSGS. TRPC5 is a calcium-permeable ion channel that has been implicated in the pathogenesis of FSGS by triggering a Rac1-TRPC5 disease pathway. TRPC5 represents a target for therapeutic intervention to halt progression of FSGS to kidney failure. It is currently in phase II stage of development.

• Research programme: focal segmental glomerulosclerosis therapeutics - Delta 4

Delta 4 is working on its lead program which aims at developing a therapy for Focal Segmental Glomerulosclerosis (FSGS). Delta 4 concluded pre-clinical development with promising results. The Company initiated patent filing and is currently preparing for clinical testing of selected drug candidates.

Further product details are provided in the report

Focal Segmental Glomerulosclerosis: Therapeutic Assessment

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

• Major Players in Focal Segmental Glomerulosclerosis

There are approx. 15+ key companies which are developing the therapies for Focal Segmental Glomerulosclerosis. The companies which have their Focal Segmental Glomerulosclerosis drug candidates in the most advanced stage, i.e. phase III include Retrophin and others

• Phases

DelveInsight’s report covers around 15+ products under different phases of clinical development like

• Late-stage products (Phase II and Phase II/III)
• Mid-stage products (Phase II and Phase II/III)
• 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

Focal Segmental Glomerulosclerosis pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

• Infusion
• Intravenous
• Intradermal
• Intramuscular
• Intranasal
• Oral
• Parenteral
• Subcutaneous
• Topical.
• Molecule Type

Products have been categorized under various Molecule types such as

• Gene therapies
• Small molecule
• Vaccines
• Polymers
• Peptides
• Monoclonal antibodies
• Product Type

Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

Focal Segmental Glomerulosclerosis: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Focal Segmental Glomerulosclerosis 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 Focal Segmental Glomerulosclerosis drugs.

Report Highlights

• The companies and academics are working to assess challenges and seek opportunities that could influence Focal Segmental Glomerulosclerosis R&D. The therapies under development are focused on novel approaches to treat/improve Focal Segmental Glomerulosclerosis.
• In November 2015, Retrophin announced that the European Commission (EC) has granted sparsentan orphan drug designation for the treatment of focal segmental glomerulosclerosis (FSGS).
• In January 2015, Retrophin announced that sparsentan received orphan drug designation from the US Food and Drug Administration (FDA) for the treatment of FSGS.
• In October 2018, Variant Pharmaceuticals announced that it has filed an application with the FDA for orphan drug designation for VAR 200 for Focal Segmental Glomerulosclerosis.

Focal Segmental Glomerulosclerosis Report Insights

• Focal Segmental Glomerulosclerosis Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Focal Segmental Glomerulosclerosis 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 Focal Segmental Glomerulosclerosis drugs?
• How many Focal Segmental Glomerulosclerosis drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Focal Segmental Glomerulosclerosis?
• What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the Focal Segmental Glomerulosclerosis 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 Focal Segmental Glomerulosclerosis and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• Dimerix Bioscience Pty Ltd
• Astellas Pharma
• Complexa
• Aurinia Pharmaceuticals
• FibroGen
• Retrophin
• Genzyme, a Sanofi Company
• Vertex Pharmaceuticals
• ChemoCentryx
• Goldfinch
• Delta 4

Key Products

• Propagermanium
• Bleselumab
• CXA-10
• Voclosporin
• FG-3019
• VX-147
• Fresolimumab
• CCX140-B
• RE-021
• GFB-887
• Research programme: focal segmental glomerulosclerosis therapeutics

Contents

Introduction
Executive Summary
Focal Segmental Glomerulosclerosis: 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
Focal Segmental Glomerulosclerosis – DelveInsight’s Analytical Perspective
In-depth Commercial Assessment
  Focal Segmental Glomerulosclerosis companies’ collaborations, Licensing, Acquisition -Deal Value Trends
Focal Segmental Glomerulosclerosis Collaboration Deals
  Company-Company Collaborations (Licensing / Partnering) Analysis
  Company-University Collaborations (Licensing / Partnering) Analysis
Late Stage Products (Phase III)
  Comparative Analysis
Sparsentan: Retrophin
  Product Description
  Research and Development
  Product Development Activities
Mid Stage Products (Phase II)
  Comparative Analysis
CXA-10: Complexa
  Product Description
  Research and Development
  Product Development Activities
GFB-887: Goldfinch Bio
  Product Description
  Research and Development
  Product Development Activities
Drug profiles in the detailed report…..
Early Stage Products (Phase I)
  Comparative Analysis
Drug Name: Company Name
  Product Description
  Research and Development
  Product Development Activities
Drug profiles in the detailed report…..
Pre-clinical and Discovery Stage Products
  Comparative Analysis
Research programme: focal segmental glomerulosclerosis therapeutics - Delta
  Product Description
  Research and Development
  Product Development Activities
Inactive Products
  Comparative Analysis
Focal Segmental Glomerulosclerosis Key Companies
Focal Segmental Glomerulosclerosis Key Products
Focal Segmental Glomerulosclerosis- Unmet Needs
Focal Segmental Glomerulosclerosis- Market Drivers and Barriers
Focal Segmental Glomerulosclerosis- Future Perspectives and Conclusion
Focal Segmental Glomerulosclerosis Analyst Views
Focal Segmental Glomerulosclerosis Key Companies
Appendix

List Of Tables

LIST OF TABLES

Table 1 Total Products for Focal Segmental Glomerulosclerosis
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

LIST OF FIGURES

Figure 1 Total Products for Focal Segmental Glomerulosclerosis
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