This “Primary Sclerosing Cholangitis- Pipeline Insight, 2020” report provides comprehensive insights about 20+ companies and 20+ pipeline drugs in Primary Sclerosing Cholangitis 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
Primary Sclerosing Cholangitis Understanding

Primary Sclerosing Cholangitis: Overview

Primary sclerosing cholangitis (PSC) is characterized by inflammation in the bile ducts (cholangitis) that leads to scarring (sclerosis), narrowing of the ducts, and a buildup of bile in the liver. Early signs and symptoms include extreme tiredness, abdominal pain, and itchiness. As the condition worsens it may cause jaundice, an enlarged spleen, and eventually liver cirrhosis and failure. Other complications may include weight loss, vitamin deficiency, and osteoporosis. Many people with PSC develop other autoimmune conditions such as inflammatory bowel disease, type 1 diabetes, celiac disease, or thyroid disease. PSC is also a risk factor for cancer of the bile ducts (cholangiocarcinoma).

Symptoms

Symptoms of PSC include:
Itchy skin.
Extreme tiredness (fatigue)
Belly pain.
Yellowing of the skin and eyes, called jaundice.
Chills and fever from infection of bile ducts
Diagnosis

A diagnosis of primary sclerosing cholangitis is made based upon a thorough clinical evaluation, a detailed patient history, identification of characteristic findings, and a variety of specialized tests.

Treatment

There is no specific, universal treatment for individuals with PSC. Treatment is directed toward the specific symptoms that are apparent in each individual and at slowing the progression of the disorder. Endoscopic surgery to remove blockages and enlarge narrowed bile ducts may be of benefit to help prevent liver deterioration in certain cases. Lost vitamins should be replaced when required to prevent complications related to these deficiencies. Antibiotics may be useful in controlling inflammation or infection. Individuals with PSC are encouraged to follow a normal healthy diet and to avoid alcohol or only have alcohol in small amounts.

Primary Sclerosing Cholangitis Emerging Drugs Chapters

This segment of the Primary Sclerosing Cholangitis 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.

Primary Sclerosing Cholangitis Emerging Drugs

Cenicriviroc: Tobira Therapeutics

Cenicriviroc (Tobira Therapeutics), or CVC, is an oral, potent immunomodulator that blocks chemokine receptors that can cause liver damage and lead to liver disease.

IMU-838: Immunic

IMU-838 is a small molecule investigational drug (vidofludimus calcium) under development as an oral tablet formulation for the treatment PSC. Of By inhibiting dihydroorotate dehydrogenase (DHODH), a key enzyme of pyrimidine de novo biosynthesis, metabolically activated T and B immune cells experience metabolic stress, and the release of Th1 and Th17 key cytokines including IL-17A, IL-17F and IFNg is inhibited, thereby reducing inflammation. In preclinical studies of vidofludimus, the active moiety and free acid form of IMU-838, apoptosis (or programmed cell death) was induced in activated T cells, which Immunic believes may also play a crucial role in the activity of the drug by further dampening the inflammatory response.

Primary Sclerosing Cholangitis: Therapeutic Assessment

This segment of the report provides insights about the different Primary Sclerosing Cholangitis drugs segregated based on following parameters that define the scope of the report.

Major Players in Primary Sclerosing Cholangitis

There are approx. 20+ key companies which are developing the therapies for Primary Sclerosing Cholangitis. The companies which have their Primary Sclerosing Cholangitis drug candidates in the most advanced stage, i.e. phase III include, Gilead Sciences/Phenex Pharmaceuticals.

Phases

This report covers around 20+ products under different phases of clinical development like
Late stage products (Phase III)
Mid-stage products (Phase II)
Early-stage product (Phase I) along with the details of
Pre-clinical and Discovery stage candidates
Discontinued & Inactive candidates
Route of Administration
Primary Sclerosing Cholangitis 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
Products have been categorized under various Molecule types such as
Monoclonal Antibody
Peptides
Polymer
Small molecule
Gene therapy
Product Type
Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

Primary Sclerosing Cholangitis: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Primary Sclerosing Cholangitis 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 Primary Sclerosing Cholangitis drugs.

Report Highlights
The companies and academics are working to assess challenges and seek opportunities that could influence Primary Sclerosing Cholangitis R&D. The therapies under development are focused on novel approaches to treat/improve Primary Sclerosing Cholangitis.
In November 2018, Gilead Sciences announced that treatment with GS-9674, an investigational, selective, nonsteroidal farnesoid X receptor (FXR) agonist, led to significant improvements in liver biochemistry and markers of cholestasis in patients with primary sclerosing cholangitis (PSC). Data were presented at The Liver Meeting 2018 in San Francisco.
Primary Sclerosing Cholangitis Report Insights
Primary Sclerosing Cholangitis Pipeline Analysis
Therapeutic Assessment
Unmet Needs
Impact of Drugs
Primary Sclerosing Cholangitis 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 Primary Sclerosing Cholangitis drugs?
How many Primary Sclerosing Cholangitis drugs are developed by each company?
How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Primary Sclerosing Cholangitis?
What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Primary Sclerosing Cholangitis 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 Primary Sclerosing Cholangitis and their status?
What are the key designations that have been granted to the emerging drugs?
Key Players
Biotie Therapies
Tobira Therapeutics, Inc.
Pliant Therapeutics, Inc.
NGM Biopharmaceuticals, Inc
Mirum Pharmaceuticals, Inc.
Genentech, Inc.
EuroPharma
Intercept Pharmaceuticals
HighTide Biopharma Pty Ltd
Gilead Sciences
Dr. Falk Pharma GmbH
Key Products
BTT1023
Cenicriviroc
PLN-7480
NGM282
LUM001
Erlotinib (Tarceva)
Curcumin
OCA
HTD1801
Cilofexor
Simtuzuma
norUDCA