Primary Biliary Cholangitis - Pipeline Insight, 2023

Primary Biliary Cholangitis - Pipeline Insight, 2023

DelveInsight’s, “Primary Biliary Cholangitis - Pipeline Insight, 2023,” report provides comprehensive insights about 18+ companies and 20+ pipeline drugs in Primary Biliary 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 Biliary Cholangitis Understanding

Primary Biliary Cholangitis: Overview

Primary biliary cholangitis (PBC; formerly known as primary biliary cirrhosis) is an autoimmune liver disorder characterized by the progressive destruction of intrahepatic bile ducts, leading to cholestasis, cirrhosis, and liver failure. PBC leads to inflammation and scarring of the small bile ducts (the ‘plumbing’ system of the liver which transports bile, the substance that helps digest fat). When PBC is very severe, it can lead to yellow discoloration of the skin (jaundice), which occurs when bilirubin levels rise above 2 to 3mg/dL or 34 to 51 µmol/L). PBC is divided into four stages from stage 1 (early disease, without significant scarring of the liver) to stage 4 (cirrhosis). Although the exact cause of PBC is unknown, it is thought that it is likely due to a combination of factors such as autoimmune (when a person’s own immune system attacks their body), genetic, and environmental factors.

Symptoms

The most common symptoms of PBC are:

Fatigue

Pruritus (itchiness)

Jaundice

Patients usually are asymptomatic at presentation but may experience fatigue or have symptoms of cholestasis (e.g. pruritus, steatorrhea) or cirrhosis (e.g. portal hypertension, ascites).

Diagnosis

Laboratory tests reveal cholestasis, increased IgM, and, characteristically, antimitochondrial antibodies in the serum. Liver biopsy may be necessary for diagnosis and staging. The diagnosis of PBC requires the presence of:

High alkaline phosphatase (ALP, a liver blood test) along with

Positive anti-mitochondrial antibody (+AMA)

If AMA testing is negative, then a patient would need a liver biopsy to confirm the diagnosis of PBC, since a number of diseases can cause a high ALP.

Treatment

A medication called ursodeoxycholic acid (also called UDCA, URSO) mimics a naturally occurring bile acid. This medication can improve liver function and delay the development of fibrosis (scar tissue) in the liver, which in turn can delay or eliminate the potential for PBC to progress towards liver failure and/or liver transplantation. Vitamins A, D, E and K can be prescribed, usually when severe cholestasis and jaundice is present. Complications of liver deterioration can often be controlled. As an example, a salt-restricted diet and medication (usually water pills) can be prescribed following accumulation of fluid in the abdomen.

""Primary Biliary Cholangitis - Pipeline Insight, 2023"" report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Primary Biliary Cholangitis pipeline landscape is provided which includes the disease overview and Primary Biliary Cholangitis treatment guidelines. The assessment part of the report embraces, in depth Primary Biliary Cholangitis commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Primary Biliary Cholangitis collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

Report Highlights

The companies and academics are working to assess challenges and seek opportunities that could influence Primary Biliary Cholangitis R&D. The therapies under development are focused on novel approaches to treat/improve Primary Biliary Cholangitis.

Primary Biliary Cholangitis Emerging Drugs Chapters

This segment of the Primary Biliary Cholangitis report encloses its detailed analysis of various drugs in different stages of clinical development, including phase III, 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 Biliary Cholangitis Emerging Drugs

Elafibranor: Genfit

Elafibranor is a dual agonist of the PPARα and PPARδ. Meaning the drug candidate acts simultaneously on the two nuclear receptors, which both play an important role in numerous processes involved in the development of NASH and its co-morbidities. It has shown highly significant results in its Phase 2 Study in patients with primary biliary cholangitis (PBC), while maintaining a favorable tolerability profile and lack of demonstrated safety concerns. Currently, it is in Phase III stage of clinical trial evaluation to treat Primary Biliary Cholangitis. It has been granted Breakthrough Therapy Designation by the FDA for the treatment of Primary Biliary Cholangitis (PBC) in adults with inadequate response to ursodeoxycholic acid (UDCA), as well as Orphan Drug Designation by the FDA and the EMA (European Medicines Agency).

ASC42: Gannex Pharma

ASC42 is a novel non-steroidal, selective, potent FXR agonist. It has shown positive results in its Phase I Study in patients with primary biliary cholangitis with no pruritus observed and LDL-C remained within normal range at the human therapeutic dose of 15 mg. The drug is in Phase II stage of its clinical studies for Primary Biliary Cholangitis.

OP-724: Ohara Pharmaceutical

OP-724 is a synthetic small molecule which is an inhibitor of cyclic AMP response element-binding protein (CREB)-binding protein (CBP)/β-catenin. Ohara Pharmaceutical obtained development rights for OP-724 from PRISM BioLab in 2018 . The drug is in Phase I stage of its clinical studies for Primary Biliary Cholangitis.

Further product details are provided in the report……..

Primary Biliary Cholangitis: Therapeutic Assessment

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

Major Players in Primary Biliary Cholangitis

There are approx. 18+ key companies which are developing the therapies for Primary Biliary Cholangitis. The companies which have their Primary Biliary Cholangitis drug candidates in the most advanced stage, i.e. phase III include, Genfit, Zydus Discovery and others.

Phases

DelveInsight’s 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 Biliary 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

Intravenous

Subcutaneous

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 Biliary Cholangitis: Pipeline Development Activities

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

Primary Biliary Cholangitis Report Insights

Primary Biliary Cholangitis Pipeline Analysis

Therapeutic Assessment

Unmet Needs

Impact of Drugs

Primary Biliary 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 Biliary Cholangitis drugs?

How many Primary Biliary 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 Biliary Cholangitis?

What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the Primary Biliary 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 Biliary Cholangitis and their status?

What are the key designations that have been granted to the emerging drugs?

Key Players

Genfit

Zydus Discovery

Ohara Pharmaceutical

Chia Tai Tianqing Pharmaceutical Group Co., Ltd.

Novartis

CymaBay Therapeutics

Key Products

Elafibranor

Saroglitazar

ASC42

LJN452

OP-724

Saroglitazar