“Primary Biliary Cholangitis (PBC) – Pipeline Insight, 2020,” report provides comprehensive insights about 10+ companies and 10+ 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 ?mol/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 (eg, pruritus, steatorrhea) or cirrhosis (eg, 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 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 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 is a dual agonist of the PPAR? and PPAR?. 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, paving the way for advancement into phase 3 clinical trial in 2020. It has recently 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).
Saroglitazar Magnesium: Zydus Discovery
Saroglitazar Magnesium is an investigational molecule in USA, undergoing clinical evaluation for treatment of liver diseases like NAFLD with polycystic ovary syndrome (PCOS), Nonalcoholic Steatohepatitis (NASH) and Primary Biliary Cholangitis (PBC). Nonclinical studies have demonstrated that Saroglitazar, because of its unique mechanism of engaging PPAR alpha and PPAR gamma, favorably affects all components of NASH including steatosis, ballooning, inflammation and fibrosis.
TQA3526: Chia Tai Tianqing Pharmaceutical Group Co., Ltd.
TQA3526 is a modified bile acid and FXR agonist. FXR is a key regulator of bile acid synthesis and transport. Bile acids are used by the body to help with digestion. It is hypothesized that regular treatment with TQA3526 will improve liver function in persons with Primary Biliary Cirrhosis (PBC).
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. 10+ 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
The report covers around 10+ 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
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
- Intravenous
- Molecule Type
Products have been categorized under various Molecule types such as
- Small molecules
- Proteins and Peptides
- 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 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.
Report Highlights
August 2020: Chemomab Ltd., a clinical-stage biotech company focusing on discovery and development of innovative therapeutics for fibrosis-related diseases, announced that the European Commission (EC) has granted Orphan Drug Designation (ODD) to its lead drug candidate CM-101 for the treatment of patients with Primary Sclerosing Cholangitis (PSC), based on a positive opinion from the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA).
August 2020: CymaBay Therapeutics, Inc. announced positive topline results from ENHANCE, a placebo-controlled, randomized, Phase 3 study evaluating the safety and efficacy of seladelpar for the treatment of primary biliary cholangitis (PBC). Seladelpar is a potent and selective peroxisome proliferator-activated receptor delta (PPAR?) agonist that has demonstrated anti-cholestatic and anti-inflammatory effects in clinical studies for PBC.
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
- Merck
- Chia Tai Tianqing Pharmaceutical Group Co., Ltd.
- Cara Therapeutics, Inc.
- Chemomab Ltd.
- CymaBay Therapeutics
Key Products
- Elafibranor
- Saroglitazar
- Emtricitabine
- TQA3526
- CR845
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