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Accelerating Primary Biliary Cholangitis Drug Development

Primary biliary cholangitis (PBC) presents significant therapeutic challenges due to its complex autoimmune pathology and limited treatment options. Protheragen is a specialized partner in preclinical drug development for PBC, offering a comprehensive suite of services that span target validation, lead optimization, and IND-enabling studies. Leveraging deep scientific expertise and advanced technology platforms, Protheragen delivers robust preclinical solutions tailored to the unique demands of PBC therapeutics. The company’s integrated approach ensures rigorous data quality, translational relevance, and strict adherence to regulatory standards, positioning clients for successful clinical advancement. Protheragen’s commitment to scientific excellence and innovation accelerates the discovery and development of novel therapies, driving meaningful progress toward improved outcomes for patients with primary biliary cholangitis.

What is Primary Biliary CholangitisTargets for Primary Biliary CholangitisDrug Discovery and Development ServicesWhy Choose Us

What is Primary Biliary Cholangitis

Primary Biliary Cholangitis (PBC) is a chronic, progressive autoimmune liver disease characterized by the immune-mediated destruction of small intrahepatic bile ducts. The exact cause is multifactorial, involving genetic susceptibility—evidenced by familial clustering and HLA associations—and environmental triggers such as infections or chemical exposures. This immune attack leads to chronic nonsuppurative cholangitis, progressive loss of bile ducts, and impaired bile flow (cholestasis), which over time causes hepatic fibrosis and can progress to cirrhosis. PBC predominantly affects middle-aged women and is associated with the presence of highly specific antimitochondrial antibodies (AMAs). Clinically, PBC often presents with nonspecific symptoms such as fatigue and pruritus in early stages, but can progress to jaundice, xanthomas, and complications of liver failure in advanced disease. Diagnosis is based on elevated cholestatic liver enzymes (notably alkaline phosphatase), the presence of AMAs, and, when necessary, liver biopsy findings. Imaging is used to exclude other causes of cholestasis. First-line treatment is ursodeoxycholic acid, which improves bile flow and delays disease progression. For patients who do not respond adequately, obeticholic acid or investigational agents like elafibranor and seladelpar lysine may be considered. Early diagnosis and treatment are crucial to improving outcomes and quality of life.

Launched Drugs

Structure Generic Name CAS Registry Number Molecular Formula Molecular Weight
img-923978-27-2-elafibranor-rec-inn-usan elafibranor (Rec INN; USAN) 923978-27-2 C22 H24 O4 S 384.489
img-851528-79-5-free-base928821-40-3-seladelpar-lysine-rec-innm seladelpar lysine (Rec INNM) 851528-79-5 (free base); 928821-40-3 C21 H23 F3 O5 S . C6 H14 N2 O2 . 2 H2 O 626.683
img-459789-99-2-obeticholic-acid-prop-inn-usan obeticholic acid (Prop INN; USAN) 459789-99-2 C26 H44 O4 420.625
img-128-13-2-ursodeoxycholateursodeoxycholic-acid-prop-inn-janu ursodeoxycholate; ursodeoxycholic acid (Prop INN; JAN); ursodiol (USAN; BAN) 128-13-2 C24 H40 O4 392.572

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Targets for Primary Biliary Cholangitis

Targets in Clinical or Later Phases of Development

Target Name Gene Symbol
NFE2 like bZIP transcription factor 2 NFE2L2
nuclear receptor subfamily 1 group H member 4 NR1H4
peroxisome proliferator activated receptor delta PPARD
peroxisome proliferator activated receptor alpha PPARA
insulin INS
apolipoprotein A1 APOA1
fibroblast growth factor 2 FGF2
hydroxysteroid 11-beta dehydrogenase 1 HSD11B1
nuclear receptor subfamily 3 group C member 1 NR3C1
Nuclear factor kappa-light-chain-enhancer of activated B cells

Primary biliary cholangitis (PBC) involves a complex interplay of immune-mediated injury, cholestatic dysfunction, and metabolic disturbances, with several molecular targets playing central roles in disease pathogenesis. CXCL10, a chemokine upregulated by interferon-gamma and NF-κB, orchestrates immune cell recruitment to the bile ducts, amplifying local inflammation and driving autoimmune destruction of cholangiocytes. In the regulation of bile acid synthesis and transport, CYP7A1 acts as the rate-limiting enzyme, while NR1H4 (FXR) serves as a master regulator, repressing CYP7A1 and promoting bile acid efflux. Disruption of these pathways leads to toxic bile acid accumulation and hepatocellular injury. Additionally, APOA1 and PPARA are key mediators of lipid metabolism and anti-inflammatory responses, with APOA1 facilitating cholesterol efflux and PPARA modulating genes involved in fatty acid oxidation and inflammation. Together, these targets reflect the multifaceted pathophysiology of PBC.

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Drug Discovery and Development Services

In Vitro Efficacy Testing ServicesIn Vivo Model DevelopmentPK/PD Study ServicesIn Vivo Toxicity Assessment ServicesBiomarker Analysis Services
In Vitro Efficacy Testing Services

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Our In Vitro Efficacy Testing Service accelerates Primary Biliary Cholangitis (PBC) drug discovery through robust, disease-relevant screening platforms. We offer comprehensive biochemical, cell-based, and reporter assays to assess compound efficacy, mechanism of action, and pathway modulation. Key targets include FXR, PPARs, immune signaling proteins, and cholangiocyte pathways. Methodologies such as FRET, HTRF, luciferase, ELISA, and arrestin recruitment enable precise evaluation of ligand binding, receptor activation, and gene transcription. Quantitative pharmacological parameters like EC-50, IC-50, and Kd guide lead optimization, ensuring informed candidate selection and supporting efficient preclinical development for PBC therapies.

Apolipoprotein A1 C-X-C Motif Chemokine Ligand 10
Cytochrome P450 Family 7 Subfamily A Member 1 Nuclear Receptor Subfamily 1 Group H Member 4
Nuclear Receptor Subfamily 3 Group C Member 1 Peroxisome Proliferator Activated Receptor Alpha
Peroxisome Proliferator Activated Receptor Delta Peroxisome Proliferator Activated Receptor Gamma
Phosphodiesterase 4B Sphingosine-1-Phosphate Receptor 4
Sphingosine-1-Phosphate Receptor 5 Superoxide Dismutase 1

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Why Choose Us

Choosing Protheragen as your partner in developing new therapeutics for Primary biliary cholangitis means entrusting your project to a team with specialized expertise in this challenging field. At Protheragen, we bring together dedicated professionals with deep experience in Primary biliary cholangitis research and drug development, supported by state-of-the-art technology platforms that enable innovative solutions at every stage of the preclinical process. Our proven track record in delivering reliable and effective preclinical drug development services demonstrates our commitment to excellence and client satisfaction. Protheragen upholds the highest quality standards and maintains strict regulatory compliance, ensuring that every project meets both scientific and industry requirements. We are deeply committed to advancing the treatment landscape for Primary biliary cholangitis and work tirelessly to support the discovery and development of novel therapeutics that can make a meaningful difference for patients. With Protheragen, you benefit from a trusted partner dedicated to professionalism, reliability, and the shared goal of improving patient outcomes in Primary biliary cholangitis.

FAQs for Our Services

Q: What are the key preclinical research challenges specific to developing new drugs for Primary biliary cholangitis (PBC)?

A: Preclinical research for PBC is challenged by the limited availability of robust animal models that accurately recapitulate the human disease pathology, particularly the autoimmune-mediated destruction of intrahepatic bile ducts. Additionally, the heterogeneity in disease progression and the need to evaluate both anti-fibrotic and immunomodulatory effects require a comprehensive approach, including the use of humanized models and advanced in vitro systems. Our company addresses these challenges by leveraging state-of-the-art animal models, organoid cultures, and customized assay development to ensure translational relevance and predictive validity.

Q: What are the main regulatory considerations for preclinical drug development targeting Primary biliary cholangitis?

A: Regulatory agencies such as the FDA and EMA require a clear demonstration of both safety and efficacy in preclinical studies before advancing to clinical trials. For PBC, it is essential to provide data on pharmacodynamics, pharmacokinetics, and toxicology, with a focus on liver-specific endpoints and potential off-target effects. Our team is experienced in designing preclinical programs that align with regulatory expectations, including the use of validated biomarkers and endpoints relevant to PBC, and we offer regulatory consulting to facilitate successful IND/CTA submissions.

Q: What technical aspects are critical in preclinical research for Primary biliary cholangitis drug candidates?

A: Technical success in PBC preclinical research depends on the selection and validation of appropriate models, the use of sensitive and specific biomarkers for disease progression and drug response, and the integration of advanced imaging and molecular techniques. Our services include high-throughput screening, transcriptomic and proteomic profiling, and the development of custom assays to monitor bile acid metabolism, immune cell infiltration, and fibrosis in preclinical models.

Q: What are the typical timeline and cost considerations for preclinical development of drugs for Primary biliary cholangitis?

A: Preclinical development for PBC typically spans 18-30 months, depending on the complexity of the program and the number of studies required. Costs can vary significantly, ranging from $1 million to $5 million, influenced by the selection of models, the extent of safety and efficacy testing, and regulatory requirements. Our company offers flexible project management and budgeting options, providing clients with transparent timelines and milestone-based pricing to optimize resource allocation and accelerate development.

Q: What are the key success factors in preclinical drug development for Primary biliary cholangitis?

A: Success in preclinical PBC drug development relies on early identification of translational biomarkers, the use of predictive disease models, and a strong understanding of the disease mechanism. Collaboration with experts in hepatology, immunology, and regulatory affairs is also crucial. Our integrated approach combines scientific expertise, cutting-edge technologies, and regulatory insight to maximize the likelihood of advancing promising candidates to clinical development.

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