Use of SGLT2 Inhibitors to Treat Primary Biliary Cholangitis

Novel use patent covering SGLT2 inhibitor compounds for the treatment of primary biliary cholangitis (PBC), a progressive autoimmune cholestatic liver disease.

About this asset

PBC remains an area of significant unmet medical need with a limited number of approved therapeutic options. This asset extends the Islet Sciences PSC / PBC franchise and offers a complementary commercial pathway alongside the company's other hepatobiliary IP.

Key characteristics

Autoimmune Cholestatic Disease
Targets a recognized rare hepatobiliary indication.
Validated Mechanism
Builds on the established SGLT2 inhibitor class.
Franchise Synergy
Strategically aligned with the PSC asset for portfolio licensees.
Global Optionality
Suitable for worldwide, regional, or co-development structures.

Potential applications

Primary biliary cholangitisRare-disease pipelinesSGLT2 franchise extension
Autoimmune Cholangiopathy

When the immune system targets its own bile ducts.

In PBC, autoreactive immune populations selectively destroy the small intrahepatic bile ducts — driving chronic cholestasis, fibrosis, and progressive liver injury.

AMA+
≈95% of patients
9:1
Female predominance
Small
Intrahepatic ducts targeted
Chronic
Lifelong autoimmune process
AUTOANTIBODY-MEDIATED DESTRUCTION
Cellular Pathology

Small bile duct destruction — step by step.

Unlike PSC, PBC selectively targets the small intrahepatic ducts. The process is autoimmune, antibody-mediated, and slowly progressive.

PHASE 1
Intact Cholangiocyte
Healthy small bile duct epithelium maintains bile flow.
PHASE 2
AMA Recognition
Anti-mitochondrial antibodies recognize PDC-E2 antigen on cholangiocytes.
PHASE 3
T-Cell Infiltration
Autoreactive CD4+/CD8+ T-cells cluster around small ducts.
PHASE 4
Ductopenia
Progressive small bile duct loss — the histologic hallmark of PBC.
PHASE 5
Cholestasis & Fibrosis
Loss of ducts impairs bile flow; portal-based fibrosis advances.
Autoimmune Signature

The serologic fingerprint of PBC.

PBC's diagnostic certainty comes from a distinct combination of autoantibodies and cholestatic enzymes.

AMA
95%
Anti-Mitochondrial Antibodies
Hallmark — present in ~95% of PBC
ANA
50%
Antinuclear Antibodies
PBC-specific patterns (sp100, gp210)
IgM
75%
Elevated Immunoglobulin M
Characteristic serologic finding
ALP
88%
Alkaline Phosphatase
Cholestatic biomarker
GGT
80%
Gamma-Glutamyl Transferase
Confirms biliary origin
TBIL
45%
Total Bilirubin
Late-stage prognostic
Clinical Burden

A Chronic Liver Disease With Long-Term Consequences.

PBC is a chronic autoimmune cholestatic disease with sustained patient burden and ongoing therapeutic gaps in incomplete responders.

Chronic autoimmune liver disease
Immune-mediated destruction of small intrahepatic bile ducts.
Progressive bile duct injury
Damage accumulates and drives long-term disease progression.
Fatigue & pruritus burden
Symptomatic burden meaningfully affects quality of life.
Risk of fibrosis & cirrhosis
Untreated or undertreated disease can progress to cirrhosis.
Long-term disease management
Patients typically require chronic pharmacologic therapy.
Unmet need in non-responders
Incomplete responders to first-line therapy remain underserved.
PSC vs PBC — Side-by-Side Compare

Two cholestatic diseases. Two distinct biologies.

Toggle below to compare disease mechanism or clinical progression across PSC and PBC.

PBC
This page
LiverCommon bile duct
Autoimmune cholestatic disease of small intrahepatic ducts.
  • Autoimmune-mediated injury (AMA antibodies in most patients)
  • Targets small intrahepatic bile ducts
  • Chronic lymphocytic cholangitis with periductal inflammation
  • Female predominance reported in published literature
  • Slowly progressive cholestasis with pruritus and fatigue burden
PSC
LiverCommon bile duct
Fibro-inflammatory disease of large and small bile ducts.
  • Chronic fibro-inflammatory injury of biliary epithelium
  • Affects intra- and extrahepatic bile ducts
  • Progressive multifocal strictures and segmental dilations
  • Strong association with inflammatory bowel disease
  • Elevated cholangiocarcinoma and transplant risk
Patent Concept

Potential SGLT2-Based Therapeutic Application in PBC.

This patent describes compositions of SGLT2 inhibitors and their use in treating Primary Biliary Cholangitis.

The intellectual property includes SGLT2 inhibitor compositions, including oral dosage forms, directed toward preventing, partially ameliorating, or fully ameliorating symptoms associated with PBC.

Important

Therapeutic-use intellectual property. Not presented as clinically proven or as an approved therapy.

  1. SGLT2 Inhibitor Compositions
  2. Oral Dosage Forms
  3. Potential PBC Application
  4. Therapeutic-Use IP Position
Patent Science Timeline

From biology to partnership opportunity.

  1. 01
    Disease Biology
  2. 02
    Scientific Hypothesis
  3. 03
    Patent Filing
  4. 04
    Patent Publication
  5. 05
    Development Opportunity
  6. 06
    Strategic Partnership
Strategic Value

Why PBC May Represent A Valuable Specialty Pharma Opportunity.

Chronic Rare Liver Disease

Recognized rare hepatobiliary indication with persistent patient burden.

Specialty Pharma Market

Active commercial interest from hepatology-focused players.

Unmet Need in Non-Responders

Significant gap remains for patients with incomplete response to first-line therapy.

Potential Repurposing Strategy

Leverages an established and well-characterized drug class.

Oral Therapy Opportunity

Aligned with chronic disease patient preference for oral regimens.

Licensing Potential

Suitable for SGLT2 originators and hepatology specialty pharma.

Explore Development or Licensing Opportunities.

Islet Sciences is evaluating strategic partnerships, licensing discussions, and development opportunities for its therapeutic-use intellectual property portfolio.