Our Scientific Approach

Azomedicine is pioneering the development of small molecule modulators targeting mitochondrial ion channels to restore cellular metabolic homeostasis.

Our proprietary screening platform enables the discovery of highly selective compounds that can penetrate cellular and mitochondrial membranes to directly modulate ion channel activity. By restoring proper mitochondrial function, our therapeutics address the root cause of metabolic dysfunction in multiple disease states.

Key Scientific Pillars

  • Mitochondrial Ion Channel Biology: Deep understanding of MPC1, UCP2, and related channels in metabolic regulation
  • Structure-Based Drug Design: Advanced computational and crystallography approaches to optimize selectivity and potency
  • Translational Biomarkers: Development of robust pharmacodynamic markers to guide clinical development
  • Disease-Relevant Models: Comprehensive preclinical testing in validated models of metabolic disease

Pipeline

Program Target Indication Stage
AZO-101 MPC1 Agonist Type 2 Diabetes IND-Enabling
AZO-201 UCP2 Modulator Obesity Lead Optimization
AZO-301 MPC1 Agonist Mitochondrial Myopathy Preclinical
AZO-401 Dual MPC1/UCP2 Metabolic Syndrome Discovery

MPC1: Mitochondrial Pyruvate Carrier

The Mitochondrial Pyruvate Carrier (MPC1) is a critical gatekeeper of metabolic flux, controlling the entry of pyruvate into mitochondria for oxidative metabolism. Dysfunction of MPC1 has been implicated in insulin resistance, type 2 diabetes, and various metabolic disorders.

Therapeutic Rationale

Our MPC1 agonist program aims to restore proper mitochondrial pyruvate uptake, thereby enhancing glucose oxidation and improving insulin sensitivity. This approach addresses a fundamental metabolic defect observed in diabetes and metabolic syndrome.

Program Highlights

  • Highly selective MPC1 agonists with >100-fold selectivity over related transporters
  • Excellent tissue penetration and mitochondrial localization
  • Demonstrated efficacy in multiple preclinical models of diabetes
  • Favorable safety profile in toxicology studies

UCP2: Uncoupling Protein 2

Uncoupling Protein 2 (UCP2) plays a crucial role in regulating mitochondrial membrane potential and reactive oxygen species production. Modulation of UCP2 activity offers a novel approach to treating metabolic diseases and obesity.

Therapeutic Rationale

Our UCP2 modulator program focuses on fine-tuning mitochondrial efficiency to enhance energy expenditure and reduce oxidative stress. This dual mechanism offers potential benefits in both obesity and metabolic complications.

Program Highlights

  • First-in-class UCP2 modulators with optimized pharmacokinetic properties
  • Demonstrated weight reduction and improved metabolic parameters in preclinical models
  • Reduced oxidative stress markers in disease-relevant assays
  • Oral bioavailability with once-daily dosing potential