Restoring Energy Homeostasis to Treat Severe Metabolic Disorders

OrsoBio’s development programs address high unmet medical need in patients with severe metabolic disorders, including obesity, type 2 diabetes, severe hypertriglyceridemia, and NASH.

Our programs address the root cause of organ dysfunction by modulating fundamental metabolic pathways with four highly targeted and complementary mechanisms:

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Prevalence of obesity in the United States

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Type 2 diabetes patients worldwide

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Risk of pancreatitis in severe hypertriglyceridemia

Silhouettes of a male and a female human body colored with biopharmaceutical company OrsoBio brand colors in a flame motif
1

Increased fatty acid oxidation in cardiac muscle (ACC2), plus beneficial effects on plasma lipids (LXR, protonophores)

2

Hepatic DNL inhibition (LXR, protonophores), increased fatty acid oxidation (ACC2, protonophores), increased NAD+ synthesis, and improved mitochondrial function (ACMSD)

3

Increased NAD+ synthesis and improved mitochondrial function in kidneys (ACMSD)

4

Increased fatty acid oxidation in skeletal muscle (ACC2)

Each of our programs modulates central aspects of cellular energetics in key, energetically-driven organs

PROGRAM OVERVIEW

ACC2 Inhibitor (TLC-3595)

PROGRAM OVERVIEW

LXR Inverse Agonist (TLC-2716)

PROGRAM OVERVIEW

Mitochondrial Protonophores (TLC-6740, TLC-1235)

PROGRAM OVERVIEW

ACMSD Inhibition

Publications

November 10, 2023

ACMSD inhibition enhances de novo NAD+ biosynthesis and reverses NAFLD/NASH (AASLD 2023, Abstract #2273)

November 6, 2023

Preliminary safety, pharmacokinetics, and lipid lowering effects of the liver-targeted liver X receptor (LXR) inverse agonist TLC-2716 in healthy volunteers (AHA Scientific Sessions 2023, Abstract #Mo3209)

November 1, 2022

Enhanced lipogenesis associated with a high-risk GCKR variant (rs1260326) is effectively suppressed by TLC-2716, a novel, liver-targeted LXR inverse agonist in steatotic human liver organoids (AASLD 2022, Abstract #4210)

November 1, 2022

Evaluation of the safety and pharmacokinetic effects of the oral, acetyl-CoA carboxylase-2 (ACC2) inhibitor TLC-3595 in healthy volunteers (AASLD 2022, Abstract #3677)

November 1, 2022

TLC-6740, a potent liver-targeted mitochondrial protonophore, has multiple metabolic benefits in preclinical models (AASLD 2022, Abstract #2551)

November 1, 2022

TLC-2716, a potent, liver-targeted, inverse agonist of the liver X receptor (LXR), demonstrates profound reductions in hepatic and plasma lipids in dysmetabolic rodent models (AASLD 2022, Abstract #2532)

November 1, 2022

Enhancement of de novo NAD+ biosynthesis by novel ACMSD inhibitors improves mitochondrial function in iPSC-derived human liver organoid models of steatohepatitis (AASLD 2022, Abstract #2441)

Recent News

OrsoBio team members collaborated with Professor Takebe in his pioneering study in Cell on the first use of en masse human liver organoids to define the genetic basis of metabolic liver disease and potential for tailored therapeutic development.