IPG8294: First-in-Class Small Molecule Inhibitor of a NADase


Mitochondrial dysfunction plays a crucial role in the pathogenesis of various diseases, including Alzheimer's disease (AD), Parkinson's disease (PD), Huntington's disease (HD), Amyotrophic lateral sclerosis (ALS), Mitochondrial myopathy (MM), Duchenne muscular dystrophy (DMD) and other related disorders. The enzyme "Target," which possesses hydrolytic activity, has been identified as a significant contributor to the development of mitochondrial dysfunction in various pathological conditions. IPG8294 has been specifically designed to selectively target and suppress the hydrolytic activity of the aforementioned enzyme. Therefore, IPG8294 holds great potential as a therapeutic agent for effectively treating disorders associated with mitochondrial dysfunction.

Mechanism of Action


■ Mitochondrial quality control pathways are tightly regulated and interconnected, including mitochondrial fission and fusion, mitophagy, and mitochondrial biogenesis, 

■ Dysregulation of the target enzyme plays a substantial role in mitochondrial dysfunction.

■ Blockade of the target by IPG8294 holds the potential to restore mitochondrial function and improve the progression of associated diseases.

Key differentiation

■IPG8294 represents a groundbreaking small-molecule inhibitor with significant potential in addressing mitochondrial dysfunction. 

■IPG8294 is a disease-modifying agent for the treatment of mitochondrial myopathy and neurodegenerative disorders.

In vivo properties

■ IPG8294 improves mitochondrial quality in the brain of APP/PS1 mice 


TEM: The percentage of damaged mitochondria (Type II, III, IV) was significantly decreased by IPG8294 treatment (Statistic data was expressed as mean ± SEM. **** P < 0.0001, compared to vehicle)

■ IPG8294 increases expression of PSD-95 and Synaptophysin in a dose-dependent manner in the brain of APP/PS1 mice.


WB: Synapse loss was significantly prevented by IPG8294 treatment in APP/PS1 mice brain (Statistic data was expressed as mean ± SEM. *** P < 0.001, **** P < 0.0001 compared to vehicle)

Current status

■ IND application