Akava Therapeutics is focused on developing innovative small molecule therapeutics that target various mechanisms of disease, particularly in neurodegenerative disorders and cancers. Their lead compound, AKV9, is a protein aggregation inhibitor specifically designed to improve the health of upper motor neurons (UMNs) affected by conditions such as amyotrophic lateral sclerosis (ALS). AKV9 has demonstrated the ability to reduce protein aggregation, enhance axon health, and maintain the integrity of cellular structures like mitochondria and the endoplasmic reticulum. This compound has shown promising results in preclinical studies, where it improved UMN health in mouse models, suggesting potential for translation to human therapies.

In addition to AKV9, Akava Therapeutics is developing ornithine aminotransferase (OAT) inactivators aimed at treating hepatocellular carcinoma (HCC). These inactivators have been shown to inhibit tumor growth and the secretion of alpha-fetoprotein, a biomarker associated with HCC. The company is also exploring neuronal nitric oxide synthase (nNOS) inhibitors for potential applications in treating neurodegenerative diseases and melanoma. By focusing on inhibition strategies across their therapeutic pipeline, Akava Therapeutics aims to address significant unmet medical needs in both oncology and neurodegeneration, ultimately improving patient outcomes and quality of life.

United States

AKAVA Therapeutics is developing first-in-class small molecule therapeutics that inhibit protein aggregation, inhibit enzymes and inhibit cancer for a variety of neurodegenerative diseases.

Akava Therapeutics

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