Aeovian Pharmaceuticals

Aeovian Pharmaceuticals is a clinical-stage biopharmaceutical company that was founded in 2012 and was originally known as Delos Pharmaceuticals. The company was incubated at the Buck Institute for Research on Aging, which was its original founder and remains a shareholder. Aeovian is focused on developing highly selective small molecules designed to restore cellular metabolic quality control. The company's therapeutic targets address dysregulated growth and hyperactive signaling characteristic of certain rare genetic disorders and age-related diseases.

The company's core strategy revolves around the modulation of the mechanistic target of rapamycin complex 1 (mTORC1) pathway. Its business model is centered on the discovery, development, and eventual commercialization of its drug candidates, funded by significant venture capital investment. To date, Aeovian has raised $88.2 million through several funding rounds, including a $37 million Series A in 2019 and a $50 million financing in 2024 led by Hevolution. This capital is being used to advance its lead candidate through clinical trials and prepare for subsequent phases.

Aeovian's lead drug candidate, AV078, is a central nervous system (CNS) penetrant, selective mTORC1 inhibitor. It is currently in a Phase 1 clinical trial to evaluate its safety, tolerability, and pharmacokinetics in healthy volunteers. The primary indication for AV078 is the treatment of refractory epilepsy associated with Tuberous Sclerosis Complex (TSC), a rare genetic disorder caused by hyperactive mTORC1 signaling. Existing treatments for TSC inhibit both mTORC1 and mTORC2, which can lead to significant side effects; AV078's selectivity for mTORC1 aims to provide a better-tolerated and more effective therapeutic option. Beyond AV078, the company has cultivated a proprietary library of small molecules. This collection includes other potent mTORC1 inhibitors as well as inhibitors for CD38, an enzyme linked to age-related NAD+ decline and metabolic dysfunction. This positions Aeovian to potentially address a wider range of rare and age-related diseases in the future.

Keywords: Aeovian Pharmaceuticals, clinical-stage biopharmaceutical, mTORC1 inhibitor, AV078, Tuberous Sclerosis Complex, refractory epilepsy, rare genetic diseases, age-related diseases, small molecule therapeutics, CD38 inhibitors, cellular metabolic quality control, CNS penetrant, drug development, Hevolution, Apollo Health Ventures, venBio, Sofinnova Investments, Buck Institute for Research on Aging, neurotherapeutics, metabolic dysfunction