Aeovian Pharmaceuticals is a clinical-stage biopharmaceutical company dedicated to developing targeted and highly selective small molecules to restore cellular metabolic quality control.
By addressing dysregulated growth and hyperactive signaling found in certain rare genetic and age-related diseases, the company aims to create therapies that improve healthspan and combat the effects of aging.
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The Solution
Aeovian Pharmaceuticals is developing therapies targeting the rapamycin complex 1 (mTORC1) pathway, a crucial regulator of cellular growth and metabolism. By focusing on selective inhibition of mTORC1, the company aims to address various rare genetic and age-related diseases characterized by hyperactive mTORC1 signaling.
AV078: Lead Development Candidate
The company’s primary therapeutic candidate, AV078, is a central nervous system (CNS) penetrant selective mTORC1 inhibitor. In March 2024, Aeovian initiated a Phase 1 clinical trial to assess the safety, tolerability, and pharmacokinetics of AV078 in healthy adult volunteers. This study includes single and multiple ascending dose cohorts, as well as evaluations of food effects and potential drug-drug interactions.
AV078 is being developed for the treatment of refractory epilepsy associated with Tuberous Sclerosis Complex (TSC), a rare genetic disorder resulting from mutations in the TSC1 or TSC2 genes. These mutations lead to hyperactive mTORC1 signaling, contributing to various clinical manifestations, including seizures. Current treatments, such as the nonselective mTORC1/mTORC2 inhibitor everolimus, have shown limited efficacy and are often associated with dose-limiting toxicities due to off-target effects. In contrast, AV078’s selective inhibition of mTORC1 offers the potential for improved safety and efficacy in managing TSC-related epilepsy.
The Development Pipeline
Beyond AV078, Aeovian has developed a proprietary library of small molecules that are potent and selective inhibitors of either mTORC1 or CD38. These compounds have the potential to precisely target the underlying biology of multiple rare and age-related diseases, thereby restoring cellular metabolic quality control.
The development pipeline is illustrated by the following image from their official website:
The Company
Aeovian Pharmaceuticals was founded in 2012, and is headquartered in Berkeley, California, USA. The company was established with technology licensed from The Buck Institute for Research on Aging.
The company has secured significant funding to advance its research and development efforts:
- March 2024: Secured an additional $50 million in a Series A extension, led by Hevolution Foundation, contributing $20 million. Existing investors Apollo Health Ventures, Sofinnova Investments, venBio, Evotec, and b2venture also participated.
- October 2019: Raised $37 million in a Series A financing round co-led by venBio and Sofinnova Investments, with participation from Apollo Health Ventures and Evotec.
In terms of partnerships Aeovian Pharmaceuticals collaborates with various organizations to support its mission. Notably, the company has partnered with Evotec SE, a Germany-based drug discovery and development company, to advance its therapeutic programs.
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