Aeovian Pharmaceuticals is developing AV078, a selective mTORC1 inhibitor designed to treat refractory epilepsy associated with tuberous sclerosis complex (TSC), a rare genetic disorder characterized by hyperactive mTORC1 signaling. The company aims to provide a targeted therapeutic option that improves safety and efficacy compared to existing nonselective mTOR inhibitors, addressing the significant unmet medical need in managing TSC-related seizures.
Funding
Funding not disclosed
Founders
Product
Problem
Tuberous sclerosis complex (TSC) is a rare genetic disorder characterized by hyperactive mTORC1 signaling, leading to dysregulated cell growth and the development of tumors, often resulting in refractory epilepsy. Current nonselective mTOR inhibitors used to manage TSC-related seizures have tolerability issues and dose-limiting toxicities, which limit their efficacy.
Solution
Aeovian Pharmaceuticals is developing AV078, a first-in-class, CNS-penetrant, selective mTORC1 inhibitor designed to address refractory epilepsy associated with TSC. By selectively targeting mTORC1, AV078 aims to restore cellular metabolic quality control and reduce the dysregulated growth and hyperactive signaling characteristic of TSC. This targeted approach seeks to improve both the safety and efficacy profiles compared to existing nonselective mTOR inhibitors, offering a more effective therapeutic option for managing TSC-related seizures. AV078 is currently being evaluated in a Phase 1 clinical trial.
Target Audience
The primary target audience includes patients with tuberous sclerosis complex (TSC) who experience refractory epilepsy, as well as physicians specializing in neurology and rare genetic disorders.
Features
- Highly selective inhibition of mTORC1 to minimize off-target effects
- CNS penetration for direct targeting of brain-related manifestations of TSC
- Designed to improve the safety and tolerability profile compared to nonselective mTOR inhibitors
- Potential to precisely target the underlying biology of TSC-related epilepsy
- Proprietary library of small molecules with potential for treating other rare and age-related diseases