Aeovian Pharmaceuticals Secures $55 Million Series B to Advance First-in-Class mTORC1 Inhibitor for Rare Epilepsy
Key Insights
Aeovian Pharmaceuticals (search) closed an oversubscribed $55 million Series B financing to fund completion of a Phase 2 proof-of-concept study for AV078 in tuberous sclerosis complex (search)-related refractory epilepsy (search).
AV078 represents a first-in-class CNS-penetrant selective mTORC1 (search) inhibitor designed to address the underlying mTORC1-driven biology that causes seizures in two-thirds of TSC patients who remain refractory to current treatments.
The financing round was co-led by Luma Group (search) and CTI Life Sciences Fund (search), with participation from multiple investors including the TSC Alliance (search) Endowment Fund, reflecting strong conviction in the therapeutic potential.
Aeovian Pharmaceuticals (search) has successfully closed an oversubscribed $55 million Series B financing round to advance its lead candidate AV078, a first-in-class selective mTORC1 (search) inhibitor, through Phase 2 clinical development for tuberous sclerosis complex (search) (TSC)-related refractory epilepsy (search). The Berkeley-based clinical-stage biopharmaceutical company announced the funding will specifically support completion of a Phase 2 proof-of-concept study in participants with TSC-refractory epilepsy.
Addressing a Critical Unmet Medical Need
TSC represents a significant medical challenge, affecting an estimated one million people globally, including approximately 50,000 individuals in the United States, with an incidence of roughly 1 in 6,000 births. Epilepsy (search) emerges as one of the most common and difficult complications of this rare genetic disorder, with current treatments often failing to address the mTORC1 (search)-driven biology at the root of these seizures.
The condition presents particularly challenging treatment scenarios, as two-thirds of patients with TSC-related epilepsy (search) experience seizures that remain refractory to existing therapeutic options. Current treatments, including nonselective mTOR inhibitors like everolimus, are significantly limited by toxicities resulting from mTORC2 (search) inhibition.
Innovative Therapeutic Approach
AV078 was specifically engineered for highly selective mTORC1 (search) inhibition with CNS penetration to directly modulate hyperactive signaling in the brain. The compound targets metabolic quality control by selectively inhibiting mTORC1, which becomes hyperactivated in TSC due to TSC1 (search) or TSC2 (search) mutations and often drives epilepsy (search) that remains refractory in two-thirds of patients.
"Building on the success of our Phase 1 study, we believe AV078 has the potential to be a safer, better-tolerated, and more efficacious treatment option for patients with TSC," said Allison J. Hulme, Ph.D., President & CEO of Aeovian Pharmaceuticals (search). "The successful close of this financing marks an important milestone for the company and reflects the strong conviction in AV078's potential to transform outcomes for patients who have long lacked effective treatment options."
The oral drug aims to deliver improved efficacy, better tolerability, and sustained seizure control compared to existing treatments by achieving a level of selectivity and CNS penetration that has not been achievable until now.
Strong Investor Confidence and Strategic Partnerships
The financing round was co-led by new investors Luma Group (search) and CTI Life Sciences Fund (search), with participation from Foresite Capital, SymBiosis, the TSC Alliance (search) Endowment Fund, and Wilson Sonsini. Existing investors Apollo Health Ventures (search), Hevolution, Sofinnova Investments, and venBio also supported the financing.
"Aeovian is pioneering a truly differentiated approach to targeting mTORC1 (search) with a level of selectivity and CNS penetration that has not been achievable until now," said Themasap Khan, Ph.D., Co-Founder and Partner at Luma Group (search). "We believe AV078 has the potential to establish a new standard of care for TSC-related epilepsy (search), where current therapies fall short for the majority of patients."
The TSC Alliance (search)'s participation through its Endowment Fund represents a significant endorsement from the patient community. "The TSC Alliance is grateful for our longstanding partnership with Aeovian Pharmaceuticals (search) through research conducted in our TSC Preclinical Consortium," says Kari Luther Rosbeck, President & CEO of the TSC Alliance. "We commend Aeovian for developing a targeted compound addressing unmet needs in the TSC community as two-thirds of those who experience epilepsy (search) in TSC are refractory."
Strategic Leadership Expansion
As part of the Series B financing, Aeovian has appointed Themasap Khan, Ph.D., co-founder and partner at Luma Group (search), and Youssef Bennani, Ph.D., managing partner at CTI Life Sciences, to its Board of Directors. These appointments bring diverse and relevant experience from various leadership roles within the biotechnology industry as the company enters its next phase of growth.
Company Evolution and Future Outlook
The Series B represents a significant progression from Aeovian's $37 million Series A funding round completed in October 2019. According to CEO Hulme, the Series A was centered on establishing the scientific foundation, validating the biology, building the team and infrastructure needed to move into the clinic, and successfully completing the Phase 1 clinical trial.
"Aeovian was able to demonstrate meaningful progress and clarity around its clinical path, which shifted the conversation with investors toward execution, capital efficiency, and scalability," Hulme explained. "As a result, the Series B was more targeted, with deeper diligence around clinical design and long-term indication expansion, reflecting the company's evolution from platform build to clinical advancement."
Aeovian's wholly-owned pipeline extends beyond TSC, targeting metabolic quality control by selectively inhibiting mTORC1 (search) or CD38 (search). The company is developing a pipeline of highly selective inhibitors with the potential to address multiple rare and age-related diseases driven by similar biological mechanisms.