Aeovian Pharmaceuticals Raises $55 Million to Advance First-in-Class Selective mTORC1 Inhibitor for Tuberous Sclerosis Complex-Related Epilepsy

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8:00 AM on Tuesday, December 16

The Associated Press

BERKELEY, Calif.--(BUSINESS WIRE)--Dec 16, 2025--

Aeovian Pharmaceuticals, a clinical-stage biopharmaceutical company developing highly selective small molecule therapeutics that restore cellular metabolic quality control, today announced it has closed an oversubscribed $55 million Series B financing that will fund completion of a Phase 2 proof-of-concept study for its lead development candidate, AV078, in tuberous sclerosis complex (TSC)-related refractory epilepsy.

The funding round was co-led by new investors Luma Group and CTI Life Sciences Fund, with participation from Foresite Capital, SymBiosis, the TSC Alliance Endowment Fund and Wilson Sonsini. Existing investors Apollo Health Ventures, Hevolution, Sofinnova Investments and venBio also supported the financing.

Proceeds from the financing will support the advancement of AV078 into a Phase 2 proof-of-concept study in participants with TSC-refractory epilepsy. TSC is a rare genetic disorder affecting an estimated one million people globally, including ~50,000 in the United States, with an incidence of roughly 1 in 6,000 births. Epilepsy is one of the most common and difficult complications, and current treatments often fail to address the mTORC1-driven biology at the root of these seizures.

“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, Aeovian Pharmaceuticals. “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 TSC Alliance is grateful for our longstanding partnership with Aeovian Pharmaceuticals 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 in TSC are refractory. Through their investments in Series B funding, the TSC Alliance Endowment Fund, a supporting organization of the TSC Alliance, is also taking an active role in our mission to catalyze new treatments and drive research toward a cure for tuberous sclerosis complex.”

Aeovian’s wholly-owned pipeline targets metabolic quality control by selectively inhibiting mTORC1, which becomes hyperactivated in TSC due to TSC1 or TSC2 mutations and often drives epilepsy that remains refractory in two-thirds of patients. Current options, including nonselective mTOR inhibitors like everolimus, are limited by toxicities from mTORC2 inhibition. AV078 was engineered for highly selective mTORC1 inhibition with CNS penetration to directly modulate hyperactive signaling in the brain, aiming to deliver better efficacy, tolerability, and sustained seizure control.

“Aeovian is pioneering a truly differentiated approach to targeting mTORC1 with a level of selectivity and CNS penetration that has not been achievable until now,” said Themasap Khan, Ph.D., Co-Founder and Partner, Luma Group. “We believe AV078 has the potential to establish a new standard of care for TSC-related epilepsy, where current therapies fall short for the majority of patients.”

As part of the Series B financing, Aeovian has appointed Themasap Khan, Ph.D., co-founder and partner at Luma Group, and Youssef Bennani, Ph.D., managing partner at CTI Life Sciences, to its Board of Directors.

"On behalf of our Board of Directors, I am delighted to welcome Themasap and Youssef to our Board as they bring diverse and relevant experience from various leadership roles within the biotechnology industry and we look forward to their contributions as Aeovian enters its next phase of growth," said Richard Gaster, M.D., Ph.D., Chairman of the Aeovian Board of Directors.

About Aeovian Pharmaceuticals

Aeovian Pharmaceuticals is a clinical-stage biopharmaceutical company developing targeted and highly selective small molecules that restore cellular metabolic quality control by inhibiting mTORC1 or CD38. The company’s lead candidate, AV078, is a first-in-class CNS-penetrant selective mTORC1 inhibitor in Phase 2 development for refractory epilepsy associated with tuberous sclerosis complex (TSC), a rare genetic disorder driven by hyperactive mTORC1 signaling. Aeovian is developing a pipeline of highly selective inhibitors with the potential to address multiple rare and age-related diseases. For more information, visit www.aeovian.com.

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SOURCE: Aeovian Pharmaceuticals