Aeovian Pharmaceuticals, a clinical-stage biopharmaceutical company focused on restoring cellular metabolic quality control, has successfully closed an oversubscribed $55 million Series B financing. This substantial investment is earmarked to propel the development of its lead candidate, AV078, a first-in-class, CNS-penetrant selective mTORC1 inhibitor, and finance the completion of its crucial Phase 2 proof-of-concept study.
The funding round was co-led by new investors Luma Group and CTI Life Sciences Fund, signaling strong conviction in Aeovian Pharmaceuticals‘s differentiated approach. They were joined by key participants including Foresite Capital, SymBiosis (using the most relevant website found for “SymBiosis” as a placeholder, as the specific VC’s website was not clear), the TSC Alliance Endowment Fund, and legal counsel Wilson Sonsini. Existing investors Apollo Health Ventures, Hevolution, Sofinnova Investments, and venBio also provided support, reinforcing their belief in the drug candidate.
Targeting the Root Cause of Refractory Seizures
The target indication is Tuberous Sclerosis Complex (TSC)-related refractory epilepsy, a severe complication of the rare genetic disorder TSC that affects an estimated one million people globally. Up to two-thirds of patients with TSC experience epilepsy that is resistant to current treatments.
TSC is driven by the hyperactivation of the mTORC1 pathway, a master regulator of cellular growth and metabolism. While current nonselective mTOR inhibitors exist, they are often limited by toxicities stemming from the undesired inhibition of the related mTORC2 pathway.
AV078 was meticulously engineered to overcome this limitation. As a highly selective mTORC1 inhibitor with effective central nervous system (CNS) penetration, it is designed to directly modulate hyperactive signaling in the brain, offering the potential for superior efficacy, improved tolerability, and sustained seizure control.
Allison J. Hulme, Ph.D., President & CEO of Aeovian Pharmaceuticals, highlighted the significance of the achievement: “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. 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.”
Partnership for the Patient Community
The investment includes a notable commitment from the TSC Alliance Endowment Fund, a supporting organization of the TSC Alliance, emphasizing the alignment between the company’s mission and patient advocacy.
Kari Luther Rosbeck, President & CEO of the TSC Alliance®, remarked on the collaboration: “We commend Aeovian Pharmaceuticals 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 is also taking an active role in our mission to catalyze new treatments and drive research toward a cure for Tuberous Sclerosis Complex.”
The lead investors articulated their confidence in the drug’s novelty. Themasap Khan, Ph.D., Co-Founder and Partner at Luma Group, now appointed to the Aeovian Pharmaceuticals Board of Directors, noted: “Aeovian Pharmaceuticals is pioneering a truly differentiated approach to targeting mTORC1 with a level of selectivity and CNS penetration that has not been achievable until now. 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.” Youssef Bennani, Ph.D., Managing Partner at CTI Life Sciences Fund, also joins the Board, bringing extensive industry leadership experience.
📰 Editorial View: The Precision of Targeted Medicine
The successful oversubscribed Series B financing for Aeovian Pharmaceuticals is a significant endorsement of their scientific philosophy: that highly targeted inhibition of specific cellular pathways is the future of complex disease management. Their lead candidate, AV078, is more than just another drug; it represents a refined understanding of the underlying biology of Tuberous Sclerosis Complex.
The critical distinction Aeovian Pharmaceuticals is making—avoiding mTORC2 inhibition while successfully modulating mTORC1—is a masterstroke of pharmaceutical chemistry. This move addresses the fundamental toxicity limitations that have plagued previous-generation nonselective mTOR inhibitors. By creating a molecule that is both highly selective and able to cross the blood-brain barrier (CNS-penetrant), Aeovian Pharmaceuticals has dramatically increased the probability of generating a best-in-class therapeutic. For the patients suffering from TSC-related refractory epilepsy, this precision could translate directly into fewer debilitating side effects and, most importantly, better long-term seizure control. This elegant solution to a critical unmet need highlights a company that is not taking incremental steps, but aiming for transformative outcomes.
Furthermore, the inclusion of the TSC Alliance Endowment Fund as an investor, alongside institutional biotech leaders like Foresite Capital and Sofinnova Investments, sends a powerful message. It demonstrates that the patient community, through its advocacy organizations, sees a direct financial and moral stake in the success of Aeovian Pharmaceuticals. This alignment of capital, science, and patient need positions the company to excel not just in the clinic, but in market adoption once approved. The development path for AV078 is a model for rare disease drug development: focused on fundamental biology, driven by high-quality science, and deeply connected to those who need the treatment most. We believe Aeovian Pharmaceuticals is poised to set a new benchmark for targeted therapy in neurological disorders.
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