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TSC Alliance Endowment Fund's Investment in Aeovian Pharmaceuticals

The TSC Alliance Endowment Fund, an organization that supports the mission of the TSC Alliance, is making significant strides in the fight against Tuberous Sclerosis Complex (TSC). In a recent announcement, they revealed an investment in Aeovian Pharmaceuticals as part of a Series B financing round. This investment aims to advance the clinical development of AV078, a selective mTORC1 inhibitor that specifically targets central nervous system (CNS) conditions associated with TSC.

The Mission Behind the Investment

Rita DiDomenico, Chair of the TSC Alliance Endowment Fund, stated that the new equity stake aligns with the fund's investment strategy, which focuses on ventures directly related to enhancing the treatment landscape for TSC patients. Funds generated from this investment will be reinvested into TSC Alliance's mission of promoting research towards effective treatment options and striving for a cure.

A Landmark Collaboration

The roots of this partnership go back to 2018 when the TSC Alliance collaborated with Aeovian Pharmaceuticals through the TSC Preclinical Consortium. This partnership allowed for the co-funding of essential research focused on creating targeted mTORC1 inhibitors for TSC, particularly focusing on epilepsy associated with the condition.

mTORC1, a critical protein complex involved in various cellular processes, plays a crucial role in the pathology of TSC. The TSC Preclinical Consortium has been integral in identifying leading drug candidates through systematic and efficient testing processes. To date, the Consortium has collaborated with 27 industry partners and screened nearly 100 compounds.

Key Developments and Clinical Progress

The development of AV078 signifies the first candidate emerging from the TSC Preclinical Consortium's initiatives to identify effective treatment options. Steven L. Roberds, Chief Scientific Officer at the TSC Alliance, expressed excitement over the progress made, noting that initial mouse studies conducted in 2018 paved the way for enhanced clinical candidates developed in collaboration with the Consortium.

Positive results from these studies have driven the advancements of AV078, and from 2024 to 2025, it underwent Phase 1 testing on healthy volunteers—marking an important milestone in the drug's development journey.

Importance of Collaboration

Allison J. Hulme, President and CEO of Aeovian Pharmaceuticals, emphasized the role of the TSC Preclinical Consortium in accelerating AV078's development. The Consortium provided access to stringent disease-relevant models that made it possible to assess critical parameters such as selectivity, efficacy, and CNS penetration effectively.

Following the successful Phase 1 trials, Aeovian is set to initiate Phase 2 clinical trials for AV078, a significant step towards making a new treatment available for patients affected by TSC-related epilepsy. For more details regarding the clinical trial process, individuals can visit Aeovian’s official website.

Understand Tuberous Sclerosis Complex (TSC)

Tuberous Sclerosis Complex is a rare genetic disorder characterized by the growth of benign tumors in multiple organs, primarily the brain, heart, kidneys, skin, and lungs. The disorder is associated with a range of symptoms, including epilepsy, developmental delays, and behavioral issues.

The TSC Alliance plays a critical role in improving the quality of life for families affected by this complex disorder. Through initiatives like the Endowment Fund, they are dedicated to catalyzing new treatment options, expanding research efforts, and ultimately enhancing support for those impacted by TSC.

Looking Ahead

This investment marks a promising development in the journey towards better treatment solutions for TSC patients. The continuous collaboration between TSC Alliance and Aeovian Pharmaceuticals highlights the commitment to advancing research and providing hope for patients and families dealing with the challenges associated with Tuberous Sclerosis Complex. As the Phase 2 trials begin, the medical community and TSC advocates alike will be watching closely for updates on AV078’s progress.

For further information about TSC Alliance’s efforts, please check the TSC Alliance website.