TSC Alliance invests in the development of an mTORC1 selective inhibitor for tuberous sclerosis complex
19 Dec 2025
The TSC Alliance Endowment Fund, a supporting organization of the TSC Alliance, has invested in Aeovian Pharmaceuticals as part of their Series B financing in support of their Phase 2 trial of AV078, a first-in-class CNS-penetrant selective mTORC1 inhibitor in tuberous sclerosis complex (TSC).
In 2018, the TSC Alliance partnered with Aeovian Pharmaceuticals by co-funding research through the TSC Preclinical Consortium. This marked a significant step towards the development of innovative mTORC1-specific inhibitors for TSC, particularly in addressing TSC-associated epilepsy.
mTORC1 is a protein complex involved in cell growth and proliferation, known to play a pivotal role in TSC pathology. The TSC Preclinical Consortium provides a screening platform to identify lead clinical drug candidates through a systematic testing process in robust and reproducible models of TSC. Since 2016, the Preclinical Consortium has worked with 27 industry partners screening 97 compounds.
"AV078 represents the first drug candidate developed using the TSC Preclinical Consortium as the preclinical efficacy engine throughout the lead development processto identify a novel clinical candidate," said Steven L. Roberds, PhD, Chief Scientific Officer at the TSC Alliance.
"We are thrilled our early investment to create the TSC Preclinical Consortium and ongoing partnership has helped enable Aeovian's groundbreaking work with AV078," he added.
Following positive results in the first mouse study in 2018, Aeovian developed improved clinical candidates in partnership with the Preclinical Consortium over the next three years. From 2024 to 2025, AV078 completed Phase 1 testing in healthy volunteers.
"Our collaboration with the TSC Preclinical Consortium was instrumental in identifying and advancing AV078," said Allison J. Hulme, Ph.D. President & CEO, Aeovian Pharmaceuticals. "The Consortium's rigorous and disease-relevant models allowed us to rapidly evaluate selectivity, CNS penetration, and efficacy, giving us the confidence to move AV078 into clinical development. This partnership was critical in accelerating our path to Phase 2 trials."