RARE Daily

Aeovian Raises $50 Million, Doses First Cohort in Phase 1 TSC Study

April 3, 2024

Rare Daily Staff

Aeovian Pharmaceuticals, a clinical-stage company developing therapeutics that potently inhibit the mTORC1 pathway, completed a $50 million financing and said it has dosed the first cohort of participants in a phase 1 clinical trial for its lead development candidate AV078, a first-in-class CNS penetrant selective mTORC1 inhibitor for the treatment of seizures associated with tuberous sclerosis complex.

Tuberous sclerosis complex (TSC) is a rare genetic disease that causes non-cancerous tumors to grow in the brain and several areas of the body, including the spinal cord, nerves, eyes, lung, heart, kidneys, and skin. TSC is a lifelong condition. The prognosis for individuals with TSC is highly variable and depends on the severity of symptoms with some people able to lead independent, productive lives, while others have symptoms that can affect everyday life and be life-threatening. Currently there is no cure for TSC.

The financing, led by Hevolution with participation by existing investors, will support the completion of the phase 1 clinical trial in healthy volunteers and prepare for the start of a subsequent phase 2 clinical trial in adult and pediatric patients with TSC refractory epilepsy.

“TSC is a genetic disorder of mTORC1 hyperactivation. We believe that our first-in-class CNS penetrant selective mTORC1 inhibitor, AV078, has the potential to be transformative for patients with TSC refractory epilepsy, a patient population in need of therapeutic options with better efficacy and tolerability,” said Allison Hulme, president and CEO of Aeovian Pharmaceuticals. “Beyond AV078, we believe that there is vast potential for our library of proprietary compounds in treating mTORC1-mediated rare and age-related diseases.”

Supporting the transition into the clinic, Aeovian also expanded its leadership team with the appointment of seasoned industry veterans William Greene, chief investment officer at Hevolution, and Justin Gover, former CEO of GW Pharmaceuticals, to the company’s Board of Directors, and appointed Micah Zajic as chief financial officer.

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