RARE Daily

FDA Grants Aytu BioPharma Fast Track Designation for Vascular Ehlers-Danlos Syndrome Therapy

April 19, 2022

Rare Daily Staff

The U.S. Food and Drug Administration granted Aytu BioPharma Fast Track designation for its experimental therapy AR101 (enzastaurin), a protein kinase C β inhibitor, for the treatment of patients with Vascular Ehlers-Danlos Syndrome.

Vascular Ehlers-Danlos Syndrome (VEDS) is an inherited connective tissue disorder, typically caused by a mutation in the COL3A1 gene. This mutation leads to defects in type III procollagen, a major protein in vessel walls and hollow organs. Patients with this diagnosis are at significant risk for serious vascular events like dissections, pseudoaneurysms, and ruptures throughout the vasculature.

AR101 (enzastaurin) is a well characterized PKCβ inhibitor that has been evaluated in more than 50 clinical trials, with more than 3,300 patients. This includes a phase 3 study of nearly 500 patients with 3 years of enzastaurin treatment. Recent findings from animal studies, in a VEDS mouse model with similar Col3A1 mutations, have shown that the mutation is a key mediator in increased PKC/ERK pathway signaling. Additionally, in this model, treatment with an inhibitor of PKCβ significantly prevented death due to spontaneous aortic rupture.

AR101 has received Orphan Drug designation in the United States and Europe, and the AR101 Investigational New Drug application has been accepted by the U.S. FDA to begin a registrational study.

Fast Track is a process designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need. Fast Track addresses a broad range of serious conditions, and the request can be initiated by a pharmaceutical company at any time during the development process. FDA reviews the request and decides based on whether or not the drug fills an unmet medical need in a serious condition. Once a drug receives Fast Track designation, early and frequent communication between the FDA and the sponsor is encouraged throughout the entire drug development and review process.

“With this important designation now in hand, we are focused on getting the operational elements of the pivotal PREVEnt study of AR101 in place such that we can initiate it as quickly as possible, with plans to begin patient dosing by late 2022 or early 2023,” said Josh Disbrow, CEO of Aytu BioPharma.

The PREVEnt Trial is designed to enroll approximately 260 COL3A1-positive VEDS patients to assess time to arterial events leading to intervention among patients treated with AR101 compared to patients treated with standard of care. Including the planned enrollment period, the study is expected to last approximately thirty months.

Photo: Josh Disbrow, CEO of Aytu BioPharma

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