AR101 – Enzastaurin

Enzastaurin Clinical Development

PREVEnt Clinical Trial logo

Prevention of Rupture with Enzastaurin in Vascular Ehlers-Danlos Syndrome

To register to receive more information about the planned PREVEnt Trial, click the button below

Objective: To evaluate the efficacy of enzastaurin compared to placebo in preventing arterial events leading to intervention in patients with Vascular Ehlers-Danlos Syndrome (VEDS) confirmed with COL3A1 gene mutations.

VEDS/COL3A1 Overview

Upcoming Clinical Trial

Aytu BioPharma, Inc. will be sponsoring an upcoming clinical trial to evaluate the effectiveness of AR101 (enzastaurin) in preventing cardiac or arterial events in patients with Vascular Ehlers-Danlos Syndrome (VEDS) confirmed with COL3A1 gene mutations, compared to placebo. Please register to receive important updates concerning this trial.

About Vascular Ehlers-Danlos Syndrome (VEDS)

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.

Diagram 1

VEDS affects about 1 in 50,000 people worldwide. Nearly 50% of patients with this devastating condition die before the age of 50 years old. Currently, there are no FDA-approved therapies, and after diagnosis, the current standard of care is “watchful waiting.”

About AR101 (enzastaurin)

Enzastaurin is a well characterized PKCβ inhibitor that has been evaluated in over 50 clinical trials, with more than 3300 patients. This includes a Phase 3 study of nearly 500 patients with 3 years of enzastaurin treatment.1 Mutations in the COL3A1 gene have been linked to the loss of structural integrity of the extracellular matrix and increased clinical presentation of VEDS related symptoms, including arterial dissection and/or rupture. 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.2 Further investigation will be necessary to determine the potential of PKC inhibition as a treatment option.

Scientific Advisory Board

Living with VEDS

VEDS changes lives, but it does not define the people who have it. Watch the stories of those confronting VEDS with courage, conviction, and above all, hope for a better future.




The Tays Family

Aytu BioPharma is a proud sponsor of these VEDS Advocacy Groups

Annabelle’s Challenge



The Ehlers Danlos Society Logo

Ehlers-Danlos Society



The VEDS Movement logo

The VEDS Movement


1. 2. Bowen CJ et al. Targetable cellular signaling events mediate vascular pathology in vascular Ehlers-Danlos Syndrome. J Clin Invest. 2020 Feb 3;130(2):686-698.

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