AR101 – Enzastaurin

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.

Enzastaurin Clinical Development

PREVEnt Clinical Trial logo

Prevention of Rupture with Enzastaurin in Vascular Ehlers-Danlos Syndrome

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.

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.”

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.

N01161 06/22

Expanded Access Policy

Expanded access, also called compassionate use, enables patients with serious or immediately life-threatening diseases who do not meet the enrollment criteria for clinical trials in progress to gain access to investigational treatments. At this time, Aytu BioPharma does not offer an expanded access program and does not accept expanded access requests. We believe that investigational drugs should be studied in patients as part of clinical trials designed to produce data on safety and efficacy that may be used to support approval of the product, thereby leading to its broader availability for patients in need of treatment. Aytu strongly encourages patients to speak with their treating physicians and when possible to participate in clinical trials. Aytu is aware that in rare cases patients with serious life-threatening diseases are unable to participate in clinical trials and may have exhausted all available therapies. In these rare cases, Aytu may consider providing an investigational product outside of a clinical trial. However, we currently do not have an expanded access program that allows patients to have access to our investigational products prior to FDA approval. As authorized by the 21st Century Cures Act, Aytu may revise this expanded access policy at any time. Additionally, the posting of this policy by Aytu shall not serve as a guarantee of access to any specific investigational drug by any individual patient. In the event Aytu decides to consider expanded access, we will evaluate and respond to each request that it receives on a case-by-case basis. Reference information about our investigational drugs and ongoing clinical trials can be found on and If you have additional questions, please speak with your physician, or contact us at