New Aytu Advisory Board Working to Advance AR101 for vEDS

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by Margarida Maia |

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Aytu BioPharma has formed its new scientific advisory board, which will work toward advancing the global development of AR101 (enzastaurin), an antitumor agent with the potential to treat vascular Ehlers–Danlos syndrome (vEDS).

There currently are no approved treatments for vEDS, characterized by thin, translucent skin that is easily bruised, and fragile arteries, muscles, and internal organs.

The company is planning a pivotal clinical trial called PREVEnt  — Prevention of Rupture with Enzastaurin in Vascular Ehlers-Danlos Syndrome — that will evaluate the efficacy of oral AR101 in preventing heart and artery problems in patients with vEDS who carry disease-causing mutations in the COL3A1 gene.

This gene contains instructions for making a protein called type 3 collagen, a key component of the connective tissue that supports, protects, and gives structure to other tissues and organs. When COL3A1 is faulty, as in vEDS, the connective tissue, especially that of the blood vessels, is weak and is easily damaged.

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The clinical trial is designed as a randomized controlled study, meaning that patients will be selected at random to receive either AR101 or a placebo control.

Those interested in finding more information about this study can submit this form.

The new board is chaired by Harry “Hal” Dietz, MD, a professor of genetic medicine at the Johns Hopkins University School of Medicine, in Baltimore. Dietz conducted the initial work supporting the therapeutic potential of AR101 for vEDS.

Four other global advisors, whose work has focused on genetics and cardiovascular disease, will join Dietz on the board. They are Xavier Jeunemaitre, MD, PhD, professor of genetics at the University Paris Descartes, in France; Shaine Morris, MD, medical director of cardiovascular genetics at Texas Children’s Hospital and associate professor at Baylor College of Medicine; Bart Loeys, MD, PhD, professor at the Center for Medical Genetics of the Antwerp University Hospital, in Belgium, and Radboud University Medical Center in Nijmegen, Netherlands; and Peter Byers, MD, professor of medicine, pathology and medical genetics at the University of Washington.

“We are honored to have our chair, Dr. Hal Dietz, as well as our key advisors Dr. Xavier Jeunemaitre, Dr. Shaine Morris, Dr. Bart Loeys, and Dr. Peter Byers join this newly formed SAB [scientific advisory board],” Josh Disbrow, CEO of Aytu, said in a press release.

“Their collective expertise will prove invaluable as we progress toward a single pivotal study of AR101 in VEDS and seek strategies to minimize the clinical risk associated with the trial and hopefully advance this novel potential treatment option so that we can positively impact patients diagnosed with this devastating disease,” he added.

More experts are expected to join the board.

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“We look forward to adding additional key opinion leaders to the SAB to lend their expertise to the development of AR101 in VEDS,” said Disbrow.

AR101 is an experimental small molecule that inhibits a class of proteins called serine/threonine kinases. These kinases serve as signaling molecules in cells. One such kinase is called PKC beta, which is short for protein kinase C beta.

Originally developed as a treatment for a range of cancers, AR101 has been licensed to Aytu following an animal study suggesting abnormal PKC beta signaling in vEDS as a therapeutic target.

In the study, mice carrying mutations comparable in certain respects to those seen in patients with vEDS were found to have increased PKC beta signaling. Treatment with PKC beta inhibitors prevented death due to the spontaneous rupture of the aorta.