FDA Names AR101 Orphan Drug, Clears Way for Vascular EDS Trial

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by Vanda Pinto PhD |

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AR101 (enzastaurin), an orally available small molecule being developed to treat vascular Ehlers-Danlos syndrome (vEDS) by Aytu BioPharma, has been designated an orphan drug by the U.S. Food and Drug Administration (FDA).

A request to launch a pivotal clinical trial in patients was also cleared by the FDA.

Orphan drug status is given to accelerate the clinical development of investigational treatments for rare diseases, those that affect less than 200,000 people in the U.S. Benefits of the designation include FDA application fee exemptions, tax credits for clinical studies, and seven years of marketing exclusivity in the U.S. if approval is granted.

“Receiving Orphan Drug designation for AR101 underscores the unmet need for patients with VEDS, for which there are currently no FDA-approved treatments,” Josh Disbrow, CEO of Aytu, said in a press release.

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Aytu plans to launch the pivotal PREVEnt — Prevention of Rupture with Enzastaurin in Vascular Ehlers-Danlos Syndrome — trial next year. The study will investigate the efficacy of AR101 in preventing incidents that can damage the heart and arteries in vascular EDS patients who carry mutations in the COL3A1 gene.

“We’re now positioned to start the PREVEnt Trial in the first half of 2022 and look forward to taking that next step for the benefit of these patients in need of a new treatment,” Disbrow said in a separate release announcing the U.S. agency’s clearance of its request.

vEDS is typically caused by mutations in COL3A1, which provides information to make a part of a structural protein called type 3 collagen. In people with vEDS, the lack of working collagen can lead to complications that include tearing of the aorta, the body’s major blood vessel, and of the bowel.

Enrolled patients will be randomly assigned to either AR101 or to a placebo as a control group. Further trial details can be provided to those interested using this form.

AR101 inhibits an enzyme known as PKC beta, which is important for many cellular processes. Preclinical studies in a mouse model of vEDS found that blocking PKC beta prevented death due to aortic rupture, Aytu reported on the therapy’s webpage.

Enzastaurin was initially developed by Eli Lilly to treat various cancers. Denovo Biopharma, which holds worldwide rights to enzastaurin and further developed it under the investigative name DB102, had licensed the compound to Rumpus Therapeutics for certain medical indications, including vEDS.

Aytu acquired rights to develop DB102 for rare childhood onset or congenital (present at birth) disorders beyond cancers in April.