AR101 Named Orphan Drug in Europe With Trial Set to Start Mid-year – Ehlers Danlos News

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by Patricia Inacio PhD | March 7, 2022
The European Commission has granted an orphan drug designation to Aytu BioPharma’s AR101 (enzastaurin), an oral therapy candidate for Ehlers-Danlos syndrome (EDS), including the severe subtype vascular EDS (vEDS).
The commission’s decision follows a positive recommendation by the Committee for Orphan Medicinal Products, an arm of the European Medicines Agency. In the U.S., the Food and Drug Administration granted the therapy a similar status late last year.
Medicines receiving this designation are intended to treat a serious or life-threatening rare disease — affecting up to five in 10,000 people in the European Union. Benefits of the designation include protocol assistance and lower fees, as well as market exclusivity for 10 years once the medicine is approved.
“We are very excited to have been granted orphan designation by the European Commission,” Josh Disbrow, CEO of Aytu, said in a press release. “VEDS is a devastating inherited connective tissue disorder for which there are no approved treatments, and we are pleased to see the regulatory community recognize the urgent need to bring therapies to the people with rare diseases who need them.”
The company is planning its pivotal PREVEnt trial (Prevention of Rupture with Enzastaurin in Vascular Ehlers-Danlos Syndrome), this year. The trial will assess the effectiveness of AR101 in preventing potential triggers of heart and vascular damage in vEDS patients with mutations in the COL3A1 gene. This disease type is typically caused by mutations in COL3A1, which has instructions to produce a part of a protein called type III collagen. Such mutations may lead to tearing of the aorta, the body’s major blood vessel, and of the bowel.
Participants will be randomly assigned to receive AR101 or a placebo. Patients interested in further information can fill out this form.
“We are preparing to initiate our PREVEnt registrational study in the middle of this year, which will evaluate AR101 as a first-in-class treatment for VEDS in patients across study sites in both the U.S. and Europe,” Disbrow said.
AR101, a small molecule delivered orally, was designed to inhibit the PKC beta enzyme. Data from studies using a mouse model of vEDS showed that blocking PKC beta prevented death due to a rupture in the aorta, according to Aytu.
Eli Lilly originally developed enzastaurin to treat cancer. Denovo Biopharma expanded its clinical development for other indications, including vEDS, under the name DB102.
Aytu acquired the rights in April 2021 to develop the treatment for rare childhood-onset or congenital (present at birth) disorders.
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