Aytu BioPharma Announces AR101 Granted Orphan Designation in Europe

Posted March 9, 2022

The specialty pharmaceutical company, Aytu BioPharma, Inc. (Nasdaq: AYTU), announced this week that the European Commission granted orphan designation to AR101 (enzastaurin), a PKCβ inhibitor, for the treatment of vascular Ehlers-Danlos syndrome (vEDS). ⁠

This clearance will enable the company to commence its pivotal PREVEnt clinical trial in mid-2022 in patients across study sites in Europe and in the US, investigating the ability of enzastaurin to reduce the severe complications of vEDS. There are currently no FDA-approved therapies for vEDS. ⁠

What is the PREVEnt trial? ⁠

The PREVEnt Trial will see Aytu BioPharma, Inc. (Nasdaq: AYTU) enroll approximately 260 COL3A1-positive vEDS patients. The study will randomize patients 1:1, with half receiving enzastaurin 500 mg once daily along with the standard of care and half receiving a placebo once daily along with the standard of care. The study’s primary endpoint is a reduction in fatal and non-fatal arterial events (ruptures, dissections, pseudo-aneurysms). ⁠

How can people living with vEDS get involved? ⁠

The Ehlers-Danlos Society is delighted to be supporting this study, which gives much-needed hope for those living with vEDS and their families, as part of the key stakeholder group. The Ehlers-Danlos Society will help to facilitate collaboration through the vEDS working group of The International Consortium, and with the enrolment of study participants through our global registry. ⁠

We will update our community with updates on the trial and how to get involved through the global registry when enrollment begins. Join our CONNECT newsletter to stay up to date.

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