ONC201 for the Treatment of Vascular Aneurysms


A repurposed therapeutic for the preventative and treatment of aneurysms.

Key Benefits

  • A repurposed cancer drug that targets cells known to be damaged during the progression of an aneurysm.
  • The drug has been tested within an animal model of aortic aneurysms, where it prevented the severe complications of the disease and protected the vascular structures.
  • Potential to be a non-invasive medicine to reduce the high mortality rate associated with aneurysms.

Market Summary

An aortic aneurysm is a bulge in the aorta wall, which carries blood from the heart to the rest of the body. Annually aortic aneurysms cause more than 9,000 deaths, about 60% affecting men. Smoking is the condition's leading risk factor and responsible for 75% of all aortic aneurysms. Unfortunately, current treatments are limited, with most patients quickly succumbing to the disease. The global aortic aneurysm market is expected to expand due to the increasing prevalence of atherosclerosis and the rising geriatric population. The described technology has the potential to become a new pharmacologic treatment for the condition, potentially reducing the need for more invasive surgical treatment modalities.

Technical Summary

Researchers at Emory are investigating the use of a drug called ONC201 used in clinical trials to treat cancer but are repurposing it to prevent the occurrence and/or worsening of aneurysms. The drug functions by targeting the mitochondrial protease Clpp. The inventors treated mice with experimentally induced aneurysms with the drug and showed it decreases aneurysm formation and preserves vascular structures.

Developmental Stage

Proof of concept animal data.

Patent Information

App Type Country Serial No. Patent No. File Date Issued Date Patent Status
PCT PCT PCT/US2023/037103   11/9/2023   Pending
Tech ID: 23006
Published: 4/5/2023