Aurora Bio revealed data from the use of its AUR01 novel PET radiotracer for systemic amyloidosis that showed strong results in uptake and retention, but also experienced some adverse events.
The synthetic peptide radiotracer is designed to bind many forms of amyloid, including AL, ATTR and ALECT2. Currently, no radiotracers are approved in the U.S. for non-invasive quantitative measurement of systemic amyloid disease, according to a news release. Amyloid is an abnormal protein that is produced in the bone marrow, according to Mayo Clinic. It can be deposited in any tissue or organ and frequently the heart, kidneys, liver, spleen, nervous system and digestive tract.
A clinical study of 18 patients included AL, ATTR and ALECT2 patients with biopsy-proven amyloidosis and adequate renal function. The trial’s primary endpoint included safety and dosimetry estimation, with a secondary endpoint of efficacy.
The AUR01 demonstrated retention in the heart, kidneys, liver, spleen, pancreas, bone marrow, lung and adrenal gland of AL patients. Cardiac uptake of the device was observed in 83% of AL patients and 100% of ATTR patients. No clinical symptoms of cardiac disease appeared in 50% of the AL and ATTR patients with cardiac uptake, while cardiac biomarkers were normal.
Aurora observed kidney, spleen and liver retention in 67%, 42% and 42%, respectively, in AL patients. Lung retention was observed in all ATTR patients and nerve and tendon/ligament retention was observed in 75% of ATTR patients.
There was one serious adverse event observed during the trial, with a volume overload due to discontinuation of a diuretic. However, it was not deemed related to the AUR01. Other adverse events were observed but were mild. The use of potassium iodide pills, a requirement for radioprotection of the thyroid, caused the adverse events, which resulted in symptoms such as metallic taste, rash, nausea and upset stomach, the company reported.
Aurora concluded that the data indicates the ability to provide quantitative detection of systemic amyloidosis in multiple organ systems, while also having general utility in detecting and monitoring amyloid burden in many forms of amyloidosis.
“These diseases are truly systemic, yet the ability to determine the extent of organ involvement has been limited by current technologies,” Aurora CEO Spencer Guthrie said in the release. “The data also provide hope that we may be able to detect asymptomatic patients and patients early in their disease course. We are actively planning further studies to determine the ability of AUR01 to measure chance in amyloid over time, either progression or improvement post-therapy. We continue to believe this compound has the potential to change the way systemic amyloidosis is diagnosed and monitored and holds great potential to provide for efficient drug development for new targeted therapeutics, including Aurora Bio’s own therapeutic programs.”
“The ability to detect and quantify the full amyloid burden in patients could dramatically change the way we diagnose, manage and treat systemic amyloidosis patients,” added Mayo Clinic professor of medicine Morie Gertz. “One of the greatest challenges is diagnosing patients early and AUR01 holds promise in detecting amyloid early so we can intervene when treatments are most effective. Detecting asymptomatic disease could open a new avenue for developing treatments before the organ damage is severe.”
“We are thrilled about the potential for a new diagnostic tool for systemic amyloidosis,” said Amyloidosis Research Consortium CEO Isabelle Lousada. “The average patient journey to get a proper diagnosis can take years, and the clinical picture about what organs are affected is often incomplete. Now that we have entered a new stage with approved drugs for ATTR and those in development for AL, it is even more important that patients get an early diagnosis.”