Analysis of PET images indicated uptake of AUR01 (124I-p5+14) in the heart, kidneys, liver, spleen, pancreas, bone marrow, lung, choroid plexus, pituitary, and adrenal glands of Light chain amyloidosis (AL) patients
Aurora Bio, announced data for the company’s novel PET radiotracer, AUR01, in development for systemic amyloidosis, was presented by Dr. Jonathan Wall, at ASH2019, in Orlando, FL.
Spencer Guthrie, Chief Executive Officer, stated, “We are very excited about the results presented today on 1st 18 patients scanned with the investigational radiotracer, AUR01. We were excited to detect multiple organs in AL, ATTR and ALECT2 patients, and particularly asymptomatic organs. These diseases are truly systemic, yet the ability to determine the extent of organ involvement has been limited by current technologies. 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.”
Morie Gertz, Professor Medicine at the Mayo Clinic, Rochester, stated, “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. 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.”
Isabelle Lousada, CEO of the Amyloidosis Research Consortium, added, “We are thrilled about the potential for a new diagnostic tool for systemic amyloidosis. 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.”
AUR01, a synthetic peptide radiotracer (124I-p5+14), has been shown in preclinical assays and in SPECT and PET imaging of murine systemic amyloidosis, to bind many forms of amyloid, including AL, ATTR, and ALECT2.
No radiotracers approved in the US for non-invasive quantitative measurement of systemic amyloid disease
The presented Ph 1 clinical study included AL, ATTR and ALECT2 patients with biopsy proven amyloidosis and adequate renal function
The primary endpoint includes safety and dosimetry estimation with a secondary endpoint of efficacy
18 patients have been dosed and evaluated.
1 serious adverse event was observed: volume overload due to discontinuation of a diuretic, and was not deemed related to AUR01
All other AEs were mild. Related AEs were due to the use of potassium iodide pills, which are a requirement for radioprotection of the thyroid. These include metallic taste, rash, nausea and upset stomach.
Retention in the heart, kidneys, liver, spleen, pancreas, bone marrow, lung, and adrenal gland of AL patients
Cardiac uptake of the radiotracer was observed in 83% of AL patients and 100% of ATTR patients
In 50% of the patients of both AL and ATTR who had cardiac uptake, there were no clinical symptoms of cardiac disease and cardiac biomarkers were normal
Kidney, spleen and liver retention was observed in 67%, 42%, and 42% of AL patients, respectively
Nerve, tendons/ligaments, and lung retention was observed in 75%, 75% and 100% of ATTR patients, respectively
Retention of the radiotracer observed in the kidney, liver, spleen and adrenal glands in patients with ALECT2
Conclusion: Initial PET/CT image data indicate that 124I-p5+14 can provide quantitative detection of systemic amyloidosis in multiple organ systems and may have general utility in detecting and monitoring amyloid burden in many forms of amyloidosis.
Source: Company Press Release