Aurora Bio, Inc Announces Clinical Data on Lead Program, AUR01, a Novel Systemic Amyloidosis Imaging Tracer, at the Upcoming 61st American Society of Hematology Annual Meeting and Exposition

Aurora Bio, Inc www.Aurorabioinc.com today 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 1^st 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."

Presentation Highlights:
https://ash.confex.com/ash/2019/webprogram/Paper128850.html

• 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.

ABOUT AURORA BIO, INC

Aurora Bio is a clinical stage biopharmaceutical company developing a platform of immunotherapies and an imaging diagnostic for multiple systemic amyloid diseases, including AL, ATTR and ALECT2 amyloidosis. The diagnostic imaging agent is currently in Phase 1 with a planned Ph 3 to potentially initiate in 2020. The company also has 2 pre-clinical stage therapeutic programs being developed for a broad range of systemic amyloid diseases. The immunotherapies consist of a peptide-fusion antibody, as well as cell therapy utilizing engineered macrophages (CAR-M) to remove amyloid. Currently, there are no approved treatments for 29 out of 30 systemic amyloid diseases While approved therapies in ATTR, and those in development for AL and ATTR, target the pre-cursor protein of individual amyloid diseases, Aurora Bio's goal is to develop immunotherapies that treat across all systemic amyloid diseases. Aurora Bio, Inc is based in South San Francisco, CA www.aurorabioinc.com

Cautionary Statement

Some of the statements included in this press release may be forward-looking statements that involve a number of risks and uncertainties. For those statements, we claim the protection of the safe harbor for forward-looking statements contained in the Private Securities Litigation Reform Act of 1995.