Aurion Biotech Announces IOTA Cell Therapy Trial

Aurion Biotech, whose mission is to cure blindness by developing a differentiated platform of advanced therapies to treat ocular diseases, today announced preliminary findings from its IOTA trial, conducted in November 2020 and in May of this year, at the Clínica Quesada in San Salvador, El Salvador.

In the IOTA trial, 50 patients diagnosed with corneal endothelial disease were treated with a transformational cell therapy procedure first developed by Professor Shigeru Kinoshita and colleagues at Kyoto Prefecture University of Medicine (KPUM). The IOTA trial is the first corneal endothelial cell therapy procedure to be performed successfully outside of Japan, with Drs. Gabriel and Rodrigo Quesada, and U.S. surgeons Drs. Edward Holland, Elizabeth Yeu, John Berdahl, Matt Giegengack, John Vukich and Kevin Waltz participating as investigators.

"This is a landmark moment for the treatment of corneal endothelial disease," declared Dr. Holland, chief medical advisor and medical advisory board chair at Aurion Biotech. "We achieved what we set out to accomplish: in IOTA-Part 1, we were able to treat 16 patients from a single donor. In IOTA-Part 2, we treated 34 patients from a single donor. Equally important: we confirmed that this procedure is accessible for surgeons and minimally invasive for patients. The initial readout from this proof-of-concept trial is promising: patients have demonstrated improvements in visual acuity and central corneal thickness. We will continue to monitor these patients over time, to assess the safety and efficacy of the cell therapy treatment."

The IOTA trial was conducted as a proof of concept of previous studies of the cell therapy procedure developed by Professor Kinoshita and his KPUM colleagues.

"The IOTA trial has given us invaluable clinical insights and confirms that this procedure can restore vision to patients suffering from blindness resulting from corneal endothelial disease," said Greg Kunst, Aurion Biotech's chief executive officer. "Until Professor Kinoshita's astonishing innovations in corneal endothelial cell therapy, vision loss caused by corneal edema has remained largely underserved. That's because of a chronic undersupply of donor corneas and current standards of care that involve exceptionally complex procedures. Because we have acquired this intellectual property, we look forward to advancing this cell therapy through regulatory approval in the US and elsewhere, to restore vision to patients throughout the world."

Patients in the IOTA trial will be assessed periodically over twelve months according to protocol guidelines and beyond conclusion of the trial, to monitor safety and efficacy of treatment. Aurion Biotech intends to publish key findings in the future, in order to contribute to a greater medical and scientific understanding of corneal endothelial cell therapy.

About Corneal Endothelial Disease

Corneal edema is a serious, sight-threatening and debilitating condition affecting millions of people throughout the world. When corneal endothelial cells die or degrade, they do not regenerate. Ultimately, corneal endothelial cell loss can cause corneal edema and loss of vision. Although corneal transplants are effective, there are disadvantages with these procedures (DMEK/DSAEK¹):

• Limited donor organ supply. Transplants require a supply of donor corneas in a 1:1 ratio (one healthy donor cornea to treat each diseased one). However, it's estimated that there is only one donor cornea available for every 70 diseased eyes.²
• Complex surgical procedure. Although there are approximately 20,000 ophthalmologists in the US³, and approximately 1,500 corneal specialists, it's estimated that fewer than 300 actively perform the DMEK/DSAEK procedures. While DMEK has excellent outcomes, it's estimated that up to 25% of patients undergoing DSAEK may require a regraft procedure⁴.

About Corneal Endothelial Cell Therapy

The innovation from Professor Kinoshita/KPUM was to successfully propagate corneal endothelial cells in vitro, in order to inject healthy cells into the cornea, reduce corneal edema and restore vision. The safety, tolerability, and efficacy of this cell therapy is supported by clinical trials conducted in Japan. Compared to current standard of care, corneal endothelial cell therapy has several potential advantages:

• Abundant supply of corneal endothelial cells. At present, the endothelial cells from one donor can replicate to treat up to 100 eyes.
• Straightforward procedure to perform. The cell therapy procedure can be performed relatively quickly and is less complex than DMEK/DSAEK procedures.
• Patient-friendly procedure. Post-operative recovery for cell therapy is several hours, as compared to several days for DSAEK/DMEK procedures.

About Aurion Biotech

Based in Seattle, Boston and Tokyo, Aurion Biotech is a clinical stage biotech company. Our mission is to cure leading forms of blindness and transform the lives of millions of patients, by developing a platform of advanced therapies to treat ocular diseases. Our first candidate is for the treatment of corneal edema, and one of the first clinically validated cell therapies for corneal care. Healthy cells from a donor cornea are cultured in a novel, multi-step, proprietary and patented process. Cells from a single donor can be used to treat more than 100 recipient eyes. In clinical trials in Japan, patients have experienced significant and durable improvements in key measures of corneal health: visual acuity, corneal endothelial cell density and corneal thickness. The Aurion Biotech team is preparing for clinical trials in the U.S. To learn more about Aurion Biotech, a division of CorneaGen Inc., visit www.aurionbiotech.com

¹ Descemets Membrane Endothelial Keratoplasty, Descemet Stripping Automated Endothelial Keratoplasty
² Source: JAMA
³ https://www.aao.org/newsroom/eye-health-statistics#ophthalmologists
⁴ Source: EBAA 2020 Annual Report