Aurion Biotech, Inc. reported that the first patients were dosed in the pivotal phase 3 ASTRA study assessing AURN001 in patients with corneal edema secondary to corneal endothelial dysfunction (CED).
Let’s start with Aurion Biotech.
The Seattle, Washington-based clinical-stage biotech company was formed in 2022 with a focus on restoring vision loss among CED patients.
Key to this goal: The development of a proprietary, multi-step, and open cell therapy platform for advanced regenerative cell therapies—including its first investigational candidate:
- AURN001, an allogeneic cell therapy that has already received two FDA designations (more on this later, though).
Any recent news to note?
Aurion had a busy 2025, with an expansion to its executive team, Alcon purchasing a majority interest in the company, and topline data from a phase 1/2 study evaluating AURN001 for CED.
And the year before: Aurion received Pharmaceuticals and Medical Devices Agency (PMDA) approval for AURN-011 in Japan, where they have already begun commercialization.
Remind me about AURN001.
AURN001 is a first-in-class, investigational combination therapy designed to transform treatment for corneal endothelial disease, including Fuchs’ dystrophy.
- It consists of unmodified human corneal endothelial cells (neltependocel) and a rho-kinase inhibitor (ROCK, Y-27632).
Moreover: AURN001 is designed to regenerate corneal clarity and vision through an intracameral injection that has the potential to reduce treatment-related complications and support a fast post-operative recovery.
- To note: The patient recovery period is estimated to be around 2 to 3 hours.
Where is it in the clinical development process?
Already granted Breakthrough Therapy Designation (BTD) and Regenerative Medicine Advanced Therapy (RMAT) by the FDA, AURN001 is currently undergoing a phase 1/2 ABA-1 (CLARA) clinical study.
About the study: AURN001 is being evaluated in three cell doses (low, medium, and high) versus each of its individual components (neltependocel and Y-2763) randomized among patients with corneal edema secondary to CED.
- For more details on the setup and design of this trial (NCT06041256), see here.
Any data so far?
Aurion released topline findings in January 2025.
The crux of that data: The study met its primary endpoint, with the company reporting a dose-dependent response in the three AURN001 arms and statistically significant improvements in the high-dose AURN001 arm at 6 months.
In announcing the findings, Chief Medical Officer Michael Goldstein, MD, shared the company's intent to bring the high dose of AURN001 forward into proposed phase 3 pivotal trials.
Which brings us to the new phase 3 study: ASTRA.
Indeed. The multicenter, randomized, double-masked, prospective study is evaluating patients at 12 sites across the U.S.
- The setup: Will include patients randomized to AURN001 + Y-27632 (i.e., AURN001) or placebo.
- Why: To assess the efficacy and safety of a single injection dose of AURN001 compared with placebo.
And the outcome measures?
The primary endpoint is the percentage of participants who achieve a ≥15-letter improvement (3-line gain) from baseline in best-corrected visual acuity (BCVA) at Month 6.
- Secondary endpoints include: Changes from baseline in non-contact central corneal thickness (CCT) and BCVA at Month 6.
When is the study expected to conclude?
The primary completion date—in other words:when the last participant in a clinical study is examined or receives an intervention to collect final data for the primary outcome measure—is estimated for June 2027.
So what’s the plan moving forward?
Per Aurion Biotech CEO and CSO Arnaud Lacoste, PhD, MBA: ASTRA will be the foundation of AURN001’s BLA submission—and a critical step forward in addressing a very large global unmet patient need.