Aurion Biotech, Inc. recently reported topline data from the phase 1/2 ABA-1 (CLARA) study evaluating the use of AURN001, an allogeneic cell therapy, for the treatment of corneal edema secondary to corneal endothelial dysfunction (CED).
First, an Aurion refresh.
Headquartered in Seattle, Washington, Aurion is a clinical-stage biotech company working to restore partial and total vision loss in patients diagnosed with CED.
Key to this: Its platform of advanced regenerative cell therapies.
Talk about how its platform works.
The company’s proprietary, multi-step, and open cell therapy process is designed to culture and regenerate the healthy cells from a single donor cornea in one minimally-invasive procedure.
- These regenerated cells are then manufactured to produce a therapeutic treatment for +1,000 recipient eyes.
Per the company: This procedure is intended to be performed rapidly and with a patient recovery period estimated around 2 to 3 hours.
I’m having a hard time picturing this …
Check out the video below for a visual.
Now to this cell therapy.
Aurion’s first clinical candidate is a combination cell therapy designed as a one-time intracameral injection administered to the anterior chamber of the eye. It’s comprised of:
- Neltependocel (allogeneic human corneal endothelial cells)
- Y-27632 (inhibitor of Rho-associated, coiled-coil containing protein kinase [ROCK])
Didn’t it receive regulatory designations last year?
Indeed it did. Two, in fact.
Back in June 2024, the FDA granted the following key designations to AURN001:
- Breakthrough Therapy Designation
- Regenerative Medicine Advanced Therapy
See here for details on what these designations mean for the candidate—including the major significance for its clinical development and regulatory future.
Next up: the study.
What it is: A prospective, multicenter, randomized, double-masked, parallel-arm, dose-ranging phase 1/2 ABA-1 (NCT06041256).
The participants: 97 patients (aged 18-99; mean age = 71.4; 55% female) from both U.S. and Canadian clinical sites.
- Criteria: Diagnosed with corneal edema secondary to CED and requiring surgery(full- or partial-thickness endothelial keratoplasty)
Baseline numbers: Across all arms, the mean best-corrected visual acuity (BCVA) was 53.5 letters (as per the Early Treatment Diabetic Retinopathy Standard [ETDRS] visual acuity test [20/85]), while the central corneal thickness (CCT) was 676.6 microns (μm).
And the randomized setup?
Participants were randomized to the following arms:
- AURN001: 1.0 × 106 neltependocel + 100 μm Y-27632 (high cell dose)
- AURN001: 5.0 × 105 neltependocel + 100 μm Y-27632 (medium cell dose)
- AURN001: 2.5 × 105 neltependocel + 100 μm Y-27632 (low cell dose)
- 100 μM Y-27632
- 1.0 × 106 neltependocel
What was measured?
The primary endpoint was the number of responders with a >15-letter improvement (>3-line gain) from baseline in BCVA at six months.
As for secondary endpoints, these included:
- Change from baseline in BCVA and in CCT at six months
- Safety and tolerability (of AURN001)
And the findings?
We’ll start with the endpoints:
Primary: The company reported that “a dose-dependent response was observed in the three AURN001 arms,” including statistically significant improvements noted in the high-dose AURN001 arm.
- The numbers:
- 50% of responders for the AURN001 arm (p = 0.020) versus
- 14.3% of responders for the Y-27631-only arm
Secondary: A statistically significant improvement was observed for the high-dose AURN001 arm (p = 0.002) versus the Y-27631-only arm—plus, a dose response was noted in the three AURN001 arms.
- As for CCT change at six months: Aurion also reported a statistically significant improvement for the high-dose AURN01 arm (p = 0.012) compared to the Y-27631-only arm.
And beyond the targeted endpoints?
Improvement in patient-reported quality of life—as evaluated via the Visual Function Questionnaire (VFQ-25)—was also observed.
- Notably: The greatest benefit was seen in the high-dose AURN001 arm, according to the company.
Let’s talk tolerance.
In general, all treatment arms were found to be well tolerated with favorable safety profiles.
As for adverse events (AEs): There was no notable dose relationship observed in frequency of the types of AEs.
While no ocular serious AEs were reported, the most common ocular treatment-emergent AEs (TEAE) included:
- Ocular hypertension (9.3%)
- Conjunctival hemorrhage (5.2%)
- Eye pain (4.1%)
- Cystoid macular edema (3.1%)
As for the most frequently-reported non-ocular TEAE, 3.1% were attributed to COVID-19.
What did Aurion have to say about the data?
President and Chief Medical Officer Michael Goldstein, MD, MBA, expressed the company’s positive outlook on the performance of the high-dose AURN001 at six months.
“Based on these findings, combined with the generally favorable safety profile in the CLARA trial, we look forward to bringing the high dose of AURN001 forward into our proposed phase 3 pivotal trials,” he stated.
In the near future: The company is looking to present full data from the CLARA trial at medical conferences later this year.