Aurion Biotech announced that the first patient has been dosed in Canada as part of the phase 1/2 clinical trial for AURN001, its cell therapy candidate intended to treat corneal edema secondary to corneal endothelial dysfunction (CED).
Refresh me on the company.
Founded in 2021 and based in Seattle, Washington, the clinical-stage biotech company is developing a transformational platform of advanced regenerative cell therapies with a mission to restore sight.
Its first candidate: a therapy for CED (AURN001).
Let’s discuss this cell therapy process.
The company uses healthy cells from a donor cornea to regenerate in a multi-step, proprietary and open process by culturing the cells from a single donor.
The result: These cells are then manufactured to produce treatment for 100+ recipients' eyes, according to Aurion.
Its uniqueness: This minimally invasive procedure can be performed “relatively rapidly, with a recovery period of approximately 2-3 hours for patients.”
I need a visual of this.
Watch Aurion’s video below to see how this procedure works.
Alrighty, talk about AURN001.
This first candidate for Aurion is a combination cell therapy consisting of:
- Neltependocel (allogeneic human corneal endothelial cells)
- Y-27632 (an inhibitor of Rho-associated, coiled-coil containing protein kinase [ROCK])
It’s designed to be administered via a one-time, intracameral injection into the eye.
Now this trial.
The prospective, multicenter, randomized, double-masked, parallel-arm, dose-ranging phase 1/2 ABA-1, CLARA) study (NCT06041256) is enrolling an estimated 100 participants (aged 18-99) diagnosed with corneal edema secondary to CED who require surgery (full- or partial-thickness endothelial keratoplasty).
Other criteria includes:
- Best-corrected visual acuity (BCVA) between 65 Early Treatment of Diabetic Retinopathy Study (ETDRS) and 5 ETDRS letters
Note: 65 ETDRS letters is equivocal to 0.4 LogMAR or approximate 20/50 Snellen equivalent, while 5 ETDRS letters would be 1.6 LogMAR or approximate 20/800 Snellen equivalent
How is the study designed?
The trial is evaluating three differing doses—low, medium, and high—of neltependocel used in combo with Y-27632,.
Participants will be randomized to receive a single injection of AURN001 vs the contribution of each element, cells alone, and Y-27632 (ROCK) alone.
- AURN001 (high dose)
- AURN001 (medium dose)
- AURN001 (low dose)
- Neltependocel (high)
- Rho-associated protein kinase (ROCK)
And what’s being measured?
The primary outcome measure is the number of participants who achieve 15 letters or greater improvement (3-line gain) from baseline in BCVA at Month 6.
And where is this being conducted?
Across clinical sites in both the U.S. and Canada.
Note: The first U.S. patient from this trial was actually dosed back in October 2023.
And when can we expect data?
Per Clinical Trials, the study is expected to conclude by October 2025. In the meantime, however, stay tuned for interim data!