FDA grants Breakthrough Therapy Designation, RMAT to Aurion's CED cell therapy

Aurion Biotech has received two critical FDA designations for its cell therapy candidate for corneal edema secondary to corneal endothelial disease (CED): Breakthrough Therapy Designation (BTD) and Regenerative Medicine Advanced Therapy (RMAT).

Let’s start with Aurion.

Since launching 3 years ago, the Seattle, Washington-based clinical-stage biotech company has committed to developing a transformational platform of advanced regenerative cell therapies.

The end goal: To restore partial and total vision loss in patients with CED

Explain the company’s cell therapy process.

Healthy cells are taken 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.

• The significance: This minimally invasive procedure can be performed “relatively rapidly, with a recovery period of approximately 2-3 hours for patients,” per Aurion.
  • See here for a visual.

Now talk about this candidate.

As the company’s first clinical candidate, AURN001 is a combination cell therapy designed as a once-administered, intracameral injection comprised of:

• Neltependocel (allogeneic human corneal endothelial cells)
• Y-27632 (an inhibitor of Rho-associated, coiled-coil containing protein kinase [ROCK])

Got it. So what do these two designations mean?

• First up: BTD
  • What it is: This designation is intended to expedite the development and review process for drugs designed to treat a serious or life-threatening disease (ie: CED, in this case)
    • Plus: BTD drugs are eligible for all Fast Track designations
• Next: RMAT
  • What it is: This technology-specific designation is applied to investigational drugs that:
    • Meet the requirements of a regenerative medicine therapy
      • Defined as a cell therapy, therapeutic tissue-engineering product, human cell and tissue product, or any combination of these product
    • Are intended to treat, modify, reverse, or cure a serious condition
    • Have preliminary clinical evidence supporting their potential to address unmet medical needs for certain conditions

Speaking of clinical data … is there any on AURN001 yet?

The FDA has reportedly reviewed clinical data for the AURN001 program, which includes “multiple clinical trials with subjects having completed at least 12 months of follow-up,” from Japan-based research (where the candidate has already received regulatory approval), according to the company.

What else?

Also on the clinical data front,  Aurion reported in April 2024 that it had completed U.S. and Canadian patient dosing in its prospective, multicenter, randomized, double-masked, parallel-arm, dose-ranging phase 1/2 ABA-1, (CLARA) study (NCT06041256).

Click here for our coverage on its first Canadian patient dosing.

To note: The first U.S.-based patient was actually dosed in October 2023.

And how is it being designed?

An estimated 100 adult CED patients in need of surgery are being randomized to receive a single injection of AURN001 in varying doses vs the addition of each element, cells alone, and Y-27632 (ROCK):

• AURN001 (high dose)
• AURN001 (medium dose)
• AURN001 (low dose)
• Neltependocel (high)
• ROCK

For more details, including primary outcome measures, see here.

Note: The participation numbers per dose has not yet been released.

When can we expect data?

Per Clinical Trials, the study is expected to conclude by October 2025 … so we’ll keep you posted on any interim data in the meantime!

Lastly, what’s the significance of these designations?

According to Aurion’s Sterling Chung, Aurion’s vice president of Regulatory Affairs and Quality, “These designations underscore the importance of developing a potential solution for millions of patients around the world who suffer from corneal endothelial diseases.”

He added: “We look forward to working closely with the FDA to expedite the development of our cell therapy.”