Aurion Biotech Receives Breakthrough Therapy Designation and Regenerative Medicine Advanced Therapy Designation for Its Drug Candidate AURN001
19 Junio 2024 - 7:00AM
Business Wire
First allogeneic cell therapy to receive both
FDA designations for the treatment of corneal edema secondary to
corneal endothelial disease
Aurion Biotech, whose mission is to restore vision to millions
of patients with life-changing regenerative therapies, today
announced that the U.S. Food and Drug Administration (FDA) has
granted both Breakthrough Therapy Designation (BTD) and
Regenerative Medicine Advanced Therapy (RMAT) Designation for
AURN001, the Company’s allogeneic cell therapy candidate for the
treatment of corneal edema secondary to corneal endothelial
disease.
Breakthrough Therapy Designation is a process designed to
expedite the development and review of drugs that are intended to
treat a serious condition and preliminary clinical evidence
indicates that the drug may demonstrate substantial improvement
over available therapy on a clinically significant endpoint(s).
RMAT is part of the 21st Century Cures Act1. An investigational
drug is eligible for RMAT designation if: it meets the definition
of regenerative medicine therapy; is intended to treat, modify,
reverse, or cure a serious condition; and preliminary clinical
evidence indicates that the regenerative medicine therapy has the
potential to address unmet medical needs for such conditions.
Sponsor companies receiving BTD and RMAT Designation can benefit
from increased interactions with the FDA with the goal of
expediting drug development. The dual designations granted to
Aurion Biotech are based on the criteria stated above, the unmet
medical need among patients with corneal endothelial disease, and
FDA’s review of clinical data from the AURN001 program, which
includes multiple clinical trials with subjects having completed at
least 12 months of follow-up.
“Aurion Biotech is honored to receive the Breakthrough Therapy
Designation and Regenerative Medicine Advanced Therapy Designation
for AURN001,” said Sterling Chung, Vice President of Regulatory
Affairs and Quality at Aurion Biotech. “These designations
underscore the importance of developing a potential solution for
millions of patients around the world who suffer from corneal
endothelial diseases. We look forward to working closely with the
FDA to expedite the development of our cell therapy.”
This news comes on the heels of Aurion’s announcement that it
has completed enrollment and dosing of its Phase 1 / 2 CLARA trial
in the U.S. and Canada2. The CLARA clinical trial is a prospective,
multi-center, randomized, double-masked, parallel-arm cell
dose-ranging study in subjects with corneal edema secondary to
corneal endothelial dysfunction. The study will evaluate three
different doses of neltependocel used in combination with Y-27632.
Ninety-seven subjects have been randomized at US & Canadian
sites. The CLARA trial is designed to assess the safety,
tolerability, and efficacy of AURN001 for the treatment of corneal
edema secondary to corneal endothelial dysfunction. The primary
endpoint is the percentage of subjects who gain three (3) lines of
vision at six (6) months.
The company has already received regulatory approval in
Japan.
“The BTD and RMAT designations are important milestones for
people living with corneal endothelial disease who need better,
less burdensome, more accessible treatment options,” said Greg
Kunst, chief executive officer of Aurion. “I thank the Aurion team
for their hard work and dedication, and I’m grateful for the
essential contributions of clinical trial patients and their
ophthalmologists - all of whom have helped to make today’s news a
reality.”
About Corneal Endothelial Dysfunction
Corneal edema secondary to endothelial dysfunction is a
sight-threatening and debilitating condition affecting millions of
people throughout the world. When corneal endothelial cells die or
degrade, they do not regenerate. If left untreated, corneal
endothelial cell loss can cause corneal edema (swelling) and loss
of vision. Although corneal transplants are effective, there are
disadvantages with these procedures – PKP/DMEK/DSAEK – including
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), yet it’s estimated that there is only one donor
cornea available for every 70 diseased eyes. In addition,
post-operative recovery for corneal transplant patients requires
that they lie flat on their backs for up to three days, in order
for the transplant to adhere to the corneal stroma.
About Aurion Biotech
Aurion Biotech is a clinical-stage biotech company, whose
mission is to restore vision to millions of patients with
life-changing regenerative therapies. It received the prestigious
Prix Galien award for best start-up in biotech. Its first candidate
is for the treatment of corneal edema secondary to corneal
endothelial disease, and the first clinically validated cell
therapy for corneal care, having received regulatory approval in
Japan. The Company has completed enrollment and dosing of its Phase
1 / 2 clinical trial in the U.S. and Canada. Privately held, Aurion
Biotech is backed by Deerfield, Alcon, Petrichor, Flying L
Partners, Falcon Vision / KKR, and Visionary Ventures. To learn
more, visit www.aurionbiotech.com
1
https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/regenerative-medicine-advanced-therapy-designation
2
https://aurionbiotech.com/aurion-biotech-announces-completion-of-enrollment-in-phase-1-2-clinical-trial
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version on businesswire.com: https://www.businesswire.com/news/home/20240619102413/en/
Judith McGarry (corporate communications) 415-971-2900
judith.mcgarry@aurionbiotech.com
Beth Keshishian (biotech & business media)
917-912-7195 beth@bethkeshishian.com
Michele Gray (ophthalmology media) 917-449-9250
michele@mgraycommunications.com