Companies To Watch: Aurion Biotech
By Ben Comer, Chief Editor, Life Science Leader
Developing an allogeneic cell therapy for eye diseases at scale for the masses
Snapshot
Privately-held Aurion Biotech is developing an allogeneic combination cell therapy — neltependocel, which is allogeneic human corneal endothelial cells, combined with Y-27632, an inhibitor of Rho-associated, coiled-coil containing protein kinase — for the treatment of corneal edema secondary to corneal endothelial dysfunction. The company is currently testing AURN001 in a roughly 100-person Phase 1/2 study. AURN001 is Aurion’s sole clinical candidate; in June, AURN001 received the FDA’s Breakthrough Therapy Designation and Regenerative Medicine Advanced Therapy Designation, an exceedingly rare achievement.
What’s At Stake
Could AURN001 become the first allogeneic cell therapy capable of scaling up to treat over a million patients with eye diseases? That is what Aurion Biotech founding CEO Greg Kunst is gunning for, and a lot is riding on Phase 1/2 data from a multicenter dose-ranging study in the U.S. and Canada, with results slated for release early next year.
Kunst, an entrepreneur and experienced executive in ophthalmology (formerly a global marketing director at Alcon, and a VP of global marketing and access at Glaukos Corporation), hopes to improve upon the standard of care — corneal transplant — with a one-time injected combination cell therapy capable of being administered by a wide variety of ophthalmologists and cataract surgeons, not just the approximately 300 U.S. corneal specialists that perform corneal transplants. Getting a much less invasive treatment option into the hands of many more ophthalmologists could “really make a dent in addressing corneal endothelial disease at the earliest onset of disease,” says Kunst.
As depicted in a set of simulated slide images on Aurion’s website (scroll down), corneal endothelial disease progression ultimately leads to blindness. Corneal transplant, and transplanting healthy endothelium into the cornea, are effective procedures but limited by the need for cornea donors; the number of specialists available to perform the transplant; and recovery time — three days lying still on your back. AURN001, by comparison, is injected once, followed by a three-hour lie down time, after which patients can resume normal activities (it takes longer for vision to return). Kunst says Aurion can create up to 1,000 doses of AURN001 per donor.
AURN001 is a treatment invented by Shigeru Kinoshita, MD, Ph.D., at Kyoto Prefecture University of Medicine. In 2020, Kinoshita published data from a five-year human study in ophthalmology, which led to a licensing deal and the formation of Aurion, in 2021 (Kinoshita sits on Aurion’s medical advisory board). AURN001 was approved in March 2023 in Japan, under the brand name Vyznova, for the treatment of bullous keratopathy of the cornea.
A mass-produced safe and effective allogeneic cell therapy — for any indication — would be no small accomplishment. One of the key difficulties, notes Kunst, is securing sufficient CMC manufacturing, an area where “a lot of cell therapy companies die on the vine.” The most difficult task, per Kunst, is working with CDMOs to get the process right. Aurion is working with “a number of CDMOs around the world” in anticipation of commercial scale up, and is “the majority of the way there, but not all the way there yet.”
Vital Statistics
Employees: ~65
Headquarters: Seattle, WA (additional offices in Cambridge, MA and Tokyo, Japan)
Finances: $120 million Series C (first Aurion financing following Series A and B rounds under the auspices of parent company CorneaGen; Aurion now operates as an independent spinoff).
Significant Investors: Deerfield (lead investor), Alcon, KKR, Petrichor, Flying-L-Partners, Visionary Ventures
Latest Updates:
June 2024: Received FDA Breakthrough Therapy Designation and Regenerative Medicine Advanced Therapy designation
April 2024: Completed enrollment and dosing in Phase 1/2 clinical trial (CLARA) of AURN001 for the treatment of corneal edema secondary to corneal endothelial dysfunction.