Eye On the Cure Research News

May 21, 2020

Nacuity’s Emerging Anti-Oxidative Therapy Moves into Clinical Trial

The oral treatment shows promise for slowing vision loss in people with RP and Usher syndrome, regardless of genetic profile

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Foundation Fighting Blindness · Nacuity’s Emerging Anti-Oxidative Therapy Moves into Clinical Trial

Fort Worth-based Nacuity Pharmaceuticals is launching a Phase 1/2 clinical trial in Australia for NPI-001, an oral treatment designed to slow vision loss in people with retinitis pigmentosa (RP) and RP associated with Usher syndrome. The trial, known as SLO-RP (Safety and Efficacy of NPI-001 Tablets versus PLacebO for Treatment of Retinitis Pigmentosa associated with Usher Syndrome) will enroll at least 48 patients with Usher syndrome and follow them for two years. If results for SLO-RP are favorable, Nacuity plans to launch clinical trials of NPI-001 for people with RP in the US and Australia in 2021.

The Foundation is investing $7.5 million in NPI-001 development through its RD Fund, a venture philanthropy fund for emerging therapies that are approaching, or in, early-stage clinical trials. The Foundation is also providing scientific consulting for the clinical trial and therapy development. Benjamin Yerxa, PhD, chief executive officer at the Foundation, is on Nacuity’s board of directors. Rusty Kelley, PhD, vice president of investments at the Foundation, is a board observer for Nacuity.

“We are grateful to have the Foundation’s support and guidance to advance our emerging treatment into human studies, and hopefully, to patients with retinal diseases who need it,” says Halden Conner, chief executive officer and president at Nacuity. “With the Foundation as a partner, we feel well-poised to achieve our goals of getting NPI-001 across the finish line.”

NPI-001, a GMP-grade of N-acetylcysteine amide (NACA), is an experimental antioxidant drug. Researchers believe that increased oxidative stress plays a major role in retinal degeneration and vision loss across many inherited retinal diseases. Antioxidative therapies are designed to mitigate oxidative stress.

NACA is related to N-acetylcysteine (NAC), an FDA-approved antioxidant treatment for hepatotoxicity caused by acetaminophen overdose. However, NPI-001, which is manufactured using Nacuity’s patented process, is better able to penetrate retinal cells than NAC.

In Foundation-funded lab studies at Johns Hopkins University, NPI-001 reduced oxidative stress and preserved vision.

“Nacuity’s emerging therapy is based on very thoughtful and sound science. Because it is a tablet taken orally, dosing can be easily adjusted,” says Dr. Yerxa. “Furthermore, it is designed to work independent of the mutated gene causing the recipient’s vision loss. That means NPI-001 has the potential to help many people with a wide range of diseases and genetic profiles.”