Nacuity’s development engine is focused on time- and capital-efficient clinical programs involving orphan and rare pediatric disease designations with the potential for priority review vouchers.

Pipeline graphic

Nacuity’s development engine is focused on time- and capital-efficient clinical programs involving orphan and rare pediatric disease designations with the potential for priority review vouchers.

Pipeline graphic June 2023

In preclinical animal studies, NPI-001 preserved photoreceptor cells and functionality. A Phase 1 clinical trial of Nacuity’s GMP-grade NPI-001 solution in healthy volunteers was completed with no serious adverse events.

A randomized, placebo-controlled, double-masked, multicenter, Phase 1/2 clinical trial of Nacuity’s proprietary NPI-001 tablets, the SLO-RP study, is ongoing in patients with RP associated with Usher syndrome (NCT04355689). The protocol has been amended to allow continued treatment for interested participants while the core study is ongoing.

This product has been granted orphan drug designation, thus, an eligible new drug application would result in 7 years of U.S. FDA regulatory exclusivity for Nacuity.


Nacuity’s proprietary intravitreal implant contains Nacuity’s small molecule antioxidant, NPI-002, intended to slow cataract progression. It is delivered via a proprietary injector. Nacuity has completed preclinical proof of concept and toxicology testing.

Currently, Nacuity is recruiting patients undergoing vitrectomy in a randomized, controlled, masked, Phase 1/2 clinical study to assess safety and efficacy of its proprietary intravitreal implant to delay cataract progression (NCT05026632). Nacuity expects to report proof of concept results in 2024.


Nacuity has developed an oral dosage form containing a small molecule antioxidant for the treatment of nephropathic cystinosis. In vitro POC, chronic oral toxicology studies and a Phase 1 clinical trial in healthy volunteers have been successfully completed. A clinical proof of concept trial is ongoing (NCT05994534). This program has been granted orphan and rare pediatric disease designations; thus, an eligible new drug application would result in Nacuity receiving seven years of U.S. FDA regulatory exclusivity and potentially a priority review voucher.


Nacuity is partnering to develop a proprietary ophthalmic insert containing a small molecule antioxidant for the treatment of the ocular effects of cystinosis. In vivo preclinical studies with prototype insert reveal that significant levels of drug can be delivered to the aqueous humor by wearing the ophthalmic insert under the eyelid. The company has successfully completed a U.S. FDA PreIND meeting. This program has been granted orphan and rare pediatric disease designations; thus, an eligible new drug application would result in Nacuity receiving seven years of U.S. FDA regulatory exclusivity and potentially a priority review voucher.


Nacuity has developed an oral dosage form containing a small molecule antioxidant for the treatment of a rare neurological disease. Chronic oral toxicology studies and a Phase 1 clinical trial in healthy volunteers have been successfully completed. A clinical development program is being planned. This program has been granted orphan and rare pediatric disease designations; thus, an eligible new drug application would result in Nacuity receiving seven years of U.S. FDA regulatory exclusivity and potentially a priority review voucher.