NP-1
Oral dosage treatment for retinitis pigmentosa in Usher syndrome
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.
For the randomized, placebo-controlled, double-masked, multicenter, Phase 1/2 clinical trial of Nacuity’s proprietary NPI-001 tablets in patients with RP associated with Usher syndrome (NCT04355689), a 24-month efficacy assessment achieved clinical Proof of Concept.
U.S. FDA has granted Fast Track and Orphan Drug designations. An eligible new drug application would result in 7 years of U.S. FDA regulatory exclusivity.
NP-2
Intravitreal implant for delay of cataract progression
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 by early 2026.
NP-3
Oral dosage for treatment for nephropathic cystinosis
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. An eligible new drug application would result in Nacuity receiving seven years of U.S. FDA regulatory exclusivity and potentially a priority review voucher.
NP-4
Intravitreal Implant for AMD and Stargardt Disease
A recent study18 was conducted to discern relative antioxidant effects of Nacuity’s antioxidants compared to N-acetylcysteine (NAC) in various retinal cell cultures. This study revealed that NPI-001, NPI-002 and NAC exhibited varying degrees of antioxidant activity, protecting cultured rat retinal cells from a variety of stressors which were designed to mimic aspects of the pathology of different retinal diseases. A general rank order of activity was observed: NPI-001 ≥ NPI-002 > NAC. Studies are planned to further evaluate NPI-001 and NPI-002 as potential treatments for AMD and/or Stargardt disease.
NP-5
Oral dosage for treatment of a rare neurological disease
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. The first clinical trial evaluating Nacuity’s NPI-001 for the treatment of Hereditary Cystatin C Amyloid Angiopathy (HCCAA) in Iceland, is ongoing. Orphan and rare pediatric disease designations are in progress.
