N-acetylcysteine amide (NPI 001) / Nacuity Pharma - LARVOL DELTA

Nacuity Pharmaceuticals Granted U.S. FDA Fast Track Designation for NPI-001 (N-acetylcysteine amide) Tablets for the Treatment of Retinitis Pigmentosa (GlobeNewswire) - "Nacuity Pharmaceuticals...today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to NPI-001 (N-acetylcysteine amide) tablets, Nacuity’s proprietary investigational therapy for the treatment of patients with retinitis pigmentosa (RP)."

Fast track • Retinitis Pigmentosa

INCA: PK and PD Study of NPI-001 and Cysteamine Bitartrate (clinicaltrials.gov) - P1/2 | N=12 | Recruiting | Sponsor: Nacuity Pharmaceuticals, Inc. | Trial completion date: Jun 2024 ➔ Jun 2025 | Trial primary completion date: May 2024 ➔ Apr 2025

Trial completion date • Trial primary completion date

Safety and Efficacy of NPI-001 Tablets for RP Associated With Usher Syndrome (clinicaltrials.gov) - P1/2 | N=49 | Active, not recruiting | Sponsor: Nacuity Pharmaceuticals, Inc. | Trial completion date: May 2025 ➔ Aug 2025 | Trial primary completion date: Mar 2025 ➔ Jun 2025

Trial completion date • Trial primary completion date • Genetic Disorders • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

N-ACETYLCYSTEINE AMIDE AND DI- N-ACETYLCYSTEINE AMIDE PROTECT RETINAL CELLS IN CULTURE VIA AN ANTIOXIDANT ACTION. (PubMed, Exp Eye Res) - "These results warrant further exploration of NACA and diNACA as antioxidant therapeutics for the treatment of retinal diseases, particularly those involving oxidative stress. Furthermore, we have defined the battery of tests carried out as the "Wood, Chidlow, Wall and Casson (WCWC) Retinal Antioxidant Indices"; we believe that these are of great value for screening molecules for potential to reduce retinal oxidative stress in a range of retinal diseases."

Journal • Ophthalmology • Retinal Disorders

INCA: PK and PD Study of NPI-001 and Cysteamine Bitartrate (clinicaltrials.gov) - P1/2 | N=12 | Recruiting | Sponsor: Nacuity Pharmaceuticals, Inc. | Trial primary completion date: Dec 2023 ➔ May 2024

Trial primary completion date

Safety and Efficacy of NPI-001 Tablets for RP Associated With Usher Syndrome (clinicaltrials.gov) - P1/2 | N=49 | Active, not recruiting | Sponsor: Nacuity Pharmaceuticals, Inc. | Recruiting ➔ Active, not recruiting

Enrollment closed • Genetic Disorders • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

INCA: PK and PD Study of NPI-001 and Cysteamine Bitartrate (clinicaltrials.gov) - P1/2 | N=12 | Recruiting | Sponsor: Nacuity Pharmaceuticals, Inc. | Not yet recruiting ➔ Recruiting

Enrollment open

INCA: PK and PD Study of NPI-001 and Cysteamine Bitartrate (clinicaltrials.gov) - P1/2 | N=12 | Not yet recruiting | Sponsor: Nacuity Pharmaceuticals, Inc.

New P1/2 trial

N-Acetylcysteine amide (NACA) and dinaca inhibit HO-INDUCED cataract formation ex VIVO IN pig and rat lenses. (PubMed, Exp Eye Res) - "Based on LC-MS/MS analyses, NACA-induced reduction in opacity observed in rat lenses was attributed to enhanced cysteine and GSH levels while the diNACA-induced reduction in opacity induced did not consistently increase cysteine, cystine and GSH levels and, therefore, appears to involve a different antioxidant mechanism. These screening studies warrant further testing of NACA and diNACA as anticataract agents."

Journal • Preclinical • Cataract • Ophthalmology

Safety and Efficacy of NPI-001 Tablets for RP Associated With Usher Syndrome (clinicaltrials.gov) - P1/2 | N=48 | Recruiting | Sponsor: Nacuity Pharmaceuticals, Inc. | Trial completion date: Sep 2023 ➔ Jan 2025 | Trial primary completion date: Sep 2023 ➔ Jan 2025

Trial completion date • Trial primary completion date • Genetic Disorders • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

Evaluation of NACA and diNACA in human cystinosis fibroblast cell cultures as potential treatments for cystinosis. (PubMed, Orphanet J Rare Dis) - "NACA and diNACA were non cytotoxic to HCFs and significantly increased cell viability. Cystine reduction was determined as percent of control after incubation with 50 µM of NACA, diNACA or cysteamine in HCFs cell culture for 6, 24, 48 and 72 h. Of the three test articles, NACA exhibited most rapid and greatest potency in cystine reduction. Rank order potency for cystine reduction over time was observed, NACA > diNACA ≥ cysteamine. Therefore, further study of NACA and diNACA as potential treatments for cystinosis is warranted."

Journal • Preclinical • Gastrointestinal Disorder • Lysosomal Storage Diseases • Metabolic Disorders • Pain • Rare Diseases

Safety and Efficacy of NPI-001 Tablets for RP Associated With Usher Syndrome (clinicaltrials.gov) - P1/2; N=48; Recruiting; Sponsor: Nacuity Pharmaceuticals, Inc.; Trial completion date: Jun 2023 ➔ Sep 2023; Trial primary completion date: Jun 2023 ➔ Sep 2023

Clinical • Trial completion date • Trial primary completion date • Genetic Disorders • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

Safety and Efficacy of NPI-001 Tablets for RP Associated With Usher Syndrome (clinicaltrials.gov) - P1/2; N=48; Recruiting; Sponsor: Nacuity Pharmaceuticals, Inc.; Not yet recruiting ➔ Recruiting; Trial completion date: Feb 2023 ➔ Jun 2023; Trial primary completion date: Dec 2022 ➔ Jun 2023

Clinical • Enrollment open • Trial completion date • Trial primary completion date • Gene Therapies • Genetic Disorders • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

Safety and Efficacy of NPI-001 Tablets for RP Associated With Usher Syndrome (clinicaltrials.gov) - P1/2; N=48; Not yet recruiting; Sponsor: Nacuity Pharmaceuticals, Inc.

Clinical • New P1/2 trial