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

AD Biomarker: Safety, Tolerability and Biomarker-based Efficacy of NPI-001 (AT-001) in Subjects With MCI or Alzheimer's Disease (AD) (clinicaltrials.gov) - P2 | N=33 | Active, not recruiting | Sponsor: Arctic Therapeutics

Biomarker • New P2 trial • Alzheimer's Disease • CNS Disorders • Cognitive Disorders • Dementia

SLORP Preserve: A 24-Month Trial of NPI-001 for the Preservation of Photoreceptors in Retinitis Pigmentosa Associated With Usher Syndrome (clinicaltrials.gov) - P3 | N=80 | Not yet recruiting | Sponsor: Nacuity Pharmaceuticals, Inc.

New P3 trial • 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=49 | Completed | Sponsor: Nacuity Pharmaceuticals, Inc. | Active, not recruiting ➔ Completed

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

24-Month Trial of NPI-001 for the Preservation of Photoreceptors in Retinitis Pigmentosa Associated With Usher Syndrome (clinicaltrials.gov) - P3 | N=80 | Not yet recruiting | Sponsor: Nacuity Pharmaceuticals, Inc. | Trial completion date: Dec 2028 ➔ Jul 2029 | Initiation date: Jun 2026 ➔ Dec 2026 | Trial primary completion date: Dec 2028 ➔ Jul 2029

Trial completion date • Trial initiation date • Trial primary completion date • 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 | Active, not recruiting | Sponsor: Nacuity Pharmaceuticals, Inc. | Recruiting ➔ Active, not recruiting

Enrollment closed

NPI-001 Slows Photoreceptor Cell Loss in Adults with Retinitis Pigmentosa Associated with Usher Syndrome (ARVO 2026) - "Conclusions Oral NPI-001 tablets (250 mg, BID) significantly delayed progression of RP measured by photoreceptor cell preservation in participants with USH over 24 months compared with placebo tablets. NPI-001 is the first drug to demonstrate significant preservation of photoreceptor cells in USH, representing an important gene agnostic approach to address an unmet need in this sight-threatening disease."

Clinical • 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. | Trial completion date: Jan 2026 ➔ Aug 2026 | Trial primary completion date: Jan 2026 ➔ Jun 2026

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: Aug 2025 ➔ Apr 2026

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

24-Month Trial of NPI-001 for the Preservation of Photoreceptors in Retinitis Pigmentosa Associated With Usher Syndrome (clinicaltrials.gov) - P3 | N=80 | Not yet recruiting | Sponsor: Nacuity Pharmaceuticals, Inc.

New P3 trial • 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. | Trial completion date: Jun 2025 ➔ Jan 2026 | Trial primary completion date: Apr 2025 ➔ Jan 2026

Trial completion date • Trial primary completion date

Arctic Therapeutics Receives EMA Approval for Phase IIa Study of AT-001 in Alzheimer’s Disease (Yahoo Finance) - "ATx has initiated a Phase IIa clinical trial to evaluate the safety and biomarker-based efficacy of AT-001 in individuals with mild cognitive impairment (MCI) or mild dementia due to Alzheimer's disease (AD)."

New P2a trial • Alzheimer's Disease • Dementia

Nacuity Pharmaceuticals, Inc…announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) to NPI-001 (N-acetylcysteine amide) tablets, Nacuity’s proprietary, investigational therapy for the treatment of patients with retinitis pigmentosa (RP). (Nacuity Press Release)

Breakthrough therapy • Retinitis Pigmentosa

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