NPI-001

Phase 3Small molecule

NNacuity Pharmaceuticals, Inc.

NPI-001 is one of 3 small molecule programs targeting Retinitis Pigmentosa (RP). It is currently in Phase 3.

Indications

Total Trials

Active Trials

Competitors

Pipeline

Status by Indication[3][1]

Indication

Retinitis Pigmentosa (RP)Not yet recruiting

Alzheimer´s DiseaseActive, not recruiting

Retinitis Pigmentosa (RP)Phase 3

Not yet recruiting

Alzheimer´s DiseasePhase 2

Active, not recruiting

Competition

Competitive Landscape

4 competitors in Retinitis Pigmentosa (RP)

#DrugCompanyPhaseTrials

NPI-001

NNacuity Pharmaceuticals, Inc.

Phase 3

1 2

N-Acetylcysteine Amide

NNacuity Pharmaceuticals, Inc.

Phase 3

1 3

Intravitreal Injection of Ultevursen

LLaboratoires Thea

Phase 2

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Timeline

Expected Milestones

Q2 2027

Phase 2Active, not recruiting

Alzheimer´s Disease

NCT07741526

Q1 2029

Phase 3Not yet recruiting

Retinitis Pigmentosa (RP)

NCT07710196

Clinical

Trials by Indication[1]

Alzheimer´s Disease1 trial

A Phase 2a Multi-site, Randomized, Double-blind, Dose Escalated, Placebo Controlled Biomarker Study to Determine the Safety, Tolerability and Biomarker-based Efficacy of NPI-001 (AT-001) in Subjects With MCI (Mild Cognitive Impairment) or Mild Dementia Due to Alzheimer's Disease (AD) Aged 50 to 85 Years

Active, not recruiting·AArctic Therapeutics·Phase 2·NCT07741526·33 enrolled

Retinitis Pigmentosa (RP)1 trial

A 24-Month, Randomized, Double-Masked, Placebo-Controlled Trial of NPI-001 for the Preservation of Photoreceptors in Retinitis PigmentosaAssociated With Usher Syndrome

Not yet recruiting·NNacuity Pharmaceuticals, Inc.·Phase 3·NCT07710196·80 enrolled

Usher Syndrome1 trial

Safety and Efficacy of NPI-001 Tablets Versus Placebo for Treatment of Retinitis Pigmentosa Associated With Usher Syndrome

Completed·NNacuity Pharmaceuticals, Inc.·Preclinical·NCT04355689·49 enrolled

Sources

References

[1]
ClinicalTrials.gov — 3 trial records for NPI-001. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — NPI-001 compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for NPI-001. EMBL-EBI & Wellcome Sanger Institute.
[4]
SEC EDGAR — Nacuity Pharmaceuticals, Inc. company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Modality: Small molecule[2]
Highest Phase: Phase 3[1]
Company: NNacuity Pharmaceuticals, Inc.

NPI-001

Status by Indication[3][1]

Competitive Landscape

Expected Milestones

Q2 2027

Q1 2029

Trials by Indication[1]

Alzheimer´s Disease1 trial

Retinitis Pigmentosa (RP)1 trial

Usher Syndrome1 trial

References

Drug Info

Quick Facts

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