FORT WORTH, Texas, Jan. 21, 2025 (GLOBE NEWSWIRE) -- Nacuity Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company developing treatments for retinitis pigmentosa, cataracts and other diseases caused by oxidative stress, 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).

A drug may receive Fast Track designation if it is intended to treat a serious or life-threatening disease and demonstrates the potential to address an unmet medical need.

“Fast Track Designation represents an objective assessment by the FDA for the potential of NPI-001 tablets as a treatment for RP, a severe blinding disease,” said G. Michael Wall, Ph.D., Senior Vice President and Chief Scientific Officer of Nacuity Pharmaceuticals. “We are committed to advancing NPI-001 to address this significant unmet medical need for patients suffering from RP.”

A drug that receives Fast Track designation may benefit from more frequent interactions with the FDA throughout drug development. In addition, the Fast Track program allows for Accelerated Approval and Priority Review, if relevant criteria are met.

About Retinitis Pigmentosa
Often diagnosed in childhood or adolescence, retinitis pigmentosa (RP) is a heterogeneous group of inherited retinal diseases involving over 3,000 different mutations in more than 50 genes (Daiger et al., 2013), causing progressive night and peripheral vision loss. RP often leads to legal and sometimes complete blindness. Forms of RP and related diseases include Usher syndrome, Leber congenital amaurosis and Bardet-Biedl syndrome. An estimated 100,000 people in the U.S. have RP (https://www.fightingblindness.org/diseases/retinitis-pigmentosa). There are no FDA-approved drugs or no standard treatments for RP. A curative gene therapy, voretigene neparvovec (LUXTURNA®), exists for patients with the RPE65 mutation, about 1-6% of patients with RP (Cross et al., 2022). Nacuity researchers are working to develop a gene-agnostic treatment for RP, a significant unmet medical need.

About NPI-001
NPI-001 is a proprietary, GMP-grade formulation of N-acetylcysteine amide (NACA) tablets being developed to target oxidative stress associated with diseases such as retinitis pigmentosa. Preclinical studies indicate that NPI-001 boosts glutathione, the body’s most powerful endogenous antioxidant, to stop chemically aggressive oxygen molecules from damaging retinal cells. NPI-001 and related small molecules are potent antioxidants in several preclinical models that may lead to additional indications. In addition to Fast Track Designation, NPI-001 has also been granted Orphan Drug Designation for RP which provides seven years of U.S. FDA regulatory exclusivity for the product upon regulatory approval.

About Nacuity Pharmaceuticals, Inc.
Nacuity Pharmaceuticals is a clinical-stage leader in innovative treatments for oxidative stress. The company’s powerful, targeted therapies aim to attenuate oxidative tissue damage, a driver of blinding eye diseases and a broad spectrum of serious chronic conditions. Nacuity has three highly differentiated clinical programs ongoing for retinitis pigmentosa, cataract and cystinosis. Nacuity has operations in Fort Worth, TX, USA, and Australia, and extensive managerial and scientific domain expertise as well as backing from Foundation Fighting Blindness (https://www.fightingblindness.org/) and its venture arm RD Fund (https://www.retinaldegenerationfund.org). For more information, please visit www.nacuity.com.

Trademarks are the property of their owners.

Nacuity Media Contact
Julia Clements
267.626.1085
jclements@6degreespr.com

References:

Cross N, van Steen C, Zegaoui Y, Satherley A, Angelillo L. Retinitis Pigmentosa: Burden of Disease and Current Unmet Needs. Clin Ophthalmol. 2022 Jun 20;16:1993-2010. doi: 10.2147/OPTH.S365486. PMID: 35757022; PMCID: PMC9232096.

Daiger SP, Sullivan LS, Bowne SJ. Genes and mutations causing retinitis pigmentosa. Clin Genet. 2013 Aug;84(2):132-41. doi: 10.1111/cge.12203. Epub 2013 Jun 19. PMID: 23701314; PMCID: PMC3856531

Guidance for Industry: Expedited Programs for Serious Conditions – Drugs and Biologics, May 2014.

Wood JPM, Chidlow G, Wall GM, Casson RJ. N-acetylcysteine amide and di-N-acetylcysteine amide protect retinal cells in culture via an antioxidant action. Exp Eye Res. 2024 Nov; 248:110074. doi: 10.1016/j.exer.2024.110074. Epub 2024 Sep 7. PMID: 39251120.

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