We aim to assess the the efficiency and safety of N-acetylcysteine for prevention of thrombotic events after allogenic hematopoietic stem cell transplantation.

Start Date01 Aug 2023

Sponsor / Collaborator

The First Affiliated Hospital of Soochow University

100 Clinical Results associated with NPI-001

100 Translational Medicine associated with NPI-001

100 Patents (Medical) associated with NPI-001

283

Literatures (Medical) associated with NPI-001

01 Mar 2025·American Journal of Veterinary Research

Oxidative stress induces plasma membrane phosphatidylserine externalization in canine erythrocytes in vitro, mitigated by thiol antioxidants

Article

Author: Woolcock, Andrew D. ; Kao, Yun-Fan ; Santos, Andrea Pires dos ; Serpa, Priscila B. S. ; Moore, George E.

AbstractOBJECTIVETo determine if oxidative stress induces phosphatidylserine (PS) externalization in canine erythrocytes and if exposure to antioxidants prevents such changes.METHODSThis was an in vitro, experimental study using 5 healthy, adult, purpose-bred research Beagles. Fresh EDTA-anticoagulated blood samples were collected from each dog, and erythrocytes were harvested. For objective 1, erythrocytes were exposed to the pro-oxidant agents tert-butyl hydroperoxide (TBHP) at 2, 3, or 4 mM or 2,2′-azobis(2-amidinopropane) dihydrochloride at 30, 40, or 50 mM. For objective 2, erythrocytes were exposed to 3 mM TBHP and the antioxidant N-acetylcysteine-amide (NACA) at various concentrations (0, 1, or 3 mM). Erythrocytes incubated with benzoylbenzoyl-ATP were used as positive control, whereas erythrocytes incubated with sodium chloride medium with 0.1% bovine serum albumin, DMSO, and NACA were used as negative controls. Erythrocytes were stained with allophycocyanin-conjugated Annexin V, and PS externalization was assessed by flow cytometry. The degree of PS externalization of each sample was recorded as median fluorescence intensity and percentage of PS positivity.RESULTSTBHP at 3 and 4 mM caused increased PS externalization in canine erythrocytes. 2,2′-Azobis(2-amidinopropane) dihydrochloride at all concentrations caused increased PS externalization. N-acetylcysteine-amide at all concentrations prevented significant PS externalization measured by median fluorescence intensity and percentage of PS positivity from erythrocytes exposed to TBHP.CONCLUSIONSOxidative stress causes PS externalization in canine erythrocytes, and NACA ameliorates this effect.CLINICAL RELEVANCEFuture studies are needed to determine if increased PS externalization in erythrocytes occurs in dogs with immune-mediated hemolytic anemia and its role in promoting thromboembolism.

01 Mar 2025·Talanta

Rational engineering of ligands & metal nodes: MOF-on-MOF based probe for efficient integration of anti-interference and triple signal amplification

Article

Author: Ma, Zhanfang ; Wang, Jiaqing ; Han, Hongliang

Accurate and sensitive detection of slight fluctuations in insulin concentration is essential for the assessment and treatment of early diabetes. The requirement posed a serious challenge to the responsiveness of electrochemical sensors in analyzing slight fluctuation in concentration and their ability to accurately identify the target analyte. Therefore, a MOF-on-MOF (NH₂-MIL-88B-on-NH₂-UiO-66) probe with integrated anti-interference and signal-amplification capabilities was designed by engineering functionalized ligands and metal nodes, which ensures the sensitivity of sensors while improving the detection accuracy. Signal-amplification: the bimetallic ion nodes of MOF-on-MOF act synergistically at the sensing interface (sodium citrate doped poly-thionine/poly (3, 4-ethylenedioxythiophene) film) to impede the electron transfer, leading to a decrease in the poly-thionine (PThi) oxidation current signal. The complexed Fe³⁺ triggers a Fenton reaction, further leading to a decrease in the PThi oxidation current signal. A triple amplification of the PThi oxidation current signal response induced by changes in concentration per unit of analyte was achieved. Anti-interference: The growth of a more hydrophilic MOF on the surface of another less hydrophilic MOF and the introduction of a large number of amino groups resulted in a significant anti-interference property of the MOF-on-MOF based immunoprobes. The proposed sensor not only achieves detection limits of insulin at the fg level, but also has a signal response per unit change in insulin concentration being 5-50 times higher than existing electrochemical sensors. The integration of the hydrophilic and triple signal amplification strategy is significant in the monitoring of slight fluctuations of analyte concentrations.

27 Feb 2025·Journal of Medicinal Chemistry

Design, Synthesis, and Biological Evaluation of BODIPY-Caged Resiquimod as a Dual-Acting Phototherapeutic

Article

Author: Takahashi, Haruko ; Morioka, Norimitsu ; Taniguchi, Haruto ; Roshdy, Eslam ; Mohammed, Elsayed S. I. ; Nakamura, Yoki ; Celik, Ismail ; Abe, Manabu ; Kikuchi, Yutaka ; Ishihara, Yasuhiro

Resiquimod, an imidazoquinoline scaffold, exhibits potent immunotherapeutic activity but is associated with off-target effects, limiting its clinical utility. To address this limitation, we developed a novel BODIPY-caged resiquimod that is responsive to red light, combining photocaging and photodynamic therapy functionalities. Molecular docking studies guided identification of the optimal caging site for resiquimod, effectively masking its immune activity. BODIPY-caged resiquimod remained inactive under dark conditions, effectively masking resiquimod's immunostimulatory effects. However, red light irradiation precisely uncaged resiquimod, inducing robust immune activation, even in the presence of N-acetyl cysteine as an antioxidant. Notably, the attachment of resiquimod to BODIPY reduced the dark toxicity typically associated with BODIPY as a photosensitizer. In 3D spheroid models of HeLa and A549 cells, BODIPY-caged resiquimod demonstrated spatiotemporal control over cytotoxicity, significantly enhancing cell death only upon irradiation. This dual-function therapeutic approach highlights a "win-win" strategy: precise, red-light-mediated control of immune activation and photodynamic efficacy with reduced collateral toxicity.

News (Medical) associated with NPI-001

22 Jan 2025

·www.prnewswire.com

Arctic Therapeutics Announces €26.5M Oversubscribed Series A Financing

Funding will accelerate the company's anti-amyloid treatment for rare and common forms of dementia. As well as bringing forward a new class of treatment for inflammatory skin diseases. Sanos Group CEO Jeppe Ragnar Andersen joins the board. REYKJAVIK, Iceland and PHILADELPHIA, Jan. 22, 2025 /PRNewswire/ -- Icelandic drug discovery and development company Arctic Therapeutics (ATx) today announced the successful closing of a €26.5 million oversubscribed Series A financing from a broad syndicate of international investors. The syndicate includes the EIC Fund, Iceland's largest privately-held investment firm Kaldbakur, Investcorp-backed Sanos Group, Swiss-based Cerebrum DAO, The Lurie Family Foundation – established by US billionaire Jeffery Lurie – as well as a consortium led by early investors and co-founders of Icelandic unicorn, Kerecis, and Copenhagen-listed Chemometec. Icelandic institutional investors and family offices also participated in the round. "This funding represents a critical milestone in our journey to address significant unmet medical needs, from familial forms of dementia and Alzheimer's to novel treatments for inflammatory skin diseases. The new investors bring expertise and strategic value to the table, enabling us to accelerate the development of groundbreaking treatments," said Ivar Hakonarson, ATx co-founder and CEO. The proceeds from the Series A financing will be used to advance ATx's two frontrunners, AT-001 and AT-004, further into the clinic. Last year, the European Medicines Agency (EMA) approved a pivotal phase IIb/III clinical trial evaluating AT-001 for the treatment of Hereditary Cystatin C Amyloid Angiopathy (HCCAA), a rare form of familial dementia. The new funding will allow ATx to explore the potential of AT-001, an oral treatment designed to prevent the aggregation of harmful amyloid proteins in the brain, in other forms of familial dementia, and eventually Alzheimer's disease. "Recent advances in understanding the role of amyloid proteins and the critical importance of preventing and dissolving plaque formation in the brain underscore the transformative potential of our approach to significantly slow or even prevent these devastating diseases," Hakonarson added. In addition to the AT-001 clinical trial, ATx is also planning to launch a phase IIa clinical trial for AT-004 in Europe post-financing. The therapeutic goal of this approach is to demonstrate safety and efficacy in acne vulgaris before expanding into other inflammatory skin diseases, including atopic dermatitis, rosacea and psoriasis. Additionally, ATx announced the appointment of Jeppe Ragnar Andersen, CEO of Sanos Group, to its board of directors. "Sanos Group's proven expertise in dermatology and recent acquisition of NeuroScios, a CRO specialized in clinical studies in Alzheimer's, strengthens our alignment with ATx's therapeutic focus even further," Andersen said. "We look forward to supporting the ATx team on achieving its goal of bringing forward innovative treatments for dementia and skin diseases to millions of patients," he added. Last year, ATx was selected to join the prestigious EIC Scaling Club, an exclusive community of Europe's most promising scaleups. The Club aims to accelerate the growth of its members, with a goal of scaling 20% of them into unicorns. This aligns with the EIC Fund's investment mandate to provide funding to European companies, which have the potential to meet the world's most pressing challenges. "Investing in innovative solutions to address the global healthcare challenge of dementia is both a responsibility and an opportunity," said Svetoslava Georgieva, Chair of the EIC Fund Board. "In Europe alone, dementia affects over ten million people – placing immense strain on families, healthcare systems, and economies. By providing an equity investment in Arctic Therapeutics, the EIC Fund is supporting groundbreaking preventive treatments that have the potential to change the trajectory of this crisis, improve quality of life, and alleviate the burden on healthcare infrastructure worldwide," Georgieva added. About Arctic Therapeutics Arctic Therapeutics is an Icelandic drug discovery and development company established in 2015 as a spin-off from the US-based Center for Applied Genomics (CAG), a research center at the Children's Hospital of Philadelphia led by ATx founder Hakon Hakonarson, MD, PhD. ATx leverages the transformative power of applied genomics, steering the course of drug development towards safer, more effective treatments for some of the world's most challenging diseases. The company has operations in Iceland, the US and multiple collaborations across Europe. For more information, please visit: and follow us on LinkedIn. About Sanos Group Sanos Group is a leading specialist provider of clinical study services in the global pharma and biotech industry. The group consists of around 200 employees, mainly in Denmark and the United States, but also in Europe and Hong Kong. Last year, Sanos acquired Austrian NeuroScios, which is a contract research organization (CRO) focusing on Alzheimer's and other diseases of the central nervous system. The acquisition is part of the group's strategic development as a multi-niche CRO, adding a third specialist area to the two current scientific areas of osteoarthritis and dermatology. About EIC Fund The European Innovation Council Fund from the European Commission is an agnostic fund: it invests across all technologies and verticals, and all EU countries and countries associated to Horizon Europe. It provides the investment component of the EIC Accelerator blended finance. The European Investment Bank acts as investment adviser to the EIC Fund. The EIC Fund aims to fill a critical financing gap and its main purpose is to support companies in the development and commercialization of disruptive technologies, bridging with and crowding in market players, and further sharing risk by building a large network of capital providers and strategic partners suitable for co-investments and follow-on funding. The fund pays particular attention to the empowerment and support of female founders as well as the ambition to reduce the innovation divide among EU countries. For media inquires Gulli Arnason, Chief Strategy Officer [email protected] +354 660 0053 This information was brought to you by Cision . The following files are available for download: SOURCE Arctic Therapeutics WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Executive ChangeAcquisition

21 Jan 2025

·www.globenewswire.com

Nacuity Pharmaceuticals Granted U.S. FDA Fast Track Designation for NPI-001 (N-acetylcysteine amide) Tablets for the Treatment of Retinitis Pigmentosa

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 PigmentosaOften 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-001NPI-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 ContactJulia Clements267.626.1085jclements@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.

Orphan DrugFast TrackGene TherapyClinical Study

21 Jan 2025

·endpts.com

With support from NFL owner Jeffrey Lurie, Icelandic biotech closes Series A for oral Alzheimer's drug

A father-and-son biotech with ties to Iceland and Philadelphia has put together a €26.5 million Series A to develop oral treatments for Alzheimer’s disease and other neurodegenerative conditions. Founded in 2015, Arctic Therapeutics will attempt to push forward work by Icelandic genomics researcher Hákon Hákonarson. His son, Ívar Hákonarson, runs things as CEO. The duo raised funds from a variety of American and European investors, including the charitable foundation of billionaire Jeffrey Lurie, who owns the NFL’s Philadelphia Eagles franchise. Alzheimer’s has thwarted drug developers for decades, thanks to a string of trial failures for therapies based on the amyloid theory — the idea that the disease can be treated, or even reversed, by removing the buildup of amyloid plaque from the brain. Only recently did the FDA approve two such drugs: Eisai and Biogen’s Leqembi and Eli Lilly’s Kisunla both demonstrated modest effects at improving patients’ cognition and function. Most efforts to develop amyloid-targeting Alzheimer’s drugs, including Leqembi and Kisunla, involve administration through intravenous infusions in order to more easily cross the blood-brain barrier. Historically, oral Alzheimer’s treatments are much harder to deliver to the brain than IV therapies. The Hákonarsons say they’ve found a way to solve those issues. Arctic’s lead program, AT-001, is a prodrug of an inexpensive antioxidant called N-acetyl cysteine (NAC). Sometimes used as a supplement, it’s been around for about 60 years. It’s currently used to treat Tylenol toxicity and polycystic ovary syndrome, as well as relieve mucus buildup in the lungs, and more. The elder Hákonarson, who is the director of the genomics center at the University of Pennsylvania’s medical school, told Endpoints News that NAC does not normally cross the blood-brain barrier, but Arctic altered the chemistry to make it both fat-soluble and brain-penetrant. By doing so, Hákonarson said AT-001 can prevent the buildup of amyloid plaque from occurring and break up deposits already in the brain. “Our drug is basically blocking any new aggregation,” Hákonarson said of the amyloid plaques. “The critical thing is obviously to try to get it in early, but once you stop and you block any ongoing aggregation and precipitation, the macrophages in the brain, they just remove it.” Arctic plans to first test its hypothesis in smaller disease populations, and last year it received approval from the EMA to start a Phase 2b/3 trial in a rare, familial form of dementia called hereditary cystatin C amyloid angiopathy (HCCAA). If it works in this indication, the plan is to expand into other forms of dementia and Alzheimer’s, according to Ívar Hákonarson. The HCCAA study is expected to last about a year, and an approval in the EU could come as early as the second half of 2026, he added. Arctic’s plans for US trials and a potential launch are still being finalized, but the company is hoping that an EMA approval could help move things along in the US. The biotech also expects to partner with a larger pharma company to commercialize the drug in the US if it’s approved. “Our goal is to get this treatment to the US as soon as possible,” Ívar Hákonarson said. “That is, of course, the largest market, which we will be targeting.” Arctic has another pipeline program called AT-004 that it will develop with the funding. The treatment is a topical solution being developed for a variety of skin conditions, including psoriasis and atopic dermatitis. The company expects to launch a Phase 2a study in acne vulgaris in Europe, and it completed its pre-IND meeting with the FDA for the program last year. In addition to the Lurie Family Foundation, other investors in the Series A included EIC Fund, Kaldbakur, Sanos Group, Cerebrum DAO, early investors and co-founders of Kerecis and ChemoMetec, and several institutional and family investors from Iceland. Editor’s note: This story was updated to correct the nature of two of the Series A investors. Early investors and co-founders of Kerecis and ChemoMetec — and not the companies — invested in Arctic.

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Indication	Highest Phase	Country/Location	Organization	Date
Usher Syndromes	Phase 2	Australia	Nacuity Pharmaceuticals, Inc.Startup	03 Sep 2020
Alzheimer Disease	Phase 2	United States	Arctic TherapeuticsStartup	-
Cerebral Amyloid Angiopathy	Phase 2	United States	Arctic TherapeuticsStartup	-
Cerebral Amyloid Angiopathy, Familial	IND Approval	Iceland	Arctic TherapeuticsStartup	12 Mar 2024
Cerebral Amyloid Angiopathy, Familial	IND Approval	Iceland	Nacuity Pharmaceuticals, Inc.Startup	12 Mar 2024

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04632069 (CTgov)	Early Phase 1	Pregnancy in Diabetics \| Diabetes Mellitus \| stress oxidative	10	N acetyl cysteine + vagus nerve stimulation	ukppwsfeeh(jmnfqotdpq) = mmhxewpeir zvdvfjmmye (fiosghtczi, qeixjuxgzt - gpiwmesyyh) View more	-	29 Aug 2024
NAASO2022	Not Applicable	Nonalcoholic Steatohepatitis	13	N-acetyl cysteine 600 mg/day	(qzqxqeueys) = lvknicmdnh pdohbkzmlj (vpjibitldr ) View more	-	21 Nov 2022
				N-acetyl cysteine 1200 mg/day	(qzqxqeueys) = nfdlefgseg pdohbkzmlj (vpjibitldr ) View more
NCT01322009 (Pro-NAC, CTgov)	Phase 1/2	Brain Injuries, Traumatic	14	N-acetyl cysteine+Probenecid (Drug)	xgfyynorel(vsgpvcfbmn) = ktxhpfzegx cksedhnxkb (qelbdfiekn, bjwelggljq - jfjvgxprum) View more	-	15 Aug 2016
				Placebo (Placebo)	xgfyynorel(vsgpvcfbmn) = fprpfpyilk cksedhnxkb (qelbdfiekn, sqcbvdgstq - eakbxibvjq) View more
ARVO2008	Not Applicable	Grouped Pigmentation of the Macula	-	N-acetylcysteine amide (NACA)	ykybkkjkju(riiaojkxan) = wdtilgvrdn yxteijblzj (odxmljsgvb )	-	01 May 2008
				N-Acetylcysteine Amide (tert-butylhydroperoxide (tBH))	ykybkkjkju(riiaojkxan) = dsfriapmme yxteijblzj (odxmljsgvb )

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