NPI-001

REYKJAVIK, Iceland and FORT WORTH, Texas, March 12, 2024 (GLOBE NEWSWIRE) -- Arctic Therapeutics ehf, an Icelandic drug development company and partner Nacuity Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company developing treatments for retinitis pigmentosa, cataracts and other diseases caused by oxidative stress, today announced approval by the European Medicines Agency (EMA) for initiation of the first clinical trial evaluating AT-001 (Nacuity’s NPI-001) for the treatment of Hereditary Cystatin C Amyloid Angiopathy (HCCAA) in Iceland. HCCAA is an ultra rare, serious condition characterized by the accumulation of amyloid in the brain's blood vessels, which can lead to cerebral hemorrhage, stroke, and progressive neurological deterioration, including dementia. Current treatment options are severely limited, focusing primarily on symptom management rather than addressing the underlying cause of the disease. AT-001 is a differentiated, purified, prescription-quality, GMP-grade N-acetylcysteine amide (NACA). “EMA's approval paves the way for a comprehensive investigation into the safety and efficacy of a novel therapeutic approach designed to halt or reverse the progression of HCCAA. Our study aims to explore the potential of AT-001 in modifying the disease's course, improving patient outcomes, and ultimately, saving lives,” said Ivar Hakonarson, CEO and co-founder of Arctic. In September 2022, Nacuity and Arctic entered into an exclusive licensing agreement and strategic partnership for the development and commercialization of Nacuity’s NPI-001 under the name AT-001 for HCCAA in Iceland and related forms of amyloid angiopathies (CAAs) worldwide. Nacuity is also currently evaluating NPI-001 in Phase 1/2 clinical trials in Australia for retinitis pigmentosa associated with Usher syndrome and cystinosis. “Our proprietary NPI-001 has potential to address the myriad diseases and conditions where oxidative stress plays a role. EMA approval for the evaluation of NPI-001 tablets for treatment of HCCAA represents most importantly, a step forward in addressing HCCAA, a life threatening and underserved rare disease, and secondly, a key pharmaceutical quality milestone for the companies in the global development of NPI-001,” said G. Michael Wall, Ph.D., SVP & CSO, Nacuity. About Nacuity Pharmaceuticals, Inc. Nacuity Pharmaceuticals, Inc. ( ), is a clinical-stage leader in innovative treatments for oxidative stress. The company’s powerful, targeted therapies aim to attentuate 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 in retinitis pigmentosa, cataracts and cystinosis with the potential to be first-of-a-kind therapies and gateways to wider applications. 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 ( ) and its venture arm RD Fund ( ). About Arctic Therapeutics ehf Arctic Therapeutics ehf (AT) ( ) is an Icelandic drug 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. AT 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, USA, and multiple collaborations across Europe. Nacuity Media Contact Julia Clements Tel: 267.626.1085 jclements@6degreespr.com Arctic Media Contact Gulli Arnason gulli@arctictherapeutics.com

Around the world, most people are regularly exposed to low or moderate levels of lead, cadmium and arsenic in the environment, increasing risk of coronary artery disease, stroke and peripheral artery disease, according to a new statement. Chronic exposure to low levels of lead, cadmium and arsenic through commonly used household items, air, water, soil and food is associated with an increased risk of cardiovascular disease, according to a new American Heart Association scientific statement published today in the Journal of the American Heart Association, an open access, peer-reviewed journal of the American Heart Association. This scientific statement reviews evidence linking chronic exposure to low or moderate levels of three contaminant metals -- lead, cadmium and arsenic -- to cardiovascular diseases including coronary artery disease, stroke and peripheral artery disease. It highlights clinical and public health implications. Traditional risk factors for cardiovascular disease do not currently include environmental toxicants. The field of environmental cardiology identifies exposure to pollutants including contaminant metals as modifiable risks for cardiovascular disease. "Large population studies indicate that even low-level exposure to contaminant metals is near-universal and contributes to the burden of cardiovascular disease, especially heart attacks, stroke, disease of the arteries to the legs and premature death from cardiac causes," said Gervasio A. Lamas, M.D., FAHA, chair of the statement writing group and chairman of medicine and chief of the Columbia University Division of Cardiology at Mount Sinai Medical Center in Miami Beach, Florida. "These metals interfere with essential biological functions and affect most populations on a global scale," said vice chair of the statement writing group Ana Navas-Acien, M.D., Ph.D., a professor of environmental health sciences at Columbia University's Mailman School of Public Health and the director of the Columbia University Northern Plains Superfund Research Program in New York City. "After exposure, lead and cadmium accumulate in the body and remain in bones and organs for decades. In the U.S. alone, one large study suggested that more than 450,000 deaths annually could be attributed to lead exposure." Where are people exposed to contaminant metals? Exposure to contaminant metals most often occurs involuntarily, through activities of daily living. Lead may be found in a variety of items such as paint in old homes (lead paint was banned in the U.S. in 1978), tobacco products, second-hand smoke, contaminated foods (ground water and some pottery, ceramics and kitchenware are sources of lead contamination in food), water pipes, spices, cosmetics, electronics and industrial emissions. Cigarette smoking is a source of both lead and cadmium. Cadmium is found in nickel-cadmium batteries, pigments, plastic, ceramics and glassware, and construction products. Industrially produced fertilizers use phosphate rock that is naturally rich in cadmium, which then contaminates root vegetables and leafy green plants (including tobacco). Arsenic exposure is primarily through groundwater, which affects drinking water, soil and food grown in contaminated soil. Notably, arsenic builds up in rice more than other food crops. While exposure and risk occur across diverse populations regardless of socioeconomic level, some people experience greater exposure to toxic metals, according to the statement. Risk of exposure is higher for people who live closer to major roadways, industrial sources and hazardous waste sites; reside in older houses; or in areas where environmental regulations are poorly enforced and responses to community complaints are inadequate. "This is a global issue in which lower-income communities are disproportionately exposed to toxic metals through contaminated air, water and soil," said Navas-Acien. "Addressing metal exposure in these populations may provide a strategy to reduce cardiovascular disease disparities and advance environmental justice." What are the cardiovascular risks of contaminant metals? The scientific statement outlines global epidemiologic research confirming that lead, cadmium and arsenic are associated with premature death, due in large part to increased cardiovascular disease risk. The global research includes: What can be done about metals in the environment? Monitoring environmental metal levels and testing for metal in individuals are key steps to implement appropriate public health initiatives, the writing group suggests. Lead levels in children with symptoms of exposure are monitored by health professionals using blood tests. However, there are no monitoring guidelines or exposure limits established for contaminant metals in adults other than those required for specific types of work. Future research is needed to establish if such testing may be an effective strategy to identify and protect people at risk of cardiovascular disease. The statement authors note that decreasing metal exposure in tobacco, protecting community water systems and wells, and minimizing metal contamination in air, food and soil are all examples of public health measures that may reduce exposure to metals. "Cardiovascular health may be improved with a multi-pronged approach that recognizes environmental cardiology and includes environmental monitoring and biomonitoring of contaminant metals; controlling for sources of exposure; and developing clinical interventions that remove metals or weaken their effects on the body," said Lamas, who is also a professor of medicine at Columbia University Irving Medical Center in New York City. While there is currently no standard medical therapy to counteract the vascular impact of contaminant metals, there is research in progress to address the potential for treating individuals for exposure. Some research is assessing the effect of chelating agents, which are medications that can remove contaminant metals, especially lead and cadmium, from the body. The chelating agent binds to metals so they can be excreted. In addition, the statement suggests research is needed to investigate nutritional supplements that may reduce the effects of contaminant metals and accelerate excretion. Supplements that have shown potential based on small trials include folate and N-acetyl cysteine. This scientific statement was prepared by the volunteer writing group on behalf of the American Heart Association's Council on Epidemiology and Prevention; the Council on Cardiovascular and Stroke Nursing; the Council on Lifestyle and Cardiometabolic Health; the Council on Peripheral Vascular Disease; and the Council on Kidney in Cardiovascular Disease. American Heart Association scientific statements promote greater awareness about cardiovascular diseases and stroke issues and help facilitate informed health care decisions. Scientific statements outline what is currently known about a topic and what areas need additional research. While scientific statements inform the development of guidelines, they do not make treatment recommendations. American Heart Association guidelines provide the Association's official clinical practice recommendations. The Association receives funding primarily from individuals. Foundations and corporations (including pharmaceutical, device manufacturers and other companies) also make donations and fund specific Association programs and events. The Association has strict policies to prevent these relationships from influencing the science content. Revenues from pharmaceutical and biotech companies, device manufacturers and health insurance providers, and the Association's overall financial information are available here.