Intracranial Arterial Diseases

Group Pushes for Passage of "Ellie's Law" to Increase NIH Funding for Brain Aneurysm Research; Aims to Build Awareness of the Broad Impact of the Disease on Patients and Caregivers HANOVER, Mass., March 19, 2024 /PRNewswire/ -- The Brain Aneurysm Foundation (BAF), the leading advocacy organization supporting education, research, and policy to transform the treatment of brain aneurysms, today joined with caregivers, survivors, researchers, and physicians from across the U.S. to advocate for greater funding and to increase awareness of brain aneurysms and the long-term impact they can have on individuals and their families. Continue Reading Advocates on Capitol Hill for the 11th Annual Brain Aneurysm Foundation Advocacy Day - March 2023. The group will meet with lawmakers to discuss the prevalence of the disease, the significant economic impact – including medical expenses, lost productivity, and long-term disability – as well as the critical need for new interventions that can help better detect and treat aneurysms. It is estimated that one in fifty people in the US has a brain aneurysm, with more than 30,000 people suffering from ruptures annually, of which half are fatal. Worldwide, there are close to 500,000 deaths each year from brain aneurysms, half of which occur in people under the age of 50. "The tragic loss of our daughter last year showed us the suddenness of brain aneurysms, how they strike without warning and can take the lives of those who are young, vibrant, and otherwise healthy," said Lynne Caruso, whose 31-year-old daughter Katelyn died from a ruptured aneurysm that occurred following a morning workout. The family's extensive work to honor Katelyn, who died just months before her planned wedding, has led to their involvement in supporting increased funding for critical research. "This is an underappreciated and potentially devastating condition, and our focus is to ensure that dollars reach those working to develop new interventions that can save lives and prevent the unimaginable suffering our family and others have had to experience." Brain Aneurysm research receives a disproportionately low level of funding from the National Institutes of Health compared to other neurological disorders, with only two dollars provided annually for each person affected. The BAF is working on the passage of Ellie's Law (H.R. 902 and S. 895), which would provide dedicated research for aneurysm prevention and improve the long-term outcomes for aneurysm survivors. Ellie's Law would authorize $10 million each fiscal year for five years from the National Institute of Neurological Disorders and Stroke (NINDS) to conduct or support further comprehensive research on unruptured intracranial aneurysms. "Research shows that up to five percent of the US population may have an unruptured aneurysm, yet despite the prevalence, awareness of the condition and its risk factors remains low. Our focus is to provide education regarding the potential severity of the disease and to press for critical research funding for promising life-saving research and innovation," said Christine Buckley, executive director of the BAF. "It is an honor to be joined by so many of the families, survivors, caregivers, and physicians who are taking the time to share their stories regarding the impact of the disease and the economic impact on individuals, our health system, and society." Brain aneurysms often do not cause symptoms until they rupture, at which point they become a medical emergency. Research focused on identifying biomarkers or advanced imaging techniques may help in the detection of aneurysms prior to rupture, significantly improving patient outcomes by allowing for early interventions. There is also a significant need for new surgical techniques, endovascular procedures, and minimally invasive interventions that can lead to safer and more effective treatments for both ruptured and unruptured aneurysms. "Researchers today often rely on vital seed funding from organizations like the BAF to advance research for subarachnoid hemorrhage and other brain diseases. The BAF's support for new innovation has been essential, but there is clearly a need for additional funding sources to support research as we get closer to potential breakthroughs in the field," said Vince Tutino, an assistant professor at the Jacobs School of Medicine and Biomedical Sciences at the University of Buffalo and CEO of Neurovascular Diagnostics, Inc., which is working to develop a blood test for detecting whether brain aneurysms may rupture. "Brain scans can be cost prohibitive and are often not recommended by a physician unless there is a family history of the disease. Developing simple blood tests and other forms of detection will lead to millions of lives being saved and billions of dollars from being spent on patient care." ABOUT BRAIN ANEURYSMS A brain aneurysm is a weak, bulging area in an artery in the brain that, in some instances, can rupture. Blood then spills into the space between the skull and the brain, which is a serious type of stroke known as a subarachnoid hemorrhage. While brain aneurysms are most prevalent in people ages 35 to 60, they can also occur in children. Women over the age of 55 have a higher risk of brain aneurysm rupture than men and are generally at greater risk. Brain aneurysms are also seen at higher rates in African American and Hispanic populations. ABOUT THE BRAIN ANEURYSM FOUNDATION Founded in Boston and now based in Hanover, Massachusetts, the Brain Aneurysm Foundation is the globally recognized leader in brain aneurysm awareness, education, support, advocacy, and research funding. The foundation's mission is to provide information about and raise awareness of the symptoms and risk factors of brain aneurysms to prevent ruptures and subsequent death and disability; work with medical communities to provide support networks for patients and families; and advance research to improve patients' outcomes and save lives. Established in 1994, the foundation has a Medical Advisory Board that comprises more than 40 of the world's foremost aneurysm experts — neurologists, neurosurgeons, interventional neuroradiologists, and other brain aneurysm specialists — from leading hospitals and universities. For more information about the Brain Aneurysm Foundation, please visit bafound.org or follow us on Facebook, Twitter, Linkedin, Instagram, and YouTube. Media Contact: Lynn Nutall Director of Marketing and Communications Brain Aneurysm Foundation 617-543-3921 [email protected] SOURCE Brain Aneurysm Foundation

March 12, 2024 -- 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. Nacuity Pharmaceuticals, Inc. (http://www.nacuity.com/), 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 (https://www.fightingblindness.org/) and its venture arm RD Fund (https://www.retinaldegenerationfund.org). Arctic Therapeutics ehf (AT) (https://www.arctictherapeutics.com/) 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. The content above comes from the network. if any infringement, please contact us to modify.

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