Medical University of Gdansk

ALLSCHWIL, Switzerland I July 1, 2024 I Idorsia Ltd (SIX: IDIA) announced today that the European Commission (EC) has approved JERAYGO ™ (aprocitentan) for the treatment of resistant hypertension in adult patients in combination with at least three antihypertensive medicinal products. 1 The recommended dose is 12.5 mg orally once daily. The dose can be increased to 25 mg once daily for patients tolerating the 12.5 mg dose and in need of tighter blood pressure (BP) control. 1 Hypertension is one of the leading causes of cardiovascular disease worldwide, impacting an estimated 1.3 billion people globally. 2 Approximately 10% of these people have uncontrolled BP, despite receiving at least three antihypertensive medications from different classes, at optimal doses and they are categorized in hypertension guidelines as having resistant hypertension. 3,4 Prof. Krzysztof Narkiewicz, MD, PhD, Head of the Department of Hypertension and Diabetology, Medical University of Gdansk, Poland, commented: “JERAYGO is an oral antihypertensive therapy that is tackling a new therapeutic pathway – the endothelin system. JERAYGO has demonstrated clinically meaningful rapid and long-term reduction in blood pressure. What I was particularly impressed with, this effect was shown in patients with resistant hypertension, whose blood pressure remained uncontrolled despite receiving at least three antihypertensive medications as background therapy. In Europe, there are millions of patients with resistant hypertension, and they are at a higher risk of heart attack, heart failure, stroke, end-stage renal disease and death due to their high blood pressure. With JERAYGO, doctors now a have an effective new treatment option to help control blood pressure in these patients.” Alberto Gimona, MD, Head of Global Clinical Development & Medical Affairs, commented: “We are very proud to have gained approval for JERAYGO, the first innovative anti-hypertensive drug in 40 years, acting on the endothelin pathway, which we believe is a key player in patients with resistant hypertension. We have seen a clinically meaningful and consistent blood pressure lowering across blood pressure measurement methodologies and in subgroups of patients with serious comorbidities – for example in patients with chronic kidney disease. We also saw a marked reduction in albuminuria with JERAYGO as evidenced by a decrease in baseline UACR. I’m very pleased that the wealth of data we have generated with JERAYGO is well reflected in the label. We will now work to expand marketing authorization by also applying for JERAYGO approval in the UK, Canada, and Switzerland.” André Muller, Chief Executive Officer of Idorsia commented: “With aprocitentan, we have a largely unencumbered asset approved in the US and Europe. We continue to carefully evaluate all our funding options including potential collaborations for the commercialization of aprocitentan, while preparing to make aprocitentan available in these two key markets.” About the Phase 3 PRECISION study 1,5 The efficacy of aprocitentan was evaluated in one randomized, double-blind (DB), placebo-controlled Phase 3 multicenter study. Patients with uncontrolled blood pressure (systolic blood pressure [SBP] ≥140 mmHg) despite the use of at least three antihypertensive medicinal products and following exclusion of pseudo-resistant hypertension (e.g., white coat effect, inappropriate blood pressure measurement, secondary causes of hypertension) were considered to have resistant hypertension. The patients were switched to standardized background antihypertensive therapy consisting of an angiotensin receptor blocker (valsartan 160 mg), a calcium channel blocker (amlodipine 5 or 10 mg), and a diuretic (hydrochlorothiazide 25 mg) throughout the study. Patients with concomitant use of beta-blockers continued this treatment throughout the study, in addition to the standardized background antihypertensive therapy and study treatment. A total of 730 patients received either aprocitentan 12.5 mg, aprocitentan 25 mg, or placebo once daily during the initial 4-week DB treatment (part 1). Thereafter, patients received aprocitentan 25 mg once daily during the 32-week single-blind treatment (part 2). At the end of the 32 weeks, patients were re-randomized to receive either aprocitentan 25 mg or placebo, once daily, during the 12-week double-blind withdrawal (DB-WD) treatment (part 3). The primary efficacy endpoint was the change in sitting SBP (SiSBP) from baseline to Week 4 during DB treatment (part 1), measured at trough by unattended automated office blood pressure (uAOBP). The key secondary endpoint was the change in SiSBP measured at trough by uAOBP from DB-WD baseline (Week 36) to Week 40 (part 3). Patients had a mean age of 61.7 years (range 24 to 84 years; 34.1% were ≥ 65 and < 75 years; 9.9% were ≥ 75 years) and 59.5% were male. Patients were White (82.9%), African American (11.2%) or Asian (5.2%). The mean body weight was 97.6 kg (range 46 to 196 kg) and mean BMI was 33.7 kg/m2 (range 18 to 64 kg/m2). Patients had a medical history of type 2 diabetes mellitus (54.1%), ischemic heart disease (30.8%), central nervous system vascular disorders (23.0%), chronic kidney disease stages 3 and 4 (22.2%; 19.3% of patients had eGFR 30–59 mL/min/1.73 m2 and 2.9% had eGFR 15–29 mL/min/1.73 m2), congestive heart failure (19.6%), and sleep apnea syndrome (14.1%). 63.0% of patients had four or more antihypertensive medicinal products. Key PRECISION findings 1,5 Doses of aprocitentan 12.5 and 25 mg showed a statistically significant reduction vs placebo on SiSBP at Week 4. The treatment effect was consistent for sitting diastolic blood pressure (SiDBP). The persistence of the BP-lowering effect of aprocitentan was shown in DB-WD treatment (part 3). In patients re-randomized to placebo, the mean SiSBP increased, whereas in patients re-randomized to aprocitentan 25 mg the mean effect on SiSBP was stable, resulting in a statistically significant difference. The treatment effect was consistent for SiDBP. The effect was also consistent across SBP and DBP measured by ambulatory BP monitoring (ABPM) and assessed as daytime, night-time, and 24h periods at Week 4 and Week 40. A substantial proportion (i.e., at least 90%) of the BP-lowering effect was observed within the first two weeks of treatment with aprocitentan. The effect of aprocitentan was consistent across subgroups of age (including patients ≥ 75 years), sex, race (including patients with Black or African American origin), BMI, baseline urine albumin-to-creatinine ratio (UACR), baseline eGFR and medical history of diabetes. The most frequently reported adverse reactions with aprocitentan were edema/fluid retention (mostly peripheral edema) (9.1%, 12.5 mg; 18.4%, 25 mg) and hemoglobin decreased (3.7%, 12.5 mg; 1.2%, 25 mg). JERAYGO is contraindicated for use in women who are pregnant, breast-feeding and in women of childbearing potential who are not using reliable contraception, and patients with severe hepatic impairment. For more information on the marketing authorization of JERAYGO in the European Union, please review the Summary of Product Characteristics (SmPC) . Notes to the editor About aprocitentan The team at Idorsia has been working on the research and development of endothelin receptor antagonists for more than 30 years, successfully bringing three other molecules from this class to patients in different indications. Endothelin (ET)-1, via its receptors (ET A and ET B ), mediates a variety of effects such as vasoconstriction, fibrosis, cell proliferation, and inflammation and is upregulated in hypertension. Aprocitentan is a dual ERA that inhibits the binding of ET-1 to ET A and ET B receptors and hence the effects mediated by these receptors. 1 References About Prof. Krzysztof Narkiewicz, MD, PhD Professor Krzysztof Narkiewicz is the Head of the Department of Hypertension and Diabetology, Medical University of Gdansk, Gdansk, Poland. His research has been focused on the role of the sympathetic nervous system and metabolic factors in regulation of cardiovascular function in physiological and pathological states, and on prevention and treatment of cardiometabolic diseases including hypertension, diabetes, coronary artery disease, congestive heart failure and obstructive sleep apnea. He has published over 700 full-text publication; (> 39 000 citations; h-index: 69). He was the President of the Scientific Council of the European Society of Hypertension (2009-2011). He was a member of the Task Force for the Management of Arterial Hypertension of the European Society of Hypertension (ESH) and of the European Society of Cardiology (ESC) preparing the 2007, 2013 and 2018 Guidelines for the Management of Arterial Hypertension. He also contributed to the 2023 ESH hypertension guidelines. Prof. Krzysztof Narkiewicz serves as a consultant to Idorsia. About Idorsia Idorsia Ltd is reaching out for more – We have more ideas, we see more opportunities and we want to help more patients. In order to achieve this, we will develop Idorsia into a leading biopharmaceutical company, with a strong scientific core. Headquartered near Basel, Switzerland – a European biotech-hub – Idorsia is specialized in the discovery, development and commercialization of small molecules to transform the horizon of therapeutic options. Idorsia has a 25-year heritage of drug discovery, a broad portfolio of innovative drugs in the pipeline, an experienced team of professionals covering all disciplines from bench to bedside, and commercial operations in Europe and North America – the ideal constellation for bringing innovative medicines to patients. Idorsia was listed on the SIX Swiss Exchange (ticker symbol: IDIA) in June 2017 and has over 750 highly qualified specialists dedicated to realizing our ambitious targets. SOURCE: Idorsia

NEW YORK, April 01, 2024 (GLOBE NEWSWIRE) -- Cognition Therapeutics, Inc. (the “Company” or “Cognition”) (NASDAQ: CGTX), a clinical stage company developing drugs that treat neurodegenerative disorders, today announced it will host a virtual KOL event on Friday, April 12, 2024 from 8:00 AM to 9:30 AM ET. The live and archived event may be accessed from the investor relations section of the Cognition website. To register, please click here. The event will feature three experts who will provide insights on the unmet need and the current treatment landscape for Alzheimer’s disease, as well as anti-amyloid beta monoclonal antibody drug launches, and commercial uptake. They include: Anton Porsteinsson, MD of the University of Rochester Alzheimer's Disease Care, Research and Education Program,Martin J. Sadowski, MD, PhD, DSci of New York University School of Medicine, andEverard (Jort) Vijverberg, MD, PhD of Alzheimer Center Amsterdam and Neuroscience Amsterdam. In addition, the KOLs and Cognition management will discuss CT1812, Cognition's novel oral once-daily sigma-2 (σ-2) receptor modulator designed to displace Aβ oligomers from their binding sites on neurons. Topics will include details of its mechanism of action and ongoing clinical development program in Alzheimer’s disease, which includes the Phase 2 START Study in people with early stage disease and the Phase 2 SHINE Study in people with mild-to-moderate Alzheimer's disease. Topline results from the SHINE Study are anticipated in mid-2024. A live question and answer session will follow formal presentations. About Anton Porsteinsson, MDDr. Porsteinsson is the director of the University of Rochester Alzheimer's Disease Care, Research and Education Program (AD-CARE). In addition, he is the first William B. and Sheila Konar Professor of Psychiatry at the University of Rochester, School of Medicine and Dentistry. Dr. Porsteinsson has devoted his career to the care and study of individuals with memory disorders. He participates in the University of Rochester Memory Care Program, which serves a population with memory disorders. Internationally recognized in clinical research and considered one of the leading experts in Alzheimer's disease and dementia, his interests lie in biomarkers, imaging, and novel pharmacologic agents in the treatment of Alzheimer's disease and other dementias, both in terms of cognitive loss and behavioral disturbances. He is a leading investigator for many prominent Alzheimer's prevention and treatment trials investigating cognitive enhancers, course-stabilizing agents in dementia, as well as psychotropics for behavioral changes associated with dementia. In 1987, he received his MD from the University of Iceland School of Medicine and came to the University of Rochester in 1989, where he completed his internship and residency. He is Board certified in psychiatry, with added qualifications in geriatric psychiatry. Dr. Porsteinsson is the author and collaborator of hundreds of research publications and serves as site principal investigator for research grants from the National Institutes of Health and other leading funders. About Martin J. Sadowski, MD, PhD, DSciDr. Sadowski is an associate professor in the departments of neurology, psychiatry, and biochemistry and molecular pharmacology at the New York University School of Medicine. Research in Dr. Sadowski’s laboratory focuses on amyloid beta (Aβ) metabolism, the interaction between Aβ and apolipoprotein E (APOe), and discovery of therapeutics for Alzheimer’s disease and prion diseases. His research is supported by funding from the National Institute on Aging, American Federation for Aging Research, Alzheimer’s Association, and private philanthropy. In addition, Dr. Sadowski provides clinical service to patients with dementia and conducts clinical trials of novel Alzheimer’s therapeutics. Dr. Sadowski is a recipient of the Paul B. Beeson Career Development Award in Aging Research and the NIH K02 Independent Scientist Award. He earned MD/PhD from the Medical University of Gdansk, in Gdansk Poland and completed his postdoctoral fellowship in experimental neuropathology under the mentorship of Dr. Henry M. Wisniewski and neurology residency in New York University School of Medicine. He has authored approximately 60 papers and three patents and has served on several NIH study section panels. About Everard (Jort) Vijverberg, MD, PhDDr. Vijverberg is a staff neurologist and CNS trial specialist at the Amsterdam Alzheimer Center at Amsterdam Neuroscience, the research institute for neuroscience of Amsterdam UMC and the science faculties of Vrije Universiteit Amsterdam and the University of Amsterdam. Dr. Vijverberg has dedicated his career to finding novel ways to treat Alzheimer’s disease and other dementias with specialization in familial forms of neurodegenerative disorders associated with cognitive and behavior impairment. He is founder of The CANDIDATE Center, a research unit dedicated to central nervous system (CNS) target and drug discovery and is currently a primary investigator and/or consultant on more than ten different clinical trials in neurodegeneration, including the NEwTON study of progressive brain damage caused by traumatic head-brain injury and the Social Brain Project, which investigates individuals with behavioral changes in late adulthood that may indicate a psychiatric disorder. Dr. Vijverberg earned his PhD from Vrije Universiteit Amsterdam (VU Amsterdam) and his medical degree from Leiden Universiteit in The Netherlands. He has authored more than 60 papers and co-authored four books. About Cognition Therapeutics, Inc.Cognition Therapeutics, Inc. is a clinical-stage biopharmaceutical company engaged in the discovery and development of innovative, small molecule therapeutics targeting age-related degenerative disorders of the central nervous system and retina. We are currently investigating our lead candidate CT1812 in clinical programs in Alzheimer’s disease, dementia with Lewy bodies (DLB) and dry age-related macular degeneration (dry AMD). We believe CT1812 and our pipeline of σ-2 receptor modulators can regulate pathways that are impaired in these diseases. We believe that targeting the σ-2 receptor with CT1812 represents a mechanism functionally distinct from other current approaches in clinical development for the treatment of degenerative diseases. More about Cognition Therapeutics and its pipeline can be found at https://cogrx.com. Forward Looking Statements This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. All statements contained in this press release, other than statements of historical facts or statements that relate to present facts or current conditions, including but not limited to, statements regarding our product candidates, including CT1812, and any expected or implied benefits or results, including that initial clinical results observed with respect to CT1812 will be replicated in later trials and our clinical development plans, including statements regarding our clinical studies of CT1812 and any analyses of the results therefrom, are forward-looking statements. These statements, including statements relating to the timing and expected results of our clinical trials, involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance, or achievements to be materially different from any future results, performance, or achievements expressed or implied by the forward-looking statements. In some cases, you can identify forward-looking statements by terms such as “may,” might,” “will,” “should,” “expect,” “plan,” “aim,” “seek,” “anticipate,” “could,” “intend,” “target,” “project,” “contemplate,” “believe,” “estimate,” “predict,” “forecast,” “potential” or “continue” or the negative of these terms or other similar expressions. We have based these forward-looking statements largely on our current expectations and projections about future events and financial trends that we believe may affect our business, financial condition, and results of operations. These forward-looking statements speak only as of the date of this press release and are subject to a number of risks, uncertainties and assumptions, some of which cannot be predicted or quantified and some of which are beyond our control. Factors that may cause actual results to differ materially from current expectations include, but are not limited to: competition; our ability to secure new (and retain existing) grant funding; our ability to grow and manage growth, maintain relationships with suppliers and retain our management and key employees; our ability to successfully advance our current and future product candidates through development activities, preclinical studies and clinical trials and costs related thereto; uncertainties inherent in the results of preliminary data, pre-clinical studies and earlier-stage clinical trials being predictive of the results of early or later-stage clinical trials; the timing, scope and likelihood of regulatory filings and approvals, including regulatory approval of our product candidates; changes in applicable laws or regulations; the possibility that the we may be adversely affected by other economic, business or competitive factors, including ongoing economic uncertainty; our estimates of expenses and profitability; the evolution of the markets in which we compete; our ability to implement our strategic initiatives and continue to innovate our existing products; our ability to defend our intellectual property; impacts of ongoing global and regional conflicts; the impact of the COVID-19 pandemic on our business, supply chain and labor force; and the risks and uncertainties described more fully in the “Risk Factors” section of our annual and quarterly reports filed with the Securities & Exchange Commission and are available at www.sec.gov. These risks are not exhaustive and we face both known and unknown risks. You should not rely on these forward-looking statements as predictions of future events. The events and circumstances reflected in our forward-looking statements may not be achieved or occur, and actual results could differ materially from those projected in the forward-looking statements. Moreover, we operate in a dynamic industry and economy. New risk factors and uncertainties may emerge from time to time, and it is not possible for management to predict all risk factors and uncertainties that we may face. Except as required by applicable law, we do not plan to publicly update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise. Contact Information:Cognition Therapeutics, Inc.info@cogrx.comCasey McDonald (media)Tiberend Strategic Advisors, Inc.cmcdonald@tiberend.comMike Moyer (investors)LifeSci Advisorsmmoyer@lifesciadvisors.com

AUCKLAND, New Zealand, Jan. 26, 2023 /PRNewswire/ -- Douglas Pharmaceuticals, a New Zealand Pharmaceutical company focused on specialty generic and repurposed drug development, today announced the formation of a clinical advisory board to support its lead program R-107, an oral extended-release dose of racemic ketamine being developed as add-on or monotherapy for treatment resistant depression (TRD). "We are very excited to bring together a group of world-class experts in the field of psychiatry, to help guide our late-stage development of R-107," said Peter Surman, Chief Scientific Officer. "We believe their deep knowledge in the field, insights and contributions will be instrumental in charting an efficient course through pivotal clinical studies in TRD." The members of the clinical advisory board include: Prof. Paul Glue, MB, ChB (Otago), MD (Bristol), FRCPsych – Dr. Paul Glue is the Hazel Buckland Professor of Psychiatry at the University of Otago, New Zealand and Chair of Douglas' R-107 Trial Steering Committee. He joins in the role of Board Chair with extensive experience in psychiatric research in ketamine, including early development of R-107. His areas of interest include the pharmacology of drug treatments in psychiatry and the investigation of disease mechanisms in neurological and psychiatric disorders. He has published extensively in the fields above, with more than 250 papers and book chapters to his name. Prof. Alan F. Schatzberg, MD – Dr. Alan F. Schatzberg is the Kenneth T. Norris, Jr. Professor of Psychiatry & Behavioural Sciences at Stanford University, US. From 1991-2010, he was the Chair of the Department at Stanford. He is also the Director of the Stanford Mood Disorders Center and Emeritus Editor for Journal of Psychiatric Research. Dr. Schatzberg's current research interests include the biological bases of depressive disorders, glucocorticoid/dopamine interactions in delusional depression and pharmacologic treatment of depressive disorders, with more than 400 publications to date. Dr. Schatzberg has been extensively involved as investigator in several trials in depressive disorders. He was elected the 136th President of the American Psychiatric Association and is a member of the National Academy of Medicine. Prof. Sanjay J. Mathew, MD – Dr. Sanjay J. Mathew is a Professor of Psychiatry and Behavioural Sciences, Vice Chair for Research and Director of Mood and Anxiety Disorders Program at Baylor College of Medicine, Texas, USA. His clinical expertise is in the psychopharmacological management of adult patients with difficult-to-treat depressive and anxiety disorders. He has been the recipient of several grants related to his research in the neurobiology treatment of depressive disorders and authored over 150 manuscripts. Prof. Allan Young, MB, ChB, MPhil, PhD, FRCP (Edin.), FRCPC, FRCPsych – Dr. Allan Young is Professor of Mood Disorders and is the Director of the Centre for Affective Disorders in the Department of Psychological Medicine in the Institute of Psychiatry, Psychology and Neuroscience as well as Head of School and Vice-Dean for Academic Psychiatry at King's College London, UK. Dr. Young is the National Institute for Health Research (NIHR) Mental Health Biomedical Research Centre (BRC) Cluster and Theme lead in the Translational Therapeutics Cluster. He is the clinical academic lead in the Psychological Medicine and Integrated Care Clinical Academic Group in the South London and Maudsley NHS Trust where he is also a Consultant Psychiatrist and the head of the National Affective Disorders Tertiary Clinic. Dr. Young's research interests focus on the cause and treatments for severe psychiatric illnesses, particularly mood disorders. He is ranked as one of the world's leading scientific minds according to the Thomson Reuters Highly Cited Researchers list, ranking among the top 1% most cited for their subject field and year of publication, a mark of exceptional impact. Prof. Wiesław J. Cubała, MD, PhD – Dr. Wiesław J. Cubała is the Head of the Department of Psychiatry at the Medical University of Gdansk, Poland. His research interests focus on the psychopharmacology of mood disorders with a particular interest in ketamine/esketamine/arketamine use in major depression and bipolar disorder as well as clozapine use in treatment-resistant bipolar depression with a focus on suicidality and its rapid-cycling course. Dr. Cubała is engaged in the development of the national treatment guidelines for mood and anxiety disorders. He is also the author of over 160 publications in depressive disorders and was an editorial board member of BJPsych Open and European Neuropsychopharmacology: the journal of the European College of Neuropsychopharmacology in 2021. Dr. Cubała has been extensively involved as investigator in several trials in mood disorders including 14 trials with Rapid-Acting Antidepressants to date. Prof. Colleen Loo, MBBS (Hons), FRANZCP, MD –Dr. Colleen Loo is Professor of Psychiatry at the University of New South Wales, Sydney and Professor and leader of the Neurostimulation and Interventional Psychiatry program at the Black Dog Institute, Sydney, Australia. Her research interests focus on improving treatments for treatment-resistant depression, including electroconvulsive therapy (ECT), Transcranial Magnetic Stimulation (TMS), transcranial Direct Current Stimulation (tDCS) and ketamine. She was president of the International Society for ECT and Neurostimulation from 2018-2020. From 2011, she conducted a series of studies on ketamine treatment of depression, examining different routes of administration, dosing approaches and developing a system for safety monitoring. She has published over 300 peer reviewed papers and contributed to national and international guidelines. For further information, please contact: Stephen Waterman Managing Director PharmaVentures Ltd [email protected] +44-7931144097 About Douglas Pharmaceuticals Douglas Pharmaceuticals is New Zealand's largest privately owned pharmaceutical and healthcare company. It was founded in 1967 by pharmacist Sir Graeme Douglas and today employs over 700 people and exports to over 40 countries around the world. The Douglas mission is to 'Improve Lives' by providing innovative, competitive, and high-quality healthcare solutions. Douglas' core business is producing prescription drugs for areas including oncology, dermatology, the central nervous system, and immunology. Douglas develops, manufactures, and distributes novel and generic products, with a preference for those where there is a strong intellectual property position as well as technical complexity in areas of high unmet need. Furthermore, as of Sep 2022 a state-of-the-art research and development facility has opened in West Auckland, which aims to use world class medical research to find solutions for a range of serious illnesses. The $50MM Douglas Innovation centre is part of the long-established Douglas campus in Auckland. The three story, 4,500sq metre building was 18 months in construction. It employs 100 people and includes laboratory, pilot scale product development suites, commercial manufacturing rooms which can handle highly potent and cytotoxic products, and office space. Manufacturing capabilities include soft gelatine encapsulation, liquids, creams, and modified release oral dosage forms. For more information, please visit: R-107 R-107, an oral extended-release dose of racemic ketamine being developed as add-on or monotherapy for treatment resistant depression (TRD). Phase 2 results demonstrated a significant antidepressant effect within 8 days, dose-dependent sustained mood improvement, with maintenance of response at 3 months, high compliance at-home dosing regimen. R-107's robust safety profile is enabled by a proprietary oral extended-release tablet, which has been formulated with abuse-deterrent features. R-107 has potential for expedited regulatory pathway in the US and EU, with potential market entry as early as 2026 and a strong IP protection in place through to at least 2037 excluding potential extensions. Douglas has also collected positive responses to the R-107 Target Product Profile from Key Opinion Leaders and payers in US & EU. SOURCE Douglas Pharmaceuticals