Health Status After Switching Angiotensin-converting Enzyme Inhibitors or Angiotensin Receptor Blockers to Sacubitril-valsartan in Patients with Heart Failure with Reduced Ejection Fraction from Rural Tanzania: an Interventional Study

Angiotensin-neprilysin inhibitors (ARNI) are beneficial in patients with heart failure with reduced ejection fraction. No study evaluating ARNI has been conducted in sub-Saharan Africa (except South Africa) yet, where heart failure is a major health problem. Before implementing ARNI in Tanzania, a study evaluating the benefit and safety of ARNI in Africans is needed. The aim of this interventional pre-post study is to evaluate the health status of symptomatic patients with heart failure with reduced ejection fraction who are under a chronic heart failure therapy, before and after switching angiotensin converting enzyme (ACE) inhibitors or angiotensin receptor blockers (ARB) to ARNI. Participants will be recruited at the Heart and Lung Clinic of the St Francis Regional Referral Hospital in Ifakara in rural Tanzania during a study period of 30 months, including 10 months of follow-up. A total of 238 participants will be included. The investigators hypothesize that health status, expressed by the Kansas City Cardiomyopathy Questionnaire summary score and 6-minute walking test, will improve after switching from ACE-inhibitors or ARB to ARNI. In Tanzania, sacubitril/valsartan is registered under the name Uperio®.

Start Date01 Mar 2025

Sponsor / Collaborator

Swiss Tropical & Public Health Institute

[+1]

NCT06693674

/ RecruitingPhase 3IIT

Role of Sacubitril-Valsartan for Right Ventricular Reverse Remodeling in Adults with Tetralogy of Fallot and Ebstein's Anomaly: a Randomized Controlled Pilot Clinical Trial

The purpose of this study is to compare changes in RV structure and function, biomarkers, and patient reported outcomes between TOF patients randomized to an ARNI vs placebo.

Start Date01 Feb 2025

Sponsor / Collaborator

Mayo Clinic

[+1]

NCT06712030

/ Not yet recruitingPhase 3IIT

Effect of Pharmacological Treatment with Angiotensin Receptor/Neprilysin Inhibitors on Transthyretin Cardiac Amyloidosis and Heart Failure with Reduced Ejection Fraction (SAVA-TTR)

Prospective, randomized, multicenter, open-label clinical trial to evaluate the safety and efficacy of Sacubitril/Valsartan (Sac/Vals) compared to no initiation of the drug in patients with transthyretin cardiac amyloidosis (ATTR) and heart failure with reduced ejection fraction (LVEF ≤40%). The primary objective is to determine the impact of Sac/Vals treatment on systolic function by assessing the change in LVEF on echocardiogram at 12-month follow-up.

Start Date01 Jan 2025

Sponsor / Collaborator

Hospital Majadahonda SA

100 Clinical Results associated with Sacubitril/Valsartan

100 Translational Medicine associated with Sacubitril/Valsartan

100 Patents (Medical) associated with Sacubitril/Valsartan

522

Literatures (Medical) associated with Sacubitril/Valsartan

01 Mar 2025·BRAIN RESEARCH

Lactobacillus casei Zhang prevents hippocampal atrophy and cognitive impairment in rats with type 2 diabetes by regulating blood glucose levels

Article

Author: Liu, Sijie ; Lin, Fuchun ; Kang, Yan ; Cheng, Linlin ; Chen, Xi ; Wang, Xuxia ; Cai, Yue

PURPOSE:

Lactobacillus casei Zhang (LCZ) has health benefits, such as the ability to improve blood glucose levels in individuals with type 2 diabetes mellitus (T2DM). However, little is known about the effects of LCZ on brain structural plasticity and cognitive function in T2DM. The aims of this study were to determine whether LCZ can prevent and alleviate brain damage and memory impairment in T2DM, and to understand the mechanisms underlying the effects of LCZ in T2DM.

METHODS:

Forty-one male Sprague-Dawley rats were divided into the saline control (CON, n = 14), T2DM (n = 14) and T2DM + LCZ (n = 13) groups. Magnetic resonance imaging (MRI) was used to evaluate alterations in brain structure among these three groups. The novel object recognition and Y-maze tests were conductedto assess cognitive function. Histological and immunohistochemical analysis, including Nissl staining, Golgi-Cox staining and glial fibrillary acidic protein immunostaining, were performed to explore the pathophysiological mechanisms underlying brain structural changes.

RESULTS:

T2DM rats presented hyperglycemia, cognitive decline, hippocampal atrophy, and damage to hippocampal neurons and astrocytes. Compared with those in the T2DM groups, rats in the T2DM + LCZ group presented lower blood glucose levels, better cognitive function, a larger hippocampal volume, and more normal hippocampal neurons and astrocytes. There was no significant difference in these metrics between rats in the T2DM + LCZ and CON groups.

CONCLUSION:

Hyperglycemia-induced damage to hippocampal neurons and astrocytes may lead to hippocampal atrophy and cognitive dysfunction in T2DM. LCZ can effectively prevent this damage by regulating blood glucose levels, preventing brain atrophy and cognitive impairment in T2DM rats. These findings provide a scientific basis for the clinical application of LCZ.

01 Jan 2025·JOURNAL OF ETHNOPHARMACOLOGY

Qianyang Yuyin granules alleviate hypertension-induced vascular remodeling by inhibiting the phenotypic switch of vascular smooth muscle cells

Article

Author: Zheng, Yawei ; Fan, Yadong ; Liu, Ming ; Zhang, Siqi ; Zhang, Haitao ; Sun, Zeqi ; Zheng, Ziwen ; Jialiken, Dinala ; Fang, Zhuyuan ; Yan, Xiwu ; Zhang, Weiting

ETHNOPHARMACOLOGICAL RELEVANCE:

Qianyang Yuyin granules (QYYY) have been used clinically to treat hypertension for over two decades. Previous clinical trials have shown that QYYY can improve vascular elastic function in hypertensive patients. However, the underlying pharmacological mechanism is unclear.

AIM OF THE STUDY:

To elucidate the effects and mechanisms of QYYY on vascular remodeling using a multidisciplinary approach that includes network pharmacology, proteomics, and both in vitro and in vivo experiments.

MATERIALS AND METHODS:

The main components of QYYY were identified using ultra-high-performance liquid chromatography and high-resolution mass spectrometry. Network pharmacology and molecular docking were employed to predict QYYY's primary active ingredients, potential therapeutic targets and intervention pathways in hypertensive vascular remodeling. We induced hypertension in male C57BL/6 mice by infusing angiotensin II (Ang II) via osmotic minipumps, and performed pre-treatment with QYYY or Sacubitril/valsartan (Entresto). Blood pressure was monitored in vivo, followed by the extraction of aortas to examine pathological structural changes and alterations in protein expression patterns. The expression and location of proteins involved in the HIF-1α/TWIST1/P-p65 signaling pathway were investigated, as well as markers of vascular smooth muscle cells (VSMCs) phenotypic switch. In vitro, we studied the effects of QYYY water extract on Ang II-stimulated human aortic VSMCs. We investigated whether QYYY could affect the HIF-1α/TWIST1/P-p65 signaling pathway, thereby ameliorating apoptosis, autophagy, and phenotype switch in VSMCs.

RESULTS:

We identified 62 main compounds in QYYY, combined with network pharmacology, speculated 827 potentially active substances, and explored 1021 therapeutic targets. The KEGG pathway analysis revealed that the mechanisms of action associated with QYYY therapy potentially encompass various biological processes, including metabolic pathways, TNF signaling pathways, apoptosis, Ras signaling pathways, HIF-1 signaling pathways, autophagy-animal pathways. In hypertensive mice, QYYY restored abnormally elevated blood pressure, vascular remodeling, and inflammation with a dose-response relationship while altering abnormal protein patterns. In vitro, QYYY could inhibit abnormal proliferation, migration, intracellular Ca²⁺ accumulation and cytoskeletal changes of VSMCs. It improved mitochondrial function, reduced ROS levels, stabilized membrane potential, prevented cell death, and reduced overproduction of TGF-β1, TNF-a, and IL-1β.

CONCLUSION:

QYYY may be able to inhibit the overactivation of the HIF-1α/TWIST1/P-p65 signaling pathway, improve the phenotypic switch, and balance apoptosis and autophagy in VSMCs, thereby effectively improving vascular remodeling caused by hypertension.

01 Jan 2025·Acta Crystallographica Section E-Crystallographic Communications

Crystal structure of luliconazole

Article

Author: Chęcińska, Lilianna ; Ben, Anna

The crystal structure of luliconazole {LCZ; C14H9Cl2N3S2; systematic name: (E)-[(4R)-4-(2,4-dichlorophenyl)-1,3-dithiolan-2-ylidene](1H-imidazol-1-yl)acetonitrile} is reported. In the molecule of the title compound, the dithiolane ring adopts an envelope conformation, while the dichlorophenyl ring exhibits disorder. In the crystal packing of luliconazole, only two intermolecular C–H...N hydrogen bonds are observed. Hirshfeld surface analysis reveals that the most dominant contacts are H...N/N...H, H...Cl/Cl...H, H...H and C...H/H...C.

148

News (Medical) associated with Sacubitril/Valsartan

22 Jan 2025

·www.prnewswire.com

Chronic Heart Failure Market to Observe Stunning Growth at a CAGR of 10% by 2034 Owing to Strong Uptake of Entresto and approved SGLT2 Inhibitors along with Emergence of New Class of Therapies | DelveInsight

Several leading classes of therapies are exploring diverse mechanisms of action to treat CHF, including SGLT2 inhibitors, cardiac myosin activators, myeloperoxidase inhibitors, mineralocorticoid receptor antagonists, GLP-1 receptor agonists, cell therapies, and more. This growing focus is expected to drive the chronic heart failure market forward. In summary, the existing blockbuster therapies such as Entresto, recently approved SGLT2 inhibitors, and Soluble guanylate cyclase (sGC) stimulators, along with emerging cell therapies, peptides, monoclonal antibodies, and small molecules, together have the potential to drive the existing market along with securing a significant market share upon introduction of new entrants to the CHF treatment landscape. LAS VEGAS, Jan. 22, 2025 /PRNewswire/ -- DelveInsight's Chronic Heart Failure Market Insights report includes a comprehensive understanding of current treatment practices, chronic heart failure emerging drugs, market share of individual therapies, and current and forecasted market size from 2020 to 2034, segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan]. Key Takeaways from the Chronic Heart Failure Market Report According to DelveInsight's analysis, the market size for chronic heart failure was found to be approximately USD 5.5 billion in the US in 2023. Among the 7MM, the US contributed the largest market share of the total CHF market size and will continue to experience a steep rise in the market size throughout the forecast period. As per DelveInsight's estimate, the total diagnosed prevalent cases of CHF in the 7MM were approximately 20 million in 2023. Heart failure with preserved ejection fraction (HFpEF) contributed to most of the cases. Within class-specific diagnosed prevalence of Heart failure, NYHA class II and class III contribute the maximum. Established chronic heart failure companies with approved therapies in the market are Novartis, Otsuka, Boehringer Ingelheim/Eli Lilly, Bayer/Merck, Amgen/Servier, scPharmaceuticals, and Lexicon Pharmaceuticals/Viatris. In addition, Daiichi Sankyo and American Regent are targeting adult patients with heart failure who are iron deficient. Among all the approved therapies, Novartis and Otsuka's Entresto, approved since 2015 in the US, is still the leading therapy generating more than USD 4 billion in the 7MM in 2023. Among, Eli Lilly's Jardiance and AstraZeneca's Farxiga, both SGLT2 inhibitors, Farxiga is the leading prescribed SGLT2 inhibitor globally by volume Leading chronic heart failure companies developing emerging therapies, such as Cytokinetics, Bayer, Eli Lilly, BioCardia, Mesoblast, Tenax Therapeutics, Novo Nordisk, AstraZeneca, Boehringer Ingelheim, Bristol Myers Squibb, Intra-Cellular Therapies, Cardurion Pharmaceuticals, Rivus Pharmaceuticals, and others are developing novel chronic heart failure drugs that can be available in the chronic heart failure market in the coming years. The promising chronic heart failure therapies in the pipeline include Omecamtiv Mecarbil, KERENDIA (finerenone), Tirzepatide (LY3298176), CardiAMP Cell Therapy, Ziltivekimab (NN6018), REVASCOR (rexlemestrocel-L), Levosimendan (TNX-103), Semaglutide, Balcinrenone (AZD9977) + dapagliflozin, Mitiperstat (AZD4831), Vicadrostat (BI 690517) + Empagliflozin, Cimlanod (CXL-1427/BMS-986231), Lenrispodun (ITI - 214), CRD-740, HU6, and others. In October 2024, Viatris entered into an exclusive licensing agreement with Lexicon Pharmaceuticals for INPEFA (sotagliflozin) in all markets outside of the US and EU. In September 2024, Bayer presented the late-breaking data of Phase III FINE-HEART findings of KERENDIA (finerenone) during a Hot Line session at the European Society of Cardiology (ESC) Congress 2024. In May 2024, Eli Lilly and Company committed an additional USD 5.3 billion manufacturing investment in the company's newest Indiana site to boost API production for tirzepatide and pipeline medicines. AstraZeneca expects the data of the Phase III BalanceD-HF trial of balcinrenone (AZD9977) + dapagliflozin for heart failure in 2025. Discover which therapies are expected to grab the major chronic heart failure market share @ Chronic Heart Failure Market Report Chronic Heart Failure Overview Chronic heart failure is a long-term condition in which the heart's ability to pump blood efficiently is reduced, leading to inadequate circulation of oxygen and nutrients to meet the body's needs. The primary causes of CHF include coronary artery disease, high blood pressure, diabetes, cardiomyopathy, valvular heart disease, and previous heart attacks. Lifestyle factors like obesity, smoking, excessive alcohol consumption, and a sedentary lifestyle can also contribute to its development. The symptoms of CHF vary in severity and may include persistent fatigue, shortness of breath, swelling in the legs, ankles, or abdomen, rapid or irregular heartbeat, and difficulty exercising. Advanced cases can lead to severe fluid retention, wheezing, and an inability to carry out daily activities. Diagnosis typically involves a comprehensive medical history review and a physical examination, followed by tests like an ECG to assess heart rhythm, echocardiography to evaluate heart structure and function, blood tests, chest X-rays, and stress tests. Early diagnosis and management are crucial to improving quality of life and preventing complications. Chronic Heart Failure Epidemiology Segmentation The chronic heart failure epidemiology section provides insights into the historical and current chronic heart failure patient pool and forecasted trends for the 7MM. It helps recognize the causes of current and forecasted patient trends by exploring numerous studies and views of key opinion leaders. The chronic heart failure market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into: Total Diagnosed Prevalent Cases of Heart Failure Gender-specific Cases of Heart Failure Ejection Fraction-specific Cases of Heart Failure New York Heart Association (NYHA) Class-specific Cases of Heart Failure Type-specific Cases of Heart Failure Age-specific Cases of Heart Failure Chronic Heart Failure Treatment Market Current treatments for heart failure primarily rely on angiotensin-converting enzyme (ACE) inhibitors, angiotensin receptor blockers (ARBs), beta-blockers, and diuretics. Additional therapies, such as aldosterone antagonists, amiodarone, antiplatelets, anticoagulants, calcium channel blockers, and nitrates, are also utilized in managing the condition. Most treatment regimens involve a combination of therapies, with beta-blockers being among the most commonly prescribed. Among the approved treatments, Novartis' ENTRESTO has emerged as a leading drug in the congestive heart failure market. Initially slow to gain traction, ENTRESTO has become one of Novartis' key growth drivers, with revenue expected to increase in the coming years. Following its approval for use in patients with preserved ejection fraction, ENTRESTO's market presence has grown significantly. However, ENTRESTO faces growing competition. The entry of new drugs, including SGLT2 inhibitors (such as FARXIGA and JARDIANCE) and VERQUVO, poses a challenge to ENTRESTO's dominance. Additionally, Novartis is set to lose ENTRESTO's exclusivity in the United States in 2025, raising the risk of generic competition. The company is actively engaged in several patent lawsuits to protect its position. SGLT2 inhibitors have also gained a foothold in the CHF market. Although FARXIGA and JARDIANCE are established drugs, they are relatively new in this therapeutic area. The outlook for SGLT2 inhibitors in heart failure is promising, but AstraZeneca and Lilly/Boehringer will have limited time to solidify their positions, as FARXIGA's US patent expires in 2025 and JARDIANCE's in 2028. The recently approved SGLT2 inhibitor sotagliflozin may face challenges in capturing market share from these established treatments. To know more about chronic heart failure treatment guidelines, visit @ Chronic Heart Failure Management Chronic Heart Failure Pipeline Therapies and Key Companies Omecamtiv Mecarbil: Cytokinetics KERENDIA (finerenone): Bayer Tirzepatide (LY3298176): Eli Lilly and Company CardiAMP Cell Therapy: BioCardia Ziltivekimab (NN6018): Novo Nordisk REVASCOR (rexlemestrocel-L): Mesoblast Levosimendan (TNX-103): Tenax Therapeutics Semaglutide: Novo Nordisk Balcinrenone (AZD9977) + dapagliflozin: AstraZeneca Mitiperstat (AZD4831): AstraZeneca Vicadrostat (BI 690517) + Empagliflozin: Boehringer Ingelheim Cimlanod (CXL-1427/BMS-986231): Bristol Myers Squibb Lenrispodun (ITI - 214): Intra-Cellular Therapies CRD-740: Cardurion Pharmaceuticals HU6: Rivus Pharmaceuticals Discover more about chronic heart failure drugs in development @ Chronic Heart Failure Clinical Trials Chronic Heart Failure Market Dynamics The chronic heart failure market dynamics are expected to change in the coming years. An increasing prevalence of cardiovascular diseases, fueled by aging populations and lifestyle-related risk factors such as obesity, hypertension, and diabetes, has significantly raised the demand for effective CHF treatments. Advances in medical technology, including innovative drug therapies, cardiac devices, and regenerative medicine, are expanding treatment options and improving patient outcomes. Growing awareness about heart health and early diagnosis, supported by government and non-government initiatives, is also boosting market expansion. Additionally, the rising focus on personalized medicine and the development of therapies targeting the underlying molecular mechanisms of CHF are creating lucrative opportunities. The increasing healthcare expenditure and the availability of reimbursement policies in developed and emerging economies further bolster the market's growth trajectory. Furthermore, potential therapies are being investigated for the treatment of chronic heart failure, and it is safe to predict that the treatment space will significantly impact the chronic heart failure market during the forecast period. Moreover, the anticipated introduction of emerging therapies with improved efficacy and a further improvement in the diagnosis rate are expected to drive the growth of the chronic heart failure market in the 7MM. However several factors may impede the growth of the chronic heart failure market. A key challenge is the underdiagnosis and delayed recognition of CHF due to overlapping symptoms with other conditions and the lack of widespread access to advanced diagnostic tools in many regions. Economic constraints, particularly in low- and middle-income countries, limit patient access to costly therapies, including advanced drugs and devices. Additionally, adherence to treatment regimens remains low due to the complexity of management protocols and the long-term nature of care, leading to suboptimal outcomes. Regulatory hurdles and lengthy approval processes for novel therapies further impede the introduction of innovative treatments. Finally, a limited understanding among general practitioners about newer guidelines and therapies reduces the referral rates to specialists, creating gaps in patient care. Addressing these barriers requires concerted efforts in education, affordability, and healthcare infrastructure. Scope of the Chronic Heart Failure Market Report Therapeutic Assessment: Chronic Heart Failure current marketed and emerging therapies Chronic Heart Failure Market Dynamics: Key Market Forecast Assumptions of Emerging Chronic Heart Failure Drugs and Market Outlook Competitive Intelligence Analysis: SWOT analysis and Market entry strategies Unmet Needs, KOL's views, Analyst's views, Chronic Heart Failure Market Access and Reimbursement Download the report to understand which factors are driving chronic heart failure market trends @ Chronic Heart Failure Market Trends Table of Contents Related Reports Chronic Heart Failure Epidemiology Forecast Chronic Heart Failure Epidemiology Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology as well as the chronic heart failure epidemiology trends. Chronic Heart Failure Pipeline Chronic Heart Failure Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key CHF companies, including Zensun (Shanghai) Sci & Tech, Cytokinetics, Mesoblast, Shanghai Hongyitang Biopharmaceutical Technology, Tasly Pharmaceuticals, Ionis Pharmaceuticals, Berlin Cures, CardioCell, Help Therapeutics, Eli Lilly and Company, Chong Kun Dang Pharmaceutical, Antlia Biosciences, Cardiol Therapeutics, among others. Acute Coronary Syndrome Market Acute Coronary Syndrome Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key ACS companies, including Novartis, Boehringer Idorsia Pharmaceuticals, Viatris, Recardio, AstraZeneca, Faraday Pharmaceuticals, DalCor Pharmaceuticals, Roche, Jiangsu Vcare PharmaTech, CeleCor Therapeutics, Novo Nordisk, Bristol Myers Squibb, Johnson & Johnson Innovative Medicine, CellProthera, BioCardia, Kancera, Amgen, Arrowhead Pharmaceuticals, Abcentra , among others. Acute Coronary Syndrome Pipeline Acute Coronary Syndrome Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key acute coronary syndrome companies, including Janssen Research & Development, LLC, DalCor Pharmaceuticals, Hyloris Pharmaceuticals, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Phase 3Drug ApprovalLicense out/in

21 Jan 2025

·www.globenewswire.com

Pharming to nominate biopharmaceutical leader Fabrice Chouraqui as new Executive Director and Chief Executive Officer

Leiden, the Netherlands, January 21, 2025: Pharming Group N.V. (“Pharming” or “the Company”) (EURONEXT Amsterdam: PHARM/Nasdaq: PHAR) announces today that the Board of Directors has nominated Fabrice Chouraqui to become Pharming’s new Executive Director and Chief Executive Officer, succeeding Sijmen de Vries. Pharming will nominate Mr. Chouraqui for the appointment as Executive Director and Chief Executive Officer for a term of four years at an upcoming Extraordinary General Meeting of Shareholders (EGM). Information regarding the EGM, including the notice to convene, will be shared shortly in a separate press release. Upon the appointment of Mr. Chouraqui, Sijmen de Vries will resign from the Board of Directors. To ensure a smooth hand-over of tasks and responsibilities, Mr. de Vries will remain a strategic advisor to the new CEO until December 31, 2025. Dr. Richard Peters, Chairman of the Board of Directors, commented:“After an extensive search, the Board and I are confident that Fabrice Chouraqui is the right candidate to lead Pharming and continue to implement our growth strategy, shaping Pharming into the rare disease company of choice. Fabrice is a deeply experienced global pharmaceutical and biotechnology executive, who brings a wealth of profound global expertise and experience, across the entire biopharmaceutical value chain, to Pharming. On behalf of the entire Board of Directors, I would like to thank Sijmen de Vries for his great commitment to Pharming over the past 16 years and for creating the company that it is today, serving patients and paving the way for delivery on the company’s strategy for growth. We are grateful that Sijmen will continue to be available through the end of the year to ensure a smooth hand-over with Fabrice.” Sijmen de Vries, Chief Executive Officer, commented:“I am very pleased with the nomination of Fabrice Chouraqui as my successor. Fabrice brings strong leadership experience with global pharmaceutical companies and highly innovative biotechnology companies to Pharming. To make way for Fabrice to take over the executive leadership of the Company, I will resign from the Board of Directors upon the appointment of Fabrice by our shareholders. I look forward to supporting Fabrice, working closely together through the remainder of the year, and ensuring a smooth hand-over.” Fabrice Chouraqui commented:“I am honored to have the opportunity to take over the leadership of Pharming, building on the strong foundation and joining at a time when I can help propel the company through its next stage of growth in the rare disease space. I am impressed by Pharming’s growing portfolio of products and commercial opportunities which will pave the way for us to become the rare disease company of choice.” ProfileMr. Fabrice Chouraqui (date of birth: August 1, 1970, French national, U.S. citizen) is a global pharmaceutical executive with a record of value creation at Flagship Pioneering, Novartis and Bristol-Myers Squibb. A purpose-driven leader with a passion for bringing innovative treatments to patients, Mr. Chouraqui has a reputation for building strong followership in organizations. He is scientifically minded, with a deep understanding across the business spectrum, from research and development to access and commercialization, including strong expertise in investor management and business development. Across his career, Mr. Chouraqui has led the launch of treatments in many therapeutic areas, including highly targeted specialty care drugs and large primary care medicines. He consistently demonstrates strategic and innovative thinking to achieve long-lasting results and has a strong record of developing next generation talents and highly diverse teams. Until recently, Mr. Chouraqui was a CEO-Partner at Flagship Pioneering, one of the largest Biotech Venture Capital firms in the US. Mr. Chouraqui also served as CEO of Cellarity, Inc., one of the companies created by Flagship Pioneering. In his CEO capacity, Mr. Chouraqui was leading the development of Cellarity, a company which is driving a radically new approach to drug discovery by working at the intersection of AI and biology. Mr. Chouraqui raised over $250M and attracted world-renowned investors. He initiated a collaboration with Novo Nordisk and had Cellarity recognized as a top biotech to watch by several sources. Today, Mr. Chouraqui holds the position of Executive Chairman at Cellarity. Prior to joining Flagship Pioneering, Mr. Chouraqui spent 10 years at Novartis. He was President of Novartis Pharmaceuticals USA from 2016 to 2019, during which he delivered strong growth despite negative price impact and significantly improved profitability. Promoted to turn around performance after the business suffered issues with recent launches, Mr. Chouraqui developed sophisticated commercial and access capabilities to meet the new market environment. He achieved strong market positions for two significant growth drivers (Cosentyx and Entresto), drove the uptake of the rare disease drug Ilaris, initiated the landmark PIONEER clinical trial in heart failure, and championed several business development initiatives. Mr. Chouraqui moved the organization’s culture from “consensus building, conservative and siloed” to “questioning, experimenting and collaborative.” He also served as Novartis representative on the board of BIO. Before leading the US organization, Mr. Chouraqui was President, Latin America & Canada (2014-2016) for Novartis Pharmaceuticals, responsible for leading a portfolio shift from established primary care medicines to innovative specialty care drugs. He also served as Global Head, Business Franchise Neuroscience (2012-2014). In this role, he relaunched Gilenya (Multiple Sclerosis) following a post-launch regulatory review and made Gilenya the largest growth contributor in the Novartis five-year strategic plan. Mr. Chouraqui also addressed late-stage pipeline gaps with the start of the siponimod program in Secondary Progressive Multiple Sclerosis (approved in 2019) and the identification of business development opportunities, which led to the in-licensing of ofatumumab in MS from GSK (launched as Kesimpta) and a partnership with Amgen on erenumab for migraine (launched as Aymovig). From 2000 to 2010, Mr. Chouraqui held multiple international roles at Bristol-Myers Squibb, ultimately serving as Vice President, Commercial Operations, Asia-Pacific and General Manager, Southeast Asia (2008-2010). He began his career in R&D roles at Roussel Uclaf and Hoechst Marion Roussel, pharmaceutical companies that were predecessors to today’s Sanofi. Mr. Chouraqui earned a MBA from INSEAD and a Doctorate in Pharmacy, a Post-Graduate Degree in Quality Assurance of Medicines, and a MSc in Biological and Medicinal Sciences from University of Paris V. Mr. Chouraqui shall have stepped down as Executive Chairman of Cellarity, Inc., a non-listed company, no later than upon his appointment as the new Executive Director and Chief Executive Officer of Pharming. Thereafter, Mr. Chouraqui intends to serve as a non-executive Board member of Cellarity. Mr. Chouraqui also holds the position of independent Board member of OranoMed, a non-listed (and therefore private) subsidiary of Orano Group. OranoMed is a company specialized in the research and development of Pb-212 based radioligand alpha therapies for the treatment of various types of cancers. The resume of Mr. Chouraqui is available on our website. For further public information, contact:Pharming Group, Leiden, the NetherlandsMichael Levitan, VP Investor Relations & Corporate CommunicationsT: +1 (908) 705 1696E: investor@pharming.com FTI Consulting, London, UKVictoria Foster Mitchell/Alex Shaw/Amy ByrneT: +44 203 727 1000 LifeSpring Life Sciences Communication, Amsterdam, the NetherlandsLeon MelensT: +31 6 53 81 64 27E: pharming@lifespring.nl About Pharming Group N.V. Pharming Group N.V. (EURONEXT Amsterdam: PHARM/Nasdaq: PHAR) is a global biopharmaceutical company dedicated to transforming the lives of patients with rare, debilitating, and life-threatening diseases. Pharming is commercializing and developing an innovative portfolio of protein replacement therapies and precision medicines, including small molecules and biologics. Pharming is headquartered in Leiden, the Netherlands, and has employees around the globe who serve patients in over 30 markets in North America, Europe, the Middle East, Africa, and Asia-Pacific. For more information, visit www.pharming.com and find us on LinkedIn. Forward-Looking Statements   This press release may contain forward-looking statements. Forward-looking statements are statements of future expectations that are based on management’s current expectations and assumptions and involve known and unknown risks and uncertainties that could cause actual results, performance, or events to differ materially from those expressed or implied in these statements. These forward-looking statements are identified by their use of terms and phrases such as “aim”, “ambition”, ‘‘anticipate’’, ‘‘believe’’, ‘‘could’’, ‘‘estimate’’, ‘‘expect’’, ‘‘goals’’, ‘‘intend’’, ‘‘may’’, “milestones”, ‘‘objectives’’, ‘‘outlook’’, ‘‘plan’’, ‘‘probably’’, ‘‘project’’, ‘‘risks’’, “schedule”, ‘‘seek’’, ‘‘should’’, ‘‘target’’, ‘‘will’’ and similar terms and phrases. Examples of forward-looking statements may include statements with respect to timing and progress of Pharming's preclinical studies and clinical trials of its product candidates, Pharming's clinical and commercial prospects, and Pharming's expectations regarding its projected working capital requirements and cash resources, which statements are subject to a number of risks, uncertainties and assumptions, including, but not limited to the scope, progress and expansion of Pharming's clinical trials and ramifications for the cost thereof; and clinical, scientific, regulatory, commercial, competitive and technical developments. In light of these risks and uncertainties, and other risks and uncertainties that are described in Pharming's 2023 Annual Report and the Annual Report on Form 20-F for the year ended December 31, 2023, filed with the U.S. Securities and Exchange Commission, the events and circumstances discussed in such forward-looking statements may not occur, and Pharming's actual results could differ materially and adversely from those anticipated or implied thereby. All forward-looking statements contained in this press release are expressly qualified in their entirety by the cautionary statements contained or referred to in this section. Readers should not place undue reliance on forward-looking statements. Any forward-looking statements speak only as of the date of this press release and are based on information available to Pharming as of the date of this release. Pharming does not undertake any obligation to publicly update or revise any forward-looking statement as a result of new information, future events or other information. Inside InformationThis press release relates to the disclosure of information that qualifies, or may have qualified, as inside information within the meaning of Article 7(1) of the EU Market Abuse Regulation. Attachments Fabrice Chouraqui Pharming to nominate new Executive Director and Chief Executive Officer_EN_21JAN25

Executive ChangeDrug Approval

16 Jan 2025

·endpts.com

DC appeals court halts launch of generic to Novartis' heart drug

Novartis won a potentially short-lived victory in its fight across multiple courts to keep generic versions of its blockbuster heart drug Entresto off the market. The US Court of Appeals for the DC Circuit placed a temporary pause on MSN Pharmaceuticals’ Entresto generic launch, which could’ve come as soon as Thursday. The appeals court Wednesday evening made clear that it’s only pausing the launch to consider Novartis’ emergency motion, and its decision is not based on the merits of that motion. Earlier Wednesday, the district court paved the way for the launch of MSN’s generic, denying Novartis’s motion to stop it. US District Judge Dabney Friedrich held that Novartis failed to show that it would likely succeed on the merits or suffer irreparable harm. Novartis did not immediately respond to a request for comment. These two court decisions follow closely behind a separate, pro-Novartis ruling in which the US Court of Appeals for the Federal Circuit last Friday found a key Entresto patent was valid, reversing a lower court ruling. Despite the back and forth in the courts, the FDA approved the first Entresto generics in May 2024. Analysts at Jefferies estimate generics will launch by mid-2025. That launch time will be crucial as Entresto, first FDA-approved in 2015, is one of Novartis’ top-selling drugs, bringing in more than $6 billion in 2023 sales.

Drug ApprovalPatent Infringement

100 Deals associated with Sacubitril/Valsartan

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KEGG	Wiki	ATC	Drug Bank
-	-	C09DX04	-

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Essential Hypertension	CN	Novartis Pharma Schweiz AG	24 May 2021
Hypertension	JP	Novartis Pharma KK	29 Jun 2020
Left ventricular systolic dysfunction	US	Novartis Pharmaceuticals Corp.	01 Oct 2019
Reduced ejection fraction co-occurrent and due to chronic heart failure	EU	Novartis Europharm Ltd.	19 Nov 2015
Reduced ejection fraction co-occurrent and due to chronic heart failure	IS	Novartis Europharm Ltd.	19 Nov 2015
Reduced ejection fraction co-occurrent and due to chronic heart failure	LI	Novartis Europharm Ltd.	19 Nov 2015
Reduced ejection fraction co-occurrent and due to chronic heart failure	NO	Novartis Europharm Ltd.	19 Nov 2015
Heart Failure	CH	Novartis AG	17 Sep 2015
Chronic heart failure	US	Novartis Pharmaceuticals Corp.	07 Jul 2015

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Chronic Kidney Diseases	Phase 3	KR	Chong Kun Dang Pharmaceutical Corp.	14 Oct 2024
Postmyocardial infarction syndrome	Phase 3	-	Novartis Pharmaceuticals Corp.	26 May 2021
Hydrops Fetalis	Phase 3	JP	Novartis Pharmaceuticals Corp.	07 May 2019
Erectile Dysfunction	Phase 3	DE	Novartis Pharmaceuticals Corp.	16 Apr 2019
Heart Failure, Systolic	Phase 3	DE	Novartis Pharmaceuticals Corp.	16 Apr 2019
Acute myocardial infarction	Phase 3	US	Novartis Pharmaceuticals Corp.	09 Dec 2016
Acute myocardial infarction	Phase 3	CN	Novartis Pharmaceuticals Corp.	09 Dec 2016
Acute myocardial infarction	Phase 3	AR	Novartis Pharmaceuticals Corp.	09 Dec 2016
Acute myocardial infarction	Phase 3	AU	Novartis Pharmaceuticals Corp.	09 Dec 2016
Acute myocardial infarction	Phase 3	AT	Novartis Pharmaceuticals Corp.	09 Dec 2016

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT03168568 (VASCEND, Pubmed \| JACC Adv)	Phase 4	Heart Failure	79	Sacubitril/Valsartan	hbuktiozou(vsnwvynbpq) = xdgmtfnchz kxuumkcqli (rjptauchek, 3.9) View more	Negative	01 Dec 2024
				Valsartan	hbuktiozou(vsnwvynbpq) = bdvmropdzw kxuumkcqli (rjptauchek, 2.8) View more
NCT01035255 (PARADIGM-HF, Pubmed \| Eur J Heart Fail)	Phase 3	Heart failure with reduced ejection fraction \| Chronic heart failure	8,399	Beta-blocker	abxuhdpqdk(sdjflnnyae): HR = 0.47 (95% CI, 0.32 - 0.69) View more	Positive	19 Nov 2024
				No Beta-blocker
NCT02678312 (PANORAMA-HF, Pubmed \| Circulation) Manual	Phase 2/3	Heart Failure	375	Sacubitril/Valsartan	hxmyegaxwf(eudjhqhiuj): OR = 0.91 (95% CI, 0.72 - 1.14), P-Value = 0.42 View more	Negative	25 Sep 2024
				Enalapril
NCT01920711 (PARAGON-HF, Pubmed \| Eur J Heart Fail)	Phase 3	Heart Failure	4,795	Sacubitril/valsartan	bizplcarea(tqxltcesiw) = lajyyynkjy iklmxlofwj (gytrvzczkf, 0.0 - 0.2) View more	Positive	01 Sep 2024
				Valsartan	cjqstsycnb(rbripzzusk) = kiolplllns iuiqwmubvf (usfwwxmngr ) View more
ESC2024	Not Applicable	-	24	sacubitril/valsartan (Control)	extlhepzkk(twzlrriefl) = lybqrccmsl gmuytmfdkh (qymfugyjee )	Negative	31 Aug 2024
				Sacubitril/Valsartan	extlhepzkk(twzlrriefl) = aixuffndoz gmuytmfdkh (qymfugyjee )
ESC2024	Not Applicable	Heart Failure	-	Sacubitril/Valsartan (Patients aged below 80 years)	hylbfthwen(emmdgkoodp) = rrcqxboife nyqahzydby (xspaoecqhp )	-	31 Aug 2024
				Sacubitril/Valsartan (Patients aged above 80 years)	hylbfthwen(emmdgkoodp) = jmrghiufvg nyqahzydby (xspaoecqhp )
ESC2024	Not Applicable	Heart Diseases	-	ARNI Sacubitril/Valsartan	yjipnkmmyx(cptabrkybr) = vpzcizzvvg gsvacgyskg (xwvzhdaibg, 806 - 4330)	-	31 Aug 2024
ESC2024	Not Applicable	Heart Failure	-	sacubitril-valsartan (Patients <75 years old)	gcbljjzvam(tdyeofcale) = very few patients were hospitalized for reasons related to angioedema, 16/44,743 in ≥75 y patients and 26/60,167 in those <75 y xafgqwdtdj (etfxvkmfiz )	-	30 Aug 2024
				sacubitril-valsartan (Patients ≥75 years old)
NCT02884206 (PERSPECTIVE, CTgov)	Phase 3	Chronic heart failure	592	Placebo of Valsartan+LCZ696 (LCZ696 200 mg Bid)	uhexceqzox(gwmylgptoo) = yvklwxvcvv etuizukdea (xjrkowoeee, dyuydnezil - zjnyqnmupj) View more	-	06 Aug 2024
				Placebo of LCZ696+Valsartan (Valsartan 160 mg Bid)	uhexceqzox(gwmylgptoo) = kjazvqyqrj etuizukdea (xjrkowoeee, jkepazvkes - tiybwtnzfi) View more
NCT03988634 (PARAGLIDE-HF, CTgov)	Phase 3	Heart failure with normal ejection fraction	467	valsartan matching placebo+sacubitril/valsartan (Sacubitril/Valsartan (LCZ696))	ogjrdwpfgx(zeyslznluh) = nzqqoodzqd mvwikvofzk (tkingjggxc, opczvlzsll - czujwpcrqk) View more	-	29 Jul 2024
				sacubitril/valsartan matching placebo+valsartan (Valsartan)	ogjrdwpfgx(zeyslznluh) = xemtsplqku mvwikvofzk (tkingjggxc, miszecnzyz - ihyentzukj) View more

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