Background: Sodium-glucose-cotransporter (SGLT) inhibition has been observed to reduce risk of cardiovascular events and kidney failure in persons with type 2 diabetes. People with type 1 diabetes also have increased risk of cardiovascular and kidney disease, and may benefit from SGLT-inhibition. The exact mechanism of how SGLT-inhibition benefits the kidneys are yet unknown. Change in renal hypoxia may be a factor.
Objective: The primary aim of this study is to assess the effects of 12 weeks SGLT-1 and 2 inhibition on renal oxygenation in persons with type 1 diabetes and chronic kidney disease.
Further aims are to study if renal oxygen consumption and response to SGLT-inhibition differs between people of African-Caribbean or Northern European decent.
Additionally effects on left ventricular ejection fraction, kidney function and biomarkers in blood and urine will be explored.
Method: 12 weeks treatment with oral sotagliflozin or matching placebo as intervention. Kidney oxygenation and perfusion parameters and left ventricular ejection fraction will be assessed by functional magnetic resonance imaging. Kidney function and biomarkers will be assessed according to local hospital laboratory guidelines.
Design: Randomized, double-blinded, placebo-controlled, cross over intervention study.
Study population: 69 persons with type 1 diabetes and diabetic kidney disease with albuminuria will be included, 39 at Steno Diabetes Center Copenhagen, 30 at King's College London.
Endpoints: Primary end-point: Change from 0 to 12 weeks in dynamic R2*-weighted signal after treatment with sotagliflozin compared to placebo. Secondary endpoints: Change from 0 to 12 weeks with sotagliflozin compared with placebo on renal perfusion, renal artery flow, renal oxygen consumption, renal parenchymal triglyceride fraction, renal fibrosis, left ventricular ejection fraction, urinary albumin-creatinin ratio, ketone bodies, erythropoietin, pro brain natriuretic peptide, and plasma- and urine inflammation- and fibrosis biomarkers as well as difference after 12 weeks treatment in glomerular filtration rate.
Timeframe: Inclusion of patients from february 2024. Last visit september 2025. Presentation spring 2026, publication fall 2026.

Start Date01 Feb 2025

Sponsor / Collaborator

Steno Diabetes Center A/S

[+4]

NCT06435156

/ RecruitingPhase 2IIT

A Phase 2 Double-blind Randomised Controlled Trial Studying the Effect of Sotagliflozin Versus Placebo in Individuals with Heart Failure and Type 1 Diabetes.

People with type 1 diabetes sometimes develop heart failure which can cause symptoms like breathlessness, tiredness or ankle swelling, reduced quality of life and lead to being admitted to hospital or suffering potential fatal consequences. This trial is investigating if a tablet called sotagliflozin, can improve quality of life in people with type 1 diabetes and heart failure. In addition, this trial will also assess the safety and tolerability of sotagliflozin in this population.
In previous trials that included people with type 2 diabetes and heart failure sotagliflozin was shown to improve patients' symptoms of heart failure, quality of life and reduce the chance of people with heart failure being admitted to hospital or dying. However, people with type 1 diabetes and heart failure were not included in these trials meaning that it is not known if these benefits also apply to this population.
This trial aims to recruit 320 people with type 1 diabetes and heart failure symptoms in multiple sites in the United Kingdom (UK). This trial will compare the health and quality of life of participants who take sotagliflozin tablets with participants who take placebo tablets, which is a dummy tablet that looks the same as sotagliflozin. Participants will be randomly allocated to one of two groups (i.e. one taking sotagliflozin and the other the placebo) and both the medical team and participants will not know in which group each participant is until the end of the study. Participants will be in the trial for approximately 6 months and will be given sotagliflozin or placebo tablets to take 1 per day for 4 months. The trial is expected to run for a total of 26 months.

Start Date28 Jan 2025

Sponsor / Collaborator

University Of Dundee

[+2]

NCT06618976

/ Not yet recruitingPhase 4IIT

SOTA-THROMBOSIS: Antithrombotic Activities of Sotagliflozin vs. Empagliflozin

The availability of Sodium-glucose cotransporter-2 inhibitors (SGLT2-i) has dramatically altered the management of heart failure (HF) patients, independently of their ejection fraction and glycemic status. A meta-analysis of 57 studies comparing SGLT2-I monotherapy vs. placebo or active comparator showed reductions in major cardiovascular events, but no impact on atherothrombotic events. In fact, a non-significant increase in the risk for non-fatal stroke was observed. Similar trend observed in multiple trials indicate a SGLT2-i class effect. Sotagliflozin is the first dual SGLT1/2 receptor inhibitor, that was shown to significantly reduce atherothrombotic events compared with placebo in diabetic HF patients, suggesting that dual SGLT1/2 inhibitor may have additional properties vs. SGLT2-i. The hypothesis of this study is that dual SGLT1/2 inhibition by sotagliflozin improves thrombogenic profile (i.e. reduces thrombus formation), which could make it a safer and more effective treatment option for cardiovascular (CV) patients than SGLT2-i. To test the hypothesis, the researchers will compare the antithrombotic activity of sotagliflozin vs. empagliflozin in healthy volunteers using a randomized, cross-over study design, where each participant will receive both study treatments (sotagliflozin and empagliflozin) separated by a washout period. Treatment effects will be assessed by measuring ex vivo thrombus formation using the Badimon Perfusion chamber, platelet aggregation using Multiplate Analyzer, and Thromboelastometry using RoTEM Gamma. Study assessments will be performed before initiating (baseline/pre-treatment) and after completion of each treatment.

Start Date01 Jan 2025

Sponsor / Collaborator

Icahn School of Medicine at Mount Sinai

100 Clinical Results associated with Sotagliflozin

100 Translational Medicine associated with Sotagliflozin

100 Patents (Medical) associated with Sotagliflozin

323

Literatures (Medical) associated with Sotagliflozin

01 Feb 2025·DIABETES OBESITY & METABOLISM

Effects of sotagliflozin on anaemia in patients with type 2 diabetes and chronic kidney disease stages 3 and 4

Letter

Author: Cherney, David Z. I. ; Davies, Michael J. ; Carroll, Amy K. ; Girard, Manon ; Sridhar, Vikas S. ; Banks, Phillip

01 Feb 2025·Cardiovascular drugs and therapy

Rationale and Design of the SOTA-P-CARDIA Trial (ATRU-V): Sotagliflozin in HFpEF Patients Without Diabetes

Article

Author: Fuster, Valentin ; Atallah-Lajam, Farah ; Abascal, Vivian ; Rodríguez-Capitán, Jorge ; Contreras, Johanna P ; Moreno, Pedro ; Urooj Zafar, M ; Fergus, Icilma ; Requena-Ibáñez, Juan Antonio ; Moss, Noah ; Lala, Anu ; Mitter, Sumeet ; Badimon, Juan José ; Santos-Gallego, Carlos G ; Escolar, Ginés ; Pérez, Maeve Soto ; Mancini, Donna ; Lerakis, Stamatios ; Sanz, Javier ; Lam, David

Heart failure with preserved ejection fraction (HFpEF) is now the most common form of heart failure (HF). This syndrome is associated with an elevated morbi-mortality, and effective therapies are urgently needed. Sodium-glucose co-transporter 2 inhibitors (SGLT2i) are the first pharmacological class that has demonstrated to reduce hospitalization and cardiovascular mortality in large clinical trials in HFpEF. Furthermore, the dual SGLT 1/2 inhibitor sotagliflozin has shown a reduction in cardiovascular outcomes in diabetic HF patients, regardless of ejection fraction Sotagliflozin on Cardiovascular Events in Patients with Type 2 Diabetes Post Worsening Heart Failure (SOLOIST-WHF) Trial, and prevents the development of HF in patients with diabetes and chronic kidney disease Sotagliflozin on Cardiovascular and Renal Events in Patients with Type 2 Diabetes and Moderate Renal Impairment Who Are at Cardiovascular Risk (SCORED) trial. The major objective of the Sotagliflozin in Heart Failure With Preserved Ejection Fraction Patients (SOTA-P-CARDIA) trial (NCT05562063) is to investigate whether the observed cardiorenal benefits of sotagliflozin in HF patients with diabetes can be extended to a non-diabetic population. The SOTA-P-CARDIA is a prospective, randomized, double-blinded, placebo-controlled study that will randomize non-diabetic patients with the universal definition of HFpEF (ejection fraction > 50% assessed the day of randomization). Qualifying patients will be randomized, in blocks of 4, to receive either sotagliflozin or placebo for a period of 6 months. The primary outcome is changes in left ventricular mass by cardiac magnetic resonance from randomization to end of the study between the groups. Secondary end points include changes in peak VO₂; myocardial mechanics, interstitial myocardial fibrosis, and volume of epicardial adipose tissue; distance in the 6-min walk test; and quality of life. Finally, the authors expect that this trial will help to clarify the potential benefits of the use of sotagliflozin in non-diabetic HFpEF patients.

01 Jan 2025·JACC. Advances

Novel Therapies to Reduce Rehospitalization Risk in Worsening Heart Failure

Article

Author: Lima, Ivna G C V ; Bocchi, Edimar A ; Godoy, Lucas C ; Nunes, Jairo T ; McDonald, Michael

Background:

Worsening heart failure (WHF) challenges health care with frequent rehospitalizations and reduced quality of life for patients. Despite therapeutic advances, high rehospitalization risks highlight the urgent need for new treatments.

Objectives:

This study evaluated the effectiveness of initiating novel therapies during hospitalization or vulnerable phase for WHF patients to reduce rehospitalization risks and determine the optimal treatment sequence.

Methods:

A systematic review and network meta-analysis were performed in accordance with Cochrane Collaboration and Preferred Reporting Items for Systematic Reviews and Meta-Analysis guidelines. We included randomized clinical trials from January 2013, to December 2022, sourced from PUBMED and EMBASE, comparing novel heart failure therapies against control. The primary outcome was heart failure rehospitalization.

Results:

Analysis of 9 randomized clinical trials involving 18,540 patients showed significant rehospitalization reductions with novel treatments, especially sodium-glucose co-transporter 2 inhibitors, angiotensin-neprilysin receptor inhibitor (ARNI), and ferric carboxymaltose. Sotagliflozin led to more pronounced reductions in heart failure hospitalization (OR: 0.50; 95% CI: 0.39-0.63; P < 0.0001), followed by ARNI (OR: 0.54; 95% CI: 0.35-0.83; P = 0.005), ferric carboxymaltose (OR: 0.55; 95% CI: 0.44-0.70; P < 0.0001), empagliflozin (OR: 0.56; 95% CI: 0.39-0.78; P = 0.0007), vericiguat 10 mg (OR: 0.83; 95% CI: 0.74-0.92; P = 0.0008), and finerenone 15 mg (OR: 0.59; 95% CI: 0.38-0.91; P = 0.016). These therapies, when initiated during hospitalization, markedly influenced rehospitalization outcomes.

Conclusions:

Early administration of sodium-glucose co-transporter 2 inhibitors, ARNI, and ferric carboxymaltose for WHF patients significantly reduces rehospitalization risk. Our findings support a strategic shift in WHF management, advocating for the rapid introduction of these novel therapies to enhance patient prognosis.

145

News (Medical) associated with Sotagliflozin

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

06 Jan 2025

·www.globenewswire.com

Lexicon Announces Receipt of Complete Response Letter for Zynquista™ (sotagliflozin)

Dec. 20, 2024 -- Lexicon Pharmaceuticals, Inc. (Nasdaq: LXRX) today announced it has received a complete response letter (CRL) from the U.S. Food and Drug Administration (FDA) regarding the New Drug Application (NDA) for Zynquista™ (sotagliflozin) as an adjunct to insulin therapy for glycemic control in adults with type 1 diabetes and chronic kidney disease (CKD). This expected communication from the FDA aligns with the company’s previously disclosed strategic decision to discontinue launch preparations for Zynquista and focus solely on its clinical development pipeline. "We are sincerely grateful to the patients and physicians who participated in our Zynquista™ clinical trials, and the broader diabetes community who strongly advocated for Zynquista’s approval," said Mike Exton, Ph.D., chief executive officer and director of Lexicon. “Although this was not our desired outcome for sotagliflozin in this indication, we remain steadfast in our commitment to advancing our clinical pipeline, including our near-term focus on LX9211 for diabetic neuropathic pain (DPNP) with top line data from our PROGRESS Phase 2b study anticipated in Q1 2025, and pursuing innovations that we believe can profoundly benefit patients." Lexicon is a biopharmaceutical company with a mission of pioneering medicines that transform patients’ lives. Through the Genome5000™ program, Lexicon’s unique genomics target discovery platform, Lexicon scientists studied the role and function of nearly 5,000 genes and identified more than 100 protein targets with significant therapeutic potential in a range of diseases. Through the precise targeting of these proteins, Lexicon is pioneering the discovery and development of innovative medicines to treat disease safely and effectively. Lexicon has a pipeline of promising drug candidates in discovery and clinical and preclinical development in neuropathic pain, hypertrophic cardiomyopathy (HCM), obesity, metabolism and other indications. For additional information, please visit www.lexpharma.com. The content above comes from the network. if any infringement, please contact us to modify.

NDA

02 Jan 2025

·medcitynews.com

FDA Wraps Up 2024 Handing Out Several Notable Regulatory Decisions - MedCity News

For many people, the end of the year is a mad rush to wrap things up before the holidays, and so it was for the FDA. Notable regulatory decisions include the first drug approval for a prevalent chronic condition and a novel regenerative medicine approach to help trauma patients. In one case, a new drug approval comes as its developer takes on a new identity in the new year. Here’s a look back at some highlights from a busy regulatory month: presented by Health IT Transforming Patient Care: The Role of AI-Powered Assistants The progress in artificial intelligence (AI) is reshaping patient care, across various dimensions, from facilitating faster discharge to curating treatment plans and suggesting lifestyle changes. By IT Medical Notable Firsts —The prevalent sleeping disorder obstructive sleep apnea has historically been managed with a medical device that helps breathing. Eli Lilly’s Zepbound is now the first FDA-approved drug treatment for the chronic condition. Obesity is a risk factor for sleep apnea and clinical trial results showed that weight reductions from treatment with Zepbound were accompanied by breathing improvement. Approval in obstructive sleep apnea adds another potential blockbuster indication for a metabolic medication that has fast become one of Lilly’s top-selling products. —Patients who have the rare inherited metabolic disorder familial chylomicronemia syndrome lack the ability to break down triglycerides, a type of fat from food. The only way to avoid potentially fatal complications to the pancreas is by maintaining an extremely restrictive diet. Approval of Ionis Pharmaceuticals’ olezarsen gives patients a therapeutic option. The once-monthly injected genetic medicine, which is designed to block the body’s production of a liver protein that regulates triglyceride metabolism, will be marketed under the brand name Tryngolza. Ionis previously brought drugs through late-stage development and commercialization under partnerships with larger companies. Tryngolza will be the first product Ionis commercializes on its own. —When trauma to an arm or leg requires replacement of a blood vessel, the standard treatment is grafting a vein from the patient or implanting a synthetic vein. Now there’s a new regenerative medicine option. Humacyte won FDA approval for Symvess, a bioengineered blood vessel for restoring blood flow to avoid the loss of a limb when grafting a vein from the patient is not feasible. Here’s more about the biotech’s regenerative technology. Sponsored Post The Funding Model for Cancer Innovation is Broken — We Can Fix It Closing cancer health equity gaps require medical breakthroughs made possible by new funding approaches. By Eve McDavid - CEO of Mission-Driven Tech —Mesoblast’s regulatory approval was a long time coming. In 2020 and 2023, the FDA turned down the Australian company’s application for remestemcel, an allogeneic cell therapy for graft versus host disease, an immune response that develops when donor T cells attack the recipient’s cells following a transplant procedure. Remestemcel, brand name Ryoncil, is a made from mesenchymal stromal cells sourced from the bone marrow of healthy donors. The product’s approval covers acute graft versus host disease that is refractory to treatment with steroids in patients age 2 months and older. It’s the first affirmative regulatory decision for a cell therapy made from mesenchymal stromal cells. Approvals in Immunology —Vtama, an Organon drug acquired from Roivant Sciences earlier this year, received FDA approval as a treatment for atopic dermatitis in adults and children age 2 and older. The drug is topical cream that was initially approved in 2022 as a treatment for plaque psoriasis. Approval in atopic dermatitis brings the product to a much larger dermatologic indication, albeit one served my many branded and generic medications. —In other atopic dermatitis news, Galderma landed FDA approval for nemolizumab, brand name Nemluvio. The drug is an antibody designed to block IL-31, a signaling protein associated with the itch and inflammation of the chronic skin disorder. FDA approval of Nemluvio covers use of the drug in patients age 12 and older who have moderate-to-severe atopic dermatitis. It’s the second approval in the past year for Nemluvio, which was first approved over the summer as a treatment for prurigo nodularis. Rare Disease Regulatory Decisions —Neurocrine Biosciences received approval for Crenessity, a treatment for the rare inherited hormone disorder congenital adrenal hyperplasia. The small molecule helps bring the hormone imbalance back to more normal levels. The FDA approved a pill formulation for adults and an oral solution for pediatric patients. Analysts project Crenessity could achieve blockbuster sales, pending regulatory approvals in other countries. —Novo Nordisk is best known for metabolic medicines, but its rare disease portfolio is getting a boost with FDA approval of Alhemo, a drug that reduces bleeding episodes in patients with either hemophilia A or B. Alhemo, known in development as concizumab, is an antibody designed to bind to TFPI, preventing that protein from blocking factor Xa, a different protein that plays a role in blood clotting. That’s the same mechanism of action as Pfizer’s Hympavzi, which won its FDA approval in October. Both drugs are subcutaneous injections that provide alternatives to intravenously infused hemophilia therapies. But Pfizer’s once-weekly Hmypavzi has a dosing edge over Alhemo, which must be injected once daily. —Vertex Pharmaceuticals is adding a new cystic fibrosis (CF) drug to its portfolio with FDA approval of Alyftrek, which combines three compounds in a single therapy. Like Vertex’s other CF therapies, Alyftrek is a modulator of the CFTR a protein that regulates the movement of chloride ions into and out of cells. Alyftrek’s approval is based on clinical data comparing the once-daily therapy to Trikafta, a Vertex triple combination drug initially approved in 2019 as a twice-daily CF treatment. Results showed Alyftrek was non-inferior to Trikafta on a key measure of lung function and was superior in reducing sweat chloride levels, which is a surrogate indicator of the function of CFTR proteins. Besides the dosing advantage, Alyftrek addresses 31 additional mutations that are not addressable by other CFTR modulators. The Dec. 20 approval of Alyftrek came nearly two weeks ahead of the Jan. 2 target date for a regulatory decision. Developments in Cancer Drugs —The FDA awarded accelerated approval to Merus Therapeutics drug zenacutuzumab as a treatment for advanced cases of two types of cancer: non-small cell lung cancer and pancreatic adenocarcinoma. It’s the first approval for a drug that addresses a genetic signature called an NRG1 gene fusion. Netherlands-based Merus will market the bispecific antibody under the brand name Bizengri. In a deal struck days prior to the approval announcement, Partner Therapeutics licensed U.S. commercialization rights to Bizengri. —The blockbuster AstraZeneca drug Imfinzi expanded its FDA-approved uses to include limited-stage small cell lung cancer that has not progressed following concurrent platinum-based chemotherapy and radiation therapy. The checkpoint inhibitor was first approved in 2017 for bladder cancer and added extensive-stage lung cancer as a new indication in 2020. The drug’s latest approval is based on Phase 3 results showing a 27% reduction in the risk of death compared to a placebo. AstraZeneca said the FDA decision makes Imfinzi the first immunotherapy approved for limited-stage small cell lung cancer. —Xcovery Holdings drug ensartinib, brand name Ensacove, was approved to treat adults with advanced cases of non-small cell lung cancer that is positive for ALK mutations. Patients prescribed the once-daily pill must not have previously received an ALK inhibitor. —Pfizer cancer drug Braftovi landed accelerated approval as a first-line treatment for metastatic colorectal cancer driven by the BRAF V600E mutation. The approval covers use of the drug in combination with Eli Lilly’s Erbitux and the chemotherapy regimen referred to as FOLFOX, both standard colorectal cancer therapies. Braftovi, a small molecule inhibitor of BRAF V600E, was initially approved in 2018 for advanced cases of melanoma. The drug came from Pfizer’s 2019 acquisition of Array Biopharma. —Tevimbra, a cancer immunotherapy developed by BeiGene, received FDA approval as a first-line treatment for gastric and gastroesophageal junction cancers when used in combination with chemotherapy. It’s the second FDA approval for the checkpoint inhibitor, which was approved last March for treating advanced or metastatic esophageal squamous cell carcinoma after prior treatment with chemotherapy. The new year means a new identity for BeiGene. The cancer drug developer is changing its name to BeOne Medicines. Starting Jan. 2, the company’s stock symbol on the Nasdaq will be “ONC.” Rejections, Warnings & More Bad News in Biotech —Astellas Pharma’s bid to bring patients less-frequent eye injections of its drug Izervay was rejected by the FDA. Izervay, approved for treating geographic atrophy in 2023, is administered monthly to slow progression of the vision-loss disorder. Astellas sought approval for every-other-month dosing based on two-year Phase 3 data. According to Astellas, no safety benefit/risk issues were cited but the FDA took issue with a statistical matter related to labelling language proposed by the company. The rejection will limit Izervay’s ability to compete against Apellis Pharmaceuticals’ geographic atrophy drug Syfovre, which is approved for both monthly and every-other-month dosing. —In other Astellas news, the label for menopause drug Veozah now sports a black box warning for the risk of serious liver injury. The warning follows a FDA safety communication issued in September after a postmarketing report of a patient who developed signs and symptoms of liver injury after taking the once-daily pill for about 40 days. Veozah won FDA approval in 2023 as the first in a new class of therapies for menopause. —Applied Therapeutics received a double dose of bad regulatory news. First the FDA rejected the biotech’s application for govorestat, a drug developed as a treatment for the rare metabolic disease galactosemia. According to Applied, the agency cited “deficiencies in the clinical application.” Days after the FDA complete response letter, the FDA sent Applied a warning letter. The trial and the drug are redacted in the public version of the letter, but in a regulatory filing, Applied acknowledged the FDA’s concerns are about the govorestat galactosemia trial. Applied said the warning letter identified issues with electronic data capture and a dosing error in the dose-escalation portion of the study — both of which the company believed it had already addressed with the agency. Applied said it would respond to the FDA warning letter. —Bad news keeps stacking up for Intercept Pharmaceuticals and its drug, Ocaliva, a treatment for the rare liver disease primary biliary cholangitis (PBC). On Dec. 12, the FDA issued a safety alert, warning of the risk of serious liver injury in patients without advanced liver cirrhosis. The alert came one month after the FDA turned down Intercept’s application seeking full approval for Ocaliva, which won its accelerated approval in PBC in 2016. The FDA’s rejection letter for the drug cited safety concerns. When Ocaliva first reached the market, it was the only FDA-approved second-line treatment for PBC. In the past year, drugs from Ipsen and Gilead Sciences have each won accelerated approvals as new second-line therapies for the rare disease. Two weeks after the FDA turned down full approval of Ocaliva, the European Commission revoked the conditional marketing authorization for the drug. Advanz Pharma holds rights to Ocaliva in Europe. The company said the commission decision is subject to an ongoing annulment procedure in the EU’s General Court and a ruling is expected in 2025. —The FDA turned down Zealand Pharma’s glepaglutide as a treatment for short bowel syndrome, a rare disorder that develops when the small intestine is damaged or shortened, making it difficult for the organ to absorb nutrients. The drug is a long-acting GLP-2 analog intended to enhance the intestine’s ability to absorb nutrients, reducing patients’ dependence on intravenous feeding. According to Zealand, the FDA said the application needed more evidence of efficacy and safety. The company said it would discuss the letter with the FDA and proceed with plans to seek European approval in 2025. —Lexicon Pharmaceuticals came up short in its bid to expand approval of its drug, sotagliflozin (brand name Zynquista), to include the treatment of adults with type 1 diabetes and chronic kidney disease. The FDA’s Dec. 20 rejection of the drug followed a negative FDA advisory committee vote in October. The FDA approved sotagliflozin last year as a treatment for heart failure; it’s marketed in this indication under the brand name Inpefa. —Johnson & Johnson received a complete response letter for its injectable version of Rybrevant, a drug that treats cancer driven by EGFR mutations. Intravenously infused Rybrevant was approved in 2021 as a treatment for non-small cell lung cancer. The injectable version is made with technology from Halozyme. J&J said the FDA letter flagged manufacturing issues and did not cite any concerns about the new formulation or its safety and efficacy.

Drug ApprovalImmunotherapyAccelerated ApprovalAcquisitionClinical Result

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D10669	Sotagliflozin	A10BK06	DB12713

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Heart Failure	US	Lexicon Pharmaceuticals, Inc.	26 May 2023
Diabetes Mellitus, Type 1	EU	NCI Healthcare LLC	26 Apr 2019
Diabetes Mellitus, Type 1	IS	NCI Healthcare LLC	26 Apr 2019
Diabetes Mellitus, Type 1	LI	NCI Healthcare LLC	26 Apr 2019
Diabetes Mellitus, Type 1	NO	NCI Healthcare LLC	26 Apr 2019

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Chronic Kidney Diseases	NDA/BLA	US	Lexicon Pharmaceuticals, Inc.	21 Jun 2024
Hypertrophic Cardiomyopathy without Obstruction	Phase 3	US	Lexicon Pharmaceuticals, Inc.	24 Sep 2024
Hypertrophic obstructive cardiomyopathy	Phase 3	US	Lexicon Pharmaceuticals, Inc.	24 Sep 2024
Diabetes Mellitus, Type 2	Phase 3	US	Lexicon Pharmaceuticals, Inc.	01 Nov 2016
Diabetes Mellitus, Type 2	Phase 3	US	Sanofi	01 Nov 2016
Diabetes Mellitus, Type 2	Phase 3	CA	Lexicon Pharmaceuticals, Inc.	01 Nov 2016
Diabetes Mellitus, Type 2	Phase 3	CA	Sanofi	01 Nov 2016
Diabetes Mellitus, Type 2	Phase 3	HU	Lexicon Pharmaceuticals, Inc.	01 Nov 2016
Diabetes Mellitus, Type 2	Phase 3	SK	Lexicon Pharmaceuticals, Inc.	01 Nov 2016
Hyperglycemia	Phase 3	US	Lexicon Pharmaceuticals, Inc.	01 Sep 2015

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


Pubmed Manual	Not Applicable	Diabetes Mellitus, Type 1 \| Chronic Kidney Diseases	-	Sotagliflozin 200mg (CKD)	akhvbpfeyj(xwmbczpmlc) = wjxlkhgfjt rjhyeonnup (ygnudazzek ) View more	Positive	01 Nov 2024
				Sotagliflozin 400mg (CKD)	akhvbpfeyj(xwmbczpmlc) = wzmmivpgxa rjhyeonnup (ygnudazzek ) View more
NCT03285594 (SOTA-INS, GlobeNewswire) Manual	Phase 3	Diabetes Mellitus, Type 2	-	Sotagliflozin 400 mg	ydqsxunkug(zmfstrprvo) = ljaenpvryn jhtjfmavic (nbxowmxlxi )	Positive	10 Sep 2024
				Sotagliflozin 200 mg	ydqsxunkug(zmfstrprvo) = zisrxsqhqc jhtjfmavic (nbxowmxlxi )
NCT03521934 (SOLOIST-WHF, Pubmed \| J Am Coll Cardiol)	Phase 3	Diabetes Mellitus, Type 2 \| Heart Failure	-	Sotagliflozin	khuxuyhnzn(xrodqtgaep) = The baseline KCCQ-12 score was low overall (median: 41.7; Q1-Q3: 27.1-58.3) and improved by 4 months in both groups fjlwbkycag (rvgwenasrr )	Positive	01 Sep 2024
				Placebo
ESC2024	Not Applicable	Diabetes Mellitus	-	Sotagliflozin	glakszzmyz(deuvirkrfq) = lpaqbbovpu yzhyzvbsnz (bxsmpyxzfh ) View more	-	30 Aug 2024
				Placebo	glakszzmyz(deuvirkrfq) = nkobzrkaby yzhyzvbsnz (bxsmpyxzfh ) View more
924-P (ADA2024)	Phase 3	Diabetes Mellitus, Type 2 Add-on	570	Sotagliflozin 400 mg	vqpxhkehyv(pkuoaqtehy) = inzkcusgah rudwkjughi (lfmonomuwm )	Positive	14 Jun 2024
				Sotagliflozin 200 mg	vqpxhkehyv(pkuoaqtehy) = zkgddelrut rudwkjughi (lfmonomuwm )
917-P (ADA2024)	Phase 3	Diabetes Mellitus, Type 2	770	Sotagliflozin 400 mg	yhxblxvksj(vokcandreg) = Other adverse events in the SOTA group aligned with expectations associated with SGLTi therapy fyycyjxahb (wlngmtritl ) View more	Positive	14 Jun 2024
				Empagliflozin 25 mg
NCT03315143 (SCORED, GlobeNewswire) Manual	Phase 3	Heart Failure	1,932	INPEFA® (sotagliflozin)	traiwvvmst(pmoqspfbdj) = guzvnxskum izyedyolca (rmyyulkxlb )	Positive	14 May 2024
SOLOIST-WHF and SCORED (ESC2024)	Not Applicable	obesity \| type 2 diabetes (T2D)	-	Sotagliflozin	ormvfswsfm(alevjfxgyg) = ljauuclzaq qgjwjrmltz (batymzoqkh )	Positive	14 May 2024
NCT03315143 (SCORED, ACC2024) Manual	Phase 3	Cardiovascular Diseases \| Diabetes Mellitus, Type 2 \| Chronic Kidney Diseases	10,584	Sotagliflozin	zqewkupfzn(qpxzchjili) = poemjzfcgd dzuhgeimiy (plcrkfkpfu ) View more	Positive	25 Mar 2024
				Placebo	zqewkupfzn(qpxzchjili) = lmfzoqmbno dzuhgeimiy (plcrkfkpfu ) View more
NCT03315143 (SCORED, Pubmed) Manual	Phase 3	Diabetes Mellitus, Type 2 \| Chronic Kidney Diseases	10,584	sotagliflozin	nxlwaulfsn(sqjzeycmwy) = rwpsayvhfz dtghqpdagw (mpgisbvkoo ) View more	Positive	26 Jan 2024
				placebo	nxlwaulfsn(sqjzeycmwy) = ujbanwkeah dtghqpdagw (mpgisbvkoo ) View more

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