Latest news with #NAVIGATE
Business Wire
2 days ago
- Business
- Business Wire
Azafaros Announces Initiation of two Global Phase 3 studies with Nizubaglustat in Niemann-Pick disease Type C (NPC) and GM1/GM2 gangliosidoses, respectively
LEIDEN, Netherlands--(BUSINESS WIRE)-- Azafaros, a company focused on developing treatments for the unmet needs of patients with rare lysosomal storage disorders, today announced that the first patient has been dosed in the company's pivotal, multicenter Phase 3 clinical program to evaluate the safety and efficacy of the company's lead asset, nizubaglustat, in patients with Niemann-Pick disease Type C (NPC) and GM1/GM2 gangliosidoses. 'The dosing of the first patient in our Phase 3 program with nizubaglustat is a significant achievement for Azafaros, and a huge step forward in our efforts to bring new, disease modifying treatments to patients with these seriously debilitating diseases,' Share The initiation of the two Phase 3 studies (NCT07054515) represents a major milestone in Azafaros' commitment to addressing the urgent unmet medical needs of children affected by these devastating neurodegenerative disorders. The Phase 3 program consists of two studies targeting the late-infantile and juvenile-onset forms of NPC, and GM1/GM2 gangliosidoses. The studies aim to assess the potential of nizubaglustat to alter disease progression and improve functional outcomes in these patient populations. Today's news follows the recent, successful completion of an oversubscribed series B financing, raising €132M to support the acceleration of nizubaglustat and the expansion of the company's pipeline to other indications. 'The dosing of the first patient in our Phase 3 program with nizubaglustat is a significant achievement for Azafaros and a huge step forward in our efforts to bring new, disease modifying treatments to patients with these seriously debilitating diseases,' said Stefano Portolano, Chief Executive Officer at Azafaros. 'We are deeply grateful to the patients, families, clinicians, and advocacy groups who are partnering with us to advance this promising therapy.' About the NAVIGATE trial The two 18-month randomized 2 to 1, double-blind, placebo-controlled trials will recruit patients at approximately 35 sites across 15 countries worldwide, including in the US, Europe and Latin America. The studies are expected to enroll around 70 patients. The primary endpoint for both trials is the change from baseline to Month 18 in the Scale for the Assessment and Rating of Ataxia (SARA), with both total and functional SARA scores evaluated. For more information on the Phase 3 program, please visit To enquire about trial participation, email: medinfo@ (if a professional) or patientadvocacy@ (if a patient or caregiver). To protect privacy, avoid including identifying information in the initial message. About nizubaglustat Nizubaglustat is a small molecule, orally available and brain penetrant azasugar with a unique dual mode of action, developed as a potential treatment for rare lysosomal storage disorders with neurological involvement, including GM1 and GM2 gangliosidoses and Niemann-Pick disease type C (NPC). Nizubaglustat has received Rare Pediatric Disease Designations (RPDD) for the treatment of GM1 and GM2 gangliosidoses and NPC, Orphan Drug Designations (ODD) for GM1 and GM2 gangliosidosis (Sandhoff and Tay-Sachs Diseases) and NPC, as well as Fast Track Designation and IND clearance for GM1/GM2 gangliosidoses and NPC from the US Food and Drug Administration (FDA). Additionally, nizubaglustat has been awarded Orphan Medicinal Product Designation (OMPD) for the treatment of GM1 and GM2 gangliosidoses by the European Medicines Agency (EMA) and Innovation Passport for the treatment of GM1 and GM2 gangliosidoses from the UK Medicines and Healthcare Products Regulatory Agency (MHRA). About GM1 and GM2 gangliosidoses GM1 gangliosidosis and GM2 gangliosidosis (Tay-Sachs and Sandhoff diseases) are lysosomal storage disorders caused by the accumulation of GM1 or GM2 gangliosides respectively, in the central nervous system (CNS). This results in progressive and severe neurological impairment and premature death. These diseases mostly affect infants and children, and no disease-modifying treatments are currently available. About Niemann-Pick disease Type C (NPC) Niemann-Pick disease Type C is a progressive, life-limiting, neurological, lysosomal storage disorder, caused by mutations in the NPC1 or NPC2 gene and aberrant endosomal-lysosomal trafficking, leading to the accumulation of various lipids, including gangliosides in the CNS. The onset of disease can happen throughout the lifespan of an affected individual, from prenatal life through adulthood. About Azafaros Azafaros is a clinical-stage company founded in 2018 with a deep understanding of rare genetic disease mechanisms using compound discoveries made by scientists at Leiden University and Amsterdam UMC and is led by a team of highly experienced industry experts. Azafaros aims to build a pipeline of disease-modifying therapeutics to offer new treatment options to patients and their families. By applying its knowledge, network and courage, the Azafaros team challenges traditional development pathways to rapidly bring new drugs to the rare disease patients who need them. Azafaros is supported by leading healthcare investors including Forbion, Jeito Capital, Seroba, Pictet Group, BioGeneration Ventures (BGV), BioMedPartners, Asahi Kasei Pharma Ventures, and Schroders Capital.
Business Wire
07-07-2025
- Health
- Business Wire
Segal Trials Contributes to Breakthrough Flu Prevention Study Led by Cidara Therapeutics
BUSINESS WIRE)--Segal Trials proudly announces its participation in the successful Phase 2b NAVIGATE clinical trial conducted by Cidara Therapeutics, evaluating CD388 —a novel, non-vaccine, long-acting antiviral—for the prevention of seasonal influenza. Dr. Steven Chavoustie served as the Principal Investigator for the study at Segal Trials. 'We are honored to have been part of a study that marks a potential paradigm shift in how we approach flu prevention,' said Dr. Steven Chavoustie, Principal Investigator at Segal Trials. The NAVIGATE trial enrolled over 5,000 healthy, unvaccinated adults and met both its primary and secondary efficacy endpoints, showing that single doses of CD388 conferred 76%, 61%, and 58% protection, respectively, across three dosage levels. Participants were monitored for symptomatic, laboratory-confirmed influenza over a 24-week period. CD388 was well-tolerated at all doses with no unexpected safety signals reported. 'We are honored to have been part of a study that marks a potential paradigm shift in how we approach flu prevention,' said Dr. Steven Chavoustie, Principal Investigator at Segal Trials. 'A once-per-season antiviral could significantly improve access to flu protection, especially among individuals for whom vaccines may not be effective.' Unlike vaccines, CD388 does not rely on the body's immune response, making it a promising option for those with compromised immunity or heightened risk for severe flu. This innovative approach represents a potential new standard of care for broad seasonal influenza protection. Following the trial's success, Cidara Therapeutics has submitted an End of Phase 2 meeting request to the U.S. Food and Drug Administration (FDA) to discuss the upcoming Phase 3 trial design. About Segal Trials Founded in 1998, Segal Trials is a privately held clinical research network with multiple sites across South Florida. Specializing in psychiatry, neurology, women's health, and general medicine, Segal Trials is committed to advancing medical research through high-quality Phase I–IV trials while helping sponsors meet enrollment goals.
Business Wire
25-06-2025
- Health
- Business Wire
New Hope for Chronic Nausea: Enterra Medical Begins NAVIGATE Study After Gaining FDA IDE Approval and Breakthrough Device Designation
ST. LOUIS PARK, Minn.--(BUSINESS WIRE)--Enterra Medical, Inc., a leader in innovative treatments for gastrointestinal disorders, has initiated the NAVIGATE study to evaluate the safety and effectiveness of Gastric Electrical Stimulation (GES) as a treatment for patients with chronic nausea that have normal gastric emptying. This study was approved as an IDE study by the U.S. Food and Drug Administration (FDA) and was subsequently granted Breakthrough Device designation. NAVIGATE and its potential therapy indication represent a promising advancement for a patient population with extremely limited treatment options. Unlike previous GES studies that focused on patients with refractory gastroparesis, this study is evaluating patients who experience chronic nausea with severe symptoms, despite normal gastric emptying. The NAVIGATE study will assess whether the Enterra ® Therapy System provides safe and effective improvement for nausea severity, vomiting frequency, and quality of life measures for individuals who have not found relief through available diet and drug therapies. Unlike previous GES studies that focused on patients with refractory gastroparesis, this study is evaluating patients who experience chronic nausea with severe symptoms, despite normal gastric emptying. Importantly, NAVIGATE also allows enrollment of patients who have previously undergone pyloric interventions. 'Patients with normal gastric emptying can experience symptoms equally severe as those with delayed emptying, making us question if gastric emptying status is as central to patients' symptoms as previously believed,' said Prof. Jan Tack, Global Principal Investigator for the NAVIGATE study. While patients with refractory gastroparesis have been treated with GES for over two decades, the NAVIGATE study aims to expand the patient population that may benefit from GES therapy. NAVIGATE is a multi-center, randomized, double-blind, placebo-controlled study that will enroll at least 148 subjects across leading medical institutions throughout the U.S., Europe, and the United Kingdom. The first patient was enrolled at the University of Louisville by the team of Gastroenterologist Dr. Abigail Stocker and Surgeon Dr. John Olsofka. Find all currently enrolling centers for the NAVIGATE study on About Enterra Therapy Enterra Therapy was approved by FDA in 2000 under a Humanitarian Device Exemption (HDE). A unique therapy called Gastric Electrical Stimulation (GES), Enterra Therapy delivers mild electrical pulses to the nerves and smooth muscles of the stomach to control the chronic nausea and vomiting symptoms associated with gastroparesis. To learn more about Enterra Therapy visit the company's website and follow us on LinkedIn. About Enterra Medical Enterra Medical was founded in 2022 to help people living with chronic nausea and vomiting find the relief to take back their seat at the table. Learn more and view important safety information at Enterra ® is a registered trademark of Enterra Medical, Inc. in the US, EU, and other regions. This press release contains 'forward-looking statements' concerning the development of Enterra Medical's products, the benefits and attributes of such products, and the company's expectations regarding its prospects. Forward-looking statements are subject to risks, assumptions and uncertainties that could cause actual future events or results to differ materially from such statements. These statements are made as of the date of this press release. Actual results may vary. Enterra Medical undertakes no obligation to update any forward-looking statements for any reason.
Yahoo
10-06-2025
- Health
- Yahoo
Galectin Therapeutics to Host Virtual KOL Event to Discuss Belapectin for Treatment of MASH Cirrhosis and Portal Hypertension
NORCROSS, Ga., June 10, 2025 (GLOBE NEWSWIRE) -- Galectin Therapeutics Inc. (NASDAQ:GALT), the leading developer of therapeutics that target galectin-3 for patients with MASH cirrhosis and portal hypertension, today announced that it will host a virtual key opinion leader (KOL) event on Monday, June 16, 2025 at 12:00 PM ET. To register, click here. The event will focus on the unmet need and current treatment landscape for metabolic dysfunction-associated steatohepatitis (MASH) cirrhosis and portal hypertension. Featured KOLs will include: Naga P. Chalasani, MD, Professor of Gastroenterology and Hepatology, Adjunct Professor of Anatomy, Cell Biology and Physiology, & Director of Terance Kahn Liver Research Program, Indiana University School of Medicine; and Naim Alkhouri, MD, FAASLD, DABOM, Chief Academic Officer, Summit Clinical Research, & Director of the Steatotic Liver Program, North Shore Gastroenterology, Cleveland, OH. The event will highlight results from Galectin's NAVIGATE Phase 3 clinical trial evaluating belapectin for the treatment of MASH cirrhosis and portal. Belapectin is a complex carbohydrate drug that targets galectin-3, a critical protein in the pathogenesis of MASH and fibrosis. With no approved therapies to prevent or treat varices in MASH cirrhosis, belapectin may represent the first potential treatment to address this significant unmet need. A live question and answer session will follow the formal presentations. About Naga P. Chalasani, MD Naga P. Chalasani, MD completed medical school in India before he moved to the US to pursue Internal Medicine residency at Emory University in Atlanta, followed by a fellowship in Gastroenterology and Hepatology at the same institution. He joined IU in 1997 as an Assistant Professor in the Division of Gastroenterology and Hepatology. Dr. Chalasani is currently David W. Crabb Professor of Gastroenterology and Hepatology and Adjunct Professor of Anatomy, Cell Biology & Physiology and as Director of Terance Kahn Liver Research Program at Indiana University School of Medicine. He previously served as the GI Division Chief (2007-2020), Associate Dean for Clinical Research (2017-2020) interim Chair of the Department of Medicine (2020-2021) and Vice President for Academic Affairs at Indiana University Health (2022-2024). He is a highly regarded clinician and ranked as Top Doctor continuously for over 15 years. He has been continuously funded by the National Institutes of Health (NIH) since 1999 and is currently the PI on several U01 and R01 awards from the NIH. Dr. Chalasani is considered an authority in the fields of Non-Alcoholic Fatty Liver Disease (NAFLD) and Drug Induced Liver Injury (DILI), two highly significant public health problems. He is one of the most published and most cited investigators at Indiana University, with Google Scholar H-Index 120 and 75,000 citations (as of Feb 2025). He is an elected member of the American Society of Clinical Investigation (ASCI), the American Association of Physicians (AAP) and the National Academy of Medical Sciences – India (2024). He has mentored over 75 undergraduate and graduate students, medical students, sub-specialty trainees, and junior faculty. Twenty of his mentees are recipients of federal funding (K award, U01, R21, R01, DOD, and VA Merit Review). To honor his contributions and mentorship, Naga P Chalasani Endowed Professor in Gastroenterology and Hepatology has been established at Indiana University School of Medicine in October 2022. About Naim Alkhouri, MD, FAASLD, DABOM Naim Alkhouri, MD, FAASLD, DABOM is the Chief Academic Officer at Summit Clinical Research and the Director of the Steatotic Liver Program at North Shore Gastroenterology in Cleveland, OH. Prior to joining Summit, Dr. Alkhouri served as the Chief Medical Officer and Director of the Steatotic Liver Disease program at Arizona Liver Health in Phoenix, AZ. Dr. Alkhouri is a key opinion leader in the field of MASH therapeutics and an advisor/ consultant to many pharmaceutical and biomarker development companies. He is Principal Investigator on several multicenter global MASH trials and a member of the AASLD MASLD Special Interest Group (MASLD SIG). Dr. Alkhouri has been published in over 260 publications to include publications in the New England Journal of Medicine, Lancet, JAMA, Nature Medicine, Gastroenterology, Hepatology, and Journal of Hepatology. He presents his work at both national and international medical conferences. About Galectin Therapeutics Galectin Therapeutics is dedicated to developing novel therapies to improve the lives of patients with chronic liver disease and cancer. Galectin's lead drug belapectin is a carbohydrate-based drug that inhibits the galectin-3 protein, which is directly involved in multiple inflammatory, fibrotic, and malignant diseases, for which it has Fast Track designation by the U.S. Food and Drug Administration. The lead development program is in metabolic dysfunction-associated steatohepatitis (MASH, formerly known as nonalcoholic steatohepatitis, or NASH) with cirrhosis, the most advanced form of MASH-related fibrosis. Liver cirrhosis is one of the most pressing medical needs and a significant drug development opportunity. Additional development programs are in treatment of combination immunotherapy for advanced head and neck cancers and other malignancies. Advancement of these additional clinical programs is largely dependent on finding a suitable partner. Galectin seeks to leverage extensive scientific and development expertise as well as established relationships with external sources to achieve cost-effective and efficient development. Additional information is available at Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These statements relate to future events or future financial performance, and use words such as 'may,' 'estimate,' 'could,' 'expect', 'look forward', 'believe', 'hope' and others. They are based on management's current expectations and are subject to factors and uncertainties that could cause actual results to differ materially from those described in the statements. These statements include those regarding the hope that Galectin's development program for belapectin will lead to the first therapy for the treatment of MASH, formerly known as NASH, with cirrhosis and those regarding the hope that our lead compounds will be successful in cancer immunotherapy and in other therapeutic indications. Factors that could cause actual performance to differ materially from those discussed in the forward-looking statements include, among others, full analysis of the NAVIGATE trial data may not product positive data; Galectin may not be successful in developing effective treatments and/or obtaining the requisite approvals for the use of belapectin or any of its other drugs in development; the Company may not be successful in scaling up manufacturing and meeting requirements related to chemistry, manufacturing and control matters; the Company's current clinical trial and any future clinical studies may not produce positive results in a timely fashion, if at all, and could require larger and longer trials, which would be time consuming and costly; plans regarding development, approval and marketing of any of Galectin's drugs are subject to change at any time based on the changing needs of the Company as determined by management and regulatory agencies; regardless of the results of any of its development programs, Galectin may be unsuccessful in developing partnerships with other companies or raising additional capital that would allow it to further develop and/or fund any studies or trials. Galectin has incurred operating losses since inception, and its ability to successfully develop and market drugs may be impacted by its ability to manage costs and finance continuing operations. For a discussion of additional factors impacting Galectin's business, see the Company's Annual Report on Form 10-K for the year ended December 31, 2024, and subsequent filings with the SEC. You should not place undue reliance on forward-looking statements. Although subsequent events may cause its views to change, management disclaims any obligation to update forward-looking statements. Company Contact:Jack Callicutt, Chief Financial Officer(678) 620-3186ir@ Investor Relations Contact:Kevin Gardnerkgardner@ Galectin Therapeutics and its associated logo is a registered trademark of Galectin Therapeutics Inc. Belapectin is the USAN assigned name for Galectin Therapeutics' galectin-3 inhibitor belapectin.
Yahoo
18-03-2025
- Business
- Yahoo
Cidara Therapeutics Announces Two Presentations on CD388 in Influenza at International Conference on Antiviral Research (ICAR) 2025
SAN DIEGO, March 18, 2025 (GLOBE NEWSWIRE) -- Cidara Therapeutics, Inc. (Nasdaq: CDTX), a biotechnology company using its proprietary Cloudbreak® platform to develop drug-Fc conjugate (DFC) immunotherapies, today announced two upcoming presentations at the 38th International Conference on Antiviral Research (ICAR). The conference takes place from March 17-21, 2025 in Las Vegas, Nevada. Cidara's presentations will highlight the study design, demographic information, and preliminary safety data from the ongoing Phase 2b NAVIGATE trial of CD388, as well as dose optimization models for evaluation of CD388 in a Phase 3 study. Presentation details: Title: NAVIGATE: A Phase 2b, Randomized, Double-blind, Placebo-controlled, Multicenter Dose-ranging Study to Evaluate the Efficacy and Safety of CD388, a Novel Long-acting Antiviral Conjugate, for Prevention of Influenza in Subjects not at Risk for Influenza ComplicationsPresenter: James AlexanderAbstract number: 361 Date/Time: Poster Session 1: Tuesday, March 18, 2025, 5:15-6:15pm PT; Poster Session 2: Thursday, March 20, 2025, 9-10am PT Summary: The Phase 2b NAVIGATE trial to evaluate the safety and preventative efficacy of CD388 in a real-world environment completed enrollment of more than 5,000 participants in the United States and the UK. Prior Phase 1 and Phase 2a clinical data have shown that CD388, administered by subcutaneous injection, appeared to be well-tolerated and efficacious in healthy human volunteer studies. The results of the Phase 2b trial will inform dose selection and design for the planned Phase 3 development program. Title: Real World Data-Based Modeling of Seasonal Influenza Variations to Support Clinical Dose Selection of CD388, A Novel Antiviral in Development for Prevention of Seasonal and Pandemic Influenza Presenter: Shawn FlanaganAbstract number: 352 Date/Time: Poster Session 1: Tuesday, March 18, 2025, 6:15-7:15pm PT; Poster Session 2: Thursday, March 20, 2025, 8-9am PT Summary: In trials involving community acquired infections, like influenza, variations in disease incidence over time may complicate interpretation of drug effect in Phase 2 and Phase 3 trials across multiple seasons, especially for long-acting drugs like CD388. Advanced model-based analysis was conducted to improve decision power based on simulated Phase 2 results over different flu seasons. While a standard proportions test was shown to be sensitive to influenza seasonal variations, the model-based analysis was not and increased decision power for clinical dose selection of CD388 for Phase 3. About CD388CD388 is an investigational drug-Fc conjugate (DFC) comprised of multiple copies of a potent small molecule neuraminidase inhibitor stably conjugated to a proprietary Fc fragment of a human antibody. DFCs are not vaccines or monoclonal antibodies but are low molecular weight biologics which are designed to function as long-acting small molecule inhibitors. CD388 was designed to provide universal protection against all known strains of seasonal and pandemic influenza with the potential to provide season-long protection with a single subcutaneous or intramuscular administration. Importantly, because CD388 is not a vaccine, its activity is not reliant on an immune response and thereby is expected to be efficacious in individuals regardless of immune status. More information can be found at: About Cidara TherapeuticsCidara Therapeutics is using its proprietary Cloudbreak® platform to develop novel drug-Fc conjugates (DFCs) comprising targeted small molecules or peptides coupled to a proprietary human antibody fragment (Fc). Cidara's lead DFC candidate, CD388, is a long-acting antiviral designed to achieve universal prevention of seasonal and pandemic influenza with a single dose by directly inhibiting viral proliferation. In June 2023, CD388 was granted Fast Track Designation by the U.S. Food and Drug Administration (FDA), and the Company announced completion of enrollment of its Phase 2b NAVIGATE trial in December 2024. Additional DFCs have been developed for oncology and in July 2024 Cidara received IND clearance for CBO421 which is intended to target CD73 in solid tumors. Cidara is headquartered in San Diego, California. For more information, please visit Forward-Looking StatementsThis release contains 'forward-looking statements' within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and such forward-looking statements are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. 'Forward-looking statements' describe future expectations, plans, results, or strategies and are generally preceded by words such as 'anticipates,' 'expect,' 'intends,' 'believes,' 'may,' 'plan' or 'will'. Forward-looking statements in this release include, but are not limited to, statements related to the potential of and future plans for CD388, and promising CD388 clinical data generated to date. Such statements are subject to a multitude of risks and uncertainties that could cause future circumstances, events, or results to differ materially from those projected in the forward-looking statements, such as unanticipated delays in or negative results from Cidara's clinical trials and other risks related to clinical development, delays in action by regulatory authorities, other obstacles on the enrollment of patients or other aspects of CD388 or other DFC development and other risks and uncertainties associated with Cidara's business in general. These and other risks are identified under the caption 'Risk Factors' in Cidara's Annual Report on Form 10-K for the fiscal year ended December 31, 2024 and other filings subsequently made with the SEC. All forward-looking statements contained in this press release speak only as of the date on which they were made and are based on management's assumptions and estimates as of such date. Cidara does not undertake any obligation to publicly update any forward-looking statements, whether as a result of the receipt of new information, the occurrence of future events or otherwise. INVESTOR CONTACT:Brian RitchieLifeSci Advisors(212) 915-2578britchie@ MEDIA CONTACT:Michael FitzhughLifeSci Communications(628) 234-3889mfitzhugh@ in to access your portfolio
