Clinical Trial News
FDA Upgrades Xinnate's TCP-25 Trial for Epidermolysis Bullosa to Registrational Status
• Following a successful Type C meeting with the FDA, Xinnate's upcoming STEP trial for TCP-25 in Epidermolysis Bullosa patients has been upgraded to serve as a registrational trial.
• TCP-25, an immunomodulatory peptide with dual-action capabilities targeting both inflammation and bacterial infection, previously received Orphan Drug Designation from the FDA for EB treatment.
• The STEP trial, planned to begin in 2025, is designed as an international multicenter, randomized, double-blind, placebo-controlled study to assess TCP-25's efficacy and safety in dystrophic and junctional EB patients.
Merck and imec Partner to Develop Advanced Microphysiological Systems Platform for Drug Discovery
• Merck and imec have announced a strategic partnership to develop an advanced Microphysiological Systems (MPS) platform that aims to improve drug discovery efficiency and reduce reliance on animal testing.
• The collaboration integrates organoid biology models with semiconductor hardware, incorporating biosensing and microfluidic capabilities to provide real-time insights from individual organs to multi-organ connected systems.
• The jointly developed modular system will feature standardized interfaces compatible with Merck's portfolio of stem cells and patient-derived organoids, enabling more reliable predictions of human organ responses to drug stimuli.
Enibarcimab Shows Promising Results in Biomarker-Guided Septic Shock Treatment
• Journal of Critical Care has published positive results from AdrenoMed's Phase II AdrenOSS-2 trial, showing enibarcimab improved organ function and reduced 28-day mortality in septic shock patients.
• The precision medicine approach used two biomarkers (bio-ADM and DPP3) to identify patients most likely to benefit from treatment, with mortality rates of 18% in the enibarcimab group versus 26% in placebo.
• Based on these promising findings, AdrenoMed is advancing to a confirmatory Phase IIb/III BOOST trial, potentially addressing a critical unmet need in septic shock treatment where mortality rates remain at 30-50%.
Highlighted Clinical Trials:
Radboud University Medical Center
Posted 5/23/2016
Adrenomed AG
Posted 12/12/2017
Real-World Data Confirms Trikafta's Long-Term Efficacy as Gold Standard in Cystic Fibrosis Treatment
• New real-world evidence presented at ATS 2025 validates Trikafta (elexacaftor/tezacaftor/ivacaftor) as an effective long-term therapy for cystic fibrosis patients with at least one F508del mutation.
• The study by Sutharsan and colleagues provides valuable insights into laboratory markers while demonstrating sustained clinical benefits across various outcomes in this patient population.
• These findings further establish Trikafta's position as the gold standard treatment in long-term cystic fibrosis care, reinforcing its therapeutic value for patients.
Novel Drug Combination Shows Promise for B-Cell Acute Lymphoblastic Leukemia with Reduced Toxicity
• Cambridge researchers have developed a promising combination therapy using venetoclax and inobrodib that effectively targets B-cell acute lymphoblastic leukemia (B-ALL), potentially reducing the need for toxic chemotherapy.
• The drug combination works by exploiting cancer cell metabolism, triggering ferroptotic cell death even in cells resistant to venetoclax alone, offering new hope for both pediatric and adult patients.
• Unlike current treatments that require over two years of chemotherapy or risky procedures like bone marrow transplants, this oral therapy could provide a less toxic alternative while maintaining effectiveness.
University of Virginia Expands Manning Institute of Biotechnology with $66M Additional Contract
• Skanska has secured an additional $66 million contract with the University of Virginia to construct the Paul and Diane Manning Institute of Biotechnology, bringing advanced translational research capabilities to the Commonwealth.
• The 354,000-square-foot facility will feature modular laboratories, expanded research spaces, and dedicated areas for up to 150 researchers and biotechnology companies to collaborate on cutting-edge science.
• Construction of this first-of-its-kind research program in Virginia began in December 2023 and is scheduled for completion in the fourth quarter of 2027.
NextCell Pharma and Fujifilm Irvine Scientific Form Strategic Alliance to Advance MSC Therapy Development
• NextCell Pharma has partnered with Fujifilm Irvine Scientific to combine their expertise in mesenchymal stromal cells and cell culture solutions for the life science sector.
• The collaboration will provide researchers with an integrated offering of standardized umbilical cord MSC products, optimized culture media, and cryopreservation solutions.
• This strategic alliance builds on NextCell's 2024 expansion into research-use-only MSC products and leverages Fujifilm's 50+ years of experience in developing high-quality cell culture technologies.
Regeneron Develops Novel Antibody Approaches to Enable AAV Gene Therapy Redosing
• Regeneron has demonstrated successful AAV gene therapy redosing in non-human primates using two antibody-based strategies designed to suppress neutralizing antibody formation.
• The company's approaches include a CD40 blockade method and a CD20xCD3 bispecific antibody that depletes B-cells in lymph nodes and spleen, both showing superior efficacy compared to conventional rituximab.
• These transient immunomodulation techniques could potentially overcome one of gene therapy's major limitations by allowing patients to receive multiple doses of AAV-based treatments.
VERAXA Biotech Appoints Oncology Veteran Rick Austin as Chief Scientific Officer to Advance Novel Cancer Therapies
• Dr. Rick Austin, with over 25 years of oncology drug development experience, joins VERAXA Biotech as Chief Scientific Officer to advance the company's BiTAC platform and clinical-stage pipeline.
• Austin brings extensive expertise from leadership roles at Harpoon Therapeutics, Amgen, and Tularik, where he led tumor immunology projects and enabled numerous IND filings and first-in-human studies.
• VERAXA, an emerging leader in novel cancer therapies and proposed acquisition target of Voyager Acquisition Corp., aims to leverage Austin's expertise to expand therapeutic windows for antibody drug conjugates and T-cell engagers.
New Global Review Reveals Comprehensive Landscape of Interstitial Cystitis Clinical Trials in 2025
• A newly released global clinical trials review provides extensive data on Interstitial Cystitis research, tracking trial numbers and enrollment trends across major countries worldwide.
• The report categorizes trials by region, phase, status, and sponsorship, highlighting key pharmaceutical companies including Astellas Pharma, AbbVie, Johnson & Johnson, and Pfizer as prominent sponsors.
• This comprehensive analysis serves as a strategic resource for stakeholders making investment decisions in IC research, offering insights into trial success rates and identifying optimal locations for clinical studies.