MedPath

Tagged News

Guselkumab Shows Superior Treatment Persistence Over IL-17A Inhibitors in Psoriatic Arthritis Study

Monoclonal AntibodyReal World Evidence
  • A 24-month real-world study comparing Guselkumab and subcutaneous interleukin-17A inhibitors found that patients receiving Guselkumab demonstrated higher rates of treatment adherence in psoriatic arthritis management.
  • The research analyzed treatment persistence in real-world settings, moving beyond clinical trial conditions to evaluate how patients actually adhere to these therapeutic approaches over extended periods.
  • Statistical analysis revealed significant differences in treatment persistence between the two therapeutic modalities, with Guselkumab patients more likely to remain on therapy for the full study duration.
  • The findings emphasize the importance of patient-centered care and effective communication between healthcare providers and patients to improve long-term treatment outcomes in chronic inflammatory conditions.

iRhythm's Zio Long-Term ECG Monitoring Shows Consistent Performance Across Asian and Non-Asian Populations

AIReal World Evidence
  • A large-scale analysis of over 400,000 patients demonstrates that iRhythm's Zio 14-day ECG monitoring system performs consistently across Asian and non-Asian populations, with comparable wear times and arrhythmia detection rates.
  • Despite lower baseline prevalence of cardiovascular conditions, Asian patients showed similar atrial fibrillation detection rates (14.0% vs. 14.3%) and total arrhythmia yield (79.8% vs. 82.9%) compared to non-Asian patients.
  • The findings support the generalizability of long-term continuous ECG monitoring technology across diverse patient populations, addressing concerns about racial and cultural factors affecting device performance.
  • New research published in Heart Rhythm journal reveals that 24-48-hour Holter monitoring can miss actionable arrhythmias even in patients with daily symptoms, with 64% of daily-symptom patients requiring longer monitoring periods for detection.

Real-World CAR-T Therapy Outcomes Show Promise Despite Higher Toxicity Rates in Diverse Patient Population

CAR-TReal World Evidence
  • A five-year real-world study from Montefiore Health System demonstrates CAR-T therapy achieved 50.6% complete remission rates in a predominantly minority, medically complex population with diffuse large B-cell lymphoma.
  • Patients experienced higher rates of complications including cytokine release syndrome (76%) and immune effector cell-associated neurotoxicity syndrome (49%), with cumulative five-year mortality reaching 34.2%.
  • Complete remission following CAR-T therapy was strongly associated with reduced mortality risk (adjusted hazard ratio 0.13), while pre-existing heart failure and chronic kidney disease significantly increased death risk.
  • Rare complications including severe demyelination with quadriplegia, bullous skin reactions, and bilateral parotid gland enlargement were documented in separate case reports, expanding the known toxicity spectrum of CAR-T therapy.

Inka Health Publishes Framework to Enable Cross-Border Use of Real-World Evidence in Oncology

Real World EvidenceAIPrecision MedicineRare DiseaseOncology
  • Inka Health, a subsidiary of Onco-Innovations, published a technical primer in the Journal of Clinical Evaluative Research addressing how to adapt medical evidence from one country for use in another through advanced statistical transportability methods.
  • The framework outlines three foundational principles for reliable cross-border evidence sharing: consistency, positivity, and conditional exchangeability, which will be integrated into Inka Health's AI-powered SynoGraph platform.
  • These transportability methods are particularly important for rare disease drug approvals where patient populations are small and local evidence is often lacking, potentially accelerating regulatory approvals and improving patient access to innovative therapies.
  • The primer was co-authored with experts at AstraZeneca Canada and details proven statistical techniques including matching, weighting, standardization, and bias analysis to address differences in patient populations across regions.

FDA Issues Complete Response Letter for Biohaven's VYGLXIA (troriluzole) for Spinocerebellar Ataxia

Rare DiseaseReal World Evidence
  • The FDA issued a Complete Response Letter rejecting Biohaven's New Drug Application for VYGLXIA (troriluzole) to treat spinocerebellar ataxia, a rare neurodegenerative disorder affecting approximately 15,000 Americans.
  • Biohaven's real-world evidence study demonstrated that VYGLXIA slowed disease progression by 50-70% and reduced fall risk in SCA patients, but the FDA's Office of Neuroscience declined to approve the treatment.
  • The company will pivot resources to other pipeline programs and implement a 60% reduction in annual direct R&D spending while continuing to seek a path forward for troriluzole approval.

Largest Wearable Defibrillator Study Demonstrates 100% Conversion Success in 21,612 Patients

Real World Evidence
  • The ACE-PAS study, enrolling 21,612 patients across the United States, represents the largest prospective real-world study of wearable defibrillators to date and confirms strong safety and effectiveness of Kestra's ASSURE WCD device.
  • The study achieved 100% successful conversion for ventricular tachycardia and fibrillation events while maintaining an inappropriate shock rate of only 0.0065 per patient-month, demonstrating exceptional clinical performance.
  • Patients showed high compliance with median wear time exceeding 23 hours per day, and 2.6% experienced life-threatening arrhythmias within months, highlighting the critical need for protection during vulnerable recovery periods.
  • The ASSURE system detected additional clinically relevant conditions including high-rate atrial fibrillation in 4.2% of patients and severe bradycardia in 0.3%, enabling timely clinical interventions.

UK Government Launches £650,000 Obesity Study Targeting Scotland's Most Deprived Communities

Real World EvidenceAI
  • The UK government has awarded £650,000 to launch the Scotland CardioMetabolic Impact Study (SCoMIS), a groundbreaking research initiative targeting 3,000-5,000 participants from Scotland's most deprived communities to evaluate real-world impact of GLP-1 weight-loss medicines.
  • The study represents a collaboration between Novo Nordisk, IQVIA, and Scottish universities to assess incretin-based treatments' effectiveness in primary care settings, with full launch planned for 2026 and potential funding extending to 2029-2030.
  • More than one in three adults in Scotland's most deprived areas live with obesity, making this research critical for addressing health inequalities and potentially guiding NHS commissioning decisions for weight-loss medications.
  • The study will examine four key areas: delivery effectiveness in NHS care, weight loss outcomes in disadvantaged populations, impact on healthcare costs, and broader societal benefits including work productivity and reduced sick leave.

GENFIT Showcases ACLF Pipeline Advances at AASLD 2025 with Three Novel Therapeutic Programs

Real World Evidence
  • GENFIT will present new preclinical data on three ACLF therapeutic programs at AASLD 2025, including G1090N (nitazoxanide reformulation), SRT-015 (ASK1 inhibitor), and CLM-022 (NLRP3 inflammasome inhibitor).
  • The company's lead ACLF program G1090N is expected to deliver clinical safety data and early efficacy markers by the end of 2025.
  • Partner Ipsen will present additional data on Iqirvo (elafibranor) from ongoing studies in primary biliary cholangitis and primary sclerosing cholangitis.
  • GENFIT will also present real-world evidence studies and biomarker research highlighting diagnostic challenges and patient management issues in ACLF.

Real-World Safety Analysis Reveals New Adverse Events Associated with Fenofibric Acid for Hyperlipidemia Treatment

Real World Evidence
  • A comprehensive analysis of 2,293 adverse event reports from WHO VigiAccess and FDA databases confirmed known safety risks of fenofibric acid while identifying several previously unreported potential adverse effects.
  • The study detected drug-related safety signals across 23-26 organ system categories, revealing new potential adverse events including gout, hypoglycemia, and various musculoskeletal complications.
  • Researchers emphasize the critical importance of monitoring patients during the first three months of fenofibric acid treatment, with particular attention needed for female patients who may experience higher rates of adverse events.

Whitehawk Therapeutics Validates PTK7 as Third Most Abundant ADC Target Across 157,000 Tumor Samples

OncologyReal World Evidence
  • Whitehawk Therapeutics presented real-world analysis of over 157,000 tumor samples confirming PTK7 as the third most highly expressed tumor marker among ADC targets, present in approximately 70% of solid tumors.
  • The study revealed stable PTK7 expression across disease stages and metastatic status, with highest expression levels observed in endometrial, ovarian, head and neck, and non-small cell lung cancers.
  • The findings support development of HWK-007, Whitehawk's PTK7-directed ADC candidate, with the company planning to submit an IND application by year-end for initial evaluation in NSCLC, ovarian, and endometrial cancers.

MedPath

Empowering clinical research with data-driven insights and AI-powered tools.

© 2025 MedPath, Inc. All rights reserved.