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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.

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.

Gilead Presents Three-Year Livdelzi Data Showing Liver Health Stabilization in Primary Biliary Cholangitis Patients

Real World EvidenceOrphan Drug
  • Gilead will present new three-year interim data from the ASSURE study demonstrating that Livdelzi (seladelpar) may help stabilize or improve liver health in patients with primary biliary cholangitis.
  • Late-breaking presentations at The Liver Meeting 2025 will include real-world treatment experience data following the withdrawal of obeticholic acid and long-term pruritus outcomes from clinical trials.
  • The company will showcase findings from more than 35 accepted abstracts across PBC and viral hepatitis research, reinforcing its commitment to advancing liver disease treatments.

RESPONSE: Response to Seladelpar in Subjects With Primary Biliary Cholangitis (PBC) and an Inadequate Control to or an Intolerance to Ursodeoxycholic Acid (UDCA)

NCT04620733CompletedPhase 3
CymaBay Therapeutics, Inc.
Posted 4/21/2021

Safety and Efficacy of Bictegravir/Emtricitabine/Tenofovir Alafenamide Versus Dolutegravir + Emtricitabine/Tenofovir Disoproxil Fumarate in Treatment Naive, HIV-1 and Hepatitis B Co-Infected Adults

NCT03547908CompletedPhase 3
Gilead Sciences
Posted 5/30/2018

Seladelpar in Subjects With Primary Biliary Cholangitis (PBC)

NCT03301506Active, Not RecruitingPhase 3
Gilead Sciences
Posted 12/12/2017

Study to Assess Efficacy and Safety of Bulevirtide in Combination With Pegylated Interferon Alfa-2a in Participants With Chronic Hepatitis Delta (CHD)

NCT03852433CompletedPhase 2
Gilead Sciences
Posted 5/31/2019

Study to Assess Efficacy and Safety of Bulevirtide in Participants With Chronic Hepatitis Delta (CHD)

NCT03852719CompletedPhase 3
Gilead Sciences
Posted 4/17/2019

Global Registry Data Confirms Survival Advantage of Mogamulizumab in Advanced Cutaneous T-Cell Lymphoma

Monoclonal AntibodyRare DiseaseReal World EvidenceOncology
  • New findings from the PROCLIPI study, spanning 19 countries and over 2,000 patients, demonstrate a meaningful overall survival advantage for advanced cutaneous T-cell lymphoma patients treated with mogamulizumab.
  • Among 371 patients with advanced mycosis fungoides and Sézary syndrome, those receiving mogamulizumab showed median overall survival of 64 months compared to 54 months for those not receiving the therapy.
  • For Sézary syndrome patients specifically, median survival reached approximately 6.5 years with mogamulizumab treatment versus 3 years for those on other systemic treatments.
  • The study represents one of the largest international registries in cutaneous T-cell lymphoma research, highlighting the power of global collaboration in rare disease evidence generation.

Shionogi's Cefiderocol Shows Superior Outcomes When Used as Early Treatment for Drug-Resistant Infections

Real World Evidence
  • Real-world data from the PROVE study demonstrates that cefiderocol achieved a 73.7% clinical cure rate when used as empiric treatment compared to 54.3% when used as salvage therapy for serious gram-negative bacterial infections.
  • The antibiotic maintained effectiveness against bacteria resistant to beta-lactam–beta-lactamase inhibitor combinations, with 90.6% of non-susceptible isolates remaining susceptible to cefiderocol.
  • Five-year surveillance data shows cefiderocol susceptibility rates have remained consistently high since its approval, including activity against metallo-beta-lactamase-carrying Acinetobacter baumannii.
  • The study analyzed 508 U.S. patients with serious infections, with 57.3% requiring intensive care and 47.6% needing organ support at treatment initiation.

Study of Cefiderocol (S-649266) or Best Available Therapy for the Treatment of Severe Infections Caused by Carbapenem-resistant Gram-negative Pathogens

NCT02714595CompletedPhase 3
Shionogi
Posted 9/7/2016

Pharming Group to Present 12 Studies on Rare Disease Treatments at 2025 ACAAI Meeting

Rare DiseaseReal World Evidence
  • Pharming Group will present 12 accepted abstracts at the 2025 ACAAI Annual Scientific Meeting in Orlando, showcasing new clinical and economic data for their rare disease portfolio.
  • Five presentations will focus on RUCONEST® for hereditary angioedema treatment, including cost-effectiveness studies and indirect treatment comparisons with competing therapies.
  • Seven studies will highlight real-world effectiveness data for Joenja® (leniolisib) in treating APDS, including pediatric trial results and caregiver burden assessments.
  • The presentations demonstrate Pharming's commitment to advancing patient care in rare genetic disorders affecting immune system function and regulation.

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