MedPath

Clinical Trial News

HKU Launches Gene Therapy Clinical Trial for Chronic Hepatitis B Cure

  • The University of Hong Kong has launched a clinical trial for a gene therapy aimed at treating chronic hepatitis B infection, potentially offering patients a cure rather than lifelong symptom management.
  • The new gene therapy works by removing the hepatitis B infected gene from the patient's genetic code to reduce viral surface antigens and halt virus integration with the human genome.
  • Initial results from nine patients show reduced surface antigen levels, with the trial aiming to enroll 40 patients by the end of 2026.
  • The therapy could eliminate the need for decades-long medication and reduce risks of liver cancer, cirrhosis, and liver failure for the 300 million people affected worldwide.

Arvinas CEO John Houston Announces Retirement Following PROTAC Breakthrough Success

Leadership ChangeTargeted Therapy
  • John Houston, Ph.D., CEO and President of Arvinas, announces plans to retire from executive roles after eight years of leadership, while remaining as Board Chairperson.
  • Under Houston's leadership, Arvinas achieved multiple industry firsts for PROTAC protein degraders, including the first positive pivotal Phase 3 trial and first new drug application.
  • The company has advanced six PROTAC programs into clinical trials and demonstrated for the first time that orally administered PROTACs can achieve pharmacodynamic activity in the central nervous system.
  • Arvinas Board of Directors has initiated a search for Houston's successor to continue advancing the company's targeted protein degradation platform across multiple therapeutic areas.

FDA Approves Retifanlimab Plus Carboplatin/Paclitaxel for Advanced Anal Cancer Following PODIUM-303 Trial Success

Drug Approval
  • The FDA approved retifanlimab in combination with carboplatin/paclitaxel for advanced anal cancer in May 2025, based on the PODIUM-303 study results.
  • The PODIUM-303 trial demonstrated improved progression-free survival (9.3 vs 7.4 months) and response rates (56% vs 44%) with the addition of retifanlimab to standard chemotherapy.
  • Current NCCN guidelines now include carboplatin/paclitaxel plus retifanlimab as a category 2B evidence-based approach for first-line treatment of advanced squamous cell anal cancer.
  • The InterAACT study previously established carboplatin/paclitaxel as standard care, showing similar response rates but improved survival and tolerability compared to cisplatin/5-FU.

Soleno Therapeutics Raises $200 Million to Fund Commercialization of First FDA-Approved Prader-Willi Syndrome Therapy

Drug ApprovalRare Disease
  • Soleno Therapeutics completed a $200 million public offering at $85 per share to fund commercialization of VYKAT XR, the first FDA-approved therapy for hyperphagia in Prader-Willi syndrome patients.
  • The company received FDA approval for VYKAT XR on March 26, 2025, marking a significant milestone for treating this rare genetic disorder.
  • Proceeds will also support regulatory and market development activities in the European Union and further research and development efforts.
  • The offering included 2,352,941 shares with underwriters holding a 30-day option to purchase an additional 352,941 shares at the same price.

Comprehensive Genomic Profiling Emerges as Essential Standard for Personalized Cancer Treatment

Precision MedicineTargeted Therapy
  • Comprehensive genomic profiling has become standard practice for non-small cell lung cancer treatment, enabling identification of actionable genomic alterations that guide personalized therapeutic decisions.
  • The integration of both tissue-based testing and plasma-based next-generation sequencing provides complementary molecular profiling capabilities throughout the patient care continuum.
  • Incomplete genomic testing can leave patients without access to potentially beneficial targeted therapies, emphasizing the critical importance of comprehensive molecular profiling for optimal outcomes.
  • Future applications include expanded serial testing for resistance monitoring and potential expansion into earlier cancer stages as precision medicine approaches continue evolving.

NALIRIFOX Emerges as Effective Alternative to FOLFIRINOX in Metastatic Pancreatic Cancer Treatment

  • The NAPOLI-3 trial in 2023 established NALIRIFOX as an effective treatment option for metastatic pancreatic adenocarcinoma, achieving 11.1 months median overall survival that matches FOLFIRINOX's benchmark.
  • NALIRIFOX demonstrated superior objective response rates of 42% compared to historical FOLFIRINOX data of approximately 30%, though cross-trial comparisons require cautious interpretation.
  • The reduced oxaliplatin dosing in NALIRIFOX makes it an attractive option for patients concerned about peripheral neuropathy, a significant long-term adverse effect of FOLFIRINOX.
  • Treatment selection between these triplet regimens requires careful consideration of patient-specific factors including baseline neuropathy risk, performance status, and individual treatment goals.

Chromophobe Renal Cell Carcinoma Shows Immune-Cold Environment and Poor Response to Checkpoint Inhibitors

  • Chromophobe renal cell carcinoma (ChRCC) demonstrates an immune-cold tumor microenvironment with significantly reduced CD8+ T-cell infiltration compared to clear cell RCC, explaining poor immunotherapy responses.
  • Patients with metastatic chromophobe RCC receiving immune checkpoint inhibitor-based therapies achieved inferior survival outcomes with median overall survival of 24.7 months versus 50.5 months in clear cell RCC patients.
  • The study identified α-intercalated cells as the cellular origin of chromophobe RCC and revealed potential therapeutic targets including ferroptosis pathways and mTOR signaling.
  • Real-world analysis showed mTOR inhibitors may offer superior outcomes in chromophobe RCC compared to immunotherapy, with median overall survival of 41.3 months versus 13.4 months in clear cell RCC patients.

Anti-GPC3 Targeted Therapies Market Expands with Novel Treatment Approaches for Hepatocellular Carcinoma

CAR-TMonoclonal AntibodyTargeted Therapy
  • The anti-GPC3 targeted therapies market is expected to surge significantly by 2040, driven by increasing cancer incidence and clinical pipeline activity across hepatocellular carcinoma, non-small cell lung cancer, and gastric cancer.
  • AstraZeneca presented promising data from the RHEA-1 first-in-human study of AZD9793, a first-in-class CD8-guided T cell engager targeting GPC3-positive advanced or metastatic HCC at ASCO 2025.
  • Bayer initiated a Phase I clinical trial for 225Ac-GPC3 (BAY 3547926), an investigational targeted alpha radiopharmaceutical designed to treat GPC3-expressing tumors in patients with advanced HCC.
  • Key pipeline therapies include ECT204 from Eureka Therapeutics, currently under evaluation in the ARYA-3 Phase I/II trial for GPC3-positive HCC patients.

Broken String Biosciences and BioLizard Partner to Develop AI-Powered SafeGuide for CRISPR Gene Editing Safety

Gene EditingAI
  • Broken String Biosciences and BioLizard have announced the development of SafeGuide, an AI tool designed to select safer guide RNAs for CRISPR gene editing applications.
  • The collaboration aims to replace current trial-and-error approaches that take one to two years and result in drug costs exceeding $4 million per patient.
  • The project is supported by a $935,000 grant from the competitive Eurostars-3 program, part of the European Partnership on Innovative SMEs.
  • SafeGuide will integrate Broken String's INDUCE-seq platform data with BioLizard's AI algorithms to predict high-efficacy, low-risk guide RNA designs.

Ultragenyx's Setrusumab Phase 3 Trial Continues After Interim Analysis Fails to Meet Early Termination Threshold

Rare DiseaseMonoclonal Antibody
  • Ultragenyx Pharmaceutical's Phase 3 Orbit study of setrusumab for osteogenesis imperfecta failed to meet the interim statistical threshold for early termination (p<0.01), leading to a 27% stock decline on July 6.
  • The Data Monitoring Committee confirmed setrusumab's excellent safety profile and allowed trials to continue, with final readouts expected by year-end 2025 using more lenient statistical thresholds.
  • Phase 2 results previously demonstrated a 67% reduction in fractures (p=0.0014), supporting the drug's potential for treating the rare bone disease affecting approximately 60,000 patients globally.
  • Osteogenesis imperfecta currently has zero approved therapies, representing a significant unmet medical need with potential peak sales estimated at $1.6 billion by Goldman Sachs.

MedPath

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

© 2025 MedPath, Inc. All rights reserved.