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Clinical Trial News

CAR-T Cell Therapies Show Promise for Severe Autoimmune Diseases at EULAR 2025

CAR-T
  • Multiple CAR-T cell therapies targeting CD19-positive B cells demonstrated clinical efficacy across severe autoimmune diseases including systemic lupus erythematosus, systemic sclerosis, and rheumatoid arthritis at EULAR 2025.
  • Resecabtagene autoleucel achieved deep B cell depletion and clinical responses in patients with systemic sclerosis, idiopathic inflammatory myopathy, and lupus, with patients remaining free of immunosuppressive therapy.
  • Novel approaches including dual CD19/BCMA targeting CAR-T cells and off-the-shelf iPSC-derived CAR-T therapies showed favorable safety profiles with no severe cytokine release syndrome or neurotoxicity.
  • Early data suggest CAR-T therapy can reset the immune system in multiple rheumatic and musculoskeletal diseases, allowing patients to achieve sustained remission without ongoing immunosuppression.

Benchling Partners with Merck to Streamline Vaccine Bioanalysis Through Integrated Digital Platform

  • Benchling and Merck have announced a collaboration to implement a unified software framework that integrates pre-clinical and clinical bioanalytical workflows onto a single platform.
  • The partnership focuses on three key areas: enhancing speed and throughput through automated data capture, streamlining workflows with unified data management, and maintaining compliance standards in regulated environments.
  • The collaboration aims to improve efficiency throughout the vaccine development lifecycle by enabling scientists to better access, analyze, and harness data insights during the development process.
  • Over 200,000 scientists currently rely on Benchling's R&D Cloud platform, which serves cutting-edge startups and more than half of the top 50 global biopharma companies.

Bristol Myers Squibb Advances Targeted Protein Degradation Platform with New Clinical Data at EHA 2025

  • Bristol Myers Squibb presented updated clinical findings on investigational CELMoD agents mezigdomide and iberdomide for multiple myeloma, and golcadomide for non-Hodgkin lymphoma at the 2025 European Hematology Association Annual Congress.
  • The company revealed first results for its first-in-class oral BCL6 ligand-directed degrader (BMS-986458) in non-Hodgkin lymphoma, expanding its targeted protein degradation portfolio.
  • These protein degraders are designed to target and degrade disease-driving proteins that are difficult to address with conventional small-molecule inhibitors, potentially enhancing existing therapies and overcoming resistance.
  • Bristol Myers Squibb anticipates pivotal study readouts for these agents within the next year as the company continues to advance its leadership in targeted protein degradation research.

Sun Pharma's Halol Facility Receives Eight FDA Observations Following Latest Inspection

  • The US FDA issued eight observations to Sun Pharma's Halol manufacturing facility in Gujarat following a Good Manufacturing Practices inspection conducted from June 2-13, 2025.
  • The facility has been under an import alert since May 2022 and remains classified as OAI (Official Action Indicated) since March 2020, restricting exports to the US market.
  • Despite the regulatory challenges, UBS maintains a buy rating with a target price of Rs 2,450, while Nomura expects limited impact from potential site resolution given the company's large revenue base.
  • Sun Pharma announced a leadership transition with Kirti Ganorkar becoming Managing Director effective September 1, 2025, while founder Dilip Shanghvi will continue as Executive Chairman.

ITM to Present Phase 3 COMPETE Trial Data at SNMMI 2025 Meeting

Targeted Therapy
  • ITM Isotope Technologies Munich will present further analysis from its successful Phase 3 COMPETE trial at the SNMMI 2025 annual meeting in New Orleans from June 21-24.
  • The COMPETE trial demonstrated that 177Lu-edotreotide (ITM-11) achieved clinically and statistically significant improvement in progression-free survival compared to everolimus in patients with gastroenteropancreatic neuroendocrine tumors.
  • Dr. Thomas Hope will deliver an oral presentation on predicting tumor uptake using pre-therapeutic imaging, while a satellite symposium will cover the broader treatment landscape and dosimetry evolution.
  • The company will also showcase its radiopharmaceutical therapy pipeline and capabilities at booth 1001 during the conference.

Regor Therapeutics to Present Phase 2a Data for Oral GLP-1 Agonist RGT-075 at ADA 2025

  • Regor Therapeutics will present Phase 2a trial results for RGT-075, an oral small molecule GLP-1 receptor agonist, at the American Diabetes Association's 85th Scientific Sessions in Chicago.
  • RGT-075 demonstrated preliminary weight reduction in Phase 1 studies with no new safety signals observed in healthy volunteers and diabetes patients.
  • The company is currently conducting the COMO-1 Phase 2b trial with approximately 240 participants, evaluating multiple doses over 36 weeks with topline data expected by end of 2025.
  • RGT-075 represents a potential breakthrough as an orally bioavailable, once-daily treatment for obesity and overweight patients with weight-related comorbidities.

Food Deserts Compound Clinical Trial Access Barriers for Breast Cancer Patients

  • A study of over 1.3 million breast cancer patients found that living in food deserts significantly reduces clinical trial participation rates.
  • Patients facing both food deserts and clinical trial deserts showed a 27% lower likelihood of trial enrollment compared to 19% for food desert residents alone.
  • The research demonstrates a compounding negative effect when patients live in areas with both limited healthy food access and distant trial sites.
  • Interventions such as establishing satellite facilities, telehealth options, and patient navigation programs could help address these systemic barriers.

FDA Delays KalVista's Sebetralstat Approval Decision Due to Resource Constraints

Drug Approval
  • The FDA has extended its review of KalVista's sebetralstat for hereditary angioedema by four weeks past the June 17 deadline due to heavy workload and limited resources.
  • Sebetralstat would become the first on-demand oral treatment for hereditary angioedema if approved, offering a convenient alternative to injectable therapies.
  • The delay represents the first instance directly attributed to FDA resource constraints following agency restructuring under the Trump administration.
  • KalVista maintains confidence in near-term approval as the FDA has not requested additional data or raised safety concerns.

Intravenous Rehydration Shows Safety in Severely Malnourished Children, Challenging WHO Guidelines

  • A randomized controlled trial involving 292 children across four African countries found that intravenous rehydration was as safe as oral rehydration for children with severe acute malnutrition and gastroenteritis.
  • The study revealed no cases of heart failure or fluid overload with IV treatment, contradicting long-held concerns about cardiac complications in malnourished children.
  • Children receiving standard oral rehydration experienced more complications, including increased need for nasogastric tubes, vomiting episodes, and emergency IV fluids for shock.
  • The findings could prompt a review of WHO guidelines that currently distinguish between rehydration strategies for malnourished and non-malnourished children.

FDA Delays Sebetralstat Approval Decision for Hereditary Angioedema Treatment Due to Resource Constraints

Drug ApprovalRare Disease
  • The FDA will not meet the June 17, 2025 PDUFA goal date for KalVista's sebetralstat NDA due to heavy workload and limited resources, with a decision expected within four weeks.
  • Sebetralstat represents a potential breakthrough as the first oral on-demand treatment for hereditary angioedema attacks in patients aged 12 and older.
  • The FDA has not requested additional data or raised safety concerns, with only labeling finalization remaining under review according to KalVista.
  • The delay affects patients with hereditary angioedema who currently rely on intravenous or subcutaneous treatments for managing painful and potentially life-threatening attacks.

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