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Clinical Trial News

XOMA Royalty Corporation to Acquire Turnstone Biologics for $0.34 Per Share Plus Contingent Value Rights

Oncology
  • XOMA Royalty Corporation announced a definitive merger agreement to acquire Turnstone Biologics for $0.34 in cash per share plus contingent value rights.
  • The Turnstone Board of Directors unanimously approved the acquisition, with stockholders holding 25.2% of shares signing support agreements.
  • The tender offer will commence by July 11, 2025, with the merger transaction expected to close in August 2025.
  • Turnstone Biologics has been focused on developing selected tumor-infiltrating lymphocyte therapy for solid tumor treatment.

Urine DNA Test Predicts Bladder Cancer Recurrence After Immunotherapy Treatment

Oncology
  • A multi-institutional study published in Science Direct demonstrates that urine-based tumor DNA (utDNA) testing can predict which bladder cancer patients face higher recurrence risk after immunotherapy treatment.
  • Researchers analyzed utDNA from 89 patients in the SWOG S1605 trial using the UroAmp test, finding that positive utDNA results correlated with poorer treatment response and increased cancer recurrence rates.
  • The study offers potential for personalized bladder cancer care by enabling earlier identification of treatment response, potentially reducing unnecessary surgeries and improving patient outcomes.
  • This approach could help tailor therapy decisions sooner for the approximately 83,000 Americans diagnosed with bladder cancer annually, particularly those with high-risk disease unresponsive to BCG treatment.

Selinexor Maintenance Therapy Shows Sustained Benefit in TP53 Wild-Type Advanced Endometrial Cancer

  • Long-term follow-up from the phase 3 SIENDO trial demonstrates that selinexor maintenance therapy provides durable clinical benefit in patients with TP53 wild-type advanced or recurrent endometrial cancer.
  • In the TP53 wild-type/mismatch repair-proficient subgroup, selinexor achieved a median progression-free survival of 39.5 months compared to 4.9 months with placebo.
  • The treatment showed consistent improvements across multiple efficacy endpoints including time to first subsequent therapy and second progression-free survival, regardless of mismatch repair status.
  • Safety profile remained manageable with nausea being the most common adverse event, occurring in 90% of selinexor-treated patients.

Maintenance With Selinexor/Placebo After Combination Chemotherapy in Participants With Endometrial Cancer [SIENDO]

NCT03555422Active, Not RecruitingPhase 3
Karyopharm Therapeutics Inc
Posted 1/5/2018

Selinexor in Maintenance Therapy After Systemic Therapy for Participants With p53 Wild-Type, Advanced or Recurrent Endometrial Carcinoma

NCT05611931RecruitingPhase 3
Karyopharm Therapeutics Inc
Posted 4/18/2023

FDA Grants Breakthrough Therapy Designation for Daraxonrasib in KRAS-Mutated Pancreatic Cancer

Targeted TherapyPrecision MedicineOncology
  • FDA has granted Breakthrough Therapy Designation to daraxonrasib, a RAS(ON) multi-selective inhibitor, for previously treated metastatic pancreatic ductal adenocarcinoma (PDAC) in patients with KRAS G12 mutations.
  • The designation represents a significant regulatory milestone for targeting KRAS mutations in pancreatic cancer, one of the most challenging malignancies to treat.
  • Daraxonrasib's multi-selective RAS(ON) inhibition mechanism offers a novel therapeutic approach for patients with limited treatment options in the metastatic setting.

Paralyzed Veteran Becomes First Neuralink Brain-Computer Interface Recipient at University of Miami

  • A paralyzed U.S. military veteran successfully received Neuralink's brain-computer interface implant at The Miami Project to Cure Paralysis, becoming the fifth participant in the PRIME Study and first at the University of Miami site.
  • The patient, known as RJ, can now control computers and smartphones with his thoughts following the April 2025 surgery and was discharged just one day after the procedure.
  • The PRIME Study is an investigational clinical trial evaluating the safety and functionality of Neuralink's intracortical brain-computer interface that creates a wireless digital connection between the brain and external devices.
  • Neuralink continues recruiting patients with limited hand function due to cervical spinal cord injury or ALS for the ongoing clinical trial.

Precise Robotically IMplanted Brain-Computer InterfacE

NCT06429735RecruitingNot Applicable
Neuralink Corp
Posted 1/9/2024

NCCN Guidelines Update Treatment Sequencing for Metastatic Gastrointestinal Stromal Tumors

  • NCCN guidelines establish a clear sequential treatment approach for metastatic GIST, with imatinib as first-line, followed by sunitinib, regorafenib, and ripretinib in subsequent lines.
  • Recent guideline updates include considerations for ripretinib in second-line for patients intolerant to sunitinib, based on quality-of-life improvements from the INTRIGUE study.
  • Adjuvant imatinib therapy duration has been extended from 3 to 6 years for high-risk resected GIST patients, based on improved recurrence-free survival data.
  • Continuous kinase inhibitor therapy is essential in GIST management, as discontinuation results in rapid disease progression demonstrated in placebo-controlled trials.

Cellular Senescence Gene Signature Reveals New Biomarkers for Idiopathic Pulmonary Fibrosis

  • Researchers identified four key cellular senescence-related genes (CDKN2A, VEGFA, SOX2, and FOXO3) that could serve as diagnostic biomarkers for idiopathic pulmonary fibrosis (IPF).
  • A comprehensive bioinformatics analysis of 122 differentially expressed genes revealed that a four-gene model achieved excellent diagnostic performance with an AUC of 0.956 in the training dataset.
  • The study validated these findings using immunofluorescence staining on lung tissue samples from IPF patients, confirming differential expression patterns compared to healthy controls.
  • These cellular senescence-related biomarkers could provide new insights into IPF pathogenesis and potentially guide development of senescence-based therapeutic interventions.

BCMA-Targeted CAR-T Therapy Achieves 96.3% Response Rate in Relapsed Multiple Myeloma Study

CAR-T
  • A novel dual nanobody CAR-T therapy targeting BCMA demonstrated a remarkable 96.3% overall response rate in 27 patients with relapsed or refractory multiple myeloma, including many with high-risk features.
  • The treatment showed a median duration of remission of 11 months with a range of 2-36 months, indicating sustained therapeutic benefit in this challenging patient population.
  • One-year survival outcomes revealed an overall survival rate of 61.1% and progression-free survival of 57.2%, representing significant clinical activity in heavily pretreated myeloma patients.
  • Real-world data on belantamab mafodotin showed more modest results with 43% overall response rate and 3.8 months median progression-free survival in relapsed/refractory multiple myeloma patients.

FDA Approves Gamifant as First Treatment for Macrophage Activation Syndrome in Still's Disease

Drug ApprovalMonoclonal AntibodyRare Disease
  • The FDA has approved Gamifant (emapalumab-lzsg) as the first-ever treatment for adults and children with macrophage activation syndrome in Still's disease, marking a significant breakthrough for patients with this life-threatening condition.
  • Clinical trials demonstrated that 54% of patients achieved complete response at week 8, with 82% reaching clinical MAS remission, providing new hope for managing severe hyperinflammation.
  • This approval addresses a critical unmet medical need for patients with MAS, offering an alternative to high-dose glucocorticoids and potentially reducing reliance on conventional therapies with significant side effects.

A Study to Investigate the Safety and Efficacy of Emapalumab, an Anti-IFN-gamma mAb in Patients With Systemic Juvenile Idiopathic Arthritis (sJIA) or Adult-onset Still's Disease (AOSD) Developing Macrophage Activation Syndrome/Secondary HLH (MAS/sHLH)

NCT03311854CompletedPhase 2
Swedish Orphan Biovitrum
Posted 2/20/2018

Evaluate Efficacy, Safety and Tolerability, PK and PD of Emapalumab in Children and Adults With MAS in Still's or SLE

NCT05001737CompletedPhase 3
Swedish Orphan Biovitrum
Posted 12/15/2021

Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS)

NCT06617429Active, Not RecruitingPhase 3
Ultragenyx Pharmaceutical Inc
Posted 12/3/2024

Cessatech Reports 75% Pain Reduction in Pediatric Acute Pain Study, Eyes US Launch by End 2025

  • Cessatech's Study 0202 demonstrated a 75% overall pain reduction in pediatric patients, significantly outperforming previous studies that showed 60-64% reduction in similar populations.
  • The company is preparing for US market launch by end 2025 following completion of stability studies and manufacturing setup with a new sterile nasal production partner.
  • Clinical investigators reported immediate visible improvements in children and reassurance for parents within minutes of treatment administration across multiple study sites.
  • The company raised approximately 15 million DKK from professional investors and family offices to support US commercialization efforts and maintain operations through 2025-2026.

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