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Clinical Trial News

Mesoblast's Ryoncil Nears Potential FDA Approval for Pediatric Graft-versus-Host Disease

  • Mesoblast's remestemcel-L (Ryoncil) has a PDUFA date of January 7, 2025, for treating steroid-refractory acute graft-versus-host disease (SR-aGVHD) in pediatric patients.
  • If approved, Ryoncil would be the first treatment option for children under 12 with SR-aGVHD, addressing a critical unmet need in this vulnerable population.
  • Industry analysts maintain a consensus Buy rating for Mesoblast, with an average price target of $11, representing a 56% upside potential.

Pelareorep Plus Paclitaxel Shows Survival Benefit in HR+/HER2- Breast Cancer

  • The combination of pelareorep and paclitaxel demonstrated a significant improvement in overall survival (OS) compared to paclitaxel alone in patients with HR+/HER2- metastatic breast cancer.
  • In the BRACELET-1 trial, the median OS was not reached in the pelareorep/paclitaxel arm, while it was 18.2 months in the paclitaxel monotherapy arm (HR, 0.48).
  • The 2-year survival rate was 64% in the pelareorep/paclitaxel group versus 33% in the paclitaxel monotherapy group, indicating a substantial survival advantage.
  • Progression-free survival (PFS) also improved with the combination therapy, showing a median PFS of 12.1 months compared to 6.4 months with paclitaxel alone (HR, 0.39).

A Study of Reolysin For Patients With Advanced/Metastatic Breast Cancer

NCT01656538CompletedPhase 2
Canadian Cancer Trials Group
Posted 2/20/2013

A Study to Assess Overall Response Rate by Inducing an Inflammatory Phenotype in Metastatic BReast cAnCEr With the Oncolytic Reovirus PeLareorEp in CombinaTion With Anti-PD-L1 Avelumab and Paclitaxel - BRACELET-1 Study

NCT04215146Active, Not RecruitingPhase 2
Oncolytics Biotech
Posted 6/10/2020

Biomarkers sNfL and sGFAP Predict Disability Worsening in Multiple Sclerosis

  • A new study identifies serum neurofilament light chain (sNfL) and serum glial fibrillary acidic protein (sGFAP) as key biomarkers for predicting disability worsening in multiple sclerosis (MS).
  • Elevated sNfL levels at MS onset correlate with increased risk of both relapse-associated worsening (RAW) and progression independent of relapse activity (PIRA).
  • High sGFAP levels, combined with low sNfL, indicate an increased risk of PIRA and poor response to current disease-modifying treatments (DMTs).
  • Measuring sNfL and sGFAP levels can enable personalized treatment strategies, optimizing the use of high-efficacy DMTs and identifying patients needing novel therapies.

EMA Finalizes Reflection Paper on Single-Arm Trials for Efficacy Assessment

  • The European Medicines Agency (EMA) has finalized a reflection paper addressing the use of single-arm trials for establishing drug efficacy, providing guidance for pharmaceutical companies.
  • The paper outlines considerations for when single-arm trials may be appropriate, particularly in rare diseases or situations with significant unmet medical needs.
  • It emphasizes the importance of robust data collection, clear endpoints, and rigorous statistical analysis to ensure the reliability of results from single-arm trials.
  • The EMA's guidance aims to harmonize the approach to evaluating single-arm trials, potentially accelerating drug development for conditions with limited treatment options.

Xofluza Significantly Reduces Influenza Transmission in Phase III Trial

  • Roche's Xofluza (baloxavir marboxil) significantly reduced influenza transmission within households in a Phase III trial, marking a potential advance in flu control.
  • The CENTERSTONE study met its primary endpoint, showing a single oral dose of Xofluza reduced the likelihood of household members contracting the virus.
  • The antiviral treatment was well-tolerated, with no new safety signals, suggesting a favorable risk-benefit profile for influenza prevention.
  • These results could improve health outcomes at both individual and community levels, especially for influenza pandemic preparedness.

Study to Assess the Safety, Pharmacokinetics, and Efficacy of Baloxavir Marboxil in Healthy Pediatric Participants From Birth to < 1 Year With Influenza-Like Symptoms

NCT03653364CompletedPhase 3
Hoffmann-La Roche
Posted 1/23/2019

Study to Assess the Efficacy of Baloxavir Marboxil Versus Placebo to Reduce Onward Transmission of Influenza A or B in Households

NCT03969212CompletedPhase 3
Hoffmann-La Roche
Posted 10/10/2019

Five Strategic Travel Solutions to Boost Clinical Trial Diversity and Patient Enrollment

  • Recent CISCRP report identifies travel to study clinics as the primary burden for clinical trial participants, highlighting a persistent barrier to patient enrollment and diversity.
  • Clinical trial sites' locations often exclude minority communities, who are disproportionately affected by certain diseases yet face significant transportation and accessibility challenges.
  • Implementing comprehensive travel support solutions, including coordination services, timely reimbursements, and specialized transport options, can significantly improve trial accessibility and diversity.

Rice and Baylor Receive $2.8 Million to Develop Cell Therapy for ARDS

  • Rice University and Baylor College of Medicine have been awarded $2.8 million by the NHLBI to develop a novel cell therapy for acute respiratory distress syndrome (ARDS).
  • The therapy uses engineered retinal pigment epithelial (RPE) cells to deliver anti-inflammatory cytokines directly to the lungs, reducing inflammation and lung damage.
  • This targeted approach aims to overcome the limitations of current cytokine therapies, such as poor biodistribution, toxicity, and immune complications, in ARDS patients.
  • The collaborative effort seeks to improve patient outcomes by creating cell therapy systems that enhance lung health and increase survival rates for individuals with ARDS.

CheckMate-9ER: Glycoproteomic Analysis Identifies Potential Biomarkers for Nivolumab Plus Cabozantinib in Advanced RCC

• Post-hoc analysis of CheckMate-9ER reveals that higher degrees of fucosylation and sialylation in serum proteins are negative prognostic factors in advanced renal cell carcinoma (RCC). • Serum glycoproteins involved in complement cascade and lipid metabolism may predict response to nivolumab plus cabozantinib versus sunitinib. • High serum levels of CO3 glycopeptide potentially predict favorable response to nivolumab plus cabozantinib, while low levels of CFAH glycopeptide also indicate benefit. • These early exploratory findings suggest protein glycosylation as a potential area for further biomarker development in RCC to predict response to anti-PD-1-based therapy.

A Study of Nivolumab Combined With Cabozantinib Compared to Sunitinib in Previously Untreated Advanced or Metastatic Renal Cell Carcinoma

NCT03141177Active, Not RecruitingPhase 3
Bristol-Myers Squibb
Posted 7/23/2017

Immix Biopharma's NXC-201 Shows Promise in AL Amyloidosis Treatment

  • Immix Biopharma's stock hits a 52-week low amidst market challenges, reflecting investor concerns about the company's financial performance.
  • NXC-201, a CAR-T therapy, demonstrates a 92% overall response rate in an ex-U.S. study for AL Amyloidosis, leading to expansion of U.S. Phase 1b/2 trial.
  • The European Commission grants orphan drug designation to NXC-201 for multiple myeloma, supporting its development for this indication.

Kiromic BioPharma Expands Deltacel-01 Trial for NSCLC to University of Arizona Cancer Center

  • Kiromic BioPharma has activated the University of Arizona Cancer Center as the fifth clinical trial site for its Deltacel-01 Phase 1 trial.
  • The trial evaluates Deltacel™ (KB-GDT-01), an allogeneic gamma delta T-cell therapy, in patients with stage 4 metastatic non-small cell lung cancer (NSCLC).
  • The primary objective is to assess safety, with secondary endpoints including objective response and overall survival in patients who have failed standard therapies.
  • Patient enrollment at the University of Arizona Cancer Center is expected to commence in the coming weeks, expanding the trial's reach.

Safety Study for a Gamma Delta T Cell Product Used with Low Dose Radiotherapy in Patients with Locally Advanced or Metastatic NSCLC or Solid Tumors with Bone Metastases

NCT06069570RecruitingPhase 1
Kiromic BioPharma Inc.
Posted 11/7/2023

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