MedPath

Clinical Trial News

NRx Pharmaceuticals Enhances Clinical Trial Reliability in Depression Studies

  • NRx Pharmaceuticals' methodology for training and monitoring study site raters demonstrates higher interrater reliability (IRR) on the MADRS depression scale.
  • The enhanced reliability in psychometric ratings has positive implications for future registration trials of NRX-101 and similar medications targeting depression.
  • In the NRX-101 study, 94.5% concordance was achieved, defined by no more than 3 points of disagreement between site and central raters.
  • The methodology enabled statistical significance with fewer participants, potentially reducing study costs and risks of failure in antidepressant drug trials.

Citius Pharma Advances Mino-Lok Approval Pathway Following FDA Meeting

  • Citius Pharmaceuticals recently met with the FDA to discuss the NDA pathway for Mino-Lok, an antibiotic lock solution for infected central venous catheters.
  • The FDA provided constructive feedback on regulatory considerations and clinical data needed to support the NDA submission for Mino-Lok.
  • Mino-Lok aims to offer an alternative to catheter removal, potentially improving the management of catheter-related bloodstream infections and reducing healthcare costs.
  • Citius Pharma remains dedicated to advancing the Mino-Lok program and will continue to update on regulatory and clinical developments.

GSK Seeks FDA Approval for Blenrep Combination Therapy in Multiple Myeloma

  • GSK's Blenrep, previously withdrawn, aims for re-approval as a combination therapy for multiple myeloma after at least one prior line of treatment.
  • The FDA has accepted GSK's application for Blenrep combined with bortezomib plus dexamethasone, or pomalidomide plus dexamethasone, with a decision expected by July 2025.
  • Phase III trials DREAMM-7 and DREAMM-8 demonstrated statistically significant improvements in progression-free survival compared to standard-of-care regimens.
  • Blenrep's potential approval could introduce competition to BCMA-targeted therapies, offering a simpler administration route than CAR-T therapy.

Belantamab Mafodotin Plus Pomalidomide and Dexamethasone (Pd) Versus Bortezomib Plus Pd in Relapsed/Refractory Multiple Myeloma

NCT04484623Active, Not RecruitingPhase 3
GlaxoSmithKline
Posted 10/1/2020

Evaluation of Efficacy and Safety of Belantamab Mafodotin, Bortezomib and Dexamethasone Versus Daratumumab, Bortezomib and Dexamethasone in Participants With Relapsed/Refractory Multiple Myeloma

NCT04246047Active, Not RecruitingPhase 3
GlaxoSmithKline
Posted 5/7/2020

Citius Pharma Gains FDA Guidance on Mino-Lok Phase 3 Program for CLABSI

  • Citius Pharmaceuticals received constructive feedback from the FDA regarding its Phase 3 Mino-Lok program, aimed at salvaging central venous catheters in patients with CLABSI.
  • The FDA's guidance supports a potential New Drug Application (NDA) submission for Mino-Lok, addressing critical aspects like in-vitro data, clinical efficacy, safety, and regulatory considerations.
  • Mino-Lok, combining minocycline, ethanol, and edetate disodium, offers a novel approach to treating catheter-related bloodstream infections, potentially reducing healthcare costs and improving patient outcomes.
  • Citius Pharma is committed to advancing the Mino-Lok program and will provide updates on regulatory and clinical developments.

Citius Pharma Gains Clarity on Mino-Lok's Regulatory Path After FDA Meeting

  • Citius Pharmaceuticals reports a productive Type C meeting with the FDA regarding its Mino-Lok program for catheter-related bloodstream infections (CRBSI).
  • The FDA provided clear and actionable guidance, supporting a potential New Drug Application (NDA) submission for Mino-Lok.
  • Mino-Lok demonstrated compelling clinical outcomes in a Phase 3 trial, offering a potential alternative to catheter removal and reducing healthcare costs.
  • Citius Pharma is committed to advancing the Mino-Lok program and will provide updates on regulatory and clinical developments.

NRx Pharmaceuticals' Methodology Achieves High Accuracy in Depression Trial Ratings

  • NRx Pharmaceuticals' novel training and monitoring methodology demonstrated a 94.5% interrater reliability in a Phase 2b/3 clinical trial for NRX-101, surpassing previous industry standards.
  • The improved rater reliability enabled the identification of statistically significant improvements in suicidality and akathisia with a smaller patient cohort.
  • The enhanced methodology is expected to reduce the costs and risks of failure in future clinical trials for NRX-101 and similar medications targeting depression.

Long COVID Patients Frustrated by Lack of FDA-Approved Treatments Despite Federal Research Funding

• Long COVID patients express frustration over the lack of FDA-approved treatments despite $1.15 billion in federal research funding. • The NIH's RECOVER initiative is criticized for prioritizing observational studies over clinical trials for potential therapies. • An additional $662 million in funding, including $300 million for clinical trials, aims to address the urgent need for effective treatments. • Researchers and patient advocates emphasize the importance of understanding biological mechanisms and prioritizing patient-centered research.

Ractigen's RAG-21 Receives FDA Orphan Drug Designation for FUS-ALS Treatment

  • Ractigen Therapeutics' RAG-21, a novel siRNA therapy targeting the FUS gene, has received FDA Orphan Drug Designation for amyotrophic lateral sclerosis (ALS).
  • RAG-21 leverages RNA interference to reduce FUS protein levels, addressing motor neuron degeneration in FUS-ALS, a particularly aggressive ALS subtype.
  • Preclinical studies demonstrate RAG-21's efficacy and safety in mitigating FUS cytoplasmic mis-localization and aggregation, offering hope for improved outcomes.
  • The FDA's designation provides incentives, including tax credits and marketing exclusivity, to accelerate RAG-21's development for this rare and severe disease.

Tislelizumab Gains Category 1 Recommendation in NCCN Guidelines for Hepatocellular Carcinoma

• Tislelizumab-jsgr (Tevimbra) has been added to the NCCN guidelines as a category 1 recommended regimen for first-line systemic therapy in HCC. • The NCCN guidelines are updated regularly, and healthcare professionals should ensure they are using the latest version to inform their practice. • Identifying biomarkers to predict response to checkpoint inhibitors remains a key challenge in HCC treatment, as many patients do not benefit from current therapies.

FDA Approves Attruby (Acoramidis) for Transthyretin Amyloid Cardiomyopathy (ATTR-CM)

• The FDA has approved Attruby (acoramidis) for treating transthyretin amyloid cardiomyopathy (ATTR-CM), offering a new hope for patients with this rare heart condition. • Attruby, a TTR stabilizer, demonstrated significant reduction in cardiovascular death and hospitalization in the ATTRibute-CM Phase 3 trial. • The approval provides an additional treatment option alongside existing therapies, potentially improving heart health and quality of life for ATTR-CM patients. • BridgeBio offers patient support programs like ForgingBridges™ to assist with access, insurance, and financial aid for Attruby treatment.

MedPath

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

© 2025 MedPath, Inc. All rights reserved.