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FDA-Approved Cancer Drug Ruxolitinib Shows Promise for Treating Cardiac Arrhythmias

  • Researchers from Johns Hopkins University and University of Chicago discovered that ruxolitinib, an FDA-approved cancer and skin condition drug, effectively inhibits CaMKII protein kinase linked to cardiac arrhythmias.
  • The team developed a novel biosensor called CaMKAR to screen nearly 5,000 FDA-approved drugs, identifying five previously unknown CaMKII inhibitors with ruxolitinib being the most effective.
  • In mouse models, a 10-minute application of ruxolitinib prevented catecholaminergic polymorphic ventricular tachycardia and rescued atrial fibrillation without causing adverse cognitive effects.
  • The discovery could enable rapid clinical translation since ruxolitinib has already been proven safe in humans, potentially offering new treatment options for arrhythmia patients resistant to standard therapies.

Zanubrutinib, Obinutuzumab, and Venetoclax Triplet Achieves High uMRD Rates in CLL/SLL

  • A phase 2 trial (BOVen) showed that zanubrutinib plus obinutuzumab and venetoclax induced high rates of undetectable minimal residual disease (uMRD) in CLL/SLL patients.
  • 92% of patients achieved uMRD in both peripheral blood and bone marrow, leading to treatment discontinuation after a median of 10 months.
  • ΔMRD400, a measure of MRD kinetics, predicted early uMRD in bone marrow and shorter therapy duration, with 60% of patients achieving it.
  • The BOVen regimen was well-tolerated, with manageable adverse events, supporting an MRD-driven approach to treatment duration in CLL/SLL.

Study of Zanubrutinib, Obinutuzumab, and Venetoclax in Patients With Chronic Lymphocytic Leukemia (CLL) or Small Lymphocytic Leukemia (SLL)

NCT03824483RecruitingPhase 2
Memorial Sloan Kettering Cancer Center
Posted 2/22/2019

Navigating Complex Validation Challenges in ATMP Analytical Methods: Key Considerations for Drug Development

  • Advanced Therapy Medicinal Products (ATMPs) face unique analytical method validation challenges due to inherent variability in starting materials, complex biological features, and limited batch availability.
  • Quality control testing methods for ATMPs can be categorized into three maturity levels: fully mature methods analogous to established biologics, common GMP QC release assays requiring adaptation, and novel techniques specific to complex molecules.
  • Regulatory compliance for ATMP analytical validation requires careful consideration of reference standards, product availability, and continuous dialogue with regulatory agencies throughout development.

Sequana Medical Announces Positive Outcomes from North American Pivotal Alfapump Study (POSEIDON)

Sequana Medical has revealed additional data from its North American pivotal study, POSEIDON, showcasing the alfapump's effectiveness in controlling ascites, improving patients' quality of life, and demonstrating a favorable survival rate. The study, focusing on patients with recurrent or refractory ascites due to liver cirrhosis, also highlighted the device's safety profile and its potential to significantly reduce the need for therapeutic paracentesis.

Emerging Role of GLP-1 Agonists in Obesity

GLP-1 agonists, initially developed for type 2 diabetes treatment, have shown significant efficacy in promoting weight loss in both preclinical and clinical studies. This review highlights the role of GLP-1 agonists in weight management, their pleiotropic effects, and their impact on hyperglycemia, insulin sensitivity, blood pressure, and cardio-metabolic and renal protection.

CardioMEMS Implant Halves Hospital Admissions for Heart Failure Patients

  • A tiny CardioMEMS sensor implanted in the pulmonary artery significantly reduces hospital admissions for heart failure patients by 44%.
  • The device monitors blood pressure fluctuations, alerting doctors to changes before they escalate into serious health issues.
  • Patients transmit data daily via a specialized pillow, enabling proactive management and reducing the need for hospitalization.
  • Early trials show improved quality of life for patients, with hopes for broader NHS evaluation and availability.

FDA Approves AVITA Medical's RECELL System for Vitiligo Skin Repigmentation

AVITA Medical has received FDA approval for its RECELL System, a groundbreaking treatment for repigmentation in patients with stable vitiligo. This one-time, point-of-care treatment utilizes autologous skin cells to safely and effectively restore pigmentation, marking a significant advancement in vitiligo care.

FDA Extends Review of GSK's Momelotinib for Myelofibrosis

  • The FDA has delayed its decision on GSK's momelotinib, a potential treatment for myelofibrosis, to September 16 to review additional data.
  • Momelotinib, acquired through GSK's $2 billion purchase of Sierra Oncology, aims to alleviate symptoms like anemia and reduce transfusion needs in myelofibrosis patients.
  • The drug targets a different protein than existing JAK inhibitors like Jakafi, offering a potential new option for patients who don't respond well to current treatments.
  • Analysts speculate the additional data may support a broader use of momelotinib, although Jakafi is expected to remain the preferred first-line treatment.

4SC's Resminostat Advances Towards European Market Approval for CTCL Maintenance Therapy

  • The EMA has accepted 4SC's Letter of Intent to Submit a Marketing Authorization Application (MAA) for resminostat, marking a key regulatory step.
  • The EMA has also accepted 'Kinselby' as the trade name for resminostat, facilitating its potential commercialization in Europe.
  • Resminostat, if approved, would be the first HDAC inhibitor for CTCL maintenance therapy in Europe, addressing a significant unmet need.
  • 4SC anticipates submitting the MAA in Q1 2024, based on positive data from the pivotal RESMAIN study.

OliX Pharmaceuticals Initiates Phase I Trial for Androgenic Alopecia Treatment

OliX Pharmaceuticals has commenced a Phase I clinical trial in Australia for OLX72021, an RNAi therapeutic aimed at treating androgenic alopecia, also known as male-pattern baldness. The trial focuses on assessing the safety, tolerability, and pharmacokinetics of a single intradermal injection of OLX72021.

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