FDA Requests Additional Trial for Novavax COVID-19 Vaccine Before Full Approval
• The FDA has asked Novavax to conduct an additional randomized, controlled study of its COVID-19 vaccine NVX-CoV2601 before considering full approval, despite the vaccine having emergency use authorization since 2022.
• Interim results from a phase 2/3 study showed Novavax's updated XBB.1.5 variant vaccine generated 5.8 times higher neutralizing antibody levels compared to the original formulation, with a favorable safety profile.
• The FDA's request represents a significant setback for Novavax, which had expected approval based on prior conversations with regulators and had already passed its April 1 PDUFA date.
CereVasc's eShunt System Receives FDA Breakthrough Designation for Pediatric Hydrocephalus Treatment
• CereVasc's eShunt System received its second FDA Breakthrough Device Designation for treating communicating hydrocephalus in pediatric patients aged 12 and older, enabling priority review and enhanced regulatory communication.
• Research published in the Journal of NeuroInterventional Surgery demonstrated that endovascular shunt placement was feasible in 67% of pediatric patients studied, with no statistically significant age-based differences in eligibility.
• The minimally invasive eShunt System previously achieved primary safety and efficacy endpoints in a US study of 30 elderly patients with Normal Pressure Hydrocephalus, with 97% showing symptom improvement.
Blue Cross Blue Shield of Massachusetts to End Coverage for Weight Loss GLP-1 Drugs in 2026
• Blue Cross Blue Shield of Massachusetts announced it will stop covering GLP-1 medications for weight loss starting January 1, 2026, while continuing coverage for diabetes treatment.
• The insurer cited unsustainable financial burden as GLP-1 drugs now account for nearly 20% of its pharmacy spend, exceeding $300 million in 2024, double the previous year's amount.
• The policy change will affect employers with fewer than 100 employees automatically, while larger employers will have the option to include or exclude weight loss GLP-1 coverage in their plans.
First Patient Treated with eShunt System in Landmark NPH Trial, Marking New Era in Hydrocephalus Treatment
• CereVasc has initiated the STRIDE pivotal trial at VCU Health, comparing their novel eShunt System to traditional VP shunts for normal pressure hydrocephalus treatment.
• The eShunt System represents the first new treatment option for NPH in over 60 years, offering a minimally invasive endovascular approach to address this condition affecting 800,000 Americans.
• The trial aims to demonstrate improved recovery times and reduced complications compared to standard VP shunts, potentially making treatment accessible to more patients with comorbidities.
OKYO Pharma Advances Phase 2a Trial for First-Ever Neuropathic Corneal Pain Treatment
• OKYO Pharma expects to complete enrollment of 48 patients in its Phase 2a trial of OK-101, the first FDA-cleared investigational drug specifically for neuropathic corneal pain, by Q2 2025.
• The company recently reported positive Phase 2b results for OK-101 in dry eye disease, showing significant improvements in conjunctival staining and symptom reduction with a favorable safety profile.
• The Phase 2a NCP trial, led by renowned expert Dr. Pedram Hamrah at Tufts Medical Center, is designed as a double-masked, randomized, 12-week placebo-controlled study.
TAICHIKNEE Trial Updates Ethics Documentation for Web-Based Tai Chi Intervention Study
• The TAICHIKNEE trial, which assesses web-based Tai Chi for knee osteoarthritis, has updated its ethics and regulatory documentation after one year.
• Researchers are comparing Tai Chi interventions to routine care for individuals with knee pain due to osteoarthritis.
• The study aims to promote mind-body approaches for managing knee osteoarthritis across healthcare systems, potentially reducing reliance on pharmacological interventions.
• Initial findings were presented at the NIH Pragmatic Trials Collaboratory’s 2024 Annual Steering Committee Meeting, highlighting the study's significance.
AI-Driven Discovery Unveils Cancer Therapy Breakthrough
Rakovina Therapeutics Inc. has synthesized groundbreaking AI-designed drug candidates targeting critical cancer treatments, potentially reshaping oncology research.
Alleviant Medical's No-Implant Atrial Shunt Receives FDA IDE and Breakthrough Designation for HFrEF
• Alleviant Medical's novel atrial shunt technology, which leaves no permanent implant, receives FDA IDE approval for a pivotal trial in HFrEF patients.
• The FDA also grants Breakthrough Device designation to Alleviant's technology for HFrEF, expediting its development and review process.
• The ALLAY-HFrEF trial will evaluate the safety and effectiveness of the Alleviant System in heart failure patients with reduced ejection fraction.
• Alleviant Medical secures $90 million in financing to expand its focus to the full spectrum of chronic heart failure.
Brolucizumab Shows Non-Inferior Visual Acuity and Superior Anatomic Outcomes Compared to Aflibercept in DME
• A recent meta-analysis of randomized controlled trials compared brolucizumab to aflibercept for treating diabetic macular edema (DME).
• Brolucizumab was found to be non-inferior to aflibercept in terms of best-corrected visual acuity from baseline.
• The study showed brolucizumab significantly improved anatomic parameters, specifically the change in central subfield thickness, compared to aflibercept.
• No statistically significant differences in ocular adverse events were observed between the two anti-VEGF therapies.
Reprieve System Shows Promise in Decongesting Acute Heart Failure Patients
• The Reprieve System demonstrated safe and effective fluid and sodium removal in patients with acute decompensated heart failure (ADHF) in a first-in-human study.
• The FASTR pilot trial showed the Reprieve System led to greater sodium excretion, urine output, and weight loss compared to optimal diuretic therapy (ODT).
• Reprieve Cardiovascular's device aims to improve patient outcomes and prevent hospital readmissions by precisely managing fluid overload and optimizing kidney function.
• The company is planning a pivotal trial (FASTR II) in the first half of 2025 to further validate the Reprieve System's efficacy and safety for ADHF treatment.
60 Degrees Pharma's Tafenoquine Receives IRB Approval for Phase II Chronic Babesiosis Study
• 60 Degrees Pharmaceuticals has received IRB approval for a Phase II study evaluating Tafenoquine for chronic babesiosis.
• The open-label study will assess the efficacy and safety of Tafenoquine over 90 days in patients with chronic babesiosis.
• Patient enrollment is expected to commence in Q3 2025, targeting those with significant functional impairment for at least six months.
• The company estimates a substantial addressable market for Tafenoquine in babesiosis, potentially exceeding 400,000 patients by 2035.
OKYO Pharma Doses First Subject in Phase II Trial of OK-101 for Neuropathic Corneal Pain
• OKYO Pharma has dosed the first subject in its Phase II clinical trial of OK-101 for neuropathic corneal pain (NCP).
• The trial is a double-masked, randomized, placebo-controlled study involving 48 patients diagnosed with NCP, confirmed via confocal microscopy.
• OK-101, a lipid-conjugated chemerin peptide agonist, targets the ChemR23 receptor on immune cells, aiming to reduce inflammation associated with corneal nerve damage.
• There are currently no FDA-approved treatments for NCP, making OK-101 a potential breakthrough therapy for this chronic condition.
Specialty Drug Coverage Disparities Create Access Barriers as PBMs Push for Benefit Integration
• Research from Tufts Medical Center reveals 15% of health plans have inconsistent coverage requirements between medical and pharmacy benefits for the same specialty drugs, creating patient access challenges.
• Vertical integration of pharmacy benefit managers (PBMs) with insurers drives the shift of specialty drugs from medical to pharmacy benefits, leading to complex navigation requirements for patients.
• White and brown bagging practices, aimed at maximizing profits through insurer-owned pharmacies, may increase system waste and burden patients with additional drug management responsibilities.
OKYO Pharma Doses First Patient in Phase 2 Trial of OK-101 for Neuropathic Corneal Pain
• OKYO Pharma has dosed the first patient in its Phase 2 clinical trial evaluating OK-101 for the treatment of neuropathic corneal pain (NCP).
• The Phase 2 trial is a randomized, double-masked, placebo-controlled study aiming to enroll 48 patients with confirmed NCP.
• OK-101 is a non-opioid therapeutic candidate designed to alleviate pain associated with corneal nerve damage, addressing an unmet need.
• The trial's primary endpoint is to measure pain relief using the Visual Analog Scale (VAS).
Synthetic THC Shows Promise in Reducing Agitation in Alzheimer's Patients
• A clinical trial demonstrated that dronabinol, a synthetic THC, significantly reduced agitation in Alzheimer's disease patients by 30%.
• The study found dronabinol to be well-tolerated, without causing common adverse effects like delirium or seizures.
• Researchers plan to conduct larger, longer-term studies to explore additional applications of medical cannabis in managing symptoms for patients and caregivers.
• Agitation affects approximately 40% of Alzheimer's patients and is a primary reason for emergency department visits and long-term care admissions.
Ribociclib Pharmacokinetics Vary Significantly in Black Patients Based on CYP3A5 Genotype
• A recent study in *Nature* explored the pharmacokinetics and pharmacogenomics of ribociclib in Black patients with metastatic breast cancer.
• The study found significant differences in ribociclib exposure based on CYP3A5 genotype, with poor metabolizers showing higher drug exposure.
• These pharmacokinetic differences may influence ribociclib efficacy and toxicity, warranting further investigation for personalized dosing.
• The findings highlight the importance of considering pharmacogenomics in optimizing cancer treatment for diverse populations.
Study Reveals Quality Gap in Online Psoriasis Treatment Information Sources
• A comprehensive analysis of Google search trends shows adalimumab dominates public interest in psoriasis treatments, with users primarily seeking information about technical details and costs.
• Commercial and government websites provide the highest quality information about psoriasis treatments, scoring over 3 out of 4 points on JAMA benchmark criteria.
• Academic and medical practice websites demonstrate lower visibility and quality scores, highlighting a need for improved online presence and content development in professional medical sources.
Milk Elimination Diet Shows Promise as First-Line Therapy for Pediatric Eosinophilic Esophagitis
• A new study suggests that eliminating animal milk alone may be as effective as a four-food elimination diet for children with eosinophilic esophagitis (EoE).
• The randomized, multi-site clinical trial compared a one-food (milk) elimination diet (1FED) with a four-food (milk, egg, wheat, soy) elimination diet (4FED) in 63 children with EoE.
• Results showed that 1FED and 4FED achieved similar remission rates and improvements in symptoms, histologic, and endoscopic features over 12 weeks.
• Starting with milk elimination alone may be an easier and more tolerable initial approach for managing EoE in children, potentially increasing patient compliance.
Quality of Life Measures Gain Traction in Cancer Clinical Trials
• A nationwide clinical trial is assessing the quality of life (QoL) in young Hodgkin lymphoma patients, marking a shift towards accessible and interpretable patient-reported outcomes.
• The addition of brentuximab vedotin to chemotherapy showed clinical superiority and fewer negative effects on QoL compared to standard treatment, highlighting the importance of reducing late-term toxicities.
• Researchers are collaborating with the NCI and FDA to integrate patient experience into the drug approval process, leveraging standardized QoL measurement tools like PROMIS.
• The study emphasizes the need for thoughtful timing and methods for collecting patient-reported outcomes to improve participation rates and data quality in clinical trials.
CereVasc's eShunt System Receives FDA Breakthrough Device Designation for Normal Pressure Hydrocephalus
• CereVasc's eShunt System has been granted Breakthrough Device Designation by the FDA for treating Normal Pressure Hydrocephalus (NPH).
• The designation facilitates priority review and enhanced communication with the FDA during clinical trials and premarket assessment.
• The eShunt System offers a minimally invasive endovascular alternative to traditional surgical treatments for communicating hydrocephalus.
• CereVasc is preparing to initiate its STRIDE pivotal study to evaluate the eShunt System, with enrollment expected to begin in the second half of 2024.
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