Socioeconomic Factors and Parental Smoking Linked to Worse MS Outcomes in Children
• Children from socioeconomically disadvantaged backgrounds show more severe brain inflammation and tissue loss when diagnosed with multiple sclerosis, according to a new study published in Neurology.
• Researchers found that parental smoking may significantly increase MS risk in genetically predisposed children, potentially triggering immune system dysfunction and brain changes visible on MRI scans.
• These findings suggest that MS development may begin in childhood, highlighting the importance of early environmental interventions and supporting the growing evidence of Epstein-Barr virus as a potential MS trigger.
AIM ImmunoTech Advances to Phase 2 Trial of Ampligen-Imfinzi Combination for Late-Stage Pancreatic Cancer
• Safety Committee grants approval for Phase 2 portion of AIM ImmunoTech's Phase 1b/2 trial evaluating Ampligen in combination with AstraZeneca's Imfinzi for late-stage pancreatic cancer treatment.
• The trial, conducted at Erasmus Medical Center in the Netherlands, addresses a critical unmet need in oncology, with pancreatic cancer claiming approximately 50,000 American lives annually.
• The combination therapy aims to enhance immune system response through Ampligen's immune reprogramming properties alongside Imfinzi's checkpoint inhibition mechanism.
AIM ImmunoTech Announces Clinical Milestones and Upcoming Studies for Ampligen
• AIM ImmunoTech highlights the continued development of Ampligen (rintatolimod) across multiple clinical programs targeting pancreatic cancer and Long-COVID.
• Several key milestones are anticipated in the next 18 months, with trials partially funded by the National Cancer Institute, AstraZeneca, and Merck.
• Positive preliminary data were reported from a Phase 1b/2 study of Ampligen and Imfinzi for late-stage pancreatic cancer, and Phase 2 data for post-COVID fatigue.
• AIM ImmunoTech received patents for Ampligen in treating endometriosis and post-COVID fatigue, and completed cGMP manufacturing of 9,042 clinical vials.
Vesper Bio Initiates Phase Ib/IIa Trial of VES001 for Frontotemporal Dementia (FTD) with GRN Mutations
• Vesper Bio has commenced a Phase Ib/IIa clinical trial, named SORT-IN-2, to assess VES001 in asymptomatic patients with GRN mutations causing frontotemporal dementia (FTD).
• VES001, an orally administered drug, aims to normalize progranulin levels by inhibiting sortilin, a protein that degrades progranulin, which is deficient in FTD(GRN) patients.
• The SORT-IN-2 trial is an open-label study conducted in the Netherlands and the United Kingdom, with dosing completion expected by mid-2025.
• Previous Phase 1a studies showed VES001 was safe, well-tolerated, and effectively increased progranulin levels in healthy volunteers, supporting its potential as a disease-modifying treatment.
European Commission Approves Emcitate (Tiratricol) as First Treatment for MCT8 Deficiency
• The European Commission has granted marketing authorization for Emcitate (tiratricol), making it the first approved treatment for monocarboxylate transporter 8 (MCT8) deficiency in the EU.
• The approval is based on data from Triac Trial I, the Erasmus University Medical Center Cohort Study, and preliminary results from Triac Trial II, demonstrating significant reductions in serum T3 concentrations.
• Emcitate is indicated for treating peripheral thyrotoxicosis in MCT8 deficiency patients from birth, addressing symptoms like increased heart rate and muscle weakness.
• Egetis Therapeutics anticipates initiating pricing and reimbursement discussions in Europe, with the first launch expected in the second quarter of 2025.
Rintatolimod and Durvalumab Combination Shows Promise in Late-Stage Pancreatic Cancer
• Preliminary data from the DURIPANC trial indicates that rintatolimod plus durvalumab demonstrates early disease control in late-stage pancreatic cancer.
• Two of three patients in the first dose cohort maintained stable disease at 6 months, with ongoing treatment and assessment.
• The combination therapy was generally well-tolerated, with no severe adverse effects observed in the first cohort.
• Improvements in quality of life were noted, correlating with stable disease, offering hope for patients with limited treatment options.
Landmark Clinical Trials of 2024: Promising Advances in Cancer, Malaria, and Genetic Diseases
• AstraZeneca's trastuzumab deruxtecan shows a 73.3% intracranial response rate in breast cancer patients with brain metastases, offering a potential new treatment option.
• Oxford University's malaria vaccine, R21/Matrix-M, demonstrates a 78% efficacy rate in young African children, marking a significant improvement over existing vaccines.
• Verve Therapeutics' VERVE-101/VERVE-102 utilizes in vivo base-editing to deactivate the PCSK9 gene mutation, potentially providing a one-time treatment for high cholesterol.
• A Netherlands Cancer Institute study suggests that neoadjuvant ipilimumab and nivolumab may eliminate the need for surgery in stage III melanoma patients, with a 72% positive response.
uniQure's Etranacogene Dezaparvovec Shows Sustained Therapeutic Benefit in Hemophilia B Patients
• Two-year follow-up data from a Phase IIb study shows that etranacogene dezaparvovec maintains stable Factor IX (FIX) activity at therapeutic levels in hemophilia B patients.
• The gene therapy demonstrated a mean FIX activity of 44.2% of normal at two years post-administration, with reduced bleeding rates and FIX replacement therapy usage.
• Long-term data from a Phase I/II study of AMT-060, uniQure's first-generation gene therapy, shows sustained clinical benefits up to five years, including increased FIX activity.
• Regulatory submissions to the FDA and EMA for etranacogene dezaparvovec are anticipated to begin in the second half of 2021, incorporating data from the ongoing Phase III HOPE-B trial.
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