Clinical Trial News
RNA-Targeted Drug Discovery Summit to Address Key Challenges in Small Molecule Development
• Leading biopharmaceutical executives and researchers gather at the 3rd RNA-Targeted Drug Discovery Summit to advance RNA-targeting small molecule therapeutics development and improve patient outcomes.
• The summit aims to tackle critical challenges in RNA drug discovery, including RNA structure determination, target selection optimization, and translation into clinical development.
• Industry leaders from Scripps Research Institute, Anima Biotech, and Arrakis Therapeutics will share insights on enhancing medicinal chemistry approaches and RNA biology understanding for robust therapeutic development.
Phase 3 Trial Shows Low-Dose Carbetocin Effective for Hyperphagia in Prader-Willi Syndrome
• Levo Therapeutics' Phase 3 CARE-PWS trial found that the lower 3.2mg dose of intranasal carbetocin (LV-101) significantly reduced hyperphagia and anxiety symptoms in patients with Prader-Willi syndrome, while the higher 9.6mg dose did not meet primary endpoints.
• The oxytocin-receptor agonist was generally well-tolerated, with mild to moderate side effects including headache, flushing, and diarrhea at the effective dose, offering potential relief for a condition with no currently approved treatments for its most challenging symptoms.
• Over 98% of trial participants elected to continue receiving the treatment in the long-term follow-up phase, with maintained benefits or further improvements observed, demonstrating sustained clinical value for this rare neurodevelopmental disorder affecting approximately 1 in 16,000 births.
Highlighted Clinical Trials:
Levo Therapeutics, Inc.
Posted 11/20/2018
NHS Embraces Telemedicine Revolution: 71% of GP Consultations Now Delivered Remotely
• The UK's NHS has seen a dramatic shift in healthcare delivery, with 71% of routine GP consultations now conducted remotely, compared to just 25% before the COVID-19 pandemic.
• Health Secretary Matt Hancock has mandated that all future consultations should be teleconsultations unless clinically contraindicated, marking a significant policy shift in healthcare delivery.
• While telemedicine adoption shows promise, challenges remain including digital access inequalities, safeguarding concerns for vulnerable populations, and technological barriers for healthcare providers.
Izotropic Files Pre-Submission Application With U.S. FDA for Breast CT Imaging System
Izotropic Corporation has taken a significant step towards market approval for its Breast CT Imaging System by filing a Pre-Submission Application with the U.S. FDA. This move marks a pivotal moment in the company's journey to revolutionize breast cancer imaging, aiming to provide a less painful and more efficient diagnostic tool compared to traditional mammography.
Nivolumab Plus Ipilimumab Shows Durable Response in MSI-H/dMMR Colorectal Cancer
• Nivolumab plus low-dose ipilimumab demonstrates robust and durable clinical benefit in first-line treatment of MSI-H/dMMR metastatic colorectal cancer.
• At a median follow-up of 29 months, the combination therapy showed deepening responses and promising progression-free and overall survival rates.
• The objective response rate increased to 69%, with a complete response rate of 13%, indicating improved efficacy with longer follow-up.
• The safety profile remained consistent, with the combination being well-tolerated and no new safety signals identified.
Ultrasound-Guided Lung Recruitment Manoeuvre Shows Promise in Reducing Postoperative Atelectasis in Children
A recent study compared the effectiveness of an ultrasound-guided lung recruitment manoeuvre with a conventional method in reducing postoperative atelectasis in children. The findings suggest that the ultrasound-guided approach may offer better outcomes in preventing intra-operative atelectasis and postoperative desaturation, though its impact on postoperative atelectasis remains uncertain.
FDA Approves Roche's Evrysdi: A More Affordable Oral Treatment for Spinal Muscular Atrophy
• Roche's Evrysdi (risdiplam) receives FDA approval for treating spinal muscular atrophy in patients 2 months and older, offering a daily oral administration option for patients.
• The drug demonstrates significant clinical improvements across multiple SMA types, including helping infants achieve key motor milestones and improving survival without permanent ventilation.
• With annual pricing between $100,000-$340,000 based on patient weight, Evrysdi presents a more cost-effective alternative to existing treatments that cost up to $2.1 million per dose.
AstraZeneca and Daiichi Sankyo Form $6 Billion Partnership for Novel TROP2-Targeting Cancer Drug
• AstraZeneca has entered a $6 billion collaboration with Daiichi Sankyo to develop DS-1062, a novel antibody-drug conjugate targeting TROP2, a protein highly expressed in multiple cancer types.
• The partnership includes an upfront payment of $1 billion from AstraZeneca, with potential additional payments of up to $5 billion based on regulatory and sales milestones.
• The companies will jointly develop and commercialize DS-1062 globally for multiple TROP2-expressing tumors, particularly focusing on lung and breast cancers, building on their previous successful collaboration with Enhertu.
Advances and Challenges in Gynecologic Cancer Treatment
Recent developments in gynecologic cancer treatment include the assessment of CBX-12 for ovarian cancer, mixed findings on Keytruda and chemotherapy combinations, and FDA approvals for new treatments. Patients are encouraged to understand their disease's molecular makeup and consider clinical trials.
FDA Grants Fast Track Designation for Abenacianine in Lung Cancer Surgery
The FDA has granted fast track designation to abenacianine, a drug aimed at enhancing the visualization of lung tumors during minimally invasive surgery, marking a significant advancement in lung cancer treatment.