UNIVERSITY OF FLORIDA
🇺🇸United States
- Country
- 🇺🇸United States
- Ownership
- Private
- Established
- 1853-01-01
- Employees
- 10K
- Market Cap
- -
- Website
- http://www.ufl.edu
Expanded GLP-1 Drug Access Could Save 42,000 Lives and Billions in Healthcare Costs Annually
• Expanding access to GLP-1 weight-loss medications like Ozempic and Zepbound could prevent more than 42,000 deaths annually in the United States, according to a recent Yale study published in PNAS.
• A separate study from Emory University found that a 25% weight reduction among obese individuals could save up to $5,442 per person annually in healthcare costs, with Medicare beneficiaries seeing the greatest savings.
• Despite their potential benefits, GLP-1 drugs face significant barriers including high costs exceeding $1,000 monthly without insurance, limited coverage policies, and ongoing supply shortages.
Dyne Therapeutics Strengthens Leadership Team with Key Scientific and Business Appointments
• Dyne Therapeutics has appointed Vikram Ranade, PhD, as Chief Business Officer and Ranjan Batra, PhD, as Chief Scientific Officer, bolstering its executive team ahead of potential product launches in 2027.
• Dr. Batra, an RNA biology expert with experience developing therapies for rare neuromuscular disorders, will lead research strategy and pipeline development for Dyne's FORCE™ platform technology.
• Current CSO Oxana Beskrovnaya, PhD, will transition to Chief Innovation Officer, focusing on maximizing the potential of Dyne's TfR1 delivery platform for new therapeutic applications in neuromuscular diseases.
FDA Approves First Treatment for Hyperphagia in Prader-Willi Syndrome
• The FDA has approved VYKAT XR (diazoxide choline) as the first therapy specifically targeting hyperphagia in Prader-Willi syndrome, offering new hope to patients and families affected by this rare genetic disorder.
• Clinical trials demonstrated VYKAT XR's efficacy through a Phase 3 randomized withdrawal study, where patients switching to placebo showed significant worsening of hyperphagia compared to those remaining on the medication.
• Soleno Therapeutics expects to make VYKAT XR available in the US by April 2025, supported by the Soleno One patient support program to facilitate access to this breakthrough treatment.
Study Reveals Ozempic's Potential as Depression Treatment in Landmark Analysis
• A major study of 30,000 patients demonstrates that GLP-1 receptor agonists like Ozempic and Wegovy may have significant antidepressant effects, opening new therapeutic possibilities.
• Diabetic patients over 66 taking GLP-1RAs showed fewer depression symptoms compared to those on traditional diabetes medications, suggesting broader mental health benefits.
• Researchers attribute the antidepressant effect to the drugs' anti-inflammatory properties, with evidence indicating reduced brain inflammation as a key mechanism.
GLP-1 Drug Exenatide Shows No Benefit in Large-Scale Parkinson's Disease Trial
• A rigorous 96-week Phase 3 trial involving 194 Parkinson's patients across six UK hospitals found that exenatide, a GLP-1 drug related to Ozempic, showed no benefit in treating or slowing disease progression.
• The double-blind study, led by UCL researchers, revealed no improvements in patient movements, symptoms, or brain imaging results, despite earlier smaller studies suggesting potential benefits.
• The disappointing results represent a setback for the half million Americans with Parkinson's disease, who currently have treatments for symptoms but no options to slow disease progression.
Amicus Therapeutics to Present Extensive Research on Fabry and Pompe Diseases at WORLDSymposium 2025
• Amicus Therapeutics will showcase 16 presentations at WORLDSymposium 2025, featuring comprehensive research on migalastat for Fabry disease and cipaglucosidase alfa plus miglustat for Pompe disease.
• Key studies include long-term patient outcomes from the FollowME Fabry Pathfinders registry and real-world evidence of treatment effectiveness in both rare diseases.
• Notable research highlights treatment satisfaction, quality of life improvements, and clinical outcomes in patients switching from existing therapies to Amicus' novel treatment approaches.
SAB-142 Shows Promise in Phase 1 Trial for Type 1 Diabetes
• SAB BIO's SAB-142 demonstrated a favorable safety profile in a Phase 1 trial, supporting chronic dosing in an outpatient setting.
• The Phase 1 trial of SAB-142 showed clinically validated multi-target immunomodulation, analogous to rabbit ATG.
• SAB BIO plans to advance SAB-142 into a Phase 2b trial in 2025 to evaluate its therapeutic potential in patients with new-onset T1D.
• Topline data from the Phase 1 trial supports SAB-142's potential as a transformative therapy for delaying the progression of type 1 diabetes.
GLP-1 Agonists Linked to Reduced Risk of 42 Health Conditions in Large Study
• A large observational study of nearly 2 million patients found that GLP-1 receptor agonists (GLP-1RAs) are associated with a reduced risk of 42 health conditions.
• The study, which included 215,000 GLP-1RA users, showed benefits beyond glucose control and weight loss, including neurological and respiratory conditions.
• While GLP-1RAs demonstrated broad benefits, the study also identified increased risks for certain conditions like kidney stones and low blood pressure.
• Researchers emphasize the need for further studies to confirm these findings and understand the long-term effects of GLP-1RA therapy.
Solid Biosciences' SGT-212 Receives FDA IND Clearance for Friedreich's Ataxia Gene Therapy Trial
• The FDA has cleared Solid Biosciences' IND for SGT-212, a gene therapy targeting both neurological and cardiac manifestations of Friedreich's ataxia.
• SGT-212 utilizes a dual route of administration, delivering full-length frataxin to the cerebellum via IDN infusion and to the heart via IV infusion.
• A Phase 1b trial is planned for the second half of 2025, assessing safety and tolerability in ambulatory and non-ambulatory adults with FA over five years.
• SGT-212 aims to address the underlying mitochondrial dysfunction in neurons and cardiomyocytes by restoring frataxin levels.
FDA Moves to Remove Oral Phenylephrine as Decongestant Due to Ineffectiveness
• The FDA is moving to phase out oral phenylephrine, a common decongestant in over-the-counter cold medicines, after determining it is no more effective than a placebo.
• This decision follows expert reviews of studies dating back to the 1960s, which revealed flaws and questionable data regarding phenylephrine's efficacy in oral form.
• Consumers may need to switch to alternatives like pseudoephedrine (available behind the counter) or nasal sprays for congestion relief.
• The FDA's decision is expected to take several months to finalize, allowing time for public comment and for drug manufacturers to reformulate or remove affected products.
BioCardia's CardiAMP Heart Failure Trial Results to be Presented at ACC 2025
• BioCardia's CardiAMP HF Phase 3 trial results will be presented at the American College of Cardiology (ACC) 2025 Scientific Sessions.
• The CardiAMP cell therapy uses a patient's own marrow cells to stimulate the body's natural healing response in the heart.
• CMS has approved reimbursement for study procedures, potentially mitigating costs and accelerating the trial's progress.
• The FDA approved a protocol amendment, allowing for the inclusion of more patients in the CardiAMP HF II trial.
Solid Biosciences' SGT-212 Receives FDA Fast Track Designation for Friedreich's Ataxia Treatment
• Solid Biosciences' SGT-212, a dual-route gene therapy for Friedreich's ataxia (FA), has received Fast Track designation from the FDA, expediting its development and review process.
• SGT-212 delivers the frataxin gene via intravenous and direct intradentate nucleus infusions, targeting both neurological and cardiac manifestations of FA.
• A Phase 1b clinical trial is planned for the second half of 2025 to assess the safety and tolerability of SGT-212 in adult FA patients.
• The Fast Track designation will allow Solid Biosciences to have more frequent interactions with the FDA and the potential to be eligible for priority review.
FDA Grants Fast Track Designation to Genascence's GNSC-001 for Osteoarthritis of the Knee
• The FDA has granted Fast Track designation to Genascence's GNSC-001, a gene therapy for osteoarthritis (OA) of the knee, expediting its development and review.
• GNSC-001 is designed to provide long-term inhibition of IL-1, a key mediator in OA, through a single intra-articular injection.
• Phase 1 trial data showed GNSC-001 was well-tolerated, with sustained IL-1Ra expression and a trend toward improved pain and function scores.
• Osteoarthritis affects over 30 million Americans, and current treatments offer only short-term symptom relief without slowing disease progression.
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