UNIVERSITY OF MIAMI

The FDA has authorized emergency compassionate use of ZyVersa Therapeutics' VAR 200 for a patient with ApoCII amyloidosis, an ultra-rare kidney disease with no effective treatment options.

Adolore BioTherapeutics published preclinical data in Molecular Therapy demonstrating biosafety and efficacy of its rdHSV-CA8* gene therapy for chronic knee pain from osteoarthritis.

The phase 2/3 Alliance A092104 trial evaluating olaparib plus temozolomide in advanced uterine leiomyosarcoma failed to meet its primary progression-free survival endpoint compared to standard care.

The FDA's Oncology Drugs Advisory Committee unanimously approved minimal residual disease (MRD) as an end point for accelerated approval of multiple myeloma therapies in April 2024, potentially reducing trial timelines from 10-15 years to just 3 years.

Alcohol-related deaths in the United States nearly doubled from 1999 to 2020, rising from 10.7 to 21.6 per 100,000 people, with women experiencing the largest relative increase despite men having higher overall rates.

• Survivorship care addresses the comprehensive needs of cancer patients after primary treatment, with approximately 20 million cancer survivors in the U.S. expected to reach 26-27 million by 2040. • Evidence-based survivorship programs that address emotional, physical, and practical needs have been shown to reduce emergency department visits and rehospitalizations while improving quality of life outcomes. • Multidisciplinary approaches including psychological support, wellness clinics, and supportive therapies are essential components of comprehensive cancer care, particularly as the aging population faces increased cancer rates and treatment complexities.

• A groundbreaking drug Qalsody, targeting the SOD-1 gene mutation, demonstrates first-ever improvements in some genetic ALS patients, marking a significant advancement in treatment. • Researchers at the University of Miami are conducting trials to evaluate Qalsody's potential in preventing ALS onset in individuals carrying the SOD-1 genetic mutation. • The breakthrough treatment, while currently applicable to a small subset of ALS patients with genetic mutations, could provide insights for treating the broader ALS population.

• Whole genome sequencing demonstrates significant potential in enhancing clinical prognostication and identifying drug resistance mechanisms in multiple myeloma patients, though high costs and time constraints currently limit routine implementation. • The technology enables identification of predictive biomarkers for treatment selection, allowing clinicians to proactively adjust therapeutic approaches and avoid ineffective treatments. • Integration of WGS into clinical practice could revolutionize personalized medicine in multiple myeloma by improving risk stratification and treatment optimization, according to expert insights.

• Provectus Biopharmaceuticals has launched VisiRose, a new entity focused on Rose Bengal Photodynamic Antimicrobial Therapy (RB PDAT) for eye infections. • VisiRose will commercialize RB PDAT, combining Rose Bengal Sodium (RBS) with a light-based device for infectious keratitis and other serious eye infections. • The University of Miami is a minority equity shareholder, with the therapy based on research from Bascom Palmer Eye Institute. • RB PDAT offers a non-invasive treatment option for eye infections caused by bacteria, fungi, and parasites, including multidrug-resistant organisms.

• New research reveals tobacco smoking contributes to molecular pathogenesis of myelodysplastic syndromes (MDS), with smokers showing higher rates of specific high-risk genetic mutations. • Study demonstrates clear dose-response relationship, with heavy smokers having up to 3.5 times more mutations than non-smokers and 27% higher 5-year disease progression rate. • Findings from the largest national MDS study highlight the critical importance of smoking cessation counseling at diagnosis to potentially delay disease progression and improve survival outcomes.