Vanderbilt University
🇺🇸United States
- Country
- 🇺🇸United States
- Ownership
- Private
- Established
- 1873-01-01
- Employees
- 10K
- Market Cap
- -
- Website
- http://www.vanderbilt.edu
GeoVax Advances Gedeptin Gene Therapy for Solid Tumors with Expanded Patent Portfolio and Promising Clinical Data
• GeoVax has strengthened its Gedeptin therapy platform with newly allowed patent claims covering the synergistic combination of gene therapy with radiation for treating various solid tumors.
• Clinical data presented at AACR 2025 demonstrated Gedeptin's safety profile and efficacy in heavily pretreated head and neck cancer patients, with median progression-free and overall survival of 7.0 months.
• The company is advancing to a Phase 2 trial combining Gedeptin with pembrolizumab (Keytruda) as a neoadjuvant therapy for relapsed head and neck cancer, leveraging potential synergy with immune checkpoint inhibitors.
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.
CareDx Showcases Extensive Clinical Evidence for Transplant Monitoring Solutions at ISHLT 2025
• CareDx presented 60 abstracts including 19 oral presentations at the 2025 ISHLT meeting, demonstrating the growing clinical adoption of their transplant monitoring solutions across 92 transplant centers.
• New findings from the SHORE study revealed that HeartCare is prognostic of long-term graft outcomes independent of biopsy results and is being used by clinicians to guide immunosuppression management decisions.
• Data from the ALAMO study showed AlloSure Lung may predict development of Chronic Lung Allograft Dysfunction (CLAD) and effectively monitors acute cellular rejection and treatment response in lung transplant patients.
Ziresovir Shows Breakthrough Efficacy Against RSV in Infants Under 6 Months with Long-Term Benefits
• Phase III trial results published in The Lancet Child & Adolescent Health demonstrate ziresovir is the first RSV-targeted antiviral to show significant clinical efficacy in hospitalized infants under 6 months of age.
• Treatment with ziresovir led to a 54.5% greater reduction in bronchiolitis symptoms and significantly faster viral clearance compared to placebo, with improvements in respiratory rate, wheezing, and chest retractions.
• Two-year follow-up data revealed long-term respiratory benefits, including 3.6 times lower incidence of recurrent wheezing and reduced asthma rates, suggesting early antiviral intervention may prevent chronic respiratory conditions.
Qureight's AI Imaging Platform to Support Avalyn's Inhaled Pirfenidone Trial in Pulmonary Fibrosis
• Qureight and Avalyn Pharma have formed a strategic partnership to integrate deep-learning imaging technology in the Phase 2b MIST trial evaluating inhaled pirfenidone (AP01) for progressive pulmonary fibrosis.
• The collaboration marks the first-ever use of deep-learning imaging technologies in a progressive pulmonary fibrosis clinical study, aiming to enhance treatment response measurement.
• Avalyn's inhaled pirfenidone formulation targets improved efficacy and reduced side effects compared to current oral antifibrotic therapies by delivering medication directly to affected lung tissue.
Stanford Study: Cancer Drug EBC-46 Shows Breakthrough Potential for HIV Eradication
Stanford researchers have discovered that EBC-46, a compound already known for cancer treatment, demonstrates remarkable effectiveness in targeting dormant HIV cells. The study revealed that EBC-46 analogs achieved a 90% success rate in reversing HIV latency, quadrupling the effectiveness of existing treatments and marking a significant step toward a potential HIV cure.
Trump Administration Repeals Key Drug Pricing Executive Order, Creating Uncertainty in Healthcare Reform
• President Trump's repeal of Executive Order 14087 halts significant drug pricing initiatives, including a $2 Medicare copay program for generic medications and reforms to the FDA's accelerated approval pathway.
• While the Inflation Reduction Act's core provisions remain intact, including Medicare's drug price negotiation authority and insulin cost caps, the administration's 60-day regulatory freeze creates uncertainty about future healthcare policy direction.
• Major pharmaceutical products including Biktarvy, Ozempic, and Hemlibra have already seen price increases in 2025, raising concerns about medication affordability and access for patients.
NSCLC Treatment Landscape: Biomarker Testing, Evolving Guidelines, and ADC Developments
• Biomarker testing is crucial for personalized NSCLC treatment, enabling tailored therapies for both early- and late-stage disease.
• Updated lung cancer screening guidelines expand eligibility by removing quit time and reducing pack-year criteria, potentially benefiting more at-risk patients.
• Sacituzumab govitecan monotherapy failed to improve overall survival in the EVOKE-01 trial, despite being well-tolerated.
• A biologics license application for datopotamab deruxtecan was withdrawn, with a new application planned based on data from other trials.
VU319 Shows Promise in Phase 1 Trial for Alzheimer's Disease
• VU319, an M1 positive allosteric modulator, has demonstrated encouraging results in a Phase 1 clinical trial for Alzheimer's disease and other neurodegenerative conditions.
• The trial showed signs of target engagement at the highest dose tested, with no typical side effects, suggesting a potentially improved therapeutic index.
• VU319 enhances acetylcholine activity at the M1 receptor, addressing neurotransmitter dysfunction seen in Alzheimer's and schizophrenia.
• The successful trial marks a significant milestone in Vanderbilt University's drug discovery efforts, from basic research to clinical testing.
Alzheimer's Research Faces Scrutiny Amidst Rising Concerns of Scientific Misconduct
• Neurologist Matthew Schrag highlights the increasing prevalence of research misconduct in Alzheimer's disease studies, which he believes has slowed the development of effective treatments.
• Schrag identifies image manipulation, overly perfect data, and data aligning too perfectly with hypotheses as potential red flags for research fraud.
• He emphasizes the need for greater transparency and data sharing within the scientific community to address and rectify instances of misconduct.
• Concerns are raised about the ethical implications of ongoing clinical trials based on potentially flawed data, urging regulatory agencies to prioritize patient safety.
Ifetroban Shows Promise in Improving Heart Function for DMD Patients in Phase 2 Trial
• Ifetroban, an oral thromboxane receptor antagonist, significantly improved left ventricular ejection fraction (LVEF) in Duchenne muscular dystrophy (DMD) patients.
• The FIGHT DMD trial demonstrated a 3.3% improvement in LVEF with high-dose ifetroban, contrasting with a decline in the placebo group.
• Compared to natural history controls, high-dose ifetroban showed a 5.4% overall LVEF improvement, suggesting a potential disease-modifying effect.
• Ifetroban has received Orphan Drug and Rare Pediatric Disease designations, potentially becoming the first targeted therapy for DMD-related heart disease.
FDA Approves Novel Therapies for Frostbite, Food Allergies, and Synovial Sarcoma in 2024
• The FDA approved Aurlumyn (iloprost) in February 2024, the first-ever treatment for severe frostbite, significantly reducing the risk of amputation in affected patients.
• Omalizumab (Xolair) received FDA approval in February 2024 to reduce allergic reactions following accidental food exposure, benefiting individuals with life-threatening food allergies.
• Tecelra, a T-cell receptor gene therapy, was approved in August 2024 for metastatic synovial sarcoma, marking a significant advancement in treating this rare cancer.
• The FDA has authorized 950 AI/ML-enabled medical devices, including 21 in 2024, showing progress in integrating artificial intelligence into healthcare for improved diagnostics and treatments.
$11.4 Million NIH Grant to Study Nitric Oxide Treatment for Newborns with Congenital Diaphragmatic Hernia
• A $11.4 million NIH grant funds a multi-center clinical trial to investigate the efficacy of nitric oxide in treating newborns with congenital diaphragmatic hernia (CDH).
• The NoNO Trial will randomize nitric oxide use at 19 institutions, comparing outcomes before and after de-implementation to assess its impact on oxygenation and ECMO needs.
• The study aims to understand barriers to discontinuing nitric oxide use and address key management questions in CDH, a condition affecting 1 in 2,000-4,000 live births.
• The University of Tennessee Health Science Center will serve as the clinical coordinating center, with the University of Texas Health Science Center directing data coordination.
Real-World Data Confirms High Effectiveness of RSV Vaccines in Older Adults
• A real-world study found RSV vaccines offer approximately 80% protection against severe illness and hospitalization for adults aged 60 and older.
• The study included data from multiple U.S. healthcare networks, representing 230 hospitals and 245 emergency departments nationwide.
• The analysis showed the vaccine's benefit was particularly evident in adults aged 75 and older, the most vulnerable age group.
• Findings highlight the potential for significant cost savings due to reduced RSV-related hospitalizations, estimated between $1.2 and $5 billion annually.
Cancer Vaccine Shows Promise in Blocking Early-Stage Tumor Progression in Mouse Model
• A cancer vaccine demonstrated the ability to block tumor progression in a mouse model when administered at an early stage of lesion development.
• The study found that tumor-specific T cell populations are present in early-stage lesions and can be therapeutically targeted by vaccination.
• Early treatment with the cancer vaccine effectively blocked tumor progression, even when immune checkpoint blockade therapies were ineffective.
• The research highlights the importance of timing in cancer vaccination, suggesting earlier intervention may improve long-term outcomes.
Cassava Sciences Faces Mounting Pressure to Halt Simufilam Alzheimer's Trials Amid Data Manipulation Allegations
• Cassava Sciences is under scrutiny following allegations of data manipulation in its Phase 2 trial of simufilam, an experimental Alzheimer's drug, leading to calls for a halt to its clinical programs.
• The SEC fined Cassava $40 million for allegedly misleading investors with doctored data, with accusations centered on manipulated biomarker data by a consultant, Hoau-Yan Wang.
• Independent analysis by experts like Matthew Schrag raised concerns about image tampering and the scientific rationale behind simufilam, questioning the integrity of Cassava's clinical results.
• Despite ongoing controversies and calls for transparency, Cassava Sciences continues its Phase 3 trials, with top-line data expected by the end of this year and further results in mid-2025.
Cassava Sciences' Alzheimer's Drug Simufilam Faces Scrutiny Amid Data Integrity Concerns
• Cassava Sciences' simufilam, an Alzheimer's drug, is under scrutiny following a $40 million settlement related to data manipulation allegations.
• Despite the controversy, Cassava continues Phase 3 trials with over 1,900 patients, while experts question the ethics and validity of the ongoing research.
• Concerns persist regarding data integrity, with experts suggesting regulatory agencies should intervene to protect clinical trial participants.
• The FDA has the authority to pause or terminate clinical trials if it discovers issues related to data integrity.
iTeos' TIGIT Inhibitor Shows Promise in Lung Cancer, Reviving Interest in the Target
• iTeos Therapeutics' belrestotug, combined with GSK's Jemperli, demonstrated encouraging tumor shrinkage in lung cancer patients, with overall response rates significantly higher than Jemperli alone.
• The Phase 2 trial results have sparked renewed interest in TIGIT as a therapeutic target, despite previous setbacks with other TIGIT inhibitors from companies like Roche and Merck.
• Safety concerns exist, as the combination therapy was associated with a higher incidence of serious side effects and treatment-related deaths compared to Jemperli monotherapy.
• iTeos and GSK have initiated a Phase 3 trial to further evaluate the efficacy and safety of belrestotug plus Jemperli against the current standard of care, Keytruda.
Cancer Clinical Trials Inappropriately Exclude Patients with Duffy-Null Phenotype
• A recent study reveals that many cancer clinical trials exclude individuals with the Duffy-null phenotype, a trait common in people of African and Middle Eastern descent.
• The exclusion is due to lower neutrophil counts in Duffy-null individuals, which are misinterpreted as a sign of increased infection risk during cancer treatment.
• Researchers recommend that clinical trials adjust neutrophil count criteria to include Duffy-null individuals and avoid inappropriate dose reductions.
• Addressing this inequity could improve clinical trial representation and treatment outcomes for diverse populations.
FDA Approves Cobenfy, a Novel Schizophrenia Treatment Targeting Cholinergic Receptors
• The FDA has approved Cobenfy (xanomeline and trospium chloride) as the first new class of drug for schizophrenia in over 30 years, offering a novel approach to treatment.
• Cobenfy targets muscarinic receptors, unlike traditional antipsychotics that focus on dopamine, potentially reducing side effects like weight gain and movement disorders.
• Clinical trials demonstrated Cobenfy significantly reduced schizophrenia symptoms compared to placebo, marking a transformative moment in managing this challenging condition.
• Expected to launch in late October, Cobenfy offers a new option for adults with schizophrenia, with ongoing studies exploring its potential in Alzheimer's psychosis and other conditions.
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