UNIVERSITY OF MINNESOTA

EMA Suspends Valneva's IXCHIQ Chikungunya Vaccine for Elderly Following Safety Concerns

15 days ago

• European Medicines Agency has suspended the use of Valneva's IXCHIQ chikungunya vaccine in people over 65 years old following reports of 17 serious adverse events, including two deaths, in elderly patients. • The suspension is temporary while authorities review safety data, with EMA maintaining current recommendations for IXCHIQ use in people aged 12 to 64 years, affecting over 40,000 doses administered worldwide. • Similar precautionary measures were previously implemented in the United States and France, with Valneva's Chief Medical Officer pledging full cooperation with health authorities while exploring potential updates to the product's indication.

CRISPR-Based Immunotherapy Achieves Complete Remission in Terminal Colon Cancer Patient

16 days ago

• A 35-year-old woman with stage 4 colon cancer achieved complete remission after receiving experimental CRISPR-Cas9 gene-edited immunotherapy at the University of Minnesota, marking an unprecedented outcome for advanced colorectal cancer. • The novel treatment targeted intracellular mechanisms that shield cancer cells from immune detection, using genetically modified immune cells that were specifically programmed to fight the patient's cancer. • The breakthrough case was presented at the American Association for Cancer Research Annual Meeting in Chicago, with researchers now working to replicate these results in more patients with treatment-resistant colorectal cancer.

CRISPR Gene-Editing Therapy Shows Promise in First Human Trial for Advanced GI Cancers

18 days ago

• University of Minnesota researchers conducted the first human trial using CRISPR/Cas9 to modify immune cells, demonstrating safety and potential effectiveness against advanced gastrointestinal cancers. • The innovative approach deactivated the CISH gene in tumor-infiltrating lymphocytes, enhancing their ability to recognize and attack cancer cells with one patient experiencing complete tumor regression lasting over two years. • Unlike conventional cancer treatments requiring ongoing doses, this gene-editing technique provides permanent modification to T cells in a single step, potentially offering new hope for patients with previously incurable stage IV colorectal cancer.

FDA-Approved Lenire Device Shows 91.5% Success Rate in Real-World Tinnitus Treatment Study

24 days ago

• A new study published in Nature Communications Medicine reveals that 91.5% of tinnitus patients experienced clinically meaningful improvement after 12 weeks of treatment with the Lenire bimodal neuromodulation device. • The retrospective analysis of 220 patients represents one of the largest real-world studies of tinnitus treatment, confirming results from previous clinical trials that led to FDA approval in March 2023. • Lenire works by simultaneously delivering audio tones through headphones and mild electrical pulses to the tongue, offering a promising treatment option for the estimated 25 million Americans suffering from tinnitus.

FDA Requests Additional Trial for Novavax COVID-19 Vaccine Before Full Approval

a month ago

• 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.

Trump Administration Cancels $800 Million in LGBTQ Health Research Funding

a month ago

• The Trump administration has terminated 323 NIH grants worth $806 million focused on LGBTQ health research, affecting studies on HIV prevention, cancer, and mental health. • Critical research projects halted include HIV prevention trials, studies on preventing suicide in transgender teens, and research on sexually transmitted diseases, leaving many labs forced to lay off staff. • Scientists and public health experts warn these cuts will harm healthcare for sexual and gender minorities, who make up nearly 10% of American adults, and could reverse progress in disease prevention.

Minnesota-Developed SynerFuse Implant for Chronic Back Pain Advances Toward FDA Approval

a month ago

• SynerFuse, developed by University of Minnesota and M Health Fairview specialists, combines traditional spine surgery with nerve stimulators to provide opioid-free relief for chronic back pain. • The innovative implant allows patients to control nerve stimulation via smartphone, with early trial participants reporting significant pain reduction and improved quality of life without opioid dependence. • After completing 15 successful surgeries with one year of post-operation data, SynerFuse is advancing to a national randomized clinical trial across 15-20 sites, with developers seeking fast-track FDA approval.

First Hormone-Free Male Birth Control Pill Shows 99% Efficacy in Preclinical Studies

2 months ago

• YourChoice Therapeutics' novel contraceptive YCT-529, targeting retinoic acid receptor-alpha, demonstrated 99% effectiveness in mice and successfully reduced sperm counts in non-human primates. • The drug achieved full fertility reversal within 6 weeks in mice and 10-15 weeks in primates after discontinuation, with no observed side effects in either species. • Currently in Phase 1b/2a clinical trials, YCT-529 represents a significant advancement in male contraception options, which have historically been limited to condoms and vasectomy.

GT Biopharma Secures $0.7M Through Strategic Warrant Exercise for TriKE® Platform Development

3 months ago

• GT Biopharma has secured approximately $0.7 million in gross proceeds through the exercise of existing warrants at $4.35 per share, strengthening its financial position for TriKE® platform development. • The company will issue new unregistered warrants for 604,138 shares with an exercise price of $2.02 per share, providing additional future funding potential for immuno-oncology therapeutic development. • The transaction, expected to close by February 27, 2025, includes H.C. Wainwright & Co. as exclusive placement agent, with proceeds directed towards working capital and corporate initiatives.

Ataciguat Shows Promise in Slowing Aortic Valve Stenosis Progression, Mayo Clinic Study Reveals

3 months ago

• A groundbreaking study at Mayo Clinic demonstrates that ataciguat reduced aortic valve calcification progression by 69.8% over six months compared to placebo in patients with moderate aortic valve stenosis. • The novel drug shows potential to delay or prevent valve replacement surgery, particularly benefiting younger patients who might otherwise need multiple surgeries throughout their lifetime. • Clinical trials confirm ataciguat's safety profile with minimal side effects, maintaining bone health while effectively slowing valve calcification, paving the way for upcoming Phase 3 trials.

Jord BioScience Raises Series B Funding to Advance Agricultural Microbial Solutions

3 months ago

• Jord BioScience has secured Series B funding led by Silver Blue LLC, with participation from Discovery Capital Funding and other investors to accelerate microbial technology development for agriculture. • The company leverages a proprietary bank of over 6,500 microbial isolates collected globally to enhance crop performance in major agricultural products including corn, soybeans, canola, and wheat. • Under CEO Dr. Keri Carstens' leadership, Jord BioScience aims to scale operations and fast-track biological solutions development through partnerships with leading agribusinesses.

Children's Minnesota Launches Groundbreaking Vaccine Trial for Deadly Pediatric Brain Cancer

3 months ago

• Children's Minnesota has initiated a clinical trial testing a novel vaccine-peptide combination therapy for diffuse midline glioma (DMG), a lethal pediatric brain cancer with less than 1% cure rate. • The experimental treatment, developed by University of Minnesota researchers and licensed to OX2 Therapeutics, combines an immune-stimulating vaccine with a synthetic peptide designed to overcome cancer's immune-suppressing effects. • The trial, recruiting patients aged 2-25 with DMG or high-grade gliomas, builds on promising early results where some adult patients achieved 26-month survival compared to typical 9-11 month prognosis.

GT Biopharma Doses First Patient in Phase 1 Trial of GTB-3650 for Hematologic Malignancies

4 months ago

• GT Biopharma initiated a Phase 1 trial of GTB-3650, a second-generation TriKE, for relapsed or refractory CD33-expressing hematologic malignancies. • The trial will evaluate the safety, pharmacokinetics, pharmacodynamics, and clinical activity of GTB-3650 in up to 14 patients. • GTB-3650 utilizes camelid nanobody technology to potentially improve potency and enhance binding affinity to target cells. • Initial data from the Phase 1 trial is anticipated in 2025, marking a significant milestone for GT Biopharma's clinical development.

Fecal Microbiota Transplantation Shows Promise in Preventing Graft-versus-Host Disease After Stem Cell Transplants

4 months ago

• A phase 2 clinical trial demonstrates that oral fecal microbiota transplantation (FMT) is a safe and feasible approach to prevent graft-versus-host disease (GVHD) in stem cell transplant recipients. • The study identifies donor-specific effects, with FMT from Donor 3 showing the highest microbiota engraftment rate and a favorable safety profile. • Lower pre-FMT microbiota diversity in patients was associated with better donor microbiota engraftment, suggesting a more receptive gut environment for transplanted microbes. • The ongoing randomized phase of the trial will further investigate FMT's impact on reducing severe GVHD, improving quality of life, and increasing survival rates.

FDA Approves SpringWorks' Gomekli (Mirdametinib) for Neurofibromatosis Type 1

6 months ago

• The FDA has approved Gomekli (mirdametinib) for both adult and pediatric patients with Neurofibromatosis Type 1 (NF1) who have symptomatic plexiform neurofibromas (PN) not amenable to complete resection. • Approval was based on Phase 2b ReNeu trial data, demonstrating a 41% ORR in adults and 52% in children, alongside deep and durable tumor volume reductions and manageable safety profiles. • Gomekli is the first and only medicine approved for both adults and children with NF1-PN, addressing a significant unmet need, particularly for adult patients who previously lacked approved treatments. • SpringWorks received a rare pediatric disease priority review voucher from the FDA, potentially expediting the review of future drug candidates.

FDA Approves Kebilidi, First Brain-Delivered Gene Therapy for AADC Deficiency

6 months ago

• The FDA has approved Kebilidi (eladocagene exuparvovec-tneq), a gene therapy for AADC deficiency, marking the first brain-delivered gene therapy approved in the U.S. • Kebilidi is administered directly into the brain and replaces the mutated DDC gene with a functional version, restoring dopamine and serotonin production in both adults and children. • Clinical trials demonstrated significant improvements in gross motor function in pediatric patients, with some achieving head control and the ability to sit. • Approved under the accelerated approval pathway, Kebilidi's long-term effects have shown durability for up to 10 years, offering hope for this rare genetic disorder.

Alzheimer's Drug Trials Under Scrutiny for Genetic Risk Disclosure

7 months ago

• Eisai and Eli Lilly face criticism for not fully disclosing genetic risks to participants in Leqembi and Kisunla Alzheimer's drug trials. • Volunteers with the ApoE4 gene, particularly homozygotes, faced a higher risk of brain bleeding (ARIA) during the anti-amyloid treatments. • Experts debate whether withholding genetic information undermined informed consent, balancing patient safety with scientific rigor. • Current practice now favors upfront disclosure of ApoE4 status in Alzheimer's clinical trials to allow informed decision-making.