Clinical Trial News

Presage Biosciences dosed the first cancer patient with Pure Biologics' ROR1-targeting antibody PBA-0405 in a Phase 0 study, utilizing their CIVO platform for direct tumor microenvironment analysis.

Walgreens awarded $25M for COVID-19 vaccine study; Oracle enhances AI solutions for life sciences; Merck collaborates with N-Power Medicine for expanded oncology research; AACR outlines clinical trial design considerations; Advarra centralizes patient recruitment; Heriot-Watt and ABHI collaborate for HealthTech excellence; ObjectiveHealth expands partnership with QualDerm; MD Anderson and Rice University form Cancer Bioengineering Collaborative; Univo IRB launches OneVerse platform.

Mirum's Livmarli approved for PFIC in patients 12 months and older, addressing cholestatic pruritus in rare liver disease.

Final Phase 2 study results of TPN-101 show excellent safety and potential disease-modifying effects in C9orf72-related ALS/FTD, with reduced neuroinflammation and neurodegeneration via LINE-1 inhibition. TPN-101 demonstrated significant benefits in respiratory function and ALSFRS-R scores, suggesting a global clinical benefit with longer treatment. Transposon plans to advance TPN-101 into a Phase 3 study for C9-ALS and other neurodegenerative disorders.

Sage Therapeutics and Biogen announce topline results from Phase 2 KINETIC 2 study of SAGE-324 (BIIB124) for essential tremor, showing no statistically significant dose-response relationship on primary endpoint. No further clinical development planned for SAGE-324 in essential tremor.

Pfizer announces positive Phase 3 results for hemophilia A gene therapy candidate giroctocogene fitelparvovec, showing significant reduction in annualized bleeding rate post-infusion and generally well-tolerated profile.

Vivacelle Bio starts Phase 3 trial for VBI-S, its lead asset to treat hypovolemic septic shock, using patented phospholipid nanoparticle technology to elevate blood pressure. The trial, approved by the FDA, follows a successful Phase 2a study showing 100% efficacy in increasing mean arterial blood pressure by at least 10 mmHg.

ConSynance Therapeutics receives FDA Rare Pediatric Disease Designation for CSTI-500 in Prader-Willi Syndrome, aiming to address hyperphagia and severe temper outbursts through a Triple Monoamine Reuptake Inhibitor.

FDA approves BioMarin's Brineura for children under 3 with CLN2 disease, expanding its use to all ages, potentially delaying disease onset and slowing motor function decline.

FDA approves Femlyv, the first orally disintegrating birth control pill, on July 22, 2024, providing an alternative for individuals with difficulty swallowing medication.