Clinical Trial News

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.

Xeomin, first approved in 2011 for frown lines, now also treats forehead and crow's feet lines. It's the first FDA-approved neurotoxin for simultaneous treatment of upper facial lines, containing only essential ingredients. The new indication is based on clinical studies showing its efficacy and safety, with high patient satisfaction and continued efficacy over multiple treatment cycles.

A single HIV viral load test may yield false positives for those on long-acting injectable cabotegravir (CAB-LA) PrEP, according to a multi-country study. A second test with a new blood sample reliably distinguishes true from false positives. Presented at the 2024 International AIDS Conference, the study highlights the need for optimizing HIV testing methods for CAB-LA PrEP users.