Clinical Trial News

FDA expands approval of Palforzia [Peanut (Arachis hypogaea) Allergen Powder-dnfp] to include individuals ages 1 through 3 years with peanut allergy to mitigate allergic reactions, including anaphylaxis, from accidental exposure. Palforzia, under a REMS, was initially approved in 2020 for ages 4 through 17 years. Users must still avoid peanuts in their diet.

FDA approves Leqselvi (deuruxolitinib), an oral JAK inhibitor for severe alopecia areata treatment.

Xspray Pharma received a Complete Response Letter from the FDA for Dasynoc, a CML and ALL treatment, requesting additional info on labeling and manufacturing inspection. No new clinical studies are required, and Xspray aims to address FDA concerns promptly.

NIH researchers found AKT2 alterations cause lysosome dysfunction, leading to drusen formation in dry age-related macular degeneration (AMD), suggesting AKT2 as a therapeutic target.

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.