Clinical Trial News

Biogen's nusinersen (Spinraza) showed efficacy at a higher dose than the FDA-approved regimen in the DEVOTE trial, with significant improvements on the CHOP-INTEND scale compared to a sham treatment. The higher dose was generally well-tolerated, though more serious adverse events were observed. Biogen plans to share detailed results with the SMA community and health authorities.

Palisade Bio announces successful refinement of patient selection strategies for Ulcerative Colitis (UC) based on PDE4B biomarkers, identifying 70% of adult and 90% of pediatric moderate to severe cases. The company is advancing PALI-2108, an orally administered, colon-specific PDE4 inhibitor, with a Phase 1 clinical trial for UC set to begin before year-end.

Poland emerges as a leading hub for clinical research, the FDA approves the first nasal spray for severe allergic reactions, lenacapavir offers 100% protection against HIV, Novartis' Leqvio significantly reduces LDL-C, alarming trends in men's cancer incidence are projected to 2050, Duality Biotherapeutics initiates Hong Kong IPO to fund cancer drug trials, Omny Health launches a GLP-1 data resource, an adaptive brain pacemaker improves Parkinson’s symptoms, Alzheimer’s drug Lecanemab slows cognitive decline, and AI tool AIM-MASH enhances liver disease trials.

Innovent Biologics' PD-1/IL-2a-bias Bispecific Antibody Fusion Protein (IBI363) received Fast Track Designation from the FDA for treating melanoma. Phase 1/2 trials in China, the US, and Australia show promising efficacy, with 29.7% ORR and 73.0% DCR in melanoma patients previously treated with immunotherapy. IBI363 aims to address unmet needs for immunotherapy-resistant melanoma.

Safe Pro Group's SpotlightAI™, powered by AWS, was selected by UNDP for Phase III drone survey in Ukraine, analyzing Kharkiv Oblast to detect landmines and UXO using AI and ML models, significantly enhancing land release efficiency in agricultural areas.

The FDA granted Orphan Drug Designation to Skyline Therapeutics' SKG1108, a gene therapy for retinitis pigmentosa, aiming to restore visual function by generating new photo-sensing cells. The designation qualifies SKG1108 for incentives, accelerating its development and enhancing patient access.

Researchers at the University of Montana's Center for Translational Medicine developed a cancer treatment now in human trials, leveraging a partnership with Inimmune. The compound, initially an influenza vaccine, was repurposed to boost the immune system's natural anti-cancer response. This collaboration, supported by $33 million in grants, integrates UM's academic ideas with Inimmune's business expertise, fostering innovation and potentially new cancer treatments.

Ractigen Therapeutics' RAG-18, an investigational saRNA therapy, received FDA orphan drug designation for treating DMD and BMD by activating the UTRN gene to increase utrophin production. This marks the first saRNA therapy to receive both orphan drug and rare pediatric disease designations from the FDA. RAG-18 aims to provide clinical benefit by upregulating utrophin, structurally similar to dystrophin, via RNA activation. Ractigen's pipeline includes RAG-01, which is in a phase 1 trial for NMIBC, targeting a tumor suppressor gene.

Bird flu can infect humans, causing serious illness through contact with infected birds or contaminated environments. Symptoms range from mild respiratory issues to severe pneumonia. This study evaluates the safety and immunogenicity of Bird influenza A/H5 vaccines, made from eggs at different dose levels, with two shots given 21 days apart. Study care is provided at no cost, and health insurance is not required. Participants must be 18-64 years old or 65 and older, have a BMI under 35 kg/m2, and be generally healthy.

Arsenal Biosciences raised $325M in Series C to advance CAR T-cell therapies for solid tumors, with AB-1015 for ovarian cancer and AB-2100 for kidney cancer in early clinical development.