Citing potential confounding factors, Vanda Pharmaceuticals Inc. is hoping further analysis might explain the top-line miss for its phase III study testing tradipitant in gastroparesis, hinting that regulatory submissions could still be in the offing. Investors, unsurprisingly, were less optimistic, sending shares of the Washington-based company (NASDAQ:VNDA) down nearly 18% to close Feb. 4 at $12.03.
爱丁堡大学剥离Biocaptiva ltd .)taking its cell free DNA (cfDNA) capture device to clinical trials in 2022 following promising preclinical results. The company’s platform technology is designed to increase the quantity of cfDNA available for liquid biopsy testing. Current liquid biopsy practices obtain cfDNA via a venous blood draw but the concentration of tumor-derived ctDNA can be too low for comprehensive testing. Biocaptiva’ device emerged from research at the University of Edinburgh investigating liquid biopsy technologies.
The dosing by Vincerx Pharma Inc., in mid-December of last year, of the first patient in the phase I study with VIP-152 marked the second experiment begun by the company in 2021, and added oomph to the already-growing renewal of interest in CDK9 as a therapeutic target.
The ultra-rare skin disease X-linked hypohidrotic ectodermal dysplasia (XLHED) can be a heavy burden for children, affecting structures in the skin including sweat glands, respiratory glands, and hair as well as teeth. Though lacking a specific treatment to date, a pivotal clinical trial planned by the Geneva-based nonprofit Esperare Foundation and Pierre Fabre SA could now help bring one about.
The FDA has placed Logicbio Therapeutics Inc.’s phase I/II clinical trial of LB-001, an investigational AAV genome-editing therapy for treating pediatric patients with methylmalonic acidemia (MMA), on a clinical hold. So far, four patients have been dosed in the study and two have had serious adverse events related to the candidate, the company’s lead asset.
LONDON – The first human challenge study of SARS-CoV-2 infection has reported initial results, showing it is safe to infect healthy volunteers with the virus in controlled conditions, and paving the way for the model to be used to accelerate clinical trials of COVID-19 vaccines, antivirals and diagnostics.
Applaud Medical Inc. has secured breakthrough device designation from the FDA for its acoustic enhancer technology to be used in conjunction with ureteroscopy with laser lithotripsy (URS-LL) for the fragmentation of calcium-based urinary stones. The San Francisco-based company is currently enrolling subjects in an FDA-approved pivotal trial evaluating the technology’s safety and efficacy.
As COVID-19 infections in Australia continue to mount, a dozen or so Australian compounds are winding their way through the pipeline that could offer additional protection to the vaccines and antivirals that are already provisionally approved by Australia’s TGA.
With phase Ib data published inThe Lancet Microbeshowing Suzhou Abogen Biosciences Co. Ltd.’s ARCoV had acceptable safety and induced a strong immune response, partner Walvax Biotechnology Co. Ltd. is continuing to enroll participants in a large phase III test of the vaccine.
Sio Gene Therapies Inc. is retrenching the business as it terminated the company’s AXO-Lenti-PD license agreement with Oxford Biomedica plc for treating Parkinson’s disease. Also, Sio’s CEO, Pavan Cheruvu, is leaving the company. Sio said it is deprioritizing its Parkinson’s disease program due to several factors, including resource requirements and development timelines “to reach meaningful value inflection for the program and an increasingly challenging market and regulatory environment” for the indication.