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In Brief This Week: A2 Biotherapeutics, Ellipses Pharma, Quanterix, Verismo Therapeutics

NEW YORK – The US Food and Drug Administration this week granted orphan drug designation to A2 Biotherapeutics' autologous cell therapy A2B530 for the treatment of germline heterozygous HLA-A*02-positive colorectal cancer that expresses carcinoembryonic antigen (CEA) and has lost HLA-A*02 expression. A2 is studying A2B530 in a Phase I/II trial in colorectal, pancreatic, non-small cell lung, and other solid cancers that express CEA and have lost HLA-A*02 expression. The FDA grants orphan designation to drugs for rare diseases, which provides sponsors tax credits for clinical trials, exemption from user fees, and seven years of market exclusivity for the drug after regulatory approval. 

The US Food and Drug Administration this week granted fast track designation to Ellipses Pharma's RET inhibitor EP0031/A400 for the treatment of RET-fusion positive non-small cell lung cancer. Ellipses is currently studying EP0031/A400 in a Phase I/II trial in patients with advanced RET-altered malignancies, including patients who progressed on first-generation selective RET inhibitors. With fast track designation, a sponsor can meet with the FDA more frequently to get advice on developing fast-track designated agents, submit data on a rolling basis, and apply for accelerated approval or priority review. 

Quanterix said this week that its Simoa Phospho-Tau 217 blood test has received breakthrough device designation from the US Food and Drug Administration as an aid in diagnostic evaluation of Alzheimer's disease. The test is a semi-quantitative in vitro diagnostic immunoassay for the measurement of p-tau 217 concentration in plasma using Quanterix's HD-X immunoassay system. Proposed indications include patients presenting with cognitive impairment who are being evaluated for AD risk to aid in diagnostic evaluation. 

Verismo Therapeutics said this week that it has started recruiting patients to its STAR-101 clinical trial at its second clinical trial site, the University of Texas MD Anderson Cancer Center. Previously, the University of Pennsylvania spinout had only been recruiting at UPenn, near its headquarters. The Phase I trial is designed to evaluate Verismo's autologous cell therapy, SynKIR-110, as treatment for mesothelin-overexpressing ovarian cancer, bile duct cancer, and mesothelioma. 

In Brief This Week is a selection of news items that may be of interest to our readers but had not previously appeared on Precision Medicine Online.