Travere Therapeutics Inc.’s mid-September deal with Vifor Pharma Group – a collaboration and licensing agreement for the commercialization of sparsentan in Europe, Australia and New Zealand – brought further, well-deserved attention to the dual endothelin angiotensin receptor antagonist, in the works for rare kidney disorders, specifically focal segmental glomerulosclerosis and IgA nephropathy.
It’s all systems “go” for Travere Therapeutics Inc. and its sparsentan after meetings with the FDA paved the way for filings and potential accelerated approvals in the rare kidney disease IgA nephropathy and the kidney scarring condition focal segmental glomerulosclerosis.
As the company unveiled 18-month results from the phase III study called Helios-A, Alnylam Pharmaceuticals Inc.’s president of R&D, Akshay Vaishnaw, said “new exploratory data with the cardiac endpoints are extremely encouraging.” The study is testing RNAi therapy vutrisiran in polyneuropathy associated with hereditary transthyretin-mediated (hATTR) amyloidosis, where Helios-A met all secondary endpoints measured at 18 months.
Optimism for the potential of a bifunctional fusion protein complex in development at HCW Biologics Inc. for the potential treatment of pancreatic cancer jolted company shares (NASDAQ:HCWB) skyward to $4.73 on Oct. 28, up 62%, though still far short of the company's $8 per share summer market debut. The company, founded and led by former Altor Bioscience Corp. CEO Hing Wong, gained FDA clearance to start a first-in-human phase Ib trial of the candidate, HCW-9218.
A phase III trial of Rafael Pharmaceuticals Inc.’s devimistat in pancreatic cancer has failed, sending shares in its publicly listed holding company through the floor. Shares in Rafael Holdings (NYSE:RFL) closed Oct. 28 down 73% to $8.09, for a loss of $22.08 after an independent data monitoring committee also recommended a separate phase III trial of devimistat in acute myeloid leukemia should be stopped due to lack of efficacy.
Diacarta Inc.’s diagnostic assay, Coloscape, could be the first to meet the Centers for Medicare and Medicaid Service (CMS)’s strict standards for Medicare coverage of a liquid biopsy for colorectal cancer screening if results seen in a Plos One study are borne out in larger trials and the test gets the nod from the FDA. That is a lot of ifs, but the study posted strong results: 100% specificity and 92.2% sensitivity for cancer and 95% specificity and 62.5% sensitivity for precancerous lesions.
To help better utilize real-world data in drug development, the EMA published its final guideline Oct. 26 on the use of registry-based studies to support regulatory decision-making.
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