For years, the biopharma industry has justified U.S. prescription drug prices by pointing to the high cost of years-long R&D and the too-real risk of failure. But in the current paradigm, a hefty launch price, more often than not, seems to reflect the cost of acquisition and is couched in terms of "value." That point was driven home last week when Novartis AG announced that its newly approved gene therapy, Zolgensma (onasemnogene abeparvovec), would list at $2.1 million. Read More
DUBLIN – Shares in Newron Pharmaceuticals SpA dropped by as much as 20.5% Tuesday on news that preclinical toxicity findings would delay the start of pivotal trials of its candidate schizophrenia treatment evenamide by at least six months. Read More
New data on Biomarin Pharmaceutical Inc.'s investigational treatment for severe hemophilia A, valoctocogene roxaparvovec (valrox), sparked mild debate among analysts who expressed appreciation for the gene therapy's apparent durability but uncertainty regarding interim data from a phase III test of the medicine. But overall, it "at least met – and in some cases exceeded – the majority of investors' expectations," J.P. Morgan analyst Cory Kasimov said. Though the market rendered its own verdict, driving company shares (NASDAQ:BMRN) 5.1% lower to $84.50 by Tuesday's close, the program seemed poised to retain significant interest going forward. Read More
Eli Lilly and Co. acquired worldwide rights for CNTX-0290, a somatostatin receptor type 4 agonist as a non-opioid treatment for chronic pain, from Centrexion Therapeutics Corp. Read More
HONG KONG – To get new drugs to the market faster, the South Korean government has plans that could see approval periods for biopharmaceuticals cut drastically, as part of its ongoing efforts to improve regulations and prompt greater investing in the biotechnology and health care industries, increasingly seen as a key pillar of economic growth. Read More
DUBLIN – Oxford Biomedica plc is banking £53.5 million (US$67.7 million) from a strategic investment by Novo Holdings A/S, under which the latter firm will acquire a 10.1% stake in the gene therapy firm. Read More
Characterized by large numbers of benign but highly disfiguring tumors, neurofibromatosis 1 (NF-1) is anything but a subtle disease. Because they are glaringly conspicuous, research into NF-1 has largely focused on the tumors themselves. But in the May 20, 2019, issue of PLoS ONE, researchers have reported new insights into the disease that stem from looking not at the tumors themselves but at tumor-free skin from NF-1 patients. Read More
HONG KONG – Seeking to expand its presence in Europe, Shionogi & Co. Ltd. inked a contract with Italian firm Molteni Farmaceutici SpA to distribute Rizmoic (naldemedine), its treatment for opioid-induced constipation (OIC), in Italy and Poland, and plans to export two additional drugs, cefiderocol and lusutrombopag, to the European market. Read More
Prevail Therapeutics Inc., of New York, filed to raise up to $100 million in an IPO. The gene therapy company plans to list on Nasdaq under the symbol PRVL. Morgan Stanley, Bofa Merrill Lynch and Cowen serve as joint bookrunners. Read More
Teva Pharmaceuticals Co. Ltd., of Jerusalem, agreed to an $85 million settlement with Oklahoma two days ahead of a trial on the company's role in fueling the state's opioid epidemic. Read More
Biomunex Pharmaceuticals SA, of Paris, said it established a U.S. subsidiary, Biomunex Pharmaceuticals Inc., based in Cambridge, Mass. The company's plug-and-play Bixab platform is designed to generate next-generation bi- and multispecific antibodies. Read More