Orchard Therapeutics Ltd., of London, launched with a £21 million (US$30.5 million) series A round led by F-Prime Capital. The company also inked partnerships with University College London, Great Ormond Street Hospital for Children NHS Foundation Trust, the University of Manchester, the University of California, Los Angeles, and Boston Children's Hospital for the development of gene therapies for serious and life-threatening orphan diseases. Orchard's development programs focus on the potential of ex vivo autologous hematopoietic stem cell gene therapy to restore normal gene function in primary immune deficiencies, metabolic diseases and hematological disorders. The lead candidate, a lentiviral stem cell gene therapy, is in development for severe combined immunodeficiency caused by adenosine deaminase deficiency. Read More
Evoke Pharma Inc., of Solana Beach, Calif., said it expects to provide top-line results from its pivotal phase III trial of EVK-001 in women with symptoms associated with diabetic gastroparesis early in the third quarter. The company confirmed that 205 subjects have been randomized in the trial testing the nasal delivery formulation of metoclopramide. Read More
Santhera Pharmaceuticals AG, of Liestal, Switzerland, said it submitted briefing material and a meeting request to the FDA to discuss the filing of a new drug application for Raxone (idebenone) for the treatment of Duchenne muscular dystrophy (DMD) patients not taking concomitant glucocorticoids. Read More
Merrimack Pharmaceuticals Inc., of Cambridge, Mass., reported $10 million in net revenues from first quarter U.S. sales of its pancreatic cancer drug, Onivyde (irinotecan liposome injection), which was launched in October 2015. Read More
Impax Laboratories Inc., of Hayward, Calif., is suing New York-based Turing Pharmaceuticals AG for breach of contract involving sales of Daraprim (pyrimethamine). Read More
HYDERABAD, India – India is strengthening its "track and trace" system for drugs by making it mandatory to put online information about barcodes on the packing of drugs being exported. Read More
In an economy driven by global competition, counterfeits and theft of trade secrets remain major hazards for U.S. drug and device companies, handing them an uneven playing field in some parts of the world. Read More
HONG KONG – A recent study by scientists at the Department of Ophthalmology and Visual Sciences of the Faculty of Medicine at the Chinese University of Hong Kong (CUHK) has identified a potential new therapeutic target for age-related macular degeneration (AMD), which is the leading cause of central vision loss in the elderly. Read More
Eight years after Ionis Pharmaceuticals Inc. selected then-independent Genzyme Corp. as the partner for its phase III lipid-lowering drug, mipomersen, in a potential $1.9 billion deal, the Carlsbad, Calif.-based company dealt the commercial asset, Kynamro, to start-up Kastle Therapeutics LLC for peanuts. Read More
Ideaya Biosciences Inc. CEO Yujiro Hata told BioWorld Today that "you'd be hard pressed to find a more personalized-medicine approach to cancer" than synthetic lethality, and his firm hopes to build on proof of concept brought about by the approval of Astrazeneca plc's poly ADP-ribose polymerase (PARP) inhibitor Lynparza (olaparib) in BRCA-mutated advanced ovarian cancer. Read More
Biogen Inc. unveiled plans Tuesday to sharpen its focus on neurology by breaking its hemophilia business out into a new publicly traded company. The tax-free transaction is expected to be a boon for Biogen shareholders, but analysts were split on its broader implications. Read More