In a step toward making pig organs safe for human transplant, scientists at Egenesis Bio Inc. have used multiplexed CRISPR editing to remove porcine endogenous retroviruses (PERVs) from pigs. The team's findings, which were published in the Aug. 11, 2017, issue of Science, have multiple implications.
Synergy Pharmaceuticals Inc., of New York, reported $2.3 million in U.S. sales of chronic idiopathic constipation treatment Trulance (plecanatide) during the second quarter, the first full quarter the drug was on the market. The company also recorded approximately $1.5 million in net deferred revenue for product shipped that wasn't prescribed to patients yet. Synergy lost $73.9 million, or 33 cents per share, and ended the second quarter with $82 million in cash and cash equivalents. Shares of Synergy (NASDAQ:SGYP) closed down 13 percent to $3.07 on Thursday.
Beigene Ltd., of Beijing, said it plans to sell $125 million of its American depositary shares (ADSs), which each represent 13 of its ordinary shares. Underwriters have a 30-day option to purchase up to an additional $18.75 million of ADSs. Morgan Stanley, Goldman Sachs & Co. LLC and Cowen are acting as joint book-running managers. Shares of Beigene (NASDAQ:BGNE) closed down 6 percent to $72.64 on Thursday.
Anthera Pharmaceuticals Inc., of Hayward, Calif., said the FDA granted orphan drug designation to blisibimod for the treatment of immunoglobulin A nephropathy (IgAN). Blisibimod targets B-cell activating factor, or BAFF, which has been shown to be elevated in a variety of B-cell-mediated autoimmune diseases, including IgAN, systemic lupus erythematosus and others, the company said. Anthera is currently analyzing the data from the randomized, double-blind, placebo-controlled, phase II BRIGHT-SC study of blisibimod in patients with IgAN, also known as Berger's disease.
Elucidating the role of genetics in understanding disease is a driving force for the diagnostic and biopharmaceutical industries. But, until now, much of the human genome has remained largely a mystery. Scientists have long focused on the portion that codes for proteins – but that represents only 2 percent of the human genome.
Does orphan drug designation for a specified compound bestow exclusivity on each unique formulation of the drug? That's one of the questions a U.S. district court will have to sort out in United Therapeutics Corp. v. the Department of Health and Human Services (HHS).
Galapagos NV's autotaxin inhibitor, GLPG-1690, halted worsening lung function in adults with idiopathic pulmonary fibrosis (IPF) during the 12 weeks of a small phase IIa trial, setting the stage for it to quickly move on to late-stage testing. Top-line results of the trial, called Flora, suggest '1690 might eventually challenge Roche Holding AG's Esbriet (pirfenidone) and Boehringer Ingelheim GmbH's Ofev (nintedanib).
Celgene Corp. "kicked the tires" in 2012 and took a test drive two years later, but won't be motoring off the lot with Sutro Biopharma Inc., although the companies did agree to a relationship that puts wheels on Sutro's other partnering efforts.
Apellis Pharmaceuticals Inc. extended its string of financings with a $60 million series E preferred stock round led by Sectoral Asset Management that included new investors Sofinnova, Vivo Capital, F-Prime Capital Partners, investment funds advised by Clough Capital Partners LP and Venbio Select. Existing investors Morningside Ventures, Cormorant Asset Management, Venbio Global Strategic Fund and Epidarex Capital also participated in the financing.