Chugai Pharmaceutical Co. Ltd., of Tokyo, said data from hemophilia A patients in a phase I study of bispecific antibody emicizumab, published in The New England Journal of Medicine, demonstrated a clinically acceptable safety profile and a potential benefit for preventing bleeding in patients, both with and without factor VIII inhibitors. Emicizumab is a once-weekly subcutaneous injection and has be designated a breakthrough therapy by the FDA. Read More

BioWorld’s offices will be closed Monday, May 30, in observance of the Memorial Day holiday in the U.S. No issues will be published that day. Read More

Highland Therapeutics Inc., of Toronto, said its wholly owned subsidiary, Ironshore Pharmaceuticals and Development Inc., inked an agreement with Quotient Clinical, of Nottingham, U.K., to conduct scintigraphy imaging of its inflammatory bowel disease (IBD) candidate, HLD-400, using Quotient’s Rapidfact (rapid formulation development and clinical testing) program. Ironshore will provide Quotient with technical support and access to Ironshore’s drug delivery platform, Delexis, in return for Quotient’s GMP manufacturing facility and clinical expertise in scintigraphy. Read More

Shares of Reata Pharmaceuticals Inc. (NASDAQ:RETA) climbed 18.8 percent to $13.07 from an IPO price of $11 on Thursday, in which the sale of 5.5 million shares raised net proceeds of $52.5 million. Funds from the offering, which had initially been slated to raise as much as $64 million, will be used to advance Reata’s lead antioxidant inflammation modulator (AIM), bardoxolone methyl, through a phase III trial and additional phase II programs. Another AIM, omaveloxolone, will also be moved through phase II. Read More

Researchers have identified the signals that breast cancer cells use to get into the bone marrow, as well as the separate signals that keep them there. Disrupting those signals may offer a way to prevent late relapses of hormone-driven breast cancers. Read More

DUBLIN – Xenikos BV is on track to complete recruitment in a phase I/II trial of T-Guard, a combination of two antibody-drug conjugates in development for treating steroid refractory graft-vs.-host disease (GvHD), in the coming months. The company expects to report survival data early next year, but, based on what it has seen so far, it is already contemplating a potentially pivotal phase II trial as its next step, which could lead to an application for conditional marketing authorization. Read More

Positive phase IIb data with MIN-101 in schizophrenia paves the way to pivotal testing and gives patients hope for their negative symptoms, Minerva Neurosciences Inc. spokesman William Boni told BioWorld Today. “I don’t want to use any words like ‘breakthrough,’” he said, because there needs to be more testing done, “but there is no effective treatment for negative symptoms at this point.” Read More

Shares of Ionis Pharmaceuticals Inc. (NASDAQ:IONS) were pummeled Thursday, sinking to a three-year low of $21.09, after the company clarified that “less than a handful of patients” enrolled in its phase III NEURO-TTR study evaluating IONIS-TTRRx in transthyretin (TTR) familial amyloid polyneuropathy (FAP) experienced a serious decline in platelets. The small number of events, in seriously ill patients often taking other medications, might have been a minor blip on the radar screen except that partner Glaxosmithkline plc (GSK), which holds an option to license IONIS-TTRRx exclusively, decided to postpone a phase III cardiovascular study, CARDIO-TTR, that was planned to evaluate IONIS-TTRRx in patients with TTR amyloid cardiomyopathy. Read More