By Nuala Moran Staff Writer LONDON – The gene therapy Glybera has been rejected for the third time, despite the fact that the European Medicines Agency's (EMA) Committee for Medicinal Products for Human use (CHMP) last week voted 16 to 15 in favor of approving the treatment for ultrarare inherited disorder lipoprotein lipase deficiency (LPL). Unfortunately, for a product to get the nod, it needs an absolute majority – or 17 votes – from all the members of the 32-strong committee, not just

By Mari Serebrov Washington Editor The FDA got an earful Monday at a hearing on how to best modernize clinical trials and good clinical practice (GCP), but most of the ideas have been on the table for many years, one stakeholder pointed out. "We need to go big if we are going to modernize clinical trials," Doug Peddicord, executive director of the Association of Clinical Research Organizations, told the FDA. And action, not more research, is needed, he added. Referencing earlier calls for

By Anette Breindl Science Editor Using yeast as a model system, scientists have gained new insights into the effects of selective serotonin reuptake inhibitors (SSRIs) on cells. SSRIs are a major class of antidepressants including Prozac (fluoxetine, Eli Lilly and Co.) and Celexa (citalopram, Forest Laboratories Inc.). They were developed as a way to increase serotonin signaling, which is reduced in depression. They do so, at least in part, by preventing serotonin that is released by neurons

By Mari Serebrov Washington Editor Five Prime Therapeutics Inc. added another notch to its growing list of biologics-development relationships with big pharma players, signing on with GlaxoSmithKline plc for their second partnership. Under the new collaboration, GSK will have access to the biotech's drug discovery platforms, as well as its collection of functional human secreted proteins and transmembrane receptor proteins, in up to six programs to identify first-in-class agents – be they

By Nuala Moran Staff Writer LONDON – Algeta ASA decided to go for the higher-risk, but hopefully more profitable route in U.S. commercialization of Alpharadin, exercising an option last week on a 50-50 co-promotion agreement for which it is liable for half the costs. Under a September 2009 global licensing deal with partner Bayer AG, Algeta had the right either to receive a royalty on sales or to take ascending co-promotion rights – from 25 percent up to a maximum of half the market – for

By Nuala Moran Staff Writer LONDON – Newron Pharmaceuticals SpA has found a new partner for its Phase III drug safinamide, securing a €20 million (US$26.1 million) opening payment, with milestones and royalties to come, from the Italian pharma company Zambon SpA. Safinamide became available for partnering after Merck KgaA said in October 2011 it was handing back rights to the alpha-aminoamide, an add-on therapy to dopamine agonists or levodopa in treating Parkinson's disease. That sparked the

By Mari Serebrov Washington Editor WASHINGTON – The Supreme Court is being asked to curb a growing cottage industry of shareholder lawsuits by raising the bar for class actions alleging companies have frauded the market. Courts have become all too willing to "certify inappropriate and unwieldy classes in cases alleging that a corporation provided misleading information to investors" when there's no evidence the market was misled, the Washington Legal Foundation (WLF) said in a brief filed last

By Anette Breindl Science Editor In findings that run directly counter to prevailing wisdom, researchers have found that proinflammatory responses may be protective against the progression of age-related macular degeneration (AMD), which is the leading cause of blindness in the developed world. The studies, which identify the proinflammatory cytokine interleukin-18 (IL-18) as an upstream regulator of vascular endothelial growth factor (VEGF) may lead to new ways to treat AMD and prevent its

By Nuala Moran Staff Writer LONDON – Cancer Research UK (CRUK) is joining forces with the European Investment Fund (EIF) to launch a £50 million (US$80 million) fund to invest in early stage assets and "rescue an ecosystem under threat." The Pioneer Fund, which will take cancer drugs from discovery through to Phase II before they are out-licensed, is the fifth significant fund to be announced in Europe in the past month. In common with the Pioneer Fund, the £200 million Project Sigma set up

By Mari Serebrov Washington Editor WASHINGTON – With more riding on the reauthorization of PDUFA than ever before, a Senate committee is pushing through a package of user fees with a warning against bogging it down with costly and controversial measures. Patients, jobs and innovation will be on the line if Congress doesn't reauthorize PDUFA before its Sept. 30 expiration date, the Senate Health, Education, Labor and Pensions (HELP) Committee was reminded last week in its final hearing before

By Anette Breindl Science Editor Rapamycin is a decades-old drug, but it is still learning – or rather, still teaching scientists – new tricks. This week, researchers reported new insights into how the drug affects aging and metabolism – and most saliently, that its effects on the two can be separated. After rapamycin was discovered, its first career, as an antifungal, was short-lived because it had immunosuppressive effects. Those same immunosuppressive effects, though, did lead to its

By Trista Morrison Editor In the first two parts of BioWorld Insight's Innovation Analysis, we explored how although the creation and funding of new biotechs is not necessarily synonymous with the creation and funding of innovation, there are signs that venture capitalists are more willing to invest in innovative, early stage biotechs than they were five or six years ago. That interest is critical, because a Harvard Business School analysis showed innovation typically gets funded during

By Mari Serebrov Washington Editor WASHINGTON – Leaving a lot of personalized medicine claims in patent limbo, the Supreme Court Monday told an appellate court to reconsider a challenge to Myriad Genetics Inc.'s claims on the BRCA1 and BRCA2 genes, used in diagnostics for breast and ovarian cancer. In remanding The Association for Molecular Pathology v. Myriad Genetics Inc., the Supreme Court instructed the Court of Appeals for the Federal Circuit to reconsider the case in light of its ruling

By Anette Breindl Science Writer Researchers have reported that knockout mice lacking either two specific types of major histocompatibility complex molecules or their receptor fared better after a stroke than their wild-type cousins. In fact, lacking the MHC class I molecules Kb and Db, or their receptor PirB, improved "not only the amount of damage early on, but also what happens during recovery," co-corresponding author Rona Giffard told BioWorld Today. The findings were published in the

By Nuala Moran Staff Writer EDINBURGH, Scotland – A 10-year plan to create an integrated translational medicine hub in the heart of Edinburgh is beginning to bear fruit, with the first spinouts starting to emerge from the research base and take up residence in a new bioincubator, which opens next week. Alongside the incubator, the Edinburgh BioQuarter is now home to 650 researchers in the University of Edinburgh Medical Research Institute; a 1,000-bed teaching hospital; the Scottish Center

By Mari Serebrov Washington Editor WASHINGTON – A new capital formation environment for small biotechs on their way to going public could be just days away, if the House agrees to the Senate's investor protections for crowd funding. The Senate voted 73 to 26 Thursday for the Jumpstart Our Business Startups (JOBS) Act. The House had already passed H.R. 3606, which is intended to benefit emerging growth companies by providing a five-year on-ramp to the public market, raising the SEC's

By Catherine Shaffer Staff Writer An abrupt termination by Merck & Co. Inc. of development of oral vernakalant caught Cardiome Pharma Corp., of Vancouver, British Columbia, flatfooted as it prepared to release a financial update Monday. The company scrambled to slash its cash burn guidance in half for 2012, cutting that number from about $29 million to $15 million annually going forward. "As you can appreciate, we're stunned and very disappointed," said Doug G. Janzen, Cardiome's president and

By Cormac Sheridan Staff Writer The Wellcome Trust is setting up a new for-profit investment firm that will have £200 million (US$317 million) on hand to pursue opportunities in health care and in life sciences in the UK and other parts of Europe. Codenamed "Project Sigma" for now, the new entity has named Martin Murphy, former partner at venture capital firm MVM Life Science Partners LLP, as its CEO, and Nigel Keen, an entrepreneur and investor, as its chairman. The initiative is motivated by

By Nuala Moran Staff Writer LONDON – ThromboGenics NV is getting €75 million (US$98.4 million) up front as part of a €375 million-plus-royalties deal signed last week with leading eye care specialist Alcon Inc. for the ex-U.S. rights to ocriplasmin, providing the cash ThromboGenics needs to go it alone in commercializing the vitreous macular adhesion (VMA) treatment in the U.S. Ocriplasmin, a recombinant form of human plasmin, has been filed with the European Medicines Agency, and ThromboGenics