By Mari Serebrov Washington Editor If Congress wants to curtail drug shortages, it needs to put some teeth in a PDUFA V provision that requires companies to notify the FDA six months in advance of events that could lead to a shortage, according to a panel at the annual meeting of the American Society of Clinical Oncology (ASCO). While both the House and Senate passed versions of the user fee package that call for early notification, neither grants the FDA any tools to enforce it. Richard

By Mari Serebrov Washington Editor WASHINGTON – While the differences between the House and Senate PDUFA bills are seemingly minor, they could add up to hundreds of millions of dollars. Because of provisions in the House FDA Reform Act, H.R. 5651, that would delay drug competition in some areas, the Congressional Budget Office (CBO) estimated the bill could increase federal spending by $244 million over the next decade. In comparison, the CBO projected that the bill passed by the Senate last

By Cormac Sheridan Staff Writer NeuroSearch A/S's task of securing backing for an additional Phase III trial of its Huntington's disease drug, Huntexil (pridopidine), has gotten a bit more difficult, following the release of data from a multiple-ascending-dose trial indicating that the compound causes QT interval prolongation in healthy volunteers. Huntexil – the first in a new drug class that NeuroSearch calls dopaminergic stabilizers – had not been administered previously to Huntington's

By Cormac Sheridan Staff Writer Shares in Active Biotech AB climbed more than 12 percent Monday on news that the company earned a €10 million (US$12.7 million) milestone payment from its partner, Ipsen SA, after reaching the halfway point in recruitment into a 1,200-patient, Phase III registration trial of tasquinimod in metastatic castrate-resistant prostate cancer. Lund, Sweden-based Active Biotech said it expects top-line data from the study by the end of 2014 and a regulatory filing, based

By Brian Orelli BioWorld Insight Contributing Writer The Ewing Marion Kauffman Foundation issued a report this month blaming the lackluster returns from venture capital on a lack of oversight from limited partners (LPs) that invest in the funds. Based on a strategic look at the Kauffman Foundation's experience investing its endowment in venture capital funds, the authors concluded that LPs are to blame because they haven't demanded higher returns for the riskier investment. "Institutional

By Mari Serebrov Washington Editor WASHINGTON – Drugmakers are keeping their fingers crossed as two separate bills to reauthorize PDUFA roll through Congress well ahead of its Sept. 30 expiration. S. 2516 may advance to the Senate floor as early as this week, and the House is expected to bring H.R. 5651 to the floor late this month. However, several proposed amendments, ranging from a curb on pay-for-delay settlements to importation of drugs from Canada, could stall the bills and hamper

By Cormac Sheridan Staff Writer PARIS – Despite managing to put together a star-studded lineup for a roundtable on innate immunity, which included two current Nobel prizewinners, Bristol-Myers Squibb Co. (BMS) and Innate Pharma SA found their best-laid plans were upstaged Tuesday by the French presidential inauguration at the Elysée Palace. One of the speakers, 2011 Nobel laureate Jules Hoffmann, of the University of Strasbourg, France, was a little late arriving at the roundtable due to his

By Mari Serebrov Washington Editor If patient advocacy groups have their way, it could be a lot harder and take longer for biosimilars to come to market. While the groups want the price breaks the follow-on drugs might offer, they are concerned about safety. Seemingly the most insignificant changes in manufacturing, packaging, handling and storing of biologics can have unintended consequences that can be life threatening, Dolph Chianchiano, of the National Kidney Foundation Inc., testified

By Anette Breindl Science Editor Scientists have reported they were able to improve memory in patients with amnestic mild cognitive impairment – a neurological condition somewhere between normal age-related memory loss and outright dementia that often progresses to Alzheimer's disease – by treating them with low doses of the anti-epileptic drug Keppra (levetiracetam, from Brussels, Belgium-based UCB SA). The drug appears to work by reducing activity in the hippocampus, a brain structure that is

By Trista Morrison BioWorld Insight Editor Biotech firms have long borrowed ideas from the high tech world. The venture-backed start-up business model on which the entire biotech industry is based came from high tech, and it has served both industries well despite their very different timelines and cost structures. Even so, current capital constraints have put "unprecedented strain" on biotech business models, according to Ernst & Young's Lead Analyst Gautam Jaggi, requiring a rethink of

By Trista Morrison Editor Biotech has become increasingly virtual over the last few years, and several recent studies and surveys indicate the trend is likely to continue, particularly when it comes to outsourcing of research and development. Yet some biotechs are grumbling about the high fixed costs inherent in the current contract research organization (CRO) model, while others complain about the quality of outsourced work. When Spectrum Pharmaceuticals Inc. acquired Allos Therapeutics Inc

By Anette Breindl Science Editor The Sonic Hedgehog pathway is a critical developmental pathway that is reactivated in a number of cancers. Results with targeting the pathway to treat cancers have been mixed, as drug development is wont to be. But the successes have been enough to keep trying. Roche AG unit Genentech Inc.'s and Curis Inc.'s hedgehog inhibitor Erivedge (vismodegib) was approved in January for advanced basal cell carcinoma, after previously failing in colorectal and ovarian cancer

By Mari Serebrov Washington Editor WASHINGTON – When it comes to discovering new uses for failed drugs, serendipity is about to be replaced by translational science in what National Institutes of Health (NIH) Director Francis Collins is calling a "win-win-win" pilot program for researchers, industry and patients. Under the crowd-sourcing program, NIH's National Center for Advancing Translational Sciences (NCATS) will serve as the matchmaker – hooking up failed compounds with researchers who

By Catherine Shaffer Staff Writer After dropping its price drastically from a target range of $12 to $14, Supernus Pharmaceuticals Inc., of Rockville, Md., priced an initial public offering of 10 million shares of common stock at $5 per share. But the stock (NASDAQ:SUPN) opened strong and by the end of the day had gained 37 cents, or 7.4 percent, to close at $5.37. The company, which first filed its S-1 in late 2010, will use net proceeds of $47.8 million – or $55.5 million if underwriters

By Cormac Sheridan Staff Writer Shares in Actelion Ltd. rose 13 percent Monday on news that macitentan, its dual endothelin receptor antagonist (ERA), hit the primary endpoint in a pivotal Phase III trial in pulmonary arterial hypertension (PAH), raising the prospect that the company will be able to revitalize its aging PAH franchise with a safer, more potent drug. Both the low (3 mg once daily) and the high doses (10 mg once daily) of macitentan lowered the risk of a morbidity/mortality event

By Trista Morrison Editor When Nabi Biopharmaceuticals Inc. revealed its plans to serve as a public shell for a reverse merger with Aussie biotech Biota Holdings Ltd., Raafat Fahim knew he'd face some opposition from shareholders. The biotech industry has a few trademark reverse merger success stories – like Cougar Biotechnology Inc., which saw its stock jump 150 percent in three years and secured a billion-dollar acquisition by Johnson & Johnson, and OncoGenex Pharmaceuticals Inc., which has

By Trista Morrison Editor This year, California became the seventh state to allow benefit corporations and the first state to allow flexible purpose corporations. Both are new business structures with a non-profit flare; they let companies pursue a social mission and make decisions based not only on profit maximization for shareholders, but on non-financial factors such as the interests of their employees, community and environment. Since all biotech companies are, by definition, pursuing the

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