Shares in Gentium SpA fell 20 percent last week on news that the lengthy approval process for defibrotide, a drug for the treatment and prevention of hepatic veno-occlusive disease (VOD) in patients undergoing hematopoietic stem cell transplant, had just become longer. Preliminary feedback, following an oral presentation to the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP), indicated the CHMP is likely to adopt a negative stance when it comes to a

DUBLIN, Ireland – Shares in Elan Corp. plc (NYSE:ELN) rose 5 percent to $11.14 Monday in response to an indicative $11 per-share takeover offer from Royalty Pharma. Although Dublin, Ireland-based Elan issued a critical response to the offer, which represents a 4 percent premium to its closing share price Friday, it was not an outright refusal. The investor response suggested there is an appetite in the market for an exit. The company is now in play. With the ink barely dry on its new post

BOSTON – Location, location, location: The idea that one gene equals one protein is long discredited and research into mechanisms including post-translational modification of proteins and the baroque complexities of epigenetic control is advancing understanding of how one gene can generate multiple proteins. Now an emerging area of science – looking at the spatial organization of the genome and how the physical location of a gene within the cell nucleus impacts on its expression and functioning

Following pressure from a group of dissident shareholders, Diamyd Medical AB agreed to return most of its cash to its investors, while retaining a small amount to continue two Phase II trials of its GAD vaccine for the treatment and prevention of Type I diabetes. "We were able to avoid liquidation of the company," Chairman Anders Essen-Möller, who holds 33.92 percent of the votes in the Stockholm, Sweden-based company, told BioWorld International. Rebel shareholders, representing about 30

Ablynx NV, of Ghent, Belgium, reported efficacy and safety data for its anti-IL-6R nanobody, ALX-0061, at the 24-week final analysis of the Phase II part of a Phase I/II study in patients with moderately to severely active rheumatoid arthritis on a stable background of methotrexate. The Phase II data showed ACR20, ACR50 and ACR70 scores of up to 100 percent, 75 percent and 63 percent, respectively, for patients who were consistently dosed at 3mg/kg Q4W over 24 weeks. At all doses tested, ALX

LONDON – 2013 will be a watershed year for biosimilars – biological therapies designed to be similar to existing biological therapeutic agents – delegates attending the Second Biosimilars Congregation in London heard Tuesday. While the European Medicines Agency (EMA) has not approved any new biosimilars since 2010, largely because of a lack of applications, it is now considering two license applications lodged in April and September of last year. Both are for infliximab, which is marketed as

The FDA accepted for review a new drug application for lixisenatide, the once-daily glucagon-like peptide-1 (GLP-1) receptor agonist Zealand Pharma A/S has developed in partnership with Sanofi SA, paving the way for a possible U.S. approval in Type II diabetes by early 2014. "It's a reasonable expectation that we'll hear back from the FDA within a year," David Solomon, CEO of Copenhagen, Denmark-based Zealand, told BioWorld International. "We're buoyed by the news. Everything is in place to

BOSTON – The rise in the number of new drug approvals granted by the FDA in the past three years looks encouraging – hinting as it does that the industry is putting the productivity woes of the previous decade behind it. However, volume alone is not enough to guarantee future viability: Half of the 39 approvals in 2012 were for orphan drugs with forecast peak sales of $400 million per annum. "You need peak sales of $600 million per year to cover the costs of development. Although approvals are

LONDON – One of the UK's oldest biotech companies looks to be on its way out after reporting the failure of the lead program in Parkinson's disease. Shares in Phytopharm plc fell 80 percent after the announcement Monday that the long-running trial of Cogane showed no difference between the active compound and placebo in the 400-patient Phase II trial. The Godmanchester, UK-based company has begun a strategic review, but Robin Davison, analyst at Edison Investment Research in London, said there

BOSTON – Recent advances in the tools and technology for drug discovery mean it now takes minutes and days to accomplish tasks that previously took months and weeks. One consequence of those developments has been the rise of target-based screening to become the overwhelmingly dominant approach to discovery – largely displacing the use of phenotypic screening. Rather than looking to see what effect compounds might induce in animal models, tissues and cells, the industry became fixated on looking

3SBio Inc., of Shenyang, China, said it inked an agreement and plan of merger with Decade Sunshine Ltd., an exempted company with limited liability incorporated under the laws of the Cayman Islands. Pursuant to the terms, each of the company's ordinary shares, including shares represented by American depositary shares (ADS), will be cancelled in exchange for the right to receive $2.20 in each per share, or $15.40 per ADS. The per-share consideration represents a premium of about 32.9 percent

Matthias Wymann, the Swiss scientist who discovered the first phosphoinositide 3-kinase (PI3K) inhibitor, is co-founder of a new cancer drug discovery start-up, Piqur Therapeutics AG, which recently completed a seed financing round that will enable it take forward a pipeline of next-generation kinase inhibitors. The company, a spinout from the University of Basel, where Wymann is based, is developing dual inhibitors of PI3K and mammalian target of rapamycin (mTOR). It has assembled a veritable

Biogen Idec Inc.'s move to buy all rights from Elan Corp. plc to the blockbuster multiple sclerosis (MS) therapy Tysabri (natalizumab) didn't take long for analysts to applaud, though Wall Street traders spent time digesting the $3.25 billion deal, which includes contingent payments. "Everybody's probably searching for [the answer to the question], 'Does this make sense on a net present value [NPV] basis?' We think it does," said Paul Clancy, chief financial officer of Weston, Mass.-based

LONDON – e-Therapeutics plc is copper-bottoming its future, raising £40 million (US$62.66) in a share issue which, added to existing resources of £8 million, will fund the network pharmacology specialist until 2017. As this money is dispensed through its clinical development programs, the company expects to receive £12 million in government R&D tax credits, bringing the total pot to £60 million. A biggish chunk of this cash is going into one basket, with plans to spend £25 million on getting

LONDON – The Innovative Medicines Initiative (IMI) officially launched the European Lead Factory, a €196 million (US$266 million) project in which seven pharma companies will make at least 300,000 compounds available for screening via an open platform. That will allow academic and small company partners access to industry-quality high-throughput screening and speed the translation of new target biology through to drug leads. The cash for the five-year project comes from the European Union's R&D

LONDON – Medgenics Inc. reached the higher end of its ambitions in an issue of stocks and warrants, raising $30 million to provide funding for further clinical development of its Biopump protein drug implant technology. "I think this speaks for itself: In this environment, raising $30 million is not easy," said Andy Pearlman, CEO. The aim was to raise between $20 million and $30 million, so this was at the higher end, and in the event "some folks didn't get in," Pearlman told BioWorld

LONDON – With the discovery of 24 new genes that are linked to myopia, or short-sightedness, most of the hereditary causes of this condition are now known, researchers contend. People with the faulty versions of those genes are 10 times more likely to have myopia than those without, the study found. The genes identified are involved in neurotransmission, modeling of the extracellular matrix, ion transport, retinoic acid metabolism and eye development. Caroline Klaver, professor of

4-Antibody AG, of Basel, Switzerland, and the Ludwig Institute for Cancer Research in New York signed a multitarget research and development collaboration with Recepta Biopharma SA, of Sao Paulo, Brazil, the leading Brazilian developer of therapeutic antibodies founded by the Ludwig Institute and Brazilian investors. The deal represents an expansion of a partnership the Ludwig Institute and 4-Antibody initiated more than a year ago. Financial details were not disclosed. Addex Therapeutics SA

LONDON – The UK BioIndustry Association is stepping up its campaign to improve funding for the industry through the creation of Citizens' Innovation Funds (CIFs) that will enable the general public to invest in biotech start-ups. The BIA organized a lobbying day in Parliament last week, meeting with members of parliament and treasury ministers to promote the plan. The association also has built a coalition of other groups to support CIFs, including the Confederation of British Industry and the