With additional data in the offing for its Leber's hereditary optic neuropathy (LHON) drug Raxone (idebenone), Santhera Pharmaceuticals AG has withdrawn its marketing authorization application (MAA) from the European Medicines Agency's (EMA) re-examination process and will instead submit a new file with an expanded patient database. The move will significantly delay the program but should, the company hopes, substantially improve its chances of success. Liestal, Switzerland-based Santhera

LONDON – The shape of the UK's new drug pricing regime became clearer last week when the government confirmed it is handing responsibility for assessing the value of medicines to the National Centre for Health and Clinical Excellence (NICE), a body that has repeatedly clashed with the industry over its decisions on whether or not drugs should be funded by the National Health Service. Currently, NICE gets to pass judgement once a product has marketing authorization and the price is agreed; when

LONDON – The cannabis-based drug specialist GW Pharma plc has filed for a listing on Nasdaq, with the ambition of raising up to $50 million to turbo-charge development of its preclinical pipeline and expand manufacturing capacity in anticipation of the U.S. launch of its lead product Sativex. "It's important for strategic reasons that we engage U.S. investors," Justin Gover, CEO told BioWorld International. The development of the company's product pipeline is increasingly focused toward the U.S

LONDON – AstraZeneca plc is once again taking the ax to its R&D operations, announcing plans to relocate 2,500 roles and cut about 1,600 jobs, mainly in the UK and U.S., as it consolidates discovery and development in Cambridge, UK; Gaithersburg, Maryland; and Mölndal, near Gothenburg, in Sweden. The recently appointed CEO of AstraZeneca, Pascal Soriot said the aim is to increase the company's R&D productivity. The changes "put science at the heart of everything we do," he said. This will

Sinovac Biotech Ltd.'s enterovirus 71 (EV71) vaccine looks good in preliminary results from Phase III data assessing efficacy, immunogenicity and safety against hand, foot and mouth disease (HFMD). The data showed that it was 95.4 percent efficacious against the disease in about 10,000 healthy infants between the ages of 6 months and 35 months. The rate of serious adverse events among infants receiving the vaccine was 2.2 percent, compared to 2.6 percent for those receiving a control vaccine, a

India's Drug and Cosmetics Act purports to keep clinical trials kosher under something called Schedule Y, but the law – which has not been amended since 2005 – lacks rules for mandatory practice and does not specify what recourse trial participants have when things go wrong. Where contract research organizations (CROs) fit into the picture is not fully explained, though CROs do a great deal of clinical work in India. India's Ministry of Health & Family Welfare is asking for comment from drug

ThromboGenics NV is banking a €45 million (US$58.8 million) milestone payment from its partner, Alcon, a unit of Novartis AG, following formal European Union (EU) approval for Jetrea (ocriplasmin) for treating patients with vitreomacular traction (VMT), including those with a macular hole up to 400 microns in diameter. A second payment of €45 million will follow on the first EU sale of the drug, which are expected to occur in the coming weeks. The news was hardly unexpected, as the product was

LONDON – The genome of the tapeworm has been sequenced, providing researchers with a panoply of genes that may provide new targets for treatment of those parasites. Scientists hope that, by identifying tapeworm genes that are similar to human genes, it may be possible to select candidate drugs that are already approved for use against other conditions, such as cancer. Members of the multinational consortium that took part in the sequencing effort have already started testing laboratory cultures

Newron Pharmaceuticals SpA and its partner, Zambon SpA, remain on track to file for approval for safinamide as add-on therapy in Parkinson's disease in the fourth quarter, following the release of pivotal Phase III data at the annual meeting of the American Academy of Neurology in San Diego this week. The drug attained the primary endpoints in two Phase III trials, MOTION, which recruited 679 patients with early stage disease, and SETTLE, which recruited 549 patients with mid-to-late-stage

Acuvax Ltd., of Brisbane, Australia, will acquire Biolife Science Ltd., also of Brisbane, and seeks to generate up to $5 million for Phase II trials of Biolife's HER-Vaxx vaccine for breast cancer, a potential alternative to Herceptin (trastuzumab, Roche AG). Acuvax is publicly listed on the Australian Securities Exchange (ASX:ACU) and will undergo a 200-to-1 share consolidation, after which it will seek to raise up to $5 million through placement of up to 25 million shares at A20 cents (US21

LONDON – Start-up Helmedix Pty Ltd. has raised A$1.25 million (US$1.29 million) in seed funding, providing a springboard to the commercialization of molecules based on immune-modulating peptides used by helminth parasites to suppress the immune response of their hosts. "This is at a very early stage; the funding will cover the early stages of work to try and make variants around these compounds and to tease out the mode of action," said Stephen Thompson, a director of Helmedix and partner at

AmVac AG, of Zug, Switzerland, disclosed new preclinical data on its vaccine candidate AMV602 for the prevention of infections caused by respiratory syncytial virus (RSV). Last fall, the company selected the candidate as the most promising for further development among a range of potential leads. In preclinical studies, researchers at the University of Siena, Italy, have confirmed the candidate's excellent safety and efficacy, showing that even reduced doses of AMV602 trigger effective immune

LONDON – Cell Medica Ltd. has attracted Kurt Gunter, a leader of cell therapy in the U.S., to be its chief medical officer, in one of five senior appointments that will see the London-based company scaling up to a global operation. Gunter is currently president of the International Society for Cellular Therapy and previously was involved in efforts to commercialize cell therapies at Hospira Inc., ViaCell Inc. and Transkaryotic Therapies. Before working in industry, he was acting deputy director

NeuroVive Pharmaceutical AB extended its drug pipeline by picking up a series of polyketide-based preclinical cyclophilin inhibitors from Biotica Technology Ltd., a UK pioneer of polyketide engineering, which is currently in administration. Financial terms were not disclosed, but the agreement covers a potent class of polyketides called sangamides, which are derived from a naturally occurring molecule called Sanglifehrin A, as well as technology platform assets in the form of microbial strains

LONDON – Progenitor Labs Ltd. has raised £4 million (US$5.8 million) in seed financing from the GlaxoSmithKline plc venture fund SR One, enabling the company to further develop its platform technology for screening for small molecules that promote the differentiation of endogenous adult stem cells. Progenitor is a spinout from another start-up company, Plasticell Ltd., based in London, which has developed CombiCult, a high-throughput system for the discovery of optimal protocols for

LONDON – Study of a family affected by an inherited neurological disease has helped to reveal the identity of an enzyme that could point the way to new therapies for cancer and cardiovascular disease. The existence of the enzyme, which plays a role in maintaining cell survival and DNA repair, has been predicted for at least 30 years and has even been shown to operate in cell cultures. But, until it was linked with the gene defect in the family studied, no one knew what it was. Now an

LONDON – Mesoblast Ltd. has raised A$170 million (US$174.6 million) in a private placing, bringing total cash reserves for the stem cell specialist to A$332 million and providing funding for a Phase III trial in treating degenerating spinal discs. The shares will be issued at A$6.30, a 2.2 percent discount to the closing price of A$6.44 the day before the private placing was announced on March 6. The new shares represent less than 15 percent of the shares in issue so shareholder approval was

LONDON – It was money galore for UK biotech on Monday as the government Biomedical Catalyst fund announced its second round of grants, with 51 companies bagging a total of £47.2 million (US$70.2 million). Taken with the first round of grants, that brings the amount disbursed since the £180 million fund was set up a year ago to £96 million, with 115 companies receiving funding to help technologies across the valley of death and toward the market. And this is having one of the desired effects – of

AbbVie Inc. and InterMune Inc. could derail the European Medicines Agency's (EMA) carefully timetabled introduction of a new data transparency regime on Jan. 1, 2014, by seeking court injunctions preventing the disclosure of clinical trial data for their respective principal products, Humira (adalimumab) and Esbriet (perfidenone). According to the website of the Court of Justice of the European Union in Luxembourg, InterMune has lodged an application initiating proceedings on Feb. 11, while