• Amarin Corp. plc,of London, said the FDA indicated that an additional Phase III trial demonstrating robust results, in conjunction with confirmatory evidence from the existing clinical data, might be sufficient to support a new drug application for Miraxion in Huntington's disease. The company now is determining the design for the Phase III trial and is deciding whether to move forward on its own or seek a partner to defray costs. Amarin previously conducted two Phase III studies of Miraxion in HD patients, both of which missed their primary and secondary endpoints, though the drug showed efficacy in a subset of patients. Further analysis of those data indicated that a clinical benefit could be seen from a longer treatment period with Miraxion.

• Beike Biotechnology, of Shenzhen, China, established a laboratory facility for the research of cell reprogramming and gene engineering through a joint agreement with the Shenzhen Graduate School of China's Tsinghua University. The lab will conduct research on stem cells, the nuclear transfer and reprogramming of cells, monoclonal antibodies known as "magic bullets" for treating disease and other cell-engineering innovations.

• BioLineRx Ltd., of Jerusalem, signed a worldwide exclusive license agreement with Yissum, the technology transfer company of the Hebrew University of Jerusalem, and Hadasit, the technology transfer company of Hadassah medical organization, for the development and commercialization of BL-4060, a small-molecule drug candidate for the treatment of cancer. Financial terms of the license were not disclosed. BL-4060 is an analogue of the fatty molecule ceramide, a strong pro-apoptotic agent that induces apoptosis, indicated for the treatment of cancer.

• Cellectis SA, of Romainville, France, delivered a meganuclease targeting the RAG1 gene to a research team at the Children's Hospital in Boston. The RAG1 gene is mutated in certain severe combined immunodeficiency (SCID) patients, and until recently, the only possible therapy for those patients was a bone marrow transplant for reconstitution of their entire immune system. A gene therapy tested in France in 2000 resulted in several of the treated patients developing leukemia, which seemed to be linked to the insertion of the gene therapy vector in the vicinity of a particular gene. Meganucleases represent an alternative approach to the random insertion of a gene drug, since they entail the correction of the defective gene itself and thus make for a safer form of gene therapy. Cellectis already delivered a meganuclease to the Necker children's hospital in Paris in August to test its potential for repairing the defective gene in cell lines carrying a X-SCID mutation, in which the disease is linked to a mutation of the IL2RG gene.

• Crucell NV, of Leiden, the Netherlands, said the FDA granted fast-track designation to the firm's rabies monoclonal antibody cocktail. Results of a Phase I clinical trial indicated that the cocktail is well tolerated, provides the expected neutralizing activity and can be safely co-administered in combination with a standard rabies vaccine. In other news, Crucell and DSM Pharmaceutical Products, of Parsippany, N.J., have granted licensing rights for their PER.C6 cell line to Daiichi Sankyo, of Tokyo. Financial terms of the agreement were not disclosed.

• Innate Pharma SA, of Marseille, France, has begun regulatory preclinical development of IPH 2201, a new monoclonal antibody targeting NK (natural killer) cells. It is the latest in a series of products derived from Innate's NK cell biology platform, which are being developed in collaboration with Novo Nordisk A/S, of Bagsvaerd, Denmark. The two companies concluded a three-year agreement in March 2006, under which Novo Nordisk is the exclusive licensee for all products that emerge from this platform up to March 2009. The most advanced is IPH 2101, which currently is being tested in two Phase I clinical trials. The Danish company will take over the preclinical and clinical development of IPH 2201, possibly up to regulatory approval. A third antibody to emerge from the NK cell platform, IPH 23XX, is at the exploratory phase and has just attained an intermediate milestone, and Innate said it could enter preclinical development in 2008. Innate Pharma received undisclosed milestone payments from Novo Nordisk for the latest stages reached in the development of both IPH 2201 and IPH 23XX.

• Kiadis Pharma BV, of Amsterdam, the Netherlands, agreed to provide Rhitol during a limited period of time to requesting physicians participating in the ongoing Phase I/II trial, which closed for enrollment and is expected to finish at the end of this year. Rhitol is in development for severe, chronic, steroid-refractory graft-vs.-host disease patients who have exhausted other treatment options. Kiadis anticipates beginning a Phase III study in 2008.

• Medical Marketing International Group plc, of Cambridge, UK, agreed to terms with its partners, King's College London (KCL), Queen Mary University of London and the inventing scientists for an option to acquire their shareholdings in Viratis Ltd, MMI's subsidiary which is developing HIV and Hepatitis B therapies based on gene silencing. Since its formation, MMI's shareholding in Viratis has increased from 50 percent to 72 percent by funding development program. That soon will reach 75 percent, at which point MMI will have the right to acquire the remaining shares in Viratis in exchange for new MMI shares.

• Novexel, of Paris, initiated a Phase II clinical trial of NXL 103 (PI + PII), an oral antibiotic of the streptogramin class, which destroys bacteria in multi-resistant strains such as S. pneumoniae, H. influenzae and S. aureus, including those resistant to methicillin (SARM). The multicenter trial, which is being carried out in Bulgaria, Chile, Croatia, Estonia, Germany, Peru, Poland, Romania, South Africa and Ukraine, will test two doses of NXL103 vs. a reference treatment in 100 patients suffering from community pneumonia. Sanofi-Aventis, of Paris, has an option on NXL 103 up to the Phase IIa stage, and under that agreement the launch of this trial will result in the French pharmaceutical company paying Novexel a quarterly rights fee.

• Novogen Ltd., of Sydney, Australia, said study results revealed that NV-52, a synthetic flavonoid, may be useful for maintaining remission in inflammatory bowel disease (IBD). No adverse effects were detected during Phase Ia and Phase Ib trials of the compound in healthy volunteers in Australia. NV-52 has been shown to be effective in suppressing colonic inflammation in laboratory mice and has not displayed any toxicity in in vitro and extensive animal toxicological studies. The compound is thought to work as a selective-thromboxane synthase inhibitor, which is believed to play a major therapeutic role in IBD due to its apparent ability to inhibit pro-inflammatory thromboxanes.

• Orexo AB, of Uppsala, Sweden, said its shareholders approved the previously proposed acquisition of Stockholm, Sweden-based Biolipox AB. Biolipox had previously agreed to acquire several assets from Vancouver, British Columbia-based Inflazyme Pharmaceuticals Ltd. in exchange for up to $11 million plus potential royalties. Inflazyme shareholders are scheduled to vote on the Biolipox deal at a special meeting on Friday.

• Pharmaxis Ltd., of Sydney, Australia, raised A$11.7 million (US$10.5 million) from its share purchase plan that closed earlier this month. Shareholders were given the opportunity to buy up to $5,000 worth of shares each at the A$3.90 price paid by institutional investors in a A$50 million placement last month. About half the company's shareholders, a total of 2,496, participated in the plan, and Pharmaxis issued about 3 million shares.

• Photocure ASA, of Oslo, Norway, said its board decided to de-merge PCI Biotech, its cancer-focused drug delivery subsidiary. PCI, which develops a method for light-directed drug delivery of therapeutic molecules directly into tumor cells, intends to seek a public listing on the Oslo Stock Exchange following completion of the de-merger process.

• Rosetta Genomics Ltd., of Rehovot, Israel, said it has initiated in vivo studies of its microRNA-based liver cancer therapeutic program in collaboration with Isis Pharmaceuticals Inc., of Carlsbad, Calif. Eight microRNAs screened and tested have been determined to lead to a decrease in liver cancer cell growth when inhibited, the firm said. Those will be tested further during the in vivo studies, the company said.

• ThromboGenics NV, of Leuven, Belgium, said it has begun preclinical development of an anti-VPAC antibody for thrombocytopenia. Thrombocytopenia, which is the reduced number of platelets in blood, is a common severe side effect of chemotherapy and increases the risk of bleeding and severity of hemorrhage. Researchers at the University of Leuven and ThromboGenics have developed a novel therapeutic approach, showing that the inhibition of VPAC could stimulate the production of platelets. VPAC is a receptor present at the surface of bone marrow cells called megakaryocytes, which, when mature, produce platelets.

• UCB SA, of Brussels, Belgium, said it has been informed by the European Medicines Agency that the Committee for Medicinal Products for Human Use adopted a negative opinion on the market authorization application in the European Union for Cimzia (certolizumab pegol) in the treatment of patients with Crohn's disease. UCB plans to appeal, and a decision is expected during the first half of 2008. Cimzia is a pegylated anti-TNF, for which UCB filed a biologics license application in early 2006. The drug was approved in Switzerland for Crohn's disease in September of this year, and UCB said preparation for a U.S. regulatory submission for Cimzia in treating rheumatoid arthritis is ongoing.

• Vectura Group plc, of Chippenham, UK, and its partner Sosei Group Corp., of Tokyo, said Novartis AG, of Basel Switzerland, has begun a Phase II safety study of QVA149 for the treatment of chronic obstructive pulmonary disease. The randomized, double-blind, placebo-controlled, multicenter study will determine the effect of QVA149 and indacaterol on mean 24-hour heart rate in patients with moderate to severe COPD. NVA237 was licensed to Novartis by Sosei and Vectura in 2005 in a deal in which the two companies could receive up to $375 million in milestones, plus royalties on sales.