• Albireo AB, of Gothenburg, Sweden, said the European Medicines Agency's Committee for Orphan Medicinal Products issued a positive opinion on an orphan application for the firm's lead hepatology product, ileal bile acid transporter inhibitor A4250, in primary biliary cirrhosis, progressive familial intrahepatic cholestasis and Alagille syndrome. The opinion has been forwarded to the European Commission for final approval.

• Antisense Pharma GmbH, of Regensburg, Germany said it has been granted orphan drug designation from the FDA for its oncology antisense compound trabedersen to treat malignant melanoma. Previously, trabedersen has received orphan drug designation by the European EMA and the FDA in high-grade glioma (malignant brain tumor) and in advanced pancreatic cancer.

• Biokine Therapeutics Ltd., of Rehovot, Israel, has received orphan drug designation from the FDA for the mobilization of hematopoietic stem cells from bone marrow into peripheral blood for collection and subsequent transplantation in patients with hematological cancers. Biokine previously received FDA approval to conduct a Phase II study for stem cell mobilization in multiple myeloma and non-Hodgkin's lymphoma patients using BKT140.

• Camargo Pharmaceutical Services, of Cincinnati, said it completed five pre-investigational new drug meetings last month with the FDA for 505(b)(2) products, including two for India-based pharmaceutical companies. Since 505(b)(2) drugs rely in part on data from already approved therapies, they can be developed and get FDA approval in as little as 30 months with fewer trials and at a lower cost. Only about 20 percent of new drugs were approved through the 505(b)(2) process in 2006. Today, when the standard 505(b)(1) path can take as long as 15 years and $1 billion in investment, more than 80 percent of new, small-molecule drugs are approved through the 505(b)(2) process, Camargo noted.

• Catalent Inc., of San Diego, began supplying Abilify OD Tablets (orally disintegrating tablets) using its Zydis fast-dissolve drug delivery technology to Otsuka Pharmaceutical Co. Ltd., of Tokyo. Otsuka has developed Abilify OD for schizophrenia and bipolar disorder. Catalent will make a multimillion dollar investment in its Zydis ODT operations in Swindon, UK, to support the demand for such formulations.

• Chimerix Inc., of Research Triangle Park, N.C., said it inked a deal giving Whitehouse Station, N.J.-based Merck & Co. Inc. exclusive worldwide rights to CMX157, its lipid acyclic nucleoside phosphonate in development to treat HIV infection. Under the terms, Merck will take over development and commercialization of the oral nucleoside reverse transcriptase inhibitor in exchange for a $17.5 million up-front payment and up to $151 million in milestones, plus royalties on future sales. CMX157 has completed a Phase I study in healthy volunteers.

The FDA has approved Turdorza Pressair (aclidinium bromide inhalation powder) by Forest Laboratories Inc., of New York, and Almirall SA, of Barcelona, Spain, for chronic obstructive pulmonary disease, including chronic bronchitis and emphysema. Forest said it is the first long-acting inhaled anticholinergic agent approved in more than eight years for COPD. In trials, Turdorza Pressair showed statistically significant improvements in bronchodilation. In other news from Forest, shareholder Carl Icahn and the company are continuing their battle over the leadership and direction of the firm in advance of an Aug. 15 annual meeting. Forest released a presentation highlighting its recent achievements and encouraging shareholders to vote for its slate of directors, which includes five new independent members. Icahn sent an open letter to shareholders, promoting his slate, which includes Daniel Ninivaggi, president of Icahn Enterprises LP, and Eric Ende, who was appointed to Genzyme Corp.'s board following a proxy fight between that company and Icahn. The letter, sent Tuesday, is critical of Forest's current leadership and questions the independence of its directors.

• Medicago Inc., of Quebec City, successfully completed a rapid fire test of its production of an H1N1 virus-like particles (VLP) influenza vaccine, its fifth milestone under a $21 million Technology Investment Agreement with the Defense Advanced Research Projects Agency to develop a vaccine facility in Research Triangle Park, N.C., and demonstrate the scalable manufacturing of its plant-expressed VLP vaccines at that facility. The milestone, which involved producing at least 10 million doses of the vaccine in 30 days, triggered a $1 million payment. Medicago has received $19.8 million in other milestone payments under the agreement.

• Merck & Co. Inc., of Whitehouse Station, N.J., said that it signed an agreement with Yamasa Corp., of Choshi, Japan, to develop EFdA (4'-ethynyl-2-fluoro-2'-deoxyadenosine), a nucleoside reverse transcriptase inhibitor candidate that is in preclinical studies and has shown antiviral activity toward highly resistant HIV strains. In addition, Merck announced plans to advance an internally developed candidate, MK-1439, a non-nucleoside reverse transcriptase inhibitor, into a Phase IIb trial.

• Mesoblast Ltd., of Melbourne, Australia, reported positive results in a large animal model of rheumatoid arthritis (RA) following a single intravenous injection of its allogeneic immunomodulatory adult mesenchymal precursor cells (MPCs). The study was conducted on 30 sheep with established collagen-induced arthritis, comparing a single intravenous injection of allogeneic MPCs at one of three doses (0.3, 1 and 2 million MPCs/kg) to saline. Thirty days later, joint synovial tissues from arthritic sheep were examined. The findings demonstrated that MPCs are immunoregulatory and concomitantly suppress the activation and proliferation of T-cells, monocytes and synoviocytes seen in active rheumatoid arthritis.

• SomaGenics Inc., of Santa Cruz, Calif., said a new study showed that the two structurally distinct types of small synthetic hairpin RNA (sshRNA), right-loop or R-type and left-loop or L-type, have different mechanisms of action, and each is different from the mechanisms of action of standard siRNAs and shRNAs. Unlike larger hairpin RNAs and most microRNAs, the sshRNAs don't require the Dicer enzyme for activity in cells. The study was published online this week in Nucleic Acids Research.

• Sunesis Pharmaceuticals Inc., of South San Francisco, said data showed that its lead candidate vosaroxin, because of its resistance to P-glycoprotein-mediated drug efflux and lack of chemical reactivity, should minimize chemotherapy adverse effects such as cardiotoxicity. Development of the drug is featured in a peer-reviewed paper published in the August 2012 issue of Expert Opinion on Investigational Drugs. Vosaroxin, a first-in-class anticancer quinolone derivative, is being evaluated in the Phase III VALOR trial to treat relapsed or refractory acute myeloid leukemia.

• SuppreMol GmbH, of Munich, Germany, signed a licensing agreement with the University Hospital of Regensburg to develop SM401, a humanized anti-IL-3 monoclonal antibody for early diagnosis and treatment of rheumatoid arthritis (RA). SuppreMol will advance preclinical development of the compound in RA and simultaneously will validate a sensitive human IL-3 diagnostic assay for patient stratification. Early development of SM401 is being funded by a €1.37 million (US$1.65 million) grant from the German Federal Ministry of Education and Research.