• BioMarin Pharmaceutical Inc., of Novato, Calif., has initiated a Phase I/II trial for BMN 701, a fusion protein of insulin-like growth factor 2 and acid alpha glucosidase in development for the treatment of Pompe disease. The open-label study will evaluate the safety, tolerability, pharmacokinetics, pharmacodynamic and clinical activity of BMN 701 administered as an intravenous infusion every two weeks at doses of 5 mg/kg, 10 mg/kg and 20 mg/kg. The company expects to enroll approximately 30 patients between the ages of 13 and 65 with late-onset Pompe disease for a treatment period of 24 weeks.

• Geron Corp., of Menlo Park, Calif., announced enrollment of the first patient in a Phase II trial to evaluate the activity of the company's telomerase inhibitor drug, imetelstat (GRN163L), in patients with essential thrombocythemia (ET). ET is a chronic disorder that arises in the hematopoietic stem cells in the bone marrow. The leukemic stem cells produce aberrant clones of platelet-forming cells, which results in increased numbers of circulating platelets. The trial is a Phase II, open-label study of imetelstat as a single agent in patients with ET who have failed or are intolerant to at least one prior therapy or who refuse standard therapy, such as hydroxyurea, anagrelide or interferon-alpha.

• GlaxoSmithKline plc, of London, and Prosensa Therapeutics, of Leiden, the Netherlands, said the first patient commenced treatment in a Phase III study investigating GSK2402968 in ambulant boys with Duchenne's muscular dystrophy (DMD), who have a dystrophin gene mutation amenable to an exon 51 skip. The randomized, placebo-controlled study will enroll 180 patients, from up to 18 countries. It's designed to assess the efficacy and safety of GSK968 6mg/kg, once weekly, compared to placebo, for 48 weeks in ambulant boys older than 5 years with DMD. The primary efficacy endpoint is a measure of muscle function using the six minute walking distance test.

• Koronis Pharmaceuticals Inc., of Seattle, said Phase IIa trial results showed that the frequency of specific drug-induced mutations in the HIV genome can be significantly increased by administering KP-1461, a drug based on the firm's Viral Decay Acceleration platform. Data were published in the Jan. 14, 2011, issue of PLoS One. Koronis is planning a follow-on Phase II trial to determine the treatment duration required to achieve a clinically meaningful decrease in a patient's viral load.

• Merck Serono, a division of Merck KGaA, of Darmstadt, Germany, reported further analysis of the large, randomized Phase II OPUSa study demonstrating an association between early tumor shrinkage and long-term median overall survival of more than two years for patients with KRAS wild-type metastatic colorectal cancer treated with Erbitux (cetuximab) plus Folfox standard chemotherapy. The correlation was not seen in the chemotherapy-alone arm of the study. The study will be presented at the annual Gastrointestinal Cancers Symposium of the American Society of Clinical Oncology.

• Peregrine Pharmaceuticals Inc., of Tustin, Calif., and Tucson, Ariz., initiated an investigator-sponsored trial for patients with HER2-negative metastatic breast cancer, which accounts for 75 percent of metastatic breast cancers. The open-label Phase I study will treat patients with Peregrine's investigational monoclonal antibody bavituximab in combination with the chemotherapy agent paclitaxel. Currently, Peregrine's bavituximab is being evaluated in randomized Phase II trials in front-line non-small-cell lung cancer (NSCLC), second-line NSCLC, pancreatic cancer and hepatitis C.

• Pluristem Therapeutics Inc., of Haifa, Israel, completed a parallel scientific advisory process with the European Medical Agencies (EMA) and the FDA regarding the company's planned clinical development program for PLX-PAD. Based on positive feedback from the agencies, Pluristem said it is now in a position to advance toward two clinical studies with its PLX-PAD cells: a joint FDA-EMA Phase II/III study of PLX-PAD for critical limb ischemia and a Phase II study for intermittent claudication under the FDA and the Paul Ehrlich Institute, the German competent authority in the European Union. The primary endpoint of the study will be major amputation-free survival rate (amputations and death) at 12 months from the initial treatment with PLX-PAD or placebo.

• Targacept Inc., of Winston-Salem, N.C., reported positive top-line results from a Phase II proof-of-concept trial to assess TC-5619 as an augmentation therapy to improve cognition in patients with schizophrenia. In the trial, TC-5619 met the protocol criteria for a positive result on the primary efficacy outcome measure, the Groton Maze Learning Task of the CogState Schizophrenia Battery, and was well tolerated. Analyses of the full dataset from the trial are ongoing. Based on the outcome of the trial, AstraZeneca plc, of London, has the right to license TC-5619 on terms specified in the parties' December 2005 collaboration agreement focused in cognitive disorders including schizophrenia. If AstraZeneca exercises its right to license TC-5619, the agreement provides for AstraZeneca to make a $30 million payment to Targacept and to assume responsibility for and fund all future development and commercialization. In that event, Targacept would be eligible to receive additional payments of up to $212 million, contingent on the achievement of development, regulatory, first commercial sale and first detail milestone events for three indications, as well as stepped double-digit royalties on any future TC-5619 product sales. AstraZeneca said it will decide whether to exercise its license right in the first half of 2011.