• Ablynx NV, of Ghent, Belgium, said results from the open-label extension of its Phase Ib study of antithrombotic ALX-0081 in patients with stable angina undergoing percutaneous coronary intervention supported those of the original Phase I study and collectively provided proof of concept that the drug is safe and well tolerated and acts as a potent inhibitor of platelet aggregation. The 22-patient trial was designed to test ALX-0081, which inhibits von Willebrand Factor, on vWF-mediated clotting, measured using platelet aggregation biomarkers, including RICO (ristocetin cofactor). All 20 patients who received ALX-0081 experienced complete RICO inhibition that was statistically significant compared to placebo.

• AVI BioPharma Inc., of Bothell, Wash., reported initial efficacy data from an ongoing Phase Ib/II trial of AVI-4658 in Duchenne's muscular dystrophy, showing that analysis of the post-treatment biopsies from patients in the first four cohorts found that those in the 2 mg/kg and 4 mg/kg cohorts (three of three) demonstrated correctly spliced mRNA for dystrophin. One of those patients, in the 2 mg/kg cohort, showed robust expression for dystrophic protein by western blot and immunofluorescent analysis. Treatment with AVI-4658 in the three patients led to accurate skipping of exon 51, which is believed to be necessary to restore the mRNA reading frame for functional dystrophin expression with this class of mutations. No RNA or protein expression signal was detected in patients from the 0.5 mg/kg or 1 mg/kg cohorts after completing treatment.

• Eisai Inc., of Woodcliff Lake, N.J., reported that eritoran tetrasodium (E5564) appeared to be well tolerated in patients with severe sepsis in a Phase II trial published in the January 2010 issue of Critical Care Medicine. The study, which evaluated two doses of the drug, given in 45 mg every 12 hours, was not powered to demonstrate statistical significance in reduction in mortality, though results showed a 26.6 percent mortality rate for high-dose eritoran vs. 33.3 percent for placebo. Eritoran is a Toll-like receptor 4 antagonist.

• Ferring Pharmaceuticals SA, of Saint-Prex, Switzerland, said results from Phase III pivotal study subanalyses, reported in European Urology, showed that prostate cancer patients receiving Firmagon (degarelix) had a statistically significant greater probability of prostate-specific antigen recurrence-free survival compared with those taking leuprolide. Firmagon is a gonadotropin-releasing hormone antagonist approved in both the U.S. and Europe.

• Intra-Cellular Therapies Inc., of New York, reported Phase I data showing that ITI-007, its lead antipsychotic drug, produced dose-related and long lasting occupancy of three key targets of psychotropic drug action, as seen using positron emission tomography. Those targets are serotonin 5-HT2A and dopamine D2 receptors, as well as the serotonin reuptake transporter. At 10 mg, the first evidence is seen of D2 receptor occupancy in the basal ganglia.

• LEO Pharma A/S, of Ballerup, Denmark, said its subsidiary, Peplin Inc., reported positive results from two Phase III trials of PEP005 (ingenol mebutate) gel in actinic (solar) keratosis lesions on head treatment areas including the face and scalp. Both studies, which each enrolled 250 patients, met their primary endpoints, with statistically significant clearance of the AK lesions vs. vehicle. One additional trial is ongoing to test PEP005 in lesions on nonhead locations, including the trunk and extremities. Data from that trial are expected toward the end of the first quarter of 2010.

• Medicago Inc., of Quebec City, reported positive interim results from a Phase I study of its H5N1 Asian influenza vaccine candidate, showing that it was safe and well tolerated and induced a solid immune response. The study enrolled 48 healthy volunteers between the ages of 18 and 60 to receive two doses of either Medicago's vaccine at 5 mcg, 10 mcg or 20 mcg or placebo. Preliminary results showed that 81 percent of immunized subjects developed an immune response against the H5N1 virus after the second immunization. The vaccine also induced the production of antibodies cross-reacting with two other strains of H5N1 Asian flu, suggesting the vaccine's potential for cross-protection.

• Neurocrine Biosciences Inc., of San Diego, is advancing NBI-98854, a selective vesicular monoamine transporter 2 inhibitor, into a multiple repeated-dose Phase I study, following results from a single-ascending-dose study in healthy male volunteers that showed the drug was generally safe and well tolerated. The company said it will begin preparation for a Phase II proof-of-concept study to be initiated later in 2010.

• Resverlogix Corp., of Calgary, Alberta, started dosing patients in its Phase II trial of RVX-208, an oral, small-molecule therapy to treat atherosclerosis, in patients with stable coronary artery disease. The 18-week, randomized study will enroll about 280 patients with stable CAD for 13 weeks. The primary objective is to determine if RVX-208 will produce an increase in plasma ApoA-1 levels compared to placebo, while secondary objectives are to examine the safety and tolerability, compare dose and time response relationships for ApoA-1 over time and to examine and key reverse cholesterol makers such as Alpha 1 HDL. The company said a second parallel study is expected to involve patients with unstable acute coronary syndrome and will include the use of intravascular ultrasound.