• Accentia Biopharmaceuticals, of Tampa, Fla., reported positive interim data from its pivotal Phase III clinical trial for SinuNase, for chronic sinusitis. In a letter to shareholders, CEO Francis O'Donnell Jr., said that interim, blinded, intent-to-treat data on the primary endpoint (complete resolution of both cardinal symptoms) for approximately 80 percent of patients in the study showed that about 20 percent of all patients are achieving the primary endpoint of complete resolution of both cardinal symptoms and another 23 percent are achieving complete resolution of one or the other cardinal symptom at 16 weeks. He noted that it is important to remember that 50 percent of the patients received SinuNase and that 50 percent received a placebo control that had no antifungal activity. He said he expected that final results, due to be unblinded and reported in March, will show a highly statistically significant outcome. He said the firm currently plans to seek an expedited approval from the FDA through the Subpart H pathway, which allows for accelerated, conditional approval of therapeutics for serious unmet clinical indications.

• Alexion Pharmaceuticals Inc., of Cheshire, Conn., said it has started dosing patients in a study to evaluate the safety, efficacy and pharmacology of Soliris (eculizumab) as a treatment for paroxysmal nocturnal hemoglobinuria (PNH), a rare, acquired genetic blood disorder defined by hemolysis in which patients' red-blood cells are destroyed. The trial, known as AEGIS, is expected to enroll about 25 patients with PNH at clinical sites throughout Japan. Patients will be dosed with Soliris for 12 weeks after enrollment. The primary efficacy endpoint of the study is reduction of hemolysis from baseline. The study also will measure the effect of Soliris on other clinical manifestations of PNH, including blood transfusion requirements, thromboses and kidney function. Patients' overall quality of life, including fatigue, also will be assessed, the company said.

Allos Therapeutics Inc., of Westminster, Colo., initiated a Phase IIb trial of PDX (pralatrexate) in advanced non-small-cell lung cancer. The randomized, 160-patient study will compare PDX to Tarceva (erlotinib, OSI Pharmaceuticals Inc. and Genentech Inc.) according to a primary endpoint of overall survival. PDX is a small-molecule chemotherapeutic that inhibits dihydrofolate reductase, a folic acid-dependent enzyme involved in DNA synthesis. Shares of Allos (NASDAQ:ALTH) rose 93 cents, or 15.2 percent, to close at $7.06 on Monday.

• Alseres Pharmaceuticals Inc., of Hopkinton, Mass., completed enrollment of a target 48 patients in its Phase I/IIa trial of Cethrin for acute spinal cord injury. Interim data from the trial showed that 27 percent of patients improved their sensory and motor function, a 400 percent increase over published results obtained using the standard of care. Additional data will be released throughout the year, and a Phase IIb trial is slated to begin in the first half of 2008. Cethrin contains a recombinant protein that inactivates the Rho enzyme to promote axon regeneration.

• Amicus Therapeutics Inc., of Cranbury, N.J., said that interim results of a Phase II clinical trial of Plicera (isofagamine tartrate) demonstrated that the drug was safe and well tolerated in patients with Gaucher disease, a lysosomal storage disorder caused by inherited genetic mutations in the GBA gene, which result in deficient activity of the enzyme acid beta-glucosidase, also known as glucocerebrosidase. The study enrolled 30 patients with Gaucher disease currently receiving enzyme replacement. Amicus said it expects to present complete results of the study in March at the American College of Medical Genetics annual meeting in Phoenix.

• Biogen Idec, of Cambridge, Mass., and Elan Corp., of Dublin, Ireland, said safety data from patients on commercial and clinical therapy worldwide for Tysabri showed a favorable benefit-risk profile. In the U.S., approximately 12,900 patients are on Tysabri therapy commercially, and internationally, about 7,500 patients were on the therapy commercially. There have been no cases of progressive multifocal leukoencephalopathy since re-launch in the U.S. and launch internationally in July 2006. The drug was pulled from the U.S. market in February 2005, only a few months after gaining approval for multiple sclerosis, and ongoing trials in MS and CD were halted after two patients contracted progressive multifocal leukoencephalopathy, a potentially fatal brain infection. Further analysis and review by the FDA led to the drug's return to the market, with limited use in MS patients. (See BioWorld Today, Aug. 2, 2007.)

• BioSante Pharmaceuticals Inc., of Lincolnshire, Ill., initiated a Phase III safety study with female sexual dysfunction drug LibiGel (transdermal testosterone gel). The randomized, double-blind, placebo-controlled, event-driven study will evaluate the cardiovascular risk of using testosterone in women and was required by the FDA as part of the LibiGel new drug application. BioSante must complete 12 months of the study prior to filing and will continue to monitor patients for an additional four years afterward. The FDA also required two efficacy studies, one of which is under way and the other of which is slated to begin in early 2008.

• Chelsea Therapeutics International Ltd., of Charlotte, N.C., said it started a Phase II trial of Droxidopa in patients with intradialytic hypotension, a condition characterized by a decrease in systolic blood pressure by 20 mm Hg or a decrease in mean arterial pressure by 10 mm Hg that affects about 25 percent of all hemodialysis patients. The double-blind, placebo-controlled, dose-response study is comparing 400-mg and 600-mg Droxidopa, an orally active synthetic precursor of norepinephrine, with placebo in 75 patients at 15 sites in the U.S. Following a two-week run-in period to establish baseline, patients in the three-arm study will receive a single oral dose of Droxidopa or placebo one hour before each dialysis treatment over a four-week period. The study will compare the change in mean blood pressure and symptomatic improvement during the final two weeks of treatment to baseline established before drug treatment.

• Coronado Biosciences Inc., of San Diego, said that the first patient has been dosed in a Phase I clinical trial of CNDO101 (5-imino-13 deoxy-doxorubicin), a fourth-generation doxorubicin analogue designed to eliminate cardiotoxicity without compromising efficacy. The dose-escalation study in up to 30 adult patients with advanced cancer will investigate the safety and preliminary efficacy of CNDO101.

• Evotec AG, of Hamburg, Germany, successfully completed a Phase I safety and tolerability study with EVT 302, a reversible and highly selective inhibitor of MAO-B in development for smoking cessation. The preliminary results confirmed the good tolerability profile of EVT 302. The preliminary data indicated that EVT 302 was well tolerated in young and elderly subjects up to the highest dose levels tested. Adverse events classified as possibly treatment related were transient and mostly of mild intensity; only very few moderate AEs were reported, and no severe or serious AEs occurred.

• Exelixis Inc., of South San Francisco, said it has started a Phase I/II trial of XL184, a small molecule that simultaneously inhibits the mesenchymal epithelial transition, rearranged during transfection and vascular endothelial growth factor receptor tyrosine kinases, in patients with non-small-cell lung cancer who have had progressive disease while on a regimen containing erlotinib. In the initial Phase I part of the study, safety and pharmacokinetics of combining XL184 with erlotinib will be evaluated. The primary endpoint of the Phase II part of the study is overall response rate.

• GTC Biotherapeutics Inc., of Framingham, Mass., completed the identification of historical patient records to serve as the control arm in its trial of the antithrombotic drug ATryn in patients with hereditary antithrombin deficiency. The company expects to report top-line results from the trial by the end of the month and file a biologics license application in mid-2008. GTC's partner LEO Pharma A/S, of Ballerup, Denmark, has begun launching the product in Europe.

• Hawaii Biotech Inc., of Honolulu, said it has received approval from the FDA to start a 24-patient safety study in healthy human volunteers with its recombinant, subunit West Nile vaccine. The trial, expected to start in April, will be conducted at a single site in Hawaii. The firm said it anticipates having results available by the end of 2008. There currently is no vaccine for the prevention of West Nile virus, which has infected about 25,000 U.S. residents since 1999, resulting in more than 1,000 deaths.

• MAP Pharmaceuticals Inc., of Mountain View, Calif., has initiated a Phase III clinical trial evaluating Unit Dose Budesonide (UDB), its nebulized version of budesonide, for the potential treatment of pediatric asthma. UDB is an inhaled corticosteroid designed to be administered more quickly and to provide efficacy at lower doses than conventional nebulized budesonide. The trial is a multicenter, randomized, double-blind, placebo-controlled study in approximately 360 asthmatic children, 12 months to 8 years of age. Patients are randomized to either 0.25mg UDB, 0.135mg UDB or placebo given twice a day over a 12-week treatment period. The primary efficacy endpoint for the study is the change in nighttime and daytime composite symptom scores (cough, wheeze and breathlessness).

• Maxygen Inc., of Redwood City, Calif., released interim data from a Phase II trial of MAXY-G34 in breast cancer patients with chemotherapy-induced neutropenia. Patients in the first cohort of the trial received 10 mcg/kg of MAXY-G34 and met the safety criteria with no serious adverse events or immunogenicity issue reported. The second cohort, which will receive 30 mcg/kg of MAXY-G34, is now enrolling. MAXY-G34 is a recombinant version of granulocyte colony stimulating factor designed to improve on Neulasta (pegfilgrastim, Amgen Inc.).

• Morphotek Inc., an Exton, Pa.-based subsidiary of Eisai Co. Ltd., initiated a Phase II trial of the monoclonal antibody MORAb-009 plus gemcitabine in the first-line treatment of pancreatic cancer. The randomized, double-blind, placebo-controlled trial will enroll 152 patients. MORAb-009 inhibits mesothelin, a cell-surface protein associated with pancreatic tumor growth and metastasis.

• Orexigen Therapeutics Inc., of San Diego, said that results of a Phase IIb clinical trial of Empatic, a fixed-dose combination of zonisamide sustained release and bupropion sustained release, showed that weight loss through 48 weeks for patients receiving 360 mg in the intent-to-treat group was 14 percent and 15.1 percent in the completer group. The primary trial objective was to determine the optimal dose ratios of bupropion and zonisamide. The trial randomized 623 obese patients and included only a minimal diet and exercise intervention.

• Pipex Pharmaceuticals Inc., of Ann Arbor, Mich., initiated a Phase II trial of Coprexa (tetrathiomolybdate) in Alzheimer's disease. The 150-patient, double-blind, placebo-controlled trial is being supported partially by a grant from the Italian Ministry of Health. Coprexa is an oral, small-molecule, anti-copper agent that also is in clinical trials for idiopathic pulmonary fibrosis and primary biliary cirrhosis and is under FDA review for Wilson's disease.

• Sangamo BioSciences Inc., of Richmond, Calif., said it has started a randomized, single-blind, placebo-controlled, multicenter Phase II clinical trial of SB-509 in patients with mild-to-moderate diabetic neuropathy. The study is designed to evaluate the safety and efficacy of SB-509, an injectable formulation of plasmid DNA that encodes a zinc finger DNA-binding protein transcription factor designed to upregulate the vascular endothelial growth factor A gene, and the pharmacokinetics of stem cell mobilization into the bloodstream after treatment with varying doses of the therapy.

• Sangart Inc., of San Diego, started testing its lead product, Hemospan, in a Phase II study in chronic critical limb ischemia patients. The trial's primary endpoint is tissue oxygenation measured noninvasively, and the company said the goal is to demonstrate that Hemospan can increase oxygen supply to severely compromised tissue. Hemospan is a hemoglobin-based oxygen transport agent in development as an oxygen therapeutic and as an alternative to blood transfusions.

• Santarus Inc., of San Diego, announced positive results from a recently completed clinical trial with immediate-release Zegerid (omeprazole/sodium bicarbonate) Capsules 40 mg/1,100 mg and two leading proton pump inhibitors (PPIs), Protonix (pantoprazole sodium) delayed-release tablets 40 mg and Prevacid (lansoprazole) delayed-release capsules 30 mg. The study evaluated the effect of morning PPI dosing on 24-hour gastric acid control in patients with symptoms of gastroesophageal reflux disease. The study data indicated that the percent time gastric pH was greater than 4 for patients taking Zegerid and was approximately 43 percent longer than patients treated with Protonix (p>0.001) and approximately 22 percent longer than patients treated with Prevacid (p=0.005).

• Semafore Pharmaceuticals Inc., of Indianapolis, initiated a Phase I trial of its lead PI3 kinase inhibitor, SF1126, in multiple myeloma. The single-agent, dose-escalating, multiple-dose trial will enroll up to 30 patients and is being conducted with the Multiple Myeloma Research Consortium. Semafore also is evaluating SF1126 in a Phase I solid tumor trial and expects to release the data this year.

• Trius Therapeutics Inc., of San Diego, started its first Phase I trial of TR-701, an antibacterial drug candidate in patients with serious Gram-positive bacterial infections, including those caused by Methicillin-resistant Staphylococcus aureus and other drug-resistant strains. The 88-subject trial will evaluate oral dosages of the drug and will compare its properties to those of Zyvox (linezolid, Pfizer Inc.), the only marketed oxazolidinone drug.

• VIA Pharmaceuticals, of San Francisco, said it has enrolled the first patient in its Phase II clinical trial that uses positron emission tomography with fluorodeoxyglucose tracer (FDG-PET) to measure the ability of VIA-2291, a small-molecule drug that targets inflammation in the blood vessel wall, to reduce vascular inflammation in treated patients. The FDG-PET trial is one of three concurrent, ongoing Phase II trials testing VIA-2291 in a variety of clinical settings, which are intended to provide new visibility to the role of inflammation in cardiovascular disease and the potential of VIA-2291 to address vascular inflammation, the firm said.

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