Amicus Therapeutics Inc., of Cranbury, N.J., reported positive results from clinical and preclinical studies of the pharmacological chaperone AT2220 (duvoglustat HCl) in combination with ERT for Pompe disease at the Lysosomal Disease Network World Symposium. The results from a Phase II study established human proof-of-concept that co-administration of AT2220 just prior to infusing ERT (Myozyme/Lumizyme, or rhGAA enzymes) increases GAA enzyme activity in muscle tissue compared to ERT alone

Ablynx NV, of Ghent, Belgium, reported efficacy and safety data for its anti-IL-6R nanobody, ALX-0061, at the 24-week final analysis of the Phase II part of a Phase I/II study in patients with moderately to severely active rheumatoid arthritis on a stable background of methotrexate. The Phase II data showed ACR20, ACR50 and ACR70 scores of up to 100 percent, 75 percent and 63 percent, respectively, for patients who were consistently dosed at 3mg/kg Q4W over 24 weeks. At all doses tested, ALX

Janssen Research & Development LLC, of Titusville, N.J., a unit of Johnson & Johnson, said updated Phase III results showed Zytiga (abiraterone acetate) plus prednisone continued to provide statistically significant improvements in disease progression compared to placebo plus prednisone, and longer overall survival in men with metastatic castration-resistant prostate cancer. The randomized, multicenter, placebo-controlled study also demonstrated statistically significant improvement compared to

Critical Pharmaceuticals Ltd., of Nottingham, UK, completed a second clinical study of CP024, a growth hormone nasal spray in development for growth disorders in children and adults. The results showed that CP024 was safe and well tolerated with highly reproducible pharmacokinetics. The drug strongly induced production of insulin-like growth factor, a principal mediator of growth hormone activity. Critical Pharmaceuticals said that the trial provided proof of concept for the product

Advanced Cell Technology Inc. (ACT), of Marlborough, Mass., said its clinical partner, the University of California, Los Angeles (UCLA), received FDA approval for its investigational new drug application. Steven Schwartz, professor of ophthalmology at the David Geffen School of Medicine at UCLA and retina division chief at UCLA's Jules Stein Eye Institute, plans to lead a Phase I/II study using ACT's retinal pigment epithelial (RPE) cells, derived from human embryonic stem cells (hESC), to

Biogen Idec Inc., of Weston, Mass., and Swedish Orphan Biovitrum, of Stockholm, Sweden, reported data confirming that investigational recombinant factor VIII Fc (rFVIIIFc) fusion protein and factor IX Fc (rFVIXc) fusion protein gave long-lasting protection from bleeding with fewer injections than the standard of care for hemophilia. In A-LONG, patients with hemophilia A maintained low bleeding rates with once-to-twice weekly prophylactic injections of rFVIIIFc. In B-LONG, prophylactic

Pozen Inc., of Chapel Hill, N.C., reported results from two Phase III studies of PA32540 in ischemic stroke showing that compared to EC-ASA (aspirin), treatment with PA32540 significantly reduced the rate of endoscopic gastroduodenal ulcers (2.0 percent vs. 12.4 percent; p = 0.005). The rate of adjudicated major adverse cardiac events was similar in both groups. Adverse GI events commonly lead to discontinuation of aspirin therapy, which can increase the risk of adverse cardiovascular and

Eli Lilly and Co., of Indianapolis, halted its Phase III rheumatoid arthritis (RA) program for tabalumab, an anti-B cell activating factor monoclonal antibody, for lack of efficacy. The company said the tabalumab Phase III ILLUMINATE program in systemic lupus erythematosus is continuing. In December 2012, Lilly discontinued the Phase III RA registration study FLEX-M for lack of efficacy. FLEX-M was investigating tabalumab in patients with moderate to severe RA who had an inadequate response to

Genelux Corp., of San Diego, said researchers at Memorial Sloan Kettering Cancer Center in New York treated the first patient in a Phase I trial of GL-ONC1 in malignant pleural effusion, a complication that occurs in about 30 percent of lung cancers. The safety and dose-escalation trial will test GL-ONC1 as administered intrapleurally as a single-agent therapy. The primary goal will be to establish a recommended dose, while secondary objectives include the feasibility, safety and tolerability

Acceleron Pharma Inc., of Cambridge, Mass., said it started a Phase II study testing dalantercept (ACE-041), an angiogenesis inhibitor designed to target the activin receptor-like kinase pathway, in combination with Inlyta (axitinib, Pfizer Inc.), a vascular endothelial growth factor receptor tyrosine kinase inhibitor, in patients with metastatic renal cell carcinoma. The two-part study will feature a dose-escalation portion testing the drugs in combination as a second-line treatment, while the

Teva Pharmaceutical Industries Ltd., of Jerusalem, said it enrolled the first patient in the Phase IV MODERATO study to test Azilect (rasagiline tablets) on cognitive function in adults with mild cognitive impairment in Parkinson's disease. The study will measure cognitive function using the Scales for Outcomes of Parkinson's Disease-Cognition after 24 weeks of treatment and will enroll about 170 patients.

Tekmira Pharmaceuticals Corp., of Vancouver, British Columbia, said that complete results from a Phase I trial of its RNAi therapeutic, ALN-VSP have been published. The company said that the results validate longer-term safety and tolerability of its LNP delivery platform. Alnylam Inc., of Cambridge, Mass., licensed the LNP technology from Tekmira, and is developing several products under that license. Tekmira is due a $5 million payment related to initiation of clinical trials for ALN-VSP in

Adventrx Pharmaceuticals Inc., of San Diego, said it started patient recruitment in its pivotal Phase III study of ANX-188 (purified poloxamer 188) in sickle cell disease. The EPIC study is designed to demonstrate that ANX-188 reduces the duration of vaso-occlusive crisis in patients with sickle cell disease. The duration of vaso-occlusive crisis will be measured from the time a subject is randomized to the time at which the subject receives the last dose of parenteral opioid analgesic for the

Acceleron Pharma Inc., of Cambridge, Mass., said it started a Phase II study testing ACE-536 to treat anemia in patients with myelodysplastic syndromes (MDS). The trial is designed to test the drug's safety and efficacy in patients with low-risk or intermediate-1 risk MDS, with efficacy measures including increases in hemoglobin levels, reduction in red blood cell transfusion burden and other hematologic parameters, as well as biomarkers of iron and bone metabolism. ACE-536, a modified Type II

Amgen Inc., of Thousand Oaks, Calif., reported results from the Pegfilgrastim and anti-VEGF evaluation study (PAVES), a Phase III trial, which evaluated Neulasta (pegfilgrastim) in 845 patients receiving FOLFOX or FOLFIRI and bevacizumab for first-line treatment of locally advanced or metastatic colorectal cancer. The study met its primary endpoint, with Neulasta significantly reducing the incidence of febrile neutropenia. The incidence of grade 3 or 4 febrile neutropenia in patients receiving

Seattle Genetics Inc., of Bothell, Wash., said interim results from a Phase I trial of ASG-5ME in metastatic pancreatic ductal adenocarcinoma identified the maximum-tolerated dose for weekly administration, demonstrated tolerability and provided preliminary evidence for antitumor activity. The best response for 18 patients treated at 1.2 mg/kg weekly included one patient (6 percent) who achieved a partial response, six patients (33 percent) with stable disease and four (22 percent) with

Pfizer Inc., of New York, reported data from a Phase IV study showed that Toviaz (fesoterodine fumarate) 8 mg once daily reduced urge urinary incontinence episodes in patients with overactive bladder per 24 hours from baseline to week 12, which was statistically significantly more than Toviaz 4 mg. The efficacy of both doses was statistically significant vs. placebo. In separate news, Pfizer reported data from a Phase IV study testing Pristiq (desvenlafaxine) extended-release tablets, showing

EntreMed Inc., of Rockville, Md., said it started a Phase II study, titled "A Phase II Study of Oral ENMD-2076 Administered to Patients with Advanced/Metastatic Soft Tissue Sarcoma." ENMD-2076 is designed to inhibit both Aurora kinase A and angiogenesis. Shares of EntreMed (NASDAQ:ENMD) gained 28 cents, or 15.4 percent, to close Wednesday at $2.10. Santarus Inc., of San Diego, and VeroScience LLC, of Tiverton, R.I., said data from a pivotal study testing Cycloset (bromocriptine mesylate

Advanced Cell Technology Inc., of Marlborough, Mass., said it is amending the patient treatment protocol for the remainder of its Phase I trials testing human embryonic cell-derived retinal pigment epithelial cells in Stargardt's macular dystrophy and dry age-related macular degeneration to allow patients with better vision, defined as a visual acuity of 20/100 to enroll in the studies. The company said treating patients earlier in the course of the diseases should produce a more significant