GenSpera Inc., of San Antonio, said the first patient has been treated in a Phase II trial of lead compound, G-202, in patients who have hepatocellular carcinoma (HCC). Data from the G-202 Phase Ib program in solid tumor patients showed the compound was well tolerated, with prolonged disease stabilization observed in several hepatocellular carcinoma patients who had previously progressed on sorafenib therapy. The company's technology platform combines plant-derived cytotoxin thapsigargin with a

Aeterna Zentaris Inc., of Quebec City, said the first patient was treated in its Phase II trial of targeted doxorubicin peptide conjugate AEZS-108 in chemotherapy-refractory triple-negative luteinizing hormone-releasing hormone receptor-positive metastatic breast cancer. Up to 74 patients will be involved, and the primary endpoint is median time of progression-free survival. Secondary endpoints include overall response rate and overall survival. Onyx Pharmaceuticals Inc., of South San

Mundipharma International Corp., of Cambridge, UK, said results from the StiL NHL-1 study published in The Lancet showed that a first-line treatment regimen of bendamustine plus Rituxan (rituximab, Biogen Idec Inc. and Roche AG) doubled progression-free survival compared with CHOP plus rituximab in newly diagnosed patients with indolent non-Hodgkin's lymphoma and mantle cell lymphoma (69.5 months vs. 31.2 months; p < 0.0001).

Advaxis Inc., of Princeton, N.J., said Brown University Oncology Research Group will coordinate a Phase I/II study of ADXS-HPV in 25 patients with human papillomavirus-associated anal cancer. The open-label trial will test the safety and effectiveness of ADXS-HPV when combined with standard chemotherapy and radiation treatment. The primary objectives include the evaluation of adverse events and the evaluation of six-month clinical response. BioMarin Pharmaceutical Inc., of Novato, Calif

Amicus Therapeutics Inc., of Cranbury, N.J., reported additional six-month (Stage 1) results from the first ongoing Phase III global registration study (Study 011) of investigational oral migalastat HCl monotherapy (150 mg, every-other-day) in males and females with Fabry disease who had genetic mutations identified as amenable to migalastat HCl. Stage 1 results were highlighted in an oral platform presentation at the Lysosomal Disease Network WORLD Symposium in Orlando, Fla. Study 011 consists

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

Vaximm AG, of Basel, Switzerland, said top-line data from its Phase I/II dose-escalation study of oral cancer vaccine VXM01 met key safety and tolerability endpoints. The study, VXM01-01-DE, enrolled 45 patients with inoperable pancreatic cancer at the Heidelberg (Germany) University Hospital. In addition to standard-of-care treatment, the patients received several doses of the therapeutic vaccine, which targets the tumor vasculature, or placebo. Results indicated the vaccine was safe and well

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

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

ArQule Inc., of Woburn, Mass., and Daiichi Sankyo Co. Ltd., of Tokyo, enrolled their first patient in a pivotal Phase III trial (METIV-HCC) of tivantinib (ARQ197) in hepatocellular carcinoma (HCC). The randomized, double-blind, placebo-controlled trial will enroll about 300 previously treated patients with MET-high inoperable HCC to receive tivantinib or placebo, with a primary endpoint of overall survival and a secondary endpoint of progression-free survival. (See BioWorld Today, Oct. 3, 2012

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