Cancer Research UK's Drug Development Office in London and partner AstraZeneca plc, of London, opened a three-armed study testing AZD0424 in a Phase I trial expected to recruit up to 30 patients, initially across all solid tumor types. The design of the trial later will be adapted, enabling it to separate into three separate personalized arms to test AZD0424 in different combinations alongside standard or other experimental treatments in specific patient populations. AZD0424 is designed to work

Cytos Biotechnology Ltd., of Zurich, Switzerland, said a technical issue was identified with the rubber stoppers used to close the injection vials containing clinical trial material for its Phase IIb study testing lead candidate CYT003 in allergic asthma. Cytos will replace that material with new material available at clinical sites in the second quarter. The firm expects overall guidance for the study, which will recruit about 360 adults with moderate to severe disease who are not sufficiently

Argos Therapeutics Inc., of Durham, N.C., said the first patients were enrolled in the ADAPT Phase III study testing the ability of AGS-003, a personalized dendritic cell-based immunotherapy, in combination with standard targeted therapy to extend overall survival vs. standard therapy alone in newly diagnosed patients with unfavorable risk metastatic renal cell carcinoma. About 450 patients will be enrolled. Secondary endpoints include progression-free survival, safety, overall response and

Neuralstem Inc., of Rockville, Md., said it has received FDA approval to start a Phase I trial of lead cell therapy candidate NSI-566 in chronic spinal cord injury patients. The open-label study, based on an investigational new drug application filed in 2010, will enroll up to eight patients with thoracic spinal cord injuries (T2-T12), who have an American Spinal Injury (AIS) Association A level of impairment, between one and two years after injury. The primary objective is to determine the

KaloBios Pharmaceuticals Inc., of South San Francisco, said it started dosing in a Phase II study testing KB001-A, its anti-PcrV Humaneered pegylated monoclonal antibody fragment, in chronic Pseudomonas aeruginosa infection in cystic fibrosis patients. The 16-week study will enroll 180 patients to receive repeated doses of KB001-A, with the primary endpoint defined as the time to need for antibiotics to treat worsening of respiratory tract signs and symptoms. Secondary endpoints will include

Novartis AG, of Basel, Switzerland, said data published in The Lancet showed that patients on mTOR inhibitor Afinitor (everolimus) tablets with noncancerous kidney tumors known as renal angiomyolipomas associated with tuberous sclerosis complex experienced a significant reduction in tumor size and the absence of tumor progression. In the study, 42 percent of patients on everolimus experienced an angiomyolipoma response vs. 0 percent on placebo.

BioTime Inc., of Alameda, Calif., said it submitted a clinical investigation protocol to European regulatory authorities for approval to initiate studies for its Renevia stem cell delivery platform. Renevia, a member of the firm's HyStem family of hydrogels, is a formulation designed to mimic human extracellular matrix. Studies are expected to start in the second quarter. GeoVax Labs Inc., of Atlanta, said it completed enrollment in a nine-patient Phase I/II trial testing its DNA/MVA vaccine

TapImmune Inc., of Seattle, said an interim safety analysis of the first five breast cancer patients treated with HER2/neu Class II antigens did not show any serious adverse events, so treatment of the remaining 17 patients in the Phase I study at the Mayo Clinic will progress

Advanced Cell Technology Inc., of Marlborough, Mass., reported evidence of engraftment of transplanted human embryonic stem cell-derived retinal pigment epithelial cells and visual acuity gain in a Phase I/II trial in Stargardt's macular dystrophy (SMD) and dry age-related macular degeneration (AMD). Patients were treated over 18 months, and no safety issues were raised. The company also observed persisting engraftment in more recent SMD and AMD patients, as well. Intercept Pharmaceuticals Inc

Baxter International Inc., of Deerfield, Ill., reported pivotal Phase III results showing that routine prophylaxis vs. on-demand treatment of FEIBA NF (anti-inhibitor coagulant complex [nanofiltered and vapor-heated]) in patients with hemophilia A or B reduced median annual bleed rate. Results showed the annual bleed rate dropped from 28.7 percent during FEIBA NF on-demand treatment to 7.9 percent during FEIBA NF prophylactic treatment, marking a 72.5 percent reduction. Baxter intends to file a

Achillion Pharmaceuticals Inc., of New Haven, Conn., said it completed enrollment in a pilot Phase IIa trial evaluating ACH-3102, a second-generation pan-genotypic NS5A inhibitor, in combination with ribavirin in patients with chronic genotype 1b hepatitis C virus (HCV) infection. The initial cohort enrolled eight patients to receive 12 weeks of combination treatment. To date, five patients have completed four weeks of treatment, and ACH-3102 has been well tolerated, with no serious adverse

Baxter International Inc., of Deerfield, Ill., said it submitted an investigational new drug application for hemophilia A candidate BAX 855, a full-length longer-acting recombinant Factor VIII developed to increase the half-life of ADVATE (Antihemophilic Factor [Recombinant] Plasma/Albumin-Free Method). Baxter expects to start enrolling adults in a Phase II/III study later this quarter. Boehringer Ingelheim GmbH, of Ingelheim, Germany, and Eli Lilly and Co., of Indianapolis, said top-line

Amicus Therapeutics Inc., of Cranbury, N.J., reported positive preliminary results from all four dose cohorts in its Phase II study (Study 010) evaluating the safety and pharmacokinetics of chaperone AT2220 (duvoglustat HCl) co-administered with enzyme replacement therapy (ERT) in Pompe disease (Myozyme and Lumizyme [alglucosidase alfa, or recombinant human GAA enzyme (rhGAA), Sanofi SA/Genzyme Corp.]). Myozyme and Lumizyme are the only approved treatments for Pompe disease, in which deficient

Alkermes plc, of Dublin, Ireland, reported positive top-line results from a Phase I study of antipsychotic candidate ALKS 3831, which combines a drug molecule ALKS 33 with Zyprexa (olanzapine, Eli Lilly and Co.). ALKS 3831, in development for schizophrenia, is designed to attenuate the antipsychotic-related metabolic side effect of weight gain. The study compared the mean change from baseline in body weight in 106 healthy volunteers following three weeks of once-daily, oral administration of

Astex Pharmaceuticals Inc., of Dublin, Calif., said it started a Phase II study testing SGI-110 in advanced hepatocellular carcinoma (HCC) patients who failed prior treatment with Nexavar (sorafenib, Onyx Pharmaceuticals Inc. and Bayer AG). The trial will have two stages: a 15-patient Part A portion to evaluate safety, disease control and determine methylation status of certain tumor suppressor genes known to be involved in HCC, followed by a Part B portion, which will enroll 31 additional

Aeterna Zentaris Inc., of Quebec City, agreed with the FDA on a special protocol assessment for its Phase III registration trial of AEZS-108 in endometrial cancer. The open-label, randomized trial will be carried out in North America and Europe, and will compare AEZS-108 with dosorubicin as second-line therapy in endometrial cancer. About 500 patients will be enrolled. Synergy Pharmaceuticals Inc., of New York, completed a Phase I single-ascending dose trial of SP-333 for ulcerative colitis

Synergy Pharmaceuticals Inc., of New York, said dosing has commenced in a Phase IIb trial of plecanatide to treat patients with constipation-predominant irritable bowel syndrome (IBS-C). The 350-patient trial is being conducted at 70 sites in the U.S. Participants with IBS-C will be treated with one of four doses of plecanatide (0.3, 1, 3 or 9 mg) or placebo, taken once daily over a period of 12 weeks. The trial will measure the mean change in complete spontaneous bowel movements (CSBM) over

Celator Pharmaceuticals Inc., of Princeton, N.J., treated its first patient in a Phase III, randomized, open-label trial of CPX-351 (cytarabine/daunorubicin) liposome injection compared to conventional cytarabine and daunorubicin for first-line therapy of acute myeloid leukemia. The trial is being carried out at about 50 centers in the U.S. and Canada in partnership with the Leukemia and Lymphoma Society, which has previously supported the development of CPX-351. The primary endpoint of the

Ironwood Pharmaceuticals Inc., of Cambridge, Mass., told licensor Bionomics Ltd., of Adelaide, Australia, that the planned Phase I trial of the investigational anti-anxiety drug candidate IW-2143 (BNC210) has begun in the U.S. The trial is designed to assess the safety and pharmacokinetics of IW-2143 in healthy volunteers, using single and multi-dose administration. (See BioWorld Today, Jan. 6, 2012.) Coronado Biosciences Inc., of Burlington, Mass., entered an agreement with the National