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

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

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

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

Acura Pharmaceuticals Inc., of Palatine, Ill., said it filed an investigational new drug application with the FDA for clinical testing of its hydrocodone bitartrate with acetaminophen formulated using its Aversion Technology. Acura intends to complete clinical testing for a new drug submission via the 505(b)(2) pathway in the first half of 2014. BioAlliance Pharma SA, of Paris, said the European Independent Board of Experts held its first meeting on the ReLive Phase III trial evaluating the

Merck and Co Inc., of Whitehouse Station, N.J., reported that its large study of Tredaptive (extended-release niacin/laropiprant) missed its primary endpoint. The trial enrolled 25,673 patients at high risk for cardiovascular events, and followed them for a median of 3.9 years while they received Tredaptive therapy plus statin therapy or statin therapy alone. The study found that the addition of Tredaptive did not significantly reduce the risk of coronary deaths, nonfatal heart attacks, strokes

EyeGate Pharma Inc., of Waltham, Mass., said it enrolled the last patient in a 200-subject pivotal Phase III study of EGP-437 (dexamethasone phosphate ophthalmic solution) in patients with anterior uveitis. The study's objective is to test ocular iontophoresis with EGP-437 vs. treatment with topically applied prednisolone acetate ophthalmic suspension eyedrops. The primary endpoint will measure the proportion of patients with anterior chamber cell count of zero at day 14. Top-line data are

Acasti Pharma Inc., of Laval, Quebec, said it achieved significant progress in two ongoing clinical studies with CaPre for lipid management. The registrational Phase II double-blind, placebo-controlled clinical study has completed the first of two interim analyses. The second Phase II open-label study should be completed by the end of the first quarter of 2013. It has been delayed due to the need for further patient recruitment after the approved clinical trial amendment to add an additional 4

AcelRx Pharmaceuticals Inc., of Redwood City, Calif., reported top-line data from a Phase III trial demonstrating that the Sufentanil NanoTab PCA System was noninferior (p < 0.001) to intravenous patient-controlled analgesia with morphine for the primary endpoint of Patient Global Assessment (PGA) of method of pain control over the 48-hour study period as determined by the combined percentage of patients with PGA ratings of "good" or "excellent" (78.5 percent vs. 66.1 percent, respectively

ARCA biopharma Inc., of Broomfield, Colo., said a paper published in the European Journal of Heart Failure discusses the post-hoc analyses of data from the Phase III study, designated BEST (Beta-Blocker Evaluation of Survival Trial), testing Gencaro (bucindolo hydrochloride) in heart failure. Data demonstrated that patients with established atrial fibrillation (AF) receiving Gencaro in the trial had improvements in heart failure clinical endpoints, and those receiving Gencaro were more likely

Eli Lilly and Co., of Indianapolis, provided an update on the next steps planned for solanezumab, its Phase III monoclonal antibody being studied as a potential therapy for patients with mild Alzheimer's disease. Following discussions with regulators in the U.S., Europe and Canada, Lilly plans to conduct an additional Phase III study of solanezumab in patients with the condition, but other details of the study, which will begin no later than the third quarter of next year, are yet to come.