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.

Anacor Pharmaceuticals Inc., of Palo Alto, Calif., disclosed positive results from a Phase II safety, pharmacokinetics and efficacy trial of its boron-based phosphodiesterase-4 inhibitor, AN2728, in adolescents, ages 12 to 17, with mild to moderate atopic dermatitis. The 23-patient study showed that 74 percent of patients achieved an Investigator Static Global Assessment (ISGA) score of zero or 1 after four weeks of treatment, and 35 percent achieved an ISGA score of zero or 1 with a minimum

Iroko Pharmaceuticals LLC, of Philadelphia, reported that a Phase III study of submicron indomethacin for postsurgical pain met its primary endpoint of significant pain relief compared to placebo. The trial enrolled 462 patients, who were randomized to submicron indomethacin, celecoxib or placebo. Submicron indomethacin met that goal at all doses tested. The drug was developed using technology licensed to Iroko from iCeutical Pty Ltd., which alters the pharmacokinetic absorption properties of

Isis Pharmaceuticals Inc., of Carlsbad, Calif., reported that three drugs were found to be safe and well tolerated in separate Phase I studies. The drugs, ISIS-PTP1B(Rx), ISIS-GCGR(Rx), and ISIS-GCCR(Rx), are in development for indications related to metabolic disorders including Type II diabetes. ISIS-PTP1B(Rx) is designed to target protein tyrosine phosphatase-1B, ISIS-GCGR(Rx) is designed to target the glucagon receptor and ISIS-GCCR(Rx) is designed to target the glucocorticoid receptor

Johnson & Johnson, of New Brunswick, N.J., reported that galantamine gave a significantly lower mortality rate compared to placebo in a study of patients with severe Alzheimer's disease. The galantamine group also had significantly less cognitive decline after two years compared to the placebo group. The study was terminated early due to an imbalance of deaths between the treatment and placebo group, favoring the treatment group. The study enrolled 2,051 subjects at 127 sites in 13 countries

BioAlliance Pharma SA, of Paris, said the European Independent Board of Experts held its first meeting on the ReLive Phase III trial of Livatag (doxorubicin transdrug) in primary liver cancer and recommended continuing the study without modification. Cell Therapeutics Inc., of Seattle, said interim results from a cooperative group-sponsored Phase II trial of brostallicin in combination with cisplatin for the treatment of women with metastatic triple-negative breast cancer were presented at the

Pfizer Inc., of New York, said randomized Phase II data showed that PD-0332991 in combination with letrozole significantly extended progression-free survival (PFS) compared to letrozole alone in postmenopausal women with estrogen receptor-positive human epidermal growth factor receptor-1-negative locally advanced or metastatic breast cancer. Patients in the combination arm showed a median PFS of 26.1 months vs. 7.5 months in the letrozole-only arm, a statistically significant improvement (p < 0

UCB SA, of Brussels, Belgium, said a subgroup analysis of pooled data from three long-term open-label extension trials showed a consistent safety profile with epilepsy drug Vimpat (lacosamide), with the most commonly reported treatment-emergent adverse events being dizziness, headache and nasopharyngitis. Lacosamide exposure also was associated with a reduction in seizure frequency during the study period. A post-hoc analysis of pooled data from three Phase II/III studies suggested a greater