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
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
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
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
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
ArQule Inc., of Woburn, Mass., began patient dosing in a Phase I trial with ARQ 087, an oral multikinase inhibitor with pan-FGFR (fibroblast growth factor receptor) activity. The primary objective of the trial is to determine safety, tolerability and the recommended Phase II dose. Patients with metastatic solid tumors who are refractory to available therapies or for whom no standard systemic therapy exists will be enrolled. The number of patients expected to be enrolled will depend on the
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.
Bristol-Myers Squibb Co., of New York, and Pfizer Inc., also of New York, disclosed data from a Phase III study testing Eliquis (apixaban) for the prevention of recurrent venous thromboembolism (VTE). Data from the year-long trial, published in the New England Journal of Medicine, showed that treatment with Eliquis significantly reduced the recurrence of VTE and death from any cause compared to those treated with placebo.
Active Biotech AB, of Lund, Sweden, and Ipsen SA, of Paris, said the Phase III study of tasquinimod in prostate cancer is fully enrolled, with more than 1,200 randomized patients. That achievement triggers a €10 million (US$12.9 million) milestone payment from Ipsen to Active Biotech. The study is testing tasquinimod in patients with metastatic castrate-resistant prostate cancer, with radiological progression-free survival as the primary endpoint and overall survival as a secondary endpoint
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
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
BrainCells Inc., of San Diego, said it completed a Phase I multiple-ascending-dose study of BCI-838, an oral prodrug of the company's Group II mGluR2/3 antagonist, BCI-632. BCI-838 showed a favorable safety profile, with no treatment-related trends in clinical laboratory results, vital sign measurements, 12 lead ECG results or physical examination findings, and the drug was well tolerated up to the dose levels predicted for efficacy. The company will advance the compound into a proof-of-concept
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
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