Emmaus Medical Inc., of Torrance, Calif., completed patient enrollment in a Phase III trial of L-glutamine for sickle cell disease. The randomized, double-blind, placebo-controlled trial will assess safety and efficacy of L-glutamine for sickle cell anemia and beta0-thalassemia. The trial has enrolled 225 patients at 31 sites in the U.S. (See BioWorld Today, Oct. 12, 2012.) InnaVirVax, of Evry, France, said interim results of a Phase I/IIa trial showed good safety and tolerability for the VAC
H. Lundbeck A/S, of Copenhagen, Demark, presented three-year vision data at the American Epilepsy Society meeting in San Diego. The FDA requires a risk evaluation and mitigation strategy for Sabril due to the risk of treatment-induced permanent vision loss, and the company offers an ongoing patient registry. Of the total patients enrolled in the registry, 2,676 had infantile spasms and 1,354 had refractory complex partial seizures. Because of the nature of the registry and vision testing
Theravectys SAS, of Paris, was granted authorization by the National Security Agency for Medicines and Health Products in France and the Federal Agency for Medicines and Health Products in Belgium to launch a Phase I/II trial with its anti-HIV therapeutic vaccine candidate based on lentiviral vector technology.
Aduro BioTech Inc., of Berkeley, Calif., said it completed enrollment in a Phase II trial of CRS-207 and GVAX Pancreas Cancer Vaccine eight months ahead of schedule. The vaccine regimen was given to patients with metastatic pancreatic cancer, and it was well tolerated with no unexpected adverse events. An interim analysis of safety and efficacy will be reported in the first quarter of 2013. Ariad Pharmaceuticals Inc., of Cambridge, Mass., published results of a Phase I trial of ponatinib in
Bayer AG, of Leverkusen, Germany, said its BAY 86-6150 is being investigated in a Phase II/III study, dubbed TRUST (TReatment with Unique recombinant rFVIIa STudy), and the recruitment of the first cohort is now complete. TRUST is designed to test the recombinant Factor VIIa protein in patients with hemophilia A or hemophilia B who have developed neutralizing antibodies. The study is composed of two parts: The first will involve sequential dose escalation for assessing dose response and
Advanced Cell Technology Inc., of Marlborough, Mass., said it treated three additional patients in its U.S. trials testing retinal pigment epithelial cells derived from human embryonic stem cells. A Phase I/II study is in patients with dry age-related macular degeneration, while a separate study is testing the cells in Stargardt's macular dystrophy. Anthera Pharmaceuticals Inc., of Hayward, Calif., reported additional data from its Phase IIb PEARL-SC study at the Asian Lupus Summit meeting in
Bayer AG, of Leverkusen, Germany, said results of two pivotal Phase III studies, published in The Lancet, demonstrated the efficacy of regorafenib in patients with metastatic colorectal cancer or gastrointestinal stromal tumor who have exhausted all other treatment options. Data from the CORRECT study showed regorafenib plus best supportive care significantly improved overall survival and progression-free survival compared to placebo plus BSC (6.4 months vs. 5 months and 1.9 months vs. 1.7
Accera Inc., of Broomfield, Colo., started a clinical efficacy trial examining the effects of AC-1204 in patients with mild-to-moderate Alzheimer's disease (AD). AC-1204 is a formulation of caprylic triglyceride designed to improve cognitive function in mild to moderate AD. The randomized, double-blind, placebo-controlled, parallel-group, multicenter trial will be conducted at 60 sites in the U.S. and is expected to enroll more than 400 patients. The study will evaluate the effects of once
The Journal of the American Academy of Dermatology published results from two pivotal trials by Valeant Pharmaceuticals International Inc., of Montreal, of efinaconazole 10 percent topical solution for onychomycosis, a nail infection caused by fungi. The primary endpoint of the randomized, double-blind, vehicle-controlled study enrolling 1,655 subjects was complete cure rate at week 52. In Study 1, 17.8 percent of subjects given efinaconazole achieved complete cure, compared to 3.3 percent of
MediciNova Inc., of San Diego, started enrolling a Phase IIa trial testing MN-166 (ibudilast) in prescription opioid or heroin abusers. The trial will be conducted at Columbia University and the New York State Psychiatric Institute and is funded by the National Institutes for Drug Abuse. MN-166 is a first-in-class, orally bioavailable small-molecule glial attenuator designed to suppress pro-inflammatory cytokines IL-1 beta, TNF alpha and IL-6, and may up-regulate the anti-inflammatory cytokine
Aeterna Zentaris Inc., of Quebec City, said perifosine, its oral AKT inhibitor, combined with temsirolimus was well tolerated in an investigator-driven Phase I trial in recurrent or progression malignant glioma. The study, which involves 32 patients with recurrent or progression glioma, anaplastic astrocytoma, anaplastic oligodendroglioma and transformed low-grade gliomas, currently is accruing to dose level five after two dose-limiting toxicities in the dose level seven group and dose level
Pfizer Inc., of New York, said top-line data from a Phase III study testing pregabalin controlled-release (CR) formulation in patients with fibromyalgia indicated that the product had a statistically significant positive effect compared to placebo in the primary endpoint, defined as time to loss of therapeutic response. The trial is the second of three Phase III studies testing the CR formulation for once-daily therapy. The first study in partial onset seizures did not meet its primary endpoint
Pfizer Inc., of New York, said top-line results from a Phase III study testing the 165-mg dose and the 330-mg dose of pregabalin controlled-release (CR) formulation in adults with partial onset seizures showed that the trial did not meet its primary endpoint comparing the change in seizure frequency to placebo, although both doses were well tolerated. The study is one of three Phase III trials designed to test pregabalin CR for use as a once-daily therapy. The other two studies are testing the
Adaptimmune Ltd., of Oxford, UK, said abstracts published in Blood reported encouraging preliminary results from an early phase study using patients' own T cells that have been genetically altered to attack multiple myeloma cells. The single-arm, open-label extension study is designed to test the safety, bioactivity and antitumor effects of the infusion of those T cells, which were genetically altered to express cancer testis antigens NY-ESO-1 and LAGE-1. The initial six patients have completed
CytRx Corp., of Los Angeles, presented results from a Phase Ib/II study of tumor-targeting doxorubicin conjugate aldoxorubicin (formerly INNO-206) in advanced soft-tissue sarcoma at the Connective Tissue Oncology Society meeting in Prague, Czech Republic. Patients treated with aldoxorubicin were delivered doxorubicin in a cumulative dose of more than 3.5 times the peak cumulative dose of standard doxorubicin with no observed cardiac toxicities. Trial data showed clinical benefit, defined as
Acceleron Pharma Inc., of Cambridge, Mass., said the Gynecologic Oncology Group started a Phase II study testing dalantercept, a protein therapeutic designed to target the activin receptor-like kinase 1 pathway, in patients with recurrent or persistent endometrial cancer. The open-label study will measure progression-free survival and objective tumor response. The study is receiving financial support from the National Cancer Institute. Anthera Pharmaceuticals Inc., of Hayward, Calif., said an
Merck & Co. Inc., of Whitehouse Station, N.J., presented data from a post-hoc subgroup analysis of three studies, which found that integrase inhibitor Isentress (raltegravir) demonstrated consistent long-term viral suppression and a well-established safety profile in adults co-infected with HIV-1 and hepatitis C virus and/or hepatitis B virus, compared to adults with HIV-1, at 240 weeks. Data were presented at the International Congress on HIV and Drug Therapy in HIV Infection meeting in
Bayer Inc., of Deerfield, Ill., and the Population Health Research Institute said they started the COMPASS trial, the largest clinical study to date of oral anticoagulant rivaroxaban. The Phase III study will investigate the prevention of major adverse cardiac events, including cardiovascular death, myocardial infarction and stroke in patients with coronary artery disease or peripheral artery disease. About 20,000 patients will be enrolled.
Achillion Pharmaceuticals Inc., of New Haven, Conn., said that it completed a drug-drug interaction study of sovaprevir and ACH-3102 in 24 healthy volunteers, showing no interaction and that the compounds were safe and well tolerated. The drugs are being developed for hepatitis C virus. The company also reported additional proof-of-concept data from a Phase Ib trial of ACH-2684, for hepatitis C virus. In chronic genotype 1 patients, there was a mean maximum 3.67 log10 reduction in HCV RNA
Merck and Co. Inc., of Whitehouse Station, N.J., presented early interim results from a single-arm, open-label Phase Ib study of MK-3475, its investigational immune-modulating therapy, in advanced melanoma. Based on data for 85 of 132 patients enrolled in the study to date, 43 patients (51 percent) showed an objective antitumor response and eight patients (9 percent) showed a complete response at or after the 12-week assessment. Of 27 patients previously treated with ipilimumab (Yervoy, Bristol
Emmaus Medical Inc., of Torrance, Calif., completed patient enrollment in a Phase III trial of L-glutamine for sickle cell disease. The randomized, double-blind, placebo-controlled trial will assess safety and efficacy of L-glutamine for sickle cell anemia and beta0-thalassemia. The trial has enrolled 225 patients at 31 sites in the U.S. (See BioWorld Today, Oct. 12, 2012.) InnaVirVax, of Evry, France, said interim results of a Phase I/IIa trial showed good safety and tolerability for the VAC