Adventrx Pharmaceuticals Inc., of San Diego, said it met with the FDA to review plans for ANX-188 (purified poloxamer 188), its treatment for sickle cell patients experiencing vaso-occlusive crisis. The firm said an understanding regarding certain matters for the Phase III trial design was reached, though additional interaction with the agency will be needed over the coming weeks and months to finalize plans. Adventrx is aiming to start the Phase III study in 2012.
Amicus Therapeutics Inc., of Cranbury, N.J., said it treated the first patient in an open-label Phase II drug-drug interaction study of AT2220 (duvoglustat HCl) co-administered with enzyme replacement therapy in Pompe disease patients. The purpose of the study is to evaluate whether AT2220, an oral pharmacological chaperone candidate, can be safely given with ERT Myozyme/Lumizyme (alglucosidase alfa, Sanofi SA), the only approved therapy for Pompe disease. About 16 patients who have been on stable dose and regimen of ERT for at least three months will be enrolled, and the study will measure plasma enzyme activity and protein levels during each infusion.
Anthera Pharmaceuticals Inc., of Hayward, Calif., reported interim biomarker data from the Phase IIb PEARL-SC study in systemic lupus erythematosus, which showed that weekly and monthly subcutaneous doses of blisibimod resulted in statistically significant reductions of B cells. The company remains blinded to primary efficacy data, and final results are expected in the second quarter of 2012. Blisibimod is a peptibody drug designed to act as a broad inhibitor of B-cell activating factor.
Auxilium Pharmaceuticals Inc., of Malvern, Pa., said the first patient was dosed in its Phase IIa trial of Xiaflex (collagenase clostridium histolyticum) in idiopathic adhesive capsulitis of the shoulder, also known as frozen shoulder syndrome. The trial is expected to enroll about 50 adults and will assess the drug's safety and efficacy. Top-line data are anticipated in the first half of 2013. Xiaflex previously gained approval in Dupuytren's contracture.
Ceregene Inc., of San Diego, completed enrollment in a Phase IIb trial of CERE-120, a gene therapy product in development for Parkinson's disease. The trial enrolled 51 patients at 11 medical centers in the U.S. and will evaluate motor function at 15 months to 24 months after treatment. The company expects to report data from the trial in the first quarter of 2013.
DiaMedica Inc., of Winnipeg, Manitoba, said preclinical data from an independently conducted study showed chronic administration of DM-204 prevented disease progression within an in vivo Type II diabetes model. Treatment with DM-204 resulted in statistically significant lower mean differences compared to untreated animals after 21 days for HbA1c levels, fasting blood glucose levels and systolic blood pressure levels. Data were presented at the Windhover Therapeutic Area Partnership Conference in Boston.
Idera Pharmaceuticals Inc., of Cambridge, Mass., said it was cleared by the FDA to proceed with a Phase II trial of IMO-3100, a dual antagonist of Toll-like receptor (TLR) 7 and TLR9, in patients with psoriasis based on trial protocol submitted in October. The study is expected to start in the first half of 2012.
Merrimack Pharmaceuticals Inc., of Cambridge, Mass., dosed its first patient in a Phase I trial of MM-121, a human monoclonal antibody targeting ErbB3 for solid tumors. Merrimack is developing the product under a 2009 licensing agreement with Paris-based Sanofi SA. The trial will evaluate the safety and pharmacokinetics of MM-121 in combination with chemotherapy regimens.
Santarus Inc., of San Diego, will present new data pertaining to its investigational drug Uceris (budesonide) for ulcerative colitis at a poster session at the Crohn's and Colitis Foundation's Clinical Research Conference in Hollywood, Fla. The data showed that out of 60 patients enrolled in the study, the rate of remission was 25 percent, clinical improvement was 26.7 percent, endoscopic improvement was 40 percent and symptom resolution was 33.3 percent.