ACCESS PHARMACEUTICALS, INC. (OTC Bulletin Board: ACCP) announced that it initiated an internal pre-licensing program to confirm the utility of its proprietary Cobalamin (vitamin B12) platform technology for targeted delivery of siRNA therapies. The program is considered important because, despite the widely publicized potential of RNA therapy, researchers up to now have been stymied in their efforts to design a pharmaceutical product that efficiently transports siRNA therapeutics into the cells they are designed to inhibit or kill. Access has multiple programs ongoing around use of its Cobalamin technology to facilitate oral absorption of pharmaceuticals, including previously announced collaborations with potential pharma and biotech partners.
Protalix BioTherapeutics, Inc. (NYSE-Amex: PLX), announced that additional data from the Company's pivotal Phase III clinical trial of taliglucerase alfa in patients with Gaucher disease was presented at the Annual Meeting of the Lysosomal Disease Network: WORLD Symposium 2010 in Miami, Florida, during an oral session titled, "Novel Enzyme Replacement Therapy for Gaucher Disease: Phase III Pivotal Clinical Trial with Plant Cell Expressed Recombinant Glucocerebrosidase (prGCD) - taliglucerase alfa." The oral presentation was made by Hanna Rosenbaum M.D., Director of Hematology Day Care Unit, RAMBAM Medical Center, Haifa, Israel, and study investigator.
Amicus Therapeutics Presents Positive Data Update From Phase 2 Extension Study Of Amigal TM For Fabry Disease
Amicus Therapeutics (Nasdaq: FOLD) announced additional positive preliminary data from its ongoing Phase 2 extension study of its investigational drug Amigal™ (migalastat HCl) for Fabry disease at the Lysosomal Disease Network WORLD Symposium in Miami, Florida. The Company also presented encouraging data from preclinical studies evaluating the combination of pharmacological chaperones and enzyme replacement therapy (ERT) for Fabry disease and Pompe disease as well as from preclinical studies examining the use of pharmacological chaperones for the treatment of Parkinson's disease. Phase 2 Extension Study Overview Twenty-six subjects completed either 12 or 24 weeks of treatment with migalastat HCl during the initial Phase 2 studies and twenty-three subjects enrolled in a separate, voluntary long-term extension study designed to evaluate the long-term safety and efficacy of migalastat HCl.
Shire Presents Positive Efficacy And Safety Data For Velaglucerase Alfa In Treatment Of Naive Patients With Type 1 Gaucher Disease
Shire plc (LSE: SHP, NASDAQ: SHPGY), the global specialty biopharmaceutical company, presented positive results from its first Phase III study (TKT 032) evaluating safety and efficacy of velaglucerase alfa, its investigational enzyme replacement therapy for the treatment of Type 1 Gaucher disease. The data were presented in an oral presentation at the Lysosomal Disease Network (LDN) World Symposium in Miami, Florida. Data from a pediatric subgroup of this study and five year follow-up results from a long-term Phase I/II extension study (TKT025 EXT) conducted in adults were also reported and add to the available data on the long-term safety and efficacy of velaglucerase alfa in patients with Type 1 Gaucher disease.
Many American women are prescribed estrogen to combat the negative effects of menopause, such as bone loss and mood swings. Now, new evidence from a Tel Aviv University study suggests that hormone replacement therapy might also protect them - and younger women - from schizophrenia as well. Prof. Ina Weiner of Tel Aviv University's Department of Psychology and her doctoral student Michal Arad have reported findings suggesting that restoring normal levels of estrogen may work as a protective agent in menopausal women vulnerable to schizophrenia. Their work, based on an animal model of menopausal psychosis, was recently reported in the journal Psychopharmacology.
A team of scientists from The Scripps Research Institute has found that a specific stress hormone, the corticotropin-releasing factor (CRF), is key to the development and maintenance of alcohol dependence in animal models. Chemically blocking the stress factor also blocked the signs and symptoms of addiction, suggesting a potentially promising area for future drug development. The article, the culmination of more than six years of research, will appear in an upcoming print edition of the journal Biological Psychiatry. "I'm excited about this study, " said Associate Professor Marisa Roberto, who led the research. "It represents an important step in understanding how the brain changes when it moves from a normal to an alcohol-dependent state.