Amicus Therapeutics, of Cranbury, N.J., said the FDA indicated that the data from the completed Phase II studies of Amigal, an oral therapy for Fabry disease, support the start of Phase III development and agreed that Amigal meets the criteria to be considered for accelerated approval. Furthermore, the FDA did not oppose the use of a surrogate primary endpoint, pending further discussion and final agreement on the Phase III trial design. The successful Phase II studies in patients with Fabry disease, a lysosomal storage disorder, showed that Amigal was generally safe and well tolerated at all doses evaluated.
Karo Bio AB, of Hudinge, Sweden, announced a successful Phase IIb placebo-controlled, randomized, double-blind study evaluating eprotirome, in combination with statin, for treatment of dyslipidemia. The results showed that eprotirome, in a dose-dependent manner, significantly lowered LDL-cholesterol, triglycerides and lipoprotein(a) when added to statin. The compound was safe and well tolerated.
Orthogen AG, of Düsseldorf, Germany, published a two-year study of Orthokine therapy in osteoarthritis of the knee in Osteoarthritis and Cartilage. The study compared the effects of injections of Orthokine to the injections of hyaluronic acid, a common treatment for arthritis or placebo in 310 arthritis patients over two years, and results showed that patients treated with Orthokine scored significantly better than those treated with hyaluronic acid or placebo on measures of pain and joint function.
Pozen Inc., of Chapel Hill, N.C., said the FDA has provided an initial response to its special protocol assessment for PA32540, which was submitted in June. It planned to meet with the FDA to discuss its responses for the proposed Phase III studies, including the inclusion/exclusion criteria, the stratification plan, the primary endpoint and the statistical methodology to be used in the study. Pozen also plans to discuss an additional pharmacodynamic study requested by the FDA, which would support the bioequivalence study previously conducted for PA32540.
Schering-Plough Corp., of Kenilworth, N.J., said top-line results from a planned interim analysis of a Phase II study of boceprevir, its investigational oral hepatitis C protease inhibitor, showed a high rate of sustained virologic response in treatment-naïve patients with chronic hepatitis C virus genotype 1 receiving boceprevir-based combination therapy. Safety data from the study showed that the most common adverse events reported in the boceprevir arms were fatigue, anemia, nausea and headache. The study results sent shares of rival Vertex Pharmaceuticals Inc., (NASDAQ:VRTX) of Cambridge, Mass., which makes boceprevir competitor telaprevir, down $2.75, or 8.4 percent, to close at $29.95.
Theratechnologies, of Montreal, reported Phase III data showing that patients treated with tesamorelin for 52 weeks experienced sustained improvements in belly and body appearance distress, as well as improvements in patient-reported belly profile. Data were presented at the International AIDS Conference in Mexico City. Theratechnologies previously reported that the two Phase III studies met their primary endpoints, measured by visceral adipose tissue. It intends to file a new drug application for tesamorelin in HIV-associated lipodystrophy, with targeted market approval by the end of 2009.