• Cell Therapeutics Inc., of Seattle, said an article summarizing preclinical and clinical data, including pharmacology and pharmacokinetics, safety and efficacy data in Phase I and Phase II studies in myelofibrosis and lymphoma, for pacritinib, an oral JAK2/FLT3 inhibitor, was published in Drugs of the Future. Authors concluded that the trials to date have supported the safety, efficacy and predictable pharmacokinetic profile of the drug. In the two studies in myelofibrosis patients, pacritinib led to disease response, improvement in splenomegaly and lowered white blood cell count. Pacritinib is in a Phase III study, which has been dubbed PERSIST-1, in myelofibrosis patients.

• CSL Behring, of King of Prussia, Pa., reported data showing clinical efficacy of a once-weekly dosing regimen of recombinant fusion protein linking coagulation Factor IX with albumin (rIX-FP). The Phase I/II trial enrolled 17 hemophilia B patients, 13 of whom received weekly prophylactic treatment for approximately 11 months and four of whom received on-demand treatment only when bleeding occurred. Prophylaxis patients maintained weekly treatment for the entire study, with mean and median annualized spontaneous bleeding rates of 1.26 and 1.13, respectively. Bleeding was treated successfully with two or fewer infusions of rIX-FP, with 95.3 percent of events treated with a single infusion. Following a single infusion of 25 IU/kg rIX-FP, the mean FIX activity level was 3.8 percent and 2.7 percent above baseline at day seven and 14, respectively, and the half-life of rIX-FP was 94 hours. The findings were presented during an oral session at the International Society on Thrombosis and Hemostasis congress in Amsterdam.

• Ultragenyx Pharmaceutical Inc., of Novato, Calif., disclosed interim 24-week data from a 48-week Phase II study of UX001 in 47 patients with hereditary inclusion body myopathy (HIBM), a progressive muscle-wasting disease. The study compared treatment with a total daily dose of 6 g or 3 g of UX001 with placebo. UX001, an oral sialic acid extended-release tablet, is designed to replace the deficient sialic acid substrate in patients with HIBM. The data showed dose-dependent improvement in muscle strength relative to placebo in some muscle groups, particularly in the upper extremities at the 6 g dose. These changes were statistically significant or trended toward significance, and were more pronounced in those patients who had greater walking ability at baseline, a predefined subset. Other clinical endpoints did not reveal changes at the interim assessment. Creatine kinase levels showed a trend to improvement in the 6-g dose group compared with placebo. UX001 appeared to be well tolerated with no serious adverse events observed to date in either dose group.