New Medical Therapies™

Latest New Medical Therapy Results

Week of February 8, 2010

Keryx reported positive results from a phase II trial of perifosine for advanced Waldenstrom's Macroglobulinemia. This open label, single agent trial enrolled 37 subjects with relapsed or refractory disease. The subjects received 150 mg perifosine orally every night for six back to back 28 day cycles. Stable or responding subjects were allowed to continue therapy until progression. Of the 37 subjects, four achieved a partial response (11%), nine achieved a minimal response (24%), and 20 showed stable disease (54%). Overall, 89% of subjects were reported to have stable disease or better, while 11% demonstrated progression. The median progression-free survival was 12.6 months, with a median overall survival of 26 months. Perifosine was generally well-tolerated.

NeuroSearch released positive results from a phase III trial of Huntexil for the treatment of Huntingtons disease. This European-based, randomized, double-blind, placebo-controlled study, MermaiHD (Multinational EuRopean Multi-centre ACR16 study In Huntingtons Disease), enrolled 420 subjects. The subjects received either 45 mg Huntexil once daily (QD), 45 mg Huntexil twice daily (BID) or placebo over 26 weeks of treatment. The primary efficacy endpoint was the effect of Huntexil on motor function measured by the modified Motor Score, mMS - a subscale of the Unified Huntingtons Disease Rating Scale. Treatment with Huntexil 45 mg BID demonstrated a statistically significant improvement compared to placebo (p<0.02). Other measures of motor symptoms were also reached with statistical significance in the 45 mg arm versus placebo, including Total Motor Score (p<0.001), Eye Movements (p<0.002) and Dystonia (p <0.001). Treatment with Huntexil 45 mg QD showed some improvements on these motor function domains, but did not reach statistical significance. Huntexil was generally very well tolerated with an adverse event profile similar to placebo.

PROLOR issued positive results from a phase I trial of hGH-CTP, their long -acting version of human growth hormone. This randomized, double-blinded, placebo-controlled, single-dose, dose escalating study enrolled 24 healthy subjects at the Tel-Aviv Medical Center in Israel. The subjects received one of three doses of hGH-CTP (4mg, 7mg, or 21mg) or placebo. All safety and tolerability endpoints were met with each of the doses. The potential clinical efficacy of hGH-CTP was assessed by measuring the extent to which hGH-CTP induced insulin-like growth factor-1 (IGF-1) in subjects. Based on this measure, data suggests that the daily injections currently required by conventional hGH-users can potentially be replaced with just two monthly injections of hGH-CTP.

Vertex issued positive results from a phase IIa trial of VX-809 for the treatment of cystic fibrosis (CF). This randomized, double-blind study enrolled 89 adult subjects homozygous for the F508del mutation. The subjects received once daily doses of VX-809 (25 mg, 50 mg, 100 mg or 200 mg) or placebo, in addition to standard therapies, for 28 days. At both the 100 mg and 200 mg dose levels, a statistically significant decline in sweat chloride, a diagnostic hallmark that occurs in all CF patients, was observed compared to placebo (-6.13 mmol/L and -8.21 mmol/L , respectively). A dose response for change in sweat chloride was observed across all four dose groups. VX-809 was well-tolerated across all treatment arms.