Pompe Disease

October 29, 2007

Amicus reported positive results from two phase I trials of AT2220 for the treatment of Pompe Disease. These double-blind, placebo-controlled, dose escalation studies were designed to evaluate the safety, tolerability and pharmacokinetics of AT2220. In a single ascending dose study, 32 subjects received oral doses of AT2220 (50, 150, 300, or 600 mg) or placebo. In a multiple ascending dose study, 24 subjects received oral doses of AT2220 (50, 150, or 450 mg/day) or placebo for 7 days. Treatment was determined to be safe and well tolerated at all doses tested. In the multiple ascending dose study all possible drug-related adverse events were mild and resolved spontaneously. AT2220 was orally bioavailable with a plasma half-life of 4 to 5 hours. Based on the results, Amicus plans to initiate phase II trials early in 2008.

MediciNova issued negative results from a phase IIa trial of MN-305 for the treatment of insomnia. This randomized, double-blind, placebo-controlled, four-period crossover dose-response study enrolled 90 subjects with primary insomnia in the US. Each subject received three doses of MN-305 (1 mg, 3 mg and 6 mg) and placebo, administered orally approximately 60 minutes before bedtime, for seven consecutive nights. The primary endpoint, statistical significance in the reduction of Wake (time) After Sleep Onset (WASO), was not met. Treatment was well tolerated at all doses tested. Based on the results, MediciNova plans to discontinue the development of MN-305 for insomnia and focus on other potential indications.

TorreyPines reported positive results from a phase IIb trial of tezampanel for the treatment of migraine headaches. This randomized, double-blind, placebo-controlled, parallel-group study enrolled 306 subjects in the US. The subjects were equally randomized to one of four treatment arms: tezampanel 40 mg, 70 mg, or 100 mg or placebo administered as a single, subcutaneous dose. The primary endpoint was headache pain response at two hours post-dose. Statistical significance was observed in the 40 mg arm, with improvement in headache response reported in 78.2% of the subjects versus 58.7% in the placebo arm (p=0.033). This response was sustained through 24 hours post-dose. Statistical significance was not reached in the 70 mg arm (63.5%; p=0.890) or the 100 mg arm (57.1%; p=0.890). The 40 mg arm achieved the secondary endpoints as well, with improvements over placebo in nausea (p=0.014), photophobia (p=0.056) and phonophobia (p=0.227). Treatment was well tolerated, with no reports of serious adverse events. Based on the results, TorreyPines plans to commence phase III trials in 2008.