September 18, 2017
Eli Lilly reported results of a phase III study of galcanezumab for migraine. In the 12-month, open-label study, 270 patients were randomized to treatment with galcanezumab 120mg or galcanezumab 240mg. Galcanezumab was associated with a statistically significant reduction in the number of monthly migraine headache days with both doses (5.6 days for 120mg and 6.5 days for 240mg, p<0.001 for both dosing groups). Notably, there was no clinically meaningful difference in the rate of adverse events between galcanezumab 120mg and 240mg dosing groups. The most commonly reported adverse events (≥10%) in both dosing groups included injection site pain, nasopharyngitis and upper respiratory tract infection. Serious adverse events were reported by three patients in the 120mg dosing group and seven patients in the 240mg group. Lilly plans to submit a Biologics License Application (BLA) to the FDA for galcanezumab in the second half of 2017, followed by submissions to other regulatory agencies around the world.
July 3, 2017
Alder BioPharmaceuticals issued results of a phase III study of eptinezumab for frequent episodic migraine. PROMISE 1 was a double-blind, randomized, placebo-controlled study evaluating eptinezumab (300mg, 100mg or 30mg) administered by intravenous infusion once every 12 weeks through week 24. PROMISE 1 met the primary endpoint: highly statistically significant reductions in monthly migraine days. Significant Day 1 clinical benefit was a ≥50% reduction in the proportion of patients experiencing a migraine on Day 1 post-dose. There were significant 75% responses at all key time points: ~1/3 of patients achieved a ≥75% reduction in migraine days through four and 12 weeks. An average of one in five patients had 100% response: no migraines in any given month, months one through six. Enrollment is on track for PROMISE 2, a second pivotal phase III study that focuses on chronic migraine, and the company is on track to submit a BLA with the FDA in 2018.
June 5, 2017
Teva Pharmaceutical Industries announced positive results from a phase III HALO study of fremanezumab, an investigational treatment for the prevention of migraine. In the chronic migraine (CM) study, patients treated with fremanezumab experienced statistically significant reduction in the number of monthly headache days of at least moderate severity vs. placebo (-2.5 days) during the 12 week period after first dose, for both monthly (-4.6 days p<0.0001) and quarterly (-4.3 days p<0.0001) dosing regimens. Similar to the Phase II trials, both patients that were on monotherapy and stable doses of prophylactic medications were included in the trial. In addition, patients treated with fremanezumab experienced significant improvement compared to placebo on all secondary endpoints for both monthly and quarterly dosing regimens, including: response rate, onset of efficacy, efficacy as monotherapy, and disability. The results were positive, and of 13 hierarchical comparisons, p was <0.0001 in 12 of them, being 0.0004 in the remaining. The most commonly-reported adverse event in the study was injection site pain, with similar rates in the placebo and active groups. Based on these results, Teva plans to submit a Biologics License Application to the FDA for fremanezumab later this year.
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.