1 February 2012. Recent company reports reveal that clinical trials of two different Alzheimer’s disease immunotherapies survived futility analyses. These are interim measures designed to assess whether the trial is likely to produce meaningful results. In a January 31 conference call, Lilly announced that EXPEDITION and EXPEDITION2 trials of solanezumab, a humanized mouse monoclonal antibody against Aβ (see ARF related news story), would continue as planned, based on a data-monitoring committee assessment of safety and futility data. The latter stipulated that one of two primary endpoints in either of the Phase 3 trials should have a 30 percent chance of being positive when the trials ended, according to Ronika Pletcher, Director of Investor Relations at Lilly. The data indicated that hurdle was passed, but in a conference call, Pletcher emphasized that the analysis does not say anything about efficacy.
Both EXPEDITION trials aimed to enroll around 1,000 volunteers, to be given either a placebo or solanezumab every four weeks for 80 weeks. The primary outcome measures are change in cognition and activities of daily living. Both trials will end later this year and efficacy analysis is expected before the end of the year.
And last week, Baxter International Inc., the medical products and services company based in Deerfield, Illinois, announced that it plans to conduct a second Phase 3 trial of its Gammagard for patients with Alzheimer’s. The decision came after a futility analysis within the ongoing Phase 3 trial. "Upon reviewing the futility analysis, the Data Safety Monitoring Board stated that the trial could continue without modification," wrote Brian Kyhos, a spokesperson for Baxter, in an e-mail to ARF. "The data we receive from the first ongoing study and the confirmatory study will be used to support a regulatory filing."
A small Phase 2 trial of two dozen patients with mild to moderate AD hinted at positive effects on brain atrophy and cognition (see ARF related news story). No data are available yet for the ongoing Phase 3 trial, which is being conducted in collaboration with the Alzheimer's Disease Cooperative Study (ADCS), a national research consortium funded by the National Institute on Aging. That will run until February 2013.
In both Phase 3 trials, about 400 patients with mild to moderate AD from centers in North America, Europe, Japan, and Australia will receive either 200 or 400 milligrams of the intravenous immunoglobulin preparation (IVIg) per kilogram body weight every other week for 70 weeks. "The primary objective is to evaluate the efficacy of IVIg treatment on change in cognitive performance and functional activities," wrote Kyhos.
IVIg is made from the pooled plasma of healthy human blood donors. It contains antibodies that have been reported to target amyloid, as well as central nervous system anti-inflammatory properties. Currently, IVIg is FDA-approved for patients with certain immune disorders.—Gwyneth Dickey Zakaib and Tom Fagan.