After weeks of anticipation, Eli Lilly released complete data for studies of its solanezumab Alzheimer's compound and results indicated that cognitive decline was slowed by 42 percent in patients in the early stages of the disease in one trial, and there were also positive signs from another trial, although the showing was not statistically significant.
However, a pre-specified secondary analysis of pooled data yielded a 34 percent reduction in cognitive decline in patients with mild Alzheimer's, which was statistically significant. Although the overall showing was mixed, the results, nonetheless, underscore impressions of the preliminary findings released in August that suggested the compound may prove successful at combating symptoms in some early-stage patients.
“This isn’t the home run that is going to get you a medication by the end of the year, but it is very encouraging because with subsequent studies this could be a viable therapeutic option,” Maria Carrillo, vp of medical and scientific relations at the Alzheimer’s Association tells Bloomberg News. “We need more options for our patients and families. We are very encouraged by what this could mean."
Further studies will be needed and Lilly expects to hold talks with the FDA. In August, the drugmaker released results indicating solanezumab did not work two late-stage trials that tested effectiveness in slowing both cognitive and functional decline in mild-to-moderate Alzheimer's patients. However, Lilly also noted there were positive finding without offering specifics (back story).
The data was discussed today at the annual meeting of the American Neurological Association, which is being held in Boston. While one trial yielded a 42 percent reduction in cognitive decline, the second trial revealed a 20 percent decline, although this finding was statistically significant. When pooled together, the results indicated a 34 percent decline. There was no mention of functional improvement, though (here is the Lilly statement).
Alzheimer is a form of dementia that affects 36 million people worldwide and about 5 million in the US. Lilly hopes that its solanezumab compound, which attaches to a protein called beta amyloid that builds up into clumps in the brains of Alzheimer’s patients, will prevent clumps from forming. However, the study results suggest that the compound is most effective in patients with mild Alzheimer's, which is where Lilly is expect to focus its efforts.
About 2,000 patients were tested in the two solanzemab trials trials and about two-thirds had the mild form of the disease, Bloomberg notes. However, it remains unclear why the drug showed a statistically significant effect in only one trial. Nonetheless, the findings cheered investors, who boosted Lilly stock, which has gained about 15 percent since the release of top-line data in August.
"We feel the today's results surpassed most investor expectations and that enthusiasm for solanezumab will likely rise from their current low levels," writes Sanford Bernstein analyst Tim Anderson in an investor note today. "These results are very encouraging because trends, or in some cases, statistical significance, were seen across multiple measurements including cognition and biomarkers. This clearly supports Lilly performing additional clinical studies and moves solanezumab one step closer towards being a real therapy, albeit in a future year."
So what's next? Anderson expects Lilly will have to run "one or more" Phase III trials before being able to file for FDA approval. This means, however, that agency approval may not come until 2016 or so. "It remains only a low-likelihood," he writes, "theoretical possibility that Lilly could file for approval on the current data set" on an 'as is' basis, even though there is a great unmet medical need.
brain pic thx to tocaboca on flickr