- An experimental drug from Eli Lilly has hit the main goal of a study testing it in people with early Alzheimer's disease, results which appear to be buoying investor optimism in the program and the company.
- The study measured cognition and daily function using a tool that combines elements from two popular scales for evaluating the impact of Alzheimer's on patients. According to Lilly, participants who received its drug, called donanemab, declined 32% slower than those who got placebo. The trial also looked at a handful of so-called secondary measures, and while there were "consistent improvements" in all of them, Lilly said some did not reach statistical significance.
- Lilly didn't offer much specific data, but said full results of the study will be presented at a medical congress and submitted for publication in a peer-reviewed clinical journal. The company also plans to discuss the results with regulators to determine the next steps for its program. In the meantime, Lilly shares rose almost 14% Monday morning, roughly adding $18 billion to its market value.
Like many Alzheimer's drugs in development, donanemab targets a protein called amyloid beta. Research indicates that, when misfolded, these amyloid proteins can clump together and form sticky plaques that lead to neurodegeneration.
Despite massive amounts of time and investment, amyloid-targeting drugs have almost universally failed. AstraZeneca, Johnson & Johnson, Merck & Co. and Roche are just some of the powerful pharmaceutical firms to hit major setbacks getting these medicines to market. Lilly, too, has notched several high-profile defeats of its own.
Yet, with donanemab, Lilly thinks it's found something promising.
The company touts how donanemab, which actually goes after a modified form of amyloid beta known as N3pG, has been shown to quickly clear amyloid plaques. The way in which the drug works, combined with the positive results from this latest trial, "give us confidence in donanemab and support its rapid and deep plaque clearance for the potential treatment of Alzheimer's disease," Daniel Skovronsky, Lilly's chief scientific officer, said in a Jan. 11 statement.
Skovronsky isn't alone is his excitement. Al Sandrock, the head scientist at Biogen, said Monday during a presentation at the J.P. Morgan Healthcare Conference that Lilly's results are good news for Alzheimer's patients and research, and offer "additional reason to hope" for a disease-modifying therapy. Currently, there are no treatments approved by the Food and Drug Administration for the underlying causes of Alzheimer's.
Sandrock's words are perhaps to be expected, since Biogen is awaiting an FDA approval decision for its own amyloid-targeting drug, called aducanumab. He noted how there are many similarities between Biogen's and Lilly's drugs, and between the studies designed to prove those drugs' merits.
Lilly's trial could also be seen as a double-edged sword, however. Analysts at Cowen & Co. noted, for example, that it was a bit surprising to see Lilly's drug miss on some of the secondary endpoints, given that trial had such a specific design.
"While it is great to have a successful trial in Alzheimer's disease, results are mixed," wrote Steve Scala of Cowen. "The effect size is small despite a tailor-made endpoint, and there were misses on some secondary endpoints, despite donanemab nearly completely wiping out plaques."