- Eli Lilly said Tuesday it has begun submitting an application to get a closely watched, experimental drug for Alzheimer's disease approved. What's more, the company announced plans to run a late-stage clinical trial that would test the drug, called donanemab, against Biogen's Aduhelm, the first and only medicine cleared in the U.S. to treat Alzheimer's directly.
- Lilly is filing its application piecemeal to the Food and Drug Administration, which has agreed to give it an expedited review. If approved, some analysts expect donanemab could become the backbone to a multibillion-dollar Alzheimer's business for Lilly, although the same was said for Aduhelm, which has struggled commercially in its first few months on the market.
- The head-to-head trial against Aduhelm, meanwhile, should start enrolling participants with early, symptomatic Alzheimer's before the end of the year. Lilly said the study will test whether its drug is superior to Biogen's at clearing the toxic brain plaques that research suggests are integral to development of the disease.
With few treatment options and millions of patients in the U.S. alone, Alzheimer's has long been one of the biggest targets for drug developers.
Yet, the disease has been even more in the spotlight since June, when Aduhelm was cleared for market. While historic, the approval was also controversial. The data used to support Biogen's application were mixed and complicated. The company and the FDA also maintained an unusually close relationship before and during the review process. By July, the FDA's acting commissioner was calling for an investigation into these interactions to determine whether they violated violated agency rules.
One other repercussion of Aduhelm's approval is that it set somewhat of a standard for the data needed to get an Alzheimer's drug to market. The FDA noted how it based its decision on Aduhelm's ability to reduce aggregates of a protein called amyloid beta — an effect which the agency said was "reasonably likely to predict a clinical benefit."
For Lilly, this precedent appears to have bolstered confidence in the prospects for donanemab. The company said in April it would not seek an accelerated approval of the drug despite having just disclosed positive data from a mid-stage trial. But by June, only weeks after the Aduhelm approval, Lilly had reversed course, announcing plans to file an application with the FDA before the end of the year.
The updates given Tuesday, alongside Lilly's third quarter earnings, confirm the company has begun submitting that application.
In a conference call, Anne White, head of Lilly's neuroscience unit, noted the challenges experienced by Biogen in launching Aduhelm, laying out her expectations for "modest use of these medicines" until there's Phase 3 data confirming patient benefit.
"By clearing plaque faster and deeper, we believe ... we've optimized the chances for showing compelling benefits in Phase 3," White added. (Lilly is testing donanemab versus placebo in a Phase 3 study, with results expected in 2023.)
As for the study testing donanemab against Aduhelm, the planned design raises a few questions, according to Evercore ISI analyst Umer Raffat. Key among them is whether it will look at any measures of clinical efficacy, in addition to the primary goal of comparing amyloid plaque clearance.
"The holy grail of clinical trials is a very rare sight … a head to head trial ... especially in new drug classes," Raffat wrote in a note to clients. "However, I don't know if I would call this the holy grail of [amyloid beta] trials – for few reasons."
Aside from donanemab, Lilly revealed Tuesday that it has stopped development of zagotenemab, a different experimental drug for Alzheimer's. Lilly also scrapped another of its closely watched drugs, tanezumab, which was being studied as a new treatment for osteoarthritis and cancer pain.
Lilly ended the third quarter with $6.8 billion in revenue, up 18% year over year. Revenue from its diabetes drugs Trulicity and Jardiance, and from its breast cancer treatment Verzenio, grew 45%, 26% and 43%, respectively, from the same three-month period a year prior.