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Eli Lilly Gearing Up to Submit Anti-Amyloid Alzheimer's Drug to FDA for Full Approval in Q2

3D illustration showing amyloid plaques in Alzheimer's disease

NEW YORK – Eli Lilly will seek regulatory approval in the US for its amyloid-targeting Alzheimer's drug donanemab following a promising Phase III data readout, the pharmaceutical giant said Wednesday.

Lilly officials said the company will submit data from the TRAILBLAZER-ALZ 2 study for traditional approval with the US Food and Drug Administration during the second quarter and proceed with global regulatory submissions "as quickly as possible."

According to Lilly, the TRAILBLAZER-ALZ 2 study met its primary endpoint by demonstrating that patients with early symptomatic Alzheimer's disease and intermediate levels of the tau brain protein had 35 percent slower cognitive and functional decline on donanemab compared to placebo over 18 months, based on the integrated Alzheimer's Disease Rating Scale (iADRS).

"We are extremely pleased that donanemab yielded positive clinical results," Daniel Skovronsky, Lilly's chief scientific and medical officer and president of Lilly Research Laboratories, said in a statement. "This is the first Phase III trial of any investigational medicine for Alzheimer's disease to deliver 35 percent slowing of clinical and functional decline."

The FDA previously rejected a bid from Lilly for accelerated approval of donanemab based on Phase II data showing the drug cleared amyloid. The agency said Lilly didn't have enough data from patients who had been taking donanemab for at least 12 months.

The TRAILBLAZER-ALZ 2 trial also met multiple secondary endpoints including demonstrating that donanemab-treated patients had 36 percent slower cognitive decline compared to patients on placebo as measured by another scale, the Clinical Dementia Rating-Sum of Boxes (CDR-SB). One-third of patients on donanemab achieved amyloid clearance at six months, and 71 percent achieved clearance after one year.

Investigators also enrolled a smaller number of people with high levels of tau at baseline, representing later-stage disease. When that group was combined with the intermediate tau population, donanemab-treated patients experienced a 22 percent and 29 percent slower decline on iADRS and CDR-SB, respectively, compared to patients on the placebo arm.

About 1.6 percent of patients experienced serious amyloid-related imaging abnormalities (ARIA) on the drug, including two individuals whose deaths were attributed to ARIA.

If approved, donanemab would be the third Alzheimer's drug on the market that seeks to treat the disease by attacking beta-amyloid clusters in the brain.

The FDA controversially greenlighted Biogen and Eisai's Aduhelm (aducanumab) for Alzheimer's based on its ability to clear amyloid through the accelerated approval pathway in 2021. Aduhelm was met with skepticism from doctors and insurers over whether the drug had demonstrated adequate clinical benefits, with Medicare opting to restrict insurance coverage of the drug.

Biogen has since cut commercialization efforts for Aduhelm to focus on marketing its latest Alzheimer's drug, Leqembi (lecanemab). Leqembi, another anti-amyloid drug developed by Biogen and Eisai, received accelerated approval in January, and Eisai has submitted a supplemental biologics license application for traditional approval.

The US Centers for Medicare & Medicaid Services in February said it would continue to limit coverage of Alzheimer's therapies targeting amyloid, including Leqembi, pending further evidence of clinical benefits. Lilly officials hope its Phase III findings will spur the agency to reconsider that coverage decision.

"We believe our data meets the 'high level of evidence' … CMS has described as the trigger for reconsideration of its National Coverage Determination," Anne White, Lilly's executive VP and president of Lilly Neuroscience, said in a statement. "People with early Alzheimer's disease need and deserve full coverage and access for approved therapies."