Eli Lilly (LLY) shares moved lower Friday after the drugmaker said its request for an accelerated review of its developing Alzheimer's treatment was rejected by the U.S. Food & Drug Administration.
Eli Lilly, which says it will publish Phase 3 trial data of the drug, known as donanemab, by the middle of the year, said the FDA sent a so-called 'complete response letter' outlining issues that the drugmaker needs, including "the limited number of patients with at least 12 months of drug exposure data provided in the submission."
The drugmaker said the speed at which donanemab reduced amyloid plaque -- proteins found in spaces between nerve cells that can disrupt communication to the brain -- meant that some patients left the study after six months, reducing its overall same size.
Last year, Eli Lilly said donanemab, showed a 32% reduction in a composite measure of cognition and daily function in patients suffering early symptomatic Alzheimer's disease following a Phase 2 trial known as Trailblazer.
"We look forward to our upcoming confirmatory Trailblazer-ALZ 2 Phase 3 results and subsequent FDA submission, which we've always seen as the most impactful next steps for patients," said Anne White, Eli Lilly's Neuroscience president. "We anticipate this study will confirm the benefit and safety profile we observed in the Trailblazer-ALZ 2 Phase 2 study and believe that patients and physicians will be well served by having the full Phase 3 data available alongside our Phase 2 data when they need to make treatment decisions."
"We are committed to working with the FDA to ensure the fastest possible path to bring this potential medicine to patients in need," she added.
Eli Lilly shares were marked 1.4% lower in early Friday trading to change hands at $346.14 each, a move that would trim the stock's six-month gain to around 7.5%.
Late last year, European drugmaker Roche AG RHHBY said that trials of its gantenerumab, a drug used to treat patients with early-stage Alzheimer's, failed to reach the primary endpoint of preserving cognitive functions affected by the disease.
The results leave Biogen, which unveiled better-than-expected results from a late-stage study of its Alzheimer's treatment developed with Japan-based partner Eisai Co Ltd., as well as Eli Lilly, as potential market leaders in the multi-billion market.
Alzheimer's disease is a progressive brain disorder that affects more than 50 million people around the world. To date, no drug has been found to address the disease, which can accelerate into dementia and other more serious cognitive conditions.
Treatment pricing, however, has been a controversial issue and the U.S. Centers of Medicare and Medicaid Services noted in September that its decision to only cover treatment with Aduhelm -- Biogen's FDA-approved Alzheimer's drug -- if patients are enrolled in a clinical trial as a major factor in lowering overall premiums for Medicare Part B.
Biogen was forced to slash the price of Aduhelm by around 50%, to $28,200 per year, in order to help expand its potential reach following the CMS decision late last year.
Aduhelm, Biogen noted in earlier studies, has been show to erode amyloid beta, a plaque which builds up around the brain and can lead to neuron damage.
The FDA approved Aduhelm for the treatment of Alzheimer’s through its accelerated approval pathway, which can be used for a drug for a serious or life-threatening illness that provides a meaningful therapeutic advantage over existing treatments, last year.
