U.S. Food and Drug Administration to Convene Advisory Committee Meeting to Discuss the TRAILBLAZER-ALZ 2 Study of Donanemab

Rhea-AI Summary

Eli Lilly and Company announces FDA advisory committee meeting to discuss Phase 3 trial of donanemab for Alzheimer's disease, delaying expected FDA action in 2024. The trial shows promising results but raises safety concerns.

Positive

• None.

Negative

• The delay in expected FDA action on donanemab due to the upcoming advisory committee meeting may impact investor sentiment and stock performance.
• Safety concerns related to amyloid related imaging abnormalities (ARIA) and other risks associated with donanemab could potentially affect the drug's commercial success and regulatory approval.
• The need for further evaluation by the FDA regarding the safety and efficacy of donanemab in early symptomatic Alzheimer's disease may indicate potential challenges in gaining regulatory approval.

Medical Research Analyst

The convening of the FDA's Peripheral and Central Nervous System Drugs Advisory Committee to discuss the Phase 3 TRAILBLAZER-ALZ 2 trial signifies a pivotal moment for Eli Lilly's donanemab. The focus on the efficacy and safety of donanemab in treating early symptomatic Alzheimer's disease underscores the complexity of developing treatments for neurodegenerative conditions. The unique trial design, with its limited-duration dosing regimen based on amyloid plaque assessment and inclusion of participants based on tau levels, represents a novel approach in the field of Alzheimer's research. This method could potentially streamline the treatment process and reduce long-term drug exposure, which may mitigate side effects.

However, the key risk associated with donanemab, namely amyloid related imaging abnormalities (ARIA), warrants careful consideration. ARIA can be serious and life-threatening and its prevalence in trial participants is a critical factor for both the FDA's assessment and physician confidence in prescribing the medication post-approval. The broader implications for Eli Lilly hinge on the FDA's final decision, as approval could mean entering a market that affects millions of Americans, while rejection or requests for additional data could significantly delay commercial availability.

Financial Analyst

From a financial perspective, the FDA's decision to convene an advisory committee meeting after the anticipated action date is noteworthy. Such a delay in the regulatory process can have tangible implications for Eli Lilly's stock market performance, as investors often react to uncertainties surrounding drug approvals. While the company has stated that this action does not change its 2024 financial guidance, the ultimate approval or disapproval of donanemab will likely have a material impact on future revenue streams.

The investment in Alzheimer's disease treatments is a high-risk, high-reward area, given the significant unmet medical need and the potential market size. However, the costs associated with prolonged clinical trials and the possibility of additional data requirements from the FDA could strain Eli Lilly's R&D budget. The company's ability to navigate the regulatory landscape and effectively communicate the benefits of donanemab will be critical in maintaining investor confidence during this extended review period.

Market Research Analyst

Alzheimer's disease represents a substantial market, affecting an estimated 6-7.5 million Americans in its early symptomatic stages. The demand for effective treatments is high and the introduction of a successful therapy like donanemab could disrupt the current market dynamics. The clinical benefits demonstrated in the TRAILBLAZER-ALZ 2 study, particularly the efficacy in earlier stages of the disease and the limited-duration treatment regimen, position donanemab as a potential game-changer in Alzheimer's care.

It is essential to consider the competitive landscape, which includes other amyloid plaque-targeting therapies. The advisory committee's review of donanemab will be compared against these existing treatments. The outcome of this meeting could influence prescribing patterns and insurance coverage decisions, ultimately shaping the market share that donanemab could capture. The long-term market potential for donanemab will depend on the balance between its clinical efficacy, safety profile and cost-effectiveness compared to alternative therapies.

INDIANAPOLIS, March 8, 2024 /PRNewswire/ -- Eli Lilly and Company (NYSE: LLY) today announced that the U.S. Food and Drug Administration (FDA) expects to convene a meeting of the Peripheral and Central Nervous System Drugs Advisory Committee (PCNS) to discuss the Phase 3 TRAILBLAZER-ALZ 2 trial, which evaluated the efficacy and safety of donanemab in early symptomatic Alzheimer's disease. 

The FDA has informed Lilly it wants to further understand topics related to evaluating the safety and efficacy of donanemab, including the safety results in donanemab-treated patients and the efficacy implications of the unique trial design of the TRAILBLAZER-ALZ 2 study, including its limited-duration dosing regimen that allowed patients to complete treatment based on an assessment of amyloid plaque and the inclusion of participants based on tau levels.

The date of the advisory committee meeting for donanemab has yet to be set by the FDA, and, as a result, the timing of expected FDA action on donanemab will be delayed beyond the first quarter of 2024. While it is unusual for an advisory committee to occur after the anticipated FDA action date, the advisory committee meeting for donanemab follows similar meetings for the two other amyloid plaque-targeting therapies the FDA has approved.

"We are confident in donanemab's potential to offer very meaningful benefits to people with early symptomatic Alzheimer's disease. It was unexpected to learn the FDA will convene an advisory committee at this stage in the review process, but we look forward to the opportunity to further present the TRAILBLAZER-ALZ 2 results and put donanemab's strong efficacy in the context of safety. We will work with the FDA and the stakeholders in the community to make that presentation and answer all questions," said Anne White, executive vice president of Eli Lilly and Company, and president of Lilly Neuroscience.

TRAILBLAZER-ALZ 2 is a Phase 3, double-blind, placebo-controlled study to evaluate the safety and efficacy of donanemab in participants ages 60-85 years with early symptomatic Alzheimer's disease (MCI or mild dementia due to Alzheimer's disease) with the presence of confirmed Alzheimer's disease neuropathology. Alzheimer's disease is a progressive and fatal disease that in its early symptomatic stages affects 6-7.5 million Americans. The trial enrolled 1,736 participants, across eight countries, selected based on cognitive assessments in conjunction with amyloid plaque imaging and tau staging by positron emission tomography (PET) imaging.

Compared to participants in similar trials of other amyloid plaque-targeting therapies, the TRAILBLAZER-ALZ 2 participants were more progressed in their disease. All groups of trial participants, regardless of tau level, benefited from treatment with donanemab, with patients in earlier stages of the disease experiencing the strongest results. Donanemab also demonstrated clinical benefits using a limited-duration treatment regimen, with nearly half of clinical trial participants completing their course of treatment in six or 12 months. The key risk associated with donanemab is amyloid related imaging abnormalities, or ARIA, which can be serious and life-threatening. Other most commonly reported risks include infusion-related reactions, headache and nausea.

The Phase 3 TRAILBLAZER-ALZ 2 study results were published in the Journal of the American Medical Association (JAMA). Lilly continues to study donanemab in multiple clinical trials.

This action does not result in a change to Lilly's 2024 financial guidance.

About Lilly
Lilly is a medicine company turning science into healing to make life better for people around the world. We've been pioneering life-changing discoveries for nearly 150 years, and today our medicines help more than 51 million people across the globe. Harnessing the power of biotechnology, chemistry and genetic medicine, our scientists are urgently advancing new discoveries to solve some of the world's most significant health challenges: redefining diabetes care; treating obesity and curtailing its most devastating long-term effects; advancing the fight against Alzheimer's disease; providing solutions to some of the most debilitating immune system disorders; and transforming the most difficult-to-treat cancers into manageable diseases. With each step toward a healthier world, we're motivated by one thing: making life better for millions more people. That includes delivering innovative clinical trials that reflect the diversity of our world and working to ensure our medicines are accessible and affordable. To learn more, visit Lilly.com and Lilly.com/news, or follow us on Facebook, Instagram and LinkedIn. P-LLY

Cautionary Statement Regarding Forward-Looking Statements
This press release contains forward-looking statements (as that term is defined in the Private Securities Litigation Reform Act of 1995) about donanemab as a potential treatment for people with early symptomatic Alzheimer's disease and reflects Lilly's current beliefs and expectations. However, as with any such undertaking, there are substantial risks and uncertainties in the process of drug research, development, and commercialization. Among other things, there is no guarantee that future study results will be consistent with study findings to date, that donanemab will prove to be a safe and effective treatment, or that donanemab will receive regulatory approval. For further discussion of these and other risks and uncertainties, see Lilly's Form 10-K and Form 10-Q filings with the United States Securities and Exchange Commission. Except as required by law, Lilly undertakes no duty to update forward-looking statements to reflect events after the date of this release.

Refer to:  J.K. Wall; jkwall@lilly.com; 317-433-5328 (Media)
                 Joe Fletcher; jfletcher@lilly.com; 317-296-2884 (Investors)

 

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SOURCE Eli Lilly and Company

What did Eli Lilly and Company announce regarding donanemab?

Eli Lilly and Company announced that the FDA will convene an advisory committee meeting to discuss the Phase 3 trial of donanemab for Alzheimer's disease.

What is the expected impact of the delay in FDA action on donanemab?

The delay in FDA action may impact investor sentiment and stock performance.

What are the safety concerns associated with donanemab?

Safety concerns include amyloid related imaging abnormalities (ARIA), infusion-related reactions, headache, and nausea.

Where were the Phase 3 TRAILBLAZER-ALZ 2 study results published?

The Phase 3 study results were published in the Journal of the American Medical Association (JAMA).

How many participants were enrolled in the TRAILBLAZER-ALZ 2 study?

The trial enrolled 1,736 participants across eight countries.