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FDA grants traditional approval for Leqembi for the treatment of Alzheimer's disease

STOCKHOLM, July 7, 2023. BioArctic AB's partner Eisai announced today that the U.S. Food and Drug Administration, FDA, has approved the supplemental Biologics License Application, sBLA, supporting the traditional approval of LEQEMBI (lecanemab-irmb) 100 mg/mL injection for intravenous use, making LEQEMBI the first and only approved treatment shown to reduce the rate of disease progression and to slow cognitive and functional decline in adults with Alzheimer's disease (AD). In conjunction with the approval the Centers for Medicare and Medicaid Services, CMS, announced that Medicare will provide broad coverage of LEQEMBI according to the FDA approved label provided that real-world evidence is collected in an easy-to-use patient registry.[1]

LEQEMBI's traditional approval is based on Phase 3 data from Eisai's large, global Clarity AD clinical trial, in which LEQEMBI met its primary endpoint and all key secondary endpoints with statistically significant results and confirmed the clinical benefit of LEQEMBI. The primary endpoint was the global cognitive and functional scale, Clinical Dementia Rating Sum of Boxes (CDR-SB). LEQEMBI treatment reduced clinical decline on CDR-SB by 27% at 18 months compared to placebo in an early Alzheimer's disease[2] population. Additionally, the secondary endpoint of AD Cooperative Study-Activities of Daily Living Scale for Mild Cognitive Impairment (ADCS MCI-ADL), as measured by people caring for patients with AD, noted a statistically significant benefit of 37%. This measures the ability of patients to function independently, including being able to dress, feed themselves and participate in community activities. [3]

"The full, traditional approval of LEQEMBI in the US, combined with the broad Medicare reimbursement, is a paradigm-shifting step in the fight against Alzheimer's disease," said Gunilla Osswald, CEO of BioArctic. "Doctors in the US will now have a tool to combat this terrible chronic disease already at an early stage, with the potential to provide clinically meaningful benefit for patients and their families. More than two decades of research and development has led up to this moment, and I am impressed by the diligent efforts of our partner Eisai to ensure that this important innovation can now reach the patients BioArctic was founded to serve. I am full of gratitude and hope for the future."

On June 9, 2023, the FDA's Peripheral and Central Nervous System Drugs (PCNS) advisory committee voted unanimously that the data from Eisai's Clarity AD clinical trial confirmed the clinical benefit of LEQEMBI for the treatment of AD. Committee members also confirmed the overall risk-benefit of LEQEMBI.

Importantly, following FDA's traditional approval of LEQEMBI, CMS confirmed that broader coverage of LEQEMBI is now available and released more details on the registry, including the easy-to-use data submission process. The CMS-facilitated registry is now available for healthcare professionals to submit required patient data to CMS. We are pleased that Medicare will cover this important therapy for appropriate patients. This will facilitate reimbursement for and access to LEQEMBI across a broad range of healthcare settings in the United States.

"More than 25 years after my initial discoveries, it is fantastic to finally see LEQEMBI becoming available to the patients who have always been my focus," said Professor Lars Lannfelt, Co-founder of BioArctic and inventor of LEQEMBI. "From the start, my idea was to find a way to clear the soluble amyloid beta (Aβ) oligomers and protofibrils, the most neurotoxic forms of Aβ, that continuously accumulate in the brain of Alzheimer disease patients. LEQEMBI was generated to do just that. But even if this is a great step forward, it is important for BioArctic and others to continue research to provide more options for patients."

Eisai serves as the lead of lecanemab development and regulatory submissions globally with both Eisai and Biogen co-commercializing and co-promoting the product and Eisai having final decision-making authority. BioArctic has the right to commercialize lecanemab in the Nordic region and currently Eisai and BioArctic are preparing for a joint commercialization in the region.

Indication
LEQEMBI is indicated for the treatment of Alzheimer's disease. Treatment with LEQEMBI should be initiated in patients with mild cognitive impairment or mild dementia stage of disease, the population in which treatment was initiated in clinical trials.

About LEQEMBI (lecanemab-irmb)
LEQEMBI (lecanemab-irmb) is the result of a strategic research alliance between BioArctic and Eisai. LEQEMBI is a humanized immunoglobulin gamma 1 (IgG1) monoclonal antibody directed against aggregated soluble (protofibril) and insoluble forms of amyloid-beta (Aβ). LEQEMBI is an amyloid beta-directed antibody indicated as a disease-modifying treatment for Alzheimer's disease (AD). In the US, LEQEMBI was granted accelerated approval by the US Food and Drug Administration (FDA) on January 6, 2023, and traditional approval on July 6, 2023. Treatment with LEQEMBI should be initiated in patients with mild cognitive impairment or mild dementia stage of disease, the population in which treatment was initiated in clinical trials. There are no safety or effectiveness data on initiating treatment at earlier or later stages of the disease than were studied.

Eisai has also submitted applications for approval of lecanemab in Japan, EU, China, Canada, Great Britain, and South Korea. In Japan and China, the applications have been designated for priority review, and in Great Britain, lecanemab has been designated for the Innovative Licensing and Access Pathway (ILAP), which aims to reduce the time to market for innovative medicines.

Eisai has completed a lecanemab subcutaneous bioavailability study, and subcutaneous dosing is currently being evaluated in the Clarity AD (Study 301) open-label extension (OLE). A maintenance dosing regimen has been evaluated as part of the Phase 2b study (Study 201) OLE as well as the Clarity AD (Study 301) OLE. Separate supplemental Biologics License Applications for subcutaneous dosing and a maintenance dosing regimen is planned to be submitted to the FDA at the end of the first quarter 2024.

Since July 2020 Eisai's Phase 3 clinical study (AHEAD 3-45) for individuals with preclinical AD, meaning they are clinically normal and have intermediate or elevated levels of amyloid in their brains, is ongoing. AHEAD 3-45 is conducted as a public-private partnership between the Alzheimer's Clinical Trial Consortium that provides the infrastructure for academic clinical trials in AD and related dementias in the U.S, funded by the National Institute on Aging, part of the National Institutes of Health and Eisai.

Since January 2022, the Tau NexGen clinical study for Dominantly Inherited AD (DIAD) is ongoing, where lecanemab is given as a background anti-amyloid treatment when exploring combination therapies with anti-tau treatments. The study is conducted by Dominantly Inherited Alzheimer Network Trials Unit (DIAN-TU), led by Washington University School of Medicine in St. Louis.

About the collaboration between BioArctic and Eisai
Since 2005, BioArctic has a long-term collaboration with Eisai regarding the development and commercialization of drugs for the treatment of Alzheimer's disease. The most important agreements are the Development and Commercialization Agreement for the lecanemab antibody, which was signed 2007, and the Development and Commercialization agreement for the antibody BAN2401 back-up for Alzheimer's disease, which was signed 2015. In 2014, Eisai and Biogen entered into a joint development and commercialization agreement for lecanemab. Eisai is responsible for the clinical development, application for market approval and commercialization of the products for Alzheimer's disease. BioArctic has right to commercialize lecanemab in the Nordic under certain conditions and is currently preparing for commercialization in the Nordics together with Eisai. BioArctic has no development costs for lecanemab in Alzheimer's disease and is entitled to payments in connection with regulatory approvals, and sales milestones as well as royalties on global sales.

About BioArctic AB
BioArctic AB (publ) is a Swedish research-based biopharma company focusing on disease-modifying treatments for neurodegenerative diseases, such as Alzheimer's disease, Parkinson's disease and ALS. BioArctic focuses on innovative treatments in areas with high unmet medical needs. The company was founded in 2003 based on innovative research from Uppsala University, Sweden. Collaborations with universities are of great importance to the company together with its strategically important global partner Eisai in Alzheimer disease. The project portfolio is a combination of fully funded projects run in partnership with global pharmaceutical companies and innovative in-house projects with significant market and out-licensing potential. BioArctic's Class B share is listed on Nasdaq Stockholm Large Cap (ticker: BIOA B).

[1] https://www.cms.gov/newsroom/press-releases/statement-broader-medicare-coverage-leqembi-available-following-fda-traditional-approval

[2] Mild cognitive impairment due to AD and mild AD dementia is collectively referred to as early AD

[3] Full results of the Clarity AD study were presented at the Clinical Trials on Alzheimer's Disease (CTAD) 2022 conference and simultaneously published in the peer-reviewed medical journal,The New England Journal of Medicine, November 29, 2022.

Posted: July 2023

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