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Anti-microtubule binding region-tau antibody being investigated as a potential disease-modifying therapy to slow or delay progression of disease
Fast Track Designation recognizes the potential of anti-MTBR tau to be an important treatment option for patients with Alzheimer's disease
Bristol Myers Squibb (NYSE: BMY) today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to BMS-986446, a potential best-in-class anti-microtubule binding region-tau (anti-MTBR-tau) antibody currently in Phase 2 development for the treatment of early Alzheimer's disease. Fast Track Designation is intended to facilitate the development and expedite the review of investigational drugs that treat serious conditions and fill an unmet medical need.
Alzheimer's disease — the most common type of dementia in adults — is a progressive, multifaceted and devastating neurodegenerative disease in which significant changes occur in the brain that cause neurons to die over time. These changes include the accumulation and spread of pathological tau, an abnormal form of the tau protein. Pathological tau protein fragments containing the microtubule binding region (MTBR) appear to have a key role in the underlying pathology of Alzheimer's disease. By neutralizing the spread and promoting the clearance of pathological tau, BMS-986446 aims to modify the underlying course of the disease with the ultimate goal of slowing or delaying disease progression.
"The FDA's Fast Track Designation for BMS-986446 underscores the urgent need for innovative therapies for Alzheimer's disease and recognizes the potential of this investigational anti-MTBR-tau antibody to meaningfully alter the trajectory of disease progression," said Laura Gault, senior vice president, head of development, Neuroscience, Bristol Myers Squibb. "Bristol Myers Squibb is taking a continuum of care approach to Alzheimer's disease, studying investigational medicines such as those targeting tau to change the course of the disease as well as several symptomatic treatment options that can address severe shifts in behavioral symptoms, such as psychosis and agitation, that significantly impact patients and their caregivers."
In preclinical models, BMS-986446 demonstrated significant reductions in tau uptake and spread, protection against behavioral deficits and was localized with tau pathology in Alzheimer's brain tissue. BMS-986446 was also shown to be safe and well tolerated across three dose cohorts in a Phase 1 study of healthy participants. The ongoing Phase 2 study is fully enrolled and includes several biomarkers of tau and amyloid-beta biology, as well as clinical outcome measures, to evaluate the impact of BMS-986446 on disease progression.
About BMS-986446
BMS-986446 is a humanized monoclonal antibody that targets multiple domains of the microtubule binding region of tau, a highly pathogenic tau fragment associated with neurofibrillary tangle formation and cognitive decline in Alzheimer's disease. BMS-986446 binds to specific regions of the tau protein (R1–R3 within the microtubule-binding domain) to prevent the cell-to-cell spread of tau and tau uptake into cells. It also activates microglia—the brain's immune cells—through its Fc receptor function, promoting the clearance of tau via phagocytosis.
About the TargetTau-1 Phase 2 Trial (NCT06268886)
TargetTau-1 is a randomized, double-blind, placebo-controlled, global Phase 2 proof-of-concept study designed to evaluate the efficacy, safety and tolerability of multiple doses of BMS-986446 in participants with early Alzheimer's disease. The study aims to determine whether targeting MTBR-tau can modify disease progression. In addition to clinical outcome measures, the trial integrates a comprehensive biomarker strategy to assess tau and amyloid-beta biology.
About Alzheimer's Disease
Alzheimer's disease is a progressive, multifaceted and devastating neurodegenerative disease and the most common type of dementia in adults. Changes in the brain disrupt communication between neurons, impacting memory, cognition and behavior. As a result, Alzheimer's disease has a significant impact on the day-to-day lives of those it directly affects, as well as on their families, caregivers and friends, resulting in considerable shifts in interpersonal relationships. There remains a critical need for disease-modifying therapies that can slow or delay the progression of Alzheimer's disease as well as therapies that manage and ease neurobehavioral symptoms.
Bristol Myers Squibb: Transformational Research Advancing Neuroscience
Bristol Myers Squibb is inspired by a single vision – transforming patients' lives through science. Neurological and neuropsychiatric conditions represent some of the greatest challenges of our time, bringing life-altering hardships to patients, caregivers and families throughout the course of these devastating diseases. Our researchers are committed to the pursuit of breakthrough science to develop life-changing medicines that modify disease and treat symptoms to improve quality of life. We have established a diverse neuroscience portfolio, including assets across a wide range of therapeutic modalities and mechanisms in conditions such as Alzheimer's disease, schizophrenia, multiple sclerosis and more. With industry-leading capabilities, including our in-house neuroimaging program, we seek bold solutions to improve the treatment landscape. Evolution is in our DNA at Bristol Myers Squibb. We are motivated by the rapid progress of scientific knowledge within neuroscience and have an unwavering commitment to advance the most promising innovations to deliver transformational results for patients.
About Bristol Myers Squibb
Bristol Myers Squibb is a global biopharmaceutical company whose mission is to discover, develop and deliver innovative medicines that help patients prevail over serious diseases. For more information about Bristol Myers Squibb, visit us at BMS.com or follow us on LinkedIn , X , YouTube , Facebook and Instagram .
Cautionary Statement Regarding Forward-Looking Statements
This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 regarding, among other things, the research, development and commercialization of pharmaceutical products. All statements that are not statements of historical facts are, or may be deemed to be, forward-looking statements. Such forward-looking statements are based on current expectations and projections about our future financial results, goals, plans and objectives and involve inherent risks, assumptions and uncertainties, including internal or external factors that could delay, divert or change any of them in the next several years, that are difficult to predict, may be beyond our control and could cause our future financial results, goals, plans and objectives to differ materially from those expressed in, or implied by, the statements. These risks, assumptions, uncertainties and other factors include, among others, that future study results will be consistent with the results to date, that BMS-986446 may not achieve its primary study endpoints or receive regulatory approval for the indication described in this release in the currently anticipated timeline or at all, any marketing approvals, if granted, may have significant limitations on their use, and, if approved, whether BMS-986446 for such indication will be commercially successful. No forward-looking statement can be guaranteed. It should also be noted that Fast Track Designation does not change the standards for FDA approval. Forward-looking statements in this press release should be evaluated together with the many risks and uncertainties that affect Bristol Myers Squibb's business and market, particularly those identified in the cautionary statement and risk factors discussion in Bristol Myers Squibb's Annual Report on Form 10-K for the year ended December 31, 2024, as updated by our subsequent Quarterly Reports on Form 10-Q, Current Reports on Form 8-K and other filings with the Securities and Exchange Commission. The forward-looking statements included in this document are made only as of the date of this document and except as otherwise required by applicable law, Bristol Myers Squibb undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events, changed circumstances or otherwise.
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