June 6, 2025
Source: drugdu
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Alzheimer's disease (AD) is a disease that scares countless families. It has an insidious onset, continues to progress, and is irreversible. Brain lesions have already occurred quietly 15 to 20 years before obvious symptoms appear. Moving the diagnosis and intervention window forward is a key measure to reduce the incidence of AD.
In decades of exploration, existing drugs have always been trapped in the cage of "delaying symptoms". Now, with the maturity of diagnostic technology and the emergence of a new generation of drugs targeting beta-amyloid protein (Aβ), the academic community sees hope of "pressing the pause button on the disease".
In 2021, Aducanumab received accelerated approval from the FDA, becoming the first new drug approved by the FDA for AD, for the treatment of AD-related mild cognitive impairment (MCI) and mild AD.
In 2023, the FDA approved Lecanemab for the treatment of early AD with mild cognitive impairment or mild dementia.
In 2024, the FDA urgently approved a new drug called "Donanemab" for the treatment of early symptomatic Alzheimer's disease. This drug is highly anticipated and even hailed as a "memory eraser."
In 2025, Sanofi agreed to acquire Vigil Neuroscience and obtained the latter's oral small molecule VG-3927 for the treatment of Alzheimer's disease (AD), entering the AD treatment field.
It is obvious that AD is at a historical turning point from "invincible" to "preventable and controllable". Every memory that is reignited may be the key to unlocking the future. Perhaps this moment will be the beginning of the end of Alzheimer's disease!
Disease status
AD diagnosis and treatment market continues to expand
1. AD has a complex pathogenesis and is the main subtype of dementia
AD is a neurodegenerative disease with progressive cognitive dysfunction as its main clinical feature. Its pathological characteristics include Aβ plaque deposition, neurofibrillary tangles (NFTs) formed by hyperphosphorylation of Tau protein, neuronal loss and synaptic damage. As the most common cause of dementia (accounting for about 70% of cases), AD has become a major public health problem causing disability and death among the elderly worldwide.
2. The increasing prevalence of AD drives the continued expansion of the diagnosis and treatment market
As the global population ages, the number of people with dementia is expected to surge from 55 million in 2019 to 139 million in 2050[1]. According to the World Health Organization, the global cost of AD and related dementias was US$1.3 trillion per year in 2019, and this cost is expected to reach US$2.8 trillion in 2030. Against this backdrop, the AD diagnosis and treatment market will see significant growth. It is expected that the global AD diagnosis and treatment market size will increase from US$7.65 billion in 2024 to US$9.78 billion in 2029, with a compound annual growth rate of 5.06%.
The incidence and economic burden of AD and related dementias in China are equally severe. In 2021, there were 16.99 million patients with Alzheimer's disease and other dementias in China, with a prevalence of 1194.2 per 100,000 people[2]. It is predicted that by 2030, 2040 and 2050, the number of AD patients over 60 years old in China will reach 19.11 million, 24.71 million and 27.65 million respectively, and the corresponding direct economic burden is expected to reach 973.8 billion yuan, 1692.2 billion yuan and 2544.8 billion yuan[3]. China is currently accelerating into a stage of deep aging, which is driving the continuous expansion of the AD diagnosis and treatment market, bringing new opportunities and challenges to the development of the industry.
A diagnostic revolution
From PET to blood tests
Clinically, the progression of Alzheimer's disease is divided into five stages: preclinical AD, mild cognitive impairment caused by AD, and mild, moderate, and severe dementia. In the early stages of the disease (i.e., preclinical and early stages caused by Alzheimer's disease), patients show slow and variable disease progression, with little evidence of cognitive decline. As the severity of the disease increases (mild, moderate, and severe Alzheimer's disease), significant impairment in cognitive areas, functional decline, and behavioral changes become apparent.
The most commonly used clinical methods are imaging examination, cerebrospinal fluid biomarker examination, and blood biomarker examination. The technical means involved mainly include MRI, CT, PET, immunodiagnosis, mass spectrometry, etc. [3]. At present, the main diagnostic method for AD is combined diagnosis.
PET imaging is the "gold standard" for diagnosis, but its high cost limits its clinical popularity. The current mainstream technologies include three categories: Aβ-PET, Tau-PET, and FDG-PET. Among them, Aβ-PET has become the most mature commercial solution due to its value in early diagnosis, differential diagnosis, and treatment follow-up. In 2023, Xiantong Pharmaceutical's fluoro[18F]betabenone injection was approved by NMPA. It is the first Aβ imaging agent approved in China and the first PET imaging agent approved in China in nearly two decades. This breakthrough provides a powerful "weapon" for the early, accurate, and non-invasive diagnosis of AD in China.
Cerebrospinal fluid testing is highly traumatic and has poor patient compliance, so only Roche has been approved in China. Cerebrospinal fluid testing requires samples to be obtained through lumbar puncture. Its trauma leads to poor compliance among elderly patients and is difficult to use for large-scale screening. Currently, only Roche Diagnostics' AD series test kits were approved through Hainan's clinical urgent need channel in 2024, which can quantitatively detect Aβ-42, p-Tau and t-Tau proteins. Although this technology has the advantage of precision, the operational threshold and patient acceptance are still the main bottlenecks for its promotion.
Blood testing may be the most suitable technology for large-scale early screening of AD. Blood testing can not only detect early patients, but also be implemented on a large scale, which has obvious advantages. On May 16, 2025, the FDA officially approved the listing of the first in vitro diagnostic reagent that can assist in the diagnosis of AD through blood testing - Lumipulse G (Fujirebio Diagnostics). This product can measure the ratio of two proteins (p-Tau217 and beta-amyloid 1-42) in the blood.
As of 2024, there are 47 AD blood test kits approved by NMPA in China. The biomarkers detected by the approved kits are mainly core AD biomarkers, including plasma p-Tau181, Aβ42/Aβ40, p-Tau217, etc.
1. AD therapeutic drugs: from symptomatic treatment to disease modification
In terms of treatment, the treatment of Alzheimer's disease patients mainly includes drug therapy and non-drug therapy (such as risk factor control, physical exercise, cognitive training, etc.
At present, the AD treatment drugs that have been marketed mainly include cholinesterase inhibitors (donepezil, rivastigmine, galantamine), glutamate receptor antagonists (memantine hydrochloride) and brain-gut axis regulating drugs (mannitol sodium capsules), all of which are mainly used to improve the clinical symptoms of AD. In recent years, new drugs targeting disease-modifying therapy (DMT) have made breakthrough progress, especially monoclonal antibodies targeting Aβ have been approved for marketing, marking a new stage of disease modification in AD treatment.
Currently, there are 193 AD drugs under clinical development worldwide covering multiple targets, among which Aβ-targeted drugs still dominate with 21 under development, followed by Tau-targeted drugs (14) and cholinesterase AChE-targeted drugs (5).
2. Aβ targeted therapy: Monoclonal antibody drugs successfully verified the drugability of the target, opening a new era of disease-modifying treatment for AD
Although three Aβ monoclonal antibodies have been approved by the FDA in recent years, and on the surface, they are in the limelight for a while, their poor compliance caused by intravenous infusion, safety risks such as ARIA, and high costs (such as the annual fee of lencanezumab is $26,000) are still major limitations. The withdrawal of adunumab from the market in 2024 further warns the criticality of the efficacy-risk balance.
From the perspective of R&D strategy, the successful validation of Aβ targets has opened up space for continuous innovation for small molecule inhibitors. As the fastest-progressing oral Aβ aggregation inhibitor, the Phase III clinical data of valtrimisad (ALZ-801) is of great significance: although the primary endpoint was not reached in the overall population (11% benefit, p=0.607), it showed significant efficacy in the early MCI subgroup (ADAS-Cog13 improved by 52%, p=0.0041) and brain protection (the benefit ratios of hippocampal volume, whole brain cortical thickness and whole brain volume were 26% (p=0.0042), 35% (p<0.0001) and 22% (p=0.0267) respectively). This result not only verifies the unique value of small molecule drugs in early disease intervention, but its oral administration and safety advantages also highlight its differentiated competitiveness relative to monoclonal antibodies.
Although existing Aβ monoclonal antibodies can delay cognitive decline, their benefit-risk ratio and accessibility still restrict their widespread application. The industry urgently needs to optimize the administration method (such as oral preparations), develop precise biomarkers, and explore combination therapies to break through the limitations of single targets.
3. Tau targeted therapy: The first new drug HMTM is expected to be launched in the near future
Tau protein is the main component of neurofibrillary tangles, another major pathological feature of Alzheimer's disease. Currently, there are more than 40 Tau protein targeted therapeutic drugs under active development worldwide, of which 10 have entered the mid-to-late stage of research. The main development strategies include: Tau protein post-translational modification regulators, Tau protein aggregation inhibitors, Tau protein expression inhibitors, and active and passive immunotherapy based on Tau protein.
Among them, the fastest progress is the first oral anti-Tau protein therapy developed by Zhuorui Pharmaceuticals, hydrogen methylthionine mesylate (HMTM). This therapy has submitted an application for marketing authorization in the UK in July 2024 and is expected to become an innovative drug for the treatment of mild cognitive impairment and mild to moderate dementia caused by Alzheimer's disease.
summary
The diagnosis and treatment of Alzheimer's disease is undergoing a revolutionary transformation from "treating the symptoms" to "treating the cause".
With the popularization of technologies such as blood biomarkers and AI imaging diagnosis, the accuracy of early screening has been significantly improved; breakthroughs in disease-modifying therapies, especially the success of Aβ targeted drugs, have made "reversing the course of the disease" possible for the first time; and the collaborative innovation of medical insurance payment and commercial insurance will greatly lower the threshold for the accessibility of innovative therapies.
In the next five years, precise diagnosis, personalized treatment and diversified payment will become the main themes of the industry, rewriting the "forgotten fate" of hundreds of millions of patients.
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