Drug Database
AD

AD-201 (AD 201 / AD201)

✓ Approved

Addpharma · Small Molecule · Small Molecule

What is AD-201?

AD-201 is a small molecule developed by Addpharma. It is approved for therapeutic indications via unknown.

Drug Profile

Brand NamesAD 201, AD201
CompanyAddpharma
Drug ClassSmall Molecule
RouteUnknown
StatusApproved
Sources: ClinicalTrials.gov, Addpharma

Therapeutic Indications

AD-201 is developed for 2 unique indications across 2 therapeutic areas.

Therapeutic AreaConditionPhase
Vascular disordersHypertension✓ Approved
Metabolism and nutrition disordersHyperlipidaemia✓ Approved

Related Research Articles

PubMedJournal of nanobiotechnology2026-11-12

A peripheral proteomic signature of Alzheimer's disease is identified in the plasma extracellular vesicles of mild cognitive impairment patients from a memory clinic: the BIOPEXAL study.

Capdevila-Bayo Maria M, Rossi Rosanna R, de Rojas Itziar I, Puerta Raquel R et al.

Alzheimer's disease (AD) is commonly diagnosed when neuronal damage is already established and irreversible. Achieving an accurate differential diagnosis in the preclinical and mild cognitive impairment (MCI) stage is one of the greatest challenges nowadays. Nanotechnological analysis of plasma extracellular vesicles (pEVs) are gaining attention as a promising tool for the early detection of AD pathology. This study aims to evaluate the proteomic profile of pEVs from patients with MCI and AD dementia to explore their potential as AD screening tools. pEVs were isolated by ultracentrifugation from 144 patients with MCI A-T-, MCI A+T+, and AD dementia. Nanoparticle tracking analysis and cryo-TEM were used to characterize the pEVs. CSF, serum and pEVs proteomics were carried out by using the multiplex PEA technology of Olink® proteomics, Inflammation and Neurology Explore 384 panels (768 proteins). Characterization results showed that isolated plasma fraction corresponded in shape, size and concentration to EVs. Many pEVs neurology proteins involved in AD pathology significantly correlated (r > ± 0.30, p < 0.05) with their CSF homonyms, but not with their serum's. pEVs' proteome correlated with common AD signatures (CSF Aβ42 and pTau181, plasma pTau181, MMSE, NBACE, and Qalb) showing similar patterns to those observed with CSF biomarkers. Several pEVs neurology proteins didn't exhibit differences between the MCI A+T+ and AD dementia groups, whilst they did with MCI A-T-. Proteins in pEVs showed strong correlations with several measures of brain atrophy in MRI. Several neurology pEV proteins predicted conversion from MCI to AD dementia. Moreover, some of these showed a significant diagnostic accuracy of AD pathology. Preliminary results suggest that EVs biomarker signature could reflect AD pathology in the prodromal stages of AD continuum. However, further experiments are still needed for a better understanding of EVs' role in AD development and pathology dissemination.

PMID 43015837
Read full article →
PubMedNeurological research2026-11-12

Platelets in Alzheimer's disease: pathogenic mechanisms and therapeutic implications.

Al-Jehani Hosam H, Al-Kuraishy Hayder M HM, Eliwa Duaa D, Batiha Gaber El-Saber GE

This review examines the role of platelets, the primary peripheral source of amyloid precursor protein (APP) and amyloid-beta (Aβ), in Alzheimer's disease (AD) pathogenesis, focusing on the bidirectional feedback loop between peripheral platelet over-activation and central neuropathology, alongside potential antiplatelet therapies. A narrative review was conducted evaluating literature on platelet biological functions, pathological shifts in platelet APP processing in AD, mechanisms of blood-brain barrier (BBB) breakdown, and the therapeutic potential of antiplatelet agents (e.g. aspirin, clopidogrel) and adenosine A2A receptor modulators. In AD patients, platelet APP expression is markedly upregulated, shifting toward amyloidogenic processing via decreased α-secretase and increased β-secretase activity. Activated platelets breach the BBB, release pro-inflammatory mediators into the brain parenchyma, and promote cerebral Aβ deposition. Reciprocally, central AD pathology further drives systemic platelet activation, creating a self-reinforcing disease loop. A clear, bidirectional relationship may exist between activated platelets and AD neuropathology. Because peripheral platelets actively fuel central amyloid accumulation and neuroinflammation, targeting platelet activation may be a viable strategy to mitigate AD risk and slow disease progression.

PMID 43016150
Read full article →
PubMedCurrent aging science2026-11-12

Glymphatic Dysfunction in Alzheimer's Disease: Mechanisms, Biomarkers, and Emerging Therapeutic Perspectives.

Mehrabadi Shima S, Samarghandian Saeed S

Alzheimer's disease (AD) is a neurodegenerative disorder characterized by progressive cognitive decline, with hallmark pathological features including amyloid-beta plaques, tau tangles, and neuronal loss. The glymphatic system, a recently discovered network responsible for brain waste clearance, plays a crucial role in maintaining cerebral homeostasis by facilitating the removal of neurotoxic metabolites, including amyloid-beta. Disruption of glymphatic function has emerged as a key factor in AD pathophysiology, contributing to the accumulation of toxic proteins and accelerating neurodegenerative processes. This narrative review explores the complex interplay between glymphatic dysfunction and AD. We discuss the mechanisms of the glymphatic system, emphasizing its reliance on cerebrospinal fluid (CSF) and interstitial fluid flow to clear metabolic waste, and how this process is closely linked to sleep cycles. In AD, impaired glymphatic function, exacerbated by aging and sleep disturbances, accelerates the buildup of amyloid-beta and tau, driving neurodegeneration. Furthermore, sleep disturbances commonly observed in AD patients further hinder glymphatic activity, creating a vicious cycle that exacerbates cognitive decline. Emerging therapeutic approaches aimed at enhancing glymphatic function-including sleep regulation, pharmacological agents, and lifestyle interventions-are promising, but most findings remain preliminary and largely based on animal studies or early clinical observations. These strategies should therefore be considered exploratory until validated in large-scale human trials. By restoring efficient waste clearance, such interventions may offer novel avenues for slowing or preventing AD progression.

PMID 43016269
Read full article →
PubMedJACC. Case reports2026-11-12

Aortic Dissection After Stage 1 Palliation.

Pook Caitlin N CN, Ganigara Madhusudan M, Javois Alexander J AJ, Wong Joshua H JH et al.

Pediatric aortic dissection (AD) is rare and usually associated with hypertension or aortopathies. In this case, we present a Stanford type B dissection in an asymptomatic patient with single ventricle heart disease without known genetic aortopathy following stage 1 palliation. Dissection was diagnosed on routine catheterization and confirmed on computed tomography angiography. There was no progression, so no surgical intervention was pursued. He underwent stage 2 palliation at age 6 months and continues to do well. AD is rare in pediatrics, especially infants without aortopathy. A single iatrogenic case report after hybrid palliation has been described. Our patient had at least 2 risk factors, aortic coarctation and intermittently elevated blood pressure, that could have predisposed him to dissection. AD is rarely seen following the Norwood operation. Stanford type B AD can be safely medically managed in pediatric patients as in the adult guidelines.

PMID 43018521
Read full article →
PubMedInternational journal of general medicine2026-11-12

Multi-Omics Mendelian Randomization Identifies DDX17 as a Potential Mediator Linking Gut Microbiota-Immune-Metabolism Axis to Alzheimer's Disease.

Xiao Zhen Z, Qin Yaman Y, Dong Mingyou M, Wei Suyuan S et al.

The gut microbiota-immune-metabolism axis has been implicated in Alzheimer's disease (AD) pathogenesis; however, the key host molecular targets linking peripheral dysregulation to central neurodegeneration remain elusive. In this study, we systematically integrated large-scale GWAS data covering 1,903 gut microbiota taxa, 14,696 immune cell features, and 34,843 metabolite features with AD GWAS summary statistics, using two-sample Mendelian randomization (MR) to identify causal genes. These genes were intersected with differentially expressed genes from three GEO datasets, followed by protein-protein interaction network construction using four Cytoscape algorithms (MCC, EPC, DMNC, Degree) and feature selection via three machine learning methods (Support Vector Machine, Random Forest, LASSO). Single-cell transcriptomics (GSE188545) localized key gene expression, Support Vector Machine virtual knockout (scTenifoldKnk) analyzed downstream regulatory networks, with in vitro validation performed in okadaic acid-treated SH-SY5Y cells using RT-qPCR and Western blotting. MR analysis revealed multiple causal associations among microbial taxa, immune phenotypes, metabolites, and AD. Integration of MR causal genes with DEGs, PPI network analysis, and machine learning refinement prioritized six key genes: BYSL, DDX17, HSPA6, RRN3, C4B, and NCKAP1L. Among them, only DDX17 showed robust expression in AD-associated neuronal populations in single-cell transcriptomics, whereas the other five genes showed low expression and may act through non-cell-autonomous mechanisms. Virtual knockout predicted that the PI3K/AKT pathway was the most significantly enriched downstream of DDX17 perturbation. In vitro, DDX17 was significantly upregulated in an OA-induced AD cell model, and siRNA-mediated knockdown partially reversed tau hyperphosphorylation (total P-tau and P-tau Thr217), while restoring AKT phosphorylation at Thr308 and Ser473. These findings suggest that DDX17 is a potential downstream host candidate linking peripheral dysregulation to tau pathology in AD, partially through PI3K/AKT pathway modulation. However, these results are primarily hypothesis-generating, and direct mechanistic and in vivo validation remain essential.

PMID 43017601
Read full article →
PubMedBMC geriatrics2026-11-12

An impaired excitatory-inhibitory balance in the right inferior temporal gyrus of patients with Alzheimer's disease correlated with executive function.

Liu Xiaozheng X, Guo Zhongwei Z, Fu Bingqi B, Jia Xize X et al.

Preliminary research indicates that an imbalance between excitation and inhibition may be an early pathological feature of Alzheimer's disease. However, the relevant brain networks, neurotransmitters and genetic mechanisms are still unclear. This study enrolled 27 patients with Alzheimer's disease (AD), 86 patients with mild cognitive impairment MCI, and 85 healthy controls (HC), all aged 65 or older, from the ADNI3 cohort in May 2024. We employed analysis of covariance to compare abnormalities in the excitation-inhibition balance across the three groups. We performed a transcriptomic analysis of abnormal excitation-inhibition imbalance. To elucidate the characteristics of the underlying brain networks, we extracted brain regions exhibiting differences in the balance of excitation and inhibition for seed-based functional connectivity analysis. Compared to the HC group, AD exhibits a significant putative excitatory-inhibitory imbalance in the right inferior temporal gyrus and superior orbital frontal gyrus. The Hurst exponent of the right inferior temporal gyrus was significantly correlated with executive function. Transcriptomic analysis revealed that the abnormal excitation-inhibition imbalance observed in AD patients is significantly associated with GABA and mGluR5 receptors, as well as with genes that are enriched in the GABAergic synapse pathway. Functional connectivity analysis using the right inferior temporal gyrus as a seed point revealed significant abnormalities in the frontoparietal control network in AD patients. Our findings suggest that an imbalance between excitation and inhibition in the inferior temporal gyrus is a potential pathological mechanism underlying AD. This imbalance also leads to functional abnormalities in the frontoparietal control network associated with executive functions.

PMID 43015644
Read full article →

+9996 more articles available with a free account

Sign up free to view all articles →

Ask about AD-201