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NCT04886063

Open Label Study of ATH-1017 for Treatment of Mild to Moderate Alzheimer's Disease

Terminated Phase 2, PHASE3 Results posted Last updated 1 April 2025
What this trial tests

Phase 2, PHASE3 trial testing ATH-1017 in Alzheimer Disease in 423 participants. Terminated before completion.

Timeline
30 June 2021
Primary endpoint
23 October 2024
23 October 2024

Quick facts

Lead sponsorLeonaBio
PhasePhase 2, PHASE3
StatusTerminated
Study typeINTERVENTIONAL
Allocationna
Designsingle group
Maskingnone
Primary purposetreatment
Enrollment423
Start date30 June 2021
Primary completion23 October 2024
Estimated completion23 October 2024
Sites1 location across United States

Drugs / interventions tested

Conditions studied

Sponsor

LeonaBio — full company profile →

Who can join

Adults 55 to 85, any sex, with Alzheimer Disease. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability] Primary · Up to 173 weeks (study termination)

Description - To determine the safety and tolerability of ATH-1017 in subjects with mild to moderate Alzheimer's disease (AD) who completed the 26-week randomized treatment in Study ATH-1017-AD-0201 or Study ATH-1017-AD-0202

GroupValue95% CI
ATH-1017 40 Milligrams (mg)323
ATH-1017 40 Milligrams (mg)100

Adverse events — posted to ClinicalTrials.gov

Time frame: Up to 173 weeks; the protocolled 206-week treatment duration ended early due to study termination.. Reporting threshold: 4%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

ATH-1017 40 Milligrams (mg)
Serious: 54/423 (13%)
Deaths: 4/423

Serious adverse events (58 terms)

ReactionSystemATH-1017 40 Milligrams (mg)
COVID-19Infections and infestations
PneumoniaInfections and infestations
Urinary tract infectionInfections and infestations
FallInjury, poisoning and procedural complications
SeizureNervous system disorders
Atrial fibrillationCardiac disorders
Respiratory syncytial virus infectionInfections and infestations
ArthralgiaMusculoskeletal and connective tissue disorders
Metabolic encephalopathyNervous system disorders
AnaemiaBlood and lymphatic system disorders
Monoclonal B-cell lymphocytosisBlood and lymphatic system disorders
Aortic valve incompetenceCardiac disorders
Atrioventricular block second degreeCardiac disorders
BradycardiaCardiac disorders
Coronary artery stenosisCardiac disorders
Myocardial infarctionCardiac disorders
Early onset familial Alzheimer's diseaseCongenital, familial and genetic disorders
FaecalomaGastrointestinal disorders
Oesophageal food impactionGastrointestinal disorders
Small intestinal obstructionGastrointestinal disorders
Chest painGeneral disorders
CholecystitisHepatobiliary disorders
AppendicitisInfections and infestations
COVID-19 pneumoniaInfections and infestations
CellulitisInfections and infestations
Other adverse events (6 terms — click to expand)

ReactionSystemATH-1017 40 Milligrams (mg)
Injection site reactionsGeneral disorders
COVID-19Infections and infestations
Urinary tract infectionInfections and infestations
FallInjury, poisoning and procedural complications
EosinophiliaBlood and lymphatic system disorders
Upper respiratory tract infectionInfections and infestations

Most-reported serious reactions: COVID-19, Pneumonia, Urinary tract infection, Fall, Seizure, Atrial fibrillation, Respiratory syncytial virus infection, Arthralgia.

Data from ClinicalTrials.gov NCT04886063 adverse events section.

Sponsor's own description

The objective of this study is to determine the safety and tolerability of fosgonimeton (ATH-1017) in subjects with mild to moderate Alzheimer's disease who completed the 26-week randomized treatment in Study ATH-1017-AD-0201 or Study ATH-1017-AD-0202.

Publications & conference data

6 peer-reviewed publications reference this trial (live from Europe PMC):

  1. Alzheimer's disease drug development pipeline: 2022.
    Cummings J, Lee G, Nahed P, Kambar MEZN, et al · · 2022 · cited 369× · PMID 35516416 · DOI 10.1002/trc2.12295
  2. Alzheimer's disease drug development pipeline: 2023.
    Cummings J, Zhou Y, Lee G, Zhong K, et al · · 2023 · cited 326× · PMID 37251912 · DOI 10.1002/trc2.12385
  3. Alzheimer's disease drug development pipeline: 2024.
    Cummings J, Zhou Y, Lee G, Zhong K, et al · · 2024 · cited 209× · PMID 38659717 · DOI 10.1002/trc2.12465
  4. Considerations in the clinical use of amyloid PET and CSF biomarkers for Alzheimer's disease.
    Leuzy A, Bollack A, Pellegrino D, Teunissen CE, et al · · 2025 · cited 43× · PMID 40042435 · DOI 10.1002/alz.14528
  5. Unveiling the multifaceted pathogenesis and therapeutic drugs of Alzheimer's disease: A comprehensive review.
    Peng L, Zhang Z, Li Q, Song Z, et al · · 2024 · cited 3× · PMID 39629139 · DOI 10.1016/j.heliyon.2024.e39217
  6. Fosgonimeton attenuates amyloid-beta toxicity in preclinical models of Alzheimer's disease.
    Reda SM, Setti SE, Berthiaume AA, Wu W, et al · · 2024 · cited 3× · PMID 38599894 · DOI 10.1016/j.neurot.2024.e00350

Verify or expand the search:

Other trials of ATH-1017

Trials testing the same drug.

Other recruiting trials for Alzheimer Disease

Currently open trials in the same condition.

Other LeonaBio trials

Trials by the same sponsor.

Verify against primary sources

Data sources for this page

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT04886063.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing