Phase 3
N=549
ATH-1017 for Treatment of Mild to Moderate Alzheimer's Disease
Alzheimer Disease · Dementia of Alzheimer Type
Bottom Line
View on ClinicalTrials.gov: NCT04488419 ↗Enrolled (actual)
549
Serious AEs
5.3%
Results posted
Apr 2025
Primary outcome: Primary: Global Statistical Test (GST) Score — -0.126; -0.208 Z-score
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- ATH-1017 (Drug); Placebo (Drug)
- Age
- Adult, Older Adult · 55+ yrs
- Sex
- All
- Sponsor
- LeonaBio
- Primary completion
- Jul 2024
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Global Statistical Test (GST) Score |
-0.126; -0.208 | — |
| SECONDARY Alzheimer's Disease Assessment Scale-Cognitive Subscale 11 (ADAS-Cog11) Change From Baseline |
-0.39; -1.09 | — |
| SECONDARY Alzheimer's Disease Cooperative Study - Activities of Daily Living, 23-Item Version (ADCS-ADL23) Change From Baseline |
-0.02; 0.65 | — |
| SECONDARY Plasma Neurofilament Light Chain (NfL) Concentrations Change From Baseline |
2.95; -0.96 | — |
Summary
This study is designed to evaluate safety and efficacy of fosgonimeton (ATH-1017) in the treatment of mild to moderate Alzheimer's disease with a randomized treatment duration of 26-weeks.
Eligibility Criteria
Key Inclusion Criteria
- Age 55 to 85 years
- Mild-to-moderate AD dementia subjects, MMSE 14-24, CDR 1 or 2 at Screening
- Clinical diagnosis of dementia, due probably to AD, by Revised National Institute on Aging-Alzheimer's Association criteria (McKhann, 2011)
- Body mass index (BMI) of ≥ 18 and ≤ 35 kg/m2 at Screening
- Reliable and capable support person/caregiver
- Treatment-free (subjects not receiving acetylcholinesterase inhibitor [AChEI] treatment), defined as:
- Treatment-naïve, OR
- Subjects who received an AChEI in the past and discontinued at least 4 weeks prior to Screening
Key Exclusion Criteria
- History of significant neurologic disease, other than AD, that may affect cognition, or concurrent with the onset of dementia
- Subject has atypical variant presentation of AD, if known from medical history, particularly non-amnestic AD
- History of brain MRI scan indicative of any other significant abnormality
- Diagnosis of severe major depressive disorder even without psychotic features.
- Significant suicide risk
- History within 2 years of Screening, or current diagnosis of psychosis
- Myocardial infarction or unstable angina within the last 6 months
- Clinically significant cardiac arrhythmia (including atrial fibrillation), cardiomyopathy, or cardiac conduction defect (note: pacemaker is acceptable)
- Subject has either hypertension or symptomatic hypotension
- Clinically significant ECG abnormality at Screening
- Chronic kidney disease with estimated glomerular filtration rate (eGFR) 2 times the upper limit of normal, or Child-Pugh class B and C
- Malignant tumor within 3 years before Screening
- Memantine in any form, combination or dosage within 4 weeks prior to Screening
- Acetylcholinesterase inhibitors in any dosage form
- The subject has received active amyloid or tau immunization (i.e., vaccination for Alzheimer's disease) at any time, or passive immunization (i.e., monoclonal antibodies for Alzheimer's disease) within 6 months of Screening
Data sourced from ClinicalTrials.gov (NCT04488419). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.