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ATH-1017

Phase 2

Alzheimer Disease | Small molecule | Neurology |LeonaBio, Inc.|Last Updated: Apr 4, 2025

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedDouble-BlindPLACEBO_CONTROLLEDDMCBiomarker
Total Trials2
Total Enrollment631
FDA Designations
No designations recorded
Clinical Trials (2)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT04491006A Study of ATH-1017 in Mild to Moderate Alzheimer's DiseasePHASE2 COMPLETED 77Nov 23, 2020May 20, 2022Jun 12, 202313 United States, Australia
NCT04488419ATH-1017 for Treatment of Mild to Moderate Alzheimer's DiseasePHASE2 COMPLETED 554Sep 28, 2020Jul 15, 2024Apr 4, 20251 United States
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Study Endpoints
Primary Endpoints
Event-related Potential (ERP) P300 Latency at Baseline
At Baseline (Day 1)

ERP P300 was a method of recording brain activity elicited by external stimuli, for example (e.g.), an oddball auditory stimulus, particularly of working memory access. The participant had to perform a task related to auditory stimuli in order to assess the P300 component (latency). The stimulus consisted of an oddball paradigm with 2 sound stimuli. Stimuli were presented through headphones and auditory stimulation for P300 was assessed in a recording lasting up to 10 minutes. It was calculated as the average across the pre-dose values at Baseline visit. Baseline was defined as Day 1.

Global Statistical Test (GST) Score
Baseline and Week 26

The Global Statistical Test (GST) score is a composite of cognition and function, calculated as the average of two change from baseline z-scores; the z-scores are calculated for the change from baseline scores for cognition (Alzheimer's Disease Assessment Scale-Cognitive Subscale \[ADAS-Cog11\]; lower value indicates improvement) and function (Alzheimer's Disease Cooperative Study - Activities of Daily Living, 23-item version \[ADCS-ADL23\] score; higher value indicates improvement). GST is a standardized score relative to the population mean. Therefore, a GST score of 0 is representative of the population mean. Since GST is a composite of two endpoints, a negative ADCS-ADL23 score is used in deriving GST. Therefore, a lower score indicates improvement, and a higher score indicates worsening. There are no defined clinically relevant thresholds for this GST score.

Secondary Endpoints
Alzheimer's Disease Assessment Scale-Cognitive Subscale (ADAS-Cog11) at Baseline
At Baseline (Day 1)
Alzheimer's Disease Assessment Scale-Cognitive Subscale 11 (ADAS-Cog11) Change From Baseline
Baseline and Week 26
Alzheimer's Disease Cooperative Study - Activities of Daily Living, 23-Item Version (ADCS-ADL23) Change From Baseline
Baseline and Week 26
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Low DoseEXPERIMENTALDaily subcutaneous (SC) injection of Low Dose ATH-1017
High DoseEXPERIMENTALDaily subcutaneous (SC) injection of High Dose ATH-1017
PlaceboPLACEBO_COMPARATORDaily subcutaneous (SC) injection of Placebo
DosageEXPERIMENTALDaily subcutaneous (SC) injection of 40mg ATH-1017
Interventions
NameTypeDescription
ATH-1017DRUGDaily subcutaneous (SC) injection of ATH-1017 in a pre-filled syringe
PlaceboDRUGDaily subcutaneous (SC) injection of Placebo in a pre-filled syringe
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Eligibility Criteria
Age Range55 Years — 85 Years
SexALL
Healthy VolunteersNo
Study Sites13

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) * Reliable and capable support pers...

Countries:United StatesAustralia
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