Recent Updates
Recently added Catalysts

ALXN2060

Phase 3

Symptomatic Transthyretin Amyloid Cardiomyopathy | Small molecule | Cardiovascular |AstraZeneca PLC|Last Updated: Jan 9, 2026

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
Premium
Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
Premium
Trial Design
UNCONTROLLEDBiomarker
Total Trials1
Total Enrollment25
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT04622046A Phase 3 Study of ALXN2060 in Japanese Participants With Symptomatic ATTR-CMPHASE3 COMPLETED 25Nov 13, 2020Aug 21, 2025Jan 9, 202611 Japan
Unlock Drug Trial Details
Study Endpoints
Primary Endpoints
Part A: Change From Baseline To Month 12 Of Treatment In Distance Walked During The Six-minute Walk Test (6MWT)
Baseline, Month 12

The 6MWT measures how far a participant can walk in 6 minutes. Change from baseline was calculated as the difference between the distance walked during the 6MWT at 12 months and the distance walked during the 6MWT at baseline. To determine the baseline, at least 2 6MWTs were conducted \> 24 hours to ≤ 3 weeks apart prior to the first dose of ALXN2060. The baseline 6MWT is the average of the total distance walked by participants for the 2 qualifying 6MWTs that met all the protocol-defined criteria. Least squares mean change from baseline data were adjusted for baseline measures and visits.

Parts A and B: Number of Cardiovascular (CV)-Related Hospitalizations Over A 30-month Period
30 months

CV-related hospitalization was defined as the mean number of CV-related hospitalizations per participant per year over a 30-month period. CV-related hospitalizations were also reported as adverse events and were reviewed and adjudicated by an independent Clinical Events Committee (CEC).

All-cause Mortality (ACM) Over A 30-month Period
30 months

ACM was assessed as time from the date of first initiation of study treatment to the date of death during a 30-month period, and was analyzed using Kaplan-Meier analysis. Data are reported for the number of participants with ACM over the 30-month period.

Secondary Endpoints
Parts A and B: Change From Baseline In Distance Walked During The 6MWT
Baseline, Months 6, 9, 18, 24 and 30
Parts A and B: Change From Baseline In The Kansas City Cardiomyopathy Questionnaire Overall Score (KCCQ-OS)
Baseline, Months 6, 9, 12, 18, 24 and 30
Parts A and B: Number of Participants With Treatment-emergent Adverse Events (AEs), Serious Adverse Events (SAEs) and AEs Leading to Treatment Discontinuation
Up to Month 30
Unlock Study Endpoints
Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
ALXN2060EXPERIMENTALParticipants will receive ALXN2060.
Interventions
NameTypeDescription
ALXN2060DRUGALXN2060 tablets will be administered twice daily at a dose of 800 milligrams.
Unlock Study Design Details
Eligibility Criteria
SexALL
Healthy VolunteersNo
Study Sites11

Inclusion Criteria: 1. Established diagnosis of ATTR-CM with either wild-type TTR or a variant TTR genotype. 2. History of heart failure evidenced by at least 1 prior hospitalization for heart failure or clinical evidence of heart failure without prior heart failure hospitalization manifested by si...

Countries:Japan
Unlock Eligibility Criteria