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ACH-0144471

Phase 2

Paroxysmal Nocturnal Hemoglobinuria | Small molecule | Other |AstraZeneca PLC|Last Updated: Mar 14, 2023

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
UNCONTROLLED
Total Trials1
Total Enrollment8
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT03181633A Long-Term Treatment Study of ACH-0144471 in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)PHASE2 COMPLETED 8Jun 22, 2017Jan 4, 2022Mar 14, 20234 Italy, New Zealand +1
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Study Endpoints
Primary Endpoints
Change From Baseline in LDH Level at Week 25
Baseline, Week 25

Change from Baseline = Serum LDH levels at Week 25 - Baseline Serum LDH levels. Baseline was the baseline value from the primary Study ACH471-100.

Change From Baseline in Hgb Level in the Absence of RBC Transfusion at Week 25
Baseline, Week 25

Change from Baseline = Hgb levels at Week 25 - Baseline Hgb levels. Baseline was the baseline value from the primary Study ACH471-100.

Change From Baseline in Reticulocyte Counts at Week 25
Baseline, Week 25

Change from Baseline = reticulocyte count at Week 25 - Baseline reticulocyte count. Baseline was the baseline value from the primary Study ACH471-100.

Number of RBC Units Transfused
Baseline up to Week 169
Number of RBC Transfusion Instances
Baseline up to Week 169
Change From Baseline in PNH Clone Size at Week 25
Baseline, Week 25

The PNH clone size refers to the percentage of PNH-affected cells versus normal cells within the total cell population. Change from Baseline = PNH clone size at Week 25 - Baseline PNH clone size. Baseline was the baseline value from the primary Study ACH471-100.

Change From Baseline in AP Complement Functional Activity at Week 25
Baseline, Week 25

Serum AP functional activity was measured by the Wieslab functional immunoassay method. Change from Baseline = Serum AP functional activity at Week 25 - Baseline Serum AP functional activity. Baseline was the baseline value from the primary Study ACH471-100.

Change From Baseline in Free Hgb at Week 25
Baseline, Week 25

Change from Baseline = free Hgb at Week 25 - Baseline free Hgb. Baseline was the baseline value from the primary Study ACH471-100.

Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), Grade 3 and Grade 4 Adverse Events (AEs), And AEs Leading To Discontinuation
Baseline up to 4.5 years

An AE was as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related. An SAE was an AE that met at least 1 of the following criteria: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization for the AE, persistent or significant disability/incapacity or substantial disruption of the ability to conduct normal life functions, congenital anomaly/birth defect (in the child of a participant who was exposed to the study drug), important medical event or reaction. The intensity of an AE was graded according to the Common Terminology Criteria for Adverse Events (CTCAE) Adverse Event Severity Grading Table. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.

Secondary Endpoints
Change From Baseline in LDH Level at Weeks 49 and 169
Baseline, Weeks 49 and 169
Change From Baseline in Hgb Level in the Absence of RBC Transfusion at Weeks 49 and 169
Baseline, Weeks 49 and 169
Change From Baseline in Reticulocyte Counts at Weeks 49 and 169
Baseline, Weeks 49 and 169
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
ACH-0144471EXPERIMENTALAll participants will receive ACH-0144471 during the treatment period.
Interventions
NameTypeDescription
ACH-0144471DRUGACH-0144471 will be administered to all participants enrolled in the study.
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites4

Inclusion Criteria: * Study designed to include up to 12 participants who completed treatment in Study ACH471-100 and demonstrated clinical benefit from ACH-0144471 with no significant safety or tolerability concerns. * Negative pregnancy test for females prior to dosing and throughout the study. ...

Countries:ItalyNew ZealandSouth Korea
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