Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
ACH-0144471 · 1 trial · 1 indication
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 = Hgb levels at Week 25 - Baseline Hgb levels. Baseline was the baseline value from the primary Study ACH471-100.
Change from Baseline = reticulocyte count at Week 25 - Baseline reticulocyte count. Baseline was the baseline value from the primary Study ACH471-100.
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.
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 = free Hgb at Week 25 - Baseline free Hgb. Baseline was the baseline value from the primary Study ACH471-100.
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.
| Arm | Type | Description |
|---|---|---|
| ACH-0144471 | EXPERIMENTAL | All participants will receive ACH-0144471 during the treatment period. |
| Name | Type | Description |
|---|---|---|
| ACH-0144471 | DRUG | ACH-0144471 will be administered to all participants enrolled in the study. |
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. ...
ACH-0144471 is an investigational small molecule being studied for the treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH), a rare hematologic disorder. It is currently in Phase 2 clinical development and is not yet approved by regulatory authorities.
ACH-0144471 is being developed by AstraZeneca PLC, a multinational pharmaceutical company listed on the stock exchange under the ticker symbol AZN. The drug is in Phase 2 clinical trials for Paroxysmal Nocturnal Hemoglobinuria.
ACH-0144471 is in Phase 2 clinical development. It is an investigational drug and has not been approved by the FDA or other regulatory agencies. Its safety and efficacy are still being evaluated in clinical trials.
ACH-0144471 has one completed Phase 2 clinical trial registered under NCT03181633, titled 'A Long-Term Treatment Study of ACH-0144471 in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)'. The trial enrolled 8 participants and was conducted in Italy, New Zealand, and South Korea.
The mechanism of action of ACH-0144471 has not been disclosed in available information. It is a small molecule being investigated for the treatment of Paroxysmal Nocturnal Hemoglobinuria, but its specific molecular target is not publicly detailed.