HOW DOES A DRUG GET DEVELOPED?

DISCONTINUED: Stages of Development for TAK-831

The drug development process can be thought of as a series of stages, and drugs must successfully pass through each stage to become available to patients. This treatment has been evaluated, and the program has been discontinued. Thus, it is not in the pipeline.

Based on studies in FA mouse model, there was preliminary evidence that TAK-831 might provide symptomatic benefit to neurological and motor function. It was these studies in the mouse model that lead to a clinical trial of TAK-831 in individuals with FA

2017: A Phase II clinical trial of TAK-831 evaluated efficacy, safety and tolerability in 65 individuals with FA, ages 18-55. Multiple US sites participated in the study including Children’s Hospital of Philadelphia, Ohio State University, University of Iowa, University of Florida, University of South Florida and University of California Los Angeles.

In addition to safety and tolerability, this study evaluated upper limb function and manual dexterity using the nine-hole pegboard test as well as other neurological and performance assessments.

2018: While TAK-831 was found to be safe and well-tolerated, it did not demonstrate efficacy based on primary and key secondary endpoints and development was stopped.