Amylyx Pharmaceuticals announced recently that it would end its phase 2b trial of its drug AMX0035 for the treatment of progressive supranuclear palsy (PSP). The trial will not proceed to phase 3 due to a lack of impact of the drug on participant outcomes after 24 weeks, but the work done still advances the field’s understanding of PSP’s biology.

No Differences Between Test and Placebo Groups

According to the company, the group treated with AMX0035 did not show differences from the placebo group in outcomes designed to test the effectiveness of the drug. The primary measure used to analyze participants was the Progressive Supranuclear Palsy Rating Scale, which assesses multiple areas of disease severity. Safety data were consistent with the prior phase 1 study, with the drug being generally well-tolerated by participants. Because of the lack of significant results, Amylyx is ending the current trial and will not continue with a phase 3 trial and an open-label extension.

Trial Still Advances Understanding of PSP’s Biology

PSP is one of three FTD disorders featuring primarily movement-based symptoms, and like corticobasal syndrome and Alzheimer’s disease, is associated with dysfunction of the tau protein. While scientists know that the formation of abnormal accumulations of dysfunctional tau is a key driver of PSP, other elements that may contribute to the disorder are not well understood.

Amylyx’s experimental drug   While not the desired effect, the data from the trial provide valuable insights into the underlying biology of PSP. Narrowing down potential causes of a disorder and which targets are effective can help scientists prioritize approaches with a greater chance of success.

Similarly to the FOCUS-C9 trial, which ended under similar circumstances, researchers can also gain understanding into trial designs which are feasible, formulations of drugs which are feasible, and the data can also provide more insights into the behavior of specific biomarkers which were included in the trial.

Participate in FTD Research

Are you interested in participating in other studies for FTD? Sign up at the FTD Disorders Registry to learn of studies recruiting participants when they match your interests. You may also be eligible for the online Registry Research Study to share insights about diagnosis, family history, lived experience, genetic testing, medical records, and more. Numbers have power. The more participants, the more we can help researchers design studies for which people are ready and willing to participate.

Learn more about the many other options to join in research on the AFTD website. If you have questions or concerns about research participation, contact the AFTD HelpLine at 1-866-507-7222 or info@theaftd.org.