ORION Trial Ends, Reinforcing the Need for Continued FTD Research

Amylyx Pharmaceuticals recently announced that its ORION program, testing AMX0035 in progressive supranuclear palsy (PSP), did not meet its primary or secondary endpoints at 24 weeks. As a result, the company has discontinued the Phase 2b trial and deferred plans for a Phase 3 study.

While the results are disappointing, they still enrich scientific understanding. As Amylyx’s Chief Medical Officer noted, the findings “inform the PSP trial literature as well as deepen scientific understanding of this devastating disease,” and the company expressed gratitude to the participants, families, and sites involved.

Why This Matters to the FTD Community

At the FTD Disorders Registry, we know that every study – whether it meets its goals or not – adds valuable knowledge. Results like ORION are not unexpected; in fact, most trials do not advance, by design. This is how the drug development process ensures that only the most promising and safe interventions move forward. Each step, including those that stop early, provides critical insights into trial design, what does and does not work, and how we can ultimately reach effective treatments for FTD and related disorders.

Registry Director Carrie Milliard, MS, CGC, CCRC emphasizes:

“Each study brings us closer, regardless of the outcome. Trials like ORION show us where to look next, what to test, and how to adapt. Your willingness to participate transforms uncertainty into actionable insight.”

Moving Forward

• Every trial matters: Even negative results guide future studies.
• Participation is essential: People who join studies and the FTD Disorders Registry provide the data researchers rely on.
• Hope remains: Each step strengthens the path toward treatments and, one day, a cure.

Thank you to everyone who contributes to research and keeps momentum moving forward. Progress is only possible because of you.