Amylyx Ends ORION Trial

The pharmaceutical company Amylyx, Inc. announced results from an interim analysis of its Phase2b ORION trial testing AMX0035 in people with progressive supranuclear palsy (PSP). The Phase2b ORION trial tested whether AMX0035 is safe and tolerable in people with PSP, and assessed its effect on disease progression as measured by the PSP Raing Scale (PSPRS) over a 52-week double-blind phase. The analysis showed that AMX0035 did not show differences compared to placebo on primary or secondary outcome measures.

Based on these results, Amylyx will discontinue the Phase 2b ORION trial and open label extension and will not initiate the Phase 3 portion of the trial. Amylyx shared that this decision was not driven by any safety concerns. The decision to discontinue the ORION program is due to AMX0035 not showing differences compared to placebo on slowing PSP disease progression.

If you are participating in the ORION trial, you will receive guidance from your trial site about next steps.   

You can track current and future trials testing other therapeutics here or on clinicaltrials.gov. There are other drugs for PSP are in earlier stages of testing that may enter trials over the next few years.

“Clinical trials are, by design, experiments—and in neurodegenerative diseases, they are especially difficult. While this outcome is disappointing for our community, every study adds to our knowledge and brings us closer to breakthroughs. Encouraging progress in Alzheimer’s and Parkinson’s research shows us that success is possible, and we believe PSP is closer to benefiting from that momentum” – Kristophe Diaz, PhD, CEO , CurePSP.  

CurePSP remains committed to supporting efforts to test potential treatments for people with PSP. Access to new treatments is of the highest priority for our organization and our community. There are currently no disease-modifying treatments for PSP, and we will continue to stay closely connected to biotechnology and pharmaceutical partners pursuing trials in PSP in support of our mission to treat and cure PSP.  

You can contact CurePSP at info@curepsp.org if you have additional questions.

View the Amylyx press release here: https://investors.amylyx.com/news-releases/news-release-details/amylyx-pharmaceuticals-discontinue-orion-program-amx0035