Amylyx Pharma to drop ORION program

Cambridge, USA-based drug developer Amylyx Pharmaceuticals (Nasdaq: AMLX) yesterday announced the decision to discontinue the ORION program of AMX0035 (sodium phenylbutyrate [PB] and taurursodiol [TURSO, also known as ursodoxicoltaurine]) in adults living with progressive supranuclear palsy (PSP).

Amylyx explained that AMX0035 did not show differences compared to placebo on primary or secondary outcomes at Week 24. Based on these results, the company will discontinue the Phase IIb trial and open-label extension and will not initiate the Phase III portion of the program. Safety data were consistent with safety data from prior studies, and AMX0035 continued to be generally well-tolerated.

“We set a high bar for AMX0035 in PSP and made a commitment to base our decision-making on the totality of the data and the potential for clinically meaningful outcomes for those living with PSP. While we are disappointed in these results, we believe these data will inform the PSP trial literature as well as deepen scientific understanding of this devastating disease. We extend our gratitude to the participants, their families and care partners, the ORION sites, and the entire PSP community for their collaboration on this study,” said Dr Camille Bedrosian, chief medical officer at Amylyx.