ALS News Today: First Patient Dosed in a Phase 1 Study Testing AMX0114 in ALS

The first patient has been dosed in a Phase 1 clinical study testing AMX0114, an experimental therapy being developed by Almylyx Pharmaceuticals for amyotrophic lateral sclerosis (ALS). The treatment targets calpain-2, a protein believed to contribute to nerve cell damage in ALS.

Called LUMINA (NCT06665165), the study was cleared to begin in the U.S. after the Food and Drug Administration (FDA) lifted a clinical hold in January, allowing Amylyx to start screening, enrolling, and dosing participants. The trial aims to recruit up to 48 people with ALS at multiple clinical sites throughout North America. Currently, enrollment is ongoing at a site in Ontario.

Participants will be randomly assigned to receive either AMX0114 or a placebo via intrathecal injection, that is, into the spinal canal. Each participant will receive up to four doses, given once a month. For every person receiving a placebo, three will receive the therapy, which is being tested across multiple ascending doses. Early results from the first participants are expected this year.