Amylyx Pharmaceuticals

Historical price data

Amylyx Pharmaceuticals is dedicated to creating new possibilities for people living with debilitating neurodegenerative diseases by targeting key cellular pathways of neurodegeneration. The company achieved a landmark success with the U.S. FDA approval of RELYVRIO (AMX0035) for ALS in September 2022, transitioning from a clinical-stage to a commercial-stage entity. Its strategy involves maximizing the global potential of RELYVRIO while advancing its next-generation candidate, AMX0114, and exploring the application of its dual-mechanism platform in other diseases like Wolfram syndrome and progressive supranuclear palsy (PSP). Amylyx faces the critical challenge of commercial execution and confirming the clinical benefit of its lead asset in an ongoing confirmatory trial.

A dual-mechanism platform targeting endoplasmic reticulum stress and mitochondrial dysfunction, key pathways in neuronal cell death, applied through small molecule combinations and next-generation antisense oligonucleotides.

12 drugs in pipeline, 5 in Phase 3

Total raised: $255M

In ALS, Amylyx competes with Mitsubishi Tanabe's edaravone, generic riluzole, and Biogen's tofersen for a genetic subset. Its differentiation is based on oral administration, a novel dual-mechanism, and survival data. In PSP and Wolfram syndrome, it faces little to no competition from approved disease-modifying therapies.