Amylyx Pharmaceuticals Inc
NASDAQ:AMLX
Amylyx Pharmaceuticals Inc
Amylyx Pharmaceuticals, Inc. operates as a biopharmaceutical company, which provides disease-modifying solutions for neurodegenerative diseases. The company is headquartered in Cambridge, Massachusetts and currently employs 205 full-time employees. The company went IPO on 2022-01-07. The firm is developing therapies for treatment paradigm for amyotrophic lateral sclerosis (ALS), and range of neurodegenerative diseases by keeping neurons alive. The firm is pursuing commercialization of its product candidate, AMX0035. AMX0035 is an investigational product comprised of two complementary active agents, sodium phenylbutyrate (PB) and taurursodiol (TURSO) also known as ursodoxicoltaurine. AMX0035 is designed to target the endoplasmic reticulum and mitochondrial-dependent neuronal degeneration pathways in ALS and other neurodegenerative diseases. AMX0035 keep neurons alive under a variety of different conditions and stresses, including in in vitro models of neurodegeneration, endoplasmic reticulum stress, mitochondrial dysfunction, oxidative stress and disease-specific models of a variety of other conditions, as well as in vivo models of Alzheimer’s Disease (AD) and multiple sclerosis.
Amylyx Pharmaceuticals, Inc. operates as a biopharmaceutical company, which provides disease-modifying solutions for neurodegenerative diseases. The company is headquartered in Cambridge, Massachusetts and currently employs 205 full-time employees. The company went IPO on 2022-01-07. The firm is developing therapies for treatment paradigm for amyotrophic lateral sclerosis (ALS), and range of neurodegenerative diseases by keeping neurons alive. The firm is pursuing commercialization of its product candidate, AMX0035. AMX0035 is an investigational product comprised of two complementary active agents, sodium phenylbutyrate (PB) and taurursodiol (TURSO) also known as ursodoxicoltaurine. AMX0035 is designed to target the endoplasmic reticulum and mitochondrial-dependent neuronal degeneration pathways in ALS and other neurodegenerative diseases. AMX0035 keep neurons alive under a variety of different conditions and stresses, including in in vitro models of neurodegeneration, endoplasmic reticulum stress, mitochondrial dysfunction, oxidative stress and disease-specific models of a variety of other conditions, as well as in vivo models of Alzheimer’s Disease (AD) and multiple sclerosis.
Trial timeline: LUCIDITY enrollment is complete, final patients expected to be randomized and dosed this month, with top-line data anticipated in Q3 2026.
Primary goal: The company is focused on avexitide (90 mg once daily) and preparing NDA sections and commercial readiness to enable a potential 2027 launch if LUCIDITY is positive.
Market size: Amylyx estimates ~160,000 people living with post-bariatric hypoglycemia (PBH) in the U.S., based on literature and independent claims analyses.
Prior efficacy signal: Management highlighted prior avexitide data — a 64% least-squares mean reduction in the composite rate of Level 2 and Level 3 hypoglycemic events with p=0.0031 in one Phase II dataset (also discussed elsewhere as ~66% effect in a Phase IIb context).
Financial position: Cash and marketable securities were $317 million at 12/31/2025, providing an anticipated cash runway into 2028 to fund LUCIDITY readout and potential approval/launch activities.
Pipeline progress: AMX0318 (long-acting GLP-1 antagonist) nominated as development candidate; IND-enabling work underway with an IND target in 2027. AMX0114 (ALS) received Fast Track designation and cohort 2 enrollment in LUMINA expected to complete this month.
