Symptomatic PBH affects approximately 8% of people who have undergone bariatric surgery and can lead to debilitating hypoglycemic events. It is estimated that approximately 160,000 of the more than two million people who have undergone bariatric surgery in the last decade are currently living with symptomatic PBH.^1-4 Learn more about PBH.

FDA Breakthrough Therapy, Orphan Drug Designation for hyperinsulinemic hypoglycemia (which includes PBH)

LUCIDITY will be a multicenter, randomized, double-blind, placebo-controlled Phase 3 clinical trial designed to study the efficacy and safety of avexitide in people living with PBH.

Avexitide has demonstrated highly statistically significant and clinically meaningful data with a well-tolerated safety profile across five clinical trials in PBH. Amylyx expects to begin a Phase 3 program for avexitide in PBH in Q1 2025, with data readout anticipated in 2026.

Congenital hyperinsulinism (HI) is a rare disease with limited therapeutic options characterized by hypersecretion of insulin leading to severe, persistent hypoglycemia in infants and young children.

FDA Breakthrough Therapy, Rare Pediatric Disease Designation, Orphan Drug Designation for hyperinsulinemic hypoglycemia (which includes congenital HI)

There is a clear link between avexitide’s proposed mechanism of action and the cause of hypoglycemia in congenital HI, a significant unmet need faced by this community, as well as statistically significant data from three prior avexitide clinical trials in this indication. Amylyx is engaging physicians and community experts in discussions around next steps for clinical development.

Wolfram syndrome is a rare, monogenic neurodegenerative disease that progressively impacts multiple organ systems. Wolfram syndrome is characterized by childhood-onset diabetes mellitus, optic nerve atrophy, and neurodegeneration. Learn more about Wolfram syndrome.

FDA Orphan Drug Designation, European Commission Orphan Drug Designation

HELIOS is a 12-participant, single-site, single-arm, open-label, Phase 2 trial designed to evaluate the safety and tolerability of AMX0035 and various measures of endocrinologic, neurologic, and ophthalmologic function in adult participants living with Wolfram syndrome.

In October 2024, Amylyx announced positive topline results from HELIOS. We continue to engage with FDA and other stakeholders to inform a Phase 3 program and expect to provide an update in 2025.

PSP is a sporadic, rare, fatal neurodegenerative disease that can affect movement, gait, balance, cognition, eye movements, swallowing, and speech. PSP is considered a tauopathy based on the strong genetic link between tau variants and disease development and the presence of abnormal tau protein deposits in the brain. Learn more about PSP.

ORION is a global, randomized, double-blind, placebo-controlled Phase 2b/3 clinical trial evaluating AMX0035 for the treatment of PSP.

Data from an interim analysis of ORION are expected in mid-2025.

ALS is a relentlessly progressive and fatal neurodegenerative disorder caused by motor neuron death in the brain and spinal cord. Motor neuron loss in ALS leads to deteriorating muscle function, the inability to move and speak, respiratory paralysis, and, eventually, death. Learn more about ALS.

LUMINA will be a multicenter, randomized, double-blind, placebo-controlled, multiple ascending dose Phase 1 clinical trial evaluating the safety and biological activity of AMX0114 in people with ALS.

Amylyx expects to initiate LUMINA in Canada in the beginning of 2025. Amylyx expects early cohort data from LUMINA in 2025.