Amylyx Pharmaceuticals (AMLX) Q2 FY26 earnings call: guidance and Q&A

When was the last patient visit for LUCIDITY, what factors are key for database lock, and how is the EAP progressing, including patient recruitment and demand?

The last patient visit for LUCIDITY was mid-to-late July. The team is diligently working on data cleaning and ensuring all data matches for database lock. The EAP is in its early days, open to LUCIDITY completers and prior avexitide trial participants, with great enthusiasm for participation.

“The initial phase, if you will, of the EAP as we abbreviate it, is for those individuals in LUCIDITY who have completed the study, double-blind period, of course, and then the open-label extension. And then they had the opportunity to roll over into the EAP.”

asked by Seamus Fernandez · answered by Camille Bedrosian

Does the Phase 3 patient population resemble Phase 2, and what mechanisms are in place to monitor injection compliance?

LUCIDITY was designed to replicate Phase 2, enrolling participants consistent with Phase 2/2b populations. A rigorous eligibility assessment was conducted, and the Amylyx team, in partnership with clinical sites, maintained close oversight and monitoring to ensure compliance with study protocol and drug administration.

“We designed LUCIDITY with the Phase 2 studies in mind, the highly successful statistically significant outcomes for those Phase 2 studies. And we're enrolling participants who are very consistent with the populations that were enrolled in the Phase 2 and Phase 2b.”

asked by Joseph Thome · answered by Camille Bedrosian

What evidence is needed for a broad PBH label (including sleeve patients), and how does the 160,000 patient prevalence estimate translate into a realistic 5-year treated population?

The company will advocate for a broad PBH label due to common pathophysiology. If restricted to Roux-en-Y, an efficient study would be needed for expansion. Approximately 120,000 of the 160,000 PBH patients had Roux-en-Y. Commercial launch will initially focus on centers with 50-100 known patients, then expand through education.

“of the current 160,000 people with PBH in the United States, about 120,000 had Roux-en-Y gastric bypass leading to their PBH.”

asked by Mike · answered by Justin Klee

What is the concentration of PBH patients at specific sites, and what should be the expected placebo response in the LUCIDITY study?

Some centers have 50-100 PBH patients, validated by medical affairs. For LUCIDITY, the company powered conservatively (90% power to detect a 35% treatment effect with up to a 50% placebo effect), based on prior Phase 2 data showing a 55% reduction against placebo, acknowledging this is the first Phase 3 pivotal study in PBH.

“we powered the study to be 90% powered to detect a 35% treatment effect with even an up to 50% placebo effect.”

asked by Marc Goodman · answered by Justin Klee

What are KOLs saying about avexitide's use in gastric/upper GI surgeries, what is the development plan for AMX0318, and how does the new ICD-10 code affect payer conversations?

KOLs see potential for avexitide in other surgery-induced hypoglycemias (e.g., gastrectomy) due to similar mechanisms. AMX0318 is in IND-enabling studies, with a development plan to be outlined. The new ICD-10 code recognizes PBH and is helpful for tracking/diagnosis, but is not necessary for reimbursement, as patients are already identifiable via claims analysis.

“ICD-10 codes, they are certainly helpful for tracking and diagnosing patients. And oftentimes the coding, the ICD-10 codes are used for epidemiology. From a payer perspective, an ICD-10 code is not necessary for reimbursement.”

asked by Ananda Ghosh · answered by Dan Monahan