Amylyx Pharmaceuticals, Inc.

Overview

• Clinical-stage pharmaceutical company focused on therapies for rare and high unmet-need conditions.
• Pipeline includes endocrine/metabolic disorders and neurodegenerative diseases, with programs supported by preclinical data, biomarkers, and defined clinical outcomes.
• Employees: 136 full-time (as of December 31, 2025).

Core product candidates and focus

• Avexitide

  • Mechanism: GLP-1 receptor antagonist.
  • Indications: Post-bariatric hypoglycemia (PBH) and congenital hyperinsulinism (Congenital HI).
  • Regulatory designations: FDA Breakthrough Therapy (PBH, HI); Rare Pediatric Disease (Congenital HI); Orphan Drug (HI).
  • Status: Lead asset; Phase 3 program in PBH (LUCIDITY) with supporting Phase 2 and Phase 1/2 data.

• AMX0035 (fixed-dose combination of sodium phenylbutyrate and taurursodiol)

  • Current focus: Wolfram syndrome.
  • Prior approvals: Previously marketed as RELYVRIO (U.S.) and ALBRIOZA (Canada) for ALS; U.S. and Canadian marketing has ceased.
  • Status: Phase 2 HELIOS data in Wolfram syndrome showing sustained benefits; regulatory and formulation considerations ongoing across jurisdictions.

• AMX0114

  • Mechanism: Antisense oligonucleotide targeting CAPN2 (calpain-2) mRNA.
  • Indication: ALS.
  • Status: Phase 1 LUMINA trial underway.

• AMX0318

  • Mechanism: GLP-1 receptor antagonist for PBH and other rare diseases.
  • Status: Candidate selected January 2026; IND-enabling studies in progress; IND targeted for 2027. Identified via collaboration with Gubra A/S.

Clinical programs and status highlights

• Avexitide (PBH)

  • LUCIDITY (post-Roux-en-Y PBH): Phase 3, ~75 participants, multicenter, randomized, double-blind, placebo-controlled across ~21 U.S. sites.
  • Key design points: 3:2 randomization to 90 mg daily versus placebo; screening/run-in/treatment/exit phases; potential 32-week open-label extension.
  • Status and timeline: Recruitment complete; final eligible patients expected randomized and dosed in Q1 2026; topline data expected Q3 2026; potential commercial launch in 2027 if approved.
  • Prior studies: Phase 1/2 and Phase 2b studies showed dose-dependent reductions in hypoglycemic events and favorable pharmacodynamic effects.

• AMX0035 (Wolfram syndrome and other indications)

  • HELIOS (Wolfram syndrome): Phase 2 open-label trial in adults. Week 24 topline data showed improvement in pancreatic beta-cell function; Week 48 data showed sustained improvements in HbA1c, time in target glucose range, and visual acuity.
  • Ongoing interactions with FDA regarding a potential Phase 3 trial.

• AMX0114 (ALS)

  • LUMINA Phase 1: Started April 2025. Cohort 1 (n=12) completed enrollment and initial safety data reported December 2025; Cohort 2 enrollment began December 2025 (n=12).

• AMX0318 (PBH / rare diseases)

  • IND-enabling work underway with an IND target in 2027.

• Corporate action

  • July 2024: Acquisition of substantially all rights and assets from Eiger related to avexitide development, manufacture, and commercialization.

Intellectual property

• Avexitide

  • U.S. issued patents: 23.
  • Foreign granted patents: 257.
  • U.S. patent applications pending: >15; foreign applications pending: >115.
  • Portfolio includes in-licensed families (UPenn/CHOP, Stanford, etc.) and company-owned families around exendin(9-39) and related formulations.

• AMX0035

  • Portfolio covers 15 patent families with 156 issued patents and pending applications globally.
  • Claims include compounds/ratios of phenylbutyrate and bile acids (e.g., TURSO, 4-PBA), methods for treating neurodegenerative diseases, co-formulations, and specific uses in Wolfram syndrome.
  • Some U.S. patent families have potential protection extending into the 2030s and 2040s, depending on jurisdiction and term adjustments.

• AMX0114

  • One patent family pending in the U.S., EU and other jurisdictions; anticipated protection through the mid-2040s for issued patents.

Manufacturing and regulatory

• Manufacturing: Reliance on third-party contract manufacturing organizations (CMOs) for avexitide, AMX0035, AMX0114 and AMX0318; FDA cGMP compliance and related quality systems in place.
• Regulatory strategy: Pursues designations such as Breakthrough Therapy, Orphan, Rare Pediatric Disease, Fast Track and Priority Review, and engages EU pathways including centralized MAA, PRIME and orphan exclusivity. The company plans for potential post-approval requirements and monitors evolving pricing and reimbursement frameworks.

Financial position and operating results

• Employees: 136 full-time (as of December 31, 2025).
• Revenue: 2023 revenue of $380.8 million from RELYVRIO/ALBRIOZA (ALS products); U.S. and Canadian marketing of those products has ceased.
• Profitability and balance sheet: Accumulated deficit of $751.4 million (as of December 31, 2025).
• Cash runway: Existing cash, cash equivalents and marketable securities are expected to fund operations into 2028.
• Commercial status: No products currently approved for commercial sale in the U.S. or globally; clinical and regulatory programs ongoing.

Summary

Amylyx is a clinical-stage company developing therapies for rare endocrine/metabolic and neurodegenerative conditions. Lead programs include avexitide for PBH and congenital HI and AMX0035 for Wolfram syndrome. Additional programs (AMX0114 and AMX0318) address ALS and PBH/other rare diseases. The company has an extensive global IP portfolio, outsources manufacturing to CMOs, and advances multiple clinical programs with planned regulatory filings and milestones through 2026–2027.