What Amylyx Pharmaceuticals does

Amylyx Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company developing therapies for rare endocrine and neurodegenerative diseases with high unmet medical need. The company was founded in 2013 by Josh Cohen and Justin Klee and completed its initial public offering on NASDAQ under the ticker AMLX in January 2022. Its pipeline is modality-agnostic and currently comprises four investigational programs: avexitide, a first-in-class GLP-1 receptor antagonist being developed for post-bariatric hypoglycemia (PBH) and congenital hyperinsulinism; AMX0035, an oral fixed-dose combination of sodium phenylbutyrate and taurursodiol targeting endoplasmic reticulum stress and mitochondrial dysfunction, being developed for Wolfram syndrome; AMX0114, an antisense oligonucleotide targeting calpain-2 for amyotrophic lateral sclerosis (ALS); and AMX0318, a long-acting GLP-1 receptor antagonist being developed in collaboration with Gubra A/S.

The company's core value driver is avexitide, which was acquired from Eiger BioPharmaceuticals in July 2024 and is being evaluated in the pivotal Phase 3 LUCIDITY trial in PBH, with enrollment of 78 participants completed in March 2026 and topline data expected in Q3 2026. If positive, Amylyx plans an immediate NDA submission and a potential 2027 U.S. commercial launch into a market of approximately 160,000 U.S. PBH patients with no FDA-approved therapies. AMX0035, AMX0114, and AMX0318 provide optionality across Wolfram syndrome, ALS, and next-generation GLP-1 antagonism respectively, supported by Orphan Drug, Breakthrough Therapy, and Fast Track regulatory designations.

The business model is currently a pre-commercial clinical-development model. The company previously generated product revenue from RELYVRIO/ALBRIOZA (AMX0035) for ALS in the U.S. and Canada following FDA approval in September 2022, but voluntarily withdrew the product in April 2024 after the Phase 3 PHOENIX trial failed to meet its primary endpoint, triggering a roughly 70% workforce reduction. Commercialization strategy for avexitide includes a U.S. specialty pharmacy and rare-disease field force model, with a Chief Commercial Officer (Dan Monahan) hired in preparation for a 2027 launch. As of May 2026, Teva Pharmaceutical Industries announced an agreement to acquire Amylyx for $700 million upfront plus up to $200 million in milestone payments, with closing expected in late Q2 or early Q3 2026, which would end Amylyx's status as an independent public company.

Name

Amylyx Pharmaceuticals

Legal name

Amylyx Pharmaceuticals, Inc.

Website

https://amylyx.com

Company type

Public

Founded year

2013

Operating status

Operating

Headcount range

101–250 employees

Short description

Amylyx Pharmaceuticals is a clinical-stage biopharmaceutical company developing investigational therapies for rare endocrine and neurodegenerative diseases, with lead candidate avexitide in Phase 3 development for post-bariatric hypoglycemia and additional programs in Wolfram syndrome and ALS.

Ownership category

akta.pro rank

Product category

Clinical-Stage Rare Disease Biopharmaceuticals

NAICS

Pharmaceutical Preparation Manufacturing (325412), Biological Product (except Diagnostic) Manufacturing (325414), Research and Development in Biotechnology (except Nanobiotechnology) (541714)

SIC

Pharmaceutical Preparations (2834)

akta.pro primary industry

Neurology & CNS Specialty Pharmaceuticals (HLAIACAC)

akta.pro secondary industry

Neurology/CNS Gene Therapies (HLAAACAH)

Keywords

• Rare disease therapeutics
• Clinical stage biopharmaceutical
• Neurology drug development
• Endocrine disorder therapies
• Antisense oligonucleotide therapy

Headquarters

HQ city

Cambridge

HQ country

United States

HQ region

North America

Offices6 records

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Revenue model

1. Drug commercialization (planned — avexitide): Amylyx is preparing for potential commercial launch of avexitide in 2027 for post-bariatric hypoglycemia. No FDA-approved therapies currently exist for PBH, representing a potential first-in-class opportunity. Avexitide would be commercialized as a prescription pharmaceutical product administered via subcutaneous injection. The company has a Chief Commercial Officer (Dan Monahan) and is conducting NDA-readiness preparations.
2. Former commercial product (discontinued — RELYVRIO): Amylyx previously generated product revenue from RELYVRIO (AMX0035) in the U.S. and ALBRIOZA in Canada for ALS treatment. Revenue was generated through product sales to patients and payers. Following the Phase 3 PHOENIX trial failure in March-April 2024, the company voluntarily discontinued marketing authorizations for both products in the U.S. and Canada and provided free drug access through early 2025 to patients who chose to continue therapy. This revenue stream is no longer active.
3. Potential acquisition proceeds: In May 2026, Teva Pharmaceutical Industries announced agreement to acquire Amylyx Pharmaceuticals for $700 million upfront plus up to $200 million in milestone payments, expected to close in late Q2 or early Q3 2026. If completed, this would represent a significant liquidity event, though it would end Amylyx as an independent public company.

Pricing tiers

Go-to-market motion3 records

Distribution channels5 records

Marketing channels6 records

Core offering

Amylyx Pharmaceuticals is a clinical-stage biopharmaceutical company developing investigational therapies for rare endocrine and neurodegenerative diseases. Its pipeline includes avexitide (a first-in-class GLP-1 receptor antagonist for post-bariatric hypoglycemia), AMX0035 (oral fixed-dose combination of sodium phenylbutyrate and taurursodiol for Wolfram syndrome), AMX0114 (antisense oligonucleotide targeting calpain-2 for ALS), and AMX0318 (long-acting GLP-1 receptor antagonist). The company is preparing for a potential 2027 commercial launch of avexitide in the U.S.

Product overview

Amylyx Pharmaceuticals is a clinical-stage biopharmaceutical company developing four investigational therapies across endocrine conditions and neurodegenerative diseases: Avexitide (GLP-1 receptor antagonist for post-bariatric hypoglycemia and congenital hyperinsulinism), AMX0035 (oral combination for Wolfram syndrome), AMX0114 (antisense oligonucleotide for ALS), and AMX0318 (long-acting GLP-1 receptor antagonist for PBH). These are drugs in clinical development, not commercial software products.

Differentiator

Problem solved

Functional benefit

Brands

• ALBRIOZA: Brand name for AMX0035 approved in Canada (with conditions) for ALS treatment
• RELYVRIO

Products and services

• Avexitide Avexitide is a first-in-class glucagon-like peptide-1 (GLP-1) receptor antagonist injectable peptide designed to bind to the GLP-1 receptor on pancreatic islet beta cells and inhibit the effect of excessive GLP-1 to mitigate hypoglycemia. It is being developed for post-bariatric hypoglycemia (PBH) following bariatric surgery and congenital hyperinsulinism (HI). It holds FDA Breakthrough Therapy Designation, Orphan Drug Designation, and Rare Pediatric Disease Designation.
• AMX0035 AMX0035 is an oral fixed-dose combination of sodium phenylbutyrate (PB) and taurursodiol (TURSO), two small molecules that target endoplasmic reticulum (ER) stress and mitochondrial dysfunction — the two cellular processes central to neuronal cell death and neurodegeneration. It is being developed for Wolfram syndrome and received FDA and European Commission Orphan Drug Designations.
• AMX0114 AMX0114 is an antisense oligonucleotide (ASO) administered intrathecally that targets calpain-2 (CAPN2), a calcium-dependent protease and critical effector of axonal (Wallerian) degeneration in ALS and other neurodegenerative diseases. AMX0114 cleaves neurofilament light chain (NfL) and TDP-43, proteins associated with ALS pathology. It received FDA Fast Track Designation for ALS in May 2025.
• AMX0318 AMX0318 is a long-acting glucagon-like peptide-1 (GLP-1) receptor antagonist identified through Amylyx's research collaboration with Gubra A/S. It has completed extensive preclinical evaluation including stability, solubility, potency, in vivo pharmacokinetics and pharmacodynamics, and tolerability studies. It is being developed for post-bariatric hypoglycemia and other rare diseases.

Quantifiable outcome

• Avexitide demonstrated statistically significant reductions in hypoglycemic events in 5 prior Phase 1 and Phase 2 clinical trials in PBH, with over 50% reduction in daily rates of hypoglycemia in Phase 2 trials.
• +3 more outcomes

Named customers1 record

Segments4 records

Ideal customer profiles3 records

Technology focussed No

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature4 records

Partnerships

Two partnerships are on record, tiered core.

• Gubra A/ScoreStrategic or Co-development Partner · 1 December 2024Amylyx entered a research collaboration with Danish biotech company Gubra A/S (December 2024) to develop a novel long-acting GLP-1 receptor antagonist. Through this collaboration, AMX0318 was selected as a development candidate for post-bariatric hypoglycemia and other rare diseases. Gubra is an industry leader in peptide-based drug discovery. Amylyx has exclusive rights to development candidates identified through the collaboration. Gubra is eligible to receive over $50 million in success-based milestones plus mid-single digit royalties, including a $4 million payment for AMX0318 candidate selection. IND-enabling studies for AMX0318 are underway with an IND targeted for 2027.
• Eiger BioPharmaceuticalscoreOEM/ Whitelabel/ Licensing Partner · 1 July 2024Amylyx acquired avexitide (a Phase 3-ready GLP-1 receptor antagonist) from Eiger BioPharmaceuticals in July 2024 for an undisclosed upfront amount. Avexitide had been developed by Eiger for post-bariatric hypoglycemia and congenital hyperinsulinism, receiving FDA Breakthrough Therapy Designation and Orphan Drug Designation. The acquisition added a near-commercial asset to Amylyx's pipeline, representing the company's strategic pivot from ALS following the RELYVRIO withdrawal.

Scale indicators14 records

Recent moves7 records

Expansion highlights6 records

Direct peers

• Sage Therapeutics: CNS specialty biopharma with a similar profile of a single approved CNS drug, post-launch setbacks, and pipeline pivots — making it a close comparable in CNS rare-disease commercial and development cycles.
• Eiger BioPharmaceuticals: Original developer and licensor of avexitide (the GLP-1 receptor antagonist that became Amylyx's lead asset after Amylyx acquired it in July 2024). Directly comparable in target indications (PBH, congenital hyperinsulinism) and mechanistic approach.
• Ionis Pharmaceuticals: Antisense oligonucleotide (ASO) platform company most directly comparable to Amylyx's AMX0114 (calpain-2 ASO) program for ALS; both develop intrathecally administered CNS-directed ASOs for rare neurological indications.

Broad incumbents

• BridgeBio Pharma: Rare-disease focused biopharma with multiple programs across different modalities and small patient populations — a structural comparable for Amylyx's multi-asset rare-disease thesis.
• Alnylam Pharmaceuticals: Nucleotide-based (RNAi) rare-disease drug developer with regulatory designations and rare-disease commercial infrastructure; comparable for Amylyx's oligonucleotide direction and rare metabolic disease focus.
• BioMarin Pharmaceutical: Established rare-disease specialty pharmaceutical company with a multi-asset rare-disease portfolio and global commercial infrastructure — a relevant incumbent comparable for Amylyx's planned rare-endocrine and neurodegenerative launches.
• Ultragenyx Pharmaceutical: Rare-disease biotech with multiple modalities (biologics, ASO, gene therapy) targeting metabolic and neurological rare diseases — closely comparable platform breadth and pipeline to Amylyx's modality-agnostic strategy.

Emerging players

• Regenxbio: Gene therapy platform company with CNS-directed rare disease programs and selective modality use — comparable in CNS rare-disease therapeutic ambition and pipeline approach.
• Lexeo Therapeutics: Cardiac and CNS gene therapy clinical-stage biotech with CNS-directed rare-disease programs — comparable as a small rare-disease biotech advancing oligonucleotide/gene-therapy modalities into rare indications.
• Scholar Rock: Clinical-stage rare-disease biotech with a CNS/spinal muscular atrophy focus and platform technology — comparable in size, stage, and late-stage clinical development priorities.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat5 records

Key risks6 records

Key highlights7 records

Customer concentration

• Xx.com/amylyxpharma
• Facebookfacebook.com/pages/category/Local-Business/Amylyx-Pharmaceuticals-Inc-286929921438696
• LinkedInlinkedin.com/company/amylyx

Compliance4 records

Revenue estimate

Valuation estimate

Number of profiles

Profiles15 records

Subsidiaries4 records

Funding overview

Funding rounds10 records

Investors18 records

Funding detail is available on the Subscription and Enterprise plan.Contact sales →

M&A

Investments

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about Amylyx Pharmaceuticals