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• RELYVRIO / ALBRIOZA: A prescription medication developed for the treatment of amyotrophic lateral sclerosis (ALS).

Amylyx Pharmaceuticals (AMLX) primarily sold its product, RELYVRIO (also known as ALBRIOZA in Canada), to other companies, specifically **pharmaceutical wholesalers**.

According to Amylyx Pharmaceuticals' SEC filings (e.g., their Form 10-K for the fiscal year ended December 31, 2023), the company indicated that a significant majority of its gross product revenue was concentrated with two unnamed major pharmaceutical wholesalers. For the year ended December 31, 2023, one customer accounted for 50% of gross product revenue, and another accounted for 35%.

While Amylyx does not publicly name these specific customers in their filings, the major players in the U.S. pharmaceutical wholesale industry, who typically serve as the primary customers for drug manufacturers, include:

• McKesson Corporation (NYSE: MCK)
• Cencora (NYSE: COR) (formerly AmerisourceBergen Corporation)
• Cardinal Health, Inc. (NYSE: CAH)

These companies are responsible for distributing pharmaceutical products from manufacturers to pharmacies, hospitals, and other healthcare providers across the United States, making them the critical link in the supply chain for drug developers like Amylyx.

Important Note: On March 6, 2024, Amylyx Pharmaceuticals announced its decision to voluntarily remove RELYVRIO/ALBRIOZA from the market based on the results of the Phase 3 PHOENIX trial. As a result, the company will cease commercial sales of the product, significantly impacting its future customer relationships and revenue from these wholesalers.

• Catalent, Inc. (CTLT)
• CordenPharma

Joshua Cohen, Co-Chief Executive Officer & Co-Founder

Joshua Cohen co-founded Amylyx Pharmaceuticals in 2013 and serves as its Co-Chief Executive Officer. He co-invented AMX0035, a proprietary combination of sodium phenylbutyrate and taurursodiol, for neurodegenerative diseases. Mr. Cohen oversaw the company's growth from a dorm room concept at Brown University to a global, publicly traded pharmaceutical company. He led global regulatory approvals of AMX0035 for ALS in Canada (ALBRIOZA™) and the U.S. (RELYVRIO™). With a background in biomedical engineering from Brown University, he founded the Brown Biotechnology Investment Group and published research at the National Institutes of Standards and Technology and in the Journal of Pharmaceutical Sciences. He was named to Business Insider's "30 Under 40 in Healthcare" and PM360's "ELITE in the Drug Researchers and Developers" lists in 2020, and PM360's 2022 ELITE Entrepreneurs list.

Justin Klee, Co-Chief Executive Officer & Co-Founder

Justin Klee co-founded Amylyx Pharmaceuticals in 2013 and serves as its Co-Chief Executive Officer. Alongside Joshua Cohen, he co-invented AMX0035 and oversaw the company's development into a global, publicly traded pharmaceutical company from its origins as a concept at Brown University. Mr. Klee played a key role in the global regulatory approvals of AMX0035 for ALS in Canada and the U.S. He holds a background in neuroscience from Brown University and previously conducted research in neural systems at Brown and in neurophysiology and Alzheimer's disease at Harvard Medical School. He was recognized in Business Insider's "30 Under 40 in Healthcare" and PM360's "ELITE in the Drug Researchers and Developers" lists in 2020, and PM360's 2022 ELITE Entrepreneurs list.

James M. Frates, Chief Financial Officer

James M. Frates joined Amylyx Pharmaceuticals as Chief Financial Officer in January 2021. Prior to Amylyx, he served as the Chief Financial Officer at Alkermes from July 1998 to January 2021, where he was instrumental in growing the company from a clinical-stage entity to an international enterprise generating over $1 billion in revenue and employing more than 2,000 people. Mr. Frates managed Alkermes through numerous acquisitions and financings in debt, equity, and private markets. Before his tenure at Alkermes, he worked as an investment banker at Robertson Stephens and Morgan Stanley, specializing in biotechnology. He is a graduate of Harvard College and earned an MBA from Harvard Business School. He also serves on the Board of Sage Therapeutics, Inc.

Camille L. Bedrosian, MD, Chief Medical Officer

Dr. Camille L. Bedrosian serves as the Chief Medical Officer of Amylyx Pharmaceuticals. She brings nearly 30 years of experience in medical affairs, clinical development, and research, particularly focusing on rare and serious diseases. Her career includes significant roles at other pharmaceutical companies such as Ultragenyx, Alexion, and ARIAD, where she led medical affairs, regulatory, and clinical development initiatives.

Gina M. Mazzariello, Chief Legal Officer & General Counsel

Gina M. Mazzariello holds the position of Chief Legal Officer and General Counsel at Amylyx Pharmaceuticals.

The clear emerging threat for Amylyx Pharmaceuticals (AMLX) was the potential failure of the confirmatory Phase 3 PHOENIX trial for their main product, Relyvrio (Albrioza), for the treatment of Amyotrophic Lateral Sclerosis (ALS). This threat materialized when the company announced on March 8, 2024, that the PHOENIX trial did not meet its primary endpoint or key secondary endpoints. As a direct consequence of this clinical trial failure, Amylyx subsequently announced its decision to remove Relyvrio from the market in the United States and Canada, which constitutes a fundamental threat to its primary revenue stream and business model.

Amylyx Pharmaceuticals (AMLX) is currently focusing on several investigational therapies after withdrawing its Amyotrophic Lateral Sclerosis (ALS) drug, Relyvrio/Albrioza, from the market in April 2024.

The company's pipeline includes:

• Avexitide: This investigational therapy is for post-bariatric hypoglycemia (PBH) and congenital hyperinsulinism (HI). Approximately 160,000 of the more than two million people who have undergone bariatric surgery in the last decade are currently living with PBH in the United States.
• AMX0035 (repurposed): This therapy is being investigated for Wolfram syndrome and progressive supranuclear palsy (PSP).
• AMX0114: This is an investigational therapy for amyotrophic lateral sclerosis (ALS).

The global market for amyotrophic lateral sclerosis is expected to reach $1.5 billion by 2033. In the United States, approximately 30,000 people are affected by ALS, while Europe (including the European Union and the United Kingdom) has an estimated ALS population of over 30,000.

• Launch and Commercialization of Avexitide for Post-Bariatric Hypoglycemia (PBH): Amylyx expects to complete recruitment for the pivotal Phase 3 LUCIDITY trial of avexitide, a GLP-1 receptor antagonist for PBH, in the first quarter of 2026, with topline data anticipated in the third quarter of 2026. The company aims for a potential commercial launch of avexitide in 2027, if approved, and is actively preparing for this launch. Avexitide has received FDA Breakthrough Therapy and Orphan Drug Designations, highlighting the significant unmet need in the PBH market and its potential to be a first-in-class treatment.
• Expansion of AMX0035 into New Indications (Wolfram Syndrome and Progressive Supranuclear Palsy): Despite challenges with its ALS indication, Amylyx is continuing to develop AMX0035 (also known as Relyvrio/Albrioza) for other neurodegenerative diseases. Positive topline data from the Phase 2 HELIOS trial of AMX0035 in Wolfram syndrome showed improvement or stabilization across disease measures, and Amylyx plans to meet with the FDA to inform a Phase 3 program, with a pivotal trial potentially initiating in the second half of 2026. Additionally, the company anticipates interim analysis data from a Phase 2b/3 trial of AMX0035 in progressive supranuclear palsy (PSP) in the third quarter of 2025. These new indications represent potential new markets and revenue streams for AMX0035.
• Advancement of AMX0114 for Amyotrophic Lateral Sclerosis (ALS): Amylyx is progressing its AMX0114 program, an antisense oligonucleotide targeting calpain-2 for ALS. The company initiated the Phase 1 LUMINA clinical trial for AMX0114 in late 2024 or early 2025, with early cohort data expected in 2025. Successful progression through clinical trials could establish AMX0114 as a future revenue driver in the ALS market.
• Strategic Focus on Orphan Markets and Pipeline Development: Amylyx has emphasized its commitment to advancing its late-stage pipeline in orphan markets with high unmet needs. The company's overall strategy involves leveraging its expertise in neurodegenerative and rare endocrine conditions to develop and commercialize treatments for patient populations with limited therapeutic options. This pipeline-driven approach, including both wholly-owned assets and acquired programs like avexitide, forms a crucial foundation for long-term revenue generation beyond existing products. Analysts forecast significant revenue growth for Amylyx, projecting an annual increase of 73.4%.

Share Repurchases

• Amylyx Pharmaceuticals had a quarterly stock buyback of -$0.072 million for June 30, 2025.

Share Issuance

• In September 2025, Amylyx Pharmaceuticals priced an underwritten public offering of 17,500,000 shares of its common stock at $10.00 per share, expecting gross proceeds of $175.0 million. The net proceeds from this offering were approximately $191 million, extending the company's expected cash runway into 2028.
• In January 2025, the company announced the pricing of an underwritten public offering of 17,142,857 shares of its common stock at $3.50 per share, with expected gross proceeds of approximately $60.0 million.
• The number of shares outstanding for Amylyx Pharmaceuticals increased by 15.02% in the last year.

Capital Expenditures

• Capital expenditures for the last 12 months were -$36.23 million.
• The company's cash runway is expected to extend into 2028, with capital allocated for Avexitide commercial readiness, research and development, and general corporate purposes.