# Amylyx Pharmaceuticals > Official Amylyx Pharmaceuticals business agent. Amylyx Pharmaceuticals is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts that… ## Ask it a question Amylyx Pharmaceuticals answers for itself at https://api.hailera.com/mcp/amylyx. A client that speaks MCP can connect to that address; the tool is ask_amylyx and the protocol is 2026-07-28. Where this file and the agent disagree, the agent is current. ## About Amylyx Pharmaceuticals Who we are Amylyx Pharmaceuticals is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts. The company was founded in 2013 by Josh Cohen and Justin Klee and is listed on NASDAQ as AMLX. Amylyx brings together experience across therapeutic development through commercialization in numerous diseases, with a focus on tackling serious diseases that have high unmet needs. Mission Amylyx has an audacious mission to develop novel therapies for communities with high unmet needs. The company pursues opportunities with urgency, rigorous science, and a strong commitment to the communities it serves. What we focus on Amylyx is advancing investigational therapies across several diseases, including: - Post-bariatric hypoglycemia (PBH) - Congenital hyperinsulinism (HI) - Wolfram syndrome - Amyotrophic lateral sclerosis (ALS) - Other rare diseases where there is clear unmet medical need Our values Amylyx describes a culture of caring built around five core values: - Be audacious – Envision new possibilities, seek untried approaches, and innovate for people living with serious diseases. - Be curious – Ask questions, listen, and learn in a culture of continuous improvement and shared knowledge. - Be authentic – Be honest and transparent with communities and colleagues. - Be engaged – Deeply engage with community members and care providers to understand real needs. - Be accountable – Put people living with high unmet needs first and turn promising ideas into urgent action on their behalf. Source: https://www.amylyx.com ## Where Amylyx Pharmaceuticals works Lists Hereford and Cambridge as a location. Names Massachusetts as a service area. Areas beyond these are not published. ## Hours and contact If you would like to contact Amylyx, you can use the topic-specific email addresses and phone number below or submit an inquiry through the contact form on the company website. General contact details - General information - Email: Info@amylyx.com - Phone (U.S.): +1 (617) 682-0917 Topic-specific contacts - Investor relations - Email: Investors@amylyx.com - Media relations - Email: AmylyxMediaTeam@amylyx.com - Clinical trial information - Email: ClinicalTrials@amylyx.com - Medical information, adverse events, and product quality complaints - Email: MedInfo@amylyx.com - Advocacy information - Email: Advocacy@amylyx.com - Patient support - Primary contact: MedInfo@amylyx.com - Business development and partnering - Email: Partnering@amylyx.com Online contact form An online contact form is available on the Amylyx website for general inquiries. Visitors can provide their name, email address, and message and must consent to receiving electronic communications in accordance with the Amylyx Privacy Notice. Response times are not guaranteed; for urgent medical issues, individuals should contact a healthcare professional or local emergency services rather than relying on the website contact form. Source: https://www.amylyx.com ## Careers, Culture, and Benefits at Amylyx Working at Amylyx Amylyx describes itself as Rooted in Connection®, emphasizing strong connections between the company and the communities it serves, between questions and solutions, and between insights and actions. Team members describe Amylyx as: - Mission-driven and focused on people living with serious diseases and high unmet needs. - Collaborative and supportive, with colleagues who care about science, each other, and the communities they serve. - A place where authenticity, transparency, and professional growth are encouraged. Current job openings and detailed role descriptions are listed on the Careers page of the Amylyx website. Culture and values Amylyx highlights a culture of caring built around five core values: - Be audacious – Boldly seek new approaches and innovate for people living with serious diseases. - Be curious – Foster continuous learning, ask questions, and generously share knowledge. - Be authentic – Be honest and transparent with communities, partners, and colleagues. - Be engaged – Deeply engage with community members and healthcare providers to understand their journeys and needs. - Be accountable – Put people living with high unmet needs first and act with urgency on their behalf. Work environment Key aspects of the work environment described on the site include: - Hybrid-remote model with headquarters in Cambridge, MA. - Company-wide in-person "Anchor Weeks" several times a year and regular internal meetings (Amylyx Exchange) to stay connected. - Emphasis on learning and development, recognition, and meaningful opportunities to contribute. Overview of employee benefits (as described on the website) Amylyx notes that benefits are an important part of its total rewards approach. Highlights include: - Health and wellness - Medical, dental, and vision insurance options for employees and their families. - Tax-advantaged accounts such as Flexible Spending Accounts (FSA), Health Savings Accounts (HSA) for eligible medical expenses, Dependent Care FSA, and Limited Purpose FSA for dental and vision expenses. - Work-life balance and flexibility - Flexible paid time off that can be used for vacation, personal needs, school events, or appointments. - Hybrid work environment. - Financial and family support - 5% employer contribution to a 401(k) retirement plan. - Employee Assistance Program (EAP) for mental health, work-life solutions, financial resources, and legal guidance. - Paid parental leave. - Mobile phone reimbursement. Specific benefit details, eligibility, and availability may vary by role and location. For the latest information, candidates should review the Careers section on the Amylyx website or speak with Amylyx recruiting or HR. Source: https://www.amylyx.com ## Investigational Therapies and Diseases Amylyx Focuses On Amylyx is advancing a pipeline of tailored therapeutic approaches for serious diseases with high unmet needs. Investigational programs are matched to diseases where they may have the greatest impact, based on mechanistic rationale, defined clinical outcomes and biomarkers, and preclinical data. _All programs and compounds described here are based on information published by Amylyx and may be investigational or subject to regulatory review. This document does not provide medical advice or treatment recommendations._ Post-bariatric hypoglycemia (PBH) and congenital hyperinsulinism (HI) Avexitide - Avexitide is a glucagon-like peptide-1 (GLP-1) receptor antagonist that Amylyx has added to its pipeline as a Phase 3–ready investigational therapy. - It has FDA Breakthrough Therapy designation. - Amylyx is evaluating avexitide in: - Post-bariatric hypoglycemia (PBH), including in people with PBH following Roux-en-Y gastric bypass (RYGB). - Congenital hyperinsulinism (HI). - The Phase 3 LUCIDITY clinical trial of avexitide in PBH after RYGB completed enrollment, and Amylyx has announced positive topline data from LUCIDITY for the treatment of PBH (as reflected in the company's news and homepage). Wolfram syndrome AMX0035 in Wolfram syndrome - AMX0035 is a proprietary oral combination of sodium phenylbutyrate (PB) and taurursodiol (TURSO, also known as ursodoxicoltaurine outside the U.S.). - AMX0035 received Orphan Drug Designation from the FDA and from the European Commission for the treatment of Wolfram syndrome. - Amylyx is studying AMX0035 in Wolfram syndrome in the Phase 2 HELIOS clinical trial. - The company has reported positive interim and positive topline data from HELIOS for people living with Wolfram syndrome. Amyotrophic lateral sclerosis (ALS) About ALS (high-level overview) - Amyotrophic lateral sclerosis (ALS) is a progressive and fatal neurodegenerative disorder caused by motor neuron death in the brain and spinal cord. - Motor neuron loss leads over time to deteriorating muscle function, loss of movement and speech, respiratory failure, and ultimately death. - ALS is considered a rare disease, affecting as many as approximately 30,000 adults in the U.S. and about 3,000 in Canada, with most cases being sporadic. Role of calpain-2 and AMX0114 - Scientific data highlight calpain-2 (CAPN2), a protein involved in neurofilament biology, as an important contributor to axonal degeneration in neurodegenerative diseases such as ALS. - Elevated CAPN2 levels and its role in cleaving neurofilament light chain (NfL) and TDP-43 support CAPN2 as a potential therapeutic target in ALS. - AMX0114 is an Amylyx-developed investigational antisense oligonucleotide (ASO) designed to target CAPN2 for the potential treatment of ALS. - In preclinical studies, AMX0114: - Produced potent, dose-dependent, and durable reductions in CAPN2 mRNA and calpain-2 protein levels. - Improved neuronal survival in disease-relevant models, including a model of TDP-43 ALS. - Reduced extracellular NfL levels across multiple models of neuronal injury. - AMX0114 was reported as well-tolerated in in vivo preclinical safety studies. LUMINA clinical trial of AMX0114 in ALS - LUMINA is a Phase 1, multinational, randomized, double-blind, placebo-controlled, multiple ascending dose clinical trial of AMX0114 in ALS. - The trial is designed to evaluate safety, tolerability, pharmacokinetics, and pharmacodynamics. - Amylyx has reported that: - The first participant was dosed in April 2025. - Cohort 1 (n=12) and Cohort 2 (n=12) have been fully enrolled. - Cohort 1 safety data showed AMX0114 was generally well-tolerated with no treatment-related serious adverse events. - Presentation of biomarker data from Cohort 1 is expected in the first half of 2026 (per the company's ALS program information). AMX0035 in ALS and other neurodegenerative conditions - AMX0035 was originally developed by Amylyx with the hypothesis that it could target two pathways relevant to slowing progression in neurodegenerative diseases. - Key ALS-related milestones include: - Phase 2 CENTAUR clinical trial in ALS met its primary endpoint. - AMX0035 received Orphan Drug Designation from the FDA and EMA for ALS. - AMX0035 received regulatory approval for ALS under the brand names RELYVRIO (U.S.) and ALBRIOZA (Canada). - A global Phase 3 PHOENIX clinical trial in ALS was conducted to evaluate AMX0035 in a larger and broader population. - PHOENIX did not meet its prespecified primary endpoint. Following these results, Amylyx began a process with the FDA and Health Canada to voluntarily discontinue the marketing authorizations for RELYVRIO and ALBRIOZA. - According to the company's timeline, Amylyx committed to providing free access through early 2025 to people already on therapy who, together with their doctor, decide to continue. - AMX0035 has also been studied in Alzheimer's disease, with findings from the PEGASUS trial suggesting engagement of multiple pathological pathways related to neurodegeneration. AMX0318 and other rare diseases AMX0318 - AMX0318 is a long-acting GLP-1 receptor antagonist that Amylyx selected as a development candidate for PBH and other rare diseases through a research collaboration with Gubra A/S. - IND-enabling studies for AMX0318 are underway, according to the company's pipeline updates. Summary Amylyx is focused on building an ambitious pipeline of investigational therapies for: - Post-bariatric hypoglycemia (PBH) - Congenital hyperinsulinism (HI) - Wolfram syndrome - Amyotrophic lateral sclerosis (ALS) - Additional rare diseases where GLP-1 receptor antagonism or related mechanisms may be relevant For detailed and up-to-date information on specific clinical trials, eligibility, or access programs, individuals should refer to official Amylyx communications, clinical trial registries, or contact the company or their healthcare provider directly. Source: https://www.amylyx.com ## What Amylyx Pharmaceuticals has not published yet These are things people ask Amylyx Pharmaceuticals that its published information does not yet cover. - prices - product availability - treatment efficacy or clinical outcomes - safety outcomes or side effect rates - regulatory approvals or labelling status - clinical trial eligibility or enrollment - insurance or reimbursement coverage - guarantees, cures, or timeframes for access Ask anyway — the agent will say plainly that it is not published rather than guess. ## Where this comes from https://www.amylyx.com