This section highlights FDA-related milestones and regulatory updates for drugs developed by Amylyx Pharmaceuticals (AMLX).
Over the past two years, Amylyx Pharmaceuticals has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
AMX0035, AMX0318, and AMX0114. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
Select a button below to view the list of FDA events for that drug.
AMX0035 FDA Regulatory Timeline and Events
AMX0035 is a drug developed by Amylyx Pharmaceuticals for the following indication: To treat amyotrophic lateral sclerosis (ALS).
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- AMX0035
- Announced Date:
- May 15, 2026
- Indication:
- To treat amyotrophic lateral sclerosis (ALS)
Announcement
Amylyx Pharmaceuticals, Inc announced that Week 24 and Week 48 results from the Phase 2 open-label HELIOS clinical trial of AMX0035, an oral, fixed-dose combination of sodium phenylbutyrate and taurursodiol, in adults living with Wolfram syndrome have been published in The Journal of Clinical Investigation, a peer-reviewed medical journal.
AI Summary
Amylyx Pharmaceuticals said Week 24 and Week 48 results from its Phase 2 HELIOS clinical trial of AMX0035 have been published in The Journal of Clinical Investigation. AMX0035 is an oral medicine made from sodium phenylbutyrate and taurursodiol, and the study is testing it in adults living with Wolfram syndrome, a rare genetic disease.
HELIOS is a single-site, open-label trial with 12 adults and can follow patients for up to 208 weeks, plus a short safety follow-up period. The published results add new data on how the treatment performed over time. Amylyx said it plans to share more long-term HELIOS results at an upcoming scientific meeting.
Read Announcement- Drug:
- AMX0035
- Announced Date:
- August 27, 2025
- Indication:
- To treat amyotrophic lateral sclerosis (ALS)
Announcement
Amylyx Pharmaceuticals, Inc. announced the decision to discontinue the ORION program of AMX0035 (sodium phenylbutyrate [PB] and taurursodiol [TURSO, also known as ursodoxicoltaurine]) in adults living with progressive supranuclear palsy (PSP).
AI Summary
Amylyx Pharmaceuticals announced it will discontinue the ORION program testing AMX0035—a fixed-dose combination of sodium phenylbutyrate and taurursodiol—in adults with progressive supranuclear palsy (PSP). This decision follows Phase 2b results showing no difference between AMX0035 and placebo on primary or secondary outcomes at Week 24.
Although AMX0035 continued to be generally well-tolerated and safety data aligned with earlier studies, the lack of efficacy led Amylyx to end both the Phase 2b trial and its open-label extension. The company will not move forward with a planned Phase 3 study in PSP.
Amylyx expressed gratitude to participants, families and study sites for their support. The company is now focusing on its pivotal Phase 3 LUCIDITY trial of avexitide, ongoing AMX0035 development in Wolfram syndrome, and early ALS studies of AMX0114, with a cash runway expected through 2026.
Read Announcement- Drug:
- AMX0035
- Announced Date:
- August 12, 2024
- Indication:
- To treat amyotrophic lateral sclerosis (ALS)
Announcement
Amylyx Pharmaceuticals, Inc announced the publication of exploratory analyses on cerebrospinal fluid (CSF) biomarkers from participants with Alzheimer's disease (AD) from the Phase 2 PEGASUS trial.
AI Summary
Amylyx Pharmaceuticals announced the publication of exploratory analyses on cerebrospinal fluid (CSF) biomarkers from participants with Alzheimer’s disease in the Phase 2 PEGASUS trial. The study explored how AMX0035, a combination of sodium phenylbutyrate and taurursodiol, may affect key biomarkers associated with neurodegeneration. Researchers observed that treatment with AMX0035 resulted in consistent changes in the CSF levels of several biomarkers, including those related to tau pathology, synaptic and neuronal degeneration, and gliosis. Specifically, reductions were seen in phosphorylated tau181 and total tau, as well as in biomarkers of neuronal damage such as neurogranin and FABP3. These results support the idea that AMX0035 could engage multiple pathological pathways involved in Alzheimer’s disease, offering further insight into its potential to slow or mitigate neurodegeneration in patients with varying severity of the disease.
Read Announcement- Drug:
- AMX0035
- Announced Date:
- August 2, 2024
- Indication:
- To treat amyotrophic lateral sclerosis (ALS)
Announcement
Amylyx Pharmaceuticals, Inc. announced the European Commission (EC), based on a positive opinion issued by the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA), has granted Orphan Drug Designation for AMX0035, Amylyx' proprietary, fixed-dose combination of sodium phenylbutyrate (PB) and taurursodiol (TURSO; also known as ursodoxicoltaurine outside of the U.S.) for the treatment of Wolfram syndrome.
AI Summary
Amylyx Pharmaceuticals announced that the European Commission has granted Orphan Drug Designation for its drug AMX0035, following a positive opinion from the Committee for Orphan Medicinal Products of the European Medicines Agency. AMX0035 is a proprietary, fixed-dose combination of sodium phenylbutyrate and taurursodiol and is being developed for treating Wolfram syndrome—a rare, progressive, and life-threatening disorder. Wolfram syndrome causes childhood-onset diabetes, vision loss, hearing decline, and neurological problems, and there are currently no approved treatments for the disease.
This designation recognizes the potential of AMX0035 to provide a significant benefit over existing approaches. In addition to the designation, Amylyx is conducting a Phase 2 HELIOS trial to further evaluate the drug’s impact on endocrine, metabolic, and neurodegenerative aspects of Wolfram syndrome, with topline data expected this fall.
Read Announcement- Drug:
- AMX0035
- Announced Date:
- August 2, 2024
- Indication:
- To treat amyotrophic lateral sclerosis (ALS)
Announcement
Amylyx Pharmaceuticals, Inc announced that Topline data for all 12 participants from Phase 2 HELIOS trial studying impact of AMX0035 on endocrine, metabolic, and neurodegenerative aspects of Wolfram syndrome anticipated fall 2024
AI Summary
Amylyx Pharmaceuticals announced that topline data is expected this fall 2024 from its Phase 2 HELIOS trial, which is studying the impact of AMX0035 on Wolfram syndrome. The trial involves 12 participants and focuses on the drug’s effects on endocrine, metabolic, and neurodegenerative aspects of the disease. Interim results from eight participants showed that AMX0035 may help stabilize or even improve pancreatic function, blood sugar control, and vision—areas that typically worsen over time in Wolfram syndrome. The full data release will include Week 24 assessments for all trial participants, providing important insights into the potential benefits of AMX0035. This study is a significant step toward addressing the urgent unmet needs of those affected by this rare and progressive condition, which is marked by early-onset diabetes, vision loss, and neurological decline.
Read Announcement
AMX0318 FDA Regulatory Events
AMX0318 is a drug developed by Amylyx Pharmaceuticals for the following indication: for post-bariatric hypoglycemia.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- AMX0318
- Announced Date:
- January 8, 2026
- Indication:
- for post-bariatric hypoglycemia
Announcement
Amylyx Pharmaceuticals, Inc. announced the selection of AMX0318, a long-acting glucagon-like peptide-1 (GLP-1) receptor antagonist, as a development candidate for post-bariatric hypoglycemia (PBH) and other rare diseases.
AI Summary
Amylyx Pharmaceuticals announced that AMX0318, a long-acting glucagon-like peptide-1 (GLP-1) receptor antagonist, has been chosen as a development candidate for post-bariatric hypoglycemia (PBH) and other rare diseases. The peptide was discovered through a research collaboration with Gubra, which used an AI-driven peptide discovery platform to optimize candidates.
AMX0318 met key criteria in preclinical testing, showing robust chemical stability, high solubility, strong in vitro potency, evidence of in vivo efficacy and tolerability, and a pharmacokinetic profile consistent with long-acting administration. These attributes supported its nomination as a development candidate.
Amylyx plans to start IND-enabling studies in 2026 and is targeting an IND submission in 2027, pending successful completion of those studies. Under the collaboration terms, Gubra will receive a $4 million milestone at handover and is eligible for more than $50 million in success-based payments plus mid-single-digit royalties on worldwide sales.
Read Announcement
AMX0114 FDA Regulatory Timeline and Events
AMX0114 is a drug developed by Amylyx Pharmaceuticals for the following indication: Treatment of Amyotrophic Lateral Sclerosis.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- AMX0114
- Announced Date:
- December 5, 2025
- Indication:
- Treatment of Amyotrophic Lateral Sclerosis
Announcement
Amylyx Pharmaceuticals, Inc. announced the presentation of early safety and tolerability data from its Phase 1 LUMINA trial of AMX0114 and results from ongoing work characterizing biomarkers of AMX0114 target engagement at the 36th International Symposium on ALS/MND (MNDA) held from December 5-7 in San Diego, California.
AI Summary
Amylyx Pharmaceuticals presented early safety and tolerability results for AMX0114 from the Phase 1 LUMINA trial at the 36th International Symposium on ALS/MND in San Diego. In cohort 1 (n=12), AMX0114 was generally well tolerated and there were no treatment‑related serious adverse events reported.
The multinational, randomized, double‑blind, placebo‑controlled, multiple ascending dose LUMINA trial is assessing safety, tolerability, pharmacokinetics and pharmacodynamics of AMX0114, an antisense oligonucleotide targeting calpain‑2. Amylyx also presented ongoing work characterizing biomarkers of target engagement, including calpain‑2 activity and neurofilament light (NfL). Based on the safety data, the company plans to open enrollment for the second cohort in Canada later this month and in the U.S. in January. Cohort 1 biomarker data are expected in the first half of 2026.
Investigators said the early safety profile supports advancing the study and that the biomarker analyses will help show whether AMX0114 engages its intended target and affects disease‑relevant biology in people with ALS. Conference abstracts and presentations are available through the meeting and Amylyx’s events page.
Read Announcement- Drug:
- AMX0114
- Announced Date:
- June 3, 2025
- Indication:
- Treatment of Amyotrophic Lateral Sclerosis
Announcement
Amylyx Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to AMX0114, an investigational antisense oligonucleotide (ASO) targeting calpain-2 for the treatment of people living with amyotrophic lateral sclerosis (ALS).
AI Summary
Amylyx Pharmaceuticals announced that the FDA has granted Fast Track designation to its investigational AMX0114. This antisense oligonucleotide is designed to target calpain-2, a protein involved in the axonal degeneration seen in amyotrophic lateral sclerosis (ALS). Calpain-2 is known to play a key role in advancing ALS, a devastating and progressive disease with few treatment options.
The Fast Track status aims to facilitate a quicker development and review process by allowing more frequent interactions with the FDA. Preclinical studies have shown that AMX0114 can reduce calpain-2 activity, improve neuronal survival, and lower levels of nerve damage biomarkers. The company is moving forward with the Phase 1 LUMINA trial and anticipates early data later in 2025, marking an important step toward addressing the unmet needs of people living with ALS.
Read Announcement- Drug:
- AMX0114
- Announced Date:
- January 21, 2025
- Indication:
- Treatment of Amyotrophic Lateral Sclerosis
Announcement
Amylyx Pharmaceuticals, Inc announced that the U.S. Food and Drug Administration (FDA) has lifted the clinical hold placed on the Phase 1 clinical trial of AMX0114, an investigational antisense oligonucleotide (ASO) targeting calpain-2 for people living with amyotrophic lateral sclerosis (ALS).
AI Summary
Amylyx Pharmaceuticals announced that the FDA has lifted the clinical hold on its Phase 1 clinical trial for AMX0114, an investigational antisense oligonucleotide targeting calpain-2 in people with ALS. With approval now granted, the company is set to open trial sites across the U.S. for screening, enrollment, and dosing. The study, known as the LUMINA trial, will explore the safety and biological activity of AMX0114, assessing key ALS biomarkers such as neurofilament light levels. About 48 patients with ALS will be enrolled in a multicenter, randomized, placebo-controlled study, with participants receiving doses every four weeks. This milestone clears the way for further evaluation of AMX0114’s potential to slow axonal degeneration and offer a new treatment approach for those living with ALS, marking an important step forward in addressing unmet needs in this challenging disease.
Read Announcement
