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Early pipeline

AMX0114

Also known as calpain-2 antisense oligonucleotide

Bottom line

Very early: a first safety test in people with ALS. The lowest dose caused no serious drug side effects but did not change nerve-damage markers. Higher doses are still being tested, and most drugs at this stage do not reach approval.

Very early. This drug is in its first safety trial in people and has not been tested for whether it helps in ALS. Most drugs at this stage never reach approval. More about early drugs

AMX0114 is an experimental genetic medicine given by spinal injection. It is meant to lower calpain-2, a protein thought to help break down the long nerve fibres that motor neurons use to reach muscles. It is in its first trial in people, LUMINA, which tests safety in about 48 people with ALS at rising doses. There is no evidence yet that it helps.

Information checked 5 Nov 2026

Evidence: Results pending

Studies: 1 running

Trials are running, but there are no efficacy results yet.

How we decide the overall verdict: How to read this site.

At a glance

CompanyAmylyx PharmaceuticalsExplanation and sources

Amylyx is a Nasdaq-listed company in Cambridge, Massachusetts, which also developed AMX0035 (see its own page). Its main focus is now avexitide, for low blood sugar after weight-loss surgery. AMX0114 is the ALS drug it is currently testing in people, and Amylyx designed it in-house.

Sources

  1. Amylyx Pharmaceuticals Reports Second Quarter 2026 Financial ResultsAmylyx Pharmaceuticals · 6 Aug 2026 · Company press release
  2. Amylyx Pharmaceuticals Announces First Participant Dosed in Phase 1, Multiple Ascending Dose LUMINA Trial of AMX0114 in People Living with ALSAmylyx Pharmaceuticals · 9 Apr 2025 · Company press release
How it worksSpinal injection to lower a protein that breaks down nerve fibresCalpain-2 (CAPN2) antisense oligonucleotideExplanation and sources

AMX0114 is an antisense oligonucleotide (ASO), a short strand of genetic material that blocks the instructions a cell uses to make one protein, here calpain-2. Calpain-2 is an enzyme that helps take apart axons, the long fibres that connect nerve cells to muscles, and too much of it is thought to drive nerve damage in ALS. In lab and animal studies AMX0114 lowered calpain-2 and improved nerve cell survival, but most drugs that work in the lab or in mice do not work in people.

Sources

  1. Amylyx Pharmaceuticals Receives U.S. FDA Fast Track Designation for AMX0114 for the Treatment of ALSAmylyx Pharmaceuticals · 3 Jun 2025 · Company press release
  2. Amylyx Pharmaceuticals Reports Second Quarter 2026 Financial ResultsAmylyx Pharmaceuticals · 6 Aug 2026 · Company press release
StagePhase 1Phase 1 in people with ALS, about 48 people · Dosing: people with alsExplanation and sources

LUMINA is the first time AMX0114 has been given to people. It tests four rising doses in groups of 12 people with ALS. The first person was dosed in April 2025; the first three groups (12.5, 25 and 50 mg) were fully enrolled by August 2026, and a fourth, higher dose is planned. The FDA gave the drug Fast Track status in June 2025, which can speed up later review but says nothing about whether it works.

Sources

  1. Amylyx Pharmaceuticals Announces First Participant Dosed in Phase 1, Multiple Ascending Dose LUMINA Trial of AMX0114 in People Living with ALSAmylyx Pharmaceuticals · 9 Apr 2025 · Company press release
  2. Amylyx Pharmaceuticals Receives U.S. FDA Fast Track Designation for AMX0114 for the Treatment of ALSAmylyx Pharmaceuticals · 3 Jun 2025 · Company press release
  3. Amylyx Pharmaceuticals Corporate PresentationAmylyx Pharmaceuticals · Company website
  4. Amylyx Pharmaceuticals to Present Data from First-in-Human, Dose-Ranging Phase 1 LUMINA Trial of AMX0114 in ALS at ENCALS 2026Amylyx Pharmaceuticals · 23 Jun 2026 · Company press release
Best reported effectSafety study; no effect data yetSafety; calpain-2, NfL and other blood and spinal-fluid markersExplanation and sources

LUMINA is a safety study and is too small and short to show whether AMX0114 slows ALS. In the first group of 12 people (lowest dose), nerve-damage markers such as NfL stayed near their starting levels. Amylyx said this was not unexpected at such a low dose. Results from higher doses have not been reported.

This is the most favourable result reported. See all key studies for the full picture.

Sources

  1. Amylyx Pharmaceuticals to Present Data from First-in-Human, Dose-Ranging Phase 1 LUMINA Trial of AMX0114 in ALS at ENCALS 2026Amylyx Pharmaceuticals · 23 Jun 2026 · Company press release
  2. Advancing AMX0114 for ALS: Updates From The LUMINA Phase 1 Study (ENCALS 2026 poster)Amylyx Pharmaceuticals · 24 Jun 2026 · Conference presentation
Who it's forPeople with ALS, symptoms began within the last 2 yearsAll ALS · Spinal injection every 4 weeksExplanation and sources

The trial is open to adults with ALS whether or not they have a known ALS gene, whose first symptoms began less than 24 months ago and whose breathing capacity is at least 65% of normal. Participants can stay on their usual ALS medicines. The drug is given by lumbar puncture, a needle into the lower back.

Sources

  1. Advancing AMX0114 for ALS: Updates From The LUMINA Phase 1 Study (ENCALS 2026 poster)Amylyx Pharmaceuticals · 24 Jun 2026 · Conference presentation
  2. AMX0114 in Adult Participants With Amyotrophic Lateral Sclerosis (NCT06665165)ClinicalTrials.gov · Trial registry
What's nextSafety and marker results from the higher-dose groups of LUMINAExpected 2027Explanation and sources

Amylyx has not given a date for results from the higher doses. The trial registry estimates the main data will be collected by February 2027 and the study will finish in October 2027.

Sources

  1. AMX0114 in Adult Participants With Amyotrophic Lateral Sclerosis (NCT06665165)ClinicalTrials.gov · Trial registry
  2. Amylyx Pharmaceuticals Corporate PresentationAmylyx Pharmaceuticals · Company website
Can I join?Recruiting in the USA and CanadaRecruiting nowExplanation and sources

The registry lists LUMINA as recruiting at about 14 sites in the US and Canada. Each person has a 1 in 4 chance of getting placebo. The third dose group filled in August 2026, so places depend on the planned fourth group opening. We found no expanded access programme.

Sources

  1. AMX0114 in Adult Participants With Amyotrophic Lateral Sclerosis (NCT06665165)ClinicalTrials.gov · Trial registry
  2. Amylyx Pharmaceuticals Corporate PresentationAmylyx Pharmaceuticals · Company website

Signal: Active

Signal measures how often, and how reliably, Amylyx Pharmaceuticals tells the public about this drug. It says nothing about whether the drug works. A quiet company isn't necessarily a bad sign, but regular, on-time updates help patients plan.

Last company update6 Aug 2026 (3 months ago)12 / 40
Company updates in the last 12 months635 / 35
Promises kept2 of 2 on time25 / 25
Signal score72 / 100 · Active

Announced milestones

70 or more is Active, 45 or more is Steady, 20 or more is Quiet, below 20 is Silent. Full method.

Timeline

10 events, newest first. Company updates are marked; independent news and regulator decisions are shown too.

2026

  1. Investor updateFrom the companyAmylyx Pharmaceuticals

    Third dose group enrolling

    In its quarterly report Amylyx said the 50 mg group was enrolling; its later investor presentation said this group filled in August 2026.

  2. ConferenceFrom the companyAmylyx Pharmaceuticals

    Lowest dose did not change nerve-damage markers

    Ahead of the ENCALS meeting, Amylyx reported that markers such as NfL stayed near baseline at the lowest dose, with no drug-related serious side effects. Higher doses are still being tested.

  3. Investor updateFrom the companyAmylyx Pharmaceuticals

    Second dose group fully enrolled

    Amylyx said enrolment of the second group of 12 people (25 mg) was completed in March 2026.

  4. Investor updateFrom the companyAmylyx Pharmaceuticals

    Year-end update on LUMINA

    Amylyx repeated that the lowest dose was well tolerated and said marker data from the first group would be presented in the first half of 2026.

2025

  1. ConferenceFrom the companyAmylyx Pharmaceuticals

    First safety data from the lowest dose

    At the International Symposium on ALS/MND, Amylyx reported no treatment-related serious side effects in the first 12 people, and said it would open the second, higher dose group.

  2. Investor updateFrom the companyAmylyx Pharmaceuticals

    First dose group fully enrolled

    In its quarterly report Amylyx said the first group of 12 people had been enrolled in September 2025, with safety data due in December and marker data in the first half of 2026.

  3. NewsALS News Today

    ALS News Today reports Fast Track status

    ALS News Today reported the Fast Track designation and that the first LUMINA participant had been dosed at a site in Ontario.

  4. Press releaseFrom the companyAmylyx Pharmaceuticals

    FDA grants Fast Track designation

    Amylyx announced Fast Track status for AMX0114 in ALS. This can mean more contact with the FDA and a faster review later, but it is not a judgement that the drug works.

  5. Press releaseFrom the companyAmylyx Pharmaceuticals

    First participant dosed in LUMINA

    Amylyx said the first person with ALS had received AMX0114 in its Phase 1 trial, which aims to enrol about 48 people in North America. It expected early data later in 2025.

2024

  1. Trial registryClinicalTrials.gov

    LUMINA trial registered

    The first trial of AMX0114 in people with ALS was posted on ClinicalTrials.gov.

All sources for this page

  1. Amylyx Pharmaceuticals Announces First Participant Dosed in Phase 1, Multiple Ascending Dose LUMINA Trial of AMX0114 in People Living with ALSAmylyx Pharmaceuticals · 9 Apr 2025 · Company press release
  2. Amylyx Pharmaceuticals Receives U.S. FDA Fast Track Designation for AMX0114 for the Treatment of ALSAmylyx Pharmaceuticals · 3 Jun 2025 · Company press release
  3. AMX0114 receives FDA fast track status for the treatment of ALSALS News Today · 10 Jun 2025 · News
  4. Amylyx Pharmaceuticals Reports Third Quarter 2025 Financial ResultsAmylyx Pharmaceuticals · 6 Nov 2025 · Company press release
  5. Amylyx Pharmaceuticals Announces New Safety and Tolerability Cohort 1 Data of AMX0114 in ALS from First-in-Human LUMINA TrialAmylyx Pharmaceuticals · 5 Dec 2025 · Company press release
  6. Amylyx Pharmaceuticals Reports Fourth Quarter and Full Year 2025 Financial ResultsAmylyx Pharmaceuticals · 3 Mar 2026 · Company press release
  7. Amylyx Pharmaceuticals Reports First Quarter 2026 Financial ResultsAmylyx Pharmaceuticals · 7 May 2026 · Company press release
  8. Amylyx Pharmaceuticals to Present Data from First-in-Human, Dose-Ranging Phase 1 LUMINA Trial of AMX0114 in ALS at ENCALS 2026Amylyx Pharmaceuticals · 23 Jun 2026 · Company press release
  9. Advancing AMX0114 for ALS: Updates From The LUMINA Phase 1 Study (ENCALS 2026 poster)Amylyx Pharmaceuticals · 24 Jun 2026 · Conference presentation
  10. Amylyx Pharmaceuticals Reports Second Quarter 2026 Financial ResultsAmylyx Pharmaceuticals · 6 Aug 2026 · Company press release
  11. Amylyx Pharmaceuticals Corporate PresentationAmylyx Pharmaceuticals · Company website
  12. AMX0114 in Adult Participants With Amyotrophic Lateral Sclerosis (NCT06665165)ClinicalTrials.gov · Trial registry