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CuATSM

Also known as Cu-ATSM, Cu(II)ATSM, copper-ATSM

Bottom line

Early open-label results looked striking, but they had no placebo group. The placebo-controlled trial meant to test them finished in 2021 and its results were never released, so there is no real evidence that CuATSM helps people with ALS.

CuATSM is a copper-containing pill that was meant to protect nerve cells by delivering copper to cells with damaged energy machinery. A small open-label Phase 1 study in Australia raised hopes in 2019, but the placebo-controlled Phase 2/3 trial that followed finished years ago and its results were never released. The company has since rebranded and moved on to other work, and ALS organisations now list CuATSM as no longer in development.

Information checked 5 Nov 2026

Evidence: Early signal only

Studies: 1 against · 2 mixed · 1 running

Only small, open-label or historical-comparison studies so far. Promising at best; not proof.

Slower decline in open-label Phase 1 only

0.29 points lost a month vs about 1.02 expected from historical records, on the 48-point ALSFRS-R (no placebo group)

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

At a glance

CompanyCollaborative Medicinal Development (now Vivat Therapeutics)Explanation and sources

CuATSM was developed by Collaborative Medicinal Development (CMD), a small private company in California that licensed it from the University of Melbourne and ran its trials in Australia. The company's website now says CMD has become Vivat Therapeutics, whose lead drug is for age-related muscle loss, not ALS. We could not find when the rebrand happened.

Sources

  1. Collaborative Medicinal Development home page (now Vivat Therapeutics)Collaborative Medicinal Development · Company website
  2. Collaborative Medicinal Development, LLC Reports That Lead Drug Modifies ALS ProgressionPR Newswire · 7 Jan 2019 · Company press release
  3. Motor neurone disease (MND) breakthrough: patient trial shows impressive clinical resultsUniversity of Melbourne · News
How it worksDelivers copper to stressed cells to protect them from damageCopper delivery / peroxynitriteExplanation and sources

CuATSM is a small molecule that carries copper into the brain and spinal cord. The idea was that it releases copper mainly in cells whose mitochondria (energy factories) are failing, helping copper-dependent proteins like SOD1 work properly and mopping up a harmful molecule called peroxynitrite. It worked well in SOD1 mice, but those results did not need to translate to people.

Sources

  1. Motor neurone disease (MND) breakthrough: patient trial shows impressive clinical resultsUniversity of Melbourne · News
  2. What's the story with CuATSM?ALS Canada · Patient organisation
  3. CuATSM (drugs no longer in development)ALS/MND Alliance · Patient organisation
StageDiscontinuedPhase 2/3 finished in 2021–2022; results never releasedExplanation and sources

After the Phase 1 study, a placebo-controlled Phase 2/3 trial of up to 80 people started in Australia in September 2019, followed by a continuation study that the registry lists as completed in February 2022. No results have been published or announced. The ALS/MND Alliance now lists CuATSM among drugs no longer in development.

Sources

  1. CuATSM Compared With Placebo for Treatment of ALS/MND (NCT04082832)ClinicalTrials.gov · Trial registry
  2. Continuation study of CuATSM in ALS/MND (NCT04313166)ClinicalTrials.gov · Trial registry
  3. CuATSM treatment trialMND Association · Patient organisation
  4. CuATSM (drugs no longer in development)ALS/MND Alliance · Patient organisation
What happenedPhase 2/3 finished but results never released; company moved to other diseases15 Feb 2022Explanation and sources

The continuation study of the Phase 2/3 trial is listed as completed on 15 February 2022, the last trial activity we found. No results followed, and as of 2025 the ALS Therapy Development Institute noted results still had not been shared. The company now presents itself as Vivat Therapeutics working on a different drug, and patient organisations list CuATSM as no longer in development.

Sources

  1. Continuation study of CuATSM in ALS/MND (NCT04313166)ClinicalTrials.gov · Trial registry
  2. Copper-ATSM as a Potential Treatment for ALSALS Therapy Development Institute · Patient organisation
  3. Collaborative Medicinal Development home page (now Vivat Therapeutics)Collaborative Medicinal Development · Company website
  4. CuATSM (drugs no longer in development)ALS/MND Alliance · Patient organisation
Best reported effectSlower decline in open-label Phase 1 only0.29 points lost a month vs about 1.02 expected from historical records, on the 48-point ALSFRS-R (no placebo group)Explanation and sources

In the open-label Phase 1 study (32 people, 29 finished), people on the highest dose lost about 0.29 ALSFRS-R points per month, compared with about 1.02 expected from historical records of other patients. There was no placebo group, so this comparison is weak. The placebo-controlled Phase 2/3 trial that should have confirmed or refuted it never reported results. A 2023 study of brain and spinal cord tissue from six treated people who died found no clear protection of nerve cells, though there were fewer inflammatory immune cells (microglia) in the spinal cord.

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

Sources

  1. Collaborative Medicinal Development, LLC Reports That Lead Drug Modifies ALS ProgressionPR Newswire · 7 Jan 2019 · Company press release
  2. What's the story with CuATSM?ALS Canada · Patient organisation
  3. Treatment with the copper compound CuATSM has no significant effect on motor neuronal pathology in patients with ALSNeuropathology and Applied Neurobiology · 15 Jun 2023 · Peer-reviewed paper
  4. Copper-ATSM as a Potential Treatment for ALSALS Therapy Development Institute · Patient organisation
Who it's forAdults with sporadic or familial ALS (trials in Australia only)All ALS · Pill, dailyExplanation and sources

The trials enrolled adults with ALS in Australia, regardless of whether the disease was inherited. CuATSM was taken by mouth once a day.

Sources

  1. CuATSM Compared With Placebo for Treatment of ALS/MND (NCT04082832)ClinicalTrials.gov · Trial registry
  2. CuATSM treatment trialMND Association · Patient organisation
Can I join?No trials runningExplanation and sources

There is no way to get CuATSM through a trial or an access programme. The main Phase 2/3 study's registry entry was never updated and shows an "unknown" status.

Sources

  1. CuATSM Compared With Placebo for Treatment of ALS/MND (NCT04082832)ClinicalTrials.gov · Trial registry
  2. Continuation study of CuATSM in ALS/MND (NCT04313166)ClinicalTrials.gov · Trial registry
  3. CuATSM (drugs no longer in development)ALS/MND Alliance · Patient organisation

Timeline

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

2025

  1. NewsALS News Today

    Mouse study tests CuATSM in a three-drug combination

    An Australian lab study reported that CuATSM combined with two other drugs helped SOD1 mice. This was academic animal research, not a sign that human development has restarted.

2023

  1. PublicationNeuropathology and Applied Neurobiology

    Post-mortem study finds no clear nerve protection

    Researchers compared spinal cord and brain tissue from six people who took CuATSM with six who did not and found no significant difference in nerve cell numbers or TDP-43 build-up.

2022

  1. Trial registryClinicalTrials.gov

    Continuation study listed as completed

    The follow-on study for Phase 2/3 participants is recorded as completed. This is the last trial activity we found; no results were posted or announced.

2019

  1. Trial registryClinicalTrials.gov

    Placebo-controlled Phase 2/3 trial starts in Australia

    The registry lists the start of a 24-week trial comparing CuATSM with placebo in up to 80 people in Australia.

  2. Press releaseFrom the companyCollaborative Medicinal Development

    Company reports slower decline in open-label Phase 1

    Collaborative Medicinal Development said people taking the top dose in its open-label Phase 1 study declined more slowly than expected from historical records. There was no placebo group.

All sources for this page

  1. Collaborative Medicinal Development home page (now Vivat Therapeutics)Collaborative Medicinal Development · Company website
  2. Collaborative Medicinal Development, LLC Reports That Lead Drug Modifies ALS ProgressionPR Newswire · 7 Jan 2019 · Company press release
  3. Motor neurone disease (MND) breakthrough: patient trial shows impressive clinical resultsUniversity of Melbourne · News
  4. What's the story with CuATSM?ALS Canada · Patient organisation
  5. Copper-ATSM as a Potential Treatment for ALSALS Therapy Development Institute · Patient organisation
  6. CuATSM (drugs no longer in development)ALS/MND Alliance · Patient organisation
  7. CuATSM treatment trialMND Association · Patient organisation
  8. CuATSM Compared With Placebo for Treatment of ALS/MND (NCT04082832)ClinicalTrials.gov · Trial registry
  9. Phase 1 Dose Escalation and PK Study of Cu(II)ATSM in ALS/MND (NCT02870634)ClinicalTrials.gov · Trial registry
  10. CuATSM Phase 2a study confirms disease-modifying effects in patients with sporadic ALS observed in the Phase 1 study (abstract 1338)Neurology (AAN 2020 abstract) · 14 Apr 2020 · Conference presentation
  11. Continuation study of CuATSM in ALS/MND (NCT04313166)ClinicalTrials.gov · Trial registry
  12. Treatment with the copper compound CuATSM has no significant effect on motor neuronal pathology in patients with ALSNeuropathology and Applied Neurobiology · 15 Jun 2023 · Peer-reviewed paper