CNM-Au8

Clene is a small company based in Salt Lake City, Utah, and CNM-Au8 is its only drug, also studied in multiple sclerosis. Money is tight: it had $9.9 million in cash at the end of September 2026, which it says lasts only until the middle of the first quarter of 2027. More money from warrants depends on the FDA accepting its application for review.

The gold crystals are meant to speed up chemical reactions that cells use to produce energy, helping stressed motor neurons and their support cells survive and work better, and reducing harmful oxidative stress. This mechanism comes mostly from lab studies and is not proven in people.

Clene submitted a New Drug Application in October 2026 asking for accelerated approval, a pathway where the FDA can approve a drug based on a marker (here NfL) that is "reasonably likely" to predict benefit, with a later trial needed to confirm it. The FDA first decides whether to accept the file for review, then whether to approve. A confirmatory Phase 3 trial, RESTORE-ALS, has not started.

In the HEALEY ALS Platform Trial, CNM-Au8 did not slow decline on the ALS functional score compared with placebo, and the earlier RESCUE-ALS trial also missed its main goal. In HEALEY, NfL, a blood marker of nerve damage, rose about 8% on placebo and fell about 2% on CNM-Au8 over 24 weeks, an exploratory finding. Company analyses, many done after the fact or using comparison groups from outside the trial, link NfL drops to longer survival. It is not yet proven that lowering NfL with this drug helps people live longer or function better.

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

HEALEY and the expanded access programmes included adults with sporadic or familial ALS. Clene's application covers ALS generally rather than one genetic form; the exact proposed label has not been published.

After an application is submitted, the FDA first decides whether it is complete enough to review. Clene has not given a date for this decision. Separately, it plans to start the RESTORE-ALS Phase 3 trial in the second quarter of 2027.

ClinicalTrials.gov lists one CNM-Au8 expanded access programme (NCT05281484) as available at participating US sites; ask your ALS clinic whether it takes part. The NIH-funded programme (NCT06408727) is listed as temporarily not available. No placebo-controlled trial is enrolling; RESTORE-ALS is planned for 2027.

• CNM-Au8 expanded access (EAP01/02/06) (NCT05281484) available · USA
• NIH-funded expanded access (EAP04) (NCT06408727) unknown · USA