CNM-Au8 (REPAIR-PD)

The research record

This is the project's file for this landmark, as written and checked. Sources are listed at the end; anything not yet confirmed is marked "Not yet verified".

Who

Clene Nanomedicine (Salt Lake City); lead author of the congress abstract R. Glanzman, with Richard Dewey (Holladay, USA, per the abstract listing). Results were presented at the International Parkinson and Movement Disorder Society Virtual Congress in September 2021 (abstract 73), summarised in a Clene release on 9 November 2021, and posted to the registry in May 2024.

What happened

Parkinson's brains show failing energy machinery (landmark 11). CNM-Au8 is a suspension of gold nanocrystals that the company says speeds the cell's energy reactions and lifts the NAD/NADH ratio (a measure of the cell's energy state).

• Design. Phase 2, open-label, sequential-cohort, single site, not randomised (investigators blinded to dose). 13 people with Parkinson's diagnosed within 3 years (REPAIR-PD) took CNM-Au8 daily for 12 weeks; 11 people with multiple sclerosis (REPAIR-MS, a sister study) were analysed alongside. Brain energy was measured by phosphorus magnetic resonance spectroscopy before and after. The registry results list one Parkinson's arm, 30 mg a day; the company says 15 mg or 30 mg were used.
• Result. In both groups pooled (24 people), the brain NAD+/NADH ratio rose by about 10.4% (0.589 units, p=0.037, paired t-test; company release). In the Parkinson's group alone the rise was not statistically significant: the registry gives a mean change of 0.39 (SD 0.80), p=0.1077, and the company p=0.11.
• Safety. No serious adverse events in the 13 people (registry).
• Not measured. No placebo group. Clinical measures (gait, balance, MDS-UPDRS) were listed as exploratory; I did not read their posted values.

Why it moved us toward a cure

It showed a brain-energy marker could be moved by an oral drug and read with a scan, the same measure used in the UDCA study (landmark 150). It does not show any benefit to patients. The later NOPARK result (landmark 172) shows that raising NAD does not guarantee a slowing effect.

Outcome

Open, but weak: a signal on one imaging marker in 13 people. In its 2021 release Clene listed a planned Phase 2 "RESCUE-PD" efficacy trial, but no Parkinson's trial of CNM-Au8 beyond this one is registered on ClinicalTrials.gov (19 Oct 2026); the company's later work is in ALS and MS.

Sources

• ClinicalTrials.gov NCT03815916 (API results section, 19 Oct 2026; start 19 Dec 2019, primary completion 7 June 2021)
• MDS Congress 2021 abstract 73
• Clene Q3 2021 release (9 Nov 2021) via SEC filings
• Not yet verified: any peer-reviewed publication of REPAIR-PD; the pooled numbers (company release only); the posted clinical measures; the statement that the later work is in ALS and MS (from search snippets, not read).