MND Australia


Phase 2

What is the RESCUE-ALS Study?

This is a multi-centre randomised, double-blind, parallel group, placebo-controlled Phase 2 study of the efficacy, safety, pharmacokinetics, and pharmacodynamics of CNM-Au8. CNM-Au8 is an oral, gold nanocrystal liquid suspension designed to assist cells with the energy they need to function well. It works by supporting bioenergetic cellular reactions, and helping to remove the toxic byproducts of cellular metabolism that add to the breakdown of motor neurons in ALS.

The drug has been developed by Clene Nanomedicine.

Preclinical studies have shown the nanomedicine improves nerve cell survival and response to cellular stress, and lowers intracellular levels of toxic reactive oxygen species. CNM-Au8 was seen to improve motor function in rodent models ALS, multiple sclerosis, and Parkinson’s disease.

CNM-Au8 was also found to be safe in a Phase 1 trial of escalating doses in healthy volunteers.

What is involved?

The randomised, double-blind and placebo-controlled Phase 2 study, called RESCUE-ALS, will enroll up to 42 people, ages 40 to 80, with newly evident disease symptoms at two sites in Australia. Symptoms are considered newly evident if they began within 24 months of screening for this study, or 12 months of a probable or definite ALS diagnosis.

Participants will be treated with CNM-Au8 or given a liquid placebo once each morning for 36 consecutive weeks (about eight months), in addition to standard care. Those who complete this treatment period may move into a separate and open-label study, where all will receive CNM-Au8.

The objective of the RESCUE-ALS study is to demonstrate that improvements in brain bioenergetic cellular support in early symptomatic ALS patients treated with CNM-Au8 will help preserve motor neurons survival and function. This safety and efficacy was study due to finish in April 2021.

How far off is a treatment?

A possible treatment resulting from this study is years away. This research will provide preliminary data on the safety and efficacy of CNM-Au8 as well as best dosing regimes.