- Two “Compassionate Use” Expanded Access Programs (EAPs) provided access to treatment with CNM-Au8 to more than 250 people living with amyotrophic lateral sclerosis (ALS).
- A significant survival benefit (p=0.0001) was observed in EAP participants treated with CNM-Au8 compared to historical ALS disease progression controls (participants untreated with CNM-Au8) in two independent analyses: a 68% decreased risk of all-cause mortality compared to PRO-ACT matched controls, and a 57% decreased risk of all-cause mortality compared to
ALS Natural History Consortium matched controls. - CNM-Au8 30 mg treatment was well-tolerated, without a single serious adverse event attributed to CNM-Au8, and no significant safety findings reported.
A pooled survival analysis of EAP participants treated with CNM-Au8 30 mg was compared to two independent datasets derived from PRO-ACT and the
The results in the EAP participants versus the matched controls demonstrated a significant survival benefit for each comparison:
- The PRO-ACT dataset is the largest publicly available repository of longitudinal ALS clinical trial data, containing more than 12,000 records of trial participants:
- CNM-Au8 EAP vs. PRO-ACT matched controls: The baseline risk-adjusted hazard ratio demonstrated a 68% decreased risk of all-cause mortality with CNM-Au8 treatment (HR: 0.320, 95% CI: 0.178 – 0.575, p = 0.0001).
- CNM-Au8 EAP vs. PRO-ACT matched controls: The baseline risk-adjusted hazard ratio demonstrated a 68% decreased risk of all-cause mortality with CNM-Au8 treatment (HR: 0.320, 95% CI: 0.178 – 0.575, p = 0.0001).
The ALS/MND Natural History Consortium data set contained approximately 1,700 records of people living with ALS from researchers across nine academic sites collecting recent real-world data:- CNM-Au8 EAP vs.
ALS/MND Natural History Consortium matched controls: The baseline risk-adjusted hazard ratio demonstrated a 57% decreased risk of all-cause mortality with CNM-Au8 treatment (HR: 0.433, 95% CI: 0.282 – 0.663, p = 0.0001).
- CNM-Au8 EAP vs.
Analyses including all 256 EAP participants compared to the 220 matched controls also showed statistically significant survival benefits with log-rank p-values of p < 0.0001 and p=0.006 for the
“The long-term safety and survival data from the CNM-Au8 expanded access programs add to the available data supporting CNM-Au8 moving rapidly to Phase 3 testing in ALS,” said
“These EAP results help us better understand how people living with more advanced disease respond to treatment,” said
An EAP is an FDA-regulated pathway that allows patients with a serious or immediately life-threatening condition to gain access to an investigational drug outside of clinical trials when no comparable or satisfactory alternative therapy is available.
The Sean M. Healey &
The second EAP (EAP02) was started in 2021 for people living with ALS who did not qualify for participation in the concurrent HEALEY ALS Platform Trial, which is a perpetual multi-center, randomized, double-blind, placebo-controlled clinical trial program designed to evaluate the efficacy and safety of multiple investigational products in people living with ALS. EAP02 is sponsored by
As previously announced, treatment with CNM-Au8 produced consistent survival and delayed time to ALS clinical worsening data in two independent Phase 2, randomized, placebo-controlled, double-blind ALS trials and their open-label extensions. The Phase 2 HEALEY ALS Platform Trial in the
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