Clene Inc.
For the period ended Jan 12, 2026. Filed Jan 12, 2026.
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Jan 12, 2026 · Filed Jan 12, 2026
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Contents
EX-99.1 · Alignment with Potential FDA Accelerated Approval Framework for CNM-Au8
Alignment with Potential FDA Accelerated Approval Framework for CNM-Au8
| 1. | NfL trajectory is independently associated with increased mortality risk in ALS Analyses across two large, independent ALS cohorts (APST, Answer ALS) demonstrated that longitudinal NfL increases were robustly and consistently associated with increased mortality risk (p<0.001), independent of baseline NfL and clinical covariates. Participants in the highest NfL slope categories experienced a significant 2.3-2.6-fold increased risk of death compared to patients with lower NfL slopes (p<0.001), with a clear NfL slope-dose response relationship observed across tertiles. |
| 2. | CNM-Au8 treatment consistently reduces NfL levels, a critical biomarker in ALS Concordant evidence from the HEALEY ALS Platform Trial 24-week double-blind period and the externally controlled NIH-sponsored EAP study at Week 36 demonstrated statistically significant and reproducible NfL reductions following CNM-Au8 treatment (HEALEY Week 24 Plasma NfL GMR 0.905, p=0.0403; NIH EAP Plasma NfL Week 36 full analysis set AUC difference −0.090, p=0.0373). These findings demonstrate consistent pharmacodynamic effects of CNM-Au8 on a clinically relevant ALS biomarker across independent study designs. |
| 3. | NfL reduction is quantitatively associated with improved survival Analyses of large ALS patient datasets demonstrate that even modest reductions in NfL are associated with improved survival. Across cohorts, the NfL reduction observed with CNM-Au8 treatment (approximately 9–10%) was associated with an approximately 8–13% lower risk of death, while larger NfL reductions were associated with proportionally greater survival benefit—a clinically meaningful benefit in a rapidly progressive disease with median survival of 2–4 years. |
These findings were robust across multiple complementary statistical methods, including parametric survival modeling, joint longitudinal–survival models, and Cox regression approaches designed to address missing biomarker data and survivorship bias. These analyses describe associations observed across ALS patient populations based on statistical models and do not directly represent estimates of treatment effect on survival. For example, from the HEALEY ALS Platform Trial long-term follow-up, CNM-Au8 30 mg treatment resulted in statistically significant survival improvement compared to concurrently randomized controls (HR: 0.272, 95% CI: 0.096 – 0.772, p=0.014) with 93% of participants alive at month 12, a pre-specified analysis timepoint.
Together, these results support the biological and clinical relevance of NfL trajectory as a prognostic biomarker in ALS and provide quantitative context for interpreting NfL changes observed in interventional clinical studies.
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