Clene Inc. (Nasdaq: CLNN) Advances Towards FDA Meeting with New Biomarker Data
SALT LAKE CITY, January 12, 2026 (GLOBE NEWSWIRE) – Clene Inc. (Nasdaq: CLNN), a leader in innovative treatments for neurodegenerative diseases like amyotrophic lateral sclerosis (ALS) and multiple sclerosis (MS), has announced a significant development: the FDA has granted the company an in-person Type C meeting scheduled for the first quarter of 2026. This meeting will focus on Clene's promising biomarker data which may support an upcoming New Drug Application (NDA).
Key Insights on Biomarkers and ALS Treatment
Recent analyses from large independent ALS cohorts demonstrate that modest reductions of approximately 10% in neurofilament light chain (NfL) levels are significantly linked to a lower risk of mortality. This finding positions NfL reduction as a potential surrogate endpoint for accelerated approval pathways. In addition, novel exploratory findings indicate that patients receiving CNM-Au8 30 mg who show a decline in the IGFBP7 biomarker had a markedly reduced mortality risk by 78% (HR 0.22, p=0.01) in the HEALEY ALS Platform Trial.
- In-Person FDA Meeting: Scheduled for Q1 2026.
- NfL Reductions: Linked to lower mortality risk.
- IGFBP7 Findings: Associated with a significant survival advantage.
Evidence Supporting NfL as a Biomarker for Accelerated Approval
Clene's pre-meeting briefing package submitted to the FDA includes analysis that highlights statistically significant reductions in both NfL and glial fibrillary acidic protein (GFAP) from the HEALEY ALS Platform Trial. These findings, which were previously announced in December 2025, provide survival evidence that could inform the FDA's consideration of CNM-Au8 for accelerated approval.
The package addresses critical FDA inquiries, such as:
- The clinical significance of observed NfL declines.
- The reproducibility of the NfL decline seen in the HEALEY ALS Platform Trial.
- Linking NfL declines to measurable clinical outcomes like survival.
Correlations and Potential Pathways for CNM-Au8
The data reveals a strong association between NfL levels and mortality rates in ALS, as shown in two independent studies (APST and Answer ALS). Higher NfL slopes correlated with a significantly increased risk of death (2.3-2.6 times) compared to those with lower NfL slopes.
Furthermore, evidence from the 24-week double-blind period of the HEALEY ALS Platform Trial and a NIH-sponsored Expanded Access Program indicates that CNM-Au8 reliably reduces NfL levels—a critical biomarker in ALS. Notably:
- Cohorts treated with CNM-Au8 showed a 9-10% reduction in NfL.
- This reduction correlated with an 8-13% lower risk of death.
- Long-term follow-up indicated a significant survival improvement with CNM-Au8 (HR: 0.272, p=0.014).
Exploratory Findings on IGFBP7
Clene has identified Insulin-like Growth Factor Binding Protein 7 (IGFBP7) as another biomarker that could signify the effectiveness of CNM-Au8 treatment. During the HEALEY ALS Platform Trial's double-blind phase, a decline in IGFBP7 was associated with a reduction in mortality by 78% among responders (HR: 0.22, p=0.012).
This finding suggests IGFBP7's potential role as a 'mechanistic hub' in a wider biomarker response to CNM-Au8 treatment, supported by the correlation with other relevant biomarkers and data indicating that lower IGFBP7 levels may protect against ALS progression.
CEO Statement and Future Outlook
Rob Etherington, Chief Executive Officer of Clene, expressed optimism about the forthcoming FDA meeting: “ALS remains a devastating disease with limited therapeutic options, and the field urgently needs biomarkers that can meaningfully inform drug development. We appreciate the FDA’s willingness to engage in a detailed discussion of our biomarker and survival data.”
As Clene prepares for its Type C meeting, the company aims to present comprehensive evidence in support of NfL and other emerging biomarkers to seek guidance on regulatory pathways for CNM-Au8, including the possibility of accelerated approval.