Clene’s CNM-Au8 Shows Statistically Significant Difference in Plasma Neurofilament Light (NfL) levels in the HEALEY ALS Platform Trial

• CNM-Au8 treatment demonstrated significantly reduced plasma neurofilament light chain (NfL) levels compared to placebo at 24 weeks (p=0.04)
  
• Clene plans to discuss the totality of the survival and time-to-event results from the full CNM-Au8 clinical data set, including the plasma NfL biomarker data, at an upcoming FDA meeting to accelerate the path toward approval

SALT LAKE CITY, June 15, 2023 (GLOBE NEWSWIRE) -- Clene Inc. (NASDAQ: CLNN) (along with its subsidiaries, “Clene”) and its wholly owned subsidiary, Clene Nanomedicine, Inc., a clinical-stage biopharmaceutical company focused on revolutionizing the treatment of neurodegenerative diseases, today reported new data demonstrating a statistically significant reduction of plasma neurofilament light chain (NfL) levels for CNM-Au8 treated participants compared to placebo after 24 weeks of treatment in the double-blind, placebo-controlled period of the HEALEY ALS Platform Trial.

NfL is a key biomarker of neurodegeneration. NfL is released from neurons following axonal injury, especially in people living with ALS, where higher levels of NfL have been found to predict more rapid decline in clinical function and increased mortality risk. Surrogate biomarkers such as NfL have recently been used to support an FDA approval for the treatment of ALS.

Six-Month Plasma NfL Biomarker Findings from the Regimen C (CNM-Au8) Arm in the HEALEY ALS Platform Trial

The results are based on an analysis of the plasma NfL biomarker across all Regimen C participants (CNM-Au8 or placebo, n=161), representing a broad ALS population, as the least-square mean (LS mean) change of the natural logarithm (Ln) of the plasma NfL values with the standard error (SE):

• CNM-Au8 treatment reduced plasma NfL levels compared to placebo; LS Means on a Ln Scale for the 24-week difference of plasma NfL: CNM-Au8 = -0.024 (SE: 0.024); placebo = +0.076 (SE: 0.042); CNM-Au8 vs. placebo difference of LS Means on a Ln Scale = -0.100 (SE: 0.048), p=0.040.
  

ALS Participants at the Greatest Risk of Disease Progression Showed Consistent Benefit

In addition to the full analysis across all Regimen C participants, sensitivity analyses showed consistent significant reduction in plasma NfL levels versus placebo observed in specific populations generally considered at greater risk of ALS disease progression, including:

• Faster progressors (baseline pre-treatment ALSFRS-R slope >0.45 points/month (post hoc, n=107); Difference of LS Means on a Ln Scale (SE) = -0.144 (0.058); p=0.014.
• Definite or probable ALS diagnosis per El Escorial criteria (post hoc, n=125); Difference of LS Means on a Ln Scale (SE) = -0.124 (0.054); p=0.023.
• Higher mortality risk (baseline plasma NfL > median, post hoc, n=79); Difference of LS Means on a Ln Scale (SE) = -0.150 (0.068); p=0.031.