Clene's Amyotrophic Lateral Sclerosis Candidate Disappoints In Mid-Stage Study

Clene Inc CLNN has announced topline data from RESCUE-ALS phase 2 trial evaluating CNM-Au8 for early amyotrophic lateral sclerosis (ALS).
The trial did not meet the primary or secondary endpoints, Motor Unit Number Index (MUNIX), and forced vital capacity (FVC) at week 36.
In a pre-specified analysis of limb onset ALS subset, CNM-Au8 demonstrated a significant treatment effect in MUNIX at week 12 and a trend for improvement at week 36.
Limb onset ALS accounts for approximately 70% of the ALS population.
MUNIX is a neurophysiological biomarker that estimates the number of functioning lower motor neurons serving selected muscles.
Clinically relevant exploratory endpoints demonstrated significant benefits with CNM-Au8 treatment, including slowing ALS disease progression and improved quality of life.
In addition, the data showed evidence for a potential long-term survival benefit when comparing the survival of the trial population to the validated ENCALS predictive model.
CNM-Au8 was found to be well-tolerated through 36 weeks of daily oral dosing. There were no reported serious adverse events (SAEs) related to CNM-Au8 treatment.
Treatment-emergent adverse events were predominantly mild-to-moderate in severity.
Related Link: Clene's Nanotherapeutic Improves Energy Production, Brain Utilization In Patients With Parkinson's, Multiple Sclerosis.
Price Action: CLNN shares are down 20.20% at $4.39 during the market session on the last check Tuesday.