New Data from Clene’s RESCUE-ALS Study to be Presented at 2022 ENCALS Meeting - Seite 2

Poster 3: RESCUE-ALS Trial Results: A Phase 2, Randomized, Double-Blind, Placebo-Controlled Study of CNM-Au8 to Slow Disease Progression in ALS

• The results from the RESCUE- ALS study provide evidence of safety and suggest efficacy of CNM-Au8 in patients with early ALS. The 36-week blinded period of RESCUE-ALS suggested efficacy benefits with CNM-Au8 treatment versus placebo: slowing ALS disease progression (p=0.0125), decreasing the proportion of participants with a 6-point decline in the ALS Functional Rating Scale Revised (ALSFRS-R) (p=0.035) and improving quality of life as measured by the ALS Specific Quality of Life (ALSSQOL-SF) questionnaire (p=0.018). 
  

Poster 4: Interim ALS Specific Quality of Life Results from the Long-Term Open Label Extension of RESCUE-ALS, a Double-Blind, Placebo-Controlled Study of CNM-Au8 to Slow Disease Progression in ALS

• CNM-Au8 treatment resulted in improved quality of life assessed by the ALS Specific QOL short-form scale [ALSSQOL-SF), (p=0.018) during the 36-week double-blind period). Furthermore, during the OLE, subjects originally randomized to CNM-Au8 continued to maintain stable ALSSQOL-SF scores through 84 weeks or more of treatment. Ex-placebo participants who transitioned to the CNM-Au8 treatment during the long-term OLE demonstrated similar stability.
  

Poster 5: Preliminary Biomarker Findings from the RESCUE-ALS Double-Blind, Placebo-Controlled Study of CNM-Au8 to Slow Disease Progression in ALS

• CNM-Au8 treated participants in the RESCUE-ALS study demonstrated decreased levels of markers of neurodegeneration, specifically plasma ubiquitin C-terminal hydrolase L1 and urinary neurotrophin receptor p75 extracellular domain normalized to creatine levels, compared to placebo over the 36-week double-blind period.
  

“These emerging data highlight the significantly reduced risk of mortality following early and sustained treatment with CNM-Au8.,” said Rob Etherington, President and CEO of Clene. “The magnitude of the effect, more than 60% reduction in risk versus delayed treatment, provides hope for people with ALS and their caregivers. Based on these emerging survival data, the RESCUE-ALS open-label has been extended indefinitely, and we are excited to understand how CNM-Au8 treatment may continue to keep people with ALS alive and with stable quality of life.”