VISIONARY-MS Trial Results Featured in Platform Presentation at PACTRIMS 2022: CNM-Au8® Demonstrated Global Neurological Improvement in Stable MS Patients as Adjunct to Background Disease Modifying Therapies

•    CNM-Au8 demonstrated low contrast vision improvement and global neurological improvement (low contrast vision, cognition, upper extremity function, and walking speed) in stable MS patients as adjunct to background immunomodulating disease modifying therapies (DMTs)   
 
•    No approved MS DMTs have shown global neurological improvement in stable MS patients, a significant unmet medical need in MS   
 
•    CNM-Au8 treatment was well-tolerated, and no significant safety findings were observed   
  
 

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 announced that the VISIONARY-MS trial results were featured as a platform presentation by Professor Michael Barnett, MBBS FRACP PhD at the 14th Annual Singapore Pan-Asian Committee on Treatment and Research in Multiple Sclerosis (PACTRIMS) Congress held November 24-26.

The platform presentation titled, " VISIONARY-MS Top-line Results: A Phase 2, Randomized, Double-Blind, Parallel Group, Placebo-Controlled Study to Assess the Safety and Efficacy of CNM-Au8, a Catalytically Active Gold Nanocrystal Suspension in Relapsing Multiple Sclerosis ," provided proof-of-concept evidence for global neurological improvement as assessed by the modified Multiple Sclerosis Functional Composite (mMSFC), evaluating low contrast vision, cognition, upper extremity function, and walking speed with CNM-Au8 as adjunct to approved background immunomodulatory disease modifying therapies (DMTs) in stable MS patients.

VISIONARY-MS investigated the efficacy and safety of CNM-Au8 (15 mg or 30 mg daily) versus placebo over 48 weeks in stable relapsing remitting MS participants with chronic optic neuropathy. Nearly all participants (92%) were treated with highly effective DMTs as background standard of care.

• The primary endpoint, evaluating change in low contrast letter acuity (LCLA) compared to placebo at week 48, demonstrated significant improvement (mITT population, least squares [LS] mean difference, 3.13; 95% CI: -0.08 to 6.33, p = 0.056).
 
• Secondary endpoint of global neurological improvement, the modified Multiple Sclerosis Functional Composite (mMSFC), mean standardized change compared to placebo at week 48, demonstrated significant improvement (mITT population, LS mean difference, 0.28; 95% CI: 0.04 to 0.52, p = 0.0207).
 
• Consistent improvements favoring CNM-Au8 were observed across paraclinical biomarkers, including multifocal visual evoked potential amplitude and latency, measurements of retinal structure with optical coherence tomography, and novel MRI endpoints examining myelin and axonal integrity. These data provided consistent supportive evidence from neurophysiology, retinal imaging, and novel MRI markers for the potential neuroprotective and remyelinating effects of CNM-Au8 treatment.
 
• Placebo treated patients generally worsened across clinical and paraclinical measures during the 48-week period.
 
• CNM-Au8 was well-tolerated, and no significant safety findings were observed.
  
 

"Remyelination and neuroprotection are key unmet needs for patients with multiple sclerosis," said Professor Michael Barnett, one of the trial's key clinical advisors. "MS patients continue to progress despite current standard of care with increasing cognitive and functional deficits accumulating over time. The Phase 2 VISIONARY-MS trial results demonstrated promising efficacy of the cellular energetic nanocatalyst, CNM-Au8, across remyelination and neuroprotection domains. When these results are confirmed by a future, larger Phase 3 study, CNM-Au8 would be a remarkable advance for patients with MS as an adjunct to conventional anti-inflammatory DMTs."

As announced in February 2022, the VISIONARY-MS trial was stopped prematurely due to COVID-19 pandemic operational challenges, enrolling 73 out of the 150 planned participants. Due to limited enrollment, the threshold for significance was pre-specified at p=0.10 prior to database lock and submitted to the FDA in the statistical analysis plan (SAP). The primary analysis was conducted in the modified intent to treat (mITT) population, which censored invalid data, including data from a single site with LCLA testing execution errors (n=9), and the timed 25-foot walk data from one subject at another site with a change in mobility assist device. The ITT results incorporating the invalid data were not significant, though directionally consistent with the mITT results.

Michael Hotchkin, Clene's Chief Development Officer, said, "Despite the operational challenges presented by COVID and the primary endpoint (LS mean difference, 3.13; 95% CI: -0.08 to 6.33, p = 0.056) marginally exceeding the traditional p=0.05 statistical threshold, Clene and its MS expert advisors believe these results strongly support the hypothesis that improving brain energetic metabolism results in improved neurological function when CNM-Au8 is administered as adjunct to standard immunomodulatory disease-modifying MS therapies. For the first time in a well-treated, stable MS patient population, CNM-Au8 treatment demonstrated improved vision, based on low contrast letter acuity, and global neurological improvement, based on the mMSFC composite of low contrast vision, cognition, upper extremity function, and walking speed. Clene plans to initiate a fully powered Phase 3 study to demonstrate improved global neurological function in patients with progression independent of relapse activity (PIRA), the most urgent unmet need in MS today, following consultation with regulatory authorities."

Robert Glanzman, MD FAAN, Chief Medical Officer at Clene, added, "CNM-Au8's demonstration of neurological improvement in stable MS patients is very encouraging. The currently available MS disease-modifying therapies are very successful at limiting relapses but do not address disease progression independent of relapse activity. These data provide independently assessed clinical and quantitative physiological evidence that support the potential neuroprotective and remyelinating effects of CNM-Au8. We are very pleased to see the potential effectiveness of CNM-Au8 demonstrated in this trial."

  About CNM-Au8 ^®  
CNM-Au8 is an oral suspension of gold nanocrystals developed to restore neuronal health and function by increasing energy production and utilization. The catalytically active nanocrystals of CNM-Au8 drive critical cellular energy producing reactions that enable neuroprotection and remyelination by increasing neuronal and glial resilience to disease-relevant stressors. CNM-Au8 ^® is a federally registered trademark of Clene Nanomedicine, Inc.

  About Clene  
Clene is a clinical-stage biopharmaceutical company focused on revolutionizing the treatment of neurodegenerative disease by targeting energetic failure, an underlying cause of many neurological diseases. The company is based in Salt Lake City, Utah, with R&D and manufacturing operations in Maryland. For more information, please visit www.clene.com or follow us on Twitter , LinkedIn and Facebook.  

  Forward-Looking Statements  

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Source: Clene Inc.