Clene to submit new CNM-Au8 data to FDA ahead of meeting
Added trial analyses aimed at addressing FDA questions
Clene said it planned to give the U.S. Food and Drug Administration (FDA) new data on CNM-Au8 for the treatment of amyotrophic lateral sclerosis (ALS) ahead of a meeting on a path to accelerated approval of the therapy.
The company said it would submit a briefing book containing new analyses of data from clinical trials, designed to address FDA comments from a previous meeting in which regulators indicated that available data at the time did not meet the criteria for accelerated approval.
Clene will meet again with regulators in the third quarter and seek feedback on its plan to submit a regulatory application under the FDA’s accelerated approval pathway, according to a company press release.
That program allows the FDA to conditionally approve a therapy that fills an unmet need for a serious medical condition based on a surrogate clinical trial endpoint, or a biomarker that demonstrates the treatment is likely to offer clinical benefit.
The company would later need to show direct clinical trial evidence that the treatment does indeed improve clinical outcomes — that is, that it slows disease progression or extends survival — before it can be converted to full approval.
NfL levels at issue
Clene expects that an accelerated approval application would be based on clinical trial data demonstrating that CNM-Au8 significantly lowers levels of neurofilament light chain (NfL), an established biomarker of nerve cell damage whose levels correlate with disease progression in a number of neurodegenerative conditions.
The briefing book contains new analyses from completed clinical studies, Clene said. The company aims to address the FDA’s request to see more information about how CNM-Au8’s mechanism relates to NfL reductions, and in turn, how NfL reductions are associated with better clinical outcomes in ALS.
It will show additional NfL data from the trials, more robust analyses of the survival and functional benefits from CNM-Au8 that have been observed so far, and additional evidence supporting CNM-Au8’s mechanism, according to the company.
Clene said it believes the new analyses “further demonstrate the potential for CNM-Au8 as a treatment in people living with ALS, consistent with the accelerated approval standards.”
The company will publicly provide insights into these new analyses later this year. It will also announce feedback from the FDA after the meeting.
CNM-Au8 is an oral liquid suspension of gold nanoparticles that’s designed to support the energetic needs of nerve cells and protect them against forms of cellular damage that contribute to neurodegeneration.
Findings across CNM-Au8’s clinical trials and expanded access programs have demonstrated that CNM-Au8 can slow disease progression, delay clinical worsening, and extend survival for ALS patients.
Most recently, analyses from the CNM-Au8 arm of the HEALEY ALS Platform Trial (NCT04297683) showed that the experimental therapy lowered the risk of death by nearly 60% relative to external placebo-treated patients after up to 3.5 years of treatment. It also led to gradual declines in NfL over 1.5 years.
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