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The National Institutes of Health (NIH) has awarded about $45.1 million toward an expanded access program (EAP) of <a href="https://alsnewstoday.com/cnm-au8/">CNM-Au8</a>, an oral therapy candidate for <a href="https://alsnewstoday.com/what-is-als/">amyotrophic lateral sclerosis</a> (ALS).
The EAP, commonly referred to as compassionate use, will make CNM-Au8 available to U.S. patients who aren&#8217;t eligible for clinical trials that are testing the experimental therapy. It will join two other CNM-Au8 EAP programs that have enrolled more than 200 participants.
The programs are expected to provide additional clinical data to support the safety and effectiveness of CNM-Au8, which will be reviewed by regulatory agencies ahead of an approval.
“This EAP study will give ALS patients who don’t meet the criteria to enroll in a clinical trial an opportunity to try CNM-Au8 as a novel investigational therapy,” Jinsy A. Andrews, MD, a professor of neurology at Columbia University, said in a <a href="https://www.globenewswire.com/news-release/2023/10/05/2755532/0/en/National-Institutes-of-Health-Awards-45-1-Million-NINDS-Grant-for-CNM-Au8-Study-in-ALS.html">press release from Clene Nanomedicine</a>, the therapy’s developer.
The program&#8217;s four-year grant comes from the NIH&#8217;s National Institute of Neurological Disorders and Stroke (NINDS), and is supported by the Accelerating Access to Critical Therapies (ACT) for ALS Act, which was signed into law in 2021. The grant was awarded to <a href="https://clene.com/">Clene</a> in collaboration with Columbia University and <a href="https://www.synapticure.com/">Synapticure</a>.
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<h3>Making CNM-Au8 accessible remotely</h3>
The EAP will be led by Andrews, Eric Anderson, MD, vice president of clinical operations and care delivery at Synapticure, and Benjamin Greenberg, MD, head of medical at Clene.
It will enroll patients in all 50 states, including those in remote and rural areas, via Synapticure’s telemedicine neurology clinic and several nationwide clinics.
The program will monitor CNM-Au8&#8217;s safety, its impact on survival and disease progression, and changes in key disease-related biomarkers.
“Programs like this help to advance research and much-needed innovation in ALS,” Andrews said. &#8220;We are truly excited to be a part of this grant with Clene and Columbia and to support people living with ALS by providing access to treatments that could meaningfully impact the course of their disease.&#8221;
She said Synapticure&#8217;s virtual platform will aid in reaching people in remote areas who haven&#8217;t been able to access investigational treatments like CNM-Au8. “We are grateful to NINDS for recognizing how a virtual platform like Synapticure can provide expanded access programs in a remote capacity,&#8221; Andrews said.
<h3>What is CNM-Au8?</h3>
CNM-Au8 is an oral liquid suspension of gold nanoparticles that&#8217;s believed to slow ALS by supporting the energetic needs of nerve cells and other cells of the nervous system. It can also protect them from oxidative stress, a type of cellular stress implicated in the neurodegenerative disease.
It&#8217;s being investigated in the Phase 2 <a href="https://clinicaltrials.gov/ct2/show/NCT04098406">RESCUE-ALS trial (NCT04098406)</a> and its <a href="https://clinicaltrials.gov/ct2/show/NCT05299658">open-label extension (NCT05299658)</a>, as well as in an arm of the <a href="https://clinicaltrials.gov/ct2/show/NCT04297683">HEALEY ALS platform trial (NCT04297683)</a>.
Data from the studies have demonstrated CNM-Au8 is safe and can <a href="https://alsnewstoday.com/news/cnm-au8-seen-to-extend-survival-by-more-than-1-5-years-in-als-trial/">slow disease progression</a> and <a href="https://alsnewstoday.com/news/long-term-survival-benefit-seen-cnm-au8-healey-als-analyses-trial/">extend survival</a> in patients.
“Clene has demonstrated evidence of consistent safety and improved survival for CNM-Au8 across a broad ALS population in two independent Phase 2 trials and an ongoing EAP with up to 3.8 years of follow-up,” Greenberg said. “This new EAP provides access to CNM-Au8 for more people living with ALS and enables the collection of survival, safety, and biomarker data in a population not studied in clinical trials. These data can help provide confirmatory support for the existing trial data Clene has gathered in its clinical trials.&#8221;

Tag: expanded access program (EAP)

New EAP will make CNM-Au8 available outside of trials