Clene expanding CNM-Au8 expanded access program in US
Compassionate use program now to enroll 80% more patients than first planned
Clene Nanomedicine has nearly doubled the enrollment cap for a soon-to-launch expanded access program (EAP) to allow more patients to receive CNM-Au8 — its investigational treatment for amyotrophic lateral sclerosis (ALS) — outside of clinical trials.
Also known as a compassionate use program, the EAP will provide a regulated pathway for as many as 180 patients across the U.S. to gain access to the treatment when no other options are available. This will mean that patients living in rural communities, who typically have more difficulties in accessing research centers where clinical trials are conducted, may now be able to access the drug candidate.
The planned recruitment of 180 patients represents an 80% expansion of the previous enrollment target for the EAP. The expanded access program is slated to launch early this month.
“The Clene team recognized that there was great need in the ALS community for more access to promising treatments,” Austin Rynders, Clene’s vice president of clinical operations, said in company press release, adding that multiple departments at Clene “put forth an extraordinary effort to expand the size of the … EAP without increasing the size of the budget.”
Patient visits beginning soon in CNM-Au8 expanded access program
With the first patient visits slated for early June, the program will run in collaboration with Columbia University and Synapticure as part of the Accelerating Access to Critical Therapies for ALS Act. It will be supported by a four-year National Institutes of Health (NIH) grant totaling more than $45 million.
“We are grateful for the NIH’s recognition to fund this important initiative and are pleased to provide this EAP for the ALS community,” said Rob Etherington, who is Clene’s CEO.
To date, more than 250 patients have gained access to CNM-Au8 under the company’s first two EAP initiatives, launched in 2019 at Massachusetts General Hospital (NCT04081714) and in 2021 at 16 sites of the Northeast ALS Consortium (NCT05281484).
Pooled data from those patients, who had advanced disease and were treated with CNM-Au8 at a daily oral dose of 30 mg, showed that the therapy was well tolerated and prolonged survival compared with what would be seen without treatment.
It’s important to note that this is not just a standard EAP. … A lot of time, careful thought, and effort have resulted in a study that will yield important insights into the real-world impact of CNM-Au8 treatment on the disease course of ALS.
The third expanded access program (NCT06408727) aims to collect real-world data on safety, and to determine how CNM-Au8, given at a daily oral dose of 30 mg, affects survival and disease progression in patients with a diagnosis of ALS. The study will run for up to 148 weeks, or nearly three years, and biomarker data also will be collected.
“It’s important to note that this is not just a standard EAP,” said principal investigator Jinsy A. Andrews, MD, of Columbia University, a principal investigator on the grant.
“A lot of time, careful thought, and effort have resulted in a study that will yield important insights into the real-world impact of CNM-Au8 treatment on the disease course of ALS,” Andrews said.
CNM-Au8 is a drinkable solution of gold nanocrystals — tiny, orderly arranged lattices of gold ions — designed to restore the levels of energy in nerve cells and scavenge harmful molecules that cause damaging oxidative stress in neurodegenerative diseases like ALS.
Clinical trials have shown benefit of CNM-Au8 in ALS patients
Two earlier clinical studies, HEALEY (NCT04414345) and RESCUE-ALS (NCT04098406), tested CNM-Au8 in ALS patients. These trials showed that the therapy delayed functional decline and reduced the risk of death when taken for up to 2.5 years. There were no serious side effects with the treatment’s use.
“CNM-Au8 treatment has been associated with lowered risk of death and delayed clinical worsening — all while being very well-tolerated, in both of our independent Phase 2 clinical studies,” Etherington said.
“Data from EAP programs, which usually include a broader ALS population than clinical trials,” have been consistent with those clinical studies, which “can supplement the safety and other meaningful data gathered from clinical studies,” Etherington said.
Patients can participate in the third EAP, called ACT-EAP, either via a local research institution or through Synapticure’s virtual clinical site. Synapticure is a company that offers personalized virtual care to people living with neurodegenerative diseases, as well as support to their caregivers.
The expansion of ACT-EAP means that patients living in remote and rural areas will be able to participate via only virtual visits. Those in local research institutions also may choose to have in-person or virtual visits.
“We are pleased to be the first-ever fully virtual clinical site to participate in an EAP — or any clinical study for that matter,” said Peter Wallach, chief financial officer and co-founder of Synapticure. “With a nationwide presence, we enable people living with ALS access to investigational medicines like CNM-Au8 who have not previously had the option.”
About the Author
