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Clene, and its wholly owned subsidiary Clene Nanomedicine, have launched an Extended Access Program (EAP) in the United States to allow certain people with amyotrophic lateral sclerosis (ALS) to access its investigational oral therapy CNM-Au8 .
Called CNMAu8.EAP02, the program will focus on patients who are not eligible to participate in the HEALEY ALS Platform Trial (NCT04297683) – the first multi-regimen study in ALS – Clene announced in a statement. Press.
EAPs are intended to make specific investigational therapies – those whose benefits are considered to outweigh the potential risks – available outside of clinical trials for people with serious or life-threatening conditions with little or no adequate treatment.
The HEALY Platform Trial is currently testing CNM-Au8 and four other potential ALS therapies at the same time to accelerate the development of those deemed most promising, while reducing costs.
Patients for the EAP will be recruited from three of HEALEY’s participating sites, with the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital leading the trial. The first two sites to be included are Holy Cross Hospital in Fort Lauderdale, Florida, and Hospital for Special Care in New Britain, Connecticut.
“We are honored to collaborate with the Healey Center for ALS in this critically important effort to provide access to CNM-Au8 to people with ALS who may benefit from it,” said Robert Glanzman, MD, Chief Medical Officer by Clene.
“The Healey Center is an exceptional partner and we proudly share its goal of developing life-saving therapies for the treatment of ALS,” added Glanzman.
Notably, CNMAu8.EAP02 represents Clene’s second EAP for ALS, the first, named CNMAu8.EAP01 (NCT04081714), currently providing 40 patients with early access to CNM-Au8. Launched in 2019, also in partnership with the Healey Center for ALS, the initial program includes patients treated for up to two years.
Problems in the mitochondria, the powerhouses of the cell, as well as oxidative stress, have been linked to several neurodegenerative diseases, including ALS. Oxidative stress is an imbalance between the production of harmful free radicals – which occurs primarily in the mitochondria – and the ability of cells to detoxify them.
CNM-Au8 is a stable suspension of pure gold nanocrystals designed to increase energy production, while protecting cells against oxidative stress.
Importantly, therapy can cross the blood-brain barrier, the highly selective membrane that blocks circulating microbes and potentially harmful molecules from reaching the central nervous system, or CNS, made up of the brain and spinal cord. The blood brain barrier is a common barrier for treatments targeting the CNS, preventing their usefulness.
As such, CNM-Au8 is expected to prevent nerve cell death and slow disease progression in patients with ALS.
Previous preclinical studies have shown that CNM-Au8 improved nerve cell survival and motor neuron function in rodent models with ALS and other neurodegenerative diseases, such as Parkinson’s disease and multiple sclerosis.
The safety, pharmacokinetics, pharmacodynamics and efficacy of therapy were evaluated in 45 adults with early-onset ALS who enrolled in the recently completed RESCUE-ALS Phase 2 trial in Australia (NCT04098406). Pharmacokinetics refers to the movement of therapy in, through, and out of the body, while pharmacodynamics refers to how it affects the body.
The first non-blind results are expected in the coming months. Intermediate blinded results suggest that therapy may, to some extent, slow the progression of ALS from its natural course, but it is still unclear whether the observed benefits come from patients receiving CNM-Au8 or those receiving a placebo.
In addition, a US-based Phase 2 trial called REPAIR-ALS (NCT03843710) is expected to assess the metabolic effects of CNM-Au8 on the CNS of up to 24 patients with ALS. The study may not be recruiting yet.
Within HEALEY, the CNM-Au8 Phase 2/3 pivotal trial arm (NCT04414345), which reached 50% of its target recruitment in March, will include up to 160 adults with sporadic or familial ALS. Participants will be randomly assigned to drink two 30 or 60 mg bottles of CNM-Au8 (120 patients) or a placebo (40 patients), per day for 24 weeks, or approximately six months.
Common goals of HEALEY include changes in disease progression – as assessed with the revised ALS Functional Rating Scale – lung function, muscle strength and survival, as well as safety measures.
Recruitment for CNM-Au8 and other trial arms is underway at more than 50 sites across the United States; More information can be found here.
The first results of the CNM-Au8 arm are expected by mid-2022. If they are positive, Clene plans to use them to support regulatory submissions to seek treatment approval for ALS.
“As CNM-Au8 progresses through testing the recording platform with the goal of becoming available to all patients with ALS, expanded access programs allow more people with ALS to benefit today of the potential of our first-class neuroreparative energy nanotherapeutics, ”said Rob Etherington, President and CEO of Clene.
“Clene is committed to supporting communities living with devastating neurodegenerative diseases, and we are doing all we can every day to reach and treat more patients,” Etherington added.
In August, the company announced two leases that will more than quadruple its manufacturing capacity of CNM-Au8.
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