New ALS drug neflamapimod chosen for UK platform study
First results from EXPERTS-ALS treatment trials expected later this year
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- Neflamapimod, Cervomed's oral therapy candidate, has been selected for testing in the EXPERTS-ALS platform study.
- The therapy targets inflammation and dysfunction in nerve cell communication, aiming to reduce nerve damage.
- Results from the first analyses in the U.K.-funded EXPERTS-ALS are expected this spring.
A U.S. biotech company’s experimental oral therapy, neflamapimod, designed to treat age-related brain disorders, has been selected for inclusion in the EXPERTS-ALS platform study, a U.K. initiative aiming to rapidly test potential treatments for amyotrophic lateral sclerosis (ALS).
Developer Cervomed announced in a company press release that its treatment candidate will be tested in EXPERTS-ALS, a multipronged study sponsored by Sheffield Teaching Hospitals NHS Foundation Trust.
The platform study is funded by the U.K.’s National Institute for Health and Care Research and several ALS-focused charities. It is designed to quickly evaluate multiple investigational therapies at once to determine whether they can reduce levels of neurofilament light chain (NfL), a marker of nerve damage.
The overall goal is to help researchers decide within a few months, and using only a small group of patients, whether to proceed to further testing.
“Neflamapimod is a promising investigational therapy for neurodegenerative diseases, and we’re excited to add it to the EXPERTS-ALS platform as we try to rapidly identify therapies that have the potential to slow disease progression in ALS,” said Chris McDermott, PhD, a neurologist at the University of Sheffield who is coleader of the initiative.
A progressive disease, ALS is marked by the gradual damage and death of motor neurons, the nerve cells that control movement. While its exact causes remain unclear, excessive inflammation in the brain and spinal cord and problems at synapses, the specialized sites where nerve cells communicate with each other, are thought to play important roles in driving the condition.
Neflamapimod aims to target both disease-related processes by binding the alpha isoform of p38 MAP kinase, a protein involved in inflammation and synapse dysfunction.
Neflamapimod has shown promise for treating dementia
The therapy has shown a favorable safety profile and early signs of effectiveness in trials involving people with dementia with Lewy bodies, another neurological disorder marked by synaptic abnormalities. A Phase 3 trial in that indication is expected to begin later this year.
“The scientific rationale for neflamapimod’s mechanism of action, combined with its positive clinical data in dementia with Lewy bodies … make it a strong candidate for our platform,” McDermott said. “We look forward to working with Cervomed and helping to further the development of neflamapimod for the ALS community.”
John Alam, MD, CEO of Cervomed, noted that ALS “places an enormous burden on patients and their families.”
“We are proud to collaborate with EXPERTS-ALS to evaluate whether neflamapimod can offer meaningful hope to this community,” Alam said.
In the neflamapimod arm of EXPERTS-ALS, about 35 adults with ALS will be treated for 18 to 24 weeks, or about four to six months. The main goal is to determine whether the therapy lowers NfL levels, which could indicate reduced nerve cell damage and a potential slowing of disease progression.
Researchers will also evaluate clinical measures of disease activity and survival as secondary outcomes. If initial results are encouraging, the arm may be expanded to include up to 80 adults with ALS.
The scientific rationale for neflamapimod’s mechanism of action, combined with its positive clinical data in dementia with Lewy bodies … make it a strong candidate for our platform.
The EXPERTS-ALS platform is right now testing three therapies, all of which are already approved for other indications. One is the antidiabetes drug metformin, while the others are the calcium channel blocker nifedipine and the Parkinson’s treatment ropinirole. The first analysis from the platform is expected this spring.
The study is being conducted at 11 centers across the U.K., with plans to expand to 17 sites. New treatments are evaluated and added to the platform every three to six months, with the next available slot anticipated for later this year.
“Inclusion in the EXPERTS-ALS platform serves to further validate neflamapimod’s potential in neurodegenerative diseases and allows for rapid evaluation of neflamapimod in ALS within a clinical trial platform purposefully built to fast-track promising therapies for that disease,” Alam said.
