CervoMed’s Experimental Drug Selected for UK Platform Trial

Rare Daily Staff

CervoMed said its lead experimental candidate, neflamapimod, has been selected for inclusion in the United Kingdom’s EXPERTS-ALS platform, a U.K.-backed initiative designed to identify and accelerate promising treatments for the rare neurodegenerative disease amyotrophic lateral sclerosis.

Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease characterized by the progressive loss of motor neurons that control movement, speech, and breathing. There are currently no approved therapies that halt or reverse disease progression.

CervoMed’s oral small-molecule therapy will be tested for the first time in people with ALS through a randomized, multicenter, open-label trial sponsored by Sheffield Teaching Hospitals NHS Foundation Trust and funded by the U.K.’s National Institute for Health and Care Research, in partnership with leading motor neuron disease charities. The trial’s first participant is expected to be dosed by the end of 2026.

Neflamapimod targets p38, an enzyme linked to neuroinflammation and neurodegeneration. The oral, brain-penetrant drug has previously shown clinical activity in other brain disorders, including dementia with Lewy bodies, and has demonstrated favorable safety and biomarker results in multiple trials.

Under the EXPERTS-ALS platform, about 35 ALS patients will initially receive neflamapimod for 18 to 24 weeks, with the potential to expand to as many as 80 participants. Investigators will assess the drug’s effect on neurofilament light chain levels — a biomarker of neuroaxonal damage correlated with disease progression and survival — along with clinical and survival measures.

James Shorter, a biochemistry and biophysics professor at the University of Pennsylvania, noted that persistent p38 activation is “a key driver of ALS pathology” and that neflamapimod’s ability to cross the blood-brain barrier “provides a compelling rationale for its inclusion” in the trial.

“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,” said John Alam, CEO of CervoMed.

CervoMed is also advancing plans to begin a phase 3 global trial of neflamapimod in dementia with Lewy bodies in the second half of this year.