CervoMed’s ex-Vertex drug has failed to reduce the severity of patients’ dementia in a phase 2 trial, halting plans for a late-stage study.
The drug in question is neflamapimod, a p38 MAP kinase alpha enzyme inhibitor that EIP Pharma picked up from Vertex in 2014. Vertex had developed the candidate as a treatment for rheumatoid arthritis, taking it as far as a midphase trial in the setting.
EIP spied another application, starting a phase 2a trial in Alzheimer’s disease in 2015. An aborted attempt to go after Huntington’s disease followed, before EIP eventually settled on dementia with Lewy bodies (DLB) as its lead indication. The results from an exploratory midphase clinical trial were strong enough to persuade the U.S. National Institute on Aging to chip in $21 million toward the costs of running a phase 2b.
When EIP merged with Diffusion Pharmaceuticals last year to create CervoMed, the phase 2 trial of neflamapimod in DLB was the combined company’s top priority.
But the results are now in, and they have failed to live up to expectations. Specifically, the study didn’t meet the statistical significance thresholds for the primary endpoint of a change in the Clinical Dementia Rating Sum of Boxes. The key secondary endpoints—which measured mobility, cognitive function and clinicians’ assessment of any improvement—were all duds as well.
CervoMed pointed to a failure to reach target levels of the drug in the blood as one potential reason for the poor results.
“Obviously, we are disappointed with these results, particularly given our prior clinical experience with neflamapimod in patients with early-stage DLB and we are investigating the reasons for the lower-than-expected plasma drug concentrations,” CervoMed CEO John Alam, M.D., said in the release.
“We continue to believe neflamapimod may have potential as a treatment for DLB, and we will thoroughly analyze the clinical and pharmacokinetic data from the trial to better understand its outcome and potential future development paths for the drug,” Alam added. “This includes data expected to be available in the first half of 2025 from the first 16 weeks of the open label extension trial which we believe may be valuable to our investigation.”
The Boston-based biotech had been gearing up for a phase 3 trial of neflamapimod in early-stage DLB, but these plans are now on ice until the full analysis of the phase 2 trial has been completed.