Treatment for Frontotemporal Dementia Receives Orphan Drug Designation from FDA

Neflamapimod (CervoMed, Boston, MA) has been granted Orphan Drug Designation by the Food and Drug Administration (FDA) as a potential treatment for individuals with frontotemporal dementia (FTD). Neflamapimod is an orally administered small molecule brain-penetrating inhibitor of p38MAP kinase. Treatment with neflamapimod was shown to reverse synaptic dysfunction in preclinical studies. Additionally, in the phase 2a AscenD-LB clinical study (NCT04001517), treatment with neflamapimod for people with dementia with Lewy bodies (DLB) was associated with reductions in dementia severity and improvements in functional mobility compared with placebo.

According to a statement from CervoMed, the ongoing, 16-week, double-blind, placebo-controlled phase 2b clinical trial, RewinD-LB (NCT05869669) is currently investigating the effects of neflamapimod on learning skills, problem solving, and memory loss in people with DLB. The study includes 159 participants with DLB, with topline results expected before the end of the year.

“Patients diagnosed with frontotemporal dementia have no available treatment options, and this rare condition is extremely burdensome to patients and caregivers alike,” said John Alam, MD, CEO of CervoMed. “Within this year, there have been multiple scientific presentations and publications that indicate neflamapimod targets specific pathogenic mechanisms associated with FTD. We are in active discussions with clinical thought leaders regarding the design of a proof-of-principle study in FTD, while continuing to prepare to advance neflamapimod into a Phase 3 trial in DLB in mid-2025.”

The FDA grants Orphan Drug Designation to investigational therapies developed to treat rare diseases and conditions based on preclinical or clinical data establishing evidence that the drug may be effective in the specific rare disease setting. Companies that receive Orphan Drug Designation receive financial assistance and support throughout the drug development process.