The FDA has granted orphan drug designation to neflamapimod, an investigational oral small-molecule therapeutic developed for the treatment of frontotemporal dementia, according to a press release from drug manufacturer CervoMed. This decision was supported by findings from the AscenD-LB phase 2a clinical trial, where neflamapimod significantly reduced dementia severity (measured by Clinical Dementia Rating Sum-of-Boxes) and improved functional mobility (assessed via the Timed Up and Go Test) compared to placebo.

CervoMed reported that the treatment showed pronounced benefits in patients with early-stage dementia with Lewy bodies (DLB) compared to the broader trial population. Additionally, neflamapimod demonstrated a favorable safety profile and was well-tolerated in two early-phase clinical trials involving 300 participants with this rare neurodegenerative condition.

Neflamapimod is currently being evaluated in the ongoing RewinD-LB phase 2b clinical trial. This study is a randomized, double-blind, placebo-controlled trial assessing the efficacy of 40 mg oral neflamapimod, administered three times daily, in 159 patients with early-stage DLB over a 16-week period. Topline results are anticipated by the end of the year.

“We are honored to receive orphan drug designation, as it validates the scientific foundation and potential of neflamapimod to address this devastating disease,” said CervoMed CEO John Alam, MD. “There are currently no treatment options for patients with frontotemporal dementia, a condition that imposes a significant burden on patients and caregivers. We are actively consulting with clinical experts to design a proof-of-concept study while preparing for a phase 3 trial, which we aim to initiate in mid-2025.”