Prothena Corporation, a clinical stage biotechnology company focused on the discovery and development of novel therapies in the neuroscience and orphan categories, announced discontinuation of development of NEOD001, an investigational antibody that was being evaluated for the treatment of amyloid light-chain (AL) amyloidosis.
The decision was based on results from the Phase 2b PRONTO study and a futility analysis of the Phase 3 VITAL study. Based on the results from the Phase 2b PRONTO study, which did not meet its primary or secondary endpoints, the Company asked the independent data monitoring committee (DMC) of the Phase 3 VITAL study to review a futility analysis of the ongoing VITAL study. The DMC recommended discontinuation of the VITAL study for futility.
The Company therefore decided to discontinue all development of NEOD001, including the VITAL study as well as the open label extension studies.
“We are deeply disappointed by this outcome, particularly for patients suffering from this devastating disease,” said Gene Kinney, PhD, President and Chief Executive Officer of Prothena. “We are surprised by the results from these two placebo-controlled studies and will continue to analyze the resulting data to share insights with our collaborators in the scientific, medical and advocacy communities. We thank all of the patients, their families, caregivers, investigators, study staff and our employees. Their participation in and commitment to these studies are indispensable to advancing our shared goal of improving the lives of patients with amyloidosis.”
Prothena Corporation is a global, clinical biotechnology company focused on the discovery and development of novel therapies in the neuroscience and orphan categories. Fueled by its deep scientific understanding built over decades of research in protein misfolding, Prothena seeks to fundamentally change the course of progressive, life-threatening diseases associated with this biology. Prothena’s pipeline of antibody therapeutic candidates targets a number of indications including Parkinson’s disease and other related synucleinopathies (PRX002/RG7935) and ATTR amyloidosis (PRX004).