Apilimod dimesylate in C9orf72 amyotrophic lateral sclerosis: a randomized phase 2a clinical trial

This trial was funded by OrphAI Therapeutics. Their role included study concept and design, funding of the participating centres, data analysis and writing of the manuscript. We thank the DSMB members, Gary Cutter, UAB School of Public Health, Colin Quinn, University of Pennsylvania, Christina Fournier, Emory University and Michael D Weiss, University of Washington. We thank Karen Jansen-West, Syneos and WWCT for biomarker and pharmacokinetics analyses. We thank Amy Perniciaro and IQVIA for clinical operations support. We thank Syneos for validation of the sGPNMB assay and analysis of clinical samples. We thank Dana Dunn for regulatory filings. We thank Dr Murat Gunel, Dr Brigette Roberts and the NEALS Consortium for support of the study. We thank Dr Jinsy Andrews, Columbia University Medical Center, for review of the manuscript. Medical writing assistance was provided by Aroga Biosciences, Inc. (San Diego, CA, USA), funded by OrphAI Therapeutics.DAS:All data requests should be submitted to Keith Fandrick (kfandrick@orphai-therapeutics.com) for consideration. Access to available anonymized data might be granted following review.