Efzofitimod for the Treatment of Pulmonary Sarcoidosis.

"Financial/Nonfinancial Disclosures The authors have reported to CHEST the following: D. A. C. has received grants from 10.13039/100014468Mallinckrodt Pharmaceuticals, Boehringer Ingelheim, the 10.13039/100015131Foundation for Sarcoidosis Research (FSR), and the Ann Theodore Foundation; serves as a consultant for Roivant Sciences and Boehringer Ingelheim; serves as a member of the adjudication committee for Pliant Therapeutics; and serves as president of the World Association for Sarcoidosis and Other Granulomatous Disorders. S. A. received conference travel support from and is an advisory board member for aTyr Pharma, Inc. C. C. W. H. has received grant funding from Mallinckrodt Pharmaceuticals. L. A. M. has received grants from the FSR, Mallinckrodt Pharmaceuticals, and the University of Cincinnati (Mallinckrodt Pharmaceuticals Foundation Grant) and serves on the scientific advisory board for FSR and the global advisory board for aTyr Pharma, Inc. O. N. O. has received travel support from aTyr Pharma, Inc., was provided equipment for conducting the current study, and serves on the scientific advisory board for FSR. P. H. S. S. has received grants the FSR, aTyr Pharma, Inc., and Novartis. S. S. and G. W. are employed by and own stock in aTyr Pharma, Inc. N. K. has received payments from aTyr Pharma, Inc., for consultancy on the statistical analysis and interpretation of data, payments from Savara Pharmaceuticals for consultancy on the design and analysis of clinical trials in respiratory diseases, and previously held stock in Roche. R. B. has received grants from 10.13039/100004326Bayer, 10.13039/100004328Genentech, Mallinckrodt Pharmaceuticals, the FSR, and Actelion; consulting fees from Mallinckrodt Pharmaceuticals, Meitheal Pharmaceuticals, Actelion, and Kinevant; and payment for speaker bureaus from Mallinckrodt Pharmaceuticals, United Therapeutics, and Boehringer Ingelheim. None declared (J. B., W. E. J., L. T. M., and N. J. S.)."

"Funding/Support Supported by 10.13039/100014933aTyr Pharma L. A. M. is supported by the 10.13039/100000002National Institutes of Health [Grants R01HL140357, R01HL142049, and R01HL136681]. P. H. S. S. is supported by the 10.13039/100000002National Institutes of Health [Grants R13HL142300and R01HL131745] and the American Thoracic Society Foundation."

"This was a randomized, double-blind, placebo-controlled multiple ascending dose study with three sequential dose cohorts with a 2:1 randomization (efzofitimod to placebo) in each cohort; the planned study size was 36 patients (ClinicalTrials.gov Identifier: NCT03824392). Patients receiving placebo from each of the three cohorts were pooled when comparing safety and efficacy between placebo and efzofitimod. The treatment period consisted of six IV administrations of study drug (efzofitimod or placebo) once every 4 weeks for a total of 20 weeks (at day 1 and weeks 4, 8, 12, 16, and 20), with the final study assessments conducted at week 24."