Intravenous Efzofitimod in Patients With Pulmonary Sarcoidosis

This study will enroll adults with histologically confirmed pulmonary sarcoidosis receiving stable treatment with oral corticosteroid (OCS), with or without immunosuppressant therapy.

• Study Sponsor: aTyr Pharma, Inc.
  
  
• Start Date: September 2022
  
  
• Estimated Primary Completion Date: December 2024
  
  
• Phase 3, randomized, double-blind, placebo-controlled, trial evaluating the efficacy and safety of comparing the efficacy and safety of intravenous (IV) efzofitimod 3 mg/kg and 5 mg/kg versus placebo after 48 weeks of treatment.

Primary Outcome Measure:

• Change from baseline in mean daily oral corticosteroid (OCS) dose post-taper [Time Frame: Baseline to Week 48]

Inclusion Criteria:

• Documented history of pulmonary sarcoidosis for at least 6 months, supported by the following evidence: documented histologically proven diagnosis of sarcoidosis by tissue biopsy and documented evidence of parenchymal lung involvement by historical radiological evidence
• Evidence of symptomatic pulmonary sarcoidosis, as demonstrated by the following criteria: Modified Medical Research Council (MRC) dyspnea scale grade of at least 1 and KSQ-Lung score ≤70
• Patients must be receiving treatment with OCS of ≥ 3 months at Day 1 with a starting dose between ≥ 7.5 and ≤ 25 mg/day ≥ 4 weeks prior to Day 1
• Body weight ≥ 40 kg and < 160 kg

Exclusion Criteria:

• Treatment with > 1 immunosuppressant therapy
• Treatment with biological immunomodulators, such as tumor necrosis factor-alpha (TNF-α) inhibitors or antifibrotics or interleukin inhibitors
• Likelihood of significant pulmonary fibrosis as shown by any 1 or more of the following: High resolution CT fibrosis > 20% within the last 12 months; FVC percent predicted (FVCPP) < 50% and KSQ-Lung score < 30
• In the opinion of the investigator, clinically significant pulmonary hypertension
• Patients with active cardiac, neuro, or renal sarcoidosis requiring organ-specific therapy in the past 2 years
• Patients with cutaneous or ocular sarcoidosis, which in the opinion of the Investigator, are at risk for exacerbation, necessitating OCS rescue or other systemic therapy
• History of Addisonian symptoms that precluded previous OCS taper attempts
• Is an active, heavy smoker of tobacco/nicotine-containing products
• History of anti-synthetase syndrome or Jo-1 positive at Screening
• Patients with active tuberculosis or those currently undergoing treatment for tuberculosis