Safety and efficacy of lenabasum in a phase 2 randomized, placebo-controlled trial in adults with systemic sclerosis
OBJECTIVE: Assess safety and efficacy of lenabasum in diffuse cutaneous systemic sclerosis (dcSSc).
METHOD(S): A double-blind, randomized, placebo-controlled Phase 2 study was conducted at nine SSc clinics in the USA. Adults with dcSSc <= 6 years duration on stable standard-of-care treatment received lenabasum (N = 27) or placebo (N = 15). Lenabasum doses were 5 mg once daily (QD), 20 mg QD, or 20 mg twice daily (BID) for 4 weeks, then 20 mg BID for 8 weeks. Safety and efficacy assessments were done at weeks 4, 8, 12, and 16.
RESULT(S): Adverse events occurred in 63% of the lenabasum group and 60% of the placebo group, with no serious adverse events related to lenabasum. Lenabasum treatment compared to placebo was associated with greater improvement in American College of Rheumatology Combined Response Index in diffuse cutaneous Systemic Sclerosis (CRISS) score and other efficacy outcomes that assessed overall disease, skin involvement, and patient-reported function. CRISS score increased in the lenabasum group during the study, reaching 0.33 versus 0.00 in the placebo group at Week 16 (p = 0.07, 2-sided mixed model repeated measures). Gene expression in inflammation and fibrosis pathways was reduced and inflammation and fibrosis improved by histological evaluation of skin biopsies from the lenabasum group compared to the placebo group (all p <= 0.05).
CONCLUSION(S): Despite a short trial duration in a small number of patients, lenabasum improved efficacy outcomes and underlying disease pathology with a favorable safety profile in this Phase 2 study in dcSSc.
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