Efficacy, Safety, and Pharmacokinetics of Golimumab in Children with Moderately-To-Severely Active Ulcerative Colitis: Results from the PURSUIT 2 Study

BACKGROUND: Golimumab is an anti-TNFα biologic agent that has been approved for adults with moderately-to-severely active ulcerative colitis (UC). We investigated the efficacy, safety, and pharmacokinetics (PK) of golimumab in biologic-naïve children with moderately-to-severely active UC. METHODS: The prospective, multicenter, open-label PURSUIT 2 study enrolled biologic-naïve children (2 to <18 years) with moderately-to-severely active UC (Mayo score 6-12; endoscopic subscore ≥2) despite conventional treatment. During the 6-week induction phase, patients received subcutaneous (SC) golimumab dosed by weight (<45 kg: 120/60 mg/m2; ≥45 kg: 200/100 mg) at weeks 0/2. Week 6 clinical responders (Mayo score decrease from baseline ≥30% and ≥3 points, with either a decrease in the rectal bleeding subscore of ≥1 or a rectal bleeding subscore of 0/1) continued golimumab dose q4w during the 48-week maintenance phase. The primary endpoint was clinical remission (Mayo score ≤2 points with no individual subscore >1) at week 6. Efficacy and PK data are presented alongside a reference adult UC population who received golimumab SC 200/100 mg at weeks 0/2 and 100 mg q4w thereafter. RESULTS: Of the 69 patients (mean age, 13.4 ± 3.3 years) enrolled, 31.9% (22/69) were in clinical remission at week 6, and of those, 54.5% (12/22) remained in clinical remission at week 54. At week 54, 31.7% (13/41) of week 6 clinical responders achieved clinical remission. Additionally, 33.3% achieved clinical remission by the Pediatric Ulcerative Colitis Activity Index (PUCAI), 56.5% clinical response by Mayo, 40.6% endoscopic improvement, and 7.2% endoscopic remission at week 6. Of those who were clinical responders at week 6, 34.1% achieved clinical remission by PUCAI, 31.7% corticosteroid-free clinical remission by Mayo, 36.6% endoscopic improvement, and 9.8% endoscopic remission at week 54. Serum golimumab concentrations through week 6 were comparable to the reference adult UC population. Through week 54, 40.6% reported serious adverse events and 13.0% serious infections. No new safety concerns were identified. CONCLUSIONS: Overall, the results of the PURSUIT 2 study support golimumab treatment for children with moderately-to-severely active UC. ClinicalTrials.gov ID: NCT03596645.