Lanadelumab Provides Sustained PK/PD Following Long-Term Treatment in Patients with Hereditary Angioedema (HAE)

Abstract

RATIONALE: Efficacy of lanadelumab in preventing HAE attacks was demonstrated in the phase 3 HELP Study (NCT02586805) and ongoing OLE (NCT02741596). Findings from an exploratory analysis evaluating efficacy by baseline HAE attack rate are presented, reflecting cumulative results through 31August18.. METHODS: Patients with ≥1 attack/4 weeks at baseline who continued from the HELP Study (rollovers) received a single 300mg lanadelumab dose on Day 0, then 300mg every 2 weeks (Q2W) after their first attack (regular dosing stage). Patients who were not HELP Study participants (nonrollovers) with ≥1 historical attack/12 weeks received 300mg Q2W from Day 0. Monthly HAE attack rate (regular dosing stage for rollovers; from Day 0 for nonrollovers) was evaluated by baseline rates of <1, 1 to <2, 2 to <3, or ≥3 attacks/month. RESULTS: In 212 patients (rollover, n5109; nonrollover, n5103), baseline HAE attack rate was <1 for 11.8% (0 rollovers, 25 nonrollovers); 1 to < 2 for 34.9% (35 rollovers, 39 nonrollovers); 2 to <3 for 14.2% (19 rollovers, 11 nonrollovers); and ≥3 for 39.2% (55 rollovers, 28 nonrollovers) of patients. With lanadelumab treatment, median (range) reductions in HAE attack rate from baseline were-92.2% (-100.0%, 852.8%) for baseline attack rate <1;-100.0% (-100.0%,-32.4%) for baseline attack rate 1 to <2;-98.1% (-100.0%,-21.7%) for baseline attack rate 2 to <3; and-96.5% (-100.0%,-32.4%) for baseline attack rate ≥3. CONCLUSIONS: Treatment with lanadelumab produced marked reductions in monthly HAE attack rates regardless of baseline attack frequency, consistent with the results of the pivotal study.