Long-term Safety and Efficacy of IncobotulinumtoxinA for the Treatment of Blepharospasm in Botulinum Toxin-naïve Subjects: Results of a Phase III Study (S28.005)

Objective: To assess the safety and efficacy of incobotulinumtoxinA for benign essential blepharospasm (BEB) in toxin-naive subjects. Background: IncobotulinumtoxinA is efficacious for BEB. This was the first, randomized, Phase III study (NCT01896895) in toxin-naive subjects. Here we present data from the complete study. Design/Methods: Subjects (18–80 years) with bilateral BEB, Jankovic Rating Scale (JRS) severity subscore ≥2, and no BEB treatment with any botulinum neurotoxin (BoNT) serotype within past ≥12 months, were enrolled. In the main period (MP), subjects were randomized (1:1:1) in a double-blind manner to single intramuscular injections of incobotulinumtoxinA 25U (12.5U/eye), 50U (25U/eye) or placebo, with an observation period (OP) of 6–20 weeks. Subjects with a need for re-injection (JRS severity subscore ≥2 at final MP visit) were eligible for the open-label extension period (EP): a single dose of incobotulinumtoxinA ≤70U (≤35U/eye) with a 6–20-week OP. Mean change from baseline in JRS severity subscore and safety were assessed. Results: Overall, 61 subjects were randomized (mean 55.0 years; 59.0% female); 55 completed the MP and 39 entered and completed the EP. At MP Week 6, JRS severity subscore significantly improved from baseline with incobotulinumtoxinA 50U versus placebo (p=0.0004), and numerically improved with incobotulinumtoxinA 25U versus placebo. Sustained improvements were seen with incobotulinumtoxinA ≤70U from EP baseline to EP Week 6 (−1.2) and to EP final visit (−0.7), and from MP baseline to EP final visit (−1.0) (all p Conclusions: IncobotulinumtoxinA showed sustained efficacy in toxin-naive subjects with BEB. Long-term safety results were in line with the known safety profile. Disclosure: Dr. Mitsikostas has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Allergan, Biogen, Electrocore, Cefaly, Eli Lilly, Merck-Serono, Novartis, Roche, Sanofi, Teva. Dr. Mitsikostas has received personal compensation in an editorial capacity for Journal of Headache and Pain. Dr. Mitsikostas has received research support from Biogen, Eli Lilly, Sanofi. Dr. Dekundy has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Merz Pharmaceuticals GmbH. Dr. Sternberg has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities as an employee at Merz Pharmaceuticals and AbbVie Deutschland GmbH & Co. KG. Dr. Althaus has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Employee of Merz Pharmaceuticals. Dr. Althaus holds stock and/or stock options in Fresenius SE, Co KGaA. Dr. Pagan has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Merz, US WorldMeds, Acadia, Teva, AbbVie, Sunovion, and Acorda. Dr. Pagan has received research support from US WorldMeds and Medtronic.