AR-13324-CS205 Clinical Pilot Study Topline Results 1
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Executive Summary Netarsudil 0.02% and 0.04% were safe and generally well tolerated in Japanese-American subjects Netarsudil 0.02% and 0.04% were efficacious in lowering IOP in Japanese-American subjects Both concentrations appear to have similar efficacy, but need larger sample size before drawing conclusions Netarsudil IOP lowering in Japanese-American subjects similar to non-japanese data from Phase 1 (low baseline IOP) and Phase 3 studies Netarsudil efficacy in this study (mean baseline IOPs 18-20 mmhg) predicts strong efficacy in low tension glaucoma 3
AR-13324-CS205 Trial Design Patients (Japanese-American within second generation) with open angle glaucoma (OAG) with IOP (unmedicated) 15 mmhg and < 35 mmhg at 8am or ocular hypertension (OHT) with IOP (unmedicated) 22 mmhg and < 35 mmhg at 8am N = 42 subjects randomized Patients randomized 1:1:1 Netarsudil 0.02% QD (PM) Netarsudil 0.04% QD (PM) Placebo QD (PM) Primary endpoints: Safety: Ocular and systemic safety during a 4-week treatment period Efficacy: Mean diurnal IOP at Week 4 ClinicalTrials.gov Identifier NCT03310580 4
IOP mmhg + SEM Efficacy Mean Diurnal IOP 23 Diurnal Average IOP 22 21 20 19.6 20.2 19 18 17 16 15 14 13 17.2 17.6 17.5 18.3 13.2 13.0 13.3 14.9 14.0 15.4 Baseline Week 1 Week 2 Week 4 12 11 Placebo (N=12) Netarsudil 0.02% (N=14) Netarsudil 0.04% (N=14) Note small sample sizes (N=12 to 14) Very different baseline IOPs for 0.02% vs 0.04%* Need to compare using change from baseline analyses *One (1) subject in Netarsudil 0.04% has a higher baseline IOP 5
D IOP mmhg + SEM Efficacy Change in Mean Diurnal IOP 0 Diurnal D IOP -1 Week 1-2 -3-2.5-2.0-2.1 Week 2 Week 4-4 -5-6 -5.0-5.3-5.0-5.2-5.7-7 -6.6-8 Placebo (N=12) Netarsudil 0.02% (N=14) Netarsudil 0.04% (N=14) 0.02% efficacy stable at -5.0 to -5.3 mmhg 0.04% efficacy bouncing around but similar to 0.02% at Week 1 and Week 4 Placebo effect similar to other studies 6
Safety/Tolerability Overview of Netarsudil There were no serious adverse events (SAEs) The most common adverse event was conjunctival hyperemia and was scored as mild in the majority of the patients 7
Mean Hyperemia Mean Hyperemia Score Over Time at 8am 3.0 2.5 2.0 Netarsudil 0.02% (N=15) Netarsudil 0.04% (N=14) Hyperemia severity 0 = none 1 = mild 2 = moderate 3 = severe 1.5 1.0 0.5 0.0 Baseline Week 1 Week 2 Week 4 Visits 8