Rhopressa TM (netarsudil ophthalmic solution) 0.02% Rocket 4 Phase 3 Topline Results 1 Exhibit 99.2 |
Important Information Any discussion of the potential use or expected success of our product candidates is subject to our product candidates being approved by regulatory authorities. The information in this presentation is current only as of its date and may have changed or may change in the future. We undertake no obligation to update this information in light of new information, future events or otherwise. We are not making any representation or warranty that the information in this presentation is accurate or complete. Certain statements in this presentation are “forward-looking statements” within the meaning of the federal securities laws. Words such as “may,” “will,” “should,” “would,” “could,” “believe,” “expects,” “anticipates,” “plans,” “intends,” “estimates,” “targets,” “projects,” “potential” or similar expressions are intended to identify these forward-looking statements. These statements are based on the Company’s current plans and expectations. Known and unknown risks, uncertainties and other factors could cause actual results to differ materially from those contemplated by the statements. In evaluating these statements, you should specifically consider various factors that may cause our actual results to differ materially from any forward-looking statements. These risks and uncertainties are described more fully in the quarterly and annual reports that we file with the SEC, particularly in the sections titled “Risk Factors” and “Management’s Discussion and Analysis of Financial Condition and Results of Operations.” In particular, the topline Rocket 4 data presented herein is preliminary and based solely on information available to us as of the date of this presentation and additional information about the results may be disclosed at any time. In addition, the preclinical research discussed in this presentation is preliminary and the outcome of such preclinical studies may not be predictive of the outcome of later clinical trials. Any future clinical trial results may not demonstrate safety and efficacy sufficient to obtain regulatory approval related to the preclinical research findings discussed in this presentation. Such forward-looking statements only speak as of the date they are made. We undertake no obligation to publicly update or revise any forward-looking statements, whether because of new information, future events or otherwise, except as otherwise required by law. 2 |
• Rhopressa™ QD met the criteria for non-inferiority to timolol BID for the primary efficacy analysis (for baseline IOP < 25 mmHg) – Also met non-inferiority for baseline IOP < 27 mmHg and <28 mmHg • Rhopressa™ QD showed stable efficacy from Week 2 to Month 3 • Consistent efficacy results across multiple statistical analysis imputations (PP/ITT/LOCF) • The main adverse event for Rhopressa™ was conjunctival hyperemia, which was reported in ~40% of patients and was scored as mild for ~85% of the patients • There were no drug-related serious adverse events and no evidence of treatment-related systemic effects Rhopressa TM Achieves Primary Clinical Endpoint 3 |
Rocket 4 Trial Design Patients randomized 1:1 Primary endpoints: • Efficacy: Mean IOP at Weeks 2 and 6 and Month 3 for subjects with baseline IOP > 20 mmHg and <25 mmHg (N = 423 subjects per protocol) • Safety: Ocular and systemic safety during a 6-month treatment period Patients with open angle glaucoma (OAG) or ocular hypertension (OHT) with IOP > 20 mmHg and < 30 mmHg at 8am, N = 708 subjects randomized at 52 US sites Rhopressa™ (AR-13324) 0.02% QD (PM) Timolol 0.5% BID (AM and PM) 4 ClinicalTrials.gov Identifier: NCT02558374 |
Baseline Demographics Rhopressa TM QD N = 351 Timolol BID N = 357 Gender, n (%) Male 143 (40.7%) 120 (33.6%) Female 208 (59.3%) 237 (66.4%) Race, n (%) White 259 (73.8%) 274 (76.8%) Black/African American 84 (23.9%) 75 (21.0%) Asian 7 (2.0%) 6 (1.7%) Multiple 0 (0%) 1 (0.3%) Other 1 (0.3%) 1 (0.3%) Age (years) < 65 165(47.0%) 164 (45.9%) >65 186 (53.0%) 193 (54.1%) Iris Color, n (%) Brown/Black 241 (68.7%) 227 (63.6%) Blue/Grey/Green 73 (20.8%) 90 (25.2%) Hazel 36 (10.3%) 40 (11.2%) Other 1 (0.3%) 0 (0%) 5 ++Data on File Based on Rocket 4 Topline Interim 3-month |
Patient Disposition Rhopressa™ QD N = 351 Timolol BID N = 357 Completed Month 3 290 (82.6%) 335 (93.8%) Discontinued Prior to Month 3 61 (17.4%) 22 (6.2%) Discontinued Adverse Event Withdrawal of Consent Non-Compliant Lost to Follow-up Lack of Efficacy Disallowed Concurrent Medication Investigator Decision Protocol Violation Other 39 (11.1%) 7 (2.0%) 1 (0.3%) 0 5 (1.4%) 1 (0.3%) 0 4 (1.1%) 4 (1.1%) 6 (1.7%) 7 (2.0%) 1 (0.3%) 0 0 2 (0.6%) 2 (0.6%) 3 (0.8%) 1 (0.3%) 6 ++Data on File Based on Rocket 4 Topline Interim 3-month |
Patient Disposition (For Baseline IOP < 25 mmHg) Rhopressa™ QD N = 214 Timolol BID N = 209 Completed Month 3 189 (88.3%) 199 (95.2%) Discontinued Prior to Month 3 25 (11.7%) 10 (4.8%) Discontinued Adverse Event Withdrawal of Consent Non-Compliant Lost to Follow-up Lack of Efficacy Disallowed Concurrent Medication Investigator Decision Protocol Violation Other 16 (7.5%) 3 (1.4%) 1 (0.5%) 0 1 (0.5%) 1 (0.5%) 0 2 (0.9%) 1 (0.5%) 4 (1.9%) 2 (1.0%) 1 (0.5%) 0 0 1 (0.5%) 1 (0.5%) 1 (0.5%) 0 7 ++Data on File Based on Rocket 4 Topline Interim 3-month |
Rhopressa TM Achieved Non-Inferiority In the Primary Efficacy Analysis (Baseline IOPs < 25 mmHg) Mean IOP at Each Time Point (Per Protocol) 8 ++Data on File Based on Rocket 4 Topline Interim 3-month efficacy |
Rhopressa TM Phase 3 Rocket 4, Per Protocol Baseline IOPs < 25 mmHg 9 Summary • Upper 95% CI 1.5 mmHg at all time points, 1.0 mmHg at majority (8/9) time points • Rocket 1: 1.0 mmHg at majority (7/9) time points • Rocket 2: 1.0 mmHg at majority (6/9) time points • Met the criteria for demonstrating non-inferiority ++Data on File Based on Rocket 4 Topline Interim 3-month efficacy |
10 Rhopressa TM Achieved Non-Inferiority For Baseline IOP < 27 mmHg ++Data on File Based on Rocket 4 Topline Interim 3-month efficacy Mean IOP at Each Time Point (Per Protocol) |
Rhopressa™ Rocket 4 Efficacy Results for Different Baseline IOPs Baseline IOP (mmHg) Non-inferiority <30 Did not meet** <28 Met <27 Met <26 Met <25* Met <24 Met <22 insufficient power *Primary endpoint ** Missed 2 time points by 0.03 mmHg 11 ++Data on File Based on Rocket 4 Topline Interim 3-month efficacy |
Rocket 4 Rhopressa™ Synergy with PGAs • Rhopressa™ synergy with prior PGA use evident at Week 2 • Observed in previous Rhopressa™ trials 12 Prior PGA No Prior PGA ++Data on File Based on Rocket 4 Topline Interim 3-month efficacy |
Safety/Tolerability Overview of Rhopressa TM • There were no drug-related serious adverse events (SAEs) • There was no evidence of treatment-related systemic effects (e.g., clinical laboratory or haematology values, heart rate or blood pressure) • The most common adverse event was conjunctival hyperemia with ~40% incidence and was scored as mild for ~85% of the patients • Other ocular AEs – AEs occurring in ~5-12% of subjects receiving Rhopressa TM included: conjunctival hemorrhage, cornea verticillata, lacrimation increased and vision blurred 13 ++Data on File Based on Rocket 4 Topline Interim 3-month safety |
Rhopressa TM Phase 3 Safety Profile Adverse Events ( 5% in any group) Rhopressa TM QD N = 351 Timolol BID N = 357 Eye Disorders Conjunctival Hyperemia 148 (42.2%) 24 (6.7%) Conjunctival Hemorrhage 41 (11.7%) 7 ( 2.0%) Cornea Verticillata 41 (11.7%) 0 ( 0.0%) Lacrimation Increased 21 ( 6.0%) 4 ( 1.1%) Vision Blurred 20 ( 5.7%) 2 (0.6%) Administration Site Conditions Instillation Site Pain 82 (23.4%) 89 (24.9%) Instillation Site Erythema 36 (10.3%) 3 (0.8%) Patients with known contraindications or hypersensitivity to timolol were excluded 14 ++Data on File Based on Rocket 4 Topline Interim 3-month safety |
0.0 0.5 1.0 1.5 2.0 2.5 3.0 Baseline Week 2 Week 6 Month 3 Rhopressa™ (N=351) No Change in Mean Hyperemia Score Over Time (Interim Month 3) • Hyperemia severity did not increase with continued dosing • Hyperemia was sporadic • Only ~16% of patients had hyperemia on each study visit day from week 2 to month 3 (similar to the rates seen for Rocket 1, Rocket 2 and Mercury 1) • In Rocket 2, only ~10% of patients had hyperemia on each study visit day from week 2 to month 12 Hyperemia severity 0 = none 1 = mild 2 = moderate 3 = severe 15 ++Data on File Based on Rocket 4 Topline Interim 3-month safety |
When Present, ~85% of Rhopressa TM Hyperemia Graded as Mild For illustrative purposes only Grade Image Description 0 None/Normal 1 Mild 2 Moderate 3 Severe 16 ++ Data on File Based on Rocket 4 Topline Interim 3-month safety |
Rhopressa™ Adverse Events Summary • Conjunctival Hemorrhage • sporadic subconjunctival petechiae • Cornea Verticillata • asymptomatic non-toxic lipid deposits • only visible via biomicroscopy evaluation • Instillation Site Pain • same incidence as timolol • transient ++ Data on File Based on Rocket 4 Topline Interim 3-month safety 17 |
Timolol Caused Statistically Significant Reduction in Heart Rate (Rocket 4) • Timolol reduced mean heart rate by 2 - 3 beats per minute (average across all patients; p < 0.001) • Despite all measures to exclude patients with possible negative sensitivity to beta-blockers 18 ++Data on File Based on Rocket 4 Topline Interim 3-month safety |
Rhopressa TM Performance Summary To Date 19 Well researched with nearly 2,000 clinical patients Once-daily efficacy demonstrated in 4 Phase 3 trials (Rocket 1, 2, 4 and Mercury 1) Stable efficacy through 12 months Synergistic/additive effect with prostaglandin analogues Well-tolerated with no evidence of treatment-related systemic effects Promising early results: e.g., trabecular meshwork anti-fibrotic/disease modification and 24-hour IOP control |
Rhopressa TM and Roclatan TM Key Milestones 20 Q1-2017: Rhopressa™ NDA re-filing expected Q2-2017: Rhopressa™ Rocket 4 Topline safety (6 mos) Q1-2016: Rhopressa™ Rocket 2 Topline safety (12 mos) Q4-2016: Rhopressa™ Rocket 4 Topline efficacy (3 mos) 2016 2017 Q1-2016: Roclatan™ P3 Mercury 2 initiated Near YE 2017: Roclatan™ NDA filing expected Q2-2017: Roclatan™ P3 Mercury 2 Topline efficacy (3 mos) Q3-2017: Roclatan™ P3 Mercury 1 Topline safety (12 mos) 1H-2017: Roclatan™ P3 Mercury 3 (EU) to be initiated Q3-2016: Roclatan™ P3 Mercury 1 Topline efficacy (3 mos) |