OCS-01 Phase 3 DIAMOND Trials Topline Results in DME May 29, 2026
These slides and the accompanying oral presentation contain forward-looking statements and information. The use of words such as “may,” “might,” “will,” “should,” “expect,” “plan,” “anticipate,” “believe,” “estimate,” “project,” “intend,” “future,” “potential,” or “continue,” and other similar expressions are intended to identify forward-looking statements. For example, all statements we make regarding our regulatory and development plans for OCS-01 in diabetic macular edema or other indications, the timing, progress and regulatory strategy for our other research and development programs, our cash runway and statements about the potential therapeutic effects of our product candidates are forward-looking. All forward-looking statements are based on estimates and assumptions by our management that, although we believe to be reasonable, are inherently uncertain. The clinical trial results presented in this presentation are topline and preliminary and subject to change, as analysis is ongoing. All forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those that we expected. Factors that may cause actual results to differ materially from current expectations include, but are not limited to: the possibility that Oculis may be adversely affected by economic, business, and/or competitive factors; Oculis' estimates of expenses and profitability; Oculis' ability to develop, manufacture and commercialize the product candidates in its pipeline; actions of regulatory authorities, which may affect the initiation, timing and progress of clinical studies or future regulatory approvals or marketing authorizations; the ability of Oculis or its partners to enroll and retain patients in clinical studies; the ability of Oculis or its partners to gain approval from regulators for planned clinical studies, study plans or sites; Oculis' ability to obtain and maintain regulatory approval or authorizations of its products, including the timing or likelihood of expansion into additional markets or geographies; the success of Oculis' current and future collaborations, joint ventures, partnerships or licensing arrangements; financial position, strategy and anticipated milestones; and other risks and uncertainties set forth in the sections entitled “Risk Factors” and “Cautionary Note Regarding Forward-Looking Statements” in documents that Oculis may from time to time file or furnish with the SEC. Any forward-looking statement speaks only as of the date on which it was made. We undertake no obligation to update or revise any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law. Cautionary note on forward-looking statements Copyright of this presentation is owned by Oculis. No part of this presentation may be reproduced in any manner without the permission of Oculis. Safe Harbor Statements
Phase 3 DIAMOND Program Topline Results Summary At this time, Oculis does not plan to pursue FDA regulatory filing with OCS-01 for DME DIAMOND-1 DIAMOND-2 Visual function: Primary endpoint not met Mean Change in BCVA from Baseline at Week 52 OCS-01: 1.2 letters Vehicle: 5.1 letters OCS-01: 4.6 letters Vehicle: 5.2 letters Retinal anatomy: Rapid and sustained reduction Mean Change in CST from Baseline at Week 52 OCS-01: -51.8 µm Vehicle: -31.7 µm OCS-01: -64.3 µm Vehicle: -18.8 µm Safety OCS-01 was well tolerated with no unexpected safety findings
Study Design and Patient Population
OCS-01 | Phase 3 DIAMOND Program in DME BCVA: best corrected visual acuity; CST: central subfield thickness; DME: diabetic macular edema; ETDRS: Early Treatment Diabetic Retinopathy Study; VEGF: vascular endothelial growth factor. Multicenter Study on the Efficacy and Safety of OCS-01 in Subjects With Diabetic Macular Edema. ClinicalTrials.gov identifier: NCT05066997 Study of the Efficacy and Safety of OCS-01 Eye Drops in Subjects With Diabetic Macular Edema (DIAMOND-2). ClinicalTrials.gov identifier: NCT06172257 Evaluate the safety and efficacy of OCS-01 vs vehicle for the treatment of DME in two adequate and well-controlled global Phase 3 clinical trials Age 18-85 years Confirmed diagnosis of DME Diabetes mellitus 1 and 2 ETDRS BCVA letter score: 65-24 Retinal thickness (CST) ≥ 310 µm Primary endpoint: Change in BCVA ETDRS letter score at Week 52 Key secondary endpoint: % with a ≥ 15 ETDRS letter gain in BCVA at Week 52 CST change vs Baseline Objective Endpoints Study Population Screening OCS-01 Vehicle End of trial Randomization 1:1 46 weeks Maintenance Phase 3x/day 6 weeks Induction Phase 6x/day
ITT Population Diamond Patient Disposition AE: adverse event; ITT: intention-to-treat. DIAMOND-1 DIAMOND-2 Screen failures n=285 OCS-01 n=200 Vehicle n=203 Completed 52 weeks n=164 Completed 52 weeks n=163 Discontinued study Withdrawn: n=19 AE: n=9 Lost to follow-up: n=12 Other: n=0 Discontinued study Withdrawn: n=12 AE: n=13 Lost to follow-up: n=10 Other: n=1 Randomized n=404 Screened n=689 Screen failures n=312 OCS-01 n=200 Vehicle n=201 Completed 52 weeks n=168 Completed 52 weeks n=168 Discontinued study Withdrawn: n=13 AE: n=8 Lost to follow-up: n=5 Other: n=7 Discontinued study Withdrawn: n=11 AE: n=7 Lost to follow-up: n=9 Other: n=5 Randomized n=401 Screened n=713 1 patient randomized, not dosed
DIAMOND-1 DIAMOND-2 Parameter OCS-01 (N=200) Vehicle (N=203) OCS-01 (N=200) Vehicle (N=201) Age, mean years (SD) 63.5 (9.77) 64.2 (9.57) 65.0 (8.68) 64.7 (8.33) Sex, n (%) Male Female 120 (60.0) 80 (40.0) 111 (54.7) 92 (45.3) 113 (56.5) 87 (43.5) 108 (53.7) 93 (46.3) Duration of DME, mean months (SD) 21.95 (33.866) 25.20 (34.352) 23.62 (32.916) 24.16 (40.082) BCVA, mean ETDRS letter score (SD) 58.2 (7.37) 57.8 (8.02) 57.2 (9.21) 57.2 (7.62) CST, mean µm (SD) 458.8 (117.35) 453.4 (128.22) 457.9 (145.77) 466.0 (147.83) IOP, mean mm Hg (SD) 14.8 (2.95) 14.9 (3.08) 14.6 (2.95) 14.6 (2.78) Treatment status, n (%) Naïve Previously-treated 109 (54.5) 91 (45.5) 102 (50.2) 101 (49.8) 101 (50.5) 99 (49.5) 97 (48.3) 104 (51.7) Lens status, n (%) * Phakic Pseudo phakic 125 (62.5) 75 (37.5) 128 (63.1) 75 (36.9) 119 (59.5) 80 (40.0) 118 (58.7) 83 (41.3) ITT Population Diamond Baseline Demographics BCVA: best corrected visual acuity; CST: central subfield thickness; DME: diabetic macular edema; ETDRS: Early Treatment Diabetic Retinopathy Study; ITT: intention-to-treat; SD: standard deviation. *1 patient in DIAMOND-2 OCS-01 arm was aphakic
Efficacy
Mean Change in CST, Baseline to Week 52 CST: central subfield thickness; ITT: intention-to-treat; LS: least squares; SE: standard error. Mean Change in CST Baseline to Week 52 DIAMOND-1 ITT Population 1 6 12 18 30 24 36 42 48 52 0 P <0.0001 P <0.0001 P <0.0001 P <0.0001 P <0.0001 P <0.0001 P =0.0013 P =0.0023 P =0.0396 P NS
Mean Change in CST, Baseline to Week 52 CST: central subfield thickness; ITT: intention-to-treat; LS: least squares; SE: standard error. Mean Change in CST Baseline to Week 52 DIAMOND-2 ITT Population 1 6 12 18 30 24 36 42 48 52 0 P <0.0001 P <0.0001 P <0.0001 P <0.0001 P <0.0001 P =0.0002 P <0.0001 P <0.0001 P <0.0001 P=0.0016
ITT Population BCVA: best corrected visual acuity; CI: confidence interval; ETDRS: Early Treatment Diabetic Retinopathy Study; ITT: intention-to-treat; LS: least squares; SE: standard error. Visual Function: Primary Endpoint Mean Change in BCVA from Baseline at Week 52 Mean Change in BCVA from Baseline at Week 52 DIAMOND-1 Mean Change in BCVA from Baseline at Week 52 DIAMOND-2 P value NS P value NS
ITT Population BCVA: best corrected visual acuity; ETDRS: Early Treatment Diabetic Retinopathy Study; ITT: intention-to-treat. Visual Function: Key Secondary Endpoint ≥15-Letter Gain in BVCA at Week 52 ≥15-Letter Gain from Baseline at Week 52 DIAMOND-1 ≥15-Letter Gain from Baseline at Week 52 DIAMOND-2 P value NS P value NS
Safety
Safety Summary OCS-01 was well tolerated, with no unexpected adverse events observed. Overall safety profile was consistent with that of previous trials. As expected, elevated IOP and cataracts were higher in the OCS-01 treated patients in line with chronic use of steroid in DME.
Summary
OCS-01 DIAMOND TLR Summary and Pipeline Prioritization Despite showing a rapid, substantial and sustained reduction in retinal thickness in patients treated with OCS-01, primary (mean change in BCVA) and key secondary (≥15-letter gainers) endpoints for both trials were not met at Week 52. Based on the results, at this time Oculis does not plan to pursue an FDA regulatory filing for OCS-01 in DME. Oculis will strategically focus resources on advancing our late-stage portfolio, including the Privosegtor platform, starting with the PIONEER program for Privosegtor in optic neuropathies, and the PREDICT-1 trial for Licaminlimab to drive precision medicine in dry eye disease. Financial position remains strong with $278 million in cash, cash equivalents, and short-term investments as of March 31, 2026, providing cash runway into 2H 2029.
Q&A