Oculis Holding Ag · NASDAQ:OCS
Oculis — Investor Day (February 2024)
110 slides from Oculis's investor day presentation, Feb 28, 2024
Slide 1: R&D Day February 28, 2024
Exhibit 99.1 R&D Day February 28, 2024
Slide 2: Safe Harbor Statements
Safe Harbor Statements Cautionary note on forward-looking statements These slides and the accompanying oral presentation, as well as slides and presentations made today by others, contain forward-looking statements and information as defined in the Private Securities Litigation Reform Act of 1955, a
Slide 3: Agenda
9:00 - 9:05 Welcome & Speaker Introduction Sylvia Cheung AM 9:05 - 9:15 Opening Remarks & Oculis Overview Riad Sherif, MD AM OCS-01 in Diabetic Macular Edema 9:15 – 9:25 DIAMOND Program Update Arshad Khanani, MD, MA, FASRS AM Potential of OCS-01 in Clinical Practice: 9:25 – 9:35 • Retina specialist
Slide 4: OCS-01 for Diabetic Macular Edema
OCS-01 for Diabetic Macular Edema A topical Optireach® formulation of dexamethasone designed to reach the retina David Boyer, M.D. Eric Donnenfeld, M.D. Pravin Dugel, M.D. Keck School of Medicine, University of Southern California, and Retina Vitreous New York University Oculis Board of Directors As
Slide 5: OCS-02 (licaminlimab) in Dry Eye Disease
OCS-02 (licaminlimab) in Dry Eye Disease TNFα inhibitor eye drop formulation developed with a proprietary antibody fragment technology Christophe Baudouin, Anat Galor, Eric Donnenfeld, M.D. M.D., Ph.D. M.D., M.S.P.H. New York University Quinze-Vingts National Ophthalmology Bascom Palmer Eye Institut
Slide 6: Our Purpose
Our Purpose To drive innovation to save sight and improve eye care
Slide 7: Strong Start to 2024 after a Successful 2023
Strong Start to 2024 after a Successful 2023 Mar May June Aug Aug Nov Dec Dec Feb Feb OPTIMIZE RELIEF DIAMOND Enrollment in NASDAQ DIAMOND Stage 1 Public Offering LEOPARD OPTIMIZE-2 Ph3 DIAMOND-2 Ph 3 Topline Ph2b Stage 2 Ph 3 RELIEF Ph2b Ph 3 Topline Gross Proceeds: PoC Initiated Initiated Initiate
Slide 8: Experienced Leadership Team with Successful Track Record
Experienced Leadership Team with Successful Track Record Committed to build an industry leader in ophthalmic innovation ✓ Highly experienced leadership team ✓ Expertise in drug development leading to approvals and launches of >40 approved drugs globally ✓ Expertise in public company management and l
Slide 9: Major Innovations Addressing Highly Meaningful Unmet Medical Needs
3 Major Innovations Addressing Highly Meaningful Unmet Medical Needs OCS-05 OCS-02 (Licaminlimab) OCS-01 ® Promising neuroprotective agent Antibody fragment technology OPTIREACH enables eye drops for neuro retina diseases enables biologic eye drop treating retinal disease To address neurological dam
Slide 10: Innovative, Diversified and Late-stage Pipeline
Innovative, Diversified and Late-stage Pipeline Product Investigational 2023 Upcoming Pre-clinical Phase 1 Phase 2 Phase 3 Candidate(s) Indication(s) Accomplishments Key Catalysts Positive DIAMOND Stage 1 DIABETIC MACULAR EDEMA Initiated Ph3 Stage 2 OCS-01 Positive OPTIMIZE-1 ® INFLAMMATION AND PAIN
Slide 11: DME is a Large and Growing Market with Critical Unmet Needs
DME is a Large and Growing Market with Critical Unmet Needs OCS-01 eye drops: potential to expand pool of treated DME patients & improve outcomes for those currently treated Growing DME patient Only invasive Late start 1 population size treatments approved of treatment Global DME Patients DME – Trea
Slide 12: OCS-01 Would Be Able to Address All Segments
OCS-01 | Would Be Able to Address All Segments while Leading 2 Segments Alone: Early Intervention and Treatment Customization Patient presents with DME with DME DME with moderate to severe 43% 33% 24% DME symptoms mild visual recent onset visual impairment impairment Diagnosed by OCT 1 1 1 DME Disea
Slide 13: Large and Growing DED Opportunity
Large and Growing DED Opportunity Market still underpenetrated and unsatisfied Dry Eye Rx drug market in Significant unmet need and market opportunity 1 G7 countries in 2021 • Large and growing unmet medical need with ~10 million Corticosteroids Secretagogues, 1,2 (ophthalmic), 4% diagnosed moderate
Slide 14: OCS-02 Positive Ph 2 Results in Dry Eye Disease and Uveitis
OCS-02 | Positive Ph 2 Results in Dry Eye Disease and Uveitis Programs advancing into Phase 2b Positive Phase 2 / PoC Advancing into studies in DED and Uveitis Phase 2b for both indications DED#1 85 patients Phase 2 PoC Phase 2b to evaluate signs in DED Successfully completed (with secondary endpoin
Slide 15: OCS-02 Potential to Transform Treatment of Moderate-to-Severe Dry Eye
OCS-02 | Potential to Transform Treatment of Moderate-to- Severe Dry Eye Patients with Novel Anti-TNFα Eye Drop 2 2 20 million Moderate (35%) Severe (14%) diagnosed U.S. 1 DED patients 1,2 ~ 10 million diagnosed moderate-to-severe DED patients in the U.S. Current 3 Only 13% feel their chronic DED is
Slide 16: OCS-01 in Diabetic Macular Edema
OCS-01 in Diabetic Macular Edema
Slide 17: OCS-01 for Diabetic Macular Edema
OCS-01 for Diabetic Macular Edema A topical Optireach® formulation of dexamethasone designed to reach the retina David Boyer, M.D. Eric Donnenfeld, M.D. Pravin Dugel, M.D. Keck School of Medicine, University of Southern California, and Retina Vitreous New York University Oculis Board of Directors As
Slide 18: DIAMOND Program Update
DIAMOND Program Update Arshad Khanani, MD, MA, FASRS Reno School of Medicine, University of Nevada, and Sierra Eye Associates Co-Primary Investigator On behalf of the DIAMOND Trial Investigators 18 18
Slide 19: Disclosures
Disclosures Arshad M. Khanani, MD, MA, FASRS is the director of clinical research at Sierra Eye Associates and clinical associate professor at the University of Nevada. Dr. Khanani reports consultancy to AbbVie, Adverum, Alcon, Amgen, Annexin, Annexon, Apellis Pharmaceuticals, Aviceda Therapeutics,
Slide 20: OCS-01 Phase 3 DIAMOND Program in DME
OCS-01 | Phase 3 DIAMOND Program in DME Evaluating OCS-01 efficacy and safety STAGE 1 STAGE 2 COMPLETED TWO IDENTICAL 52-wk GLOBAL PIVOTAL TRIALS INITIATED ALL COMERS: 2/3 Naïve, 1/3 Previously Treated 6 weeks 6 weeks 6 weeks 46 weeks N=350-400/trial N=148 OCS-01 OCS-01 OCS-01 OCS-01 (6x/day) (3x/da
Slide 21: Key Takeaways From OCS-01 DIAMOND Stage 1
5 Key Takeaways From OCS-01 DIAMOND Stage 1 Robust statistically significant improvement in vision and reduction in retinal edema vs vehicle 7.2-letter gain in BCVA vs baseline at week 6, increasing to 7.6 at week 12 25.3% of patients gained ≥15 letters at week 6, increasing to 27.4% at week 12 Rapi
Slide 22: 7.2-letter gain in BCVA vs baseline at week 6
5 Key Takeaways From OCS-01 DIAMOND Stage 1 7.2-letter gain in BCVA vs baseline at week 6, increasing to 7.6 at week 12 BCVA ETDRS LETTER CHANGE AT WEEK 6 BCVA ETDRS LETTER CHANGE AT WEEK 12 Primary Endpoint Secondary Endpoint 9.0 9.0 P = 0.007 P = 0.016 7.6 6.0 6.0 7.2 3.0 3.0 3.7 3.1 0.0 0.0 OCS-0
Slide 23: Key Takeaways From OCS-01 DIAMOND Stage 1
5 Key Takeaways From OCS-01 DIAMOND Stage 1 Robust statistically significant improvement in vision and reduction in retinal edema vs vehicle 7.2-letter gain in BCVA vs baseline at week 6, increasing to 7.6 at week 12 25.3% of patients gained ≥15 letters at week 6, increasing to 27.4% at week 12 Rapi
Slide 24: 25.3% of patients gained ≥15 letters at week 6
5 Key Takeaways From OCS-01 DIAMOND Stage 1 25.3% of patients gained ≥15 letters at week 6, increasing to 27.4% at week 12 * ≥15-LETTER GAINERS AT WEEK 6 ≥15-LETTER GAINERS AT WEEK 12 30 30 25 27.4% 25 P = 0.015 P = 0.009 25.3% 20 20 15 15 10 10 9.8% 5 7.5% 5 0 0 OCS-01 VEHICLE OCS-01 VEHICLE N=100
Slide 25: Key Takeaways From OCS-01 DIAMOND Stage 1
5 Key Takeaways From OCS-01 DIAMOND Stage 1 Robust statistically significant improvement in vision and reduction in retinal edema vs vehicle 7.2-letter gain in BCVA vs baseline at week 6, increasing to 7.6 at week 12 25.3% of patients gained ≥15 letters at week 6, increasing to 27.4% at week 12 Rapi
Slide 26: Rapid reduction in retinal edema already at week 2
5 Key Takeaways From OCS-01 DIAMOND Stage 1 Rapid reduction in retinal edema already at week 2 CHANGE IN CST AS ASSESSED BY SD-OCT 30.0 OCS-01 (N=100) 20.0 10.2 5.4 Vehicle (N=48) 10.0 1.1 -1.4 0.0 -16.0 -10.0 -20.0 -30.0 -40.0 P = 0.004 P < 0.0001 -50.0 -60.0 -53.5 -61.6 -59.0 -63.5 -61.4 -70.0 -80
Slide 27: Key Takeaways From OCS-01 DIAMOND Stage 1
5 Key Takeaways From OCS-01 DIAMOND Stage 1 Robust statistically significant improvement in vision and reduction in retinal edema vs vehicle 7.2-letter gain in BCVA vs baseline at week 6, increasing to 7.6 at week 12 25.3% of patients gained ≥15 letters at week 6, increasing to 27.4% at week 12 Rapi
Slide 28: Well-tolerated with no unexpected AEs
5 Key Takeaways From OCS-01 DIAMOND Stage 1 Well-tolerated with no unexpected AEs Treatment-Emergent Adverse Events Treatment-Emergent Serious Adverse Events >2.0% in the OCS-01 arm or OCS-01 (n=100) Vehicle (n=48) OCS-01 (n=100) Vehicle (n=48) n (%) n (%) n (%) n (%) >4.0% in the vehicle arm Any oc
Slide 29: Minimal mean IOP increase across induction and maintenance
5 Key Takeaways From OCS-01 DIAMOND Stage 1 Minimal mean IOP increase similar across induction and maintenance CHANGE IN IOP ACROSS INDUCTION AND MAINTENANCE 35 30 OCS-01 (N=100) 25 Vehicle (N=48) 20 15 10 2.79 2.39 2.10 1.61 0.64 5 0.44 0.0 0.08 0 -0.05 -0.17 -5 2 4 6 8 12 Safety population Weeks M
Slide 30: Key Takeaways From OCS-01 DIAMOND Stage 1
5 Key Takeaways From OCS-01 DIAMOND Stage 1 Robust statistically significant improvement in vision and reduction in retinal edema vs vehicle 7.2-letter gain in BCVA vs baseline at week 6, increasing to 7.6 at week 12 25.3% of patients gained ≥15 letters at week 6, increasing to 27.4% at week 12 Rapi
Slide 31: DIAMOND Program Key Milestones
DIAMOND Program Key Milestones 2023 2024 May January US Investigators’ Meeting Stage 1 topline data Dallas, TX December February FPFV DIAMOND-1 FPFV DIAMOND-2 FPFV (First patient first visit). 31 31 31 31
Slide 32: DIAMOND Program Key Milestones
DIAMOND Program Key Milestones 2023 2024 May January US Investigators’ Meeting Stage 1 topline data Dallas, TX December February FPFV DIAMOND-1 FPFV DIAMOND-2 FPFV (First patient first visit). 32 32 32 32
Slide 33: DIAMOND US Investigators' Meeting
DIAMOND US Investigators’ Meeting High engagement and strong attendance with 95% of US sites represented “Informative training and presentations that will give me confidence going into the study and screening my first patient” “Energetic speakers that were personable and passionate about the study a
Slide 34: Key Takeaways From OCS-01 DIAMOND Stage 1
5 Key Takeaways From OCS-01 DIAMOND Stage 1 Robust statistically significant improvement in vision and reduction in retinal edema vs vehicle 7.2-letter gain in BCVA vs baseline at week 6, increasing to 7.6 at week 12 25.3% of patients gained ≥15 letters at week 6, increasing to 27.4% at week 12 Rapi
Slide 35: Potential of OCS-01 in Clinical Practice: Retina Specialist
Potential of OCS-01 in Clinical Practice: Perspective from a retina specialist Ramin Tadayoni, MD, PhD Oculis Chief Scientific Officer, Rothschild Foundation Hospital, Paris, France President of EURETINA 35 35
Slide 36: Disclosures
Disclosures* Grants and consulting for Novartis, AbbVie, Allergan, and Bayer Consulting for Alcon, Genentech, Roche, Thea, KHB, Apellis, Iveric Bio, Optic2000, Zeiss and Oculis *Disclosures listed are prior to Dr. Tadayoni appointment as Oculis Chief Scientific Officer. 36 36 36 36
Slide 37: DME Is a Leading Cause of Vision Loss in Working-Age Adults
1 DME Is a Leading Cause of Vision Loss in Working-Age Adults Only invasive Unmet needs for ~37M therapies are available early intervention 2,3* for DME, which have a high & inadequate responders DME patients worldwide 4 treatment burden to anti-VEGFs * 37M estimated population calculated based on 2
Slide 38: Real-World Data Highlights Unmet Need of Many DME Patients
Real-World Data Highlights Unmet Need of Many DME Patients TREATMENT RATE IN 1,a 13,410 NEWLY DIAGNOSED US DME PATIENTS 2 60% Unmet need for early intervention 74% 40% of patients treated with anti-VEGF 40% 3 have an inadequate response 26% “Inadequate response” as <5-letter BCVA improvement 28 Days
Slide 39: Challenges in the Current Management of DME Patients
Challenges in the Current Management of DME Patients 1 Recent onset and mild patients not treated due to risk / benefit of invasive therapy 2 Multifactorial disease involving inflammation requiring a different MOA 3 Patients discontinuing treatment due to burden associated with invasive therapy 3 Pa
Slide 40: Diabetic Patient With Bilateral DME at Two Different Stages
PATIENT CASE Diabetic Patient With Bilateral DME at Two Different Stages 56-year-old female: presenting for loss of vision in her right eye MRx Has known bilateral diabetic retinopathy RIGHT LEFT 20/63 20/25 Type 2 diabetic for 18 years, blood A1c levels ~8% RIGHT LEFT LASTING DME EARLY DME DME (Dia
Slide 41: Diabetic Patient With Bilateral DME at Two Different Stages
PATIENT CASE Diabetic Patient With Bilateral DME at Two Different Stages RIGHT LEFT Early DME Lasting DME Anti-VEGF Anti-VEGF Resisting DME Cured DME IVT steroid 20/32 Sequelae of DME Normal looking 20/20 LASTING DME EARLY DME DME (Diabetic macular edema); IVT (Intravitreal). 41 41 41 41
Slide 42: Diabetic Patient With Bilateral DME at Two Different Stages
PATIENT CASE Diabetic Patient With Bilateral DME at Two Different Stages RIGHT LEFT SEQUELAE OF DME NORMAL LOOKING Inadequate response to monthly anti-VEGF Early intervention preserved left eye vision (final 20/32) (final 20/20) Anti-VEGF–resistant DME Usually not treated or exceptionally Today: Tod
Slide 43: Potential of Topical OCS-01 to Ease DME Patient Burden
Potential of Topical OCS-01 to Ease DME Patient Burden and Improve Patient Care Patients with an inadequate response to anti-VEGF Complementary eye drop for anti-VEGF–treated patients Early intervention for patients currently untreated DME (Diabetic macular edema); IVT (Intravitreal). 43 43 43 43
Slide 44: Potential of OCS-01 in Clinical Practice: Cornea/Cataract Expert
Potential of OCS-01 in Clinical Practice: Perspective from a cornea/cataract expert Elizabeth Yeu, MD Eastern Virginia Medical School, Virginia Eye Consultants, and President of ASCRS 44 44
Slide 45: Disclosures
Disclosures Elizabeth Yeu, MD, is the president of the American Society of Cataract and Refractive Surgery (ASCRS) and is an ophthalmologist at Virginia Eye Consultants. Dr. Yeu reports consultancy to AcuFocus, Adaptilens, Advanced Vision Group, Alcon, Aldeyra, Abbvie, Aurion, Avellino, Bausch & Lom
Slide 46: OCS-01 Presents an Opportunity for Retina and Non-Retina Ophthalmologists
OCS-01 Presents an Opportunity for Both Retina and Non-Retina Ophthalmologists For early intervention by any ophthalmologist LANDSCAPE OF US EYE CARE POPULATION diagnosing DME 1 3,000 retina specialists For inadequate responders to current SoC either as: 8,000 anterior segment 2 physicians Standalon
Slide 47: Diabetic Patient Presenting for Routine Cataract Evaluation
PATIENT CASE Diabetic Patient Presenting for Routine Cataract Evaluation 65-year-old male: presenting for cataract evaluation Has had painless progressive loss of vision RIGHT No known DME on arrival Type 2 diabetic for 25 years, blood A1c levels ~7-8% Date Exam SPH CYL Axis ADD DVA NVA 09Jan2024 Le
Slide 48: Initial imaging suggests something beyond cataracts
PATIENT CASE Diabetic Patient Presenting for Routine Cataract Evaluation Initial imaging suggests something beyond cataracts is causing vision loss RIGHT LEFT NUMEROUS BRIGHT SPOTS (LIPID DEPOSITION) SPARSE RED SPOTS (INTRARETINAL HEMORRHAGING) SEVERAL RED SPOTS (INTRARETINAL HEMORRHAGING) Color fun
Slide 49: Further imaging confers intraretinal bleeding
PATIENT CASE Diabetic Patient Presenting for Routine Cataract Evaluation Further imaging confers intraretinal bleeding due to diabetic damage RIGHT LEFT INTRARETINAL HEME IS MORE APPARENT INTRARETINAL HEME IS MORE APPARENT Fundus autofluorescence 49 49 49 49
Slide 50: Macular edema is worse in left eye than right eye
PATIENT CASE Diabetic Patient Presenting for Routine Cataract Evaluation Macular edema (CSME) is worse in left eye than in right eye RIGHT LEFT MINIMAL MACULAR EDEMA PROMINENT CSME AND VESSEL LEAKAGE RESPONSIBLE FOR LOSS OF VISION Optical coherence tomography (Green line indicates transverse plane i
Slide 51: Diabetic Patient Presenting for Routine Cataract Evaluation
PATIENT CASE Diabetic Patient Presenting for Routine Cataract Evaluation “Mr. Smith, your cataracts are a similar in size in both eyes but, I agree it is slightly larger in the left eye. If your only disease were the cataracts, then your vision should be about the same, but, as you’ve specifically n
Slide 52: Diabetic Patient Presenting for Routine Cataract Evaluation
PATIENT CASE Diabetic Patient Presenting for Routine Cataract Evaluation “So, it is ESSENTIAL that I send you over to the retina specialist for an urgent evaluation within the next 7-10 days in order to get treatment.” INSTEAD, I HOPE TO BE SAYING ONE DAY “It is essential that I get you STARTED on t
Slide 53: Potential of Topical OCS-01 to Ease DME Patient Burden
Potential of Topical OCS-01 to Ease DME Patient Burden and Improve Patient Care Treatable DME patients are typically working individuals Potential for co-management of patients Early intervention treatment option for non-retina specialists DME (Diabetic macular edema). 53 53 53 53
Slide 54: DME Q&A Session
DME Q&A Session Dr. Pravin Dugel Dr. Riad Sherif
Slide 55: OCS-02 (Licaminlimab) in Dry Eye Disease
OCS-02 (Licaminlimab) in Dry Eye Disease February 28, 2024
Slide 56: OCS-02 (licaminlimab) in Dry Eye Disease
OCS-02 (licaminlimab) in Dry Eye Disease TNFα inhibitor eye drop formulation developed with a proprietary antibody fragment technology Christophe Baudouin, Anat Galor, Eric Donnenfeld, M.D. M.D., Ph.D. M.D., M.S.P.H. New York University Quinze-Vingts National Ophthalmology Bascom Palmer Eye Institut
Slide 57: Unmet Needs in Dry Eye Disease
Unmet Needs in Dry Eye Disease Eric Donnenfeld, MD New York University 57 57
Slide 58: Disclosures
Disclosures Eric Donnenfeld, MD is a Clinical Professor of Ophthalmology at New York University and former president of ASCRS. Dr. Donnenfeld reports consultancy to Aeon, Allegro, Allergan , Alcon, Aurion, Avellino Labs, Bausch & Lomb, CorneaGen, Covalent, CRST, Crystilex, BVI, Blephex, Dompe, ELT S
Slide 59: Significant Unmet Need for Treating Patients with Dry Eye Disease
Significant Unmet Need for Treating Patients with Dry Eye Disease Multifactorial disease High demand for novel ~110M people 1* with substantial impact therapies due to with DED in G7 on quality of life and limitations of current 3 1 functional vision treatments ~10M diagnosed moderate to severe 1,2
Slide 60: Despite New Treatment Options, Patient Satisfaction Remains a Challenge
Despite New Treatment Options, Patient Satisfaction Remains a Challenge 1 Rx Treatments 2016 2021 2002 2018 2020 2023 OTC tears In-office procedures ~13% of diagnosed patients with DED are being 2 treated with a prescription medicine in the US Only 13% of patients feel that their chronic dry eye 3 i
Slide 61: High Unmet Need for Novel Treatments for DED
High Unmet Need for Novel Treatments for DED EFFICACY ONSET TOLERABILITY Clinical relief is not achieved Many patients experience a Commonly reported adverse for many patients despite slow onset of action with events include dysgeusia and several approved therapies current therapeutic options instil
Slide 62: Innovations in DED Lag in Comparison to Other Specialties
Innovations in DED Lag in Comparison to Other Specialties The rise of TNF-α inhibitors SYSTEMIC INFLAMMATORY 26 years DISEASE TREATMENTS Global Sales: First anti-TNF: 3 ENBREL®: $9.4B (2015) 1 2 Cyclosporine Infliximab 3 REMICADE®: $8.1B (2015) 2 Etanercept * 4 HUMIRA® : $21.6B (2022) 1983 1998 2002
Slide 63: Inhibition of TNF-α is Widely Used to Treat Inflammatory Diseases
Inhibition of TNF-α is Widely Used to Treat Inflammatory Diseases Five FDA-approved TNF-α inhibitors indicated as systemic therapy for several inflammatory diseases TNF-α is central in other diseases Crohn’s disease ENBREL® Ulcerative colitis CIMZIA® Plaque psoriasis REMICADE® Arthritis SIMPONI® Uve
Slide 64: TNF-α inhibitors have markedly improved disease management
TNF-α inhibitors have markedly improved the disease management and treatment outcomes for patients with inflammatory disorders Ophthalmology could be the next chapter Leone GM, et al. J Clin Med. 2023;12(4):1630. 64 64 64 64
Slide 65: OCS-02 Potential to Become First Topical Anti-TNF-α Treatment
OCS-02 (Licaminlimab) – Potential to Become the First Topical Anti-TNF-α Treatment for Inflammatory Ophthalmic Diseases * Topical OCS-02 (Licaminlimab) eye drops Illustration of fragment technology Illustration of fragment technology scFv* IgG Fab (OCS-02) Illustration of fragment technology scFv* I
Slide 66: The Treatment Landscape for DED Is Evolving
The Treatment Landscape for DED Is Evolving Artificial tears Cyclosporine and TNF-α Inhibitors T-cell modulators Clinical trials suggest OCS-02 (Licaminlimab) has the potential to change DED treatment DED (Dry eye disease). 66 66 66 66
Slide 67: TNF Inhibition in Inflammatory Eye Diseases
TNF Inhibition in Inflammatory Eye Diseases Christophe Baudouin, MD, PhD, FARVO President, European Dry Eye Society Director of University Hospital Institute, IHU-FORESIGHT Sorbonne and Paris Saclay Universities Paris, France Professor and Chair of Ophthalmology, Quinze-Vingts National Ophthalmology
Slide 68: Disclosures
Disclosures Christophe Baudouin, MD, PhD, FARVO is the president of the European Dry Eye Society and Director of University Hospital Institute, IHU-FORESIGHT Sorbonne and Paris Saclay Universities; Professor and Chair of Ophthalmology, Quinze-Vingts National Ophthalmology Hospital. Dr. Baudouin repo
Slide 69: Key Mechanisms that Drive DED: The Vicious Circle Theory
S y s t e y m g r i e c l l d A r u g s O / s c s g e u l v u i a r t r a d s v l u r a e r s g c i e e r p r p o y T n i g r a L s e A m i t S i s I r d K a u i l / l r u r / e g g s f e e i r r a s r r c y a i t l i v a e h C D N G e M u / r s i o t Key Mechanisms that Drive DED: The Vicious Circl
Slide 70: Factors Contributing to Inflammation in DED
1,2 Factors Contributing to Inflammation in DED Initial ocular or extraocular inflammation/autoimmunity Hyperosmotic stress Mechanical stress Neurogenic inflammation Toxic pro-inflammatory drugs/preservatives T-cell activation (TH17) following dessicative stress DED (Dry eye disease). 1. Baudouin C,
Slide 71: Evidence of Inflammation in DED: Goblet Cell Depletion
1,2 Evidence of Inflammation in DED: Goblet Cell Depletion Tear film instability/ Tear film instability Deficiency /imbalance Goblet cells Tear/cell Tear/cell Goblet normal state Goblet hyperosmolarity hyperosmolarity ce cel ll l l lo os ss s Cell Cytokine release Loss of immune damage homeostasis M
Slide 72: Inflammatory Cells, Neuromas Associated to Severe DED
Inflammatory Cells, Neuromas Associated to Severe DED DED (Dry eye disease). 72 72 72 72
Slide 73: HLA-DR Langerin CD95 (Fas) APO2.7 (Apoptosis) HLA-DR (Human leukocyte antigen – DR isotype). 73
HLA-DR Langerin CD95 (Fas) APO2.7 (Apoptosis) HLA-DR (Human leukocyte antigen – DR isotype). 73 73 73 73
Slide 74: TNF-α Induces Ocular Epithelial Cells to Express Pro-Inflammatory Markers
TNF-α Induces Ocular Epithelial Cells to Express Pro-Inflammatory Markers ** * 140000 120000 * P < 0.004 with non-Sjögren ** P < 0.0001 with normal 100000 ** ** Sjögren 80000 ** Non-Sjögren 60000 Normal eyes 40000 20000 0 HLA-DR CD40 HLA-DR expression is dependent CD40 belongs to the TNF receptor fa
Slide 75: OCS-02 Has a Dual MOA and is a Potent Inhibitor of TNF-α
Illustration of fragment technology scFv* IgG Fab (OCS-02) Regions that bind to neutralize TNF� Framework for OCS-02 optimized for stability Standard and other drug-like antibody properties frameworks OCS-02 (Licaminlimab) Has a Dual MOA and is a Potent Inhibitor of TNF-α DUAL MOA: ANTI-INFLAMMATORY
Slide 76: The Dual MOA of OCS-02 Targets Two Key Mechanisms of DED
S y s t e y m g r i e c l l d A r u g s O / s c s g e u l v u i a r t r a d s v l u r a e r s c g i e e r p r p o y T n i g r a L s e A m i t S i s I r d K a u i l / l r u r / e g g s f e e i r r a s r r c y a i t l i v a e h C D N G e M u / r s i o t The Dual MOA of OCS-02 Targets Two Key Mechanism
Slide 77: OCS-02 (Licaminlimab) Clinical Data to Date
OCS-02 (Licaminlimab) Clinical Data to Date Victor Perez, MD Bascom Palmer Eye Institute, Miller School of Medicine, University of Miami 77 77
Slide 78: Disclosures
Disclosures Victor Perez, MD, is Professor of Ophthalmology, Director of Cornea Research at Bascom Palmer Eye Institute at the University of Miami. Dr. Perez reports consultancy to Bausch & Lomb. Parion Sciences and Shire. Dr. Perez is a consultant for Oculis. 78 78 78 78
Slide 79: OCS-02 Positive Phase 2 Results in DED and Uveitis
OCS-02 (Licaminlimab) Positive Phase 2 Results in DED and Uveitis Focus of Today’s Presentation DED 1 DED 2 Uveitis * ESBA-105 OCS-02 OCS-02 1 Phase 2 PoC 2 3 Phase 2 PoC Phase 2 PoC 85 patients 131 patients 32 patients Primary endpoint achieved Primary endpoint achieved Demonstrated treatment effec
Slide 80: OCS-02 Phase 2a Trial in Patients with Severe DED
OCS-02 (Licaminlimab) Phase 2a Trial in Patients with Severe DED OBJECTIVES Phase 2a trial evaluating the efficacy, safety and tolerability of OCS-02 in reducing ocular symptoms in patients with severe DED TRIAL PRIMARY SECONDARY SAFETY DESIGN ENDPOINT ENDPOINT ASSESSMENTS Randomized, double-masked,
Slide 81: OCS-02 PoC Phase 2a: Trial Design
OCS-02 (Licaminlimab) PoC Phase 2a: Trial Design 2 weeks 4 weeks 6 weeks, randomized OCS-02 ophthalmic solution (TID) Vehicle (TID) Screening Patients were told they would be randomized to vehicle or OCS-02 Vehicle (TID) Patients who respond to artificial tears will not be randomized DED (Dry eye di
Slide 82: OCS-02 Phase 2a: Patient Disposition Through Study Completion
OCS-02 (Licaminlimab) Phase 2a: Patient Disposition Through Study Completion OCS-02 (N=69) Vehicle (N=65) Mean (±SD) age 61.7 years (±13.05) 58.8 years (±14.48) Sex 88.4% female 83.1% female Race 73.9% White 80.0% White Mean (±SD) baseline 77.9 (±13.89) 80.3 (±12.56) * ocular discomfort score Advers
Slide 83: OCS-02 Phase 2a: Significantly Improved DED Symptoms
OCS-02 (Licaminlimab) Phase 2a: Significantly Improved DED Symptoms Primary endpoint – Change from baseline in global ocular discomfort score* at Day 29 MEAN CHANGE IN GLOBAL OCULAR * DISCOMFORT SCORE AT DAY 29 OCS-02 (N=67) Vehicle (N=64) 0 -3.6 -2 (±1.49) OCS-02 statistically significantly reduced
Slide 84: OCS-02 Phase 2a: Treated Group Had Clinically Meaningful Results
OCS-02 (Licaminlimab) Phase 2a: Treated Group Had Clinically Meaningful Results Secondary endpoint – Percentage of high responder patients at Day 29 HIGH RESPONDER RATE P = 0.018 20 There was a greater percentage of high responder patients treated with OCS-02 17.9 % compared to vehicle at Day 29 (P
Slide 85: OCS-02 Phase 2a: Well Tolerated by Patients
OCS-02 (Licaminlimab) Phase 2a: Well Tolerated by Patients OCS-02 (n=69) Vehicle (n=65) Patients with at least one TEAEs, n (%) 13 (18.8%) 9 (13.8%) Related to study treatment 2 (2.9%) 2 (3.1%) Patients with any serious TEAEs, n (%) 0 (0%) 1* (1.5%) Deaths 0 (0%) 0 (0%) Nonfatal serious TEAE 0 (0%)
Slide 86: Consistent Results in Ocular Discomfort Improvement with ESBA105
Consistent Results in Ocular Discomfort Improvement with ESBA105* Rapid onset with significant change from baseline as of Day 14 (post hoc analysis) MEAN CHANGE FROM BASELINE ESBA105 Vehicle 6 N 56 29 4 Statistically significant 2 0 -2 -4 -6 -8 -10 -12 -14 -16 -18 -20 0 1 2 3 4 5 6 7 8 9 10 11 12 13
Slide 87: OCS-02 Positive Results Shows Potential as New Treatment Option
OCS-02 (Licaminlimab) Positive Results Shows Potential as New Treatment Option for Patients Suffering from DED ✓ Significantly reduced ocular discomfort ✓ Significantly greater percentage of high responder patients ✓ Rapid onset of action ✓ Well-tolerated with no unexpected adverse events reported P
Slide 88: Precision Medicine Potential in Dry Eye Disease
Precision Medicine Potential in Dry Eye Disease Anat Galor, MD, MSPH Bascom Palmer Eye Institute, Miller School of Medicine, University of Miami 88 88
Slide 89: Disclosures
Disclosures Anat Galor, MD, MSPH is a Professor of Ophthalmology, Bascom Palmer Eye Institute at the University of Miami. Dr. Galor reports consultancy to Alcon, Allergan, Brightstar MEM, B&L, Novaliq, Oyster Point, Shire and Tarsus. Dr. Galor is a consultant for Oculis. 89 89 89 89
Slide 90: Potential for Precision Medicine Approach to Predict Response
Potential for Precision Medicine Approach to Predict Response OCS-02 (Licaminlimab) Genetic Biomarker Assessment – a prespecified analysis in the Phase 2a trial OCS-02 demonstrated DED is multifactorial Precision medicine significant reduction of and has a heterogeneous is an innovative approach to
Slide 91: Pharmacogenetic Analysis to Identify Patient Biomarkers
Pharmacogenetic Analysis to Identify Patient Biomarkers Prespecified pharmacogenetics analysis in the OCS-02 (Licaminlimab) Phase 2a trial PHARMACOGENETIC ANALYSIS 86 vehicle and OCS-02–treated patients (n=43/group) Patient DNA isolated from whole blood was analyzed Patient samples screened for SNPs
Slide 92: Significant Association Between CC Gene-variant and Response
Significant Association Between CC Gene-variant and Response to OCS-02 (Licaminlimab) Treatment TNFRSF1A (TNF-α receptor 1) LS Mean Treatment Genotype SE (90% CI) P Change CC (n=4) -29.48 6.52 (-40.34, -18.61) OCS-02 CT (n=25) -0.09 3.52 (-6.01, 5.83) <0.0001 TT (n=14) -3.90 3.51 (-9.79,1.99) CC (n=
Slide 93: Genetic Biomarker May Be Associated with Treatment Response
Genetic Biomarker May Be Associated with Treatment Response CC genotype correlates with decreased inflammatory cytokines in the tear film Cytokine expression in OCS-02 (Licaminlimab) & vehicle groups IL-1β IL-8 TNF-α IL-1β mRNA Expression IL-8 mRNA Expression TNF-α mRNA Expression 300 60 40 30 240 4
Slide 94: SNP CC also Correlates with Response to Anti-TNF-α
SNP CC also Correlates with Response to Anti-TNF-α in Other Autoimmune / Inflammatory Diseases Table 1. Polymorphisms reported to influence TNF-i response 1,* Psoriatic arthritis, a chronic inflammatory in other autoimmune / inflammatory diseases disease, impacts joints in up to 30% of Drug Gene Rs
Slide 95: OCS-02 Results Are Promising for the Management of DED
OCS-02 (Licaminlimab) Results Are Promising for the Management of DED • Significantly reduced ocular discomfort Potential as a novel • Significantly greater percentage of high responder patients treatment option • Rapid onset of action for DED patients • Well-tolerated with no expected adverse event
Slide 96: RELIEF Phase 2b Ongoing Trial
RELIEF Phase 2b Ongoing Trial George Ousler, MS Senior Vice President, Anterior Segment Ora, Inc. 96 96
Slide 97: Disclosures
Disclosures George Ousler, MS is the Senior Vice President, Anterior Segment at Ora, Inc. 97 97 97 97
Slide 98: FDA Draft Guidance on Developing Drugs for Treatment of Dry Eye
FDA Draft Guidance on Developing Drugs for Treatment of Dry Eye TRIAL DESIGN CONSIDERATIONS SIGNS OF DED CAN INCLUDE: • Conjunctival staining • Safety and efficacy should be demonstrated in at least two • Corneal staining adequate and well-controlled, multicenter independent trials • Decreased Schir
Slide 99: OCS-02 RELIEF Phase 2b Trial
OCS-02 (Licaminlimab) RELIEF Phase 2b Trial Evaluating the safety and efficacy of OCS-02 for the treatment of moderate-to-severe DED Randomized Evaluation of Licaminlimab’s Efficacy and Safety for Dry Eye Disease SAFETY ENDPOINTS TRIAL DESIGN EFFICACY ENDPOINTS Randomized, double-masked, Staining, r
Slide 100: OCS-02 RELIEF Phase 2b Trial in Moderate-to-Severe DED
OCS-02 (Licaminlimab) RELIEF Phase 2b Trial in Moderate-to-Severe DED 2 weeks 2 weeks 6 weeks, randomized 2 weeks OCS-02 ophthalmic solution (TID) Follow-up Artificial tears (TID) Screening Run-in phase Vehicle (TID) Follow-up Patients who respond to artificial tears will not be randomized DED (Dry
Slide 101: RELIEF Program Key Updates
RELIEF Program Key Updates 2023 2024 December EXPECTED Q2 ’24 STUDY INITIATION Top line results First Patient First Visit Publications & presentations following top line results February PRESENT Randomization complete 10 101 1
Slide 102: Dry Eye Disease Q&A Session
Dry Eye Disease Q&A Session Dr. Pravin Dugel Dr. Riad Sherif
Slide 103: Closing Remarks
Closing Remarks
Slide 104: OCS-02 Potential to Become First anti-TNFα Eye Drop for DED and Uveitis
OCS-02 | Potential to Become First anti-TNFα Eye Drop for DED and Uveitis • Next generation eye drop targeting core inflammation in DED & Uveitis Novel anti-TNFα biologic eye drop for ocular • Anti-TNFα with clinically proven anti-inflammatory & inflammation anti-necrotic/apoptotic MOA 1,2 High unme
Slide 105: OCS-02 Potential to Transform Treatment of Moderate-to-Severe Dry Eye
OCS-02 | Potential to Transform Treatment of Moderate-to- Severe Dry Eye Patients with Novel Anti-TNF-α Eye Drop 2 2 20 million Moderate (35%) Severe (14%) diagnosed U.S. 1 DED patients 1,2 ~ 10 million diagnosed moderate-to-severe DED patients in the U.S. Current 3 Only 13% feel their chronic DED i
Slide 106: Positive OCS-01 Ph 3 DIAMOND-1 Stage 1 Results
Positive OCS-01 Ph 3 DIAMOND-1 Stage 1 Results Trial Results validated induction and maintenance regimen to optimize OCS-01 efficacy potential in DME with robust statistical significance Objectives Met OCS-01 demonstrated robust statistically significant improvement in vision and reduction in retina
Slide 107: OCS-01 Would Be Able to Address All Segments
OCS-01 | Would Be Able to Address All Segments while Leading 2 Segments Alone: Early Intervention and Treatment Customization Patient presents with DME with DME DME with moderate to severe 43% 33% 24% DME symptoms mild visual recent onset visual impairment impairment Diagnosed by OCT 1 1 1 DME Disea
Slide 108: OCS-01 Multi Program Asset for Front and Back of the Eye
OCS-01 | Multi Program Asset for Front and Back of the Eye DME, Ocular Surgery, and CME • Potential to be the first topical and non-invasive treatment for DME Transformative Eye Drop 1,2 • Total addressable U.S. patient population for DME ~1.3M • Two Phase 3 trials in DME (DIAMOND-1 and DIAMOND-2) M
Slide 109: Multiple Near-Term Value Inflection Points for All Three Assets
Multiple Near-Term Value Inflection Points for All Three Assets 2023 ACHIEVEMENTS Key corporate and clinical milestones: Advanced innovative pipeline development: ✓ NASDAQ listing and $146M raise✓ Initiated OCS-01 Phase 3 DME DIAMOND-1 trial ✓ Positive OCS-01 DME Phase 3 (Stage 1) ✓ Initiated OCS-01
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