We have the expertise and resources to redefine the retinal disease market

SIGNIFICANT MARKET OPPORTUNITY 

Retinal disease is one of the leading causes of blindness1,2

1.8 million people are living with wet age-related macular degeneration (AMD) in the U.S.3

Incremental improvements in durability have delivered substantial market opportunity 

The anti-VEGF market for wet AMD has grown from $0.4B to $15B in 20244

Significant need for more durable treatment remains

90% of patients currently require injections every 1-3 months5

40% of patients discontinue treatment in the first year due to this heavy treatment burden6

Frequent injections may result in decreased adherence and retinal fluid fluctuations, which are associated with poor long-term outcomes7

Aiming to redefine treatment

AXPAXLI™ (also known as OTX-TKI), an investigational axitinib hydrogel administered by intravitreal injection, combines a highly potent and selective tyrosine kinase inhibitor (TKI) with our proven proprietary hydrogel. Phase 1 data provided a compelling foundation to advance toward Phase 3, with the goal to dramatically improve durability and long-term outcomes for patients with wet AMD, and the potential to impact other retinal disease.8-12

AXPAXLI

An injection comprised of axitinib in a single hydrogel has the potential to provide continuous and consistent drug delivery for up to 12 months11,18

Nothing contained herein should be considered a solicitation, promotion or advertisement for any drug including the ones under development on this website. 

All investigational product candidates are currently undergoing clinical evaluation. This content is not intended to convey any conclusion of safety or efficacy, and there is no guarantee that any product candidate will successfully complete development or gain FDA approval or other regulatory authority approval. 

Redefining development

Complementary wet AMD registrational trials designed
to show durability, repeatability, and flexibility of AXPAXLI

Superiority Trial

Non Inferiority Trial

Patient selection

Randomization after anti-VEGF loading

Randomization after anti-VEGF loading

Trial design

Enable Q12 dosing on label

Enable superiority claim on label

Enable Q6M dosing on label

Shows efficacy compared to standard of care

FDA alignment

Special Protocol Assessment (SPA) agreement

Type C, other written responses

Superiority Trial

Non Inferiority Trial

Patient selection

Randomization after anti-VEGF loading

Randomization after anti-VEGF loading

Trial design

Enable Q12 dosing on label

Enable superiority claim on label

Enable Q6M dosing on label

Shows efficacy compared to standard of care

FDA alignment

Special Protocol Assessment (SPA) agreement

Type C written response

Redefining outcomes

AXPAXLI has expansive market potential with a target retinal disease population of 10.4 million in the U.S. alone3

Registrational Trials Ongoing

1.8M

Patients with Wet Age-Related Macular Degeneration3

Registrational Trials in Planning   

2.8M

Patients with Moderate-Severe Non-Proliferative Diabetic Retinopathy3

1.7M

Patients with
Diabetic Macular Edema3

FUTURE OPPORTUNITIES

1.5M

Patients with Retinal Vein Occlusion3

2.6M

Patients with Proliferative Diabetic Retinopathy3

Each dot represents ~24,000 patients

RETINA EXPERIENCE

Our team has unmatched expertise and an impressive track record of innovation in retinal disease

> 0

clinical trials designed or led by our team 

> 0

combined years of treating patients with retinal disease

> 0

treatment launches
across retinal disease  

References

1. Wang W, et al. Int J Mol Sci. 2018;19(6):1816. 2. Di Carlo E, et al. J Clin Med. 2021;10(15):3297. 3. Market Scope. 2024 Retinal Pharmaceuticals Market Report: Wet AMD and Other Exudative Diseases. St. Louis, MO: Market Scope, LLC. 2024. 4. Data on file 01892. Anti-VEGF Revenue. Ocular Therapeutix, Inc. 5. Market Scope. Ophthalmic Market Trends: Quarterly US Retina Edition. St. Louis, MO: Market Scope, LLC. 2025. 6. Khanani AM, et al. Ophthalmology Retina. 2020;4(2):122-133. 7. Evans RN, et al. JAMA Ophthalmol. 2020;138(10):1109. 8. Zhao Y, et al. Oncologist. 2015;20(6):660-673. 9. Gross-Goupil M, et al. Clin Med Insights Oncol. 2013;7:269-277. 10. Liang C, et al. Mol Ther Oncolytics. 2022;24:577-584. 11. Blizzard CD, Inventors, Ocular Therapeutix, Inc., Assignee. Ocular implant containing a tyrosine kinase inhibitor. US Patent 11,439,592 B2. September 13, 2022. 12. Tyson SL, et al. J Cataract Refract Surg. 2019;45(2):204-212. 13. Sawhney AS, et al., Inventors, Incept, LLC, Assignee. Drug delivery through hydrogel plugs. US Patent 8,409,606 B2. April 2, 2013. 14. Blizzard C, et al. Clin Ophthalmol. 2021:15 2055–2061. 15. Boyer DS, et al. Evaluating Safety, Tolerability and Biological Activity of OTX-TKI, a Hydrogel-Based, Sustained-Release Intravitreal Axitinib Implant, in Subjects with Neovascular Age-Related Macular Degeneration. Presented at: American Academy of Ophthalmology Annual Meeting; November 13-15, 2020; Virtual. 16. Goldstein MH, et al. Invest Ophthalmol Vis Sci. 2020;61(7):4266. 17. McGrath M, et al. Invest Ophthalmol Vis Sci. 2014;55:472. 18. Data on file DOF 030. Ocular Therapeutix, Inc.

AXITINIB targets

AXITINIB is a highly potent and selective small molecule pan-VEGFR inhibitor 9-11 

ELUTYXTM delivers

ELUTYX is a fully bioresorbable, clinically established hydrogel platform that is designed for sustained and targeted drug delivery, aiming to maximize efficacy to provide optimal disease control12-17