OTX-TKI (axitinib intravitreal implant)

SUSTAINED-RELEASE PRODUCT CANDIDATE FOR RETINAL DISEASES

OTX-TKI is an investigational bioresorbable, hydrogel implant incorporating axitinib, a small molecule tyrosine kinase inhibitor with anti-angiogenic properties being evaluated for the treatment of wet age-related macular degeneration (wet AMD) and other retinal diseases.#

Existing Treatments

  • Require intravitreal injections every 4-8 weeks1,2
  • May cause endophthalmitis, ocular hemorrhage, damage to the lens or retinal detachment due to repeated injections3,4
  • May cause discomfort, eye pain, decreased vision, and floaters3

Product Candidate Attributes

  • Targeting sustained release for 6 months or longer
  • Broad anti-angiogenic profile (small molecule)
  • Small fiber (25-27G needle) with minimal/no visual impact
  • Preservative-free

Phase 1 Clinical Trial currently Ongoing

Plan to initiate mid-2021:

  • US Phase 1 clinical trial
  • Ex-US Phase 2 clinical trial

Caution: NEW DRUG – OTX-TKI is currently undergoing clinical evaluation and is limited by law to investigational use only. This product has not been approved by the FDA as safe or effective.

OTX-TKI fiber to rod. Video shown in real time in simulated vitreous humor.

“We are particularly encouraged with new interim Phase 1 data in OTX-TKI that supports the product’s safety profile and demonstrates evidence of biological activity in patients with wet AMD across all three dose groups.”

– Michael Goldstein, President, Ophthalmology and CMO – 11/11/2020

About Wet Age-Related Macular Degeneration

Age-related macular degeneration (AMD) is a leading cause of severe, irreversible vision loss.5 In the United States, the estimated number of people with AMD is expected to more than double from 2.07 million in 2010 to 5.44 million by 2050.5 The most common form of AMD is the non-exudative or dry form, which afflicts most patients and represents the early and intermediate stages of the disease.6 As the disease progresses, approximately 15% of patients will develop the more advanced neovascular (wet) form of AMD.6 Neovascular AMD causes vision loss due to abnormal new blood vessel growth and hyperpermeability and associated retinal vascular in the macula, which is primarily stimulated by local upregulation of vascular endothelial growth factor (VEGF).7 Without prompt and continuous treatment to control this exudative activity, patients develop irreversible vision loss.6 With proper treatment, patients may maintain visual function for a period of time and may temporarily regain lost vision.8 Challenges with current therapies include repeated intraocular injections every 1-2 months, treatment-related adverse events, patient compliance, and lack of vision improvement.9

Intracameral injection of OTX-TKI implant (image)

Intravitreal injection of OTX-TKI implant

Relative size of OTX-TKI implant (image of implant with a penny)

Relative size of OTX-TKI implant

REFERENCES: 1. EYLEA Full Prescribing information 2018 2. Lucentis full Prescribing Information 2019 3. Bochot A, Fattal E. Liposomes for intravitreal drug delivery: a state of the art. J Control Release. 2012;161(2):628-634. 4. Falavarjani KG, Nguyen QD. Adverse events and complications associated with intravitreal injection of anti-VEGF agents: a review of literature. Eye (Lond). 2013 Jul;27(7):787-94. doi: 10.1038/eye.2013.107. Epub 2013 May 31. PMID: 23722722; PMCID: PMC3709385. 5. National Eye Institute. Age-Related Macular Degeneration (AMD) Data and Statistics, 2019. Available: https://www.nei.nih.gov/learn-about-eye-health/resources-for-health-educators/eye-health-data-and-statistics/age-related-macular-degeneration-amd-data-and-statistics. Accessed: 22 January 2021. 6. Gehrs KM, Anderson DH, Johnson LV, Hageman GS. Age-related macular degeneration–emerging pathogenetic and therapeutic concepts. Ann Med. 2006;38:450-471. 7. Kvanta A, Algvere PV, Berglin L, Seregard S. Subfoveal fibrovascular membranes in age-related macular degeneration express vascular endothelial growth factor. Invest Ophthalmol Vis Sci. 1996;37:1929-1934. 8. Comparison of Age-related Macular Degeneration Treatments Trials (CATT) Research Group, Maguire MG, Martin DF, et al. Five-Year Outcomes with Anti-Vascular Endothelial Growth Factor Treatment of Neovascular Age-Related Macular Degeneration: The Comparison of Age-Related Macular Degeneration Treatments Trials. Ophthalmology. 2016;123(8):1751-1761. doi:10.1016/j.ophtha.2016.03.045. 9. Okada M, Mitchell P, Finger RP, et al. Nonadherence or nonpersistence to intravitreal injection therapy for neovascular age-related macular degeneration: a mixed-methods systematic review. Ophthalmology. 2021;12:234-247.