Ocular Therapeutix, Inc. announced patient enrollment has concluded for its phase 1 clinical trial assessing OTX-TKI for the treatment of non-proliferative diabetic retinopathy (NPDR).
Refresh me on OTX-TKI.
OTX-TKI is an investigational, bioresorbable hydrogel intravitreal implant with anti-angiogenic properties and designed to continuously deliver axitinib (a potent tyrosine kinase inhibitor [TKI]) for DR.
To note, it is also being developed for the potential treatment of wet age-related macular degeneration (AMD) and other vascular endothelial growth factor (VEGF)-mediated retinal diseases.
OTX-TKI for wet AMD sounds familiar …
It should! The company recently reported 12-month topline data from its phase 1 study (NCT04989699) assessing OTX-TKI for wet AMD.
See here for the findings.
Gotcha. So how is the implant delivered?
The intravitreal implant delivers axitinib via a 25G needle and using the company’s proprietary Elutyx technology platform—a bioresorbable polymer matrix hydrogel developed to provide localized, sustained drug delivery.
What’s the target release time?
According to Ocular Therapeutics: 9 to 12 months.
Talk about this trial
The multicenter, prospective, double-masked, randomized, parallel group phase 1 HELIOS trial (NCT05695417) has enrolled 21 patients across the United States.
Participants are being randomized 2:1 to receive either 600 µg of OTX-TKI via a single implant with axitinib or sham injections.
Give me more on patient criteria.
Patients are 18+ years of age with a diagnosis of type 1 or type 2 diabetes and moderately severe to severe NPDR in the study eye (Diabetic Retinopathy Severity Scale [DRSS] levels of 47 or 53, as determined by a reading center).
Their study eye’s best-corrected visual acuity (BCVA) Early Treatment Diabetic Retinopathy Study (ETDRS) letter score must also be ≥69 letters (approximate Snellen equivalent of 20/40 or better).
What’s being measured?
Primary outcomes are the safety, durability, and tolerability of OTX-TKI through 12 months.
Secondary outcomes include, from baseline up to Week 52: :
- Changes in the BCVA, as determined by ETDRS letter score
- Changes in central subfield thickness (CSFT)
- Proportion of patients receiving rescue therapy
- Changes in the DRSS
When can we expect data?
The company expects to report interim 6-month data by Q1 2024.
So what’s next?
Pending favorable interim data and acquiring the necessary funding, according to Ocular Therapeutix, a phase 3 pivotal trial for DR could potentially launch by early 2024.
Significance?
In the words of the company’s CMO Rabia Gurses Ozden, MD: “We believe the same attributes that make OTX-TKI a compelling product candidate for the treatment of wet AMD—the ease of use of an office-based injection and the potential for long-term durability that reduces the treatment burden and need for frequent, invasive injections—could establish OTX-TKI as the standard of care in the treatment of diabetic retinopathy.”