27 September 2022 - - US-based biopharmaceutical company Ocular Therapeutix, Inc. (NASDAQ: OCUL) has released interim 7-month data from its US Phase 1 clinical trial evaluating OTX-TKI, the company's axitinib intravitreal hydrogel implant being developed for the treatment of wet age-related macular degeneration (wet AMD) and other retinal diseases, the company said.

The data will be presented in more detail at the upcoming American Academy of Ophthalmology 2022 annual meeting Subspecialty Day being held in Chicago by Dilsher Dhoot, MD on Friday, September 30th at 3:29 CT and can be accessed approximately two hours after the presentation's conclusion for 90 days by visiting the investors section of the company's website.

The US-based Phase 1 clinical trial is a prospective, multi-center, randomized, controlled study that is evaluating a 600 µg dose of OTX-TKI in a single implant, with a 2 mg aflibercept injection four weeks after the implant, compared to 2 mg aflibercept injections administered every 8 weeks in subjects previously treated with anti-VEGF therapy.

The trial is designed to assess the safety, durability and tolerability of OTX-TKI, and to assess biological activity in subjects by measuring visual acuity and anatomical changes of the retina using optical coherence tomography.

The clinical trial enrolled a total of 21 subjects at six clinical sites in the US, who were randomized 3: 1 to the OTX-TKI arm or the on-label every 8-week aflibercept injection arm.

Based on the data cutoff of August 24, 2022, interim data from the US-based Phase 1 clinical trial showed that the single OTX-TKI implant was generally well tolerated with a favorable safety profile.

There were no drug-related ocular or systemic serious adverse events observed. One SAE of endophthalmitis was observed in the OTK-TKI arm which occurred following the mandated aflibercept injection at Month 1 and assessed by the investigator as related to the injection procedure.

There were no reported adverse events such as elevated IOP, retinal detachment, retinal vasculitis, or implant migration into the anterior chamber observed in the OTX-TKI arm, and no subjects dropped out of either arm.

There was one subject randomized to the OTX-TKI arm who was incorrectly given aflibercept instead of sham injections at the subject's Month 3 and Month 5 visits.

These were not rescue treatments as the subject did not meet any rescue criteria.

Since this subject was not treated according to protocol, the subject has been excluded from the analysis of biological activity (OTX-TKI: n=15 and Aflibercept: n=5) but included in the safety analysis (OTX-TKI: n=16 and Aflibercept: n=5).

The interim results showed subjects treated with a single OTX-TKI implant demonstrated stable and sustained best corrected visual acuity (mean change from baseline of -1.3 letters) and central subfield thickness (mean change from baseline of +9.2 µm) in the OTX-TKI arm at 7 months, which was comparable with the aflibercept arm dosed every 8 weeks (mean change from BVCA baseline of -1 letter; mean change from CSFT baseline of +0.4 µm).

The data also showed that 80% of subjects in the OTX-TKI arm were rescue-free up to 6 months and 73% of subjects in the OTX-TKI arm were rescue-free up to 7 months. (Table 1). These data have not been reviewed by FDA.

Based on the positive data, the company plans to complete its analysis of the interim US Phase 1 data, meet with the FDA to discuss potential future clinical trial requirements and, subject to those discussions, initiate a Phase 2 wet AMD clinical trial in the third quarter of 2023.

The company also plans to follow subjects in the Phase 1 trial at least until their respective one-year anniversaries of initial dosing, in accordance with the clinical trial protocol.

Given the potential broad applicability of OTX-TKI to other retina diseases, the company also plans to initiate a US-based Phase 1 clinical trial to evaluate OTX-TKI for the treatment of diabetic retinopathy in the first quarter of 2023.

OTX-TKI is protected by a composition of matter patent, US Patent Number 11,439,592, with an expiration date of March 2041.

Additional US and foreign patent applications are pending.

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