Ocular Therapeutix, Inc. (NASDAQ:OCUL, "Ocular"))), an integrated biopharmaceutical company committed to redefining the retina experience, today announced additional positive Week 52 data from the SOL-1 Phase 3 superiority trial of AXPAXLI (also known as OTX-TKI), its investigational product candidate for the treatment of wet age-related macular degeneration (wet AMD). The additional post-hoc analyses, presented at the 14th Annual Vit-Buckle Society (VBS) Meeting, reinforce the superior durability, strong sustained disease control, and generally well-tolerated safety seen with AXPAXLI in SOL-1.

"The new analyses of the SOL-1 Phase 3 data further strengthen our conviction in AXPAXLI's potential to redefine retina treatment. A clear drug profile has emerged for AXPAXLI: a product candidate with durability that is unmatched in the wet AMD space while demonstrating excellent sustained disease control," said Pravin U. Dugel, MD, Executive Chairman, President and Chief Executive Officer of Ocular Therapeutix. "Several analyses presented at VBS reinforce this profile. As retina specialists, anatomic control measured with OCT guides our treatment decisions. The time-to-CSFT increase analyses provide powerful additional evidence of AXPAXLI's efficacy. After Week 8, it takes a median of 39 weeks to reach a clinically meaningful increase of ≥30 uM in CSFT, and even longer to reach ≥75 uM. This is simply an unprecedented level of sustained disease control and allows us to re-imagine the management of wet AMD for patients. These data continue to increase our confidence that AXPAXLI may be adopted broadly and immediately, if approved. Most importantly, we remain on track to submit our NDA based on SOL-1 alone, subject to planned formal U.S. FDA discussions. The FDA Commissioner recently noted that the Agency expects this new default framework for approval based on a single pivotal trial to be phased in over the next six months or so, aligning with our goal of bringing AXPAXLI to patients as soon as possible."

Highlights from the data presented at VBS include:

  • Strong overall efficacy profile with unmatched durability: Achieved statistical significance in the first three of five key secondary endpoints tested in hierarchical order. In addition, six other pre-specified secondary endpoints measuring clinically significant functional and anatomic outcomes were met with statistical significance.
  • Sustained disease control (post-hoc analysis): Subjects in the AXPAXLI arm had significantly lower risk in anatomic worsening from Week 8 compared to the aflibercept (2 mg) arm. Week 8 was chosen to give both arms adequate time from the last aflibercept (2 mg) injection and start at a similar reference timepoint.
    • The median time to ≥30 uM Central Subfield Thickness (CSFT) increase from Week 8 was 39 weeks in the AXPAXLI group and 16 weeks in the aflibercept (2 mg) group, a 23-week difference. An estimated hazard ratio of 0.7 indicates that, at any time from Week 8 to Week 52, subjects in the treatment group experienced a 30% lower risk of the event (descriptive p=0.0028) compared with the control group.
    • The median time to ≥75 uM CSFT increase from Week 8 was 46 weeks in the AXPAXLI group and 24 weeks in the aflibercept (2 mg) group, a 22-week difference. An estimated hazard ratio of 0.5 indicates that, at any time from Week 8 to Week 52, subjects in the treatment group experienced a 50% lower risk of the event (descriptive p<0.0001) compared with the control group.
  • Sustained visual outcomes (post-hoc analysis): Visual acuity gains achieved during the loading phase were generally maintained up to Week 52 with AXPAXLI across screening BCVA quartile subgroups. The magnitude of vision improvements was influenced by BCVA at screening. For example, AXPAXLI subjects in the lowest vision quartile group at screening had the greatest visual gains with +11.8 ETDRS letters compared to +8.5 letters in the aflibercept (2 mg) arm at Week 52. The mean vision at screening in this arm is similar to baseline BCVA in prior wet AMD studies. In comparison, subjects in the best vision quartile at screening had essentially no change in vision at Week 52 (-0.5 vs +1.1 letters; AXPAXLI vs aflibercept) as they started with almost 20/20 vision.
  • Sustained visual outcomes in rescue-free subjects (post-hoc analysis): Observed difference for change in BCVA from baseline at Week 24 in the rescue-free subjects was -1.9 ETDRS letters in the AXPAXLI arm (+7.5 letters from screening) vs -2.6 letters in the aflibercept arm (+6.0 letters from screening). Of note, 81% of the AXPAXLI subjects were rescue-free at Week 24. At Week 36, 75% of AXPAXLI subjects remained rescue-free and lost <1 additional letter from Week 24 (+6.6 letters from screening).
  • Well-tolerated profile: For all AXPAXLI subjects with a vitreous floater adverse event reported, drug particles are no longer visible (mean time of 20 weeks). Further analysis continues to illustrate the appearance of floaters corresponds closely to expected hydrogel bioresorption and drug elution without adversely affecting vision.