Shares of Ocular Therapeutix Inc. (OCUL) got a little boost Monday after the company dropped some fresh, positive numbers. We're talking about the long-term data from its big Phase 3 trial for Axpaxli, an experimental treatment for wet age-related macular degeneration (AMD). For a company that's all about retinal diseases, this is the main event.
Now, you might remember that back in February, the initial data from this same trial—the SOL-1 study—was a bit of a mixed bag for investors. It showed Axpaxli worked, but maybe not as spectacularly as some had hoped. The new data, looking out to a full year, tells a more complete and arguably more compelling story about what this drug can do.
The headline here is about holding the line. The new analysis shows that for patients taking Axpaxli, the disease progressed more slowly. Specifically, their risk of what's called "anatomic worsening"—basically, physical damage getting worse in the back of the eye—was a lot lower than for patients getting the current standard treatment, Regeneron Pharmaceutical Inc.'s (REGN) Eylea (aflibercept).
Let's put some numbers on that. The median time until patients saw a concerning increase in retinal thickness was 39 weeks for the Axpaxli group. For the Eylea group, it was just 16 weeks. That's a 23-week difference. The math suggests patients on Axpaxli had a 30% lower risk of that kind of worsening happening between weeks 8 and 52 of the study. For more severe progression, the risk was cut in half.
On the vision front, the gains patients made early on in the trial mostly stuck around through the one-year mark. The people who started with the worst vision actually saw the biggest benefit, gaining nearly 12 letters on an eye chart compared to about 8.5 letters for those on Eylea. Patients who started with near-normal vision, as you'd expect, didn't see much change either way.
The data also showed consistency. Among patients who didn't need any extra "rescue" therapy during the study, outcomes remained stable. At the six-month mark, 81% were rescue-free and had gained about 7.5 letters in vision. By nine months, 75% were still going strong without rescue, holding onto most of that improvement.
All of this adds up to what the company calls a "strong efficacy profile." The drug hit statistical significance on several key secondary goals and met a bunch of other pre-specified endpoints related to how well patients could see and the physical state of their eyes. It's the kind of thorough data package you want to have when you walk into the FDA's office.
Speaking of which, Ocular says it's still on track to submit a New Drug Application for Axpaxli. The latest figures seem designed to bolster that case, showing not just that the drug works, but that it provides a durable defense against the disease getting worse over time.
Ocular Therapeutix shares were up 0.63% at $8.84 at the time of publication.











