Aiming to reduce the treatment burden, Ocular Therapeutix is focused on helping patients with retinal disease envision a brighter future.
According to the 2023 Retinal Pharmaceuticals Market Report, more than 410 million people around the world struggle with retinal disease. In the U.S., the most common retinal disorder that causes severe vision loss and blindness among older adults is wet age-related macular degeneration (wet AMD), which occurs when abnormal blood vessels beneath the retina leak fluid into the macula, the part of the eye responsible for central vision. If not effectively controlled, that fluid can cause swelling, vision distortion, and permanent damage to the retina—vision loss so profound it can impact a patient’s ability to participate in daily life.
Yet, despite tremendous medical advancements over the past two decades, the wet AMD patient experience remains stymied by substantial treatment burden, including regular injections that are hard for patients to maintain.
“A significant problem in retina today isn’t efficacy—it’s durability,” says Dr. Pravin Dugel, executive chairman, president, and CEO of Ocular Therapeutix, a biopharmaceutical company redefining the retina experience across drug development, treatment, and outcomes. “Therapies only work if patients can stay on them.”
While anti-vascular endothelial growth factor (VEGF) therapies transformed retinal disease management and can deliver extraordinary benefits to patients, real-world outcomes often fall short of clinical trial success stories because of these challenges. With current treatment regimens, 90% of patients require anti-VEGF injections every one to three months, but approximately 40% of those patients discontinue treatment within the first year. Ocular Therapeutix aims to change this.
“The issue is not whether anti-VEGF therapy works. It’s whether patients can remain on treatment long enough to benefit from it,” says Dugel. “The next generation of retinal therapies will increasingly be judged by their ability to reduce treatment burden while maintaining strong disease control.”
Unlike anti-VEGF treatments that target VEGF extracellularly, a new class of investigational product candidates under clinical development in retina, tyrosine kinase inhibitors (TKIs), block VEGF receptors intracellularly. This approach focuses on the receptor pathways responsible for angiogenesis and vascular leakage, two key drivers of retinal disease activity. And maybe most notably, if approved, could require less frequent injections.
Ocular is aiming to transform the treatment of retinal disease by focusing on reducing the therapeutic burden in the hope of preserving vision for the long term. “Every improvement that allows patients to remain on treatment and maintain disease control has implications not only for the patient, but also for their families, caregivers, and society as a whole,” says Dugel. “We already have therapies that can preserve vision. The question now is how we help more patients realize those benefits over years and even decades of treatment.”