Ocular Therapeutix outlined the clinical progress across its registrational program for Axpaxli in wet age-related macular degeneration (wet AMD), plans to advance Axpaxli in non-proliferative diabetic retinopathy (NPDR), and the company’s strategic outlook for 2025 in its presentation at the 43rd Annual J.P. Morgan Healthcare Conference (JPM 2025).

“Wet AMD is the leading cause of blindness in the United States, but it does not need to be. Diabetic retinopathy is the leading cause of blindness in the working population in the US, but it does not need to be. We have a proven target, VEGF, and proven treatments for decades, the anti-VEGFs. Unfortunately, we also have a treatment regimen that is simply not sustainable for many patients. Our mission at Ocular is to correct this,” said Pravin U. Dugel, MD, executive chairman, president and CEO of Ocular Therapeutix, in a press release.

Dr. Dugel continued, “Last year, we started with wet AMD. We recruited SOL-1 well ahead of schedule and, yesterday at JPM 2025, I announced that we have already enrolled 311 patients in various stages of loading and randomization in SOL-R, our second registrational study in wet AMD. The historic pace of recruitment in both studies underscores the enthusiasm in the retina community for Axpaxli. In 2025, we will also target diabetic retinopathy. Our HELIOS study has demonstrated that a single injection of Axpaxli has the potential to reduce the risk of vision loss in NPDR to literally zero at 48 weeks. In HELIOS, every single patient with non-center involved diabetic macular edema (DME) treated with a single injection of Axpaxli, improved at week 48. Therefore, Axpaxli has the potential to not only prevent vision loss, but also to treat existing vision threatening complications, such as DME.”

Dr. Dugel concluded, “In 2025, Ocular will target the two most impactful global diseases in retina, wet AMD and diabetic retinopathy. We believe we have the infrastructure, finances and expertise to succeed and redefine the global retina experience.”

Highlights from the J.P. Morgan presentation included: 

• Complementary wet AMD registrational program. Ocular’s wet AMD registrational program for Axpaxli is comprised of two complementary studies, SOL-1 and SOL-R, strategically designed with the intent of de-risking patient populations, aligning with regulatory standards, enhancing each other’s enrollment, and providing a broad evaluation of Axpaxli's durability, repeatability, and flexibility. SOL-1 is being conducted under a Special Protocol Agreement (SPA) with the FDA, while SOL-R received alignment with the FDA through a written Type C response. The FDA previously agreed that together, these studies could constitute 2 adequate and well-controlled trials to support a potential new drug application and label for Axpaxli in wet AMD.
• SOL-1 (phase 3, wet AMD) randomization complete in December 2024. In total, more than 300 subjects were randomized across greater than 100 clinical trial sites located in the US and Argentina. SOL-1 is a superiority trial comparing a single Axpaxli injection to a single aflibercept (2 mg) injection in treatment naïve wet AMD subjects with a 9-month primary endpoint and up to 2-year follow-up. SOL-1 is the first registrational trial for Axpaxli in wet AMD and was designed to establish Axpaxli's durability, and potentially enable a superiority claim on a potential future label. The company expects topline results for SOL-1 to be available in the fourth quarter of 2025.
• SOL-R (phase 3, wet AMD) outstanding enrollment progress to date. 311 subjects enrolled across various stages of loading and randomization in the US and South America as of Jan. 10, 2024. SOL-R is a non-inferiority trial comparing repeat Axpaxli injections every 6 months to repeat aflibercept (2 mg) injections every 8 weeks, with a 56-week primary endpoint. SOL-R is the second registrational trial for Axpaxli in wet AMD and was designed to inform real world treatment decisions, establish Axpaxli's safety and efficacy with repeat dosing, and provide commercially relevant data.
• Ocular plans to seek FDA feedback in H1 2025 on the clinical trial design for Axpaxli in NPDR. Following positive results from the phase 1 HELIOS trial of Axpaxli shared in 2024, Ocular plans to continue clinical development in NPDR. In the HELIOS trial, after a single Axpaxli injection, no patients receiving Axpaxli (N=13) developed a vision threatening complication (VTC) at 48 weeks compared to nearly 40% in the sham-treated patients (N= 8). Further, all patients in the Axpaxli arm who presented with non-center-involved diabetic macular edema (non-CI-DME) at baseline (N=8) had improvement in their DME based on optical coherence tomography (OCT) image analysis at week 48 compared to none of the sham-treated subjects (N=3). All subjects who received Axpaxli showed diabetic retinopathy severity scale (DRSS) improvement or stability, while any worsening of DRSS occurred only in sham-treated subjects.

Arshad M. Khanani, MD, MA, FASRS, director of clinic research at Sierra Eye Associates, Reno, NV, noted in the press release, “Real-world evidence shows that 40 to 50% of patients with wet AMD discontinue their injections due to the high treatment burden, putting them at increased risk of blindness. This issue is even more pronounced in patients with diabetic retinopathy. My team, our patients, and I are excited to support the SOL studies, and delighted that Ocular will soon target diabetic retinopathy, a huge unmet need in our space. If approved, I believe Axpaxli will be rapidly adopted, as it has the potential to significantly reduce treatment burden with a durability of 6-12 months."

Patricio G. Schlottmann, MD, director of the research department at the Charles Ophthalmic Center and ophthalmology department director at Organización Médica de Investigación in Buenos Aires, Argentina, also added, “Wet AMD and diabetic retinopathy are global diseases in which millions of patients around the world lose vision because we have an unsustainable treatment regimen. I continue to enthusiastically support the SOL programs in wet AMD and am particularly happy that Ocular will also target diabetic retinopathy. If approved, I believe Axpaxli will be enthusiastically adopted and has the potential to positively impact millions of patients around the world.”