NexThera Begins U.S. Trial with First Patient Dosed for Wet AMD Eye Drop NT-101
NexThera Co., Ltd., a biotechnology company at the forefront of ophthalmic innovation, announced a pivotal milestone in its drug development journey: the initiation of a Phase 1/2a clinical trial for its investigational eye drop therapy, NT-101, for the treatment of wet age-related macular degeneration (wet AMD). The first patient was successfully enrolled on April 8, 2025, at a trial site in Pennsylvania, marking the official commencement of a clinical study that could significantly reshape the standard of care for retinal diseases.
This trial is particularly notable because NT-101 is the inaugural clinical asset developed using NexThera’s proprietary eye drop-based drug delivery platform—an advanced formulation technology designed to non-invasively transport therapeutics to the deepest regions of the eye, specifically the retinal tissue. By circumventing the need for intravitreal injections, which are currently the norm for wet AMD treatment, NT-101 promises to provide a safer, more convenient, and patient-friendly alternative.
Breaking Away from the Needle: A New Paradigm in Retinal Drug Delivery
Traditionally, the management of wet AMD—a chronic, vision-threatening condition characterized by abnormal blood vessel growth beneath the retina—has relied heavily on intravitreal injections of anti-VEGF (vascular endothelial growth factor) agents. These biologics inhibit the formation of new blood vessels that can leak fluid and blood into the retina, leading to vision loss. While effective in controlling disease progression, anti-VEGF therapy requires recurrent injections into the vitreous cavity of the eye every 1–3 months, often indefinitely.
This burdensome regimen is associated not only with high treatment costs and logistical challenges but also with significant patient discomfort, risk of infection, retinal detachment, and other adverse effects. Moreover, there is growing concern over the diminishing efficacy of anti-VEGF agents in certain patient populations over time, which further highlights the urgent need for new treatment modalities.
NexThera’s NT-101 aims to eliminate these pain points. Leveraging a unique combination of intrinsic peptides and engineered carrier proteins, the formulation facilitates targeted drug delivery directly to the retina via topical administration. The drug is applied as an eye drop—non-invasive, easy to self-administer, and amenable to frequent dosing without the complications of needle-based delivery.
Scientific Innovation: The Mechanism Behind NT-101
The science behind NT-101’s delivery system is rooted in biomimetic principles. Its active pharmaceutical ingredients include proprietary peptides that target angiogenic pathways implicated in wet AMD, including but not limited to VEGF signaling. These peptides are formulated alongside carrier proteins designed to enhance ocular surface retention and facilitate trans-corneal and posterior segment penetration.
In preclinical studies, the combination of these components demonstrated not only effective delivery to the retinal tissue but also therapeutic activity that was comparable, if not superior, to that of existing monoclonal antibodies and fusion proteins used in current wet AMD therapies. The results suggest that NT-101’s dual mechanism—angiogenesis inhibition and neuroprotection—may offer comprehensive disease control by both halting vision-threatening neovascularization and preserving optic nerve integrity.
This multi-targeted approach represents a shift from traditional mono-target therapies and positions NT-101 as a next-generation treatment option in the field of retinal medicine.
Phase 1/2a Trial Design and Objectives
The Phase 1/2a trial, now underway, will enroll a total of 30 patients diagnosed with wet AMD across four prominent clinical sites located in Pennsylvania, Maryland, North Carolina, and Texas. Each site is staffed by experienced investigators with deep expertise in ophthalmic diseases and a strong track record in conducting rigorous, multi-modal clinical trials.
The study is designed to evaluate both the safety and preliminary efficacy of NT-101. It follows a parallel-cohort design, where enrolled participants will be randomized to receive either a low or high dose of NT-101 eye drops. The dosing schedule includes twice-daily administration—once in the morning and once in the evening—for a period of four weeks, followed by an observation phase of an additional four weeks, making the total study duration eight weeks per participant.
Key endpoints for this study include:
- Safety and tolerability, assessed through adverse event reporting, ocular examinations, and laboratory analyses.
- Central subfield thickness (CST), a key biomarker for fluid accumulation and disease activity in the macula.
- Best-corrected visual acuity (BCVA), a critical clinical metric used to evaluate changes in central vision.
While this is an early-stage trial, the data collected will lay the groundwork for larger, more definitive studies in the future, and will help inform optimal dosing, treatment duration, and efficacy parameters.
A Potential Game Changer in Retinal Care
NexThera’s pursuit of a non-invasive treatment for wet AMD stems from its long-term commitment to revolutionize ocular drug delivery and improve patient outcomes. The company believes that NT-101, if successful, could significantly reduce the treatment burden associated with intravitreal injections, not only by eliminating procedural risks but also by offering a cost-effective and scalable solution.
From a health economics standpoint, NT-101 could alleviate both direct and indirect costs associated with frequent clinic visits, transportation, lost productivity, and long-term management of injection-related complications. For aging populations around the world, many of whom are affected by AMD and struggle with access to specialized care, a self-administered topical therapy could greatly improve quality of life and adherence to treatment.
Moreover, NexThera envisions broader applications for its proprietary delivery platform, which may be adaptable to other retinal diseases such as diabetic macular edema (DME), retinal vein occlusion (RVO), and geographic atrophy. This could create a pipeline of novel therapies that follow the same needle-free model.
Industry Implications and Future Outlook
As the global ophthalmology market continues to grow—driven by aging demographics, rising prevalence of chronic eye diseases, and technological innovation—the need for disruptive therapeutic approaches is becoming increasingly evident. NexThera’s NT-101 represents a potential leap forward not only for wet AMD patients but also for the broader pharmaceutical and biotech sectors, where the transition from invasive to non-invasive therapies is a key area of interest.
With the launch of this Phase 1/2a clinical trial, NexThera is sending a clear signal to the industry: the future of retinal treatment may lie not in the needle, but in the dropper.
CEO SaeGwang Park expressed optimism about the trial’s potential, stating, “The initiation of this first-in-human study for NT-101 is a major milestone for NexThera. We are confident in the science and look forward to sharing clinical results that could pave the way for a paradigm shift in how we treat serious retinal diseases like wet AMD.”
As NT-101 enters clinical evaluation, all eyes will be on NexThera and its novel platform. If the drug proves safe and effective in human trials, it could usher in a new era in ophthalmology—one where powerful biologics can be delivered painlessly, efficiently, and affordably, right from the comfort of a patient’s home.
NexThera’s continued investment in R&D and its vision for transformative ocular therapies position it as a rising player in the field. The outcomes of this trial will be closely watched not only by patients and clinicians, but also by potential partners and investors seeking to support the next big innovation in eye care.
