The FDA grants the company agreement under a Special Protocol Assessment to evaluate TRS01 ophthalmic solution for the treatment of non-infectious uveitis, including uveitic glaucoma.
Tarsier Pharma has received agreement from the US Food and Drug Administration (FDA) under a Special Protocol Assessment (SPA) for the Tarsier-04 phase 3 trial investigating TRS01 eye drops (Dazdotuftide) for the treatment of non-infectious uveitis, including uveitic glaucoma.1
Announced on January 16, 2024, Tarsier has received written agreement from the FDA that the clinical trial protocol and planned statistical analysis of the Tarsier-04 phase 3 trial will address objectives supporting regulatory submission. An SPA agreement with the federal health agency signals concurrence with the adequacy and acceptability of specific elements of the overall protocol design for a study intended to support a future marketing application.
“The SPA agreement with the FDA provides a clear regulatory path for TRS01,” Daphne Haim-Langford, PhD, founder and CEO of Tarsier, said in a statement.1 "The approved protocol of the upcoming Tarsier-04 trial was designed to replicate the TRS4Vision trial, with revised endpoints that are aimed to emphasize the benefit/risk profile of TRS01, based on lessons learned from the TRS4Vision trial."
Tarsier-04 is designed as a multi-center, randomized, double-masked, active-controlled phase 3 trial evaluating the efficacy and safety of TRS01 eye drops, with a plan to enroll up to 300 patients in the US with non-infectious uveitis, including patients with uveitic glaucoma. The primary endpoint in the indicated Tarsier-04 protocol achieved a strong result in the TRS4Vision trial, with the company indicating their belief the endpoint of the phase 3 trial will be met.
“The primary endpoint in this FDA-approved protocol is one that achieved an alpha <0.01 in the TRS4Vision trial, thus we have strong reasons to believe that the endpoints of Tarsier-04 will be met, and this SPA agreement with the FDA is bringing TRS01 much closer to patients in need,” Haim-Langford said.1
TRS01, or dazdotuftide, is a first-in-class drug designed for the treatment of ocular blinding diseases of the back- and front- of the eye. Dazdotuftide is a bio-inspired novel proprietary technology platform and has been found to modulate macrophages from inflammatory macrophages (M1) to IL-10-secreting anti-inflammatory macrophages (M2).
The company is developing the drug as an eye drop formulation (TRS01) for front-of-the-eye indications and a slow-release biodegradable intravitreal injection (TRS02) for back-of-the-eye blinding indications, including diabetic macular edema (DME) and non-proliferative diabetic retinopathy. The company indicated the drug’s unique mechanism of action of immunomodulation suggests the potential to treat debilitating eye diseases with sustained effect and minimal risk of intraocular pressure elevation.
Non-infectious uveitis is the third leading cause of blindness in the US – despite available treatment options, nearly two-thirds of patients with the disease reach visual impairment and 22% meet legal blindness criteria.2 Current treatment methods involve topical steroids to resolve inflammation, but the use of topical steroids carries a high risk of IOP elevation and subsequently, a risk of uveitic glaucoma.
“After over 40 years of treating non-infectious uveitis and actively participating in efforts to develop new therapies, drawing confidence from the positive outcomes of the TRS4Vision trial, I'm optimistic that we're closer than ever to controlling inflammation without increasing the risk of glaucoma and that TRS01 can serve as a new first-line-treatment for non-infectious uveitis replacing topical steroids,” Alan Palestine, MD, director of the center for ocular inflammation at the University of Colorado department of ophthalmology, said in a statement.1
Tarsier has indicated it is focused on ramping up preparations for the trial start-up activities.