Tarsier Pharma receives FDA agreement under Special Protocol Assessment for Tarsier-04 Phase 3 Trial

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Tarsier Pharma Ltd announced it has received agreement from the FDA under a Special Protocol Assessment (SPA) for the clinical trial protocol and planned statistical analysis of the Tarsier-04 Phase 3 trial to evaluate TRS01 eye drops for the treatment of non-infectious uveitis including uveitic glaucoma.

A SPA agreement indicates concurrence by FDA with the adequacy and acceptability of specific critical elements of overall protocol design for a study intended to support a future marketing application.

Daphne Haim-Langford, PhD, founder and CEO of Tarsier, noted in the company’s news release the SPA agreement with the FDA provides a clear regulatory path for TRS01.

“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,” she said in the release. “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.”

Alan Palestine, MD, director Center for Ocular Inflammation at the University of Colorado Department of Ophthalmology, pointed out that patients dealing with non-infectious uveitis endure a lifelong struggle to safeguard their vision.

“Despite current treatments, a majority of patients eventually suffer vision loss, significantly impacting their quality of life,” he said in the news release “The fear of blindness, affecting 22% of patients, adds another layer of devastation.

Moreover, Palestine noted there currently are FDA-approved, steroid-free topical medications for this condition.

“An eye drop product that effectively resolves inflammation without raising intraocular pressure is a long-standing, so far unachievable, goal in our field,” he said. “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."

Tarsier received written agreement from the FDA that the clinical trial protocol and planned statistical analysis of the Tarsier-04 Phase 3 trial of TRS01 address objectives supporting regulatory submission and a potential future marketing application in this indication. Tarsier-04 will be a multi-center, randomized, double-masked, active-controlled Phase 3 trial designed to evaluate the efficacy and safety of TRS01 eye drops in non-infectious uveitis including patients with uveitic glaucoma, and is planned to enroll up to 300 patients in the U.S.

"We are grateful for the FDA's professional support and guidance", said Ms. Zohar Milman, M.Sc, COO of Tarsier. "With this swift agreement, we are now focused on gearing up preparations toward the trial start-up activities.".

About Tarsier Pharma

Tarsier Pharma is a clinical-stage biopharmaceutical company, focused on the discovery, development, and commercialization of first-in-class pharmaceutical therapies to treat immune-mediated blinding diseases in the front and back of the eye.

About TRS01

Dazdotuftide (formerly known as TRS) is a first-in-class drug with a new and unique mechanism of action, for the treatment of ocular blinding diseases of back- and front- of the eye. Dazdotuftide is a bio-inspired novel proprietary technology platform, based on a New Chemical Entity (NCE) with a disruptive mechanism of action. Dazdotuftide was found to modulate macrophages from inflammatory macrophages (M1) to IL-10 secreting ant-inflammatory macrophages (M2).

Tarsier is developing dazdotuftide as an eye drop formulation (TRS01) for front-of-the-eye indications and as a slow-release biodegradable intravitreal injection (TRS02) targeting back-of-the-eye blinding indications with an underlying inflammatory pathology, such as Diabetic Macular Edema, Non-Proliferative Diabetic Retinopathy, and others. Due to dazdotuftide's unique mechanism of action of immunomodulation, we believe it has the potential to treat these debilitating diseases with a sustained effect and minimal risk of IOP elevation.