FDA Approves Tarsier Pharma's SPA for Phase 3 TRS01 Trial in Non-infectious Uveitis

Tarsier Pharma, a biopharmaceutical company at the clinical stage, has announced that it has received an endorsement from the U.S. Food and Drug Administration (FDA) through a Special Protocol Assessment (SPA) for the Tarsier-04 Phase 3 clinical trial. This trial is designed to assess the efficacy of TRS01 eye drops in treating non-infectious uveitis, a condition that can lead to uveitic glaucoma.

The SPA agreement signifies that the FDA has acknowledged the adequacy and acceptability of the critical elements of the study's protocol and statistical analysis, which are intended to support a future marketing application. Daphne Haim-Langford, the founder and CEO of Tarsier Pharma, expressed that this agreement provides a clear path for TRS01 and that the Tarsier-04 trial's protocol was crafted to replicate the previous TRS4Vision trial with revised endpoints to highlight the drug's benefit/risk profile.

The primary endpoint of the FDA-approved protocol had already achieved a significant alpha level in the TRS4Vision trial, which gives Tarsier strong confidence that the Tarsier-04 trial will meet its endpoints. Prof. Palestine from the University of Colorado's Department of Ophthalmology emphasized the lifelong struggle of patients with non-infectious uveitis to preserve their vision and the significant impact of vision loss on their quality of life. Currently, there are no FDA-approved, steroid-free topical medications available for this condition. The development of an eye drop that can effectively reduce inflammation without increasing intraocular pressure is a significant goal in the field.

The Tarsier-04 trial is a multi-center, randomized, double-masked, active-controlled Phase 3 study that will evaluate the efficacy and safety of TRS01 eye drops in treating non-infectious uveitis, including patients with uveitic glaucoma. The trial plans to enroll up to 300 patients across the United States.

Zohar Milman, M.Sc, COO of Tarsier, expressed gratitude for the FDA's support and guidance and stated that the company is now focused on preparing for the trial's startup activities.

Tarsier Pharma is dedicated to discovering, developing, and commercializing innovative pharmaceutical therapies to treat immune-mediated blinding diseases affecting both the front and back of the eye. TRS01, formerly known as Dazdotuftide, is a first-in-class drug with a novel mechanism of action. It is based on a New Chemical Entity (NCE) and has the ability to modulate macrophages from inflammatory to anti-inflammatory types, secreting IL-10. Tarsier is developing this drug in two forms: as eye drops for front-of-the-eye conditions and as a slow-release biodegradable intravitreal injection for back-of-the-eye conditions with inflammatory pathology.

Non-infectious uveitis is a chronic autoimmune eye disease and a major cause of blindness. Despite current treatments, a significant number of patients still suffer from visual impairment, with a notable percentage meeting legal blindness criteria. The standard treatment, topical steroids, while effective in reducing inflammation, carries the risk of increasing intraocular pressure and can lead to glaucoma. The prevalence of glaucoma in eyes with uveitis is relatively high, especially in chronic cases. The development of TRS01 aims to provide a new first-line treatment option that could replace topical steroids and potentially offer a more effective and safer alternative for patients.