Tarsier Pharma Announces Successful Type C Meeting with FDA for TRS01 in Noninfectious Uveitis
November 13, 2023
Agreement on registrational program’s primary and key secondary Endpoint to reflect TRS01 positive risk/benefit profile for patients with noninfectious uveitis and uveitic glaucoma
TRS01 has the potential to be the first line treatment option for noninfectious uveitis and specifically uveitic glaucoma
Tel-Aviv., Nov. 13th, 2023 — Tarsier Pharma, Ltd., a clinical-stage ophthalmic biopharmaceutical company focused on developing and commercializing first-in-class new therapies for the treatment of blinding ocular diseases, today announced the successful outcome of a Type C meeting with the U.S. Food and Drug Administration (FDA), supporting the advancement of TRS01 eye drop formulation for the treatment of noninfectious uveitis into a registrational program based on the recently completed TRS4Vision trial.
“We are pleased to have FDA agreement on the primary and key secondary endpoints for the registrational program of TRS01 which we believe has the potential to be first-in-class first line treatment for noninfectious uveitis and specifically in uveitic glaucoma” said Zohar Milman, MSc. Chief Operational Officer of Tarsier. “We are grateful for the FDA’s support and guidance and look forward to continue the professional discussions as we advance TRS01 development.”
The Type C meeting was supported by results from the previously completed TRS4Vision trial – a randomized, double-masked, active-controlled Phase 3 trial that was designed to evaluate the efficacy and safety of TRS01 eye drop formulation in noninfectious uveitis patients including uveitic glaucoma patients. In the TRS4Vision trial, TRS01 was found to be active in clearing inflammation, reducing ocular pain and exhibited a compelling risk/benefit profile, minimizing the concerns of Intraocular Pressure (IOP) increase during the course of treatment.
The upcoming trial is intended to replicate the TRS4Vision trial, with a revised endpoints in accordance with FDA discussions. The revised endpoints specifically designed to emphasize the risk/benefit profile of TRS01. The primary endpoint chosen has already attained statistical significance in the TRS4Vision trial, with a p-value <0.01.
“The results from the TRS4Vision trial demonstrates that TRS01 has a positive risk/benefit profile for the most vulnerable eyes that already reached the stage of uveitic glaucoma,” stated Quan Dong Nguyen, MD, MSc, FAAO, FARVO, FASRS, Professor of Ophthalmology, Medicine, and Pediatrics at the Byers Eye Institute, Stanford University School of Medicine, and who is an international leader in Uveitis and Ocular Inflammatory Diseases and the President of the International Ocular Inflammation Society (IOIS) “There is growing evidence on the substantial risk of increased IOP in uveitis and a clear understanding that we need a steroid-free first line treatment for the active phase of anterior non-infectious uveitis. The data thus far support that the TRS01 profile has the potential to slow or prevent clinically meaningful progression of glaucoma in noninfectious uveitis and serve as first line treatment in uveitic glaucoma,” said Professor Nguyen.
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.
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.
About Uveitic Glaucoma
Non-infectious uveitis is a chronic autoimmune disease of the eye and the third leading cause of blindness. Despite available treatment options, 67% of patients suffering from non-infectious uveitis reaching visual impairment and 22% are meeting a legal blindness criterion. The current treatment for active non-infectious anterior uveitis is topical steroids. Since their introduction to the eye 70 years ago, topical steroids have been used as first line treatment due to their potency in resolving inflammation. While potent in resolving inflammation, topical steroids carry a high risk of intraocular pressure (IOP) elevation mediated by steroids’ receptors, and often if treatment is ceased without long tapering period, a recurrent rebound inflammation occurs. In some patients, IOP elevation leads to glaucoma (i.e., uveitic glaucoma). Uveitic glaucoma is a challenging disease to treat, with a unique pathophysiology, disease course, prognosis, and a significantly more difficult clinical course to manage than anterior uveitis without glaucoma. The overall prevalence of glaucoma in eyes with uveitis (i.e., uveitic glaucoma) varies from 10 to 20 percent but is much more common in chronic uveitis and can be as high as 46 percent.