EyeGate Pharma Completes Target Enrollment in Phase 2 Study to Evaluate PP-001 for the Treatment of Ocular Surface Inflammation
EyeGate Pharmaceuticals announced that it has completed target enrollment of 21 patients in the phase 2 proof-of-concept (POC) study evaluating its lead product candidate, PP-001, in patients with ocular surface inflammation due to ocular surface diseases including dry eye. PP-001, an immune-modulating molecule, is an inhibitor of dihydroorotate dehydrogenase (DHODH) and is first-in-class for ophthalmology indications.
“This is an important clinical milestone and step forward in advancing PP-001 as a new treatment option for patients with dry eye and a range of ocular surface and systemic diseases,” Brian Strem, PhD, President and Chief Executive Officer of EyeGate, said in a company news release. “We believe our proof-of-concept study will further validate the clinical utility of PP-001, which has the potential to overcome off-target side effects and safety issues associated with DHODH inhibitors with greater specificity and best-in-class picomolar potency. Additionally, this study will inform our clinical strategy for dry eye disease in the US and more broadly, guide the development of our pipeline programs as we explore opportunities to maximize the therapeutic potential of our platform. We look forward to providing topline data in Q4 2021.”
The randomized, double-masked, placebo-controlled POC study conducted at a site in Vienna, Austria is designed to evaluate the safety, tolerability, and efficacy of PP-001 in patients with ocular surface inflammation due to dry eye disease. In this study, a total of 21 patients are treated for 12 days with 0.15% of PP-001 or placebo. An IND filing for dry eye disease in the U.S. is expected in Q4 2021.
PP-001 is a novel, non-steroidal immunomodulatory small molecule. PP-001 is a highly specific nanomolar potent inhibitor of DHODH, an essential enzyme of the de novo pyrimidine pathway. Inhibition of this pathway also downregulates expression of IFN-γ and IL-17, two hallmark cytokines of Th1 and Th17 cells responsible for inflammatory diseases of the eye. Further, PP-001 reduces the host cell pyrimidine pool, which leads to inhibition of replication of activated immune cells.