Developed for the treatment of severe eye diseases, such as non-infectious posterior uveitis. Ready for clinical Phase II. Activities to establish commercial partnerships are ongoing.
Type of substance:
Small molecule
Effect:
Affects immune cells, contributing to an anti-inflammatory effect
On this page:
Laquinimod in Brief
The project focuses on inflammatory eye diseases where current treatments may be insufficient or lacking. Laquinimod is being developed for the treatment of severe inflammatory eye diseases, such as non-infectious uveitis.
It has been shown in experimental models of autoimmune/inflammatory diseases that laquinimod targets the aryl hydrocarbon receptor (AhR) that is present in antigen-presenting cells and involved in the regulation of these cells. By targeting the AhR, antigen presenting cells are re-programmed to become tolerogenic, so that instead of activating pro-inflammatory T cells, regulatory T cells with anti-inflammatory properties are activated leading to a dampening of the inflammation.
Reduces pro-inflammatory T cells
Increases regulatory T cells
Suppresses inflammation
Non-infectious uveitis (NIU) is the inflammation of the uveal tract (iris, ciliary body, and choroid), but can also lead to an inflammation of nearby tissues, such as the retina, the optic nerve, and the vitreous humor, in the absence of an infectious cause.
Laquinimod Reduces Inflammation in the Posterior Part of the Eye
Laquinimod will be developed as a new treatment for non-infectious uveitis and has the potential to be used in the 1st line of treatment as an add-on to corticosteroids, as well as in the 2nd line of treatment for patients that have failed corticosteroid treatment.
Laquinimod has been evaluated in experimental autoimmune uveitis (EAU), a well-established model that closely mimics human non-infectious uveitis. In models induced by retinal peptides, resulting in either chronic or relapsing disease, laquinimod consistently demonstrated a significant, dose-dependent reduction in disease severity. Both oral administration and administration of laquinimod eye drops were effective, underscoring a robust anti-inflammatory effect in preclinical models.
The distribution of laquinimod in the eye has been investigated in rabbits following administration of eye drops containing laquinimod. High concentrations of laquinimod were detected in the cornea and external tissues after 30 minutes, followed by lower levels at later time points. After 4 and 8 hours, respectively, the substance was also detected in the posterior part of the eye, in the retina/choroid, at concentrations comparable to plasma levels.
Partner: Stanford MedicineGorcUSA
An eye drop formulation of laquinimod was developed to enable clinically relevant intraocular levels based on the molecule’s physicochemical properties. A clinical Phase I study in healthy volunteers has been completed, and a Phase I biodistribution study has been conducted in patients scheduled to undergo vitrectomy.
The Phase I study in healthy volunteers showed that the eye drop formulation was well tolerated and demonstrated a favourable safety profile at dose levels where therapeutic concentrations are expected to be achieved. More information about the study is available at clinicaltrials.gov (NCT05187403).
In the biodistribution study, 10 patients received laquinimod eye drops at three different dose levels for two weeks prior to surgery. Laquinimod was detected in both the vitreous body and the anterior chamber of the eye in a dose-dependent manner, demonstrating that the substance reaches the posterior parts of the eye. The results also confirmed that laquinimod achieves therapeutically relevant concentrations in the posterior segment of the eye, supporting plans to advance into Phase II development in patients with uveitis. Activities to establish commercial partnerships are ongoing.
During the years of late-stage clinical product development, data on the clinical efficacy and safety of oral laquinimod were generated in more than 5,000 patients, primarily patients with multiple sclerosis (MS), corresponding to more than 14,000 patient-years of exposure. Extensive datasets, including a regulatory package with preclinical and clinical safety data and full commercial-scale CMC documentation, have been generated.
Completed
Phase I
The primary objective of the study was safety and tolerability, and the secondary readouts included ocular toxicity, pharmacokinetics and exposure.
Completed
Phase I
The study was conducted at the Byers Eye Institute at Stanford University, US, and the principal investigator was Quan Dong Nguyen, MD, MSc, FAAO, FARVO, FASRS, Professor of Ophthalmology, Medicine and Pediatrics at Stanford University School of Medicine.
A collection of Publications
Ophthalmol Sci. 2026 Apr 3;6(6):101186. doi: 10.1016/j.xops.2026.101186. eCollection 2026 Jun
bioRxiv [Preprint]. 2025 May 25:2025.05.20.654165. doi: 10.1101/2025.05.20.654165
Presenterades på International Ocular Inflammation Society (IOIS) 2023 Meeting i Berlin, 6-9 September, 2023
Neurol Neuroimmunol Neuroinflamm 2021 Jan 6;8(2):e946