Lux Biosciences, Inc.

Uveitis

The Disease: Uveitis is a collective term for variety of ophthalmic conditions that result in chronic inflammation of the eye. Uveitis can be of infectious or autoimmune origin and is generally classified by anatomical location. Anterior uveitis affects the front of the eye, intermediate and posterior uveitis affect the back of the eye and panuveitis affects all parts of the eye. There is substantial evidence indicating the involvement of T-lymphocytes, key immune system cells involved in inflammatory processes, in the development of autoimmune uveitis.

Uveitis is an under-diagnosed and under-recognized medical condition that causes ocular pain and loss of vision. Uveitis afflicts the young – the median age at time of diagnosis is 39 years - and the disease typically persists chronically. The general course of uveitis is similar to other autoimmune diseases. The disease flares causing inflammation in the eye and symptoms such as ocular pain, redness vision loss, and floaters and then remits with treatment. The goal of treatment is to reduce the frequency and intensity of disease flares, as active inflammation is believed to cause cumulative damage to the eye resulting in persistent loss of vision. Experts estimate that 10-22% of new cases of blindness in the United States result from this disease. In the US alone, approximately 350,000 people suffer from uveitis; of these, approximately 130,000 are afflicted with the more severe forms, intermediate, posterior and panuveitis. These forms are typically not responsive to treatment with topical corticosteroids, and require systemic treatment. The chronic use of oral corticosteroids, the only systemic therapy approved by FDA for these forms of the disease, is burdened with multiple side effects including osteoporosis, hypertension, hyperglycemia, hypercholesterolemia, impaired wound healing, weight gain, cosmetic effects, and mood disorders, and in the eye cataract formation and glaucoma. The SUN (Standardization of Uveitis Nomenclature) Working Group, a group of international uveitis experts, has therefore recommended to limit the use of corticosteroids to oral doses of no more than 10 mg/day, and use corticosteroid sparing agents (none of which are approved) to keep the inflammation under control. However, in a recent publication it was found that in actual medical practice it appears difficult to achieve this goal: the average corticosteroid dose used to control inflammation among some 600 patients reviewed was 25 mg, a dose commonly associated with major corticosteroid morbidities.

Lux Biosciences is currently developing LUVENIQ™ , an orally administered immunosuppressant for noninfectious uveitis involving the intermediate and/or posterior segment of the eye.

The Market Opportunity: There is a major need for a safe, effective, and approved medication in uveitis. In North America and Europe, approximately 250,000 patients combined are afflicted with noninfectious uveitis involving the intermediate and posterior segments of the eye; i.e., the target group for LUVENIQ. As uveitis affects a young patient population, the socio-economic impact of the disease is greater than that of age-related macular degeneration (AMD) or diabetic macular edema (DME). In addition, a new medication may have the potential to change the course of the disease leading to better outcomes, such as a major delay in the mean time to vision loss, similar to the drugs found to be disease modifying in rheumatoid arthritis. The dearth of available treatment options for the treatment of uveitis at the current time is, indeed, comparable to the situation in rheumatoid arthritis in the early 1990’s, before the advent of novel disease-modifying therapies. Rheumatoid arthritis and certain types of uveitis share other similarities:

both result in chronic inflammation of the affected organ/tissue leading to severe, degenerative conditions unless treated aggressively;
both are autoimmune diseases and may often be associated with other autoimmune diseases, such as juvenile rheumatoid arthritis or psoriasis;
both are, in principle, amenable to similar types of treatments, directed at activated immune cells (Th-1 and Th-17); and
both are relatively rare, affecting less than 1% of the population.

The rheumatoid arthritis market has experienced major growth – from approximately $100M worldwide in 1990 to more than $10B today due to the introduction of new medications that were proven to be safe and effective. The uveitis market appears to have similar growth potential. FDA approval of a medication for uveitis would establish a new standard of care for this sight-threatening condition.