Aldeyra’s broad product candidate pipeline applies to many diseases that may affect diverse aspects of the body. We are developing product candidates that can be administered orally or by injection to treat disease systemically.
Immune-Mediated Systemic Diseases
Immune-mediated systemic diseases, such as autoimmune disease, are generally chronic conditions characterized by excessive and misdirected inflammatory responses. In aggregate, autoimmune diseases and related systemic inflammatory disorders represent tens of millions of patients in the United States, with aggregate drug sales expected to exceed $74 billion by 2022. In 2018, three of the top five highest-selling drugs, totaling more than $32 billion globally and $22 billion in United States sales, were prescribed for a variety of immune-mediated disorders, including Crohn’s disease, rheumatoid arthritis, psoriasis, ulcerative colitis, and ankylosing spondylitis.
The potential market for immune-modulating therapies could continue to expand as a result of growing evidence that excessive inflammation may be critical to the development and progression of cardiovascular disease, diabetes, Alzheimer’s disease, and many other common conditions that are not typically characterized as inflammatory or autoimmune diseases.
Given the complex pathophysiology of systemic immune-mediated disorders, many of which are caused by a variety of pro-inflammatory mediators, therapy often requires combinations of drugs with distinct mechanisms of action. As such, we believe novel product candidates for immune-mediated diseases are in high demand.
Aldeyra’s RASP, dihydrofolate reductase, and chaperome inhibitors are being developed to down-regulate the immune system across a number of inflammatory diseases.
Sjögren-Larsson Syndrome (SLS) is a rare systemic disease and inborn error of metabolism caused by mutations in an enzyme that metabolizes fatty (long-chain carbon) RASP, resulting in severe skin, neurological, and retinal disorders. Genetic mutation analysis suggests that there are approximately 1,300 SLS patients in the United States, and a greater number of SLS patients in Europe.
Reproxalap, Aldeyra’s lead RASP inhibitor, is being developed in SLS for the treatment of ichthyosis, a serious skin disorder characterized by excessive dryness, leading to comprise of dermal integrity, infection, and bleeding. Other RASP inhibitors in Aldeyra’s pipeline may be tested in SLS retinal or neurological diseases.