Rendering of molecules

Science & Technology

Immune-mediated diseases result from an imbalance of inhibitory and stimulatory factors that regulate the immune system. This imbalance can lead to an array of conditions including autoimmune disease, allergy, immunoproliferative disease, and cancer. Many ocular, cardiovascular, metabolic, neurological, and musculoskeletal diseases, affecting tens of millions of patients in the United States and hundreds of millions of patients worldwide, are at least partially immune-mediated.

An estimated 7% of western society suffers from some form of immune-mediated disease, and incidence has been increasing.

Given the complexity of immune dysregulation, which involves many mediators and signaling pathways, rarely is any single therapeutic approach effective, and today most immune-mediated diseases are generally considered to be inadequately treated. As such, we believe immune-mediated diseases represent considerable unmet medical need, and that demand for novel immune-modulating therapies is high.

Our Investigational New Drug Development Pipeline

Our deep and innovative development pipeline is focused on immune-mediated ocular diseases and select systemic diseases and encompasses two distinct biological mechanisms of action: Reactive Aldehyde Species (RASP) inhibition and Dihydrofolate Reductase (DHFR) inhibition. The immunological activity of our candidates generally leads to diminished levels of pathological inflammation via the down-regulation of immune cell activation or proliferation.

Our investigational new drug product reproxalap is a RASP inhibitor that has been shown to diminish ocular inflammation and has demonstrated statistically significant improvements across Phase 2 and 3 clinical trials in dry eye disease and allergic conjunctivitis. A growing body of clinical evidence supports the potential and relevance of RASP inhibition as a new and differentiated mechanism of action. As part of our RASP-Inhibitor Discovery Platform, we also are developing a range of new molecules for the treatment of retinal and systemic indications.

As we continue to execute our strategy of expanding our product candidate pipeline, we intend to license and/or acquire new immune-modulating approaches with novel therapeutic potential. In 2019, we acquired Helio Vision, Inc. and thereby obtained rights to ADX-2191, an intravitreal DHFR inhibitor for the prevention of proliferative vitreoretinopathy, a serious sight-threatening retinal disease with no approved treatment.