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 three distinct biological mechanisms of actions: Reactive Aldehyde Species (RASP) inhibition, Dihydrofolate Reductase (DHFR) inhibition, and Chaperome (CHP) 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 an aggregate of five Phase 2 clinical trials in dry eye disease and allergic conjunctivitis. In a sixth Phase 2 clinical trial, reproxalap demonstrated statistically significant and clinically relevant improvements in ichthyosis (a severe skin disorder) caused by Sjögren-Larsson Syndrome, a rare RASP-mediated disease with no approved therapy.  A growing body of clinical evidence supports the potential and relevance of RASP inhibition as a new and differentiated mechanism of action. We have discovered and are developing additional RASP inhibitors, ADX-103 and ADX-629, for the treatment of retinal disease and autoimmune disease, respectively. Additionally, in February 2018, we announced a partnership with Janssen, a Johnson & Johnson company, to develop RASP inhibitors for systemic inflammatory diseases. In the future, we may enter into additional partnerships that facilitate the development and commercialization of our product candidates.

As we continue to execute on 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. In addition, in 2016, we in-licensed the clinical-stage product candidate ADX-1612 and ADX-1615 (an oral pro-drug of ADX-1612), both of which inhibit CHP, a mechanistically differentiated approach for the potential treatment of a number of inflammatory diseases.