- Reata’s mission is to develop novel therapeutics for patients with serious and life-threatening diseases by targeting molecular pathways that regulate cellular metabolism and inflammation.
- Our lead product candidates, bardoxolone methyl and omaveloxolone, are Nrf2 activators, previously referred to as antioxidant inflammation modulators (AIMs). Nrf2 is a transcription factor for restoring mitochondrial function, reducing oxidative stress, and resolving inflammation.
- Bardoxolone methyl is currently in a pivotal Phase 2/3 (CARDINAL) study for the treatment of chronic kidney disease caused by Alport syndrome, a Phase 3 (CATALYST) study for the treatment of CTD-PAH, a Phase 2 (PHOENIX) study for treatment of rare kidney diseases, and a Phase 2 (LARIAT) study for the treatment of PH-ILD.
- Omaveloxolone is being studied in the registrational part 2 (MOXIe) study for Friedreich’s ataxia, a genetic disorder involving mitochondrial dysfunction.
Reata’s mission is to develop novel therapeutics for patients with serious and life-threatening diseases by targeting molecular pathways that regulate cellular metabolism and inflammation. Our lead product candidates, bardoxolone methyl and omaveloxolone, are Nrf2 activators, previously referred to as antioxidant inflammation modulators (AIMs). Nrf2 is a transcription factor for restoring mitochondrial function, reducing oxidative stress, and resolving inflammation.
Bardoxolone methyl is being studied in a Phase 2/3 trial in chronic kidney disease caused by Alport Syndrome called CARDINAL. In August 2017, we began enrolling patients in the Phase 3 portion of CARDINAL, which is an international, multi-center, double-blind, randomized, placebo-controlled study of the safety and efficacy of bardoxolone methyl in patients with Alport syndrome. In July 2017, we received orphan drug designation from the United States Food and Drug Administration (FDA) for bardoxolone methyl for the treatment of Alport syndrome. In November 2017, we reported the primary endpoint data for the open label, Phase 2 portion of CARDINAL. We expect one year top-line results from the Phase 3 portion to be available in the second half of 2019. Bardoxolone methyl is also being studied in trials in four other areas. In October 2017, we began activating sites in a Phase 2 trial, known as PHOENIX, to test bardoxolone methyl for the treatment of other rare kidney diseases. We anticipate initial data to be available in the second half of 2018.
In addition, bardoxolone methyl is being studied in a Phase 3 trial, known as CATALYST, for the treatment of pulmonary arterial hypertension (PAH) associated with connective tissue disease (CTD-PAH). Data from CATALYST are expected to be available during the second half of 2018. However, the trial is designed to enroll between 130 and 200 patients, and the final sample size will be determined by a pre-specified, blinded sample size re-calculation based on 6MWD variability and baseline characteristics. The timing of data availability may change if the sample size is increased due to the sample size recalculation. In 2015, the FDA granted our request for orphan drug designation for the treatment of PAH. Bardoxolone methyl is also being studied in a Phase 2 trial, known as LARIAT, for the treatment of pulmonary hypertension (PH) due to interstitial lung disease (PH-ILD) and PAH. We began enrolling patients in CATALYST in October 2016.
Omaveloxolone is being studied in the registrational part 2 of our MOXIe trial for the treatment of Friedreich’s ataxia (FA). MOXIe is a two-part Phase 2 trials, each part of which is randomized, placebo-controlled, and double-blind. Part one is a dose-escalation portion to evaluate the safety and efficacy of omaveloxolone. Data from part two have the potential to be used for registration. In June 2017, we announced data from part one of the Phase 2 MOXIe trial. In October 2017, we began enrollment of patients for part two of MOXIe. We expect data from part two to be available in the second half of 2019.
In addition to our lead Nrf2 product candidates, we have completed a Phase 1 study of RTA 901, the lead molecule in our new class of heat shock protein modulators, to evaluate its safety, tolerability, and pharmacokinetic profile in healthy adult volunteers. We encountered no safety or tolerability issues, observed an acceptable pharmacokinetic profile in the trial, and are currently evaluating various options in the design and timing of a Phase 2 trial. These compounds target pathways involved in the cellular response to misfolded proteins and mitochondrial protein import. RTA 901 and related analogs have shown promising effects in animal models of neurological disease.
Additionally, we retain several promising preclinical development programs employing both Nrf2 activators and other small molecules with different mechanisms of action. We believe that our product candidates and preclinical programs have the potential to improve clinical outcomes in numerous underserved patient populations.
The foundational biology of Nrf2 activators underlies our two lead product candidates, bardoxolone methyl and omaveloxolone. Nrf2 is a transcription factor that promotes normal mitochondrial function, increases production of antioxidants, reduces oxidative stress, and reduces pro-inflammatory signaling during the resolution of a normal, healthy inflammatory response. Since mitochondrial dysfunction, oxidative stress, and inflammation are features of many diseases, Nrf2 activators have many potential clinical applications and have been the subject of hundreds of peer-reviewed scientific papers. In addition to the Nrf2 activator programs, we have technologies in preclinical development targeting the treatment of diseases through a variety of mechanisms.
Additional information on the pharmacology of Nrf2 activators can be found here.
Beyond our platform technologies and in-licensed compounds, we are constantly working on novel science and have active internal discovery and preclinical programs in a number of significant disease areas. In addition to internal discovery, we continue to seek additional opportunities to in-license and collaboratively develop novel technologies from premier academic institutions. This operating model of combined internal discovery, in-licensing, and collaborative development guided the founding of Reata, and we continue this strategy today to maintain a pipeline of promising development programs.