According to an article from PM360, Reata Pharmaceuticals has announced that its end-of-Phase 2 meeting with the Food and Drug Administration went according to plan. The American pharmaceutical company based out of Irving, Texas, now plans to advance to Phase 3 clinical trials for its autosomal dominant polycystic kidney disease (ADPKD) candidate treatment involving the administration of bardoxolone methyl to patients.
Autosomal Dominant Polycystic Kidney Disease
Yes, it is a mouthful. ADPKD is, as you might have guessed, a genetically-inherited condition that affects the kidneys. Kidneys are essential in managing the waste in our bloodstreams, and ADPKD will over time lead to cysts developing in the organs. These cysts grow over time, and can eventually lead to a host of serious health concerns including kidney stones, high blood pressure, and eventually organ failure. There are existing treatments that make life with the condition more comfortable, but there is currently no known cure. Sadly, ADPKD does typically result in a slightly reduced life expectancy – though advances in medical technology have made it possible to, in certain cases, add years to patients lives.
ADPKD is caused by a mutation in one of two genes: PKD1 and PKD2. These genes are responsible for the production of the proteins polycystin-1 and polycystin-2, which are found in the kidneys. Most often this mutation is inherited from a parent, though in some cases (25% in the U.K.) it may develop with no prior family history. Nonetheless, the condition is relatively rare, affecting somewhere between .04% and .25% of the global population – almost 12 million people.
That might not sound like a lot of people on a global scale, but a person with ADPKD has a 50% chance of passing it to any of their children. The estimates of global rates may also be low, due to inadequate access to medical services in certain parts of the world.
Reata Aims for Phase 3 Trials
Reata dubbed its impending phase 3 trial FALCON. Phase 3 trials are only conducted on medications or therapies that have proven to be both safe and effective in treating the targeted condition, essentially confirming and elaborating on data collected in the phase 1 and 2 trials.
The company expects to open enrollment for the trial sometime in mid-2019, and will be looking to enroll some 300 patients with ADPKD. The study will be double-blind and placebo controlled, meaning neither the participant nor the researcher is aware of who is receiving bardoxolone methyl and who is receiving a placebo.
Researchers hope the expanded data pool will solidify their understanding of bardoxolone methyl and its ability to improve kidney function. Bardoxolone received orphan drug designation from both the European Council and the American FDA for the treatment of Alport syndrome, a different condition that often affects kidney function.
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