Gout Market Opportunity Overview

• Over 6 million adults in the U.S.¹ and approximately 5 million adults in the
  European Union ² have reported suffering from at least one episode of gout
• Gout is the most common form of inflammatory arthritis in men over 40 and represents a significant medical need with limited treatment options
• In the last 40 years, only one new medication has received FDA approval for the treatment of hyperuricemia associated with gout

Gout is a painful and debilitating disease caused by abnormally elevated levels of uric acid in the blood stream.  This leads to the deposition of painful, needle-like uric acid crystals in and around the connective tissue of the joints and in the kidneys resulting in inflammation, the formation of disfiguring nodules (tophi), intermittent attacks of severe pain (acute flares) and kidney damage (nephropathy).   In addition, evidence suggests that the chronic elevation of uric acid associated with gout, known as hyperuricemia, may also have systemic consequences, including an increased risk for kidney dysfunction and cardiovascular disease.

While gout is a treatable condition, there are limited treatment options, and a number of adverse effects are associated with most current therapies.  Allopurinol, a xanthine oxidase inhibitor, is the most widely-prescribed drug for the treatment of gout.  It acts by reducing the production of uric acid in the body.  Approximately 10 percent of gout patients are believed to produce too much uric acid.  The other 90 percent are considered to have a defect in their ability to excrete sufficient amounts of uric acid and are classified as “under-excretors” of uric acid.   In large clinical trials, approximately 60 percent of patients did not respond adequately to treatment with allopurinol alone.     

RDEA594 is our most advanced product candidate for the treatment of hyperuricemia and gout.  RDEA594 is a selective inhibitor of URAT1, a transporter in the kidney that regulates uric acid excretion from the body.  RDEA594 has been shown to normalize the amount of uric acid excreted by gout patients.  Since the majority of gout patients are “under-excretors”, normalizing uric acid excretion by moderating URAT1 transporter activity with RDEA594 may provide the most physiologically appropriate and effective means of reducing blood levels of uric acid (sUA) when used alone or in combination with other sUA lowering agents, such as allopurinol or febuxostat (Uloric®, Takeda Pharmaceutical Company Limited; Adenuric®, Ipsen and Menarini), the newest xanthine oxidase inhibitor currently available for the treatment of gout.

RDEA594 is currently being evaluated in a comprehensive Phase 2 development program designed to demonstrate its broad clinical utility in a wide spectrum of gout patients.  Ongoing studies in this program include a Phase 2b single-agent dose-response study evaluating the safety and urate-lowering effects of 200, 400 and 600 mg of RDEA594, a Phase 2b study evaluating RDEA594 as an add-on to allopurinol in patients that do not respond adequately to allopurinol alone, and a study in gout patients with renal impairment. 

RDEA684, our next-generation URAT1 inhibitor, has been selected as a development candidate for the treatment of hyperuricemia and gout.  Based on preclinical results, RDEA684 demonstrates many of the same positive attributes as the first-generation candidate, RDEA594, but with more than 170-times greater potency against the URAT1 transporter. 

For more information regarding these programs please click here to view our most recent company presentation.