Reata Pharmaceuticals (Irving, TX) has received a $30 million milestone payment from its licensee, Kyowa Hakko Kirin as part of a corporate agreement.
In 2017, Kyowa Hakko Kirin reported positive results from the phase 2 TSUBAKI trial of bardoxolone methyl (bardoxolone) in patients with type 2 diabetes and chronic kidney disease. Initiation of the AYAME phase 3 clinical trial to assess the efficacy and safety of bardoxolone for the treatment of diabetic kidney disease in Japan was the trigger for the payment.
In July 2017, the FDA granted orphan drug designation to bardoxolone for the treatment of Alport syndrome and pulmonary arterial hypertension. Orphan drug designation is given to treatments for diseases that affect fewer than 200,000 people in the United States. About 12,000 people in the United States have Alport syndrome and most develop end stage kidney disease. The disease also affects the ear and eye.
The orphan designation will provide Reata with development incentives, including tax credits for clinical testing, exemption from a prescription drug user fee, and seven years of market exclusivity. The European Commission likewise granted orphan drug designation in Europe to bardoxolone for treatment of Alport syndrome.
Bardoxolone is an investigational medication taken orally, once a day. It is an activator of Nrf2, a transcription factor that induces molecular pathways that decrease inflammation. The pathways help to restore mitochondrial function, reduce oxidative stress, and block signals that cause an inflammatory response.