Reata Pharmaceuticals, Inc.

Ladies and gentlemen, thank you for standing by, and welcome to the Riata Pharmaceuticals third quarter financial results and update on development programs. At this time, all participants' lines are in a listen-only mode. After the speaker's presentation, there will be a question-and-answer session. To ask a question during this session, you will need to press star 1 on your telephone. An audio recording of today's webcast will be available shortly after the call. Today on Riata's website at riatapharma.com in the Investors section. Before the company proceeds with its benchmarks, please note that the forward-looking statement disclosure in the company's press release. The company will be making forward-looking statements on today's call. There are many factors that could cause results to differ from expectations, including those noted in the company's SEC filing. Today's statements are not guaranteed of future outcomes. Please also note that any comments made on today's call speak only as of today, November 9th, 2020, and may no longer be accurate at the time of any webcast, replay, or transcript rereading. Following the prepared remarks, we will open the call up for questions. We ask that you please limit yourself to one question and one follow-up so that we can accommodate as many questions as possible. I would now like to hand the conference over to your speaker today, Vidhi Jindal, Vice President of Strategy. Please go ahead, sir.

Thank you. Good morning, and welcome to RIADA Management's call to discuss our financial results for the third quarter of 2020 and to provide a review of our development programs. This morning, we issued a press release with a summary of our financial results and the results of the baseline controlled study of imabaloxone in patients with Friedreich's ataxia. Separately, we issued a press release announcing data from our cardinal study of fardoxone methyl in patients with CKD caused by Alport syndrome and from EGLE, our long-term extension study of Vardoxone in patients with Alport syndrome. These press releases can be found on the Investors section of our website at riadopharma.com. I'm joined today by our Chief Executive Officer, Warren Huff, our Chief Research and Development Officer, Colin Meyer, and our Chief Operating and Chief Financial Officer, Manmeet Soni. Turning to slide three, I'll now turn the call over to Warren. Thanks, Vinny.

Good morning, everyone, and thank you for joining us. We have a number of important announcements and events to cover today, including the Cardinal Phase 3 Year 2 study results that we announced this morning, a regulatory update on our Alport Syndrome Program, and with respect to our Friedreich's Ataxia Development Program, the results of the MOXIE Baseline Control Study. We will also be providing a financial update. I apologize in advance for the length of the management presentation, but we have a lot to cover, and we're going to start with the Cardinal Phase 3 Year 2 study results. This morning, we announced positive top line results from the completed pivotal Cardinal Phase III study of Ardoxelone in patients with chronic kidney disease caused by Alport syndrome. Cardinal was the largest global interventional study ever conducted in Alport syndrome. Last year, we announced that we met both the primary and key secondary endpoints at the end of year one of the Cardinal study. We're very pleased to announce today that we met both the primary and key secondary endpoints at year two of the Cardinal study. Alport syndrome is a severe form of chronic kidney disease, or CKD, and many patients with the disease have a 100% lifetime risk of needing dialysis or a kidney transplant. Children and adults with the most severe form of the disease need dialysis or a kidney transplant in their 20s. It's a devastating disease that not only affects their kidneys, but also results in depression and anxiety, hearing impairment, and vision and eye problems. The patients with Alport syndrome enrolled in the phase three portion of Cardinal were losing substantial amounts of kidney function when they entered the trial. After two years in Cardinal, patients on placebo lost on average approximately nine milliliters per minute of estimated glomerular filtration rate or EGFR. The pediatric patients lost on average approximately 15 mils per minute and were at substantial risk of kidney failure within five to seven years. Importantly, more than 20% of the placebo patients experience an event that predicts kidney failure, including a 30% decline in EGFR, an EGFR of less than 15 milliliters per minute, or actual kidney failure. We refer to these events as the kidney failure composite events. By contrast, after two years of treatment, patients treated with bardoxalone lost, on average, less than one mil per minute while on treatment and 4.5 mil per minute after withdrawal of treatment. The pediatric patients treated with bardoxalone lost on average approximately one to two mil per minute on treatment and after withdrawal of treatment. Importantly, bardoxalone patients had a 50% reduced risk of one of the kidney failure composite events. In addition to the cardinal year two data, also reported data on kidney function from patients with Alport syndrome after three years of bardoxalone treatment during the EGLE long-term extension study. Increases in EGFR from baseline observed after one year of treatment were sustained on average for three years, providing additional insight into the long-term effect of bardoxalone on kidney function in these patients. From a safety perspective, the adverse event profile was consistent with prior trials. The majority of adverse events were mild to moderate in severity. Serious adverse events were reported in approximately 50% fewer bardoxelone patients compared to placebo. Urinary protein was not increased relative to placebo after two years, and bardoxelone patients reported fewer non-kidney adverse events associated with Alport syndrome. No imbalances in fluid overload events or increases in blood pressure were observed. In summary, bardoxelone had a favorable safety profile in people with Alport syndrome. Based on these efficacy and safety results, we believe that Mardoxalone has the potential to become the first therapy to show disease-modifying effects in patients with Alport syndrome. With that background on the efficacy and safety results, I'm handing it over to Colin to provide more detail on the study design, the results, and the regulatory pathway for Mardoxalone in Alport syndrome patients.