Reata Pharmaceuticals, Inc.

Thank you for standing by and welcome to the Riata Pharmaceuticals first quarter financial results update on operational progress and development programs conference call. An audio recording of today's webcast will be available shortly after the call in the investor section of Riata's website at riatapharma.com. Before the company proceeds with its remarks, please note the forward-looking statements disclosure in the company's press release. There are many factors that could cause results to differ from expectations, including those noted in the company's SEC filings. Today's statements are not guarantees of future outcomes. Please also note that any comments made on today's call apply only as of today, May 10th, 2023, 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. We are joined today by Warren Huff, RIATA's Chief Executive Officer, Manmeet Soni, President, Colin Mayer, Chief Innovation Officer, Dawn Burt, Chief Commercial Officer, Simi Khan, Chief Medical Officer, and Andrea Lowen, Senior Vice President, Regulatory Affairs. At this time, I would like to turn the call over to Warren Huss.

Good morning, everyone. We thank you for joining us today. I'll begin on slide four. On February the 28th, we announced that the FDA approved Skyclaris as the first and only FDA approved drug indicated for the treatment of Friedreich's Ataxia, or FA, in adults and adolescents aged 16 years and older. As Dawn Burr will share later in the call, our experienced and fully trained teams began engaging customers immediately following approval. We're pleased to share that we have seen strong initial demand for Skyclaris from patients and their healthcare providers. We've received approximately 500 patient start forms through early May. The patient start forms were submitted by over 250 prescribing physicians, including neurologists, primary care physicians, and other healthcare providers, providing a broad initial prescriber base. We've been working diligently to ensure we can get drug to the market as quickly as possible. we've completed the final stages of SkyClarus drug product manufacturing and packaging. As we shared on the approval call in February, we observed a process-related drug substance impurity above the reporting threshold, which required us to update the drug substance specification prior to releasing final product. We're working collaboratively with the FDA to obtain an approval of the revised drug substance specification for the process impurity as quickly as possible. We now anticipate SkyClarus commercial drug availability to be available no later than mid-August. Andrea Loewen, our SVP of Regulatory Affairs, will provide an update on this process. We understand that in some patients, symptoms of FAA manifest early in life and SkyClarus is not currently indicated for patients younger than 16 years of age. Addressing this is a top priority for us, and we're evaluating strategies to support label expansion for pediatric patients younger than 16 years of age. We're planning to request a meeting this quarter with the FDA to discuss our possible strategies. We're also planning to initiate a study to evaluate the safety, tolerability, and pharmacokinetics of OMAV in pediatric patients in the fourth quarter of this year. CME will provide additional details on our plans later in the call. Next slide. We submitted a marketing authorization application for OMAV for patients with FA in Europe in the fourth quarter of last year, and the application is currently under review. We recently received the day 120 list of questions, and as Andrea will outline later, we believe that we can adequately respond to the questions that have been raised. We're on track to provide the responses in the third quarter of this year. Beyond OMav, we continue to pursue development of our Nrf2 activator platform and are advancing additional preclinical drug candidates. As Colin will discuss, we anticipate IND filings for two additional molecules in 2024. Moving to our HSP90 program, We're developing RTA-901 for patients with diabetic peripheral neuropathic pain, or DPMP. We finalized the design for a randomized, double-blind, placebo-controlled, two-part, 12-week phase two trial of RTA-901 in patients with DPMP and plan to initiate that study during the third quarter of this year. Next slide. Earlier today, Kaiwa Kieran reported results from Ayame, Kieran's Phase III trial of bardoxelone in patients with diabetic kidney disease. As Colin will discuss, the study met the primary and key secondary endpoints, and there were no significant safety issues identified in patients receiving bardoxelone. However, there was no separation in end-stage renal disease or ESRD events between the active drug and placebo groups after three years of treatment. Because there was no improvement in ESRD events and the regulatory implications of that, we and KKC have decided to discontinue our peroxyline development activities and we've decided to refocus our capital and resources on our other programs. Lastly, we're pleased to announce this morning a new non-dilutive $275 million debt facility with funds managed by Pharmacon advisors. As Mamie will discuss, this new facility extends our cash runway through the end of 2026, and with the commercial launch of SkyClarus, puts us on a path to self-sustainability. With that summary, I'd now like to turn the call over to Dawn Burr, who will provide an update on our commercial launch for SkyClarus.

Thank you, Warren. Good morning. I'll continue on slide eight. Friedrich's ataxia represents a significant commercial opportunity, and we believe that there are approximately 6,000 patients in the United States living with FA today. Through ICD-10 claims data analyses, we see approximately 5,000 unique diagnosed FA patients that can be linked to healthcare providers, excluding approximately 10% of those diagnosed under the age of 18, The remaining 4,500, or approximately 90%, represent our total on-label addressable market. Most patients seek routine care by their local neurologist or primary care physician. Important commercial launch targets include CCRN centers or collaborative clinical research network sites, ataxia centers, and HCPs currently treating FA patients. Through the evaluation of claims data, we've identified approximately 2,500 healthcare target providers treating patients with Friedreich's ataxia. These HCPs and the diagnosed FA patients they treat are the primary focus of our commercial launch efforts this year. Next slide, please. Patients and healthcare providers have long awaited an approved treatment for Friedreich's ataxia. and this is evident through the demand we see for SkyClaris in only two months following approval. REATA REACH is our single point of contact for our patient services program and serves as the intake center for all SkyClaris patient start forms. Start forms received by REACH are an early indicator of our launch progress. Through early May, we've received approximately 500 SkyClaris patient start forms submitted by over 250 prescribing physicians, including neurologists, PCPs, and other healthcare providers. 500 FA patients represents over 10% of our current total addressable market. We are pleased with this quick uptake, which reflects strong demand. Patients are actively seeking treatment, and healthcare providers are willing to prescribe SkyClarus. Next slide. Our 2023 launch objective remains unchanged. Our goal is simple, to establish SkyClarus as the first and only effective and safe treatment approved for Friedreich's ataxia. Our commercial launch activities engage our three important stakeholders. This includes HCPs currently treating Friedreich's ataxia with patients in their practice today, on-label patients diagnosed with FA, and the payer community. With our attention on physicians currently treating patients with FA, we continue to communicate the value of SkyClarus, the significance of our clinical data, and how this data translates to a clinically meaningful impact on the disease. Additionally, we are working to ensure all HCPs understand how to access SkyClarus for their patients and the programs we offer to support utilization. FA patients and their caregivers are also important stakeholders. Because Skyclaris is the first and only drug approved for Friedreich's Ataxia, we continue to inform and educate patients and their families on this approval, and we encourage them to see their healthcare provider for treatment. To support these educational efforts, we recently launched our branded patient and HCP campaigns, highlighting the recent approval and communicating that Friedreich's Ataxia, previously untreatable, is now a treatable disease. Immediately following approval, RIADA created a strong market presence and engaged the FA community with our first branded booth at important neurology conferences, including the Muscular Dystrophy Association, the National Ataxia Foundation, and the American Academy of Neurology. Now approved, digital, social, paid search, and patient webcasts were launched in March and April to drive the awareness of SkyClarus and provide clarity on how to access treatment. Lastly, critical to our launch success and quarter our patient promise, we continue to facilitate payer coverage, access, and affordability through payer education and robust programs designed to minimize or eliminate patient out-of-pocket cost burdens. Continuing on slide number 11, Riyadh's field sales and market access teams are executing on our branded launch strategy, working to drive demand and facilitate payer coverage. Our experienced and fully trained teams began engaging customers immediately following approval on Monday, March 6th. The sales organization is working to reach and educate approximately 2,500 healthcare providers who treat most of the diagnosed FA patients in the United States. They have also engaged FA treatment centers, driving early utilization at each of the nine U.S. Centers of Excellence and implementing plans to support the needs of these highly important accounts. Our field access team consists of national account directors focused on SkyClaris coverage by top national and regional payers, and a patient access liaison team hired to educate practices on access requirements. Together, this team's primary responsibility is to facilitate patient access to the drug by working to minimize and navigate payer criteria. Since FDA approval of SkyClarus, the payer team has engaged all of the top US payers representing 90% of covered lives. We anticipate that most commercial payer policies will be established during the second half of 2023 and that plans will place SkyClarus on their specialty tier along with most rare specialty therapeutics. Our patient access liaison team has worked with local practices to educate on access requirements and facilitate rapid payer approvals through medical exception and prior authorization while awaiting payer policies. We are pleased with early payer coverage of SkyClarus, tracking as we expected for a rare, progressive, and devastating disease with no other approved treatment options. I will now turn the call over to Andrea Loewen, our Senior VP and Head of Global Regulatory Affairs for the Regulatory Update on Drug Availability and Omaviloxelone MAA status.