KemPharm, Inc.

Good day and welcome to the Zevra Therapeutics fourth quarter and full year 2022 update call. Currently, all callers have been placed in a listen-only mode and following management's prepared remarks, the call will be open for questions. If you would like to ask a question at that time, please press star one on your telephone keypad. If you need to remove yourself from the queue, please press star two. At any time, if you should need operator assistance, please press star zero. Please be advised that today's call is being recorded. I want to hand the call over to Zebra's Vice President of Investor Relations and Corporate Communications, Nicole Oshner. Thank you. You may begin.

Good morning. Thank you for joining us today to review Zebra's fourth quarter and full year 2022 clinical operational progress, and financial results. A few housekeeping items before we start. I invite you to view the webcast slides and the press release, both of which were issued this morning and can be found in the investor section of our website. As outlined on slide two, during today's presentation, we will be making several forward-looking statements. These forward-looking statements include but are not limited to the clinical and regulatory path for Aramaclimal, ASTARs, revenue milestones and expected revenue from the French EAP program, the planned next steps in our pipeline programs, the timing for enrollment, initiation, the completion and reporting of data from our clinical trial, and the upcoming annual meeting of stockholders. These statements are neither promises nor guarantees, and involve risks and uncertainties and other important factors that may cause actual results to differ materially from those discussed here. Important factors that could cause actual results to differ from forward-looking statements can be found in the risk factor section of our annual report on Form 10-K for the full year ended December 31st, 2022, expected to be filed with the SEC today. you can view the agenda for today's call on slide three. I am pleased to welcome Zebra's management team members participating in today's call. I'm joined today by our CEO, Richard Pascoe, our president and co-founder, Dr. Travis Mikkel, our chief financial officer, LaDwayne Clifton, our chief product development officer and co-founder, Crystal Mikkel, and Joshua Schaefer, Chief Commercial Officer and Executive Vice President, Business Development. I will now turn the call over to Rich Pascoe.

Thank you, Nicole, and thank you all for joining us today to review our Q4 and full year 2022 results. Before we get into the highlights of the successful year, I want to take a step back and talk about where we are today and provide an overview of our strategic evolution over the past 18 months. A lot has happened and with a new name and focus, It is important to walk our investors through how we got here and share my enthusiasm for our future. Today we are Zebra Therapeutics, a nimble and focused rare disease company. Our team has a successful track record of success in championing patients in need, as well as tackling and overcoming challenges in drug development and regulatory approval. We are leveraging our years of expertise and unique insights to chart a new course for drug development And forging pathways to success for promising product candidates. As we move Zavra forward, we will continue to leverage our legacy platform to target internally discovered rare disease product opportunities and extend our pipeline assets exclusivity through lifecycle management. In this way, we bring transformational therapies and hope to patients with rare diseases and their families and create value for our shareholders. Importantly, we are well positioned for success We have a robust pipeline of promising rare disease product candidates with multiple value-creating milestones expected over the coming year. We have a world-class leadership team with the experience of numerous successful product approvals and an equally accomplished board of directors that align with our strategic trajectory in rare disease. Supporting it all, we have a strong balance sheet. So how did we get here? We've worked hard to evolve the company's strategy over the past two years, thoughtfully led by our management team in collaboration with our board of directors and supported by our shareholders. After the approval, licensing, and commercialization of Astaris and our actions to put the company on a firmer financial footing, the management team and board paused to evaluate where we were and what we learned in getting Astaris to the market. Our objective was to determine our best path forward to create value in a timeframe and at a scale that our shareholders deserve. We concluded that we have strong capabilities and success in the scientific, development, and regulatory areas. However, focusing exclusively on a pipeline of product candidates that will eventually compete in heavily generic markets and outlicensing those products as a commercial strategy would likely fail to meet our objective of delivering a significant return on investment to our shareholders. We then worked to thoughtfully set a new course for the company, focusing on bringing the organization's existing strengths together with a refined strategic focus on rare disease indications with the goal of commercializing our products in the United States. We took a series of transformative actions that aligned perfectly with our strategy, highlighted by the acquisition of Aramoclomol, an NDA stage product candidate, upon which we intend to build commercial capabilities that will allow Zebra to create value for the benefit of shareholders. This new direction capitalizes on our strengths and cash position to enable us to retain the value of our hard-earned approvals and control our commercial destiny while delivering much needed therapeutics to patients with limited or no treatment options. It is important to note that rare disease is not an entirely new focus for us. We were already working in rare disease with our prodrug technology applied in rare sleep disorders with KP1077 for idiopathic hypersomnia. From that experience, we recognized that rare disease drug development offers several significant advantages over development in large mass market indications. First and foremost, we have the privilege of partnering with patients, families, and advocacy groups to deeply understand their needs and ensure that our therapeutic approaches are answering their biggest quality of life and disease management concerns. This ensures our products are relevant and capable of commercial success. Rare disease drug development timelines are often shorter and involve much smaller studies than larger indications, resulting in lower costs of research and development. Through the Orphan Drug Act, the company benefits from incentives such as priority review voucher, and a six-month review window for pursuing therapeutics for rare diseases. And the resulting products receive extended patent protection for longer market extensivity. Rare disease products also do not experience significant generic competition once patents expire. Moreover, with a small patient population treated primarily by specialist clinicians, we can bring those products to commercialization with our own in-house commercial team. Taking this together, we saw that rare disease drug development creates an ideal platform for maximizing value creation for patients and shareholders. This led to our decision to focus exclusively on rare disease and our strategic acquisition of Aramaquamol. Through that acquisition, we acquired a high potential product candidate for Niemann-Pick disease type C. We also onboarded an amazing European team with deep connections to the MPC patient community and significant rare disease drug development expertise. As we began focusing on rare diseases, we recognized that our name and branding as ChemPharm no longer reflected who we are as a company. So we underwent a process to redefine our messaging and brand story. The outcome of that process is our new name, Zevra Therapeutics, and branding to reflect our commitment to the rare disease patients we deserve. The word Zevra is Greek for zebra, which is the internationally recognized symbol for rare disease community. Interestingly, a group of zebras is also called a zeal or a dazzle. These expressions capture our team perfectly as we are zealous about empowering people with rare diseases. We are confident that Zebra is a name that will resonate with patients, and we envision a dazzling future for the rare disease communities that we serve. Zebra is better positioned today than at any point in its history as we work towards our key priorities to secure regulatory approval for our pipeline assets build top-tier commercial capabilities, and enhance our pipeline through internal and external efforts. We have two very strong product candidates with multiple value-creating milestones expected this year. We continue to explore new product opportunities, both through our pro-drug platform and business development activities focused on rare disease. We are a rare beast in that we already have a growing revenue stream as a development stage company, which bolsters our already strong financial position. I am confident in the team and our strong financial foundation and diverse portfolio of multiple clinical programs and in our ability to deliver value in 2023 and beyond. Importantly, we have a fully engaged and supported board of directors that has been deeply involved in our strategic evolution and can help us to advance our company. 2022 is a pivotal year for our organization and our evolution into a rare disease company with the acquisition of Aramaka Mall. and the completion of a four-year safety study of that product candidate in NPC. Our ongoing collaborative dialogue with the FDA around the program has been highly productive in preparing us for NDA resubmission. We've also made tremendous headway in our KP1077 program for rare sleep disorders over the last year, including positive data from our cardiovascular trial of SDX, a primary component of KP1077, and a Phase II trial initiation in idiopathic hypersomnia. Orphan drug designation for this product candidate in IH provides the advantages I highlighted earlier, including regulatory incentives and extended market exclusivity after approval. We continue to bolster our strong financial position, earning a one-time fee of nearly $2 million from Corium following the FDA approval of Adlarity. Our net revenue for the full year was $10.5 million. Cash equivalents and investments as of December 31st, 2022 were $102.9 million. Our available capital is expected to extend our cash runway into 2026, an enviable position for any biotech company in today's capital markets. In 2022, we began our efforts to further build our team by appointing Nicole Oshner as VP of Investor Relations and Corporate Communications. In addition, the board appointed Christopher Posner as an independent director, another great addition to the team. As you can see from this slide, we've had tremendous momentum in the fourth quarter of 2022 and the beginning of 2023 on several fronts. I want to point out a few of those. Notably, a recent development in the Aramaka Mall program was a presentation of the data from the interim analysis of the Phase 2-3 four-year safety study at the 2023 World Symposium. These data, which demonstrate the clinical benefit of Aramoclomol, will be a cornerstone of our resubmission of a new drug application expected as early as the third quarter of this year. Notably, the data suggests that Aramoclomol may reduce the progression of NPC. This underscores our understanding that if you can improve lysosomal function, you can improve outcomes for patients. We also underwent our strategic rebrand to Zebra Therapeutics in alignment with our identity as a rare disease company. and to further engage with the rare disease community we joined, the Nord Corporate Council. Since the beginning of the year, we have made several additional key appointments and changes to our leadership and board of directors, strengthening a team that I'm confident can lead Zevra into the future with success. Shown here are our executive team and our board of directors. Not only are we an accomplished group with decades of experience in R&D and commercialization leadership, but we are also a blend of legacy team members and new talent with skills that can take us into the future. The team is aligned with our direction and transformation into a rare disease company. Now I'd like to turn our attention to our Aromacromol program for Nevin Pick Disease Type C. Aromacromol is administered orally and is formulated for ease of use in varying patient circumstances. This product candidate has been studied in 10 Phase I, 4 Phase II, and three pivotal phase 2-3 trials. Aramoclomol has received orphan drug designation for NPC in the United States and the European Union, and fast track designation, breakthrough therapy designation, and rare pediatric disease designation from the FDA for NPC. If approved in the U.S., Aramoclomol would also be eligible to receive a pediatric priority review voucher. We are on track to submit the new drug application to the FDA as early as the third quarter of this year for Aramaka Mall. One of the most attractive aspects of the Aramaka Mall program is that should it be approved, we have the near-term opportunity to commercialize the product ourselves and retain the full market value for our shareholders. We believe a small and nimble commercial team could fully serve the specialists and medical centers treating the MPC patient population. The benefit of a small commercial team is the lower cost of market entry, We believe that our established and new advocacy relationships in the NPC community will help support the adoption of Aramoclomol. And we have already established market entry with this patient population through our U.S. and EU early access programs. Beyond our immediate focus on a successful U.S. commercial launch, we also see the commercial potential for Aramoclomol outside of the U.S. in the EU, Japan, China, and other geographies. Now I will highlight our KP-1077 program for rare sleep disorders, beginning with idiopathic hypersomnia, which is currently enrolling patients and dosing patients in a Phase II clinical study in the US. The market potential for idiopathic hypersomnia is also highly compelling, with limited treatment options available for this small, underserved patient segment. KP-1077 has been designated by the FDA as an orphan drug, as well as possible eligibility for fast-track review status and designation as a breakthrough treatment. In addition, we believe that if differentiated from other treatment options, pricing KP-1077 in between products like Teva Pharmaceuticals ProVisual, which is approximately a $24,000 per year treatment at the highest dose, and Jazz Pharmaceuticals Zywave, which is approximately $187,000 per year at the highest dose, could lead you to capture capturing significant market share. Because this is a rare indication, our intent is the commercialization of this product ourselves, which is aligned with our strategy for Aramocamol, and therefore allows us to keep a larger portion of the economic value. We expect interim data from the phase two trial of KP1077 in idiopathic hypersomnia by the third quarter of this year, and top line data by the end of 2023. Importantly, the interim data will also be an open-label titration phase, which is meaningful because it will allow us to start designing the Phase III study in IH. We are also planning a development program for KP1077 and narcolepsy. The IND filing for this additional program is expected during the second quarter of 2023. With that, I will turn the call over to our CFO, LeDwayne Clifton. LeDwayne?