Zevra Therapeutics, Inc.

Good morning, everyone. Thank you for joining the Zebra Therapeutics first quarter 2024 corporate updates and financial results call. Today's call is being recorded and will be made available on the company's website following the conclusion of the call. With that, I will now turn the call over to Nicole Oshner, Vice President of Investor Relations and Corporate Communications at Zebra Therapeutics.

Good morning, and thank you for joining us today to review Zevra Therapeutics' progress in the first quarter of 2024, outlining our clinical advances, operational achievements, and financial results. Before we get started, let me take a moment to provide some important information. I encourage you to access the news release which was published this morning and is available in the investor section of Zevra's website. As we begin our call, it's important to highlight that today's discussion will include forward-looking statements. Forward-looking statements are not promises or guarantees and are inherently subject to risks, uncertainties, and other significant factors that may lead to actual results differing materially from the projections made. Please refer to the risk factors section in our most recent quarterly report on Form 10-Q and other filings with the SEC an annual report on Form 10-K. I am pleased to welcome Zebra's management team members participating in today's call. I'm joined today by Neal McFarlane, President and Chief Executive Officer, LaDwayne Clifton, our Chief Financial Officer, Joshua Schaefer, our Chief Commercial Officer and Executive Vice President of Business Development, Crystal Mickel, our Chief Development Officer, and Adrian Quartel, our Chief Medical Officer. Now, I'll turn the call over to Neal.

Thank you, Nicole, and thank you all for making the time to join us today. During the first quarter, we made steady progress in executing on our strategic objectives. On our last earnings call, we announced that we were focused on three key priorities. First, to successfully launch Alprova and ensure access for patients. Second, to prepare for the potential launch of Aramacumal. And third, to advance the KP-1077 program in sleep disorders. I'm pleased to report that we are executing on all of these objectives, and today, we'll share with you a summary of our key accomplishments in the first quarter and the reasons we're optimistic for 2024 and beyond. In addition to executing on our three key priorities, we refinanced our existing debt with up to $100 million in committed capital, led by premier biotech investors, including perceptive advisors and healthcare royalty partners. This new credit facility, which LaDoyne will cover in more detail, has further strengthened our balance sheet and provides added capital flexibility to support our mission. The company continues to work through a period of significant growth and transformation, with the addition of talented people and capabilities from each of the three companies that have now come together to become one Zebra. The team is making significant progress advancing our rare disease portfolio, and in Q1, we initiated a long-range planning process including a portfolio prioritization review with a focus on building a sustainable business with reliable cash flows. Achieving our strategic objectives to commercialize Olpruva and then upon approval, successfully launching Aramacabal are key to reaching that seminal inflection point. We initiated the full commercial launch of Olpruva at the end of January 2024. As a reminder, Olpruva is indicated for the treatment of certain urea cycle disorders or UCDs, which are a group of rare genetic disorders that can cause harmful levels of ammonia to build up in the blood, potentially resulting in neurocognitive impairments, brain damage, and in some cases, coma or death. We estimate that there are approximately 2,000 people in the U.S. with UCD, of which roughly half are diagnosed and treated. The UCD market in the U.S. is estimated at approximately $350 million annually. Despite the availability of therapies, unmet needs for people living with UCD persists. We believe that I'll prove as well suited to address these needs as it provides personalized doses for each patient's requirements. It is portable and easy for a patient to take. And most importantly, it is palatable as it was formulated to overcome the challenging taste and smell associated with other formulations of sodium phenyl butyrate. Since launch, We've been focused on raising the awareness of OPRUVA and demonstrating our commitment to UCD patients. In the quarter, we had four new patient enrollments, which we define as a prescription for a patient eligible for benefits investigation or a quick start program. We are encouraged by the progress that our rare disease specialists accomplished in the first few months of launch, meeting with more than 90% of the specialists at the 40 centers of excellence that treat people with UCD. We've also assembled a team of marketers, patient services, and market access professionals, as well as medical science liaisons and patient advocates who are engaging with key stakeholders at patient events and medical conferences. Our managed care team has been working with government and commercial payers to ensure broad access for patients. we have seen meaningful growth in reimbursement coverage, which was 55% at the time we acquired Opruba to nearly 75% of covered lives as of May 1st. Overall, I'm pleased with the products we've made in the first few months of launch and look forward to reporting more details as the launch matures. Zebra's commercial footprint was established to provide a high strategic fit between Olprova and Aramocamol, given that the majority of prescribers for both products work within the same centers of excellence. If Aramocamol is approved, we believe this close proximity and overlap in patient care will allow us to realize synergies and scale with our commercial infrastructure. In fact, our team has already begun to identify and compliantly engage with key opinion leaders and prescribers who treat UCD and Niemann-Pick disease type C, or NPC. Our market access team has initiated clinical discussions with payers regarding NPC and Aromathamol. These and other synergies across our brands will allow us to accelerate the launch of Aromathamol and ensure patients have access to this much-needed therapy. As a reminder, Aromacamol is our investigational drug candidate in development for the treatment of NPC, a rare, genetic, progressive, and potentially fatal neurologic disease. If approved, Aromacamol would be the first drug indicated for the treatment of NPC in the U.S. Currently, there are approximately 900 people living with NPC, of which roughly 300 are diagnosed. Of those, approximately 70 are enrolled in our Expanded Access Program, or EAP. During the quarter, the FDA assigned a new PDUFA date of September 21, 2024, and reaffirmed its intent to present the resubmission for discussion at an advisory committee meeting for which we are thoroughly preparing. The National Neiman Pick Disease Foundation, known as MNPDF, spearheaded efforts with six other advocacy and research organizations to compile a petition of nearly 1,000 signatures from NPC patients, caregivers, and physicians with direct experience utilizing Aromalcumol that was submitted to the FDA in Q1. We were overwhelmed by the outpouring of support for Aromalcumol within the community. As the FDA review continues, we will maintain our EAP and continue working tirelessly to bring this potential therapy to patients living with this devastating rare disease. We continue to work closely with key opinion leaders to educate on Aramoclomol's disease-modifying clinical profile and raise awareness of the heterogeneous presentation of MPC. In April 2024, we presented new long-term real-world data during the Society for Inherited Metabolic Disorders. The presentation included data from EAP patients from the U.S. centers, and it demonstrated that adults treated with aromathemol, including those with and without amiglizumab use, had stable disease course, which is defined as not showing disease progression over the two years of treatment. The safety profile was consistent with that observed in the Phase 2-3 study, if approved, We intend to utilize our clinical data as well as emerging real-world evidence from our EAP to establish Aromothymol as foundational treatment for people living with MPC. Now I'd like to turn your attention to KP1077, our clinical candidate being developed as a treatment for idiopathic hypersomnia, or IH, a rare chronic sleep disorder. IH is characterized by excessive daytime sleepiness or uncontrollable need to sleep and difficulty waking. There remains an unmet need, and we estimate 37,000 people in the U.S. are currently diagnosed with IH. As you may recall, KP1077, Sirdex methylphenidate, or SDX, was designed to steadily release demethylphenidate, its active ingredient. This unique pharmacokinetic profile allows for flexible dosing to overcome these primary IH symptoms. This profile ensures that patients receive the optimal drug concentration during waking and active hours. SDX is currently designated as a Schedule IV controlled substance by the U.S. Drug Enforcement Administration. During the quarter, we reported top-line data from our Phase II study of KP1077 in patients with IH. We are encouraged by the results, which show that KP1077 is well-tolerated and demonstrates early signs of differentiated and clinically meaningful benefits. The study successfully fulfilled the objectives of informing the design of a pivotal efficacy trial, and we are planning for an end-of-Phase II meeting with the FDA in Q3. Since completing the trial and reporting top line results in March, we've been working closely with the sleep community to interpret these results. With only one approved treatment, there remains a large unmet need for therapies with different mechanisms of action to address the symptoms of IH. We look forward to presenting the full data package from our completed study at the upcoming Sleep 2024 conference in early June. As part of the strategic planning initiative kicked off in January, We completed our preliminary evaluation of the Solipilol program for the treatment of vascular Ehlers-Danlos syndrome, or VENS, which impairs COL381 connective tissue and leads to vascular and hollow organ ruptures. Solipilol's mechanism of action is designed to reduce the mechanical stress on collagen fibers within the arterial wall through vascular dilation and smooth muscle relaxation. Solipilol received orphan drug and breakthrough therapy designations from the FDA. The Phase II protocol is being conducted under a special protocol assessment, or SPA agreement, with the FDA. We recently restarted recruitment of the Phase II trial, also known as the DISCOVER trial, to support patients currently enrolled and to preserve the value of the program while we complete our portfolio review. This is a decentralized event trial design of Solipilol on VEDS-related event reduction. Solipilol is a primary treatment option in various European countries, and we believe that it could address the significant unmet need in the U.S. as there are no approved treatments for the 7,500 diagnosed patients with VEDS. Looking ahead, we have three areas of focus. First, continue to drive the launch of Olpruva. Second, to prepare for a potential adcom and launch of Aramacoma. And third, to advance KP-1077. Now I'll hand the call over to LeDwayne, who'll provide an update on our financial results.