Zevra Therapeutics, Inc.

Good afternoon, everyone. Thank you for joining the Zebra Therapeutics Q4 2023 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 calls. With that, I'll now turn the call over to Nicole Oshner, Vice President of Investor Relations and Corporate Communications for Zebra Therapeutics.

Good afternoon, and thank you for joining us today to review Zebra Therapeutics' progress in the fourth quarter and full year of 2023, 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 just published and available in the investor relations section of Zebra's website. As we proceed with this 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 10-2 and our other filings with the SEC and annual report on Form 10-K. I'm pleased to welcome Zevra's management team members participating in today's call. I'm joined by Neal McFarling, President and Chief Executive Officer, LaDwayne Clifton, our Chief Financial Officer, Joshua Schaefer, our Chief Commercial Officer and EVP of Business Development, Crystal Mickel, our Chief Development Officer, and Adrian Cortell, our Chief Medical Officer. Now, I'll turn the call over to you.

Thank you, Nicole, and thank you all for making the time to join us today. During the fourth quarter and into 2024, we made solid progress towards transforming Zevra into a commercial stage company. On our last call, we announced that we were focused on three key priorities. First, to close the ACER acquisition and deliver value to patients by commercializing Olpruva. Second, to resubmit the Arimathamol NDA. Third, to complete the Phase II trial in idiopathic hypersomnia and prepare to advance KP1077 into Phase III. I'm happy to report that we executed on all of these objectives. And I would like to take the opportunity to recognize the extraordinary effort from our entire team to deliver for people living with rare diseases. Before discussing our results, it's important to note that our financial statements for fiscal year 22, including all interim periods and the interim periods of 2023, will be restated due to a change in our warrant accounting. LaJoy will provide more details later on the call, but we believe the restatement will have no impact on the company's cash or ability to execute on our strategic priorities. Turning to the fourth quarter corporate highlights, let me start with the completion of the ACER acquisitions, which propelled us into becoming a commercial stage company, diversifying our revenue potential and providing scale. The acquisition was a natural fit with Zebra's mission, bolstering the talent on our team and bringing complementary rare disease assets, including commercially available Opruba. Opruba 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 UCDs, of which roughly half are diagnosed and treated. The UCD market in the U.S. is estimated at approximately $350 million annually. Despite the available therapies, unmet needs for people living with UCD persists. We believe that Olkruva is well-suited to address these needs as it provides personalized dosage for each patient's requirements, It's 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 that is associated with other formulations of sodium phenylbutyrate. Our commercial launch strategy is comprised of two major components. One, establishing a customer-facing team, and two, building awareness. Since the completion of the ACER acquisition in mid-November, we have made significant progress towards executing on these priorities, ensuring that people who suffer from UCDs have access to and are aware of the benefits of Olpruva. As of the end of January, we have a customer-facing team with decades of rare disease experience to support the launch of Olpruva. This team was built to be targeted and able to reach the needs of our customers and partners. most of whom are located in approximately 40 centers of excellence across the country. In addition to the sales specialists, we have marketers, patient services, and market access professionals, as well as medical science liaisons and patient advocates for engaging with key customers. While initially built to support the launch of Oprima, this same group of professionals will launch Aramacamal if approved. We've initiated several strategies that are being utilized to build awareness for El Provo, which is currently quite low. For example, we have established Quick Start, which is a 30-day free trial to allow patients and physicians to gain experience with El Provo. We're working with patient advocacy groups, the patient community, and UCD centers of excellence to drive brand recognition. We're also working with payers to ensure broad market access for patients. We've seen a meaningful growth in reimbursement coverage which was approximately 55% at the time of acquisition, to now more than 70% of covered lives. While it's too early in the launch to provide data on today's call, we're monitoring key launch performance indicators, including new patient enrollments, number of covered lives, and net revenues. As previously mentioned, the commercial footprint we established provides a high strategic fit for Aramothymol as the majority of prescribers for both products work within the same centers of excellence. If Aramothymol is approved, we believe this close proximity and overlap in patient care will allow us to realize synergies and scale with the infrastructure that we've built. As a reminder, Aromalcumol is our drug candidate in development for the treatment of Niemann-Pick disease type C, or MPC. MPC is a rare genetic, progressive, and potentially fatal neurologic disease. Earlier this month, the FDA assigned a new PDUCA date of September 21, 2024, and reaffirmed its intent to present the resubmission or discussion at an advisory committee meeting. If approved, We intend to utilize our clinical data as well as real-world evidence and the data from our expanded access program to support market access, reimbursement, and treatment decisions to establish Aramatha Mall as the foundation of treatment for people with MPC. We will continue to work closely with key opinion leaders to educate on Aramatha Mall's clinical profile and raise awareness of the heterogeneous presentation of MPC. which may include neurological and psychiatric symptoms, all of which make MPC difficult to identify and diagnose. Because of this, the time to diagnosis remains a significant unmet need in the MPC community. Therefore, we're working with patient advocates to drive early diagnosis and supporting efforts for MPC to be included in newborn screening. Together with an approved indication, these initiatives will help drive the evolution of treatment guidelines and accelerate the time to diagnosis and treatment initiation. We will continue to work with all stakeholders to develop patient services that will provide access and a positive experience. We applaud the NPC patient advocacy community who united and submitted a compelling response through an informal petition to the FDA in support of Aramathamal's approval. They received nearly 1,000 signatures from 47 states forcing their support. As the FDA review continues, Zebra will maintain our expanded access program for Aromalconol and continue working tirelessly to bring this potential therapy to patients as soon as possible. Now I'd like to turn your attention to KP1077, our clinical candidate being developed as a treatment for idiopathic hypersomnia, or IH. IH is a rare chronic sleep disorder characterized by excessive daytime sleepiness an uncontrollable need to sleep, and difficulty waking up from sleep in most instances, despite average or longer amounts of nocturnal sleep. As you may recall, Kp1077, Sert-X methylphenidate, or SDX, was designed to steadily release D-methylphenidate, its active ingredient. This unique pharmacokinetic profile allows for flexible dosing to overcome these primary IH symptoms. The design also ensures that patients receive the highest drug concentration when they need it most. SDX is designated as a Schedule IV controlled substance by the U.S. Drug Enforcement Administration. Earlier this week, we announced positive top-line data from our placebo-controlled, double-blind, proof-of-concept Phase II study evaluating the safety and tolerability of Kp1077 in patients with IH. Consistent with the interim data that we previously reported in Q4, KP1077 was well tolerated at all dose levels evaluated in the study, including the highest dose of 320 milligrams daily and at dosing regimens of either once or twice daily. The most common adverse events were insomnia, headache, anxiety, nausea, and decreased appetite. Due to KP1077's unique pharmacokinetic profile, adverse events were mostly mild in severity despite higher overall exposure levels. These data support the study's primary endpoint of safety and tolerability. Topline results from the Phase II study also showed that KP1077 produced clinically meaningful improvement in excessive daytime sleepiness, or EDS, as assessed by change from baseline in the F4 sleepiness scale during the five-week open-label titration period, which was maintained throughout the two-week double-blind withdrawal period for both dose regimens. Additionally, patients administered KP1077 showed benefit in change from baseline at the end of the open-label titration. and at the end of the double-blind withdrawal period for the IH severity scale, the sleep inertia visual analog scale, and brain fog severity scale. The study successfully fulfilled the objectives by providing key information for the design of a potentially pivotal efficacy trial, and the results of the secondary efficacy endpoints are supportive of initiating a Phase III trial of KP1077. We plan to request and end the Phase II meeting with the FDA to seek guidance on the Phase III clinical trial design. We are pleased with the top-line data and believe that KP-1077 could provide a significant benefit to the estimated 37,000 people in the U.S. who are currently diagnosed with IH. With only one FDA-approved treatment for IH, there remains an unmet need for therapies with different mechanisms of action to address symptoms including sleep inertia, excessive daytime sleepiness, and cognitive dysfunction. We look forward to presenting the results from our phase two study at the upcoming Sleep 2024 conference this summer. And in summary, we're pleased with our progress in the fourth quarter. As we enter 2024, we have three areas of focus. First, to successfully launch Olpruva and ensure access for patients. Second, to prepare for the potential launch of Arumacumab. And third, to advance KP-1077 in sleep disorders. We believe that we are all, we believe that we are well positioned to continue to execute and deliver on these key strategic objectives. Now I'll hand the call over to LeBlanc who will provide an update on our financial results and outlook.