Zevra Therapeutics, Inc.

and thank you for joining the Zephyr Therapeutics Q2 2024 Financial Results and Corporate Highlights 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 for Zephyr Therapeutics.

Good afternoon, and thank you for joining us today to review Zebra Therapeutics' progress in the second 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 afternoon and is available in the investor section of the Zebra website. As we begin our call, it's important to highlight that certain information covered in today's discussions will include forward-looking statements. We caution listeners that actual results could differ materially from these stated or implied by these forward-looking statements due to risks and uncertainties associated with the company's business. 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 those projections made. These forward-looking statements are qualified by the cautionary statements contained in the risk factors section in our most recent quarterly report on Form 10-Q and our other filings with the SEC annual report on Form 10-K. I'm pleased to welcome Zephyrus Management team members participating in today's call. I'm joined by Neal McFarlane, President and Chief Executive Officer, LeDwayne Clifton, our Chief Financial Officer, Josh Schaefer, our Chief Commercial Officer and Executive Vice President of Business Development, and Adrian Cortell, 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 second quarter, we've made steady progress executing on our strategic objectives, preparing for the advisory committee meeting and the potential launch of Aramaca Law, driving the launch of Olpruva, and advancing KP 1077 for sleep disorders. These objectives are important building blocks for our long-term strategic plan to build a sustainable rare disease company with reliable cash flows. An important element of executing against our strategy is to build and maintain our position of financial strength. During the first half of 2024, we made investor outreach a primary objective, highlighting the opportunities and catalysts for value creation that we have as a company. There has been a significant interest and growing momentum as we continue to execute our objectives. Last week, following the favorable outcome of the FDA Advisory Committee meeting focused on Aramocumal, we undertook a modestly-sized, underwritten public offering to capture that momentum and build on our base of investors as we lean into the potential of our near-term catalysts. With this funding, we have added net proceeds of approximately $64.5 million to our balance sheet, bringing our pro forma June 30th, 2024 cash, cash equivalents and investments to $113.8 million. This was the right time to demonstrate our confidence and take this important step to prepare for success. The proceeds will extend our cash runway and bolster our flexibility in executing both near and long-term objectives, including full preparation for the potential launch of Aramocumal and the flexibility to accelerate our clinical pipeline. Now, I'll share a summary of our key second quarter accomplishments and outline why we remain optimistic in our ability to deliver on our strategic plan. Let's start with Aramocumal. our product candidate for Niemann-Pick disease type C, or NPC. On August 2nd, the Genetic Metabolic Diseases Advisory Committee, or GEMDAC, voted favorably that Aromalcumol is effective in the treatment of NPC. While the vote is not binding, we believe this is an important factor as the FDA completes its review. Our PDUFA date is September 21st, which is fast approaching. We received the first round of labeling comments last Friday and are working closely with the FDA. If approved, Aromothymol would be the first drug in the U.S. indicated for the treatment of NPC, and it would be eligible for a priority review voucher. As a reminder, there are roughly 900 people in the U.S. with NPC, of which approximately one-third are diagnosed and treated. More than 70 of those patients are currently enrolled in our U.S. Expanded Access Program, or EAP. As the FDA review continues, we will maintain the U.S. EAP to ensure access for patients until commercial supply is available. In addition, subsequent to U.S. approval, we will seek regulatory approval in Europe, where an additional 70 to 80 patients are enrolled in our global EAP program. We are actively preparing for the commercial launch of Aromalcumal. Our commercial infrastructure was built to optimize the strategic fit between Olpruva and Aromalcumal. Both products address genetic metabolic diseases with multidisciplinary treatment teams that are often co-located within the same centers of excellence, allowing us to reach the majority of prescribers with a targeted commercial infrastructure. Our rare disease specialists and medical science liaisons who are currently promoting and educating on olprova and urea cycle disorders, or UCDs, are also engaging with prescribers when appropriate to raise awareness for NPC. Further, our market access team has initiated pre-approval clinical discussions with payers regarding aromathemol as a potential treatment for NPC. These pre-launch activities will help ensure patients have access once available. Now, turning to OPruva, where we continue to make process with the commercial launch initiated at the end of January 2024. Over the last few quarters, our assumption on the limited awareness of OPruva has been confirmed. However, we've made progress with healthcare providers to increase awareness levels and identified that we have more work to do to increase patient awareness. As a reminder, there are 1,100 UCD patients in the U.S., of which more than 800 are receiving treatment. The prescribing community has identified a significant number of these patients who can benefit from Opruba. While we are encouraged by their response, the number of patient enrollments is not yet where we would like it to be. During the second quarter, we had nine new patient enrollments, which we define as a prescription for a patient who's on our quick start program or one who's receiving a paid dispense. We have been working to build awareness amongst clinicians who treat UCD and to ensure market access for patients. The team has done an outstanding job of engaging HCPs at target centers of excellence and at medical conferences to build brand awareness. The team has successfully engaged the majority of clinicians and thought leaders who diagnose and treat UCD patients, which is remarkable since access to HCPs has become more challenging across the industry. Additionally, our managed care team continues to engage with government and commercial payers to ensure broad access for patients. We have increased OPROVA coverage to 75% of covered lives with improved formulary status across healthcare plans and have established comprehensive patient services programs designed to assist with the reimbursement hurdles experienced by the rare disease community. As I mentioned earlier, there are always opportunities for refinement during launch, and we are implementing changes to improve patient engagement. One key change was our transition to Orsini as our specialty pharmacy partner, who is a leader in pharmacy solutions for rare diseases. This transition completed in mid-June, including a rebalancing of our Olpuba inventory in the channel, which impacted our net sales revenue in the quarter. LeDwayne will provide more details later in the call. We believe these enhancements to our commercial infrastructure will further support the approval launch and will have a positive impact on our commercialization efforts for Aramocumal with limited incremental cost. Now I'd like to turn your attention to KP1077, our clinical candidate for the treatment of idiopathic hypersomnia, or IH, a rare chronic sleep disorder. IH is characterized by excessive daytime sleepiness and difficulty waking, also known as sleep inertia. This disease impacts approximately 37,000 people in the U.S. As you may recall, KP1077 is comprised of Cerdex methylphenidate, or SDX, which was designed to steadily release the methylphenidate, its active ingredient. Its unique pharmacokinetic profile allows for flexible dosing to overcome these primary IH symptoms and ensures patients receive the optimal drug concentration during waking hours. SDX is currently designated as a Schedule IV control substance by the U.S. Drug Enforcement Administration due to demonstrated lower risk for abuse potential. At the SLEEP 2024 meeting in June, we presented the pharmacokinetics of SDX when administered in the morning and at night. The clinical data showed peak exposure occurs the morning after a nighttime dose, when the patient needs it most to manage sleep inertia. We also reported positive results from our Phase II clinical study of Kp1077 in patients with IH. In this proof of concept study, Kp1077 was well tolerated at all dose levels, including the notably high dose of 320 milligrams daily. Adverse events throughout the study were mild, similar to other methylphenidate products, and did not lead to early discontinuations. AP 1077 showed clinically meaningful benefits in change from baseline at the end of seven weeks of treatment against secondary and exploratory endpoints, which included change in the Epworth Sleepiness Scale, the IH Severity Scale, the Sleep Inertia Visual Analog Scale, and a relatively new scale to assess the symptoms and severity of brain fog. We are encouraged by these results showing that KP 1077 is well tolerated and demonstrates clinically meaningful benefits. Importantly, the study successfully fulfilled the objectives of informing the design of a pivotal efficacy trial. We consulted with key opinion leaders, payers, and patient advocates knowledgeable in the rare sleep space to help interpret these results and have submitted a briefing book to the FDA for an end of Phase II meeting at the end of the third quarter. With only one FDA-approved treatment, there remains a large unmet need for therapies to address the symptoms of IH. We are conducting market research on the Phase II data to better understand KP1077's differentiated profile, position in the treatment landscape, and to inform our business case. Finally, we've made progress with Soliprolol, our product candidate for the treatment of vascular Euler-Danlos syndrome, or VEDS, which impairs COL3A1 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 is a primary treatment option in various EU countries, and we believe it could address the significant unmet need in the U.S., as there are no approved treatments for the 7,500 patients with VEDS. Soliprolol has received both orphan drug and breakthrough therapy designations from the FDA. During the second quarter, we restarted recruitment of the Soliprolol Phase III trial, also known as the DISCOVER trial. This decentralized, event-driven trial is being conducted under a special protocol assessment. We are encouraged by the significant interest among patients to enroll in this trial, which has exceeded our expectations. underscored the unmet need within the VEDS community, and preserves the value of the program while we conduct our portfolio review. As part of the strategic planning initiative kicked off in January, we continue to assess the value of each of our programs. Our intent is to fully understand the unmet needs of the rare disease patient community within a potential market, and then develop a solid clinical and business case for how ZEVRA can develop therapies to address those needs. Looking ahead, we have three key priorities. First, to receive approval and successfully launch Arimoclomol by leveraging the infrastructure built for Olpruva. Second, to drive the launch of Olpruva. And third, to discuss the design for a pivotal study evaluating the efficacy of Kp1077 in patients with IH in our end of phase two meeting at the end of Q3. We remain focused on execution to deliver a strong second half of 2024 and are well positioned financially to execute against those objectives. Now, LeDwayne will provide an update on our financial results.