Zevra Therapeutics, Inc. Common Stock 2026 Q2 Earnings Call

Thank you, Nichol, and welcome to everybody joining our quarterly call this afternoon. At Zevra, our mission is to redefine what is possible in bringing life-changing therapies to people living with rare disease. Our strategy to unlock long-term value through focused execution has translated into measurable results across our business. For the second quarter, our net revenue was $39.7 million, a 53% increase year-over-year. This was driven by $30.2 million in net U.S. revenue for MIPLYFFA. Our commercial progress reflects continued momentum in reaching patients and successfully establishing MIPLYFFA in combination with miglustat as a foundational treatment for Niemann-Pick disease type C, or NPC, in the U.S. As of June 30th, we had received a total of 184 prescription enrollment forms, 14 of which were received in the second quarter, bringing the total in the first six months of the year to 23.

We have strengthened our intellectual property position for MIPLYFFA with an Orange Book listing through 2041. We published long-term data from our U.S. early access program and the findings from our pediatric sub-study, further demonstrating the efficacy and safety of MIPLYFFA. We requested a re-examination of our arimoclomol Marketing Authorization Application, or MAA, by the Committee for Medicinal Products for Human Use, or CHMP, in the European Union. We continue to build a strong financial foundation with more than $260 million in cash equivalents, and investments.

Subsequently, we are advancing the re-examination process, which will provide the opportunity to address the specific grounds cited in the CHMP opinion and have our comprehensive evidence package reviewed by a new rapporteur and co-rapporteur. This process typically takes 120 days from the submission of a re-examination to the opinion from the CHMP, which is expected in Q4. Importantly, as we advance through the European regulatory process, our commitment to the patient and the NPC community remains steadfast. We will maintain access to arimoclomol for eligible patients through our global expanded access program, or EAP, which is comprised of compassionate use and reimbursed named patient programs that have enrolled 132 patients, with 10 added in the second quarter. As previously guided, we expect reimbursements beyond the French EAP to be highly variable in the first few years until the patient base has stabilized.

Ordering patterns for patients outside of Europe remain unpredictable, with some territories ordering a full year supply and others ordering on a monthly or quarterly basis. Simultaneously, we are evaluating opportunities to expand access to MIPLYFFA through the global EAP in response to strong interest from additional countries. This reflects our commitment to helping eligible patients outside the U.S. access treatment while building meaningful partnerships across the rare disease community.

Additionally, we strengthened our U.S. intellectual property position for MIPLYFFA with an Orange Book listing that provides protection through 2041. We are still awaiting the determination for the separately requested patent term extension through the U.S. Patent Office, which if granted, would fall within the Orange Book coverage period. Together, our IP portfolio provides a strong foundation for the long-term commercial opportunity for MIPLYFFA. We continue to invest in expanding and strengthening our protection beyond our current portfolio. At the same time, we are advancing celiprolol, a selective adrenoreceptor modulator that induces vascular dilation and smooth muscle relaxation for the treatment of Vascular Ehlers-Danlos Syndrome or vEDS. vEDS is a rare genetic connective tissue disorder caused by changes in the COL3A1 gene, leading to fragile walls in blood vessels and hollow organs, which can cause arterial rupture or dissection, among other complications.

Celiprolol has been shown to reduce the mechanical stress on these tissues. In our ongoing Phase III DISCOVER trial, we've enrolled a total of 66 patients, including 4 who were enrolled in the second quarter. As an event-driven study, 28 events are required to trigger the interim analysis, and we have 3 confirmed events thus far. We are focused on accelerating this program by implementing strategies to drive enrollment, including expanding our network of genetic testing centers and exploring options to advance our clinical development by engaging with the FDA. Following a Type C meeting in Q1, we remain on track to reengage with the FDA in the second half of this year. In summary, our priorities remain clear: delivering innovation for people living with rare disease, creating sustainable value for our stakeholders, and fulfilling our commitment to the patients, healthcare providers, and communities we serve.

I'll now turn the call over to Josh to share more details regarding MIPLYFFA's commercial performance.

Josh? Thank you, Neil, and good afternoon.

NPC is an ultra-rare genetic disease that causes significant neurological impairment and places a substantial burden on patients and families. Clinical manifestations can include dysfunction of ambulation, fine motor skills, swallowing, and speech. The disease and its symptoms vary significantly across patients, reflecting its complexity and severity. Earlier this year, the NPC clinical practice treatment guidelines were updated to further characterize the disease and reflect new advances in treatment since its last publication in 2018, including the addition of MIPLYFFA. Importantly, the guidelines recommend considering combination therapy for all patients with confirmed NPC. As a reminder, MIPLYFFA is differentiated as the only FDA-approved treatment for NPC with established efficacy and safety indicated for use in combination with miglustat.

The randomized controlled pivotal study demonstrated the combination halted NPC disease progression at 12 months as assessed by the rescored four-domain NPC Clinical Severity Scale, a validated measurement of NPC progression. The onset of benefit was rapid, with clinical effects sustained over the long term. MIPLYFFA's clinical data, combined with a growing awareness across the NPC community, continues to support its adoption. As Neil mentioned, we have received 184 prescription enrollment forms since launch through June 30th. The 14 we received in the second quarter included patients from Puerto Rico, reflecting our reach into U.S. territories. As we have noted previously, an enrollment form is a prescription submitted to our specialty pharmacy, which then begins the benefits investigation process to determine reimbursement eligibility.