Q2 2026 Zevra Therapeutics Inc Earnings Call

Speaker #4: Please stand by. Your meeting is about to begin.

Operator 1: Please stand by. Your meeting is about to begin. Good afternoon. Thank you for joining Zevra's Q2 2026 Financial Results and Corporate Update Conference Call. Today's call is being recorded and will be available via the investor relations section of the company's website later today. The host for today's call is Nichol Ochsner, Zevra's Vice President of Investor Relations and Corporate Communications.

Speaker #5: Good afternoon, and thank you for joining Zevra's second quarter 2026 financial results and corporate update conference call. Today's call is being recorded and will be available via the Investor Relations section of the company's website later today.

Operator: Good afternoon. Thank you for joining Zevra's Q2 2026 Financial Results and Corporate Update Conference Call. Today's call is being recorded and will be available via the investor relations section of the company's website later today. The host for today's call is Nichol Ochsner, Zevra's Vice President of Investor Relations and Corporate Communications.

Speaker #5: The host for today's call is Nichol Ochsner. ZEVRA's Vice President of Investor Relations and Corporate Communications.

Speaker #6: Thank you. And welcome to those who are joining us. Today, we will provide an overview of our recent accomplishments, followed by a review of our second quarter 2026 financial results.

Nichol Ochsner: Thank you. Welcome to those who are joining us. Today, we will provide an overview of our recent accomplishments, followed by a review of our Q2 2026 financial results. I encourage you to read our financial results news release, which was distributed this afternoon and is available in the investor section of our website. Before we begin the call, please note that certain information shared today will include forward-looking statements. Actual results may differ materially from those stated or implied by any forward-looking statements due to risks and uncertainties associated with Zevra's business.

Nichol Ochsner: Thank you. Welcome to those who are joining us. Today, we will provide an overview of our recent accomplishments, followed by a review of our Q2 2026 Financial Results. I encourage you to read our financial results news release, which was distributed this afternoon and is available in the investor section of our website. Before we begin the call, please note that certain information shared today will include forward-looking statements. Actual results may differ materially from those stated or implied by any forward-looking statements due to risks and uncertainties associated with Zevra's business.

Speaker #6: I encourage you to read our financial results news release, which was distributed this afternoon and is available in the Investor section of our website.

Speaker #6: Before we begin the call, please note that certain information shared today will include forward-looking statements. Actual results may differ materially from those stated or implied by any forward-looking statements, due to risks and uncertainties associated with Zevra's business.

Speaker #6: Forward-looking statements are not promises or guarantees, and are inherently subject to risks, uncertainties, and other important factors that may lead to actual results differing materially from the projections made, and should be evaluated together with the risk factors section in our most recent quarterly report on Form 10-Q, our annual report on Form 10-K, and our other filings with the SEC.

Nichol Ochsner: Forward-looking statements are not promises or guarantees and are inherently subject to risks, uncertainties, and other important factors that may lead to actual results differing materially from the projections made and should be evaluated together with the Risk Factors section in our most recent quarterly report on Form 10-Q, our annual report on Form 10-K, and our other filings with the SEC. This call will also reference certain non-GAAP financial measures, including adjusted net income and adjusted net income per share. Quantitative reconciliations of these measures to the most directly comparable GAAP measures, as well as information regarding the usefulness of these measures to management and investors, are included in the earnings press release issued today, which is available on our website. I am pleased to welcome Zevra's management team members participating in today's call.

Nichol Ochsner: Forward-looking statements are not promises or guarantees and are inherently subject to risks, uncertainties, and other important factors that may lead to actual results differing materially from the projections made and should be evaluated together with the Risk Factors section in our most recent quarterly report on Form 10-Q, our annual report on Form 10-K, and our other filings with the SEC.

Speaker #6: This call will also reference certain non-GAAP financial measures, including adjusted net income and adjusted net income per share. Quantitative reconciliations of these measures to the most directly comparable GAAP measures, as well as information regarding the usefulness of these measures to management and investors, are included in the earnings press release issued today, which is available on our website.

Nichol Ochsner: This call will also reference certain non-GAAP financial measures, including adjusted net income and adjusted net income per share. Quantitative reconciliations of these measures to the most directly comparable GAAP measures, as well as information regarding the usefulness of these measures to management and investors, are included in the earnings press release issued today, which is available on our website. I am pleased to welcome Zevra's management team members participating in today's call.

Speaker #6: I am pleased to welcome ZEVRA's Management Team members participating in today's call: Neil McFarlane, ZEVRA's President and Chief Executive Officer; Josh Schafer, our Chief Commercial Officer; and Justin Renz, our Chief Financial Officer.

Nichol Ochsner: Neil McFarlane, Zevra's President and Chief Executive Officer, Josh Schafer, our Chief Commercial Officer, and Justin Renz, our Chief Financial Officer. Now it's my pleasure to hand the call over to Neil.

Nichol Ochsner: Neil McFarlane, Zevra's President and Chief Executive Officer, Josh Schafer, our Chief Commercial Officer, and Justin Renz, our Chief Financial Officer. Now it's my pleasure to hand the call over to Neil.

Speaker #6: Now, it's my pleasure to hand the call over to Neil.

Speaker #7: 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.

Neil McFarlane: Thank you, Nichol. 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 Q2, our net revenue was $39.7 million, a 53% increase year over year. This was driven by $30.2 million in net US 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 US. As of 30 June, we had received a total of 184 prescription enrollment forms, 14 of which were received in Q2, bringing the total in H1 of the year to 23.

Neil McFarlane: Thank you, Nichol. 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 Q2, our net revenue was $39.7 million, a 53% increase year over year. This was driven by $30.2 million in net US revenue for MIPLYFFA.

Speaker #7: 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.

Speaker #7: A $53% increase year over year. This was driven by 30.2 million dollars in net US revenue for myPLIFA. Our commercial progress reflects continued momentum in reaching patients and successfully establishing myPLIFA in combination with MIGLESTAT as a foundational treatment for Neumann-Pick disease type C or NPC in the US.

Neil McFarlane: 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 US. As of 30 June, we had received a total of 184 prescription enrollment forms, 14 of which were received in Q2, bringing the total in H1 of the year to 23.

Speaker #7: 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.

Speaker #7: Additionally, we have strengthened our intellectual property position for myPLIFA 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 demonstrate the efficacy and safety of myPLIFA.

Neil McFarlane: We have strengthened our intellectual property position for MIPLYFFA with an Orange Book listing through 2041. We published long-term data from our US early access program and the findings from our pediatric substudy, 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. Our strategic focus is reflected in the thoughtful portfolio decisions we are making while maintaining an unwavering sense of urgency on behalf of patients. Most recently, we announced that following our oral explanation, the CHMP issued a negative opinion regarding the arimoclomol MAA.

Neil McFarlane: We have strengthened our intellectual property position for MIPLYFFA with an Orange Book listing through 2041. We published long-term data from our US early access program and the findings from our pediatric substudy, 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.

Speaker #7: We requested a re-examination of our Aramophthalmol marketing authorization application, or MAA, by the Committee for Medicinal Products for Human Use, or CHMP, in the European Union.

Speaker #7: And we continue to build a strong financial foundation with more than 260 million dollars in cash, cash equivalents, and investments. Our strategic focus is reflected in the thoughtful portfolio decisions we are making, while maintaining an unwavering sense of urgency on behalf of patients.

Neil McFarlane: We continue to build a strong financial foundation with more than $260 million in cash equivalents, and investments. Our strategic focus is reflected in the thoughtful portfolio decisions we are making while maintaining an unwavering sense of urgency on behalf of patients. Most recently, we announced that following our oral explanation, the CHMP issued a negative opinion regarding the arimoclomol MAA.

Speaker #7: Most recently, we announced that following our oral explanation, the CHMP issued a negative opinion regarding the Aramophthalmol MAA. 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.

Neil McFarlane: 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 name patient programs that have enrolled 132 patients, with 10 added in Q2. 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.

Neil McFarlane: 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.

Speaker #7: 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.

Speaker #7: We will maintain access to Aramophthalmol 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.

Neil McFarlane: 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 name patient programs that have enrolled 132 patients, with 10 added in Q2. 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.

Speaker #7: 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.

Speaker #7: 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.

Neil McFarlane: 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 US access treatment while building meaningful partnerships across the rare disease community. Additionally, we strengthened our US 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 US 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, and we continue to invest in expanding and strengthening our protection beyond our current portfolio.

Neil McFarlane: 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 US access treatment while building meaningful partnerships across the rare disease community.

Speaker #7: Simultaneously, we are evaluating opportunities to expand access to myPLIFA. Through the global EAP, in response to strong interest from additional countries. This reflects our commitment to helping eligible patients outside the US access treatment while building meaningful partnerships across the rare disease community.

Speaker #7: Additionally, we strengthened our U.S. intellectual property position for myPLIFA 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.

Neil McFarlane: Additionally, we strengthened our US 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 US 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, and we continue to invest in expanding and strengthening our protection beyond our current portfolio.

Speaker #7: Which have granted would fall within the Orange Book coverage period. Together, our IP portfolio provides a strong foundation for the long-term commercial opportunity for myPLIFA.

Speaker #7: And we continue to invest in expanding and strengthening our protection beyond our current portfolio. At the same time, we are advancing SOLIPROLOL, a selective adrenoceptor modulator that induces vascular dilation and smooth muscle relaxation for the treatment of vascular Ehlers-Danlos syndrome for vets.

Neil McFarlane: 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 four who were enrolled in Q2. As an event-driven study, 28 events are required to trigger the interim analysis, and we have 3 confirmed events thus far.

Neil McFarlane: 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.

Speaker #7: Vets is a rare genetic connective tissue disorder caused by changes in the CAL3A1 gene. Leading to fragile walls in blood vessels and hollow organs which can cause arterial rupture or dissection among other complications.

Speaker #7: SOLIPROLOL has been shown to reduce the mechanical stress on these tissues. In our ongoing phase three discovery trial, we've enrolled a total of 66 patients including four who were enrolled in the second quarter.

Neil McFarlane: 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 four who were enrolled in Q2. As an event-driven study, 28 events are required to trigger the interim analysis, and we have three confirmed events thus far.

Speaker #7: As an event-driven study, 28 events are required to trigger the interim analysis, and we have three confirmed events thus far. We're focused on accelerating this program by implementing strategies to drive enrollment.

Neil McFarlane: We're 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 re-engage with the FDA in H2 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?

Neil McFarlane: We're 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 re-engage with the FDA in H2 of this year.

Speaker #7: 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 re-engage with the FDA in the second half of this year.

Speaker #7: 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.

Neil McFarlane: 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?

Speaker #7: I'll now turn the call over to Josh to share more details regarding myPLIFA's commercial performance. Josh?

Speaker #8: 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.

Josh Schafer: 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.

Josh Schafer: 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.

Speaker #8: 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.

Speaker #8: 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.

Josh Schafer: 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.

Speaker #8: Including the addition of myPLIFA. Importantly, the guidelines recommend considering combination therapy for all patients with confirmed NPC. As a reminder, myPLIFA is differentiated as the only FDA-approved treatment for NPC with established efficacy and safety, indicated for use in combination with MYGLISTAT.

Speaker #8: 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.

Josh Schafer: 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 30 June. The 14 we received in the Q2 included patients from Puerto Rico, reflecting our reach into US 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.

Josh Schafer: 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.

Speaker #8: A validated measurement of NPC progression. The onset of benefit was rapid. With clinical effects sustained over the long term. myPLIFA's clinical data combined with a growing awareness across the NPC community continues to support its adoption.

Speaker #8: As Neil mentioned, we have received 184 prescription enrollment forms since launch through June 30. The 14 we received in the second quarter included patients from Puerto Rico, reflecting our reach into U.S. territories.

Josh Schafer: As Neil mentioned, we have received 184 prescription enrollment forms since launch through 30 June. The 14 we received in the Q2 included patients from Puerto Rico, reflecting our reach into US 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.

Speaker #8: 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.

Speaker #8: Notably, patients initiating treatment are being enrolled from centers of excellence and community practices with representation across both newly diagnosed and previously diagnosed patients. As well as adult and pediatric populations.

Josh Schafer: Notably, patients initiating treatment are being enrolled from centers of excellence and community practices with representation across both newly diagnosed and previously diagnosed patients, as well as adult and pediatric populations. This distribution supports our confidence in the estimated prevalence of 900 patients living with NPC in the US, of whom 300 to 350 are diagnosed. Our commercial strategy is centered on three key priorities: Accelerating diagnosis and treatment, driving demand, and facilitating access to MIPLYFFA. The progress we are seeing reflects the growing impact of initiatives being implemented across each of these areas. Our disease awareness campaign, Learn NPC, Read Between the Signs, is driving the identification of patients based on symptoms and has led to new patient enrollments for MIPLYFFA. Our genetic testing collaborations and our custom AI-driven targeting model are helping to support the identification and diagnosis of patients with NPC, allowing for earlier intervention.

Josh Schafer: Notably, patients initiating treatment are being enrolled from centers of excellence and community practices with representation across both newly diagnosed and previously diagnosed patients, as well as adult and pediatric populations. This distribution supports our confidence in the estimated prevalence of 900 patients living with NPC in the US, of whom 300 to 350 are diagnosed. Our commercial strategy is centered on three key priorities: Accelerating diagnosis and treatment, driving demand, and facilitating access to MIPLYFFA.

Speaker #8: This distribution supports our confidence in the estimated prevalence of 900 patients living with NPC in the US of whom 300 to 350 are diagnosed.

Speaker #8: Our commercial strategy is centered on three key priorities. Accelerating diagnosis and treatment. Driving demand. And facilitating access to myPLIFA. And the progress we are seeing reflects the growing impact of initiatives being implemented across each of these areas.

Josh Schafer: The progress we are seeing reflects the growing impact of initiatives being implemented across each of these areas. Our disease awareness campaign, Learn NPC, Read Between the Signs, is driving the identification of patients based on symptoms and has led to new patient enrollments for MIPLYFFA. Our genetic testing collaborations and our custom AI-driven targeting model are helping to support the identification and diagnosis of patients with NPC, allowing for earlier intervention.

Speaker #8: Our disease awareness campaign, Learn NPC: Read Between the Signs, is driving the identification of patients based on symptoms and has led to new patient enrollments for myPLIFA.

Speaker #8: Our genetic testing collaborations and our custom AI-driven targeting model are helping to support the identification and diagnosis of patients with NPC, allowing for earlier intervention.

Speaker #8: We also launched our Expert Connect program, which has enabled healthcare professionals who are less familiar with NPC to consult one-on-one with clinical experts to further strengthen their knowledge and confidence.

Josh Schafer: We also launched our Expert Connect program, which has enabled healthcare professionals who are less familiar with NPC to consult one-on-one with clinical experts to further strengthen knowledge and confidence. Additionally, we expanded and enhanced our digital engagement through the launch of our NPC Knowledge Hub, providing resources to help patients and caregivers navigate the treatment journey. From a market access standpoint, payer coverage remained at 69% of covered lives, consistent with our expectations. We continue to share presentations with payers on the recent update to the NPC clinical practice guidelines, as well as the growing body of newly published clinical data on MIPLYFFA. Additionally, we convene payer advisory boards with key decision-makers to inform our strategy on how best to support prescribers and patients as they navigate the reimbursement landscape.

Josh Schafer: We also launched our Expert Connect program, which has enabled healthcare professionals who are less familiar with NPC to consult one-on-one with clinical experts to further strengthen knowledge and confidence. Additionally, we expanded and enhanced our digital engagement through the launch of our NPC Knowledge Hub, providing resources to help patients and caregivers navigate the treatment journey.

Speaker #8: Additionally, we expanded and enhanced our digital engagement through the launch of our NPC knowledge hub, providing resources to help patients and caregivers navigate the treatment journey.

Speaker #8: From a market access standpoint, payer coverage remained at 69% of covered lives, consistent with our expectations. We continue to share presentations with payers on the recent update to the NPC clinical practice guidelines, as well as the growing body of newly published clinical data on myPLIFA.

Josh Schafer: From a market access standpoint, payer coverage remained at 69% of covered lives, consistent with our expectations. We continue to share presentations with payers on the recent update to the NPC clinical practice guidelines, as well as the growing body of newly published clinical data on MIPLYFFA. Additionally, we convene payer advisory boards with key decision-makers to inform our strategy on how best to support prescribers and patients as they navigate the reimbursement landscape.

Speaker #8: Additionally, we convene payer advisory boards with key decision makers to inform our strategy on how best to support prescribers and patients as they navigate the reimbursement landscape.

Speaker #8: We maintain strong relationships with the rare disease community and assembled steering committees to gather expert insights and help shape NPC disease awareness. In the second quarter, we participated in national and regional congresses including the American Academy of Neurology, the Society of Inborn Metabolic Disorders, and the Annual Pediatric Neuro Symposium.

Josh Schafer: We maintain strong relationships with the rare disease community and assembled steering committees to gather expert insights and help shape NPC disease awareness. In Q2, we participated in national and regional congresses, including the American Academy of Neurology, the Society for Inherited Metabolic Disorders, and the Annual Pediatric Neuro Symposium, to enhance scientific visibility and educate clinicians and researchers. This strong engagement has continued into Q3 with our participation at the National Niemann-Pick Disease Foundation Conference, where the importance and interest in a combination strategy for NPC treatment was highlighted prominently. Before turning the call over to Justin to review the financials, we want to reiterate our deep gratitude for the opportunity to make a meaningful impact in the lives of patients and their loved ones. We remain committed to being a trusted partner in the community by providing compassionate, patient-centered support. Justin?

Josh Schafer: We maintain strong relationships with the rare disease community and assembled steering committees to gather expert insights and help shape NPC disease awareness. In Q2, we participated in national and regional congresses, including the American Academy of Neurology, the Society for Inherited Metabolic Disorders, and the Annual Pediatric Neuro Symposium, to enhance scientific visibility and educate clinicians and researchers.

Speaker #8: To enhance scientific visibility and educate clinicians and researchers. This strong engagement has continued into the third quarter with our participation at the National Neiman Pick Disease Foundation Conference where the importance and interest in a combination strategy for NPC treatment was highlighted prominently.

Josh Schafer: This strong engagement has continued into Q3 with our participation at the National Niemann-Pick Disease Foundation Conference, where the importance and interest in a combination strategy for NPC treatment was highlighted prominently. Before turning the call over to Justin to review the financials, we want to reiterate our deep gratitude for the opportunity to make a meaningful impact in the lives of patients and their loved ones. We remain committed to being a trusted partner in the community by providing compassionate, patient-centered support. Justin?

Speaker #8: Before turning the call over to Justin to review the financials, we want to reiterate our deep gratitude for the opportunity to make a meaningful impact in the lives of patients and their loved ones.

Speaker #8: We remain committed to being a trusted partner in the community by providing compassionate, patient-centered support. Justin?

Speaker #2: Thank you, Josh. Underpinning our achievements is a strong financial foundation and a robust cash position which provide the flexibility to invest in commercial execution advance our pipeline and pursue future growth opportunities while maintaining financial discipline.

Justin Renz: Thank you, Josh. Underpinning our achievements is a strong financial foundation and a robust cash position, which provide the flexibility to invest in commercial execution, advance our pipeline, and pursue future growth opportunities while maintaining financial discipline. In addition to the financial details included in today's call, we encourage you to refer to our quarterly report on Form 10-Q for more detailed information, which we intend to file later today. In Q2 2026, we generated total net revenue of $39.7 million, which was an increase of $13.8 million, or 53%, compared to $25.9 million in Q2 2025. As a reminder, we recognize US commercial revenue when MIPLYFFA shipments are received by the specialty pharmacy. Of note, channel inventory has returned to target levels as compared to Q1, when it fell below the low end of our range.

Justin Renz: Thank you, Josh. Underpinning our achievements is a strong financial foundation and a robust cash position, which provide the flexibility to invest in commercial execution, advance our pipeline, and pursue future growth opportunities while maintaining financial discipline. In addition to the financial details included in today's call, we encourage you to refer to our quarterly report on Form 10-Q for more detailed information, which we intend to file later today. I

Speaker #2: In addition to the financial details included in today's call, we encourage you to refer to our quarterly report on Form 10-Q for more detailed information, which we intend to file later today.

Speaker #2: In the second quarter of 2026, we generated total net revenue of $39.7 million which was an increase of 13.8 million or 53% compared to 25.9 million in Q2, 2025.

Justin Renz: n Q2 2026, we generated total net revenue of $39.7 million, which was an increase of $13.8 million, or 53%, compared to $25.9 million in Q2 2025. As a reminder, we recognize US commercial revenue when MIPLYFFA shipments are received by the specialty pharmacy. Of note, channel inventory has returned to target levels as compared to Q1, when it fell below the low end of our range.

Speaker #2: As a reminder, we recognize US commercial revenue when myPLIFA shipments are received by the specialty pharmacy. Of note, channel inventory has returned to target levels as compared to Q1 when it fell below the low end of our range.

Speaker #2: The second quarter of 2026 total net revenue is comprised of $30.2 million from myPLIFA net sales in the US, $0.2 million from Opruva, $9.0 million in net reimbursements from the Global EAP for Aramaco Mall, and $0.3 million in past-due royalty revenue from Comav.

Justin Renz: The Q2 2026 total net revenue is comprised of $30.2 million from MIPLYFFA net sales in the US, $0.2 million from OLPRUVA, $9.0 million in net reimbursements from the global EAP for arimoclomol, and $0.3 million in past due royalty revenue from ComAv. Revenue from MIPLYFFA in the US grew 23% compared to Q1 of this year and 40% compared to Q2 of last year. During Q2 2026, our operating expenses were $21.0 million, which was a decrease of $3.2 million compared to the same quarter a year ago. R&D expense was $4.5 million for Q2 2026, which was an increase of $1.1 million compared to Q2 2025, due primarily to ongoing arimoclomol efforts.

Justin Renz: The Q2 2026 total net revenue is comprised of $30.2 million from MIPLYFFA net sales in the US, $0.2 million from OLPRUVA, $9.0 million in net reimbursements from the global EAP for arimoclomol, and $0.3 million in past due royalty revenue from ComAv. Revenue from MIPLYFFA in the US grew 23% compared to Q1 of this year and 40% compared to Q2 of last year.

Speaker #2: Revenue from myPLIFA in the US grew 23% compared to the first quarter of this year and 40% compared to the second quarter of last year.

Speaker #2: During the second quarter of 2026, our operating expenses were $21.0 million which was a decrease of 3.2 million compared to the same quarter a year ago.

Justin Renz: During Q2 2026, our operating expenses were $21.0 million, which was a decrease of $3.2 million compared to the same quarter a year ago. R&D expense was $4.5 million for Q2 2026, which was an increase of $1.1 million compared to Q2 2025, due primarily to ongoing arimoclomol efforts.

Speaker #2: R&D expense was $4.5 million for Q2 2026, which was an increase of $1.1 million compared to Q2 2025, due primarily to ongoing Aramco Mall efforts.

Speaker #2: SG&A expense was $16.6 million for Q2, 2026 which was a decrease of 4.2 million compared to Q2, 2025. Primarily due to a decrease in professional fees and third-party spending partially offset by an increase in personnel-related costs.

Justin Renz: SG&A expense was $16.6 million for Q2 2026, which was a decrease of $4.2 million compared to Q2 2025, primarily due to a decrease in professional fees and third-party spending, partially offset by an increase in personnel-related costs. We allocated $4.0 million for our Q2 2026 income tax provision. Net income for Q2 2026 was $8.8 million, or $0.14 per basic and diluted share, compared to net income of $74.7 million, or $1.24 per basic and $1.21 per diluted share for the same quarter of a year ago. This quarter, we incurred a mark-to-market fair value non-cash charge of $6.4 million related to our outstanding common stock warrants. Excluding this adjustment that impacts period comparison, net income for Q2 2026 would have been $15.2 million, or $0.25 per share.

Justin Renz: SG&A expense was $16.6 million for Q2 2026, which was a decrease of $4.2 million compared to Q2 2025, primarily due to a decrease in professional fees and third-party spending, partially offset by an increase in personnel-related costs. We allocated $4.0 million for our Q2 2026 income tax provision.

Speaker #2: We allocated $4.0 million for our Q2, 2026 income tax provision. Net income for the second quarter of 2026 was $8.8 million. Or $14 cents per basic and diluted share compared to net income of $74.7 million or $1.24 per basic and $1.21 per diluted share for the same quarter of a year ago.

Justin Renz: Net income for Q2 2026 was $8.8 million, or $0.14 per basic and diluted share, compared to net income of $74.7 million, or $1.24 per basic and $1.21 per diluted share for the same quarter of a year ago. This quarter, we incurred a mark-to-market fair value non-cash charge of $6.4 million related to our outstanding common stock warrants. Excluding this adjustment that impacts period comparison, net income for Q2 2026 would have been $15.2 million, or $0.25 per share.

Speaker #2: This quarter, we incurred a mark-to-market fair value non-cash charge of $6.4 million related to our outstanding common stock warrants. Excluding this adjustment that impacts period comparison, net income for Q2, 2026 would have been $15.2 million or $25 cents per share.

Speaker #2: In Q2 2025, we would have had an adjusted net loss of $2.4 million, or $0.04 per share, by excluding one-time transactions and other adjustments that contributed a net positive of $77.1 million to Q2 2025's results.

Justin Renz: In Q2 2025, we would have had an adjusted net loss of $2.4 million, or $0.04 per share by excluding one-time transactions and other adjustments that contributed a net positive of $77.1 million to Q2 2025's results. As of 30 June 2026, total cash equivalents, and investments were $260.2 million, which was an increase of approximately $23.4 million compared to 31 March 2026. In summary, we remain well-positioned with the financial capacity to execute on our strategic priorities independent of the capital markets. I'll turn the call back to Neil for his closing remarks. Neil?

Justin Renz: In Q2 2025, we would have had an adjusted net loss of $2.4 million, or $0.04 per share by excluding one-time transactions and other adjustments that contributed a net positive of $77.1 million to Q2 2025's results. As of 30 June 2026, total cash equivalents, and investments were $260.2 million, which was an increase of approximately $23.4 million compared to 31 March 2026. In summary, we remain well-positioned with the financial capacity to execute on our strategic priorities independent of the capital markets. I'll turn the call back to Neil for his closing remarks. Neil?

Speaker #2: As of June 30th, 2026, total cash, cash equivalents and investments were $260.2 million which was an increase of approximately 23.4 million compared to March 31st, 2026.

Speaker #2: In summary, we remain well positioned with the financial capacity to execute on our strategic priorities independent of the capital markets. Now I'll turn the call back to Neil for his closing remarks.

Speaker #2: Neil?

Speaker #3: Thanks, Justin. Our strong second quarter performance demonstrates the strength of our strategy, disciplined execution, and continued momentum across the business. We remain steadfast in our commitment to putting patients first.

Neil McFarlane: Thanks, Justin. Our strong Q2 performance demonstrates the strength of our strategy, disciplined execution, and continued momentum across the business. We remain steadfast in our commitment to putting patients first, a principle that is reflected in our approach to growing our US commercial business, expanding our global presence, and fueling our portfolio to deliver long-term shareholder value. With that, operator, please open the line for questions.

Neil McFarlane: Thanks, Justin. Our strong Q2 performance demonstrates the strength of our strategy, disciplined execution, and continued momentum across the business. We remain steadfast in our commitment to putting patients first, a principle that is reflected in our approach to growing our US commercial business, expanding our global presence, and fueling our portfolio to deliver long-term shareholder value. With that, operator, please open the line for questions.

Speaker #3: A principle that is reflected in our approach to growing our US commercial business, expanding our global presence, and fueling our portfolio to deliver long-term shareholder value.

Speaker #3: With that, operator, please open the line for questions.

Speaker #4: At this time, if you wish to ask a question, please press star one on your telephone keypad. You may remove yourself from the queue by pressing star two.

Operator 1: At this time, if you wish to ask a question, please press star one on your telephone keypad. You may remove yourself from the queue by pressing star two. We will take our first question from Kristen Kluska with Cantor Fitzgerald. Your line is now open.

Operator: At this time, if you wish to ask a question, please press star one on your telephone keypad. You may remove yourself from the queue by pressing star two. We will take our first question from Kristen Kluska with Cantor Fitzgerald. Your line is now open.

Speaker #4: We will take our first question from Kristen Kluska with Canter Fitzgerald. Your line is now open.

Speaker #5: Hi everyone. Thanks so much for taking the questions here. I had a couple this afternoon. First, you're sitting on a very robust balance sheet and have a continued line of sight into more revenues.

Kristen Kluska: Hi, everyone. Thanks so much for taking the questions here. I had a couple this afternoon. First, you're sitting on a very robust balance sheet and have a continued line of sight into more revenues as the MIPLYFFA business grows. Curious how you're leveraging this cash and ways you're looking to potentially explore using that. Second, wanted to talk about the EMA. You mentioned there's a new rapporteur here. Can you tell us a little bit about how that process works when there's really a new team here looking? Is there anything that you can do differently? We have seen some recent reversals, specifically in the rare disease space, so curious about your thoughts there. Thank you so much.

Kristen Kluska: Hi, everyone. Thanks so much for taking the questions here. I had a couple this afternoon. First, you're sitting on a very robust balance sheet and have a continued line of sight into more revenues as the MIPLYFFA business grows. Curious how you're leveraging this cash and ways you're looking to potentially explore using that. Second, wanted to talk about the EMA.

Speaker #5: As the myth life of business grows. So curious how you're leveraging this cash and ways you're looking to potentially explore using that. Second, wanted to talk about the EMA.

Speaker #5: You mentioned there's a new repertoire here. Can you tell us a little bit about how that process works when there's really a new team here looking?

Kristen Kluska: You mentioned there's a new rapporteur here. Can you tell us a little bit about how that process works when there's really a new team here looking? Is there anything that you can do differently? We have seen some recent reversals, specifically in the rare disease space, so curious about your thoughts there. Thank you so much.

Speaker #5: Is there anything that you can do differently? And we have seen some recent reversals specifically in the rare disease space. So curious about your thoughts there.

Speaker #5: Thank you so much.

Speaker #3: Yeah, thank you, Kristen. Maybe I'll start with your second question and then I'll pivot over to Justin to help talk a little bit about the strong financial foundation we've got.

Neil McFarlane: Thank you, Kristen. Maybe I'll start with your second question, then I'll pivot over to Justin to help talk a little bit about the strong financial foundation we've got. You are correct. In our re-examination process, we will have the opportunity to bring in a new rapporteur and co-rapporteur to further interpret the evidence that we have submitted so far. I can't actually comment on what that process will look like because I have to be thoughtful on the level of details we provide. Our confidence is grounded in the totality of the evidence and the clear unmet patient need. As you saw, we continued in our global EAP to add an additional 10 patients up to 132 patients. Those are primarily patients that are in Europe.

Neil McFarlane: Thank you, Kristen. Maybe I'll start with your second question, then I'll pivot over to Justin to help talk a little bit about the strong financial foundation we've got. You are correct. In our re-examination process, we will have the opportunity to bring in a new rapporteur and co-rapporteur to further interpret the evidence that we have submitted so far.

Speaker #3: So you are correct in our re-examination process. We will have the opportunity to bring in new repertoire and co-repertoire to further interpret the evidence that we have submitted so far.

Speaker #3: I can't actually comment on what that process will look like because I have to be thoughtful about the level of detail we provide. But our confidence is grounded in the totality of the evidence and the clear unmet patient need.

Neil McFarlane: I can't actually comment on what that process will look like because I have to be thoughtful on the level of details we provide. Our confidence is grounded in the totality of the evidence and the clear unmet patient need. As you saw, we continued in our global EAP to add an additional 10 patients up to 132 patients. Those are primarily patients that are in Europe.

Speaker #3: As you saw, we continued in our global EAP to add an additional 10 patients up to 132 patients. Those are primarily patients that are in Europe.

Speaker #3: And I think that as we continue to move forward with our re-examination process, we're looking forward to bringing new expert voices into the procedural discussions and allowing for the comprehensive evidence package to shine.

Neil McFarlane: I think that as we continue to move forward with our re-examination process, we're looking forward to bringing new expert voices into the procedural discussions and allowing for the comprehensive evidence package to shine. On that note, I'll ask Justin to talk a little more about our strong financial foundation and what we're doing moving forward.

Neil McFarlane: I think that as we continue to move forward with our re-examination process, we're looking forward to bringing new expert voices into the procedural discussions and allowing for the comprehensive evidence package to shine. On that note, I'll ask Justin to talk a little more about our strong financial foundation and what we're doing moving forward.

Speaker #3: On that note, I'll ask Justin to talk a little bit more about our strong financial foundation and what we're doing moving forward.

Speaker #2: Thank you, Neil. And thank you, Kristen. As you noted, we ended Q2 with $260.2 million in cash, cash equivalents and investments. And that really provides us meaningful flexibility.

Justin Renz: Thank you, Neil, and thank you, Kristen. As you noted, we ended Q2 with $260.2 million in cash equivalents, and investments, that really provides us meaningful flexibility. Our priorities are unchanged as we execute against our strategic plan. Namely, we're driving MIPLYFFA growth, pursue global opportunities for MIPLYFFA, continue to advance celiprolol, again, evaluate, of course, aligned external opportunities. We're going to maintain that disciplined capital allocation strategy we've always talked of. We're going to be prudent in our investments and really focus on value-creating opportunities when we can find them.

Justin Renz: Thank you, Neil, and thank you, Kristen. As you noted, we ended Q2 with $260.2 million in cash equivalents, and investments, that really provides us meaningful flexibility. Our priorities are unchanged as we execute against our strategic plan. Namely, we're driving MIPLYFFA growth, pursue global opportunities for MIPLYFFA, continue to advance celiprolol, again, evaluate, of course, aligned external opportunities. We're going to maintain that disciplined capital allocation strategy we've always talked of. We're going to be prudent in our investments and really focus on value-creating opportunities when we can find them.

Speaker #2: Our priorities are unchanged as we execute against our strategic plan. Namely, we're driving my play for growth, pursue global opportunities for my playfa, continue to advance the Webber law, and again, evaluate, of course, aligned external opportunities.

Speaker #2: So we're going to maintain that disciplined capital allocation strategy we've always talked of. We're going to be prudent in our investments and really focus on value creating opportunities when we can find them.

Speaker #5: Thank you very much.

Kristen Kluska: Thanks very much.

Kristen Kluska: Thanks very much.

Speaker #4: And we will take our next question from Sumat Kulkarni with Canaccord Genuity. Your line is open.

Operator 1: We will take our next question from Sumant Kulkarni with Canaccord Genuity. Your line is open.

Operator: We will take our next question from Sumant Kulkarni with Canaccord Genuity. Your line is open.

Speaker #3: Good afternoon. Thanks for taking

Sumant Kulkarni: Good afternoon. Thanks for taking our question. This one is about the EMA process. What exactly could you do to strengthen your, I guess, the request for re-examination? Is there anything new you can file in terms of the real-world data you've generated? I guess the best way I could ask that is what is going to be new in this request versus what was denied in the prior documents that you sent in? Thank you.

Sumant Kulkarni: Good afternoon. Thanks for taking our question. This one is about the EMA process. What exactly could you do to strengthen your, I guess, the request for re-examination? Is there anything new you can file in terms of the real-world data you've generated? I guess the best way I could ask that is what is going to be new in this request versus what was denied in the prior documents that you sent in? Thank you.

Speaker #6: our question. This one is about the EMA process. What exactly could you do to strengthen your, I guess, the request for re-examination? Is there anything new you can file in terms of the real-world data that you've generated?

Speaker #6: Or I guess the best way I could ask that is what is going to be new in this request versus what was denied in the prior documents that you sent in?

Speaker #6: Thank you.

Speaker #3: Thanks, Sumat. So I think it's really important question. The opinion that the CHMP provided reflects their assessment on the submitted evidence under this European regulatory framework that they have.

Neil McFarlane: Thanks, Sumant. I think it's a really important question. The opinion that the CHMP provided reflects their assessment on the submitted evidence under this European regulatory framework that they have. Under the re-examination process, we'll have the opportunity to focus specifically to address these specific grounds that have been cited for the refusal. We believe strongly that the comprehensive evidence package, we've got an open label extension study that goes out four years. Actually, just this week, the expanded access program data that has been published has gone out for four years. The pediatric sub-study has been published now, along with a very comprehensive safety database.

Neil McFarlane: Thanks, Sumant. I think it's a really important question. The opinion that the CHMP provided reflects their assessment on the submitted evidence under this European regulatory framework that they have. Under the re-examination process, we'll have the opportunity to focus specifically to address these specific grounds that have been cited for the refusal.

Speaker #3: Under the re-examination process, we'll have the opportunity to focus specifically to address these specific grounds that have been cited for the refusal. We believe strongly that the comprehensive evidence package—we’ve got an open-label extension study that goes out four years.

Neil McFarlane: We believe strongly that the comprehensive evidence package, we've got an open label extension study that goes out four years. Actually, just this week, the expanded access program data that has been published has gone out for four years. The pediatric sub-study has been published now, along with a very comprehensive safety database.

Speaker #3: It's actually just this week. The expanded access program data that has been published has gone out for four years. The pediatric sub-study has been published now along with a very comprehensive safety database.

Speaker #3: So this robust and comprehensive package that we've resubmitted as part of this application, we're now going to go into the further interpretation of the evidence that are specific to the questions that they or the specific grounds that were cited for the refusal.

Neil McFarlane: This robust and comprehensive package that we've resubmitted as part of this application, we're now going to go into the further interpretation of the evidence that are specific to the questions or the specific grounds that were cited for the refusal and bring it to light. That comes with new expert voices into the procedural discussion that will allow us to continue to educate the CHMP on this, again, I've said it a few times, but robust and comprehensive evidence package.

Neil McFarlane: This robust and comprehensive package that we've resubmitted as part of this application, we're now going to go into the further interpretation of the evidence that are specific to the questions or the specific grounds that were cited for the refusal and bring it to light. That comes with new expert voices into the procedural discussion that will allow us to continue to educate the CHMP on this, again, I've said it a few times, but robust and comprehensive evidence package.

Speaker #3: And bring it to light. And that comes with new expert voices into the procedural discussion that will allow us to continue to educate the CHMP on this again, I've said it a few times, but robust and comprehensive evidence package.

Speaker #6: Thanks.

Sumant Kulkarni: Thanks.

Sumant Kulkarni: Thanks.

Speaker #4: And we'll take our next question from Eddie Hickman with Guggenheim Securities. Please go ahead.

Operator 1: We'll take our next question from Eddie Hickman with Guggenheim Securities. Please go ahead.

Operator: We'll take our next question from Eddie Hickman with Guggenheim Securities. Please go ahead.

Speaker #7: Hey, good afternoon. Appreciate you taking the questions. And congrats on all the progress so far. Just two from me. On the US side, can you just give us maybe some directional color on which of the segments you're seeing a rebound in?

Eddie Hickman: Hey, good afternoon. Appreciate you taking the questions, and congrats on all the progress so far. Just two from me. On the US side, can you just give us maybe some directional color on which of the segments you're seeing

Eddie Hickman: Hey, good afternoon. Appreciate you taking the questions, and congrats on all the progress so far. Just two from me. On the US side, can you just give us maybe some directional color on which of the segments you're seeing a rebound in? Is it the GeneDx contributing to more diagnoses? Are you seeing a different level of new versus previously diagnosed, adult versus pediatric?

Eddie Hickman: a rebound in? Is it the GeneDx contributing to more diagnoses? Are you seeing a different level of new versus previously diagnosed, adult versus pediatric? Just help us understand where the biggest opportunity remaining is. Then on the ex-US side, can you maybe help us bracket the variable non-French EAP numbers? I know it's going to be up and down maybe, but can you maybe give us a min-max or some sort of bracketing for the next couple quarters on how we should model that? Appreciate it.

Speaker #7: Is it sort of the GNDX contributing to more diagnoses? Are you seeing a different level of new versus previously diagnosed? Adult versus pediatric? Just help us understand sort of where the biggest opportunity remaining is.

Eddie Hickman: Just help us understand where the biggest opportunity remaining is. Then on the ex-US side, can you maybe help us bracket the variable non-French EAP numbers? I know it's going to be up and down maybe, but can you maybe give us a min-max or some sort of bracketing for the next couple quarters on how we should model that? Appreciate it.

Speaker #7: And then on the X US side, can you sort of maybe help us bracket the sort of variable non-French EAP numbers, sort of I know it's going to be sort of up and down maybe, but can you maybe sort of give us a min-max or some sort of bracketing for the next couple of quarters on how we should model that?

Speaker #7: Appreciate it.

Speaker #3: Let me start with your second question. In regards to the global EAP, and I think you're specifically asking about the reimbursed section of that.

Neil McFarlane: Let me start with your second question in regards to the global EAP. I think you're specifically asking about the reimbursed section of that. We've said previously that our French program has been very consistent. It's approximately two and a half million dollars net per quarter or $10 million net per year. That program has been ongoing for a multitude of years, and we have about 30 patients, plus or minus, that remain very consistent. The variability in ordering patterns that come through other named patient reimbursement programs can come in. It's one patient at a time, and it can be one patient that orders for an annual basis, one patient that orders for quarterly or a monthly supply. It's really hard for me to tell you, or guide you in regards to the brackets that you're asking for because it remains unpredictable.

Neil McFarlane: Let me start with your second question in regards to the global EAP. I think you're specifically asking about the reimbursed section of that. We've said previously that our French program has been very consistent. It's approximately two and a half million dollars net per quarter or $10 million net per year. That program has been ongoing for a multitude of years, and we have about 30 patients, plus or minus, that remain very consistent.

Speaker #3: We've said previously that our French program has been very consistent. It's approximately two and a half million dollars net per quarter or $10 million net per year.

Speaker #3: And that program has been ongoing for a multitude of years. And we have about 30 patients plus or minus that remain very consistent. The variability in ordering patterns that come through other named patient reimbursement programs can come in it's one patient at a time.

Neil McFarlane: The variability in ordering patterns that come through other named patient reimbursement programs can come in. It's one patient at a time, and it can be one patient that orders for an annual basis, one patient that orders for quarterly or a monthly supply. It's really hard for me to tell you, or guide you in regards to the brackets that you're asking for because it remains unpredictable.

Speaker #3: And it can be one patient that orders for an annual basis, one patient that orders for quarterly, or a monthly supply. So it's really hard for me to tell you or guide you in regards to the brackets that you're asking for because it remains unpredictable.

Speaker #3: It's driven by the timing, the order volume, the program type, and also the geography. So I can't the only thing that I can tell you is that we remain very committed in the French EAP program, which has been consistent at about 10 million dollars net per year or two and a half, approximately two and a half million dollars per quarter.

Neil McFarlane: It's driven by the timing, the order volume, the program type, and also the geography. The only thing that I can tell you is that we remain very committed in the French EAP program, which has been consistent at about $10 million net per year or approximately two and a half million dollars per quarter. Let me switch quickly to the US business. Really great questions. The US business, we're learning more about every day. We are seeing an equal mix between children and adults. As you know, this has been primarily a child disease for many years and kind of characterizes that. Now we're seeing quite a bit of adult patients, so those learnings are coming through. We don't have a single tactic that is working to uncover the number of patients we have. Remember, we're at 184 enrollment forms now with 14 in the quarter.

Neil McFarlane: It's driven by the timing, the order volume, the program type, and also the geography. The only thing that I can tell you is that we remain very committed in the French EAP program, which has been consistent at about $10 million net per year or approximately two and a half million dollars per quarter. Let me switch quickly to the US business. Really great questions.

Speaker #3: Let me switch quickly to the US business. Really great questions. The US business, we're learning more about every day. We are seeing equal mix between children and adults as you know.

Neil McFarlane: The US business, we're learning more about every day. We are seeing an equal mix between children and adults. As you know, this has been primarily a child disease for many years and kind of characterizes that. Now we're seeing quite a bit of adult patients, so those learnings are coming through. We don't have a single tactic that is working to uncover the number of patients we have. Remember, we're at 184 enrollment forms now with 14 in the quarter.

Speaker #3: This has been primarily a child disease for many years. And kind of characterizes that. Now we're seeing quite a bit of adult patients. So those learnings are coming through.

Speaker #3: We don't have a single tactic that is working to uncover the number of patients we have. And remember, we're at 184 enrollment forms now with 14 in the quarter.

Speaker #3: This is really a integrated strategy. And I'll ask Josh to talk more about some of the things he's seen on the ground. But an integrated strategy versus a single tactic that is continuing to drive this and kind of unlocking this TAM that we think is between the 350 and 900.

Neil McFarlane: This is really an integrated strategy, and I'll ask Josh to talk more about some of the things he's seen on the ground, but an integrated strategy versus a single tactic that is continuing to drive this and kind of unlocking the TAM that we think is between the 350 and 900. Josh?

Neil McFarlane: This is really an integrated strategy, and I'll ask Josh to talk more about some of the things he's seen on the ground, but an integrated strategy versus a single tactic that is continuing to drive this and kind of unlocking the TAM that we think is between the 350 and 900. Josh?

Speaker #3: Josh?

Speaker #6: Yeah, thanks. Eddie, thanks for the question. As Neil mentioned, I wouldn't attribute the enrollments to any one tactic. In fact, we have a really well-integrated commercial strategy that's focused on accelerating diagnosis and treatment.

Josh Schafer: Yeah, thanks. Eddie, thanks for the question. As Neil mentioned, I wouldn't attribute the enrollments to any one tactic. In fact, we have a really well-integrated commercial strategy that's focused on accelerating diagnosis and treatment. We're seeing that really take place with the 23 enrollments that we've seen in the H1 of this year. Those enrollments are coming with a mix of newly diagnosed patients as well as previously diagnosed patients. It's a mix of patients in centers of excellence as well as now we're able to reach out into the community and find patients who are in community practices. We're seeing patients who are young as well as adult patients.

Josh Schafer: Yeah, thanks. Eddie, thanks for the question. As Neil mentioned, I wouldn't attribute the enrollments to any one tactic. In fact, we have a really well-integrated commercial strategy that's focused on accelerating diagnosis and treatment. We're seeing that really take place with the 23 enrollments that we've seen in the H1 of this year.

Speaker #6: And we're seeing that really take place with the 23 enrollments that we've seen in the first half of this year. Those enrollments are coming with a mix of newly diagnosed patients as well as previously diagnosed patients.

Josh Schafer: Those enrollments are coming with a mix of newly diagnosed patients as well as previously diagnosed patients. It's a mix of patients in centers of excellence as well as now we're able to reach out into the community and find patients who are in community practices. We're seeing patients who are young as well as adult patients.

Speaker #6: It's a mix of patients in centers of excellence as well as now we're able to reach out into the community and find patients who are in community practices.

Speaker #6: And we're seeing patients who are young as well as adult patients. So we're really pleased with the diversity and the distribution of enrollments that we're seeing.

Josh Schafer: We're really pleased with the diversity and the distribution of enrollments that we're seeing, most notably because it really gives us confidence in the overall addressable market that we've been talking about, which is, we believe, somewhere between 350 and 900 patients.

Josh Schafer: We're really pleased with the diversity and the distribution of enrollments that we're seeing, most notably because it really gives us confidence in the overall addressable market that we've been talking about, which is, we believe, somewhere between 350 and 900 patients.

Speaker #6: Most notably because it really gives us confidence in the overall addressable market that we've been talking about, which is we believe somewhere between 350 and 900 patients.

Speaker #7: Appreciate it. Thank you.

Eddie Hickman: Appreciate it. Thank you.

Eddie Hickman: Appreciate it. Thank you.

Speaker #4: And we will take our next question from Jason Butler with Citizens. Please go ahead.

Operator 1: We will take our next question from Jason Butler with Citizens. Please go ahead.

Operator: We will take our next question from Jason Butler with Citizens. Please go ahead.

Speaker #5: Hi. Thanks for taking the question. Just wondering if you could give us some kind of perspective on the age distribution of newly diagnosed patients.

Jason Butler: Hi. Thanks for taking the question. Just wondering if you could give us some kind of perspective on the age distribution of newly diagnosed patients. Secondly, when you look at the tools that you're using, like the AI targeting and the Expert Connect program, are you seeing any growth in the number of prescribers over the last couple of quarters? Thank you.

Jason Butler: Hi. Thanks for taking the question. Just wondering if you could give us some kind of perspective on the age distribution of newly diagnosed patients. Secondly, when you look at the tools that you're using, like the AI targeting and the Expert Connect program, are you seeing any growth in the number of prescribers over the last couple of quarters? Thank you.

Speaker #5: And then secondly, when you look at the tools that you're using like the AI targeting and the expert connect program, are you seeing any growth in the number of prescribers over the last couple of quarters?

Speaker #5: Thank you.

Neil McFarlane: Josh?

Neil McFarlane: Josh?

Speaker #3: Josh?

Speaker #7: Yeah, sure. In terms of the age distribution of newly diagnosed patients, we are seeing patients who are coming in who are both under the age of 18 as well as some who are over the age of 18.

Josh Schafer: Yeah, sure. In terms of the age distribution of newly diagnosed patients, we are seeing patients who are coming in who are both under the age of 18 as well as some who are over the age of 18. Just within this H1, we have some very specific instances. There was a newly diagnosed child who was about two years old. Symptoms had just been presented, and the parents had her tested and confirmed with NPC. On the other hand, we have a patient who is in his mid-20s and has been misdiagnosed with epilepsy for five or six years and was finally tested and confirmed to have NPC. We're really seeing a diversity of instances and age groups reinforcing the heterogeneity of the disease. This is largely driven by the tactics that we've put in place, the AI targeting, our disease awareness campaign.

Josh Schafer: Yeah, sure. In terms of the age distribution of newly diagnosed patients, we are seeing patients who are coming in who are both under the age of 18 as well as some who are over the age of 18. Just within this H1, we have some very specific instances. There was a newly diagnosed child who was about two years old. Symptoms had just been presented, and the parents had her tested and confirmed with NPC.

Speaker #7: Just within the past this first half, we have some very specific instances. There was a newly diagnosed child who was about two years old.

Speaker #7: Symptoms had just been presented and the parents had her tested and confirmed with NPC. On the other hand, we have a patient who is in his mid-20s and has been misdiagnosed with epilepsy for five or six years.

Josh Schafer: On the other hand, we have a patient who is in his mid-20s and has been misdiagnosed with epilepsy for five or six years and was finally tested and confirmed to have NPC. We're really seeing a diversity of instances and age groups reinforcing the heterogeneity of the disease. This is largely driven by the tactics that we've put in place, the AI targeting, our disease awareness campaign.

Speaker #7: And was finally tested and confirmed to have NPC. So we're really seeing a diversity of instances and age groups reinforcing the heterogeneity of the disease.

Speaker #7: And this is largely driven by the tactics that we've put in place, the AI targeting, our disease awareness campaign, all of these things are really helping to drive enrollments and again, giving us confidence that there are more patients out there yet to be diagnosed.

Josh Schafer: All of these things are really helping to drive enrollments and, again, giving us confidence that there are more patients out there yet to be diagnosed.

Josh Schafer: All of these things are really helping to drive enrollments and, again, giving us confidence that there are more patients out there yet to be diagnosed.

Speaker #3: Yeah. Jason, just to put a little bit a finer point on this, we've said that the age distribution between adults and kids is about 50/50.

Neil McFarlane: Yeah, Jason, just to put a little bit of a finer point on this, we've said that the age distribution between adults and kids is about 50/50 in what we see today. Of these 184 enrollment forms, you should think of that as half adults and half children. When it comes to the newly diagnosed patients, it's really difficult for us to be able to say and provide that level of guidance. For us, you should consider to say this is not just a childhood disease, as we've been thinking about this for many, many decades. These adult patients that are coming in today have got delayed diagnosis. They've got complex disease histories, and we're starting to unlock that. The 50/50 ratio remains of our total population.

Neil McFarlane: Yeah, Jason, just to put a little bit of a finer point on this, we've said that the age distribution between adults and kids is about 50/50 in what we see today. Of these 184 enrollment forms, you should think of that as half adults and half children. When it comes to the newly diagnosed patients, it's really difficult for us to be able to say and provide that level of guidance.

Speaker #3: And what we see today, so of these 184 enrollment forms, you should think of that as half adults and half children. When it comes to the newly diagnosed patients, it's really difficult for us to be able to say, and provide that level of guidance.

Speaker #3: So for us, you should consider to say this is not just a childhood disease as we've been thinking about this for many, many decades.

Neil McFarlane: For us, you should consider to say this is not just a childhood disease, as we've been thinking about this for many, many decades. These adult patients that are coming in today have got delayed diagnosis. They've got complex disease histories, and we're starting to unlock that. The 50/50 ratio remains of our total population.

Speaker #3: These adult patients that are coming in today have got delayed diagnosis. They've got complex disease histories. And we're starting to unlock that. So the 50/50 ratio remains of our total population.

Speaker #5: Great. Thank you.

Jason Butler: Great. Thank you.

Jason Butler: Great. Thank you.

Speaker #4: And once again, as a reminder, it is STAR 1 on your telephone keypad to ask a question. We will move next to Brandon Folks with HC Wainwright.

Operator 1: Once again, as a reminder, it is star one on your telephone keypad to ask a question. We will move next to Brandon Fowlkes with H.C. Wainwright. Your line is open.

Operator: Once again, as a reminder, it is star one on your telephone keypad to ask a question. We will move next to Brandon Fowlkes with H.C. Wainwright. Your line is open.

Speaker #4: Your line is open.

Speaker #7: Hi. Thanks for taking my questions and congrats on another good quarter. Neil, maybe just how do you feel about enrollment forms today? You said halfway through 2026.

Brandon Fowlkes: Hi. Thanks very much, Chris, and congrats on another good quarter. Neil, how do you feel about enrollment forms today? You're halfway through 2026, when we think about the full year compared to the enrollment forms you generated in 2025. It does look like things are trending up on an enrollment basis, but do you have better visibility into enrollment trends today? Are you starting to get more comfortable with sort of a quarterly level of enrollment forms you expect going forward?

Brandon Folkes: Hi. Thanks very much, Chris, and congrats on another good quarter. Neil, how do you feel about enrollment forms today? You're halfway through 2026, when we think about the full year compared to the enrollment forms you generated in 2025. It does look like things are trending up on an enrollment basis, but do you have better visibility into enrollment trends today? Are you starting to get more comfortable with sort of a quarterly level of enrollment forms you expect going forward?

Speaker #7: When we think about the full year, compared to the enrollment forms you generated in 2025, it does look like things are trending up on an enrollment basis.

Speaker #7: But do you have better visibility into enrollment trends today? And are you starting to get more comfortable with sort of a quarterly level of enrollment forms you expect going forward?

Speaker #3: Thanks, Brandon. So yes, you asked a question in regards to 2025. We had 52 enrollments for the year. And halfway through this year, we're at about 23 enrollments.

Neil McFarlane: Thanks, Brandon. Yes, you asked a question in regards to 2025, we had 52 enrollments for the year. Halfway through this year, we're at about 23 enrollments. I think what gives me a lot of confidence is really around the opportunity to unlock newly diagnosed patients. We started seeing that around this time last year. Then in Q3, we saw more newly diagnosed patients, Q4, newly diagnosed patients. These efforts that Josh just mentioned previously around our bespoke AI model that's getting the EMR data along with the claims data together and helping our reps get to places where they can educate physicians that we believe have NPC patients. It's really driving both newly diagnosed as well as the previously diagnosed patients.

Neil McFarlane: Thanks, Brandon. Yes, you asked a question in regards to 2025, we had 52 enrollments for the year. Halfway through this year, we're at about 23 enrollments. I think what gives me a lot of confidence is really around the opportunity to unlock newly diagnosed patients. We started seeing that around this time last year.

Speaker #3: I think what gives me a lot of confidence is really around the opportunity to unlock newly diagnosed patients. We started seeing that around this time last year.

Speaker #3: And then in Q3, we saw more newly diagnosed patients. Q4, newly diagnosed patients. And these efforts that Josh just mentioned previously around our bespoke AI model that's getting the EMR data along with the claims data together and helping our reps get to places where they can educate physicians that believe that we believe have NPC patients it's really driving the both newly diagnosed as well as the previously diagnosed patients.

Neil McFarlane: Then in Q3, we saw more newly diagnosed patients, Q4, newly diagnosed patients. These efforts that Josh just mentioned previously around our bespoke AI model that's getting the EMR data along with the claims data together and helping our reps get to places where they can educate physicians that we believe have NPC patients. It's really driving both newly diagnosed as well as the previously diagnosed patients.

Speaker #3: So I'm pleased with where we are through the year, but I'm even more confident about the TAM being somewhere between that 350 and 900 because we're seeing these newly diagnosed patients earlier in the launch process than we would have expected today.

Neil McFarlane: I'm pleased with where we are through the year, but I'm even more confident about the TAM being somewhere between that 350 and 900, because we're seeing these newly diagnosed patients earlier in the launch process than we would've expected today. I'm hopeful that with continued investment on our side and others in the area in NPC, we'll continue to drive that forward.

Neil McFarlane: I'm pleased with where we are through the year, but I'm even more confident about the TAM being somewhere between that 350 and 900, because we're seeing these newly diagnosed patients earlier in the launch process than we would've expected today. I'm hopeful that with continued investment on our side and others in the area in NPC, we'll continue to drive that forward.

Speaker #3: And I'm hopeful that with continued investment on our side and others in the area and NPC, we'll continue to drive that forward.

Speaker #7: Thanks very much. And then maybe just one more for me. I heard you earlier commentary on Slipperlo. But any additional color you can provide coming out of that meeting, that one-queue meeting and sort of the continued dialogue with the FDA?

Speaker #7: Thanks very much. And then maybe just one more for me. I heard you earlier commentary on Slipperlo. But any additional color you can provide coming out of that meeting, that one-queue meeting and sort of the continued dialogue with the FDA? more confident in going ahead?

Brandon Fowlkes: Thanks very much. Then maybe just one more from me. I heard your earlier commentary on celiprolol. Any additional color you can provide coming out of that Q1 meeting and sort of the continued dialogue with the FDA? Did you come out of the meeting more confident in going ahead, or what gives you confidence in terms of sort of moving that program forward coming out of that meeting? Just any color would be helpful. Thank you.

Brandon Folkes: Thanks very much. Then maybe just one more from me. I heard your earlier commentary on celiprolol. Any additional color you can provide coming out of that Q1 meeting and sort of the continued dialogue with the FDA? Did you come out of the meeting more confident in going ahead, or what gives you confidence in terms of sort of moving that program forward coming out of that meeting? Just any color would be helpful. Thank you.

Speaker #7: that program forward coming out of that meeting? Just any color would be helpful. Thank you.

Speaker #3: Well, I would coin the meeting as informative. We went into trying to find ways to accelerate the clinical development program. And the questions we asked were responded to with opportunities for us to go out and do some homework.

Neil McFarlane: Well, I would coin the meeting as informative. We went in to try and find ways to accelerate the clinical development program, the questions we asked were responded to with opportunities for us to go out and do some homework. We kind of have several approaches that we're working on here. It's a parallel track, right? One is that we want to try and find ways to accelerate the clinical development through regulatory pathways, the other is really around us and the ability to drive enrollment so we can get to the events and get to this interim analysis. As you saw last quarter, we had two confirmed events. We have three confirmed events now in Q2. We're up to 66 patients enrolled of 150 patients in the study.

Neil McFarlane: Well, I would coin the meeting as informative. We went in to try and find ways to accelerate the clinical development program, the questions we asked were responded to with opportunities for us to go out and do some homework. We kind of have several approaches that we're working on here. It's a parallel track, right?

Speaker #3: And we've kind of have several approaches that we're working

Neil McFarlane: One is that we want to try and find ways to accelerate the clinical development through regulatory pathways, the other is really around us and the ability to drive enrollment so we can get to the events and get to this interim analysis. As you saw last quarter, we had two confirmed events. We have three confirmed events now in Q2. We're up to 66 patients enrolled of 150 patients in the study.

Speaker #3: accelerate the clinical development. Through regulatory pathways. And the other is really around us and the ability to drive enrollment so we can get to the events and get to this interim analysis.

Speaker #3: As you saw last quarter, we had two confirmed events coming out of the meeting. We now have three confirmed events in Q2.

Speaker #3: We're up to 66 patients enrolled of 150 patients in the study. But we'd like to be able to find ways to put our foot on the gas and that's what our second half plans are going to be discussing with the FDA.

Neil McFarlane: We'd like to be able to find ways to put our foot on the gas, that's what our H2 plans are going to be discussing with the FDA.

Neil McFarlane: We'd like to be able to find ways to put our foot on the gas, that's what our H2 plans are going to be discussing with the FDA.

Speaker #7: Great. Thanks very much.

Brandon Fowlkes: Great. Thanks very much.

Brandon Folkes: Great. Thanks very much.

Speaker #4: And we will take our next question from Lachlan Humberry Brown with William Blair. Your line is open.

Operator 1: We will take our next question from Lachlan Hanbury-Brown with William Blair. Your line is open.

Operator: We will take our next question from Lachlan Hanbury-Brown with William Blair. Your line is open.

Speaker #6: Hey guys, thanks for the questions. Maybe the first Neil going back to sort of some of the prior questions, but just wondering what gives you confidence that you can be successful in the re-examination?

Lachlan Hanbury-Brown: Hey, guys. Thanks for the questions. Maybe the first, Neil McFarlane, going back to sort of some of the prior questions, just wondering what gives you confidence that you can be successful in the re-examination? I understand your point about confidence in the overall package, but sort of confidence in maybe being able to get the rapporteurs over the line or beyond the hurdles that they've seen in this initial review, because I don't typically associate EMA with being overly flexible. Maybe a second question, just wondering if the IP win for MIPLYFFA in the US changes how you think about your ability or maybe appetite to invest in the MIPLYFFA franchise in the US.

Lachlan Hanbury-Brown: Hey, guys. Thanks for the questions. Maybe the first, Neil McFarlane, going back to sort of some of the prior questions, just wondering what gives you confidence that you can be successful in the re-examination? I understand your point about confidence in the overall package, but sort of confidence in maybe being able to get the rapporteurs over the line or beyond the hurdles that they've seen in this initial review, because I don't typically associate EMA with being overly flexible.

Speaker #6: I understand your point about confidence in the overall package, but sort of confidence in maybe being able to get the rapporteurs over the line or beyond the hurdles that they've seen in this initial review?

Speaker #6: Because I don't typically associate EME with being overly flexible. And then, maybe a second question—just wondering if the IP win for MY platform in the US changes how you think about your ability, or maybe your appetite, to invest in the MY platform franchise in the US.

Lachlan Hanbury-Brown: Maybe a second question, just wondering if the IP win for MIPLYFFA in the US changes how you think about your ability or maybe appetite to invest in the MIPLYFFA franchise in the US.

Speaker #3: So let me start with your first question. I'm going to be thoughtful in the level of detail I provide, but our confidence is fully grounded in the totality of the evidence and the unmet need, right?

Neil McFarlane: Let me start with your first question. I'm going to be thoughtful in the level of detail I provide, but our confidence is fully grounded in the totality of the evidence and the unmet need, right? We mentioned, again, 132 patients in our global EAP. The majority of those patients are patients that are in the European Union. I think that that's an important perspective. We continue to see the demand from patients. When it comes to the CHMP and their assessment, this process that we're working through, and the stage that we're in around the re-examination process, is really around now focusing on that further interpretation of the evidence that we've provided around the open label extension, the EAP, and the pediatric sub-studies. It starts with our pivotal study, right?

Neil McFarlane: Let me start with your first question. I'm going to be thoughtful in the level of detail I provide, but our confidence is fully grounded in the totality of the evidence and the unmet need, right? We mentioned, again, 132 patients in our global EAP. The majority of those patients are patients that are in the European Union. I think that that's an important perspective.

Speaker #3: We mentioned again, 132 patients in our global EAP. The majority of those patients are patients that are in the European Union. And I think that that's an important perspective.

Speaker #3: So we continue to see the demand from patients when it comes to the CHMP and their assessment. This process that we're working through and the stage that we're in around the re-examination process is really around now focusing on that further interpretation of the evidence that we've provided around the open label extension, the EAP, and the pediatric sub-studies.

Neil McFarlane: We continue to see the demand from patients. When it comes to the CHMP and their assessment, this process that we're working through, and the stage that we're in around the re-examination process, is really around now focusing on that further interpretation of the evidence that we've provided around the open label extension, the EAP, and the pediatric sub-studies. It starts with our pivotal study, right?

Speaker #3: But it also starts with our pivotal study, right? It's primarily centered on the analysis of the pivotal study as the foundation of this application.

Neil McFarlane: It's primarily centered on the analysis of the pivotal study as the foundation of this application. We've got a positive, clinically meaningful, and statistically significant clinical study. That's what gives us the first part of confidence. The rest of it is around the comprehensive evidence package. I can't give you a probability, but what I can tell you is that we're bolstering our clinical trial with long-term clinical experience supported by multi-years of safety database with no new safety concerns. We believe strongly that the benefit risk is in favor of MIPLYFFA for patients in the EU. The second question that you had was in regards to, I'm going to take that as life cycle management and IP. Yes, we're very happy with the longer duration of intellectual property protection today.

Neil McFarlane: It's primarily centered on the analysis of the pivotal study as the foundation of this application. We've got a positive, clinically meaningful, and statistically significant clinical study. That's what gives us the first part of confidence. The rest of it is around the comprehensive evidence package. I can't give you a probability, but what I can tell you is that we're bolstering our clinical trial with long-term clinical experience supported by multi-years of safety database with no new safety concerns.

Speaker #3: So we've got a positive clinically meaningful and statistically significant clinical study. That's what gives us the first part of confidence. The rest of it is around the comprehensive evidence package.

Speaker #3: So I can't give you a probability, but what I can tell you is that we're bolstering our clinical trial with long-term clinical experience supported by multi-years of safety database with no new safety concerns and we believe strongly that the benefit risk is in favor of my platform for patients in the EU.

Neil McFarlane: We believe strongly that the benefit risk is in favor of MIPLYFFA for patients in the EU. The second question that you had was in regards to, I'm going to take that as life cycle management and IP. Yes, we're very happy with the longer duration of intellectual property protection today.

Speaker #3: The second question that you had was in regards to—I'm going to take that as life cycle management and IP. Yes, we're very happy with the longer duration of intellectual property protection today.

Speaker #3: And that does give us a lens towards looking at how we can further invest the capital Justin was talking about earlier today into the US business, but also into the European and global expansion along with the Slipperlo program.

Neil McFarlane: That does give us a lens towards looking at how we can further invest the capital Justin Renz was talking about earlier today into the US business, but also into the European and global expansion along with the celiprolol program. I leave it at that at this point in the game. I don't want to say that we have a specific action that we'll take with the current extension, but we are looking forward to maximize the value of MIPLYFFA over the long haul.

Neil McFarlane: That does give us a lens towards looking at how we can further invest the capital Justin Renz was talking about earlier today into the US business, but also into the European and global expansion along with the celiprolol program. I leave it at that at this point in the game. I don't want to say that we have a specific action that we'll take with the current extension, but we are looking forward to maximize the value of MIPLYFFA over the long haul.

Speaker #3: So I kind of leave it at that at this point in the game. I don't want to say that we have a specific action that we'll take with the current extension, but we are looking forward to maximize the value of my platform over the long haul.

Speaker #6: Got it. Thanks.

Lachlan Hanbury-Brown: John, thanks.

Lachlan Hanbury-Brown: John, thanks.

Speaker #4: And we will take our next question from Combiz Yazdi with BTIG. Your line is open.

Operator 1: We will take our next question from Kambiz Yazdi with BTIG. Your line is open.

Operator: We will take our next question from Kambiz Yazdi with BTIG. Your line is open.

Speaker #7: Hi team. Congratulations on the quarter. Just a couple for me. On the re-examination, how are you thinking about the role of a SAG meeting and KOL testimony potentially in the process?

Kambiz Yazdi: Hi, team. Congratulations on the quarter. Just a couple for me. On the re-examination, how are you thinking about the role of a SAG meeting and KOL testimony potentially in the process? Also maybe on your strong cash position, can you speak about your philosophy on share buybacks? Thank you.

Kambiz Yazdi: Hi, team. Congratulations on the quarter. Just a couple for me. On the re-examination, how are you thinking about the role of a SAG meeting and KOL testimony potentially in the process? Also maybe on your strong cash position, can you speak about your philosophy on share buybacks? Thank you.

Speaker #7: And also maybe on your strong cast position, can you speak about your philosophy on share buybacks? Thank you.

Neil McFarlane: I'll take the first one, and then I'll ask Justin to comment on our capital allocation plans. The answer to that question is, we did resubmit for re-examination, and we have requested a SAG meeting. That's part of bringing new expert voices into the procedural discussions, and we will take advantage of that. Justin?

Neil McFarlane: I'll take the first one, and then I'll ask Justin to comment on our capital allocation plans. The answer to that question is, we did resubmit for re-examination, and we have requested a SAG meeting. That's part of bringing new expert voices into the procedural discussions, and we will take advantage of that. Justin?

Speaker #3: ask Justin to comment on our capital allocation plans. The answer to that question is we did resubmit for re-examination and we have requested a SAG meeting.

Speaker #3: That's part of bringing new expert voices into the procedural discussions. And we will take advantage of that. Justin?

Speaker #6: Yeah. Thank you. And again, reiterating what we said earlier, we're in growth mode. Our priorities remain unchanged. We can start strategic plan right now and that's driving my pipe of growth, pursuing global opportunities.

Justin Renz: Yeah, thank you. Again, reiterating what we said earlier, we are in growth mode. Our priorities remain unchanged against our strategic plan right now, that is driving MIPLYFFA growth, pursuing global opportunities, advancing celiprolol, again, as we discussed, prudently at least exploring the opportunities that we might be able to invest in perhaps arimoclomol and other indications. We are going to be very much open-minded to things. We have a board, and we discuss these topics, but right now, that is not part of our current capital allocation strategy.

Justin Renz: Yeah, thank you. Again, reiterating what we said earlier, we are in growth mode. Our priorities remain unchanged against our strategic plan right now, that is driving MIPLYFFA growth, pursuing global opportunities, advancing celiprolol, again, as we discussed, prudently at least exploring the opportunities that we might be able to invest in perhaps arimoclomol and other indications. We are going to be very much open-minded to things. We have a board, and we discuss these topics, but right now, that is not part of our current capital allocation strategy.

Speaker #6: Advancing Slipperlo and then again, as we just discussed, prudently at least exploring the opportunities that we might be able to invest in perhaps Aramaco Mall and other indications.

Speaker #6: So we're going to be very much open-minded to things. So we have a board and we discuss these topics, but right now that is not part of our current capital allocation strategy.

Speaker #7: Thank you so much.

Kambiz Yazdi: Thank you so much.

Kambiz Yazdi: Thank you so much.

Speaker #4: And we will take a follow-up question from Sumant Kulkarni with Canaccord Genuity. Please go ahead.

Operator 1: We will take a follow-up question from Sumant Kulkarni with Canaccord Genuity. Please go ahead.

Operator: We will take a follow-up question from Sumant Kulkarni with Canaccord Genuity. Please go ahead.

Speaker #7: Thanks for the follow-up. So is there anything you can share about what your latest comparative intelligence tells you about the remainder of the diagnosed and treated patients in the US that are not yet on my platform, especially in terms of there being on the other approved product that's Intrabios at Neursa?

Sumant Kulkarni: Thanks for the follow-up. Is there anything you can share about what your latest competitive intelligence tells you about the remainder of the diagnosed and treated patients in the US that are not yet on MIPLYFFA, especially in terms of their being on the other approved product that is IntraBio's Aqneursa?

Sumant Kulkarni: Thanks for the follow-up. Is there anything you can share about what your latest competitive intelligence tells you about the remainder of the diagnosed and treated patients in the US that are not yet on MIPLYFFA, especially in terms of their being on the other approved product that is IntraBio's Aqneursa?

Speaker #3: Yeah. I recall that this question. It's important. We believe they're complementary mechanisms of action. And I think the treatment guidelines reinforce what we've been saying for many years now that when you have a heterogeneous disease that has multi-symptomatology and just devastating progression, it's important to have as many complementary mechanisms as possible that you can utilize I'll ask Josh to talk a little bit more about what we see in the marketplace, but what we have been seeing is the guidelines along with our current real-world evidence that patients and physicians want options and we're seeing combination therapy being a not just a recognized approach, but a more common than not approach.

Neil McFarlane: Yeah, I recall this question. It's important. We believe they're complementary mechanisms of action. I think the treatment guidelines reinforce what we've been saying for many years now, that when you have a heterogeneous disease that has multi-symptomatology and just devastating progression, it's important to have as many complementary mechanisms as possible that you can utilize. I'll ask Josh to talk a little bit more about what we see in the marketplace. What we have been seeing is the guidelines along with our current real-world evidence that patients and physicians want options, and we're seeing combination therapy being a, not just a recognized approach, but a more common than not approach.

Neil McFarlane: Yeah, I recall this question. It's important. We believe they're complementary mechanisms of action. I think the treatment guidelines reinforce what we've been saying for many years now, that when you have a heterogeneous disease that has multi-symptomatology and just devastating progression, it's important to have as many complementary mechanisms as possible that you can utilize.

Neil McFarlane: I'll ask Josh to talk a little bit more about what we see in the marketplace. What we have been seeing is the guidelines along with our current real-world evidence that patients and physicians want options, and we're seeing combination therapy being a, not just a recognized approach, but a more common than not approach.

Speaker #7: Yeah. As Neil mentioned and as we've talked about previously, Sumant, the market is really moving towards a combination therapy because the treatments that are available are very complementary to one another.

Josh Schafer: Yeah. As Neil mentioned, and as we've talked about previously, Sumant, the market is really moving towards a combination therapy because the treatments that are available are very complementary to one another. This was reinforced in the treatment guidelines that came out earlier this year. Also important in those guidelines, it was recognized that MIPLYFFA was a disease-modifying treatment and is the only treatment that's FDA-indicated in combination use with miglustat. As the market moves towards combination therapy, I think it really supports MIPLYFFA's ongoing use as foundational therapy. To your question about other products out there, we don't really see this as an either/or market. Many patients, certainly we know that many MIPLYFFA patients are receiving miglustats and perhaps other treatments.

Josh Schafer: Yeah. As Neil mentioned, and as we've talked about previously, Sumant, the market is really moving towards a combination therapy because the treatments that are available are very complementary to one another. This was reinforced in the treatment guidelines that came out earlier this year. Also important in those guidelines, it was recognized that MIPLYFFA was a disease-modifying treatment and is the only treatment that's FDA-indicated in combination use with miglustat.

Speaker #7: And this was reinforced in the treatment guidelines that came out earlier this year. Also important in those guidelines, it was recognized that my platform was a disease-modifying treatment and is the only treatment that's FDA-indicated in combination use with MEGLISTAT.

Speaker #7: So, as the market moves towards combination therapy, I think it really supports my platform's ongoing use as foundational therapy. To your question about other products out there—

Josh Schafer: As the market moves towards combination therapy, I think it really supports MIPLYFFA's ongoing use as foundational therapy. To your question about other products out there, we don't really see this as an either/or market. Many patients, certainly we know that many MIPLYFFA patients are receiving miglustats and perhaps other treatments.

Speaker #7: We don't really see this as an either-or market. Many patients certainly we know that many my platform patients are receiving MEGLISTAT and perhaps other treatments.

Speaker #7: And so we look at this really as an opportunity to continue to grow our share of those diagnosed patients and more importantly, the undiagnosed patients, the activities that we're doing now, we're seeing new enrollments coming in on a regular basis for newly diagnosed patients, all of which gives us real confidence that the overall market size is what we think it is of 350 to 900 patients.

Josh Schafer: We look at this really as an opportunity to continue to grow our share of those diagnosed patients and more importantly, the undiagnosed patients. The activities that we're doing now, we're seeing new enrollments coming in on a regular basis for newly diagnosed patients, all of which gives us real confidence that the overall market size is what we think it is of 350 to 900 patients.

Josh Schafer: We look at this really as an opportunity to continue to grow our share of those diagnosed patients and more importantly, the undiagnosed patients. The activities that we're doing now, we're seeing new enrollments coming in on a regular basis for newly diagnosed patients, all of which gives us real confidence that the overall market size is what we think it is of 350 to 900 patients.

Speaker #3: Thanks.

Sumant Kulkarni: Thanks.

Sumant Kulkarni: Thanks.

Speaker #4: Thank you. And we will move next to Jonathan Ashoff with Roth Capital Partners. Your line is open.

Operator 1: Thank you. We will move next to Jonathan Aschoff with Roth Capital Partners. Your line is open.

Operator: Thank you. We will move next to Jonathan Aschoff with Roth Capital Partners. Your line is open.

Speaker #5: Thank you. Solid quarter, guys. Can you just two things. Can you tell us anything about patient attrition, any commercial patients who are no longer taking the drug and maybe a percentage there?

Jonathan Aschoff: Thank you. Solid quarter, guys. Just two things. Can you tell us anything about patient attrition? Any commercial patients who are no longer taking the drug and maybe a percentage there? How did the end of Q2 inventory compare to the end of Q1?

Jonathan Aschoff: Thank you. Solid quarter, guys. Just two things. Can you tell us anything about patient attrition? Any commercial patients who are no longer taking the drug and maybe a percentage there? How did the end of Q2 inventory compare to the end of Q1?

Speaker #5: And how did the end of Q2 inventory compare to the end of Q2?

Speaker #3: Jonathan, I'll ask Josh to talk a little bit about the refill rate persistency and what we see in the market today. And then I'll swap it over to Justin to talk a little bit about the ending inventory.

Neil McFarlane: Jonathan, I'll ask Josh to talk a little bit about the refill rate persistency and what we see in the market today, and then I'll swap it over to Justin to talk a little bit about the ending inventory.

Neil McFarlane: Jonathan, I'll ask Josh to talk a little bit about the refill rate persistency and what we see in the market today, and then I'll swap it over to Justin to talk a little bit about the ending inventory.

Speaker #7: Yeah. With regards to attrition, well, first of all, as we noted, we've got about 69% of covered lives so we are very confident in our in the access that we're able to provide for patients, either through direct formulary for those 69% of covered lives.

Josh Schafer: Yeah. With regards to attrition, well, first of all, as we noted, we've got about 69% of covered lives. We are very confident in the access that we're able to provide for patients, either through direct formulary for those 69% of covered lives or through some sort of medical exception pathway. We're seeing that most of the patients who are enrolled to receive MIPLYFFA do indeed receive MIPLYFFA. Our refill rates and compliance rates or persistence rates are really quite high. The patients that we do lose, unfortunately, it's really a reflection more of the severity of the disease. It is an oftentimes fatal disease, and patients will progress to the point of dying. The patients who we have lost and are no longer taking MIPLYFFA, the vast majority of those are patients who have unfortunately passed away.

Josh Schafer: Yeah. With regards to attrition, well, first of all, as we noted, we've got about 69% of covered lives. We are very confident in the access that we're able to provide for patients, either through direct formulary for those 69% of covered lives or through some sort of medical exception pathway. We're seeing that most of the patients who are enrolled to receive MIPLYFFA do indeed receive MIPLYFFA.

Speaker #7: Or through some sort of medical exception pathway. And we're seeing that most of the patients who are enrolled to receive my platform do indeed receive my platform.

Speaker #7: Our refill rates and compliance rates or persistence rates are really quite high. The patients that we do lose unfortunately are it's really a reflection more of the severity of the disease.

Josh Schafer: Our refill rates and compliance rates or persistence rates are really quite high. The patients that we do lose, unfortunately, it's really a reflection more of the severity of the disease. It is an oftentimes fatal disease, and patients will progress to the point of dying. The patients who we have lost and are no longer taking MIPLYFFA, the vast majority of those are patients who have unfortunately passed away.

Speaker #7: It is a oftentimes fatal disease and patients will progress to the point of dying and the patients who we have lost and are no longer taking my platform, the vast majority of those are patients who have unfortunately passed away.

Speaker #3: Yeah. Jonathan, just to put a finer edge on that, we had discussed that patients that get on my platform seem to stay on my platform.

Neil McFarlane: Yeah. Jonathan, just to put a finer edge on that. We had discussed that patients that get on MIPLYFFA seem to stay on MIPLYFFA. Our EAP data was very supportive of that effort. The rates that we saw in terms of the patients who, as Josh mentioned, unfortunately passed away, those rates were really super small, and we're continuing to see those similar kind of rates in our long-term data so far. Now, we're almost two years in. At the end of this year, we should be able to start seeing some more persistency, and maybe we'll be able to provide that metric later on. Justin, you want to talk a little bit about inventory?

Neil McFarlane: Yeah. Jonathan, just to put a finer edge on that. We had discussed that patients that get on MIPLYFFA seem to stay on MIPLYFFA. Our EAP data was very supportive of that effort. The rates that we saw in terms of the patients who, as Josh mentioned, unfortunately passed away, those rates were really super small, and we're continuing to see those similar kind of rates in our long-term data so far.

Speaker #3: Our EAP data was very supportive of that effort. The rates that we saw in terms of the patients who as Josh mentioned, unfortunately passed away, those rates were really super small and we're continuing to see those similar kind of rates in our long-term data so far.

Speaker #3: Now, we're almost two years in. At the end of this year, we should be able to start seeing some more persistency and maybe we'll be able to provide that metric later on.

Neil McFarlane: Now, we're almost two years in. At the end of this year, we should be able to start seeing some more persistency, and maybe we'll be able to provide that metric later on. Justin, you want to talk a little bit about inventory?

Speaker #3: Justin, you want to talk a little bit about inventory?

Speaker #5: So in general, we have a channel inventory target that we work with our specialty pharmacy partner on. And our channel inventory fell below our targeted range at the end of Q1.

Justin Renz: In general, we have a channel inventory target that we work with our specialty pharmacy partner on, our channel inventory fell below our targeted range at the end of Q1. It was truly because of the shipment timing and the dynamics around that. For Q2, it was essentially back to the normal target level that we aspire to. I believe you'll see a normalization going forward of this level.

Justin Renz: In general, we have a channel inventory target that we work with our specialty pharmacy partner on, our channel inventory fell below our targeted range at the end of Q1. It was truly because of the shipment timing and the dynamics around that. For Q2, it was essentially back to the normal target level that we aspire to. I believe you'll see a normalization going forward of this level.

Speaker #5: It was truly because of shipment timing and the dynamics around that. And for Q2, it was essentially back to the normal target level that we aspire to.

Speaker #5: So I believe you'll see a normalization going forward of this level. Okay. So just a wee bit higher. Back to normal is what you're saying.

Jonathan Aschoff: Okay. Just a wee bit higher. Back to normal is what you're saying.

Jonathan Aschoff: Okay. Just a wee bit higher. Back to normal is what you're saying.

Speaker #5: Correct. Thank you very much, guys.

Justin Renz: Correct.

Justin Renz: Correct.

Jonathan Aschoff: Thank you very much, guys.

Jonathan Aschoff: Thank you very much, guys.

Speaker #4: And this does conclude the Q&A portion of today's call. I'd like to turn the call back over to Neil McFarlane for any closing remarks.

Operator 1: This does conclude the Q&A portion of today's call. I'd like to turn the call back over to Neil McFarlane for any closing remarks.

Operator: This does conclude the Q&A portion of today's call. I'd like to turn the call back over to Neil McFarlane for any closing remarks.

Speaker #3: Yeah. Thank you for joining the call today, and we look forward to keeping you apprised of our future progress. Have a great evening.

Neil McFarlane: Thank you for joining the call today, and we look forward to keeping you apprised of our future progress. Have a great evening.

Neil McFarlane: Thank you for joining the call today, and we look forward to keeping you apprised of our future progress. Have a great evening.

Speaker #4: Thank you. This brings us to the end of today's meeting. We appreciate your time and participation. You may now disconnect.

Operator 1: Thank you. This brings us to the end of today's meeting. We appreciate your time and participation. You may now disconnect.

Operator: Thank you. This brings us to the end of today's meeting. We appreciate your time and participation. You may now disconnect.

David Brown: To all locations on hold for Zevra Therapeutics' call, we have now concluded. Please disconnect at this time.

Operator: To all locations on hold for Zevra Therapeutics' call, we have now concluded. Please disconnect at this time.

Q2 2026 Zevra Therapeutics Inc Earnings Call

Demo
ZVRA

Zevra Therapeutics

Earnings

Q2 2026 Zevra Therapeutics Inc Earnings Call

ZVRA

Wednesday, August 5th, 2026 at 8:30 PM

Transcript

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