Q2 2024 Zevra Therapeutics Inc Earnings Call

August 13, 2024

Operator: John Dr. Thomas Lugo, Neil McFarlane, Oren, E. E. E. E. E. E. Please stand by, your program is about to begin. Hello, and thank you for joining the Zevra Therapeutics Q2 2024 Financial Results and Corporate Highlights. Today's call is being recorded and will be made available on the company's website following the conclusion of the call.

Unknown Executive: Hello, and thank you for joining the Zevra Therapeutics Q2 2024 financial results and corporate highlights call. Today's call is being recorded and will be made available on the company's website following the conclusion of the call.

Speaker Change: [inaudible]

Speaker Change: Hello and thank you for joining the Zebra Therapeutics Q2 2024 Financial Results and Corporate Highlights Call. Today's call is being recorded and will be made available on the company's website following the conclusion of the call. With that, I will now turn the call over to Nichol Ochsner, Vice President of Investor Relations and Corporate Communications for Zebra Therapeutics.

Nichol Ochsner: With that, I will now turn the call over to Nichol Ochsner, Vice President of Investor Relations and Corporate Communications for Zevra Therapeutics.

Nichol Ochsner: With that, I will now turn the call over to Nichol Ochsner, Vice President of Investor Relations and Corporate Communications for Zevra Therapeutics. Good afternoon, and thank you for joining us today to review Zevra Therapeutics' progress in the second quarter of 2024, outlining our clinical advances, operational achievements, and financial results. Before we get started, let me take a moment to provide some important information.

Nichol Ochsner: Good afternoon, and thank you for joining us today to review Zevra Therapeutics' progress in the second quarter of 2024, outlining our clinical advances, operational achievements, and financial results. Before we get started, let me take a moment to provide some important information. I encourage you to access the news release, which was published this afternoon and is available in the investor section of the Zevra website. As we begin our call, it's important to highlight that certain information covered in today's discussions will include forward-looking statements. We caution listeners that actual results could differ materially from those stated or implied by these forward-looking statements due to risks and uncertainties associated with the company's business.

Nichol Ochsner: Good afternoon and thank you for joining us today to review Zebra Therapeutics progress in the second quarter of 2024, outlining our clinical advances, operational achievements, and financial results. Before we get started, let me take a moment to provide some important information.

Nichol Ochsner: I encourage you to access the news release, which was published this afternoon and is available in the investor section of the Zevra website. Before we begin our call, it's important to highlight that certain information covered in today's discussions will include forward-looking statements. We caution listeners that actual results could differ materially from those stated or implied by these forward-looking statements due to risks and uncertainties associated with the company's business. Forward-looking statements are not promises or guarantees and are inherently subject to risks, uncertainties, and other significant factors that may lead to actual results differing materially from those projected.

Speaker Change: I encourage you to access the news release which was published this afternoon and is available in the investor section of the Zebra website.

Nichol Ochsner: These forward-looking statements are qualified by the cautionary statements contained in the risk factors section of our most recent quarterly report on Form 10-Q and our other filings with the SEC, including our annual report on Form 10-K. I'm pleased to welcome Zevra's management team members participating in today's call. I'm joined by Neil McFarlane, President and Chief Executive Officer, Ledwane Clifton, our Chief Financial Officer, Josh Schafer, our Chief Commercial Officer and Executive Vice President of Business Development, and Adrian Quartel, our Chief Medical Officer. Now, I'll turn the call over to Neil.

Speaker Change: As we begin our call, it's important to highlight that certain information covered in today's discussions will include forward-looking statements. We caution listeners that actual results could differ materially from these stated or implied by these forward-looking statements due to risks and uncertainties associated with the company's business.

Nichol Ochsner: Forward-looking statements are not promises or guarantees and are inherently subject to risks, uncertainties, and other significant factors that may lead to actual results differing materially from those projections made. These forward-looking statements are qualified by the cautionary statements contained in the risk factor section in our most recent quarterly report on Form 10-Q and our other filings with the SEC Annual Report on Form 10-K.

Speaker Change: Forward-looking statements are not promises or guarantees and are inherently subject to risks, uncertainties, and other significant factors.

Speaker Change: that may lead to actual results differing materially from those projections made. These forward-looking statements are qualified by the cautionary statements contained in the risk factors section.

Speaker Change: and our most recent quarterly report on Form 10-Q and our other filings with the SEC annual report on Form 10-K.

Nichol Ochsner: I'm pleased to welcome Zevra's management team members participating in today's call. I'm joined by Neil McCarline, President and Chief Executive Officer, Lydwayne Clifton, our Chief Financial Officer, Josh Schaeffer, our Chief Commercial Officer and Executive Vice President of Business Development, and Adrian Quartel, our Chief Medical Officer.

Speaker Change: I'm pleased to welcome ZEVR's management team members participating in today's call. I'm joined by Neil McFarlane, President and Chief Executive Officer, Lydwane Clifton, our Chief Financial Officer,

Neil McFarlane: Josh Schaefer, our Chief Commercial Officer and Executive Vice President of Business Development, and Adrienne Quartel, our Chief Medical Officer. Now, I'll turn the call over to Neil.

Neil McCarline: Now, I'll turn the call over to Neil. Thank you, Nicole, and thank you all for making the time to join us today. During the second quarter, we made steady progress executing on our strategic objectives, preparing for the advisory committee meeting and the potential launch of Aramaquemol, driving the launch of O'Proofa, and advancing KP 1077 for sleep disorders. These objectives are important building blocks for our long-term strategic plan to build a sustainable rare disease company with reliable cash flows. An important element of executing against our strategy is to build and maintain our position of financial strength.

Neil McFarlane: Thank you, Nichol. And thank you all for taking the time to join us today. During the second quarter, we made steady progress executing on our strategic objectives, preparing for the advisory committee meeting and the potential launch of our mock-a-mole, driving the launch of Olpruva, and advancing KP1077 for sleep disorder. These objectives are important building blocks for our long-term strategic plan to build a sustainable rare disease company with reliable cash flows. An important element of executing against our strategy is to build and maintain our position of financial strength.

Neil McFarlane: Thank you, Nichol, and thank you all for making the time to join us today.

Neil McFarlane: During the second quarter, we made steady progress executing on our strategic objectives.

Speaker Change: preparing for the advisory committee meeting and the potential launch of our mock-a-mole.

Speaker Change: driving the launch of Olpruva and advancing KP 1077 for sleep disorders. These objectives are important building blocks for our long-term strategic plan to build a sustainable rare disease company with reliable cash flows.

Speaker Change: An important element of executing against our strategy is to build and maintain our position of financial strength. During the first half of 2024, we made investor outreach a primary objective, highlighting the opportunities and catalysts for value creation that we have as a company.

Neil McCarline: During the first half of 2024, we made investor outreach a primary objective, highlighting the opportunities and catalysts for value creation that we have as a company. There has been a significant interest in growing momentum as we continue to execute our objectives. Last week, following the favorable outcome of the FDA Advisory Committee meeting focused on Aramaquemol, we undertook a modestly sized underwritten public offering to capture that momentum and build on our base of investors as we lean into the potential of our near-term catalysts. With this funding, we have added net proceeds of approximately $64.5 million to our balance sheet, bringing our pro forma June 30th, 2024 cash, cash equivalence and investments to $113.8 million.

Neil McFarlane: During the first half of 2024, we made investor outreach a primary objective, highlighting the opportunities and catalysts for value creation that we have as a company. There has been significant interest and growing momentum as we continue to execute our objectives. Last week, following the favorable outcome of the FDA Advisory Committee meeting focused on Aramakamal, we undertook a modestly-sized underwritten public offering to capture that momentum and build on our base of investors as we lean into the potential of our near-term catalyst.

Speaker Change: There has been a significant interest and growing momentum as we continue to execute our objectives.

Speaker Change: Last week, following the favorable outcome of the FDA Advisory Committee meeting focused on Arimaukamal,

Speaker Change: We undertook a modestly-sized, underwritten public offering to capture that momentum and build on our base of investors as we lean into the potential of our near-term catalysts.

Neil McFarlane: With this funding, we have added net proceeds of approximately $64.5 million to our balance sheet, bringing our pro forma June 30, 2024 cash, cash equivalents, and investments to $113.8 million. This was the right time to demonstrate our confidence and take this important step to prepare for success.

Speaker Change: With this funding, we have added net proceeds of approximately $64.5 million to our balance sheet, bringing our pro forma June 30, 2024 cash equivalents and investments to $113.8 million.

Neil McCarline: This was the right time to demonstrate our confidence and take this important step to prepare for success. The proceeds will extend our cash runway and bolster our flexibility in executing both near and long-term objectives, including full preparation for the potential launch of Aramokomal and the flexibility to accelerate our clinical pipeline.

Speaker Change: This was the right time to demonstrate our confidence and take this important step to prepare for success.

Neil McFarlane: Proceeds will extend our cash runway and bolster our flexibility in executing both near and long-term objectives, including full preparation for the potential launch of Aramakumal and the flexibility to accelerate our clinical pipeline. Now, I'll share a summary of our key second quarter accomplishments and outline why we remain optimistic in our ability to deliver on our strategic plan. Let's start with Aram Akhlamal, our product candidate for Niemann-Pick disease type C, or NPC.

Speaker Change: The proceeds will extend our cash runway and bolster our flexibility in executing both near and long-term objectives, including full preparation for the potential launch of Aramakumal and the flexibility to accelerate our clinical pipeline.

Neil McCarline: Now, I'll share a summary of our key second quarter accomplishments and outline why we remain optimistic in our ability to deliver on our strategic plan. Let's start with Aramokomal, our product candidate for Neem and Pick Disease Type C, or NPC. On August 2nd, the Genetic Metabolic Diseases Advisory Committee, or GEMDAC, voted favorably that Aramokomal is effective in the treatment of NPC. While the vote is not binding, we believe this is an important factor as the FDA completes its review. Our Padoopa date is September 21st, which is fast approaching. We received the first round of labeling comments last Friday and are working closely with the FDA.

Speaker Change: Now I'll share a summary of our key second quarter accomplishments and outline why we remain optimistic in our ability to deliver on our strategic plan.

Neil McFarlane: On August 2nd, the Genetic Metabolic Diseases Advisory Committee, or GEMDAC, voted favorably that arimoclomol is effective in the treatment of NPC. While the vote is not binding, we believe this is an important factor as the FDA completes its review.

Speaker Change: Let's start with Aaron McLemore, our product candidate for Niemann-Pick disease type C or NPC.

Speaker Change: On August 2nd, the Genetic Metabolic Diseases Advisory Committee, or GEMDAC, voted favorably that arimoclomol is effective in the treatment of NPC. While the vote is not binding, we believe this is an important factor as the FDA completes its review.

Neil McFarlane: Our PDUFA date is September 21st, which is fast approaching. We received the first round of labeling comments last Friday and are working closely with the FDA. If approved, Arimocimol would be the first drug in the U.S. indicated for the treatment of NPC, and it would be eligible for a priority review voucher. As a reminder, there are roughly 900 people in the U.S. with NPC, of which approximately one-third are diagnosed and treated. More than 70 of those patients are currently enrolled in our U.S. Expanded Access Program, or EAP.

Speaker Change: Our PDUFA date is September 21st, which is fast approaching.

Speaker Change: We received the first round of labeling comments last Friday and are working closely with the FDA. If approved, Arimocimol would be the first drug in the U.S. indicated for the treatment of NPC, and it would be eligible for a priority review voucher.

Neil McCarline: If approved, Aramokomal would be the first drug in the US indicated for the treatment of NPC, and it would be eligible for a priority review voucher. As a reminder, there are roughly 900 people in the US with NPC, of which approximately one-third are diagnosed and treated. More than 70 of those patients are currently enrolled in our US Expanded Access Program, or EAP. As the FDA review continues, we will maintain the US EAP to ensure access for patients until commercial supply is available. In addition, subsequent to US approval, we will seek regulatory approval in Europe, where an additional 70-80 patients are enrolled in our global EAP program.

Speaker Change: As a reminder, there are roughly 900 people in the U.S. with NPC, of which approximately one-third are diagnosed and treated.

Speaker Change: More than 70 of those patients are currently enrolled in our U.S. Expanded Access Program, or EAP. As the FDA review continues, we will maintain the U.S. EAP to ensure access for patients until commercial supply is available.

Neil McFarlane: As the FDA review continues, we will maintain the U.S. EAP to ensure access for patients until commercial supply is available. In addition, subsequent to U.S. approval, we will seek regulatory approval in Europe, where an additional 70 to 80 patients are enrolled in our global EAP program. We are actively preparing for the commercial launch of Aramakamal. Our commercial infrastructure was built to optimize the strategic fit between Opruva and Aramakamal.

Speaker Change: In addition, subsequent to U.S. approval, we will seek regulatory approval in Europe, where an additional 70 to 80 patients are enrolled in our global EAP program.

Neil McCarline: We are actively preparing for the commercial launch of Aramokomal. Our commercial infrastructure was built to optimize the strategic fit between O'Proofa and Aramokomal. Both products address genetic metabolic diseases, with multi-disciplinary treatment teams that are often co-located within the same centers of excellence, allowing us to reach the majority of prescribers with a targeted commercial infrastructure. Our rare disease specialists and medical science liaisons, who are currently promoting and educating on O'Proofa and urea cycle disorders, or UCDs, are also engaging with prescribers when appropriate to raise awareness for NPC. Further, our market access team has initiated pre-approval clinical discussions with payers regarding Aramokomal as a potential treatment for NPC.

Speaker Change: We are actively preparing for the commercial launch of Aramakamal. Our commercial infrastructure was built to optimize the strategic fit between Opruva and Aramakamal.

Neil McFarlane: Both products address genetic metabolic diseases with multidisciplinary treatment teams that are often co-located within the same centers of excellence, allowing us to reach the majority of prescribers with a targeted commercial infrastructure. Our Rare Disease Specialists and Medical Science Liaisons, who are currently promoting and educating on Olprova and Urea Cycle Disorders, or UCDs, are also engaging with prescribers, when appropriate, to raise awareness for NPC. In addition, our market access team has initiated pre-approval clinical discussions with payers regarding arimoclimol as a potential treatment for NPC.

Speaker Change: Both products address genetic metabolic diseases with multidisciplinary treatment teams that are often co-located within the same centers of excellence, allowing us to reach the majority of prescribers with a targeted commercial infrastructure.

Speaker Change: Our rare disease specialists and medical science liaisons, who are currently promoting and educating on Olprova and urea cycle disorders, or UCDs, are also engaging with prescribers when appropriate to raise awareness for NPC.

Speaker Change: Further, our market access team has initiated pre-approval clinical discussions with payers regarding arimoclomol as a potential treatment for NPC.

Neil McCarline: These pre-launch activities will help ensure patients have access once available.

Neil McFarlane: These pre-launch activities will help ensure patients have access once available. Now, turning to Opruva, where we continue to make progress with the commercial launch initiated at the end of January 2024. Over the last few quarters, our assumption about the limited awareness of OPRUVA has been confirmed.

Speaker Change: These pre-launch activities will help ensure patients have access once available.

Neil McCarline: Now, turning to O'Proofa, where we continue to make progress with the commercial launch initiated at the end of January 2024. Over the last few quarters, our assumption on the limited awareness of O'Proofa has been confirmed. However, we've made progress with healthcare providers to increase awareness levels and identified that we have more work to do to increase patient awareness. As a reminder, there are 1,100 UCD patients in the U.S., of which more than 800 are receiving treatment. The prescribing community has identified a significant number of these patients who can benefit from O'Proofa. While we are encouraged by their response, the number of patient enrollments is not yet where we would like it to be.

Speaker Change: Now, turning to Opruva, where we continue to make process with the commercial launch initiated at the end of January 2024.

Speaker Change: Over the last few quarters, our assumption on the limited awareness of OPRUVA has been confirmed. However, we've made progress with healthcare providers to increase awareness levels and identify that we have more work to do to increase patient awareness.

Neil McFarlane: However, we've made progress with health care providers to increase awareness levels and identify that we have more work to do to increase patient awareness. As a reminder, there are 1,100 UCD patients in the U.S., of which more than 800 are receiving treatment. The prescribing community has identified a significant number of these patients who can benefit from Olprova. While we are encouraged by their response, the number of patient enrollments is not yet where we would like it to be. During the second quarter, we had nine new patient enrollments, which we define as a prescription for a patient who's on our Quick Start program or one who's receiving a paid dispensing.

Speaker Change: As a reminder, there are 1,100 UCD patients in the U.S., of which more than 800 are receiving treatment.

Speaker Change: The prescribing community has identified a significant number of these patients who can benefit from Olprova.

Speaker Change: While we are encouraged by their response, the number of patient enrollments is not yet where we would like it to be.

Neil McCarline: During the second quarter, we had nine new patient enrollments, which we define as a prescription for a patient who's on our Quick Start program or one who's receiving a paid dispense. We have been working to build awareness amongst clinicians who treat UCD and to ensure market access for patients. The team has done an outstanding job of engaging HCPs at target centers of excellence and at medical conferences to build brand awareness. The team has successfully engaged the majority of clinicians and thought leaders who diagnose and treat UCD patients, which is remarkable since access to HCPs has become more challenging across the industry.

Speaker Change: During the second quarter, we had nine new patient enrollments, which we define as a prescription for a patient who's on our QuickStart program or one who's receiving a paid dispense.

Neil McFarlane: We have been working to build awareness amongst clinicians who treat UCD and to ensure market access for patients. The team has done an outstanding job of engaging HCPs at target centers of excellence and at medical conferences to build brand awareness. It has successfully engaged the majority of clinicians and thought leaders who diagnose and treat UCD patients, which is remarkable since access to HCPs has become more challenging across the industry. Additionally, our managed care team continues to engage with government and commercial payers to ensure broad access for patients.

Speaker Change: We have been working to build awareness amongst clinicians who treat UCD and to ensure market access for patients.

Speaker Change: The team has done an outstanding job of engaging HCPs at target centers of excellence and at medical conferences to build brand awareness.

Speaker Change: The team has successfully engaged the majority of clinicians and thought leaders who diagnose and treat UCD patients, which is remarkable since access to HCPs has become more challenging across the industry.

Neil McCarline: Additionally, our managed care team continues to engage with government and commercial payers to ensure broad access for patients. We have increased O'Proofa coverage to 75% of covered lives with improved formulary status across healthcare plans and have established comprehensive patient services programs designed to assist with the reimbursement hurdles experienced by the rare disease community. As I mentioned earlier, there are always opportunities for refinement during lunch, and we are implementing changes to improve patient engagement. One key change was our transition to Orcini as our specialty pharmacy partner, who is a leader in pharmacy solutions for rare diseases.

Speaker Change: Additionally, our Managed Care Team continues to engage with government and commercial payers to ensure broad access for patients.

Neil McFarlane: We have increased OPRUVA coverage to 75% of covered lives with improved formulary status across healthcare plans and have established comprehensive patient services programs designed to assist with the reimbursement hurdles experienced by the rare disease community. As I mentioned earlier, there are always opportunities for refinement during launch, and we are implementing changes to improve patient engagement. One key change was our transition to Orsini as our specialty pharmacy partner, who is a leader in pharmacy solutions for rare diseases. This transition, completed in mid-June, included a rebalancing of our Opruva inventory in the channel, which impacted our net sales revenue in the quarter. LeDuane will provide more details later in the call.

Speaker Change: We have increased OPRUVA coverage to 75% of covered lives with improved formulary status across health care plans and have established comprehensive patient services programs designed to assist with the reimbursement hurdles experienced by the rare disease community.

Speaker Change: As I mentioned earlier, there are always opportunities for refinement during launch, and we are implementing changes to improve patient engagement.

Speaker Change: One key change was our transition to Orsini as our specialty pharmacy partner, who is a leader in pharmacy solutions for rare diseases.

Neil McCarline: This transition completed in mid-June, including a rebalancing of our O'Proofa inventory in the channel, which impacted our net sales revenue in quarter. The Dwayne will provide more details later in the call. We believe these enhancements to our commercial infrastructure will further support the O'Proofa launch and will have a positive impact on our commercialization efforts for our Moclemall with limited incremental cost.

Speaker Change: This transition completed in mid-June, including a rebalancing of our Alprova inventory in the channel, which impacted our net sales revenue in the quarter. LeDuane will provide more details later in the call.

Neil McFarlane: We believe these enhancements to our commercial infrastructure will further support the approval launch and will have a positive impact on our commercialization efforts for Aramakamal with limited incremental costs. Now, I'd like to turn your attention to KP1077, our clinical candidate for the treatment of idiopathic hypersomnia, or IH, a rare chronic sleep disorder. IH is characterized by excessive daytime sleepiness and difficulty waking, also known as sleep inertia.

LeDuane: We believe these enhancements to our commercial infrastructure will further support the approval launch and will have a positive impact on our commercialization efforts for Aramaka Mall with limited incremental costs.

Neil McCarline: Now I would like to turn your attention to KP1077, our clinical candidate for the treatment of idiopathic hypersomnia or IH, a rare chronic sleep disorder. IH is characterized by excessive daytime sleepiness and difficulty waking, also known as sleep inertia. This disease impacts approximately 37,000 people in the U.S. As you may recall, KP1077 is comprised of CERDEX Mepofenedate or SDX, which was designed to steadily release the Mepofenedate its active ingredient. Its unique pharmacokinetic profile allows for flexible dosing to overcome these primary IH symptoms and ensures patients receive the optimal drug concentration during waking hours. SDX is currently designated as a schedule for controlled substance by the U.S.

Neil McFarlane: This disease impacts approximately 37,000 people in the U.S. As you may recall, KP1077 is comprised of SIRDEX methylphenidate, or SDX, which was designed to steadily release methylphenidate, its active ingredient. Its unique pharmacokinetic profile allows for flexible dosing to overcome these primary IH symptoms and ensures patients receive the optimal drug concentration during waking hours. SDX is currently designated as a Schedule IV controlled substance by the U.S. Drug Enforcement Administration due to its demonstrated lower risk for abuse potential.

Speaker Change: Now I'd like to turn your attention to KP1077, our clinical candidate for the treatment of idiopathic hypersomnia, or IH, a rare chronic sleep disorder.

Speaker Change: IH is characterized by excessive daytime sleepiness and difficulty waking, also known as sleep inertia. This disease impacts approximately 37,000 people in the U.S.

Speaker Change: As you may recall, KP1077 is comprised of SIRDEX methylphenidate, or SDX, which was designed to steadily release demethylphenidate, its active ingredient.

Speaker Change: Its unique pharmacokinetic profile allows for flexible dosing to overcome these primary IH symptoms and ensures patients receive the optimal drug concentration during waking hours.

Speaker Change: SDX is currently designated as a Schedule IV controlled substance by the U.S. Drug Enforcement Administration due to demonstrated lower risk for abuse potential.

Neil McCarline: Drug Enforcement Administration due to demonstrated lower risk or abuse potential. At the Sleep 2024 meeting in June, we presented the pharmacokinetics of SDX when administered in the morning and at night. The clinical data showed peak exposure occurs the morning after a nighttime dose, when the patient needs it most to manage sleep inertia. We also reported positive results from our Phase II clinical study of KP1077 in patients with IH. In this proof-of-concept study, KP1077 was well tolerated at all dose levels, including the notably high dose of 320 milligrams daily. Adverse events throughout the study were mild, similar to other methylphenidate products, and did not lead to early discontinuation.

Neil McFarlane: At the SLEAP 2024 meeting in June, we presented the pharmacokinetics of SDX when administered in the morning and at night. Clinical data showed peak exposure occurs the morning after a nighttime dose, when the patient needs it most to manage sleep inertia. We also reported positive results from our Phase II clinical study of KP1077 in patients with IH. In this proof of concept study, KP1077 was well tolerated at all dose levels, including the notably high dose of 320 milligrams daily.

Speaker Change: At the SLEAP 2024 meeting in June, we presented the Pharmacokinetics of SDX when administered in the morning and at night.

Speaker Change: The clinical data showed peak exposure occurs the morning after a nighttime dose, when the patient needs it most to manage sleep inertia.

Speaker Change: We also reported positive results from our Phase II clinical study of KP1077 in patients with IH.

Speaker Change: In this proof-of-concept study, KP1077 was well tolerated at all dose levels, including the notably high dose of 320 mg daily.

Neil McFarlane: Adverse events throughout the study were mild, similar to other methylphenidate products, and did not lead to early discontinuation. AP 1077 showed clinically meaningful benefits in change from baseline at the end of seven weeks of treatment against secondary and exploratory endpoints, which included change in the Epworth sleepiness scale. The IH Severity Scale, the Sleep Inertia Visual Analog Scale, and a relatively new scale to assess the symptoms and severity of brain fog.

Speaker Change: Adverse events throughout the study were mild, similar to other methylphenidate products, and did not lead to early discontinuation.

Neil McCarline: KP1077 showed clinically meaningful benefits in change from baseline at the end of seven weeks of treatment against secondary and exploratory endpoints, which included change in the upward sleepiness scale, the IH severity scale, the sleep inertia visual analog scale, and a relatively new scale to assess the symptoms and severity of brain fog. We are encouraged by these results showing that KP1077 is well tolerated and demonstrates clinically meaningful benefits. Importantly, the study successfully fulfilled the objectives of informing the design of a PIVO efficacy trial. We consulted with key opinion leaders, hares, and patient advocates knowledgeable in the rare sleep space to help interpret these results and have submitted a briefing book to the FDA for an end of phase two meeting at the end of the third quarter.

Speaker Change: AP 1077 showed clinically meaningful benefits in change from baseline at the end of seven weeks of treatment against secondary and exploratory endpoints which included change in the Epworth sleepiness scale,

Speaker Change: the IH Severity Scale, the Sleep Inertia Visual Analog Scale, and a relatively new scale to assess the symptoms and severity of brain fog.

Neil McFarlane: We are encouraged by these results showing that KP1077 is well tolerated and demonstrates clinically meaningful benefits. Importantly, this study successfully fulfilled the objectives of informing the design of a pivotal efficacy trial. We consulted with key opinion leaders, payers, and patient advocates knowledgeable in the rare sleep space to help interpret these results, and we have submitted a briefing book to the FDA for an end of phase two meeting at the end of the third quarter.

Speaker Change: We are encouraged by these results showing that KP1077 is well tolerated and demonstrates clinically meaningful benefits. Importantly, the study successfully fulfilled the objectives of informing the design of a pivotal efficacy trial.

Speaker Change: We consulted with key opinion leaders, payers, and patient advocates knowledgeable in the rare sleep space to help interpret these results and have submitted a briefing book to the FDA for an end-of-phase-2 meeting at the end of the third quarter.

Neil McCarline: With only one FDA-approved treatment, there remains a large unmet need for therapies to address the symptoms of IH. We are conducting market research on the phase two data to better understand KP1077's differentiated profile, position in the treatment landscape, and to inform our business case.

Neil McFarlane: With only one FDA-approved treatment, there remains a large unmet need for therapies to address the symptoms of IH. We are conducting market research on the Phase 2 data to better understand KP 1077's differentiated profile, position in the treatment landscape, and to inform our business case. Finally, we've made progress with Celiprolol, our product candidate for the treatment of vascular ehlers-danlos syndrome, or VEDS, which impairs COL3A1 connective tissue and leads to vascular and hollow organ rupture. Solipolol's mechanism of action is designed to reduce the mechanical stress on collagen fibers within the arterial wall through vascular dilation and smooth muscle relaxation.

Speaker Change: With only one FDA-approved treatment, there remains a large unmet need for therapies to address the symptoms of IH.

Speaker Change: We are conducting market research on the Phase 2 data to better understand KP-1077's differentiated profile, position in the treatment landscape, and to inform our business case.

Neil McCarline: Finally, we've made progress with saliva law, our product candidate for the treatment of vascular islo syndrome, or VEDS, which impairs call 3A1 connective tissue and leads to vascular and hollow organ ruptures. saliva law's mechanism of action is designed to reduce the mechanical stress on collagen fibers within the arterial wall through vascular dilation and smooth muscle relaxation. saliva law is a primary treatment option in various EU countries, and we believe it could address the significant unmet need in the US, as there are no approved treatments for the 7500 patients with veds. Saliva law has received both orphan drug and breakthrough therapy designations from the FDA.

Speaker Change: Finally, we've made progress with Celeprolol, our product candidate for the treatment of Vascular Ehlers-Danlos Syndrome, or VEDS, which impairs COL3A1 connective tissue and leads to vascular and hollow organ ruptures.

Speaker Change: Solipolol's mechanism of action is designed to reduce the mechanical stress on collagen fibers within the arterial wall through vascular dilation and smooth muscle relaxation.

Neil McFarlane: Filiprolol is a primary treatment option in various EU countries, and we believe it could address the significant unmet need in the U.S. as there are no approved treatments for the 7,500 patients with VEDS. Liperol has received both orphan drug and breakthrough therapy designations from the FDA. During the second quarter, we restarted recruitment of the CILIPRA Law Phase 3 trial, also known as the DISCOVER trial. This decentralized, event-driven trial, We are encouraged by the significant interest among patients to enroll in this trial, which has exceeded our expectations, underscored the unmet need within the VEDS community and preserved the value of the program while we conduct our portfolio review. As part of the strategic planning initiative kicked off in January, we continue to assess the value of each of our programs.

Speaker Change: Filipilol is a primary treatment option in various EU countries, and we believe it could address the significant unmet need in the U.S. as there are no approved treatments for the 7,500 patients with VEDS.

Speaker Change: The liperol has received both orphan drug and breakthrough therapy designations from the FDA.

Neil McCarline: During the second quarter, we restarted recruitment of the saliva law phase three trial, also known as the Discover trial. This decentralized event-driven trial is being conducted under a special protocol assessment. We are encouraged by the significant interest among patients to enroll in this trial, which has exceeded our expectations, underscored the unmet need within the VEDS community, and preserves the value of the program while we conduct our portfolio review.

Speaker Change: During the second quarter, we restarted recruitment of the CILIPRA Law Phase 3 trial, also known as the DISCOVER trial. This decentralized, event-driven trial is being conducted under a special protocol assessment.

Speaker Change: We are encouraged by the significant interest among patients to enroll in this trial, which has exceeded our expectations, underscored the unmet need within the VEDS community, and preserves the value of the program while we conduct our portfolio review.

Neil McCarline: As part of the Strategic Planning Initiative kicked off in January, we continue to assess the value of each of our programs. Our intent is to fully understand the unmet needs of the rare disease patient community within a potential market and then develop a solid clinical and business case for how Zevra can develop therapies to address those needs. Looking ahead, we have three key priorities: first, to receive approval and successfully launch Aramoklamal by leveraging the infrastructure built for approval. Second, to drive the launchable approval. And third, to discuss design for a pivotal study evaluating the efficacy of KP1077 in patients with IH in our end of phase two meeting at the end of Q3.

Speaker Change: As part of the strategic planning initiative kicked off in January, we continue to assess the value of each of our programs.

Neil McFarlane: Our intent is to fully understand the unmet needs of the rare disease patient community within a potential market and then develop a solid clinical and business case for how Zevra can develop therapies to address those needs. Looking ahead, we have three key priorities. First, to receive approval and successfully launch Aramaklamal by leveraging the infrastructure built for Ol Prueba. Second, to drive the launchable Pruva.

Speaker Change: Our intent is to fully understand the unmet needs of the rare disease patient community within a potential market and then develop a solid clinical and business case for how ZEBRA can develop therapies to address those needs.

Speaker Change: Looking ahead, we have three key priorities. First, to receive approval and successfully launch Aramaklamal by leveraging the infrastructure built for Ol Prueba.

Neil McFarlane: And third, to discuss the design for a pivotal study evaluating the efficacy of KP1077 in patients with IH at our end-of-phase-II meeting at the end of Q3. We remain focused on execution to deliver a strong second half of 2024 and are well positioned financially to execute against those objectives. Now, LaDuane will provide an update on our financial results. Thank you, Neil, and good afternoon.

Speaker Change: Second, to drive the launchable Pruva.

Speaker Change: And third, to discuss design for a pivotal study evaluating the efficacy of KP1077 in patients with IH in our end-of-Phase II meeting at the end of Q3.

Neil McCarline: We remain focused on execution to deliver a strong second half of 2024, and our well-positioned financially to execute against those objectives.

Speaker Change: We remain focused on execution to deliver a strong second half of 2024 and are well positioned financially to execute against those objectives.

Lydwayne Clifton: Now, the Dwayne will provide an update on our financial results. Thank you, Neil, and good afternoon. As we begin, I encourage you to refer to our quarterly report on foreign TimQ, which we intend to file later today for more detailed information. We have made meaningful progress during the second quarter, and our financial results also reflect discipline in our capital allocation to drive towards success in reaching our strategic objectives. Our second quarter results included net revenue of $4.4 million, which includes $3.1 million in net reimbursements from the French EAP for Aramoklamal, and $1.3 million of royalties and other reimbursements under the ASTARIS license.

Speaker Change: Now, LeDuane will provide an update on our financial results.

LaDuane Clifton: As we begin, I encourage you to refer to our quarterly report on Form 10-Q, which we intend to file later today, for more detailed information. We have made meaningful progress during the second quarter, and our financial results also reflect discipline in our capital allocation to drive success in reaching our strategic objectives. Our second quarter results included net revenue of $4.4 million, which included $3.1 million in net reimbursements from the French EAP for Aramak Lamal, and $1.3 million in royalties and other reimbursements under the Astaris License.

LeDuane: Thank you, Neil, and good afternoon.

LeDuane: As we begin, I encourage you to refer to our quarterly report on Form 10-Q, which we intend to file later today, for more detailed information.

LeDuane: We have made meaningful progress during the second quarter and our financial results also reflect discipline in our capital allocation to drive towards success in reaching our strategic objectives.

LeDuane: Our second quarter results included net revenue of 4.4 million dollars.

LeDuane: which includes $3.1 million in net reimbursements from the French EAP for Aramaclamo and $1.3 million of royalties and other reimbursements under the Astaris license.

Lydwayne Clifton: For our commercial product approval, we recognize commercial product revenue when shipments are received by our specialty pharmacy. And as we previously announced, we transitioned to a new specialty pharmacy during the quarter, which required us to ship new product and recognize returns from the prior specialty pharmacy, which all set revenue for the period.

LaDuane Clifton: For our commercial product OPRUVA, we recognize commercial product revenue when shipments are received by our specialty pharmacies. And, as we previously announced, we transitioned to a new specialty pharmacy during the quarter, which required us to ship new product and recognize returns from the prior specialty pharmacy, which all set revenue for the period. The result was de minimis revenue recognized during Q2. We believe this transition will lead to improved patient services as enrollments grow. Additionally, the cost of goods sold was inflated during Q2 due to the recognition of a $3.2 million obsolescence reserve against Opruva Inventory, which is nearing expiration.

LeDuane: For our commercial product OPRUVA, we recognize commercial product revenue when shipments are received by our specialty pharmacy.

Speaker Change: And, as we previously announced, we transitioned to a new specialty pharmacy during the quarter, which required us to ship new product and recognize returns from the prior specialty pharmacy, which offset revenue for the period.

Lydwayne Clifton: The result was the minimum revenue recognized during Q2. We believe this transition will lead to improved patient services as enrollments grow. Additionally, cost of goods sold was inflated during Q2 due to recognition of a $3.2 million off-salescence reserve against OPPROVA inventory, which is nearing expiration. This excess inventory was ordered prior to our acquisition of ACER, and the previous delay in the product's launch impacted the rate of usage, leading to the need for this reserve to be recognized in the quarter. Our R&D expenses for the second quarter were $10.5 million, which is a slight decrease compared to the first quarter of 2024.

Speaker Change: The result was de minimis revenue recognized during Q2.

Speaker Change: We believe this transition will lead to improved patient services as enrollments grow.

Speaker Change: Additionally, cost of goods sold was inflated during Q2 due to recognition of a $3.2 million obsolescence reserve against Opruva Inventory, which is nearing expiration.

LaDuane Clifton: This excess inventory was ordered prior to our acquisition of ACER, and the previous delay in the product's launch impacted the rate of usage, leading to the need for this reserve to be recognized in the quarter. Our R&D expenses for the second quarter were $10.5 million, which is a slight decrease compared to the first quarter of 2024 and primarily due to the completion of the KP 1077 Phase II trial. Selling general and administrative expenses were $12.6 million during the second quarter, reflecting our commercial team being in place for the entire quarter and actively engaged in activities to build awareness and provide patient services related to Opruva. Net loss for Q2 2024 was $19.9 million, or $0.48 per basic and diluted share, which reflects an increase driven by our investments in our commercial infrastructure.

Unknown Executive: Hello, and thank you for joining the Zevra Therapeutics Q2 2024 Financial Results and Corporate Highlights Call. Today's call is being recorded and will be made available on the company's website following the conclusion of the call.

Speaker Change: This excess inventory was ordered prior to our acquisition of Acer, and the previous delay in the product's launch impacted the rate of usage, leading to the need for this reserve to be recognized in the quarter.

Speaker Change: Our R&D expenses for the second quarter were $10.5 million, which is a slight decrease compared to the first quarter of 2024 and primarily due to the completion of the KP 1077 Phase 2 trial.

Lydwayne Clifton: And primarily due to the completion of the KP 1077 Phase 2 trial. Selling, general and administrative expenses were $12.6 million during the second quarter, reflecting our commercial team being in place for the entire quarter and actively engaged in activities to build awareness and provide patient services related to OPPROVA. Net loss for Q2, 2024, was $19.9 million, or 48 cent per basic and diluted share, which reflects an increase driven by our investments in our commercial infrastructure. At the beginning of the quarter, we announced the refinancing of our existing debt with a new $100 million credit facility, from which we took an initial draw of $60 million.

Speaker Change: Selling General and Administrative Expenses

Speaker Change: were $12.6 million during second quarter.

Speaker Change: reflecting our commercial team being in place for the entire quarter.

Speaker Change: and actively engaged in activities to build awareness and provide patient services related to OPRUVA.

Nichol Ochsner: As we begin our call, it's important to highlight that certain information covered in today's discussions will include forward-looking statements. We caution listeners that actual results could differ materially from these stated or implied by these forward-looking statements due to risks and uncertainties associated with the company's business. Forward-looking statements are not promises or guarantees and are inherently subject to risks on certainties and other significant factors that may lead to actual results differing materially from those projections made.

Speaker Change: Net loss for Q2 2024 was $19.9 million, or $0.48 per basic and diluted share, which reflects an increase driven by our investments in our commercial infrastructure.

LaDuane Clifton: At the beginning of the quarter, we announced the refinancing of our existing debt with a new $100 million credit facility from which we took an initial draw of $60 million. A second tranche of up to $20 million is available at our discretion until October 5th, 2025, and a third tranche of up to $20 million will become available upon approval of Aramakamal, in each case subject to certain terms and conditions. As of June 30, 2024, total cash, cash equivalents, and investments were $49.3 million, which was a decrease of $3.4 million compared to March 31. Use of cash during the period was $17.4 million, offset by net proceeds of $14 million from our initial draw from our credit facility. The total long-term debt was $58.3 million as of June 30, 2024.

Speaker Change: At the beginning of the quarter, we announced the refinancing of our existing debt with a new $100 million credit facility from which we took an initial draw of $60 million.

Lydwayne Clifton: A second tranche of up to $20 million is available at our discretion until October 5, 2025, and a third tranche of up to $20 million will become available upon approval of Aero-Machimal, in each case, subject to certain terms and conditions. As of June 30, 2024, total cash, cash equivalents, and investments were $49.3 million, which was a decrease of $3.4 million compared to March 31st. Use of cash during the period was $17.4 million, offset by net proceeds of $14 million from our initial draw from our credit facility. Total long-term debt was $58.3 million as of June 30, 2024.

Speaker Change: A second tranche of up to $20 million is available at our discretion until October 5th, 2025.

Nichol Ochsner: These forward-looking statements are qualified by the cautionary statements contained in the risk factor section in our most recent quarterly report on form 10Q and our other filings with the SEC annual report on form 10K.

Speaker Change: and a third tranche of up to $20 million will become available upon approval of Aramakamol, in each case subject to certain terms and conditions.

Nichol Ochsner: I'm pleased to welcome Zevra's management team members participating in today's call.

Speaker Change: As of June 30, 2024, total cash, cash equivalents, and investments were $49.3 million, which was a decrease of $3.4 million compared to March 31.

Nichol Ochsner: I'm joined by Neil McCarline, President and Chief Executive Officer, Lydwayne Clifton, our Chief Financial Officer, Josh Schaeffer, our Chief Commercial Officer, and Executive Vice President of Business Development, and Adrian Quartel, our Chief Medical Officer.

Speaker Change: Use of cash during the period was $17.4 million, offset by net proceeds of $14 million from our initial draw from our credit facility.

Speaker Change: Total long-term debt was $58.3 million as of June 30th, 2024.

Lydwayne Clifton: As Neil mentioned earlier, we successfully completed a modestly sized underwritten public offering, which brought $64.5 million in net proceeds to Zebra, along with attracting a cadre of institutional investors well known in the biotech industry as long-term supporters of innovation and solid execution. We issued approximately $10.6 million shares at a price of $6.50 per share. This offering was significantly over-subscribed, which testifies to the strong sentiment around the potential of the several upcoming value creation opportunities for Zebra. Combining the net proceeds from this offering with our existing resources, pro forma June 30, 2024, cash, cash equivalents, and investments is $113.8 million.

LaDuane Clifton: As Neil mentioned earlier, we successfully completed a modestly-sized, underwritten public offering, which brought $64.5 million in net proceeds to Zevra, along with attracting a cadre of institutional investors well-known in the biotech industry as long-term supporters of innovation and solid execution. We issued approximately 10.6 million shares at a price of $6.50 per share. This offering was significantly oversubscribed, which testifies to the strong sentiment around the potential of the several upcoming value creation opportunities for Zevra.

Speaker Change: As Neil mentioned earlier, we successfully completed a modestly-sized, underwritten public offering.

Speaker Change: which brought

Neil McFarlane: $64.5 million in net proceeds to Zebra, along with attracting a cadre of institutional investors well known in the biotech industry as long-term supporters of innovation and solid execution.

Neil McCarline: These objectives are important building blocks for our long-term strategic plan to build a sustainable rare disease company with reliable cash flows. An important element of executing against our strategy is to build and maintain our position of financial strength. During the first half of 2024, we made investor outreach a primary objective, highlighting the opportunities and catalysts for value creation that we have as a company. There has been a significant interest in growing momentum as we continue to execute our objectives.

Neil McFarlane: We issued approximately 10.6 million shares at a price of $6.50 per share.

Neil McFarlane: This offering was significantly oversubscribed, which testifies to the strong sentiment around the potential of the several upcoming value creation opportunities for Zebra.

LaDuane Clifton: Combining the net proceeds from this offering with our existing resources, pro forma June 30, 2024, cash, cash equivalents, and investments is $113.8 million. Our Cache Runway now extends into Q1 2027. Pro forma common and fully diluted shares outstanding were $52.6 million and $67.9 million, respectively. No warrants were issued in connection with this offering.

Neil McCarline: Last week, following the favorable outcome of the FDA Advisory Committee meeting focused on Aramaquemol, we undertook a modestly-sized underwritten public offering to capture that momentum and build on our base of investors as we lean into the potential of our near-term catalysts. With this funding, we have added net proceeds of approximately $64.5 million to our balance sheet, bringing our Proforma June 30th, 2024 cash, cash equivalence and investments to $113.8 million. This was the right time to demonstrate our confidence and take this important step to prepare for success. The proceeds will extend our cash runway and bolster our flexibility in executing both near and long-term objectives, including full preparation for the potential launch of Aramokomal and the flexibility to accelerate our clinical pipeline.

Neil McFarlane: Combining the net proceeds from this offering with our existing resources, pro forma June 30, 2024, cash, cash equivalents, and investments is 113.8 million dollars.

Lydwayne Clifton: Our cash runway now extends into Q1, 2027. ProForma common and fully deluded shares outstanding were $52.6 million and $67.9 million respectively.

Neil McFarlane: Our CACHE runway now extends into Q1 2027.

Neil McFarlane: Pro Forma Common and Fully Diluted Shares Outstanding were $52.6 million and $67.9 million, respectively.

Lydwayne Clifton: No warrants were issued in connection with this offering. It is important to note that our cash runway guidance is based on our current operating plan, available cash, cash equivalents, and investments, including the additional cash received through our recently completed secondary offering, and our subject to continuing compliance with our debt covenants. Our forecast includes commercial revenue from the sales of ProForma, reimbursements from the French EAP for air moclemal, and ongoing royalties under the Astaurus license agreement. It does not include commercial revenue from sales of air moclemal, nor the sale of the PRV, which would follow FDA approval.

Neil McFarlane: No warrants were issued in connection with this offering.

LaDuane Clifton: It is important to note that our cash runway guidance is based on our current operating plan, available cash, cash equivalents, and investments, including the additional cash received through our recently completed secondary offering, and is subject to continuing compliance with our debt covenants. Our forecast includes commercial revenue from the sales of Apruva. Reimbursements from the French EAP for Aramakamal and ongoing royalties under the Astoris License Agreement. It does not include commercial revenue from sales of Aramoclomol or the sale of the PRV, which would follow FDA approval.

Neil McFarlane: It is important to note that our cash runway guidance is based on our current operating plan, available cash, cash equivalents, and investments, including the additional cash received through our recently completed secondary offering, and are subject to continuing compliance with our debt covenants.

Neil McFarlane: Our forecast includes commercial revenue from the sales of Okruva, reimbursements from the French EAP for Aramakamal, and ongoing royalties under the Astoris License Agreement.

Neil McFarlane: It does not include commercial revenue from sales of Eremoclymal, nor the sale of the PRV which would follow FDA approval.

Lydwayne Clifton: We are optimistic about the outlook, and our focus is to create long-term value for shareholders through disciplined execution against our plan in support of our mission to become a leading rare disease company.

Operator: We are optimistic about the outlook, and our focus is to create long-term value for shareholders through disciplined execution against our plan in support of our mission to become a leading rare disease company. Now, we will turn the call over to the operator for questions. Thank you. At this time, if you would like to ask a question, please press star and one on your telephone. You may remove yourself from the cube by pressing stars and 2.

Neil McFarlane: We are optimistic about the outlook and our focus is to create long-term value for shareholders through disciplined execution against our plan in support of our mission to become a leading rare disease company.

Unknown Executive: Tony. Now, we will turn the call over to the operator for questions. Thank you. At this time, if you would like to ask a question, please press star and one on your telephone keypad. You may remove yourself from the cube by pressing star and two. Again, it is star and one to ask a question today. We'll take our first question from Louise Chen with Cantor. Please go ahead. Your line is open. Hi, congratulations on all the progress is quoted. Thanks for taking my questions. So I had a few for you. First question was how you think about all proof of sales in the second half of 24.

Speaker Change: Now, we will turn the call over to the operator for questions.

Speaker Change: Thank you. At this time, if you would like to ask a question, please press star and 1 on your telephone keypad. You may remove yourself from the queue by pressing star and 2.

Neil McCarline: Our Padoopa date is September 21st, which is fast approaching. We received the first round of labeling comments last Friday and are working closely with the FDA. If approved, Aramokomal would be the first drug in the US indicated for the treatment of NPC, and it would be eligible for a priority review voucher. As a reminder, there are roughly 900 people in the US with NPC, of which approximately one-third are diagnosed and treated.

Operator: Again, it is star and one to ask a question. We'll take our first question from Louise Chen. Go ahead, your line: Hi, congratulations on all the progress this quarter and thanks for taking my questions. So I had a few for you.

Louise Chen: The first question was how you think about all proof of sales in the second half of 24. And then, do you have any thoughts on initial thoughts on pricing for our remote mall if it gets approved? And the last question is just about our remote mall, patent protection, and marketing exclusivity. Thanks, Louise.

Speaker Change: Again, it is star and one to ask a question today.

Speaker Change: We'll take our first question from Luis Chen with Kantor. Please go ahead, your line is open.

Luis Chen: Hi, congratulations on all the progress this quarter and thanks for taking my questions.

Luis Chen: So I had a few for you. First question was how you think about OPRUVA sales in the second half of 2024. And then do you have any initial thoughts on pricing for our remote comal if it gets approved? And the last question is just on our remote comal, the patent protection and marketing exclusivity. Thank you.

Louise Chen: And then are you do you have any thoughts on initials? I'm pricing for our removal mall if it gets approved. And the last question is just in our removal mall, the patents protection and marketing exclusivity. Thank you. Thanks, Louise.

Neil McCarline: More than 70 of those patients are currently enrolled in our US Expanded Access Program, or EAP. As the FDA review continues, we will maintain the US EAP to ensure access for patients until commercial supply is available. In addition, subsequent to US approval, we will seek regulatory approval in Europe, where an additional 70-80 patients are enrolled in our global EAP program. We are actively preparing for the commercial launch of Aramokomal. Our commercial infrastructure was built to optimize the strategic fit between O'Proofa and Aramokomal.

Neil McFarlane: Why don't I start with the last question, and I'll tie up the pricing, and then I'll hand it off to Josh to talk a little bit about Aramathamol and Olprova. In regards to patent protection and marketing exclusivity for Aramathamol, we rely on orphan drug exclusivity for up to seven years. And then, in regards to pricing for Aramathamol, it's a little early for us to be talking about pricing, as we're just now in the labeling discussions.

Neil McCarline: Why don't I start with the last question, and I'll tee up the pricing, and then I'll hand it off to Josh to talk a little bit about our model mall and as well as old proof of. In regards to the patent protection and marketing exclusivity for our model mall, we rely on orphan drug exclusivity for up to seven years. And then in regards to pricing for our model, so we'll early for us to be talking about pricing as we're just now in the labeling discussions. But I think it's I want to make it clear to everybody that it is our goal to make aromachamol as widely available as possible as we can.

Speaker Change: Thanks, Luis. Why don't I start with the last question, and I'll tee up the pricing, and then I'll hand it off to Josh to talk a little bit about Aramathamol and as well as Olprova. In regards to the patent protection and marketing exclusivity for Aramathamol, we rely on orphan drug exclusivity for up to seven years.

Speaker Change: And then in regards to the pricing for Aromathamol, it's a little early for us to be talking about pricing as we're just now in the labeling discussions, but I think it's, I want to make it clear to everybody that it is our goal to make

Neil McCarline: Both products address genetic metabolic diseases, with multi-disciplinary treatment teams that are often co-located within the same centers of excellence, allowing us to reach the majority of prescribers with a targeted commercial infrastructure. Our rare disease specialists and medical science liaisons, who are currently promoting and educating on O'Proofa and urea cycle disorders, or UCDs, are also engaging with prescribers when appropriate to raise awareness for NPC. Further, our market access team has initiated pre-approval clinical discussions with payers regarding Aramokomal as a potential treatment for NPC. These pre-launch activities will help ensure patients have access once available.

Neil McFarlane: But I think it's I want to make it clear to everybody that it is our goal to make Aramathamol as widely available as possible as we can. And with that, I'll hand it off to Josh to talk a little bit about what we've been doing around understanding the market for Aramathamol, around what we could do for pricing, as well as Olprova sales in the second half of 2024.

Josh Schafer: And with that, I'll hand it up to Josh to talk a little bit about what we've been doing around understanding the market for aromachamol, around what we could do for pricing, as well as old proof of sales in the second half of 2024. That was regards to all the aromachamol pricing. We have conducted pretty extensive market research with payers, and we've gone out and we've tested the clinical profile and really pressured them around responding to the value proposition and the clinical value of aromachamol for patients with NPC. I have to say that it's widely been viewed as what could potentially be foundational therapy if approved by payers, but of course the final label will ultimately influence the final price and how payers view that.

Josh Schaefer: Aramakamal as widely available as possible as we can. And with that, I'll hand it off to Josh to talk a little bit about what we've been doing around understanding the market for Aramakamal, around what we can do for pricing as well as old proof of sales in the second half of 2024. Josh?

Joshua Schafer: Yeah, with regard to Aromaclomol pricing, we have conducted pretty extensive market research with payers. And we've gone out, and we've tested the clinical profile, and really pressured them around, you know, responding to the value proposition and the clinical value of Aromaclomol for patients with NPD. I have to say that it's widely been viewed as potentially being foundational therapy if approved by payers. But, of course, the final label will ultimately influence the final price and how payers view that.

Josh Schaefer: With regards to Aramakumo pricing

Josh Schaefer: We have conducted pretty extensive market research with payers.

Josh Schaefer: and we've gone out and we've tested the clinical profile and really pressured them around responding to the value proposition and the clinical value of Aramaclomol for patients with NPC.

Speaker Change: I have to say that it's widely been viewed as what could potentially be foundational therapy if approved by payers But of course the final label will ultimately influence the the final price and how payers view that

Neil McCarline: Now, turning to O'Proofa, where we continue to make process with the commercial launch initiated at the end of January 2024. Over the last few quarters, our assumption on the limited awareness of O'Proofa has been confirmed. However, we've made progress with healthcare providers to increase awareness levels, and identified that we have more work to do to increase patient awareness. As a reminder, there are 1100 UCD patients in the US, of which more than 800 are receiving treatment.

Josh Schafer: So we're working very closely with them; we're putting in place patient services and other resources to make sure that aromachamol will be as widely available to patients as possible.

Joshua Schafer: We're working very closely with them and putting in place patient services and other resources to make sure that Aramalkamo will be as widely available to patients as possible. And then with regard to Alproova in the second half, and I'd like to just remind everyone that the first half was really built around, or it was really focused on building awareness where there was very little awareness within the prescribing community about Alproova. We're very happy with the progress the team made in terms of getting in front of prescribers and payers and really building that awareness across that prescribing community.

Speaker Change: We're working very closely with them and we're putting in place patient services and other resources to make sure that Aromalchemal will be as widely available to patients as possible.

Josh Schafer: And then, with regards to all proofa in the second half. And I'd like to just remind everyone that the first half was really built or was really focused on building awareness, where there was very little awareness within the prescribing community for all proofa. We're very happy with the progress that the team made in terms of getting in front of prescribers and payers and really building that awareness across that prescribing community, and we have more than 75% covered lives for patients with UCD. We've also put in place some other resources and tactics, including a free trial program and a demonstration program, to be able to allow the physicians and prescribers to see how Proofa works.

Speaker Change: And then with regards to Alpruva in the second half, I'd like to just remind everyone that the first half was really built

Speaker Change: It was really focused on building awareness, where there was very little awareness within the prescribing community for Alproova.

Neil McCarline: The prescribing community has identified a significant number of these patients who can benefit from all proof up. While we are encouraged by their response, the number of patient enrollments is not yet where we would like it to be. During the second quarter, we had nine new patient enrollments, which we define as a prescription for a patient who's on our Quick Start program or one who's receiving a paid dispense. We have been working to build awareness amongst clinicians who treat UCD and to ensure market access for patients.

Speaker Change: We're very happy with the progress the team made in terms of getting in front of prescribers and payers and really building that awareness across that prescribing community and we have more than 75% covered lives for for patients with with UCD.

Joshua Schafer: And we have more than 75% of covered lives for patients with UCD. We've also put in place some other resources and tactics, including a free trial program and a demonstration program to be able to allow physicians and prescribers to see how Ulpruga works. And we've made a change to our specialty pharmacy. All of this, I think, bodes well for the second half.

Speaker Change: We've also put in place some other resources and tactics including a free trial program and a demonstration program to be able to allow the physicians and prescribers to see how Olpruga works and we've made a change to our specialty pharmacy.

Josh Schafer: And we've made a change to our specialty pharmacy. All of this I think bodes well for the second half, and as we really begin to focus on building more awareness within the patient community, we're looking forward to seeing increased informants.

Neil McCarline: The team has done an outstanding job of engaging HCPs at target centers of excellence and at medical conferences to build brand awareness. The team has successfully engaged the majority of clinicians and thought leaders who diagnose and treat UCD patients, which is remarkable since access to HCPs has become more challenging across the industry. Additionally, our managed care team continues to engage with government and commercial payers to ensure broad access for patients. We have increased all proof of coverage to 75% of covered lives with improved formulary status across healthcare plans and have established comprehensive patient services programs designed to assist with the reimbursement hurdles experienced by the rare disease community.

Joshua Schafer: And as we really begin to focus on building more awareness within the patient community, we're looking forward to seeing increased enrollments. Thank you. We'll take our next question from Tim Lugo with William Blair. Thanks for the question and congratulations on all the progress. It's obviously been a transitional quarter.

Speaker Change: All of this, I think, bodes well for the second half, and as we really begin to focus on building more awareness within the patient community, we're looking forward to seeing increased enrollments.

Tim Lugo: We'll take our next question from Tim Lugo with William Blair. Please go ahead. Your line is open. Thanks for the question, and congratulations on all the progress. It's been a obviously transitional quarter. I just asked her the outcome. It seems like, you know, combination use with Nicholas that is going to be maybe occurring a little bit more than I maybe had expected.

Speaker Change: Thank you.

Speaker Change: We'll take our next question from Tim Lugo with William Blair. Please go ahead, your line is open.

Tim Lugo: Thanks for the question and congratulations on all the progress. It's been a, obviously, transitional quarter.

Timothy Lugo: After the adcom, it seems like, you know, combination use with MIGLESTAT is going to be maybe occurring a little bit more than I maybe had expected. I'd just love to hear your thoughts around combination use in the real world, once approved, and how does that fit into your pricing thoughts? and then maybe also some initial.

Speaker Change: After the adcom,

Tim Lugo: It seems like, you know, combination use with Miglistat is...

Speaker Change: going to be maybe occurring a little bit more than I maybe had expected. I'd just love to hear your thoughts around combination use in the real world once approved, and how does that fit into your pricing thoughts.

Tim Lugo: I just love to hear your thoughts around combination use in the real world once approved and how does that fit into your pricing thoughts. And then maybe also some initial initial kind of feedback you've heard from the patient advocacy groups post that come. Thanks. Thanks, Tim.

Neil McCarline: As I mentioned earlier, there are always opportunities for a finement during lunch and we are implementing changes to improve patient engagement. One key change was our transition to Orcini as our specialty pharmacy partner who is a leader in pharmacy solutions for rare diseases. This transition completed in mid-June, including a rebalancing of our approved inventory in the channel, which impacted our net sales revenue in the quarter.

unknown: You know, the initial kind of feedback you've heard from the patient advocacy groups post-ADCOM. Thanks. Thanks, Tim.

Speaker Change: And then maybe also some initial kind of feedback you've heard from the patient advocacy groups post-adcom.

Neil McFarlane: I think I'll handle your first question, and then I might actually ask Josh to talk a little bit about the advocacy engagement or Adrian. I think one of the things that's really important is you get what you study when it comes to the labeling discussion.

Neil McCarline: I think let me handle your first question. And then I might actually ask Josh to talk a little bit about the advocacy engagement and Adrian. I think one of the things that's really important is you get what you study when it comes to labeling discussion. And we've studied aromachemal versus placebo with underlying routine care. And those patients could have been stratified to Bigelstad or some other kind of primary treatment that the patient had, but routine care, I think, was standard. And our discussions and in the adcom, and I think now as we're driving into our labeling discussions, you usually get what you study.

Speaker Change: Thanks, thanks Tim. I think let me handle your first question and then I might actually ask Josh to talk a little bit about the advocacy engagement and or Adrian.

Neil McCarline: The Dwayne will provide more details later in the call. We believe these enhancements to our commercial infrastructure will further support the approval launch and will have a positive impact on our commercialization efforts for our mock-em-all with limited incremental costs.

Speaker Change: I think one of the things that's really important is you get what you study.

Neil McFarlane: And we've studied arimoclomol versus placebo with underlying routine care, and those patients could have been stratified to the big list or some other kind of primary treatment that the patient received. But routine care, I think, was standard in our discussions and in the adcom. We've been, and I think now as we're driving into our labeling discussions, you usually get what you studied. And for us, that's arimoclomol versus placebo. That's the outcome that we're looking for. So I can't talk more about combination therapy at this point in the game because we're in the process of round one. But I do think that there's precedent for such a study. You get what you study.

Speaker Change: when it comes to labeling discussion. And we've studied arimoclomol versus placebo with underlying routine care.

Speaker Change: And those patients could have been stratified to Bigelstat or some other kind of primary treatment that the patient had, but routine care, I think, was standard. In our discussions and in the Adcom, and I think now as we're driving into our labeling discussions, you usually get what you studied. And for us, that's Arimothamol versus placebo. That's the outcome that we're looking for.

Neil McCarline: Now I would like to turn your attention to KP1077, our clinical candidate for the treatment of idiopathic hypersomnia or IH, a rare chronic sleep disorder. IH is characterized by excessive daytime sleepiness and difficulty waking, also known as sleep inertia. This disease impacts approximately 37,000 people in the U.S. As you may recall, KP1077 is comprised of Sirnex Meppelfenedate or SDX, which was designed to steadily release the Meppelfenedate its active ingredient. Its unique pharmacokinetic profile allows for flexible dosing to overcome these primary IH symptoms and ensures patients receive the optimal drug concentration during waking hours.

Neil McCarline: And for us, that's aromachemal versus placebo. That's the outcome that we're looking for.

Neil McCarline: So I can't talk more about combination therapy at this point in the game because we're in the process of round one. But I do think that there's precedent around study; you get what you study.

Speaker Change: I can't talk more about combination therapy at this point in the game because we're in the process of round one But I do think that there's precedent around study. You get what you study

Josh Schafer: In regards to the initial feedback from the patient advocacy organizations, let me ask Josh to talk a little bit about where we're seeing some excitement from the advocacy organizations today. Yeah, I think if anyone tuned into the advisory committee meeting, 10 days or so ago, you would have seen overwhelming support for aromachemal from prescribers, caregivers, and patients. And that really is a reflection of the benefits that many patients have received from aromachemal through our expanded access program and open label program. And as we move forward, we expect that support to really continue. I can also say that as we've gone out and we've talked with payers and physicians and done some market research and tested the profile, many of them really see Aromachemal as the first approved drug for Nimen Tick really is the foundation for therapy for these patients.

Joshua Schafer: Regarding the initial feedback from the patient advocacy organizations, let me ask Josh to talk a little bit about where we're seeing some excitement from the advocacy organizations today. Yeah, I think, if anyone tuned in to the advisory committee meeting. 10 days or so ago, you would have seen overwhelming support for Aromacomal from prescribers, caregivers, and patients. And that really is a reflection of the benefits that many patients have received from Aromacomal through our Expanded Access Program and Open Label Program.

Speaker Change: In regards to the initial feedback from the patient advocacy organizations, let me ask Josh to talk a little bit about where we're seeing some excitement from the advocacy organizations today. Yeah, I think...

Josh Schaefer: If anyone tuned in to the advisory committee meeting...

Josh Schaefer: 10 days or

Josh Schaefer: or so ago, you would have seen overwhelming support for Aromacomalt from prescribers, caregivers, and patients. And that really is a reflection of the benefits that many patients have received from Aromacomalt through our Expanded Access Program and Open Label Program.

Neil McCarline: SDX is currently designated as a schedule for control substance by the U.S. Drug Enforcement Administration due to demonstrated lower risk or abuse potential. At the sleep 2024 meeting in June, we presented the pharmacokinetics of SDX, when administered in the morning and at night. The clinical data showed peak exposure occurs the morning after a nighttime dose, when the patient needs it most to manage sleep inertia. We also reported positive results from our Phase II clinical study of KP1077 in patients with IH.

Joshua Schafer: And as we move forward, we expect that support to really continue. I can also say that as we've gone out and talked with payers and physicians and done some market research and tested the profile, many of them really see Arimoclomol as the first approved drug for Niemann-Pick, really as the foundation for therapy for these patients.

Josh Schaefer: And as we move forward, we expect that support to really continue.

Josh Schaefer: I can also say that as we've gone out and we've talked with payers and physicians and done some market research and tested the profile, many of them really see Arimoclomol as the first approved drug for Niemann-Tick really as the foundation for therapy for these patients.

Josh Schaefer: [inaudible]

Neil McCarline: In this proof of concept study, KP1077 was well tolerated at all dose levels, including the notably high dose of 320 milligrams daily. Adverse events throughout the study were mild, similar to other methylphenidate products and did not lead to early discontinuation. KP1077 showed clinically meaningful benefits in change from baseline at the end of seven weeks of treatment, again secondary and exploratory endpoints, which included change in the upward sleepiness scale, the IH severity scale, the sleep inertia, visual analog scale, and a relatively new scale to assess the symptoms and severity of brain fog.

Unknown Executive: Operator?

Oren Livnat: Yeah, as a reminder, if you'd like to ask a question today, please press the star and one keys on your telephone keypad. We'll take our next question from Orrin Livnet with HC Wing. Right, please go ahead. Your line is open. Thank you for taking the questions.

Oren Livnat: As a reminder, if you'd like to ask a question today, Starr, and our next question from Oren Livnat with HC Wing, right, please go ahead. You. Thank you for taking the questions. Let me add my congratulations on the outcome of the adcom. Just so I'm clear, you mentioned you've entered the first round of labeling comments in our discussions with the FDA. Can you say if you've actually had any explicit additional information requests or anything new you've had to provide in this process? Thanks, Oren.

Josh Schaefer: Operator.

Speaker Change: As a reminder, if you'd like to ask a question today, please press the star and one keys on your telephone keypad.

Speaker Change: We'll take our next question from Oren Livnet with HC Wainwright.

Speaker Change: Please go ahead, your line is open.

Oren Livnat: Let me add my congrats to the outcome of the adcom. Just so I'm clear, you mentioned you've entered the first round of labeling comments with the discussions with the FDA. Can you say if you've actually had any explicit additional information or requests or anything new you've had to provide in this process? Thanks, Oren. So the answer to your question is we have gotten our first round of labeling negotiations. I think your second question was a regard into any specific information requests. We have received additional information requests. As you can imagine, throughout this process, you continue to get them in regards to new data to be provided.

Oren Livnet: Thank you for taking the questions. Let me add my congrats to the outcome of the adcom.

Oren Livnet: Just so I'm clear, you mentioned you've entered the first round of labeling comments or discussions with the FDA. Can you say if you've actually had any explicit additional information requests or anything new you've had to provide in this process?

Neil McFarlane: The answer to your question is that we have got our first round of labeling negotiations. I think your second question was regarding any specific information requests. We have received additional information requests. As you can imagine, throughout this process, you will continue to receive them. In regards to new data to be provided, we have not had any requests to add new data, more so than what has been shared specifically with the advisory committee.

Oren Livnet: Thanks, Oren. So...

Neil McCarline: We are encouraged by these results showing that KP1077 is well tolerated and demonstrates clinically meaningful benefits. Importantly, the study successfully fulfilled the objectives of informing the design of a PIVO efficacy trial. We consulted with key opinion leaders, payers, and patient advocates knowledgeable in the rare sleep space to help interpret these results and have submitted a briefing book to the FDA for an end of phase two meeting at the end of the third quarter.

Speaker Change: The answer to your question is

Speaker Change: We have gotten our first round of labeling negotiations.

Speaker Change: I think your second question was a regard into any specific information requests.

Speaker Change: We have received additional information requests, as you can imagine, throughout this process, you continue to get them. In regards to new data to be provided, we have not had any requests to add new data, more so than what has been shared specifically with the advisory committee.

Neil McCarline: We have not been at had any request to add new data more so than what has been shared specifically with the Advisory Committee. All right, sorry with the FDA, not the Advisory Committee with the FDA.

Neil McFarlane: All right. Sorry, with the FDA, not the Advisory Committee, with the FDA. Okay, perfect. Assuming you're approved in late September, do you have any expectations for timing for product availability, given the significant overlap with Olprova? I assume that would be relatively quickly.

Speaker Change: All right. Sorry, with the FDA, not the Advisory Committee, with the FDA. Okay, perfect.

Oren Livnat: Okay, perfect.

Neil McCarline: With only one FDA approved treatment, there remains a large unmet need for therapies to address the symptoms of IH. We are conducting market research on the phase two data to better understand KP1077's differentiated profile, position in the treatment landscape, and to inform our business case.

Neil McCarline: Assuming or approved in late September, do you have any expectations for timing, for product availability given the significant overlap with Olpruva? I assume that would be relatively quickly, but I'm curious if you need to set up any different reimbursement support infrastructure. This is a new indication, unlike UCD. And in the NPC community and centers of excellence, I guess I'm already asked about the feedback post-AdComp. I'm just curious if there's any, are you getting the sense that there's any particular preparation or expectations there post-Adcomp? Are you hearing about warehousing of patients or any proactive outreach maybe to patients and families in anticipation of potential approval?

Oren Livnat: But I'm curious if you need to set up any different reimbursement support infrastructure, given this is a new indication, unlike UCD. And, in the NPC community and centers of excellence, you know, I guess I'm going to ask about the feedback post-adcom, but I'm just curious if there's any, you know, are you getting the sense that there's any particular preparation or expectations there post-adcoms? You know, are you hearing about warehousing of patients or any proactive outreach to patients and families in anticipation of potential approval?

Speaker Change: Assuming you're approved in late September, do you have any expectations for timing for product availability given the

Speaker Change: Significant overlap with Olpruva. I assume that would be relatively quickly. But I'm curious if you need to set up any different reimbursement support infrastructure, given this is a new indication, unlike UCD.

Neil McCarline: Finally, we've made progress with saliva law, our product candidate for the treatment of vascular, isler, danlos syndrome, or veds, which impairs call 3A1 connective tissue and leads to vascular and hollow organ ruptures, saliva law's mechanism of action is designed to reduce the mechanical stress on collagen fibers within the arterial wall through vascular dilation and smooth muscle relaxation, saliva law is a primary treatment option in various EU countries and we believe it could address the significant unmet need in the US as there are no approved treatments for the 7500 patients with veds, saliva law has received both orphan drug and breakthrough therapy designations from the FDA. During the second quarter, we restarted recruitment of the known as the discover trial.

Speaker Change: in the NPC community and centers of excellence.

Speaker Change: You know, I guess I'm already asked about the feedback post-ad, Tom, but I'm just curious if there's any...

Speaker Change: You know, are you getting the sense that there's any particular preparation or expectations there post-adcoms? You know, are you hearing about warehousing of patients or any proactive outreach maybe to patients and families in anticipation of potential approval?

Neil McCarline: Let me start by talking a little bit about the potential for our producer at the end of September. We are on track in order to be able to have product in the channel within the customer time frame of, you know, let's call it eight to twelve weeks. Maybe even smaller than eight to 10 weeks post-launch, which is fairly standard.

Oren Livnat: Let me start by talking a little bit about the potential for our PDUFA at the end of September. We are on track in order to be able to have product in the channel within the customary timeframe of, you know, let's call it 8 to 12 weeks, maybe even smaller than that, 8 to 10 weeks post-launch, which is fairly standard. Let me turn it over to Josh to talk a little bit about new infrastructure and or the needs of the NPC community post the advisory committee. Yeah, hey, Oren.

Speaker Change: Let me start by talking a little bit about the potential for our PDUFA at the end of September. We are on track in order to be able to have product in the channel within the customary time frame of, you know, let's call it 8 to 12 weeks.

Josh Schaefer: maybe even smaller than that, eight to ten weeks post-launch, which is fairly standard. Let me turn it over to Josh to talk a little bit about new infrastructure and or the needs in the NPC community post the advisory committee. Yeah, hey Oren.

Josh Schafer: Let me turn it over to Josh to talk a little bit about new infrastructure and or the needs in the NPC community post the advisory committee. As a reminder, we built the commercial team to really focus not just on all proof of, but in anticipation of an approval and launch of air mock them all. So it is really the right size to be able to optimize both products. And we also have the benefit of having a team out there now talking to prescribers about UCD. Oftentimes, these are the same physicians who are seeing NIM and TIC patients.

Neil McCarline: This decentralized event driven trial is being conducted under a special protocol assessment. We are encouraged by the significant interest among patients to enroll in the trial, which has exceeded our expectations, underscored the unmet need within the veds community, and preserves the value of the program while we conduct our portfolio.

Joshua Schafer: As a reminder, we built the commercial team to really focus not just on Alproova but in anticipation of an approval and launch of Aramaka. So it is really right-sized to be able to optimize both products. And we also have the benefit of having a team out there now talking to prescribers about UCD. Oftentimes, these are the same physicians who are seeing Niemann-Pick patients. And so our team is out there profiling some of these physicians and understanding where the patients are.

Josh Schaefer: As a reminder, we built the commercial team to really focus not just on Alproova, but in anticipation of an approval and launch of Aramak Lamal.

Josh Schaefer: So it is really right size to be able to optimize both products.

Neil McCarline: As part of the strategic planning initiative kicked off in January, we continue to assess the value of each of our programs. Our intent is to fully understand the unmet needs of the rare disease patient community within a potential market and then develop a solid clinical and business case for how Zevra can develop therapies to address those needs. Specifically, launch Aramoklamal by leveraging the infrastructure built for approval. Second, to drive the launch of approval. And third, to discuss design for a pivotal study evaluating the efficacy of KP 1077 in patients with IH in our end of phase two meeting at the end of Q3.

Josh Schaefer: And we also have the benefit of having a team out there now talking to prescribers.

Speaker Change: about UCD.

Speaker Change: Oftentimes, these are the same physicians who are seeing Niemann-Tick patients.

Josh Schafer: And so our team is out there profiling some of these physicians, understanding where the patients are. Our team on the market access side is having conversations with pairs on the clinical differentiation of air mock them all and the need to treat. So the team is in place. We feel quite confident that it's the right size for both products. And we will be well positioned to begin commercialization as soon as drug is in the channel.

Speaker Change: And so our team is out there profiling some of these physicians, understanding where the patients are. Our team on the market access side is having conversations with payers on the clinical differentiation of Arimacumol and the need to treat.

Joshua Schafer: Our team on the market access side is having conversations with payers on the clinical differentiation of Arimocamol and the need to treat. So the team is in place, and we feel quite confident that it's right-sized for both products.

Joshua Schafer: And we will be well-positioned to begin commercialization as soon as the drug is in the channel. In terms of, you know, where these patients are, we continue to work very closely with the investigators in our Expanded Access Program. As a reminder, we've got more than 70 patients here in the U.S. who are in that program, as well as other patients who are being seen by those same investigators who are eagerly awaiting the approval of Aramont.

Speaker Change: So, the team is in place. We feel quite confident that it's right size for both products. And we will be well positioned to begin commercialization as soon as the drug is in the channel.

Josh Schafer: In terms of, you know, where these patients are, we continue to work very closely with the investigators in our expanded access program. As a reminder, we've got more than 70 patients here in the US who are in that program, as well as other patients who are being seen by those same investigators who are eagerly awaiting the approval of our model.

Speaker Change: In terms of

Speaker Change: where these patients are. We continue to work very closely with the investigators.

Neil McCarline: We remain focused on execution to deliver a strong second half of 2024 and our well-positioned financially to execute against those objectives.

Speaker Change: in our Expanded Access Program. As a reminder, we've got more than 70 patients here in the U.S. who are in that program.

Lydwayne Clifton: Now, the Dwayne will provide an update on our financial results. Thank you, Neil and good afternoon.

Speaker Change: as well as other patients who are being seen by those same investigators who are eagerly awaiting the approval of Arimofim.

Josh Schafer: And I guess this lastly segwayed into the EAP patients. Is there any preparation you can do this separate or in addition to standard market pre-commercial activities with regard to this EAP population in particular since you know the patients and have relationships with them directly to some extent. Is there anything you can do to expedite their transition to a commercial product when this product is available as opposed to maybe what the process would be with a new patient. So, so thanks on a couple of things. One is, I think number one is, we should get on the table here is that we're going to continue to support these patients in the EAP program until we've got commercial supply and access for patients to be able to transition.

Joshua Schafer: And I guess just lastly, to segue into the EAP patients, is there any preparation you can do separate or in addition to standard market pre-commercial activities with regard to this EAP population in particular, since you know the patients and have relationships with them directly to some extent? Is there anything you can do to expedite their transition to commercial products?

Lydwayne Clifton: As we begin, I encourage you to refer to our quarterly report on foreign TimQ, which we intend to file later today for more detailed information. We have made meaningful progress during the second quarter, and our financial results also reflect discipline in our capital allocation to drive towards success in reaching our strategic objectives. Our second quarter results included net revenue of $4.4 million, which includes $3.1 million in net reimbursements from the French EAP for Aramoklamal, and $1.3 million of royalties and other reimbursements under the ASTARIS license.

Speaker Change: And I guess just lastly, you segued into the EAP patients. Is there any preparation you can do separate?

Speaker Change: or in addition to standard market pre-commercial activities.

Speaker Change: with regard to this EAP population in particular, since you know the patients and have relationships with them directly to some extent, is there anything you can do to expedite their transition to commercial product?

Neil McFarlane: when this product is available as opposed to maybe what the process would be with a new patient. So, Oren, a couple of things.

Speaker Change: when this product is available as opposed to maybe what the process would be with a new patient.

Neil McFarlane: One is, I think number one is, we should get on the table here, is that we're going to continue to support these patients in the EAP program until we've got commercial supply and access for patients to be able to transition. Answering your second part of the question, and we are working directly with investigators, not necessarily with patients directly, because, as you recall, the EAP program is still collecting data, and we don't have direct hands-on with patients.

Lydwayne Clifton: For our commercial product or proof up, we recognize commercial product revenue when shipments are received by our specialty pharmacy. And as we previously announced, we transitioned to a new specialty pharmacy during the quarter, which required us to ship new product and recognize returns from the prior specialty pharmacy, which all set revenue for the period. The result was the minimum revenue recognized during Q2. We believe this transition will lead to improved patient services as enrollments grow.

Speaker Change: So, thanks Orin. A couple of things. One is, I think number one is, we should get on the table here, is that we're going to continue to support these patients in the EAP program until we've got commercial supply and access for patients to be able to transition.

Josh Schafer: Answering your second part of the question, and we are working directly with investigators, not necessarily with patients directly, because recall the EAP program is still collecting data and we don't have direct hands-on with patients. The next thing you asked about is in regards to can you set up your EAP program to allow for more seamless transition so that I think I want to make it clear it's our goal to be able to transition our EAP patients to commercial supply within the first year post launch, and that will allow us to be able to move on to other other areas that we can collect data and real world data and other things that can help patients move forward.

Speaker Change: Answering your second part of the question, and we are working directly with investigators, not necessarily with patients directly, because recall the EAP program is still collecting data and we don't have direct hands-on with patients.

Neil McFarlane: The next thing you asked me about is, can you set up your EAP program to allow for a more seamless transition? Because I think I want to make it clear, it's our goal to be able to transition our EAP patients to commercial supply within the first year post-launch, and that will allow us to be able to move on to other areas where we can collect data and real-world data and other things that can help patients move forward.

Speaker Change: The next thing you asked about is in regards to can you set up

Lydwayne Clifton: Additionally, cost of goods sold was inflated during Q2 due to recognition of a $3.2 million off-salescence reserve against OPPROVA inventory, which is nearing expiration. This excess inventory was ordered prior to our acquisition of ACER, and the previous delay in the product's launch impacted the rate of usage, leading to the need for this reserve to be recognized in the quarter. Our R&D expenses for the second quarter were $10.5 million, which is a slight decrease compared to the first quarter of 2024, and primarily due to the completion of the KP 1077 Phase 2 trial.

Speaker Change: your EAP program to allow for more seamless transition.

Speaker Change: I think I want to make it clear, it's our goal to be able to transition our EAP patients to commercial supply within the first year post-launch.

Speaker Change: And that will allow us to be able to move on to other areas that we can collect data and real world data and other things that can help patients move forward. So there are things that we can do and we're working with those sites and investigators. One of them is around supply. We may give a 90 day supply, then it may go to a 60 day supply, but we're doing this in a very...

Neil McFarlane: So there are things that we can do, and we're working with those sites and investigators. One of them is around supply. We may give a 90-day supply, then it may go to a 60-day supply, but we're doing this in a very cautious way to ensure that every patient continues to have access to the EAP and supply.

Josh Schafer: So, there are things that we can do, and we're working with those sites and investigators. One of them is around supply. We may give a 90-day supply, then it may go to a 60-day supply, but we're doing this in a very cautious way to ensure that every patient continues to have access to the EAP and supply. I can't be more emphatic about the fact that we are here to make sure that we're taking care of these patients in the EAP until they can get transition.

Neil McFarlane: I can't, I can't be more emphatic about the fact that we are here to make sure that we're taking care of these patients in the EAP until they can get transitioned. Thank you so much. I appreciate it. Thank you, this does uh...

Speaker Change: and...

Speaker Change: cautious way to ensure that every patient continues to have access to the EAP and supply. I can't I can't be more emphatic about the fact that we are here to make sure that we're taking care of these patients in the EAP until they can get transitioned.

Lydwayne Clifton: Selling general and administrative expenses were $12.6 million during second quarter, reflecting our commercial team being in place for the entire quarter, and actively engaged in activities to build awareness and provide patient services related to OPPROVA. Net Loss for Q2, 2024, was $19.9 million, or 48 cent per basic and diluted share, which reflects an increased driven by our investments in our commercial infrastructure.

Oren Livnat: Thank you so much. I appreciate it. Thank you.

Speaker Change: Thank you so much, I appreciate it.

Unknown Executive: This does conclude the Q&A portion of today's call.

Operator: This concludes the Q&A portion of today's call. I would now like to turn the call back over to Neil McFarlane for any additional or closing remarks. Thank you. We continue to make solid advances towards achieving our mission of building a leading patient-focused rare disease therapeutics company. As we look to our upcoming catalysts in the second half of 2024, our priorities are clear, and we look forward to updating you in the future.

Speaker Change: Thank you. This does conclude the Q&A portion of today's call. I would now like to turn the call back over to Neil McFarlane for any additional or closing remarks.

Neil McCarline: I would now like to turn the call back over to Neil McFarlane for any additional or closing remarks. Thank you. We continue to make solid advances towards achieving our mission of building a leading patient-focused rare disease therapeutic company. As we look to our upcoming catalyst in the second half of 2024, our priorities are clear, and we look forward to updating you in the future.

Neil McFarlane: Thank you. We continue to make solid advances towards achieving our mission of building a leading patient-focused rare disease therapeutics company.

Lydwayne Clifton: At the beginning of the quarter, we announced the refinancing of our existing debt with a new $100 million credit facility from which we took an initial draw of $60 million. A second tranche of up to $20 million is available at our discretion until October 5, 2025, and a third tranche of up to $20 million will become available upon approval of AeroMachimal, in each case subject to certain terms and conditions. As of June 30, 2024, total cash, cash equivalents, and investments were $49.3 million, which was a decrease of $3.4 million compared to March 31st. Use of cash during the period was $17.4 million, offset by net proceeds of $14 million from our initial draw from our credit facility. Total long-term debt was $58.3 million as of June 30, 2024.

Speaker Change: As we look to our upcoming catalysts in the second half of 2024, our priorities are clear, and we look forward to updating you in the future. Thank you for joining us today.

Unknown Executive: Thank you for joining us today. This does conclude today's program. Thank you for your participation, and you may now. Jacks.

Neil McFarlane: Thank you for joining us today. This does conclude today's program. Thank you for your participation, and you may now disconnect. A.M. [music] OpenTreeCreating.com, Groupon, Gman Groupon reporting by Jeffrey Walshtaking, Dr. Worrell W. Wex, Ed Glenn, Joanne Rabinowitz, Matt Riddle, Mark McEnroe. [music] Bye-bye. Thank you. John F. Made By 2 originals Co-Produced By THANKS FOR WATCHING, Travel Assistant, Earned SCHMALTZ Tuning onwards. Thanks For suspend CCC Sulrin Information Microsoft Office Word Miracle Media Click Here To Watch Another Withinante Rocky Movie, [music] Omen, Omen, Omen, Tha Tha Tha Tha Tha Tha Tha Tha Tha Tha Tha Tha Tha Tha Tha Tha Tha Tha Tha Tha Tha Tha Tha Tha Tha

Speaker Change: This does conclude today's program. Thank you for your participation and you may now disconnect.

Speaker Change: I'm the one who's going to do it. I'm the one who's going to do it.

Speaker Change: Thank you very much for watching.

Speaker Change: The End

Lydwayne Clifton: As Neil mentioned earlier, we successfully completed a modestly sized underwritten public offering, which brought $64.5 million in net proceeds to Zevra, along with attracting a cadre of institutional investors well-known in the biotech industry as long-term supporters of innovation and solid execution. We issued approximately $10.6 million shares at a price of $6.50 per share. This offering was significantly over-subscribed, which testifies to the strong sentiment around the potential of the several upcoming value creation opportunities for Zevra.

Lydwayne Clifton: Combining the net proceeds from this offering with our existing resources, pro-form a June 30 of 2024, cash, cash equivalents, and investments is $113.8 million. Our cash runway now extends into Q1, 2027. Pro-form a common and fully deluded shares outstanding were $52.6 million and $67.9 million respectively. No warrants were issued in connection with this offering. It is important to note that our cash runway guidance is based on our current operating plan, available cash, cash equivalents, and investments, including the additional cash received through our recently completed secondary offering, and our subject to continuing compliance with our debt covenants.

Lydwayne Clifton: Our forecast includes commercial revenue from the sales of Opruva, reimbursements from the French EAP for Ayrmachimal, and ongoing royalties under the Astaurus License Agreement. It does not include commercial revenue from sales of Ayrmachimal, nor the sale of the PRV, which would follow FDA approval.

Speaker Change: Music

Neil McFarlane: Unknown Speaker 003 004 006 007 008 009 0010 0011 0012 0013 0014 0015 0016 0017 0018 0019 0020 0021 0022 0023 0024 0025 0026 0027 0028 0029 0031 Don't forget to subscribe to my channel! I'm, KTell Me The Stories ??? conversation An In Make Cinematographer Tom Walker Music Ivan Darion, Music Thanks for watching, plz subscribe

Unknown Executive: Now we will turn the call over to the operator for questions. Thank you. At this time, if you would like to ask a question, please press star and one on your telephone keypad. You may remove yourself from the cube by pressing star and two. Again it is star and one to ask a question today.

Speaker Change: RUTLEDGE RUTLEDGE RACE RUTLEDGE TORONTO 2015 PAN AM GAMES RUTLEDGE RACE TORONTO 2015 PAN AM GAMES

Louise Chen: We'll take our first question from Louise Chen with Cantor. Please go ahead. Your line is open. Hi, congratulations on all the progress this quarter and thanks for taking my questions. So I had a few for you. First question was how you think about all proof of sales in the second half of 24. And then are you, do you have any thoughts on initial thoughts I'm pricing for our local mall if it gets approved. And the last question is just in our our local mall, the patents protection and marketing exclusivity. Thank you. Thanks Louise.

Neil McCarline: Why don't I start with the last question and I'll T the pricing and then I'll hand it off to Josh to talk a little bit about our mock them all. And as well as old proof up in regards to the patent protection and marketing exclusivity for our mock them all. We rely on orphan drug exclusivity for up to seven years. And then in regards to pricing for our mock them all so we'll early for us to be talking about pricing as we're just now in the labeling discussions.

Neil McCarline: But I think it's I want to make it clear to everybody that it is our goal to make our mock them all as widely available as possible as we can. And with that, I'll hand it up to Josh to talk a little bit about what we've been doing around understanding the market for our mock them all around around what we could be for pricing as well as all proof of sales in the second half of 2024.

Unknown Executive: Dr. Thomas Lugo, Dr. Thomas Lugo, Dr. Thomas Lugo,

Neil McCarline: That was regards to all here mock them all pricing. We have conducted pretty extensive market research with payers and we've gone out and we've tested the clinical profile and really pressured them around. You know responding to the value proposition and the clinical value of of air and mock them all for patients with NPC. I have to say that it's widely been viewed as what could potentially be foundational therapy if approved by payers.

Neil McCarline: But of course the final label will ultimately influence the final price and how payers view that. But we're working very closely with them. We're putting in place patient services and and other resources to make sure that air mock them all will be as widely available to patients as possible. And then with regards to all proof of in the second half. And I'd like to just remind everyone that the first half was really built or was really focused on building awareness where there was very little awareness within the prescribing community for all proof of.

Neil McCarline: We're very happy with the progress the team made in terms of getting in front of prescribers and payers and really building that awareness across that prescribing community. And we have more than 75% covered lives for for patients with with UCD. We've also put in place some other resources and tactics, including a free trial program and a demonstration program to be able to allow the physicians and prescribers to see how approval works.

Neil McCarline: And we've made a change to our specialty pharmacy. All of this I think bodes well for the second half. And as we really begin to focus on building more awareness within the patient community, we're looking forward to seeing increased enrollment. Thank you.

Tim Lugo: We'll take our next question from Tim Lugo with William Blair. Please go ahead. Your line is open. Thanks for the question and congratulations on all the progress. It's been a obviously transitional quarter. I just asked her the ad calm. It seems like, you know, combination use with McListat is going to be maybe occurring a little bit more than I maybe had expected.

Neil McCarline: I just love to hear your thoughts around combination use in the real world once approved and how does that fit into your pricing thoughts. And then maybe also some initial initial kind of feedback you've heard from the patient advocacy groups post ad calm. Thanks. Thanks, Tim. I think let me handle your first question and then I might actually ask Josh to talk a little bit about the advocacy engagement and Adrian. I think one of the things that's really important is you get what you study when it comes to labeling discussion and we've studied aromachemal versus placebo with underlying routine care.

Neil McCarline: And those patients could have been stratified to Bigelstad or some other kind of primary treatment that the patient had but routine care I think was standard in our discussions and in the ad calm. And I think now as we're driving into our labeling discussions, you usually get what you studied and for us, that's aromachemal versus placebo. That's the outcome that we're looking for.

Josh Schafer: So I can't talk more about combination therapy at this point in the game because we're in the process of round one, but I do think that there's precedent around study, you get what you study in regards to the initial feedback from the patient advocacy organizations. Let me ask Josh to talk a little bit about where we're seeing some excitement from the advocacy organizations today. Yeah, I think if anyone tuned into the advisory committee meeting 10 days or so ago, you would have seen overwhelming support for aromachemal from prescribers, caregivers and patients.

Josh Schafer: And that really is a reflection of the benefits that many patients have received from aromachemal through our expanded access program and open label program. And as we move forward, we expect that support to really continue. I can also say that as we've gone out and we've talked with payers and physicians and done some market research and tested the profile. Many of them really see aromachemal as the first approved drug for nimen tick really is the foundation for therapy for these patients.

Unknown Executive: Operator. As a reminder, if you'd like to ask a question today, please press the star and one keys on your telephone keypad.

Oren Livnat: We'll take our next question from Orrin Livnet with HC Wing. Please go ahead, your line is open. Thank you for taking the questions.

Neil McCarline: Let me add my congrats to the outcome of the adcom. Just so I'm clear, you mentioned you've entered the first round of labeling comments with the discussions with the FDA. Can you say if you've actually had any explicit additional information or requests or anything new you've had to provide in this process? Thanks, Oren. So the answer to your question is we have gotten our first round of labeling negotiations. And I think your second question was a regard into any specific information requests.

Neil McCarline: We have received additional information requests as you can imagine throughout this process. You continue to get them in regards to new data to be provided. We have not been at had any request to add new data more so than what has been shared specifically with the advisory committee. All right. Sorry with the FDA, not the advisory committee with the FDA. Okay, perfect.

Neil McCarline: Assuming or approved in late September, do you have any expectations for timing, for product availability given the significant overlap with the approval? I assume that would be relatively quickly, but I'm curious if you are neat to set up any different reimbursement support infrastructure given this is a new indication, unlike UCD. And in the NPC community and centers of excellence, you know, I guess I'm already asked about the feedback post ad comp.

Neil McCarline: But I'm just curious if there's any, you know, are you getting the sense that there's any particular preparation or expectations there post ad comps? Are you hearing about warehousing of patients or any proactive outreach maybe to patients and families and anticipation of potential approval?

Josh Schafer: Let me start by talking a little bit about the potential for our producer at the end of September. We are on track in order to be able to have product in the channel within the customer time frame of, you know, let's call it eight to 12 weeks. I've maybe even smaller than eight to 10 weeks post launch, which is fairly standard. Let me turn it over to Josh to talk a little bit about new infrastructure and or the needs in the NPC community post the advisory committee.

Josh Schafer: As a reminder, we built the commercial team to really focus not just on all proof of that in anticipation of an approval and launch of air optimal. So it is really right size to be able to optimize both products and and we also have the benefit of having a team out there now talking to prescribers about UCD. Oftentimes, these are the same physicians who are seeing Neem and tick patients. And so our team is out there profiling some of these physicians, understanding where the patients are.

Josh Schafer: Our team on the market access side is having conversations with payers on the clinical differentiation of air optimal and the need to treat. So the team is in place. We feel quite confident that it's it's right size for both products. And and we will be well positioned to begin commercialization as soon as drug is in the channel. In terms of where these patients are, we continue to work very closely with the investigators in our expanded access program.

Josh Schafer: As a reminder, we've got more than 70 patients here in the US who are in that program, as well as other patients who are being seen by those same investigators who are eagerly awaiting the approval of our model.

Neil McCarline: And I guess just lastly, you segue into the EAP patients, is there any preparation you can do separate or in addition to standard market pre-commercial activities with regard to this EAP population in particular since you know the patients and have relationships with them directly to some extent? Is there anything you can do to expedite their transition to commercial product when this product is available as opposed to maybe what the process would be with a new patient?

Neil McCarline: So thanks, Oren. A couple of things. One is, I think number one is, we should get on the table here, is that we're going to continue to support these patients in the EAP program until we've got commercial supply and access for patients to be able to transition. Answering your second part of the question, and we are working directly with investigators, not necessarily with patients directly, because recall the EAP program is still collecting data, and we don't have to direct hands-on with patients.

Neil McCarline: The next thing you asked about is in regards to, can you set up your EAP program to allow for more seamless transition so that I think I want to make it clear, it's our goal to be able to transition our EAP patients to commercial supply within the first year post launch. And that will allow us to be able to move on to other areas that we can collect data and real world data and other things that can help patients move forward.

Neil McCarline: So there are things that we can do, and we're working with those sites and investigators. One of them is around supply. We may give a 90-day supply, then it may go to a 60-day supply, but we're doing this in a very cautious way to ensure that every patient continues to have access to the EAP and supply. I can't be more emphatic about the fact that we are here to make sure that we're taking care of these patients in the EAP until they can get transition.

Oren Livnat: Thank you so much, I appreciate it.

Unknown Executive: Thank you.

Neil McCarline: This does conclude the Q&A portion of today's call.

Neil McCarline: I would now like to turn the call back over to Neil McFarland for any additional or closing remarks. Thank you. We continue to make solid advances towards achieving our mission of building a leading patient-focused rare disease therapeutic company. As we look to our upcoming catalyst in the second half of 2024, our priorities are clear, and we look forward to updating you in the future.

Unknown Executive: Thank you for joining us today.

Unknown Executive: This does conclude today's program. Thank you for your participation, and you may now. Jacket.

Unknown Executive: Dr. Paret, Dr. Paret, Dr. Paret,

Q2 2024 Zevra Therapeutics Inc Earnings Call

Request a DemoDemo

ZVRA

Zevra Therapeutics

Earnings