Q1 2024 Zevra Therapeutics Inc Earnings Call
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Please standby your program is about to begin if you should need audio assistance during your call today. Please press star zero.
Unknown Executive: Thank you.
Good morning, everyone. Thank you for joining does serve ret therapeutics first quarter 2020 for corporate updates and financial results call. Today's call is being recorded and will be made available on the company's website. Following the conclusion of the call with that I will now turn the call over to Nicole Osha.
Unknown Executive: Please stand by. Your program is about to begin. If you should need audio assistance during your call today, please press star zero. Good morning, everyone. Thank you for joining the Zevra Therapeutics First Quarter 2024 Corporate Updates and Financial Results Call. Today's call is being recorded and will be made available on the company's website following the conclusion of the call. With that said, I will now turn the call over to Nichol Ochsner, Vice President of Investor Relations and Corporate Communications at Zevra Therapeutics.
Nichol L. Ochsner: President of Investor Relations and corporate communications at Zebra Therapeutics.
Nichol L. Ochsner: Good morning, and thank you for joining us today to review Zevra Therapeutics' progress in the first 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 L. Ochsner: Good morning, and thank you for joining us today to review <unk> Therapeutics progress in the first quarter of 2024 outlining our clinical advances operational achievements and financial results.
Nichol L. Ochsner: Before we get started let me take a moment to provide some important information.
Nichol L. Ochsner: I encourage you to access the news release which was published this morning and is available in the Investor section of Zevra's website. As we begin our call, it's important to highlight that today's discussion will include forward-looking statements. Forward-looking statements are not promises or guarantees and are inherently subject to risks, uncertainties, and other significant factors that may lead to actual results differing materially from the projections made.
Nichol L. Ochsner: Encourage you to access the news release, which was published this morning and is available in the Investor section observers website.
Nichol L. Ochsner: As we begin our call it's important to highlight that todays discussion will include forward looking statements.
Nichol L. 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 differ materially from the projections made please refer to the risk factors section in our most recent quarterly report on Form 10-Q.
Nichol L. Ochsner: Please refer to the risk factors section in our most recent quarterly report on Form 10-Q and other filings with the SEC, including our annual report on Form 10-K. I am pleased to welcome Zevra's management team members participating in today's call. I'm joined today by Neil McFarlane, President and Chief Executive Officer, LeDwayne Clifton, our Chief Financial Officer, Joshua Schafer, our Chief Commercial Officer and Executive Vice President of Business Development, Christal Mickle, our Chief Development Officer, and Adrian Quartel, our Chief Medical Officer. Now, I'll turn the call over to Neil.
Nichol L. Ochsner: And other filings with the SEC on annual report on Form 10-K, I am pleased to welcome <unk> management team members participating in today's call I'm joined today by Neil Macfarlane, President and Chief Executive Officer, Dwayne Clifton, Our Chief Financial Officer.
Nichol L. Ochsner: Josh what Schaffer, our Chief commercial officer, and executive Vice President of business development Crystal Nichols, Our Chief Development Officer, and Adrian <unk>, Our Chief Medical Officer, now I'll turn the call over to Neil.
Neil F. McFarlane: Thank you, Nichol, and thank you all for taking the time to join us today. During the first quarter, we made steady progress in executing on our strategic objectives. On our last earnings call, we announced that we were focused on three key priorities. First, to successfully launch El Prova and ensure access for patients. Second, to prepare for the potential launch of Aramakamal, and third, to advance the KP1077 program in sleep disorders. I'm pleased to report that we are executing on all of these objectives, and today we'll share with you a summary of our key accomplishments in the first quarter and the reasons we are optimistic for 2024 and beyond.
Neil F. McFarlane: Thank you Nicole and thank you all for making the time to join us today.
Neil F. McFarlane: During the first quarter, we made steady progress in executing on our strategic objectives.
Neil F. McFarlane: Last earnings call, we announced that we were focused on three key priorities first to successfully launch approval and ensure access for patients second to prepare for the potential launch of a remarkable and.
Third to advance the KC 10, 77 program and sleep disorders.
Neil F. McFarlane: I'm pleased to report that we are executing on all of these objectives and today, we'll share with you a summary of our key accomplishments in the first quarter and the reasons, we are optimistic for 2024 and beyond.
Neil F. McFarlane: In addition to executing on our three key priorities, we refinanced our existing debt with up to $100 million in committed capital led by premier biotech investors, including Perceptive Advisors and health care royalty partners. This new credit facility, which LeDoyne will cover in more detail, has further strengthened our balance sheet and provides added capital flexibility to support our mission. The company continues to work through a period of significant growth and transformation with the addition of talented people and capabilities from each of the three companies that have now come together to become one Zevra.
Neil F. McFarlane: In addition to executing on our three key priorities, we refinanced our existing debt with up to $100 million in committed capital led by Premier biotech investors, including perceptive advisors and healthcare royalty partners. This new credit facility, which <unk> will cover in more detail as further strength.
Neil F. McFarlane: And our balance sheet and provides added capital flexibility to support our mission.
Neil F. McFarlane: The company continues to work through a period of significant growth and transformation with the addition of talented people and capabilities for each of the three companies that have now come together to become one zebra.
Neil F. McFarlane: The team is making significant progress advancing our rare disease portfolio, and in Q1, we initiated a long-range planning process, including a portfolio prioritization review, with a focus on building a sustainable business with reliable cash flows. Achieving our strategic objectives to commercialize OPRUVA and then, upon approval, successfully launch Aramakamal are key to reaching that seminal inflection point. We initiated the full commercial launch of OPRUVA at the end of January 2024. As a reminder, OPRUVA is indicated for the treatment of certain urea cycle disorders, or UCDs, which are a group of rare genetic disorders that can cause harmful levels of ammonia to build up in the blood, potentially resulting in neurocognitive impairments, brain damage, and in some cases, coma or death.
Neil F. McFarlane: The team is making significant progress advancing our rare disease portfolio and in Q1, we initiated our long range planning process, including a portfolio prioritization review with a focus on building a sustainable business with reliable cash flows.
Neil F. McFarlane: Achieving our strategic objectives to commercialize all prove up and then upon approval successfully launching aramark. The ball are key to reaching that seminal inflection point.
Neil F. McFarlane: We initiated the full commercial launch of all prove out the end of January 2024, as a reminder, all prove is indicated for the treatment of certain urea cycle disorders or you see these which are a group of rare genetic disorders that can cause harmful levels of ammonia to buildup in the blood.
Neil F. McFarlane: Actually resulting in neurocognitive impairment brain damage and in some cases coma or death.
Neil F. McFarlane: We estimate that there are approximately 2,000 people in the U.S. with UCD, of which roughly half are diagnosed and treated. The UCD market in the U.S. is estimated at approximately $350 million annually. Despite the availability of therapies, there are still unmet needs for people living with UCD. We believe that Ulpruva is well suited to address these needs as it provides personalized doses for each patient's requirements. It is portable and easy for a patient to take. And, most importantly, it is palatable, as it was formulated to overcome the challenging taste and smell associated with other formulations of sodium phenylbutyrate.
We estimate that there are approximately 2000 people in the U S with UCD of which roughly half are diagnosed and treated.
Neil F. McFarlane: The use of the market in the U S is estimated at approximately $350 million annually.
Neil F. McFarlane: Despite the availability of therapies unmet needs for people living with UCD persist.
Neil F. McFarlane: We believe that'll prove is well suited to address these needs as it provides personalised dosage for each patient's requirements. It is portable and easy for patients to take and most importantly, it is palatable as it was formulated to overcome the challenging taste and smell associated with other formulations of sodium.
Neil F. McFarlane: Fell beautifully.
Neil F. McFarlane: Since launch, we've been focused on raising the awareness of OPRUVA and demonstrating our commitment to UCD patients. In the quarter, we had four new patient enrollments, which we define as a prescription for a patient eligible for benefits investigation or a quick start program. We are encouraged by the progress that our rare disease specialists accomplished in the first few months of launch, meeting with more than 90% of the specialists at the 40 Centers of Excellence that treat people with UCD.
Neil F. McFarlane: Since launch we've been focused on raising the awareness of all prove out and demonstrating our commitment to UCD patients.
Neil F. McFarlane: In the quarter, we had four new patient enrollments, which we define as a prescription for a patient eligible for benefits investigation or our quick start program.
Neil F. McFarlane: We are encouraged by the progress that our rare disease specialist accomplished in the first few months of launch meeting with more than 90% of the specialist at the 40 centers of excellence that treat people with UCB.
Neil F. McFarlane: We've also assembled a team of marketers, patient services, and market access professionals, as well as medical science liaisons and patient advocates who are engaging with key stakeholders at patient events and medical conferences. Additionally, our managed care team has been working with government and commercial payers to ensure broad access for patients. We have seen meaningful growth in reimbursement coverage, which was 55% at the time we acquired OPRUVA, to nearly 75% of covered lives as of May 1st. Overall, I'm pleased with the progress we've made in the first few months of launch and look forward to reporting more details as the launch matures.
Neil F. McFarlane: We've also assembled a team of marketers patient.
Neil F. McFarlane: Patient services and market access professionals as well as medical science liaisons and patient advocates who are engaging with key stakeholders at patient events and medical conferences.
Neil F. McFarlane: Our managed care team has been working with government and commercial payers to ensure broad access for patients.
Neil F. McFarlane: We have seen meaningful growth and reimbursement coverage, which was 55% at the time, we acquired all prove up to nearly 75% of covered lives as of May 1st.
Neil F. McFarlane: Raul I'm pleased with the products. We have made in the first few months of launch and look forward to reporting more details as the launch matures.
Neil F. McFarlane: That was commercial footprint was established to provide a high strategic fit between approval and a remarkable given that the majority of prescribers for both products work within the same centers of excellence.
Neil F. McFarlane: Devere's commercial footprint was established to provide a high strategic fit between O'Prova and Aramacomal, given that the majority of prescribers for both products work within the same centers of X2. If Aramata Mall is approved, we believe this close proximity and overlap in patient care will allow us to realize synergies and scale with our commercial infrastructure. In fact, our team has already begun to identify and compliantly engage with key opinion leaders and prescribers who treat UCD and Niemann-Pick disease type C, or NPC.
Neil F. McFarlane: Mark The mall is approved we believe this close proximity and overlap in patient care will allow us to realize synergies and scale with our commercial infrastructure.
Neil F. McFarlane: In fact, our team has already begun to identify and completely engaged with key opinion leaders and prescribers, who treat UCD and niemann pick disease type C or N P C.
Neil F. McFarlane: Our market access team has initiated clinical discussions with payers regarding MPC and Arimofamo. These, and other synergies across our brands, will allow us to accelerate the launch of Aromathamol and ensure patients have access to this much-needed therapy.
Neil F. McFarlane: Our market access team has initiated clinical discussions with payers regarding MPC and are remarkable.
Neil F. McFarlane: These and other synergies across our brands will allow us to accelerate the launch of our remarkable and ensure patients have access to this much needed therapy.
Neil F. McFarlane: As a reminder, our motto mall is our investigational drug candidate in development for the treatment of MPC are rare genetic progressive and potentially fatal neurologic disease.
Neil F. McFarlane: As a reminder, Arimothamol is our investigational drug candidate in development for the treatment of MPC, a rare, genetic, progressive, and potentially fatal neurologic disease. If approved, Aramacomal would be the first drug indicated for the treatment of NPC in the U.S. Currently, there are approximately 900 people living with MPC, of which roughly 300 are diagnosed. Of those, approximately 70 are enrolled in our Expanded Access Program
Neil F. McFarlane: If a proof aramark the mall would be the first drug indicated for the treatment of MPC in the U S.
Neil F. McFarlane: Currently there are approximately 900 people living with MPC of which roughly 300 are diagnosed.
Neil F. McFarlane: There was approximately 70 are enrolled in our expanded access program or EAP.
Neil F. McFarlane: During the quarter, the FDA assigned a new PDUFA date of September 21st, 2024 and reaffirmed its intent to present the resubmission for discussion at an advisory committee meeting for which we are thoroughly prepared. The National Niemann-Pick Disease Foundation, known as MNPDF. She spearheaded efforts with six other advocacy and research organizations to compile a petition of nearly 1,000 signatures from NPC patients, caregivers, and physicians with direct experience utilizing our Imoclomol that was submitted to the FDA in Q1. We were overwhelmed by the outpouring of support for our mother mall within the community.
Neil F. McFarlane: During the quarter the FDA assigned a <unk> date of September 21, 2024, and reaffirmed its intent to present the resubmission for discussion at an Advisory Committee meeting for which we are thoroughly preparing.
Neil F. McFarlane: The National Niemann pick disease Foundation notice and then Pds spearheaded efforts with six other advocacy and research organizations to compile a petition nearly 1000 signatures from MPC patients caregivers and physicians with direct experience utilizing our remarkable that was <unk>.
Neil F. McFarlane: Made it to the FDA in Q1.
Neil F. McFarlane: We were overwhelmed by the outpouring of support for our remarkable within the community.
Neil F. McFarlane: As the FDA review continues, we will maintain our EAP and continue working tirelessly to bring this potential therapy to patients living with this devastating rare disease. We continue to work closely with key opinion leaders to educate them on Aramak Lamal's disease-modifying clinical profile and raise awareness of the heterogeneous presentation of NPC. In April 2024, we presented new long-term real-world data during the Society for Inherited Metabolic Disorders. The presentation included data from EAP patients from the U.S. Centers and demonstrated that adults treated with arimoclomol, including those with and without miglostat use, had stable disease courses, which is defined as not showing disease progression over the two years of treatment.
Neil F. McFarlane: The FDA review continues we will maintain our EAP and continue working tirelessly to bring this potential therapy to patients living with this devastating rare disease.
Neil F. McFarlane: We continue to work closely with key opinion leaders to educate on Aramark remodels disease, modifying clinical profile and raise awareness of the heterogeneous presentation of MPC.
Neil F. McFarlane: In April 2024, we presented new long term real world data during the society for inherited metabolic disorders.
Neil F. McFarlane: Presentation included data from EAP patients from the U S centers and.
Neil F. McFarlane: It demonstrated that adults treated with their remarkable including those with and without the <unk> stent use had stable disease course, which is defined as not showing disease progression over the two years of treatment with <unk>.
Neil F. McFarlane: The safety profile was consistent with that observed in the Phase 2-3 study. If approved, we intend to utilize our clinical data, as well as emerging real-world evidence from our EAP, to establish our mother model as foundational treatment for people living with NPC. Now, I'd like to turn your attention to KP 1077.
Neil F. McFarlane: Safety profile was consistent with that observed in the phase II III study.
Neil F. McFarlane: If approved we intend to utilize our clinical data as well as the emerging real world evidence from our EAP to establish our mothballed as foundational treatment for people living with MPC.
Neil F. McFarlane: Now I'd like to turn your attention to K P $10 77.
Neil F. McFarlane: Our clinical candidate is being developed as a treatment for idiopathic hypersomnia, or IH, a rare chronic sleep disorder. IH is characterized by excessive daytime sleepiness or an uncontrollable need to sleep and difficulty waking. There remains an unmet need, and we estimate 37,000 people in the U.S. are currently diagnosed with IA. As you may recall, KP1077, SIRDEX methylphenidate, or SDX, was designed to steadily release d-methylphenidate into active ingredients. This unique pharmacokinetic profile allows for flexible dosing to overcome these primary IH symptoms. It also ensures that patients receive the optimal drug concentration during waking and active hours. SDX is currently designated as a Schedule IV controlled substance by the U.S. Drug Enforcement Administration.
Neil F. McFarlane: Our clinical candidate being developed as a treatment for idiopathic hypersomnia or IH, a rare chronic sleep disorder.
Neil F. McFarlane: IHS characterized by excessive daytime sleepiness or uncontrollable need to sleep and difficulty waking.
Neil F. McFarlane: There remains an unmet need and we estimate 37000 people in the U S are currently diagnosed with IH.
Neil F. McFarlane: As you May recall <unk> 77.
Neil F. McFarlane: <unk> methylphenidate for SPX was designed to steadily release D. Methylphenidate, it's active ingredient.
This unique pharmacokinetic profile allows for flexible dosing to overcome these primary IH symptoms.
Neil F. McFarlane: This profile ensures that patients receive the optimal drug concentration during waking and active hours.
Neil F. McFarlane: SPX has currently designated as a scheduled for controlled substance by the U S drug enforcement administration.
Neil F. McFarlane: During the quarter, we reported topline data from our phase II study of <unk> 77 in patients with IH. We are encouraged by the results, which showed that <unk> 77 is well tolerated and demonstrates early signs of differentiated and clinically meaningful benefits.
Neil F. McFarlane: During the quarter, we reported top-line data from our phase two study of KP1077 in patients with IE. We are encouraged by the results, which show that KP1077 is well-tolerated and demonstrates early signs of differentiated and clinically meaningful benefits. The study successfully fulfilled the objectives of informing the design of a pivotal efficacy trial, and we are planning for an end of Phase II meeting with the FDA in Q3. Since completing the trial and reporting top-line results in March, we've been working closely with the sleep community to interpret these results.
Neil F. McFarlane: <unk> successfully fulfilled the objectives of informing the design of a pivotal efficacy trial and we are planning for an end of phase II meeting with the FDA in Q3.
Neil F. McFarlane: Since completing the trial and reporting top line results in March we've been working closely with the sleep community to interpret these results.
Neil F. McFarlane: With only one approved treatment, there remains a large unmet need for therapies with different mechanisms of action to address the symptoms of IH. We look forward to presenting the full data package from our completed study at the upcoming SLEAP 2024 conference in early June. As part of the strategic planning initiative kicked off in January, we completed our preliminary evaluation of the CILIPOLOL program for the treatment of vascular ehlers-danlos syndrome, or VEDS, which impairs COL3A1 connective tissue and leads to vascular and hollow organ rupture. Oliphalol'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 F. McFarlane: With only one approved treatment there remains a large unmet need for therapies with different mechanisms of action to address the symptoms of IH we.
Neil F. McFarlane: We look forward to presenting the full data package from our completed study at the upcoming sleep 2024 conference in early June.
Neil F. McFarlane: As part of the strategic planning initiative kicked off in January we completed a preliminary evaluation of the slip below a program for the treatment of vascular eilers, danlos syndrome, or events, which impairs call 381, connective tissue and leads to vascular and hollow, Oregon ruptures.
Neil F. McFarlane: <unk> mechanism of action is designed to reduce the mechanical stress oncology fibers within the arterial wall to vascular dilation and smooth muscle relaxation.
Neil F. McFarlane: Solipolol received orphan drug and breakthrough therapy designations from the FDA. The Phase III protocol is being conducted under a Special Protocol Assessment, or SPA, agreement with the FDA. We recently restarted recruitment of the Phase C trial, also known as the DISCOVER trial, to support patients currently enrolled and to preserve the value of the program while we complete our portfolio review. This is a decentralized event trial design of Silliprol on VEDs-related event reduction. Soliprolol is a primary treatment option in various European countries. And we believe that it could address a significant unmet need in the US, as there are no approved treatments for the 7,500 diagnosed patients with VEDS.
Neil F. McFarlane: Liberal all received orphan drug and breakthrough therapy designations from the FDA.
Neil F. McFarlane: The phase III protocol is being conducted under a special protocol assessment or Spa agreement with the FDA.
Neil F. McFarlane: We recently restarted recruitment of the phase III trial also noticed at also known as the discover trial.
Neil F. McFarlane: To support patients currently enrolled and to preserve the value of the program, while we complete our portfolio review.
Neil F. McFarlane: This is a decentralized is that trial design of <unk> on beds related is that reduction.
Neil F. McFarlane: So let the wall as a primary treatment option in various European countries, and we believe that it could address the significant unmet need in the U S. As there are no approved treatments for 7500 diagnosed patients with vets.
Neil F. McFarlane: Looking ahead, we have three areas of focus first continue to drive the launch of all proven.
Neil F. McFarlane: Looking ahead, we have three areas of focus. First, continue to drive the launch of Ol Prueba. Second, prepare for a potential ADCOM and launch of Aramakumal. And third, to advance KP 1077. Now I'll hand the call over to LeDwayne, who will provide an update on our financial results.
Neil F. McFarlane: To prepare for a potential AD com and launch of our mothball and third to advanced <unk> 77.
Neil F. McFarlane: Now I will hand, the call over to Duane who will provide an update on our financial results.
LeDwayne Clifton: Thank you and good morning. Before I begin, I would encourage you to refer to our quarterly report on Form 10-Q, which we intend to file on Thursday post-market, for more detailed information. As Neil has already outlined, the first quarter was a time of solid execution as we drive the business towards the accomplishment of our strategic objective. Our financial results for the first quarter reflect our foundation of financial strength as we continued our investments in building out our commercial capabilities and in the advancement of our development program.
Duane: Thank you and good morning.
Duane: Before I begin I would encourage you to refer to our quarterly report on Form 10-Q, which we intend to file Thursday post market for more detailed information.
Duane: As Neal has already outlined first quarter was a time of solid execution as we drive the business towards the accomplishment of our strategic objectives.
Duane: Our financial results for the first quarter reflect our foundation of financial strength as we continued our investments in building out our commercial capabilities and in the advancement of our development programs.
Duane: Net revenue for the quarter was $3 $4 million, which includes $2 $2 million of net reimbursements from the French EAP for Aramark them all.
LeDwayne Clifton: Net revenue for the quarter was $3.4 million, which included $2.2 million in net reimbursements from the French EAP for Aramaclamal and 1.2 million dollars in royalties and other reimbursements under the Astoris Lights agreement. Currently, we recognize commercial product revenue when shipments are received by our specialty pharmacies. Based on the early stage of launch and inventory in the channel, OPRUBA sales were de minimis during the quarter.
Duane: $1 $2 million of royalties and other reimbursements under the <unk> license.
Duane: Currently we recognized commercial product revenue when shipments are received by our specialty pharmacy.
Duane: Based on the early stage of launch and inventory in the channel Oh prove our sales were de minimis during the quarter.
Duane: R&D expenses for the first quarter increased to $12 $3 million, which was primarily driven by the KC 10, 77 phase III trial NIH that has since been completed.
LeDwayne Clifton: R&D expenses for the first quarter increased to $12.3 million, which was primarily driven by the KB1077 Phase II trial at NIH that has since been completed, as well as our work to support the Aramakomo NDA during the ongoing review cycle and to prepare for a potential ADCOM sometime prior to the upcoming PDUFA date. Selling general and administrative expenses were $9.9 million and reflect an increase in personnel costs and professional fees associated with our commercial infrastructure.
Duane: As well as our work to support the <unk> NDA during the ongoing review cycle.
Duane: And to prepare for a potential AD com sometime prior to the upcoming <unk> date.
Duane: Selling general and administrative expenses were $9 9 million and reflect an increase in personnel costs and professional fees associated with our commercial infrastructure.
Duane: Net loss for Q1, 2024 was $16 6 million or <unk> 40 per basic and diluted share.
LeDwayne Clifton: The net loss for Q1 2024 was $16.6 million, or $0.40 per basic and diluted share. As of March 31st, 2024, total cash, cash equivalents, and securities were $52.7 million, which was a decrease of $15 million compared to December 31st, 2023. Total shares of common stock outstanding were 41.8 million, and fully diluted shares outstanding decreased by 1.4 million to 56.8 million, which includes approximately 5.6 million shares issuable upon the exercise of the warrant.
Duane: As of March 31, 2020 for total cash cash equivalents and securities were $52 7 million.
Duane: Which was a decrease of $15 million compared to December 31, 2023.
Duane: Total shares of common stock outstanding were $41 8 million and fully diluted shares outstanding decreased by $1 4 million to $56 8 million, which includes.
Duane: Approximately $5 6 million shares issuable upon exercise of warrants.
Duane: As Neil mentioned on April 10, we announced the refinancing of our existing debt with a new credit facility, which provides up to $100 million in committed capital.
LeDwayne Clifton: As Neil mentioned, on April 10th, we announced the refinancing of our existing debt with a new credit facility, which provides up to $100 million in committed capital. With the initial draw of $60 million, we have refinanced our existing debt of approximately $43 million and added an incremental $14 million in net cash proceeds to the balance sheet after fees and discounts. A second tranche of up to $20 million is available until October 5, 2025, 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.
Duane: With the initial draw of $60 million, we have refinanced our existing debt of approximately $43 million and added an incremental $14 million and net cash proceeds to the balance sheet after fees and discounts.
Duane: A second tranche of up to $20 million is available until October five 2025.
Duane: And a third tranche of up to $20 million will become available upon approval of Aramark mall in each case subject to certain terms and conditions.
Duane: By restructuring the amounts previously outstanding on two different facilities, we have simplified and extended the maturity while also providing additional non dilutive capital to support our mission.
LeDwayne Clifton: By restructuring the amounts previously outstanding on two different facilities, we have simplified and extended the maturity while also providing additional non-dilutive capital to support our mission. As a result of this transaction, and based on our current operating plan, available cash, cash equivalents, and investments are expected to extend our cash runway further into 2026, subject to continuing compliance with our debt covenant. Our forecast includes commercial revenue from sales of Olpruva, reimbursements from the French EAP for Aramakamal, and ongoing royalties under the Astoris License Agreement. It does not include commercial revenue from the sales of Aramakamal or the sale of the priority review voucher, which would follow FDA approval.
Duane: As a result of this transaction and based on our current operating plan available cash cash equivalents and investments are expected to extend our cash runway further into 2026 subject to continued compliance with our debt covenants.
Duane: Our forecast includes commercial revenue from sales of all prove up reimbursements from the French EAP for Aramark hamal and ongoing royalties under the <unk> license agreement.
Duane: It does not include commercial revenue from the sales of Aramark hamal, nor the sale of the priority review voucher, which would follow an FDA approval.
Duane: Disciplined utilization of resources will continue to be our guiding principle as we make prudent investments in our commercial and development operations, while matching those investments to our operating requirements.
LeDwayne Clifton: Disciplined utilization of resources will continue to be our guiding principle as we make prudent investments in our commercial and development operations while matching those investments to our operating requirements. We are optimistic about the opportunities we have in store for 2024. Our focus is on creating long-term value for shareholders by executing against our plan in support of our mission to become a leading rare disease company. We will now turn the call over to the operator for questions.
Duane: We are optimistic about the opportunities we have in store during 2024.
Duane: Our focus is on creating long term value for shareholders by executing against our plan in support of our mission to become a leading rare disease company.
Speaker Change: We will now turn the call over to the operator for questions.
Speaker Change: Yes.
Unknown Executive: Thank you, and at this time, if you wish to ask a question, please press star 1 on your telephone keypad. You may remove yourself from the queue by pressing star 2. We'll take our first question from Tim Lugo with William Blair. Please go ahead.
Speaker Change: Thank you and at this time, if you wish to ask a question. Please press star one on your telephone keypad, you may remove yourself from the queue by pressing star two.
Speaker Change: Our first question from Tim Lugo with William Blair. Please go ahead.
Speaker Change:
Tim Lugo: Thank you for taking the question. Regarding Aromaclomol, I know the data submitted to the agency included the use of Aromaclomol alone and in combination with Miglostat. Can you just clarify how you expect the, you know, product to eventually be used by patients? Is combination going to be more used? Is it going to be used alone more? Just, you know, what do you think the strength of data for both of those approaches is?
Tim Lugo: Thank you for taking my question.
Tim Lugo: For Aramark limo, another data submitted to the agency included.
Tim Lugo: Yes.
Tim Lugo: <unk> alone and in combination combination of Nicolas that can you just clarify how you.
Tim Lugo: Expect the.
Tim Lugo: Product to eventually be used by patients in combination is going to be more.
Tim Lugo: He is more is it going to be used alone more.
Tim Lugo: Just what do you view the strength of data for both of those approaches.
Tim Lugo: Tim Thanks for that question. So in clinical trial that was performed we indeed had patients that were both a maintenance test and not on maintenance staff.
Neil F. McFarlane: Tim, thanks for that question. So in the clinical trial that was performed, we indeed had patients who were both a miglostat and not a miglostat. And what we found is that patients, whether they were a miglostat or not a miglostat, improved while on aromoclomol. What we saw overall was that there was not a significant increase in the number of patients that were on aromoclomol, and a big difference between the improvement in patients with miglostat or without miglostat.
Tim Lugo: What we found is that patients whether they were mirrors that are not Omega staff improved while speeds.
Tim Lugo: On are multimodal.
What we saw overall is that there was one this big difference between the improvement in patients with major center without makes them. So we believe that once we go to market that are more for mobile will be the foundational therapy.
Neil F. McFarlane: So we believe that once we go to market, Aramoclomol will be the foundational therapy. You know, it is a disease-modifying treatment, and whether physicians and patients want to add miglostat to that foundational therapy is really up to them. I think it needs to be noted that miglostat is not approved for the treatment of neuromyxia, and Aramoclomol will most likely be the only approved foundational therapy for patients with neuromyxia
Tim Lugo: So it is a disease modifying treatments.
Tim Lugo: Physicians and patients want to add on <unk>. Two that's foundational therapy is really up to them I think it needs to be noted that <unk> is not approved for treatment of <unk> and there are multiple will most likely occur.
Tim Lugo: Approved foundational therapy for patients.
Speaker Change: Okay. Thank you and can you discuss it sounds like the patient advocacy groups are.
Joshua M. Schafer: Okay, thank you. And can you discuss, it sounds like the patient advocacy groups are, I guess, kind of rallying around the filing a bit. Can you maybe summarize your interactions with those groups or some of the patient advocates that you've heard from in the past quarter? It seems like it's really ramping up after the submission.
Speaker Change: I guess kind of rattling around the filing a bit can you maybe summarize your interactions with those groups or or or.
Speaker Change: Some of the patient advocacy you've heard in the past quarter. It seems like it's really ramping into the.
Speaker Change: Into the I guess.
Speaker Change: After the submission.
Speaker Change: Yes, Hi, Tim This is Josh Shanker and we.
Joshua M. Schafer: Yeah, hi Tim, this is Josh Schafer. And we have been very engaged with the patient advocacy community for several years now. And they have demonstrated their support of Aramak Lamal, most recently, signing a petition with 1000 signatures of patients, caregivers, and physicians who were in support of Aramak Lamal's submission and eventual and hopeful approval. So that certainly demonstrates their support for Aramak Lamal and for Zevra. And we remain very committed to that patient community.
Joshua M. Schafer: We have been very engaged with the patient advocacy community for for several years now and they are.
Joshua M. Schafer: They have demonstrated their support of earmark hamal, most recently signing a petition with 1000 signatures.
Joshua M. Schafer: Patients caregivers and physicians, who are in support of Aramark limos submission and eventual hopeful approval. So thats certainly demonstrates their support for <unk> and for Zebra and we remain very committed to.
Joshua M. Schafer: It's a very well organized community. They have, you know, helped to build a lot of the awareness for Aramak Lamal that we We hope we'll help with the launch at approval. And as you know, there are about 70 patients in our Expanded Access Program in the United States, plus another approximately 300 patients in the U.S. that are actively being treated, and we hope to be able to make our mock-a-mole available for all of those patients.
Joshua M. Schafer: To that patient community, it's a very well organized community.
Joshua M. Schafer: They have.
Joshua M. Schafer: Helped to build a lot of the awareness for Aramark home all that.
Joshua M. Schafer: We hope will help.
Joshua M. Schafer: Helped with the launch at approval.
Joshua M. Schafer: And as you know there are about 70 patients in our expanded access program in the United States plus another approximately 300 patients in the U S that are actively being treated and we hope to be able to make aramark hamal available for all of those patients.
Speaker Change: Alright, thank you.
Tim Lugo: All right, thank you. And I guess one last question. I believe you mentioned 300 MPC patients were diagnosed, but there's an expectation of 900 here in the U.S. Can you just talk about that difference in the differential between diagnosed and expectation? Sure, 900 is the estimated number.
Speaker Change: I guess one last question I believe you mentioned 300.
Speaker Change: Mtc patients were diagnosed but there was an expectation of 900.
Speaker Change: Here in the U S. Can you just talk about the difference in the differential between diagnosed and expectation.
Joshua M. Schafer: Sure. 900 is the estimated prevalence of the disease, but not all of those patients have been confirmed with a diagnosis. That is based on some claims data that was published in 2021, I believe. And of those 900, there are about 350 who have been diagnosed and are receiving some form of treatment, whether that's miglostat today or treatment for symptomatic disease.
Speaker Change: Sure 900 is the estimated prevalence of the disease not all of those patients are confirmed diagnosis that is based on some claims data that was published in 2021, I believe and of those 900.
Speaker Change: There are about 350, who have been diagnosed and are receiving some form of treatment, whether thats makes us stand today or where treatment for symptomatic disease.
Speaker Change: Alright, thank you.
Speaker Change: Thank you we will take our next question from Jonathan Aschoff with Roth Am Kim. Please go ahead.
Jonathan Matthew Aschoff: Thank you. We will take our next question from Jonathan Aschoff of Roth MTN. Please go ahead.
Jonathan Matthew Aschoff: Thank you. Good morning. I was curious if you might be able to help us out a little with De Minimis. I'll approve the revenue.
Jonathan Matthew Aschoff: Thank you. Good morning, I was curious if you might be able to help us out a little with de Minimis revenue. Mr. Any help there or you just wish to keep that quiet for now.
Neil F. McFarlane: Is there any help there, or do you just wish to keep that quiet for now?
Jonathan Matthew Aschoff: Yes.
Speaker Change: Good morning, Jonathan.
Neil F. McFarlane: Good morning, Jonathan. It's really a function of the sort of product that's already in the specialty pharmacies, and therefore, we had modest shipments during Q1. So our main focus during Q1 has been to build awareness, to get out and make contact with the key opinion leaders and the prescribing physicians, and we've actually touched over 90% of those folks at this point. So in future quarters, as enrollments continue to build, we'll see more pulls into the pharmacy, and therefore revenue will go up, but de minimis is de minimis.
Jonathan Matthew Aschoff: It's really a function of sort of the product it's already at the specialty pharmacies and therefore, we had modest shipments during Q1. So our main focus during Q1 has been to build awareness to get out and make contact with the key K key opinion leaders and this is prescribing physicians and we've.
Jonathan Matthew Aschoff: Touch actually over 90% of those folks at this point so in future quarters as enrollments continue to Bill, we'll see more pulls into the pharmacy and therefore revenue will go up but de Minimis is de minimis.
Jonathan Matthew Aschoff: Okay, how about time to still process all the data? If you can, you know, venture a guess there at all or maybe help us out with the enrollment percentage at present, you know, something that will give us a sense of at least when the data will come. I know it's several years.
Speaker Change: Okay, how about.
Speaker Change: Time to let protocol data if you can.
Speaker Change: Venture to guess there at all or maybe help us out with the enrollment percentage at present, something that will give us a sense of at least one the data will come I know it's several years.
Adrian Quartel: Yeah, Jonathan, thank you for that question. So we started recruitment to support patients that are currently enrolled. We are looking at Leprol as part of our whole portfolio, so it's part of a preliminary portfolio review. We understand the value of the program, so we started this program. As said, this is running a SPA agreement with the FDA, and we're looking at 48 events. We currently have 17 patients enrolled, and we will continue to enroll throughout the rest of the year.
Speaker Change: Yes telephone. Thank you for that question, so restocking recruitment to support patient that are currently enrolled.
Speaker Change: We're looking at sort of full as part of.
Speaker Change: Our whole portfolio.
Speaker Change: Part of our preliminary April.
Speaker Change: You can review.
Speaker Change: We understand the value and the programs that we started this program.
Speaker Change: <unk> business runoff Spa agreement with the FDA and we're looking at 48 Defence. We currently have 17 patients enrolled and we continue to enroll the.
Speaker Change: The rest of the year.
Speaker Change: Okay. And then also have you noticed anything on the part of Amgen regarding marketing strategy with where their key or is it just <unk>.
Jonathan Matthew Aschoff: Okay, and also, have you noticed anything on the part of Amgen regarding their marketing strategy with Reviki, or is it just, you know, absolutely not a needle mover for them?
Speaker Change: Absolutely not a needle mover for them.
Neil F. McFarlane: So, you know, we can't really comment on what Amgen is doing, but, you know, we know that there are roughly 800 patients today that are receiving some therapy for UCD, and about 25% of those patients still have some hyperammonia events, and that's probably due to poor compliance as a result of patients not being able to tolerate current therapies. We believe that Alprove is going to help solve that problem with its ability to be personalized in its dose, to be portable in the way that it's delivered, and, most importantly, the palatability that we think will lead to better outcomes for these patients.
Speaker Change: So we can't really comment on what Amgen is doing but we know that.
Speaker Change: There are roughly 800 patients today that are receiving some therapies for for UCD and about 25% of those patients still has some hyper money you make events and thats, probably due to poor compliance as a result.
Speaker Change: <unk> not been able to tolerate chronic therapies.
Speaker Change: We believe that it will prove is going to help solve that problem with its ability to.
Speaker Change: The personalized in its dose to be affordable and it's in the way that it is delivered and most importantly, the tolerability that we think will lead to better outcomes for these patients.
Speaker Change: Okay. Thanks, and lastly, just a weird little detail on your 10-K. It says on $3 30, you're at 43 4 million shares then.
Jonathan Matthew Aschoff: Okay, thanks. And lastly, just a weird little detail. On your 10K, it says on 330 that you had 43.4 million shares. Then a day later, in this press release here today, it says you have 41.8. So did you buy back 1.6 million? Or what happened?
Speaker Change: Day later.
Speaker Change: In this press release here today. It says you have 41, eight so did you buy back $1 6 million or what happened.
Speaker Change: Yeah.
LeDwayne Clifton: There were no repurchases during Q1, and so... Chairs outstanding as of the end of the year was at 41.5, and chairs outstanding as of 3.31 is 41.8. So, are you referring to the fully diluted share count, Jonathan? It's just whatever's on...
Speaker Change: There was no repurchases during Q1.
Speaker Change: And so.
Speaker Change: Shares outstanding as of.
Speaker Change: End of the year it was at $41 five and shares outstanding as of $3 31 is 41 eight so.
Speaker Change: Are you referring to the fully diluted share count Jonathan.
Jonathan Matthew Aschoff: It's just whatever's on page 1 of the 10-K. I think it's never the fully diluted number, 43.4. It's just a weird little detail.
Jonathan Matthew Aschoff: Just whenever it's on page one in the 10-K.
Speaker Change: Salt Lake is a good number.
Speaker Change: Okay.
Speaker Change: 43 points, yes, so weird little detail.
LeDwayne Clifton: Yeah, I'm not sure, but Sherry's outstanding. When you see the 10Q tomorrow, it will be 41.8.
Jonathan Matthew Aschoff: Yes, I'm not sure if its shares outstanding when you see the 10-Q tomorrow be 41 eight.
Jonathan Matthew Aschoff: Okay, thank you very much.
Speaker Change: Okay. Thank you very much.
Jonathan Matthew Aschoff: Okay.
Jonathan Matthew Aschoff: Thank you and once again that is star and one for your question well go next to Louise Chen with Cantor. Please go ahead.
Unknown Executive: Thank you. And once again, that is the star and one for your questions. We'll go next to Louise Chen with Cantor. Please go ahead. Hi.
Louise Alesandra Chen: On peak sales for Alprova, how do you think about that? And how long do you think it'll take you to get there?
Louise Alesandra Chen: Hi, Thanks for taking my question and congrats on all the progress this quarter.
Louise Alesandra Chen: I wanted to ask you on peak sales for all prove out how do you think about that and how long do you think it'll take you to get there and then another question. We get a lot is just <unk> 77, comparing it behind contracting at the other products in the market and where your competitive advantage.
Joshua M. Schafer: And then another question we get a lot is just on KP 1077, comparing it, kind of comparing it to other products in the market, and where your competitive advantage is. And then lastly, just on your new opportunities here, the Solipolo product, what is the market opportunity, and what do you think about it? And what makes you excited about this opportunity? Thank you.
Jonathan Matthew Aschoff: And then lastly, just on <unk>.
Jonathan Matthew Aschoff: New opportunities here, the silicone IOL product.
Jonathan Matthew Aschoff: What is the market opportunity and how do you think about it like what makes you excited about this opportunity. Thank you.
Jonathan Matthew Aschoff: Sure. This is Josh and I'll address the proven question.
Joshua M. Schafer: Sure, this is Josh, and I'll address the question. So, as we noted, you know, the first quarter was really focused on building awareness of the drug. We have been really pleased with the performance of the team. You know, I'd like to remind you that in the first quarter, we built an entire commercial organization of really talented, experienced rare disease experts who were out talking with specialists. They've been able to engage with about 90% of the prescribers.
Josh: So as we noted the first quarter was really focused on building awareness of the of the drug.
Josh: We have been really pleased with the performance of the team.
Josh: Like to remind you that in in the first quarter, we built our entire commercial organization of really talented experienced rare disease experts who've been out talking with the specialists they've been able to engage.
Josh: With about 90% of prescribers also importantly, we've been engaged with the patient advocacy groups and the payer community and we now have 75% of covered lives.
Joshua M. Schafer: Also importantly, we've been engaged with patient advocacy groups and the payer community, and we now have 75% of covered lives. So all of that really bodes well for what we think will be some momentum as we build into the launch. It's also important to note that these patients, like many rare disease patients, really only go in to see their physicians every six to nine months. So, you know, we're pleased with the progress that we're seeing now, but, you know, we've got more to do, really not. Yeah, leave it at that.
Josh: So all of that really bodes well for.
Josh: But we think will be some momentum as we as we build into the launch.
Josh: Also important to note that these these patients like many rare disease patients really only go in to see their physicians every six to nine months.
Josh: So.
Josh: We're pleased with the progress that we're seeing now, but we've got more to do.
Josh: Yes.
Josh: Really not.
Josh: Leave it at that.
Adrian Quartel: I'll ask Adrian to see if he can talk a little bit about the KP-1077 differentiation. Yeah, thanks for that question.
Josh: I'll ask.
Josh: Adrian to see if you can talk a little bit about the KC $10 77 differentiation.
Adrian Quartel: Yeah, thanks for that question. Obviously, angiopathic hypersomnia is actually a fairly large patient population that constitutes it. So we're talking about 37,000 patients. There is still a significant unmet medical need in that large population. We believe that KP1077 provides a unique mode of action addressing the specific symptoms of angiopathic hypersomnia, so we believe we have a differentiated product, specifically when it comes to its PK profile, its mode of action, and it's a Schedule IV drug that has proven cardiovascular safety, so we believe that it will have its own unique space within the angiopathic hypersomnia population.
Adrian: Yes. Thanks for that question, so obviously idiopathic hypersomnia, the tenants actually fairly large patient populations.
Adrian: Yeah.
Adrian: So we're talking about 37000 patients there's still a significant unmet medical need in the logical inflation, we believe that KC $10 77.
Adrian: <unk> unique motive action addressing the specific symptoms often have some gap. So we believe we have a differentiated product specifically clinical PK profile is about infection, which is scheduled for a drug that has proven cardiovascular safety. So we believe that it will have its own <unk>.
Adrian: Face within the competitive Harrison.
Adrian: Yes.
Adrian: Okay.
Speaker Change: I think you also asked a little bit about the <unk> market opportunity if I understood it as well.
Adrian Quartel: I think you also asked a little bit about the solifolol market opportunity if I understood it as well. Yeah, um, ciliprolo.
Adrian Quartel: Yeah, and Ciliprolol is used for the treatment of VEDS, of which there are about 7,500 patients in the U.S. In some European countries, it is currently being used as a standard of care for those patients, but other than that, there really is no treatment option for these patients other than surgery. So we think that if approved, Ciliprolol would really offer some benefit for these patients who have no other available treatments.
Speaker Change: Yes.
Adrian: Philip below.
Adrian: Is used for the treatment of <unk> of which there are about 7500 patients in the U S.
Adrian: In some European countries. It is currently being used as the standard of care for those patients, but other than that there really is no treatment option for these patients other than surgery. So we think that if approved <unk> would really offer some benefit for these patients who have no other available therapies.
Speaker Change: Thank you.
Speaker Change: Thank you and once again, if you would like to ask a question. Please press star one on your telephone keypad.
Louise Alesandra Chen: Thank you. And once again, if you would like to ask a question, please press star one on your telephone keypad. You may remove yourself by pressing star two. Once again, that is star and one. We'll go next to Oren Livnat on HG Rainrat. Please go ahead.
Speaker Change: Remove yourself by pressing star to once again that is star and one well go next to on Loopnet with H C. Wainwright. Please go ahead.
Speaker Change: Thanks for taking the question I just wanted to follow up on all through so.
Oren Gabriel Livnat: Thanks for taking the question. I just want to follow up on Olpruva. So I know it's very early, and you've only had four patients enrolled that you've called out here, but can you talk about where those patients come from? Are those de novo patients or switchers from a less palatable product? And what experience are you having, you know, very early on here with trying to adjudicate on whether to reimburse therapy and with these initial patients? Are you getting any pushback regarding February pricing, relative pricing, and how fast do you think you can convert these? And then, hopefully, future enrolled patients to paid therapy? Thanks.
Speaker Change: I know, it's very early in <unk>.
Speaker Change: <unk> four patients enrolled that you've called out here, but.
Speaker Change: But can you talk about.
Speaker Change: Where those patients come from are those de novo patients or <unk>.
Richard: Richard from less palatable.
Richard: <unk>.
Richard: And what experience you were having very early on here with trying to adjudicate.
Richard: To reimbursed therapy with these initial patients are you getting any pushback regarding February pricing relative pricing and how fast do you think you can convert these and then hopefully future enrolled patients to paid therapy. Thanks.
Joshua M. Schafer: Thanks for the question, Art. First of all, it's important to note that the four patients that we reported were just those new enrollments for the quarter. There are more than that currently on Alprova or who have been prescribed Alprova, and these patients are coming from a variety of different places, either switching from current therapy. We also do have some patients who are new to therapy and have not received any treatment. Like all rare disease products, there are steps through and steps edits that take place in order for patients to receive treatments for their rare diseases.
Speaker Change: Thanks, Thanks for the question art.
Speaker Change: First of all it's important to note that the four patients that we reported.
Speaker Change: Just those new enrollments for the quarter.
Speaker Change: There are more than that currently on offer.
Speaker Change: Or who have been prescribed <unk>.
Speaker Change: And these patients are coming from a variety of different places either switches from current therapy.
Speaker Change: So do you have some patients who are new to therapy or not.
Speaker Change: We have not received any any treatment.
Speaker Change: Like all rare disease products. There there are step throughs and step edits that that take place in order to for patients to receive <unk>.
Speaker Change: Treatments for their rare diseases, but we've been really pleased with the progress that we're getting from our reimbursement in covered lives. We've got 75% covered lives again, reflecting back when we closed the transaction. It was at 55. So we've made great progress there so.
Joshua M. Schafer: But we've been really pleased with the progress that we're making on reimbursement and covered lives. We've got 75% covered lives. Again, looking back when we closed the transaction, it was at 55, so we've made great progress there. You know, I think we're, we feel like we're in a good place right now and on par to be able to compete with Severain and Rivitti.
Speaker Change: I think we feel like we're in a good place right now on.
Speaker Change: On par to be able to compete with <unk> and <unk>.
Speaker Change: And if I could just follow up on what you said there can you help us understand.
Oren Gabriel Livnat: If I could just follow up on what you said there. Can you help us understand, I guess, how many patients were already on therapy? Was that under the prior ACER launch? And are those paid subjects, or are they getting a free drug now, and you're hoping to convert them to therapy? Or is the reason that revenue is diminishing just that there are paid therapy patients now, but there were just already, before your time, so to speak, there was already enough supply in the channel. So you didn't need to shift. Yeah,
Speaker Change: I guess, how many patients who are already on therapy was that under the prior Acer launch and are those.
Speaker Change: Paid subjects or are they getting free drug now and youre, hoping to convert them to therapy or is the reason that revenue is de minimis just that they are paid therapy patients now, but theyre just already from before your time so to speak.
Speaker Change: Already in the supply and the channels actually you didn't need to ship yes.
Joshua M. Schafer: Yeah, so it's a little, it's a, it's kind of a combination of all the things that you just offer there. There were a handful of patients who had received Alprova and were enrolled prior to the close of the transaction. However, most of them came late in 2023 into the first quarter and now into the second quarter. And again, they're coming from a variety of different sources, whether those are switches or
Speaker Change: Yes, so it's a little it's kind of a combination of all the things that you just offered there.
Speaker Change: There were a handful of patients who had received.
Speaker Change: I'll provide more enrolled prior to the close of the transaction. However, most of them came late in 2023 into the first quarter and now into the second quarter.
Speaker Change: And again Theyre coming from a variety of different sources, whether those are <unk>.
Speaker Change: Which is or naive.
Speaker Change: And.
Speaker Change: And no im.
Joshua M. Schafer: And I'm sorry, I was just thinking about the rest of your question here, and those are a combination of both paid patients as well as those who have gone on to our Quick Start program, which allows us to get patients on to therapy while we're investigating their benefits and attempting to convert them to paid patients as well.
Speaker Change: Im sorry, I was just thinking about the rest of your question here and those are a combination of both paid patients as well as those who have gone onto our quick start program, which allows us to get patients onto therapy, while we're.
Speaker Change: Investigating their benefits and attempting to convert them to paid patients as well.
Speaker Change: Alright, Thanks, I'll follow up with you guys. Afterwards, Yeah go ahead.
Oren Gabriel Livnat: Alright, I'll follow up with you guys afterwards, yeah. Go ahead.
Joshua M. Schafer: Oh, I think there was a third part to your question, which was really around the de minimis revenue, and that it's important to note that there is a disconnect, as mentioned, between the revenue recognition and the enrollments due to the way that we still, I'll prove it to you, put it into our specialty pharmacies, so that it's kind of a reflection of just that dynamic.
Speaker Change: Well I think there was a third parts of your question, which was really around the de Minimis revenue and that it's important to note that there is a disconnect as Wayne mentioned between the revenue recognition and the enrollment due to the way that.
Speaker Change: That we sell.
Speaker Change: I'll prove it into our specialty pharmacies, so that is kind of a.
Speaker Change: A reflection of just that dynamic.
Speaker Change: Alright, and are you hearing anything regarding relative pricing of these drugs or is there are they all expensive enough and.
Oren Gabriel Livnat: All right, and are you hearing anything regarding the relative pricing of these drugs, or are they all? and presumably some unmet need with these patients, if they're even considering your product, that it's not an issue, the potentially slightly cheaper nature of a competing product.
Speaker Change: Presumably unmet need with these patients if they're even considering your products.
Speaker Change: It's not an issue.
Speaker Change: Potentially slightly cheaper nature of competing products.
Speaker Change: Yes so.
Joshua M. Schafer: Yeah, so as you probably know, we've seen a lot of payers begin to put Revicti on their exclusion lists as a result of Revicti's high price. We are benefiting from that, given that we have a significantly lower cost, but more importantly, we are able to compete on the clinical benefits of Olpruva, and so that dynamic is certainly helping us.
Speaker Change: As you probably know we've seen a lot of payers begin to put <unk> on exclusion list as a result of predict these high price.
Speaker Change: We are benefiting from that given that we have.
Speaker Change: A significantly less.
Speaker Change: A lower cost and more importantly that we're able to compete on the clinical benefits.
Speaker Change: Well proven.
Speaker Change: And so that dynamic is certainly helping helping us.
Speaker Change: Alright, thank you.
Sumant Satchidanand Kulkarni: Thank you we will take our next question from <unk> Kulkarni with Canaccord Genuity. Please go ahead.
Oren Gabriel Livnat: Thank you. We will take our next question from Sumant Kulkarni with Canaccord Genuity. Please go ahead.
Sumant Satchidanand Kulkarni: Hello, this is Kyle Chan speaking. This is Sumant. So, two questions. Error Monocle Mode, can you compare and contrast your approach with intra bios and the potential for you to have an adcom that is back to back with that product given the proximity of the two action dates? [inaudible] The second question is, have you had mock outcomes already on Aramakamal, and what are the potential findings and those that you guys have considered surprising or counterintuitive?
Sumant Satchidanand Kulkarni: Hello, This is Jan speak English.
Jan: So two questions on <unk> can you compare and contrast your approach.
Speaker Change: And goodbye.
Speaker Change: And so for you to have an AD com that is back to Pat.
Speaker Change: That part of that given the proximity of the <unk>.
Speaker Change: And.
Speaker Change: The second question.
Speaker Change: You had moth Alcon 30 on a remarkable what are.
Speaker Change: Bindings and those that might have considered you guys have considered surprising alright got it.
Speaker Change: Got it.
Speaker Change: Thanks, Kyle I'll take the last question and then I'll hand off the question to Adrian around.
Unknown Executive: Thanks, Kyle. I'll take the last question, and I'll hand off the question about differentiation to Adrian.
Speaker Change: <unk>, yes to answer your question, Yes, we are thoroughly preparing for a potential advisory committee.
Unknown Executive: To answer your question, yes, we are thoroughly preparing for a potential advisory committee. We've had a number of red team, blue team exercises, preparing briefing books, and multiple mock ad comms coming that we're learning from and perfecting our craft as we move forward. So I feel like we're thoroughly preparing for that at this point, in regards to their being a true product being assessed for nemo-PI
We've had a number of red team Blue team exercises preparing briefing books multiple add mark add comps coming that we're learning from and perfecting our craft as we move forward. So I feel like we're thoroughly preparing for that at this point.
Unknown Executive: Yes.
Adrian Quartel: Regarding there being two products being assessed for pneumopixy, it's great for patients. I think we should start with that. I think it's important to understand that erythromoxamol, with its unique mode of action, is really a disease-modifying drug, where the intrabiotic product is a more symptomatic treatment, addressing only certain symptoms of it. The differentiated mode of action and the specific clinical profile of Arimocrimol, we believe will lead it to be the foundational treatment for patients with hemopixy, and that's kind of what we're looking forward to.
Unknown Executive: In regards to there being two products being assessed for niemann pick C. It's great for patients I think let's start with that.
Adrian Quartel: I think it's important to understand that there are multiple.
Adrian Quartel: Unique motive action as we need a disease modifying disease, where the NDA byproducts, it's more symptomatic treatment.
Adrian Quartel: <unk> symptoms of the disease, the differentiated mode of action in the specific clinical profile.
Adrian Quartel: Our multimodal, we believe will lead to be the foundational treatment for patients with <unk> and Thats kind of where we're looking forward to.
Adrian Quartel: Okay.
Speaker Change: Alright. Thanks.
Speaker Change: Thank you Kyle.
Adrian Quartel: Alright. 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 and closing remarks.
Unknown Executive: All right, and 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, Ashley.
Neil F. McFarlane: Thank you Ashley.
Neil F. McFarlane: 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 catalysts in the second half of 2024, our strategic priorities are clear, and we look forward to updating you in the future. Thanks for joining us today. Have a wonderful day.
Neil F. McFarlane: We continue to make solid advances towards achieving our mission of building a leading patient focused rare disease Therapeutics company as.
Neil F. McFarlane: As we look to our catalyst in the second half of 2024, our strategic priorities are clear and we look forward to updating you in future in the future. Thanks for joining us today have a wonderful day.
Speaker Change: Thank you and this does conclude today's program you may disconnect at any time and have a wonderful day.
Unknown Executive: Thank you. This does conclude today's program. You may disconnect at any time and have a wonderful day.
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unknown: Okay.
unknown: [music].
unknown: Uh-huh.