Operator: Ladies and gentlemen, welcome to the Arrowhead Pharmaceuticals conference call. Throughout today's recorded presentation, all participants will be in a listen-only mode. After the presentation, there will be an opportunity to ask questions. I want to hand the conference over to Vince Anzalone, Vice President of Investor Relations for Arrowhead. Please go ahead, Vince.
Speaker #1: Ladies and gentlemen , welcome to the Arrowhead Pharmaceuticals conference . Call . Though today's presentation , all participants will be in a listen only mode .
Speaker #1: After the presentation, there will be an opportunity to ask questions. I will now hand the conference over to Vincent Anzalone, Vice President of Investor Relations for Arrowhead.
Vincent Anzalone: Thank you, Victor. Good afternoon, and thank you for joining us today to discuss Arrowhead's results for its fiscal 2026 Q1, ended December 31, 2025. With us today from management are President and CEO, Dr. Chris Anzalone, who will provide an overview, Andy Davis, Senior Vice President and Head of the Global Cardiometabolic Franchise, who will provide an update on commercialization activities, Dr. James Hamilton, Chief Medical Officer and Head of R&D, who will discuss our development programs, and Dan Apel, Chief Financial Officer, who will give a review of the financials. Following management's prepared remarks, we will open the call to questions.
Vince Anzalone: Thank you, Victor. Good afternoon, and thank you for joining us today to discuss Arrowhead's results for its fiscal 2026 Q1, ended December 31, 2025. With us today from management are President and CEO, Dr. Chris Anzalone, who will provide an overview, Andy Davis, Senior Vice President and Head of the Global Cardiometabolic Franchise, who will provide an update on commercialization activities, Dr. James Hamilton, Chief Medical Officer and Head of R&D, who will discuss our development programs, and Dan Apel, Chief Financial Officer, who will give a review of the financials. Following management's prepared remarks, we will open the call to questions.
Speaker #1: Please go ahead, Vince.
Speaker #2: Thank you . Victor . Good afternoon , and thank you for joining us today to discuss Arrowhead's results for its fiscal 2026 . First quarter ended December 31st , 2025 .
Speaker #2: With us today from management are President and CEO Dr. Christopher Anzalone, who will provide an overview, and Andy Davis, Senior Vice President and Head of the Global Cardiometabolic Franchise, who will provide an update on commercialization activities.
Speaker #2: Doctor James Hamilton , Chief Medical Officer and Head of R&D , who will discuss our development programs and Daniel Apel Chief Financial Officer , who will give over a review of the financials .
Vincent Anzalone: Before we begin, I would like to remind you that comments made during today's call contain certain forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. All statements other than statements of historical fact are forward-looking statements and are subject to numerous risks and uncertainties that could cause actual results to differ materially from those expressed in any forward-looking statements. For further details concerning these risks and uncertainties, please refer to our SEC filings, including our most recent annual report on Form 10-K and our quarterly reports on Form 10-Q. I'd now like to turn the call over to Chris Anzalone, President and CEO of the company. Chris?
Vince Anzalone: Before we begin, I would like to remind you that comments made during today's call contain certain forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. All statements other than statements of historical fact are forward-looking statements and are subject to numerous risks and uncertainties that could cause actual results to differ materially from those expressed in any forward-looking statements. For further details concerning these risks and uncertainties, please refer to our SEC filings, including our most recent annual report on Form 10-K and our quarterly reports on Form 10-Q. I'd now like to turn the call over to Chris Anzalone, President and CEO of the company. Chris?
Speaker #2: Following management's prepared remarks , we will open the call to questions . Before we begin , I would like to remind you that comments made during today's call contain certain forward looking statements within the meaning of section 27 of the Securities Act of 1933 and section 21 Securities of the Exchange Act of 1934 .
Speaker #2: All statements, other than statements of historical fact, are forward-looking statements and are subject to numerous risks and uncertainties that could cause actual results to differ materially from those expressed in any forward-looking statements.
Speaker #2: For further details concerning these risks and uncertainties , please refer to our SEC filings , including our most recent annual Report on Form 10-K and our quarterly Reports on Form 10-q .
Christopher Anzalone: Thanks, Vince. Good afternoon, everyone, and thank you for joining us today. We had another quarter of strong execution across all areas of our business, and we are well-positioned to build on this progress throughout 2026 and beyond. In fact, the recent months have included some of the more significant achievements in Arrowhead's history. Let's talk about some of these. First, on 18 November 2025, Arrowhead received its first regulatory approval and began the next phase of growth as a commercial company marketing its own medicines. The FDA approved Redemplo as an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome or FCS.
Chris Anzalone: Thanks, Vince. Good afternoon, everyone, and thank you for joining us today. We had another quarter of strong execution across all areas of our business, and we are well-positioned to build on this progress throughout 2026 and beyond. In fact, the recent months have included some of the more significant achievements in Arrowhead's history. Let's talk about some of these. First, on 18 November 2025, Arrowhead received its first regulatory approval and began the next phase of growth as a commercial company marketing its own medicines. The FDA approved Redemplo as an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome or FCS.
Speaker #2: I'd now like to turn the call over to Chris Anzalone , president and CEO of the company . Chris . Thanks , Vince .
Speaker #3: Good afternoon , everyone , and thank you for joining us today had another . We quarter of strong execution across all areas of our and we are business , well to build on this positioned progress throughout 2026 and beyond .
Speaker #3: In fact , the recent months have included some of the more significant achievements in Arrowhead's history . Let's talk about some of these .
Speaker #3: First , on November 18th , 2025 , Arrowhead received its first regulatory approval and began the next phase of growth as a commercial company , marketing its own medicines .
Speaker #3: The FDA approved as an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome , or FCS . FCS is a severe , rare disease with an estimated 6500 people in the US living with genetic or clinical FCS characterized by TG levels that can be 10 to 100 times higher than normal , leading to a substantially higher risk of developing acute , recurrent , and potentially fatal pancreatitis .
Christopher Anzalone: FCS is a severe, rare disease with an estimated 6,500 people in the US living with genetic or clinical FCS, characterized by TG levels that can be 10 to 100 times higher than normal, leading to a substantially higher risk of developing acute, recurrent, and potentially fatal pancreatitis. This approval was supported by clinical data from the phase 3 Palisade study in adults with either clinically diagnosed or genetically confirmed FCS. The Palisade study demonstrated deep and durable reductions in TGs, with a median reduction of 80% from baseline and a lower numerical incidence of acute pancreatitis events compared to placebo. Arrowhead launched Redemplo independently in the US with a one Redemplo pricing model that creates one consistent price across current and potential future indications. This is important.
Chris Anzalone: FCS is a severe, rare disease with an estimated 6,500 people in the US living with genetic or clinical FCS, characterized by TG levels that can be 10 to 100 times higher than normal, leading to a substantially higher risk of developing acute, recurrent, and potentially fatal pancreatitis. This approval was supported by clinical data from the phase 3 Palisade study in adults with either clinically diagnosed or genetically confirmed FCS. The Palisade study demonstrated deep and durable reductions in TGs, with a median reduction of 80% from baseline and a lower numerical incidence of acute pancreatitis events compared to placebo. Arrowhead launched Redemplo independently in the US with a one Redemplo pricing model that creates one consistent price across current and potential future indications. This is important.
Speaker #3: This approval was supported clinical data from the phase three by study in adults with either clinically diagnosed or genetically confirmed FCS . study demonstrated deep The and durable reductions in TGS with a with a median reduction of 80% from baseline in the lower numerical incidence of acute pancreatitis events compared to placebo .
Speaker #3: Arrowhead launched independently in the with the wonder low pricing model that creates one consistent price across current and potential future indications . This is important .
Christopher Anzalone: We're committed to sustainable innovation, and this requires a rational drug pricing according to the value a medicine offers to patients and healthcare systems. Redemplo is a pancreatitis drug, and when we think about pricing, we look to those patient populations at greatest risk of acute TG-related pancreatitis. We've only had drug in channel for about 10 weeks, which included Thanksgiving, Christmas, and New Year's holidays, so it is difficult to infer too much about launch. However, initial trends in prescriptions, payer interactions, and shipments have been encouraging. To date, over 100 prescriptions for Redemplo have been received from a diverse prescriber base with geographically balanced uptake across the US. Early patient starts fall into three categories: patients transitioning from our expanded access program, patients naive to the APOC3 class, and patients switching from Olezarsen.
Chris Anzalone: We're committed to sustainable innovation, and this requires a rational drug pricing according to the value a medicine offers to patients and healthcare systems. Redemplo is a pancreatitis drug, and when we think about pricing, we look to those patient populations at greatest risk of acute TG-related pancreatitis. We've only had drug in channel for about 10 weeks, which included Thanksgiving, Christmas, and New Year's holidays, so it is difficult to infer too much about launch. However, initial trends in prescriptions, payer interactions, and shipments have been encouraging. To date, over 100 prescriptions for Redemplo have been received from a diverse prescriber base with geographically balanced uptake across the US. Early patient starts fall into three categories: patients transitioning from our expanded access program, patients naive to the APOC3 class, and patients switching from Olezarsen.
Speaker #3: We're committed to sustainable innovation, and this requires a drug pricing approach according to the value medicine offers to patients and healthcare systems.
Speaker #3: It is a pancreatitis drug. And when we think about pricing, we look to those patient populations at greatest risk of acute HTG-related pancreatitis.
Speaker #3: We've only had drug and channel for about ten weeks, which included Thanksgiving, Christmas, and New Year's holidays. So it is difficult to infer too much about launch.
Speaker #3: However , initial trends in prescriptions payer interactions and shipments have been encouraging . To date , over 100 prescriptions for ejemplo have been received from a diverse prescriber base from with geographically balanced uptake across the US Early .
Speaker #3: patient starts fall into three categories patients transitioning from our expanded access program . Patients naive to the Apoc3 class , and patients switching from Olezarsen .
Christopher Anzalone: In addition, Redemplo shipments are being made for patients with clinically diagnosed and genetically confirmed FCS. In addition to FDA approval, we announced in January 2026 that Redemplo also received approval for the treatment of FCS from Health Canada and from the Chinese National Medical Products Administration. Redemplo will be available later this year in Canada, and we anticipate it will be marketed independently by Arrowhead. Pending regulatory review and approval, we expect to potentially launch Redemplo later this year in select EU countries, and in the UK. In Greater China, Redemplo will be marketed by Sanofi. Our cardiometabolic pipeline is off to a good start with Redemplo and the ongoing phase 3 study of zodasiran in homozygous familial hypercholesterolemia, or HFH. We are actively expanding this pipeline with a number of discovery programs and, importantly, three clinical programs.
Chris Anzalone: In addition, Redemplo shipments are being made for patients with clinically diagnosed and genetically confirmed FCS. In addition to FDA approval, we announced in January 2026 that Redemplo also received approval for the treatment of FCS from Health Canada and from the Chinese National Medical Products Administration. Redemplo will be available later this year in Canada, and we anticipate it will be marketed independently by Arrowhead. Pending regulatory review and approval, we expect to potentially launch Redemplo later this year in select EU countries, and in the UK. In Greater China, Redemplo will be marketed by Sanofi. Our cardiometabolic pipeline is off to a good start with Redemplo and the ongoing phase 3 study of zodasiran in homozygous familial hypercholesterolemia, or HFH. We are actively expanding this pipeline with a number of discovery programs and, importantly, three clinical programs.
Speaker #3: In addition, shipments are being made for patients with clinically diagnosed and genetically confirmed FCS. In addition to FDA approval, we announced in January 2026 that we also received approval for the treatment of FCS from Health Canada, and approval from the Chinese National Medical Products Administration will be available later this year in Canada.
Speaker #3: And we anticipate it will be marketed independently by Arrowhead, pending regulatory review and approval. We expect to launch later this year in select EU countries and in the UK.
Speaker #3: In Greater China, the region will be marketed by Sanofi. Our cardiometabolic pipeline is off to a good start with Exemplar and the ongoing Phase 3 study of Zoroaster in homozygous familial hypercholesterolemia, or HoFH.
Christopher Anzalone: ARO-INHBE and ARO-ALK7, being developed as potential treatments for obesity, are in phase 1 and 2 studies. We also recently initiated a phase 1/2 study of ARO-DIMER-PA in patients with mixed hyperlipidemia. For our initial obesity candidates, we recently announced some early interim clinical data. ARO-INHBE enhanced weight loss and fat reduction versus Tirzepatide alone in obese patients with type 2 diabetes... More specifically, 2 administrations of ARO-INHBE at the 400 mg dose in combination with Tirzepatide achieved approximately twofold better weight loss at week 16 than Tirzepatide alone. This appears to be high-quality weight loss, as we saw an approximately threefold reduction in each of total fat, visceral fat, and liver fat measures based on week 12 MRI versus Tirzepatide alone in these patients. The ARO-ALK7 phase 1/2 study is approximately 2 quarters behind the ARO-INHBE study, but early data are encouraging.
Chris Anzalone: ARO-INHBE and ARO-ALK7, being developed as potential treatments for obesity, are in phase 1 and 2 studies. We also recently initiated a phase 1/2 study of ARO-DIMER-PA in patients with mixed hyperlipidemia. For our initial obesity candidates, we recently announced some early interim clinical data. ARO-INHBE enhanced weight loss and fat reduction versus Tirzepatide alone in obese patients with type 2 diabetes... More specifically, 2 administrations of ARO-INHBE at the 400 mg dose in combination with Tirzepatide achieved approximately twofold better weight loss at week 16 than Tirzepatide alone. This appears to be high-quality weight loss, as we saw an approximately threefold reduction in each of total fat, visceral fat, and liver fat measures based on week 12 MRI versus Tirzepatide alone in these patients. The ARO-ALK7 phase 1/2 study is approximately 2 quarters behind the ARO-INHBE study, but early data are encouraging.
Speaker #3: We are actively expanding pipeline with this a number of discovery importantly , three clinical programs and programs arrow and Arrow . Alk7 being developed as potential treatments for obesity are in phase one two studies .
Speaker #3: We also recently initiated a phase one two study of aero dimer in patients with mixed hyperlipidemia . For our initial obesity candidates , we recently announced some early interim clinical data Iroin HHB , enhanced weight loss and fat reduction versus Tirzepatide alone in obese patients with type two diabetes .
Speaker #3: More specifically , two administrations of oral ifnb at the 400mg dose in combination with tirzepatide achieved approximately two fold better weight loss at week 16 than Tirzepatide alone .
Speaker #3: This appears to be high quality weight loss , as we saw an approximately three fold reduction in each of total fat , visceral fat , and liver fat measures .
Speaker #3: Based on week 12 MRI versus Tirzepatide alone in these patients , the is phase one two study approximately two quarters behind the Inhbb study , but early data are encouraging .
Christopher Anzalone: We believe this is the first RNAi therapeutic to show adipocyte gene target silencing in a clinical trial, and we've seen dose-dependent reductions in adipose ALK7 mRNA, with a mean reduction of -88% at the 200 mg dose at week 8, and a maximum reduction of -94%. While these are very intriguing data, they are early and incomplete, so we have substantial work ahead of us before we get too excited about how these candidates could eventually be used. We will continue to run both phase 1 and 2 studies. We are expanding existing cohorts to increase power, and we are adding new cohorts to better understand these candidates and underlying biology. We intend to report additional results later in 2026.
Chris Anzalone: We believe this is the first RNAi therapeutic to show adipocyte gene target silencing in a clinical trial, and we've seen dose-dependent reductions in adipose ALK7 mRNA, with a mean reduction of -88% at the 200 mg dose at week 8, and a maximum reduction of -94%. While these are very intriguing data, they are early and incomplete, so we have substantial work ahead of us before we get too excited about how these candidates could eventually be used. We will continue to run both phase 1 and 2 studies. We are expanding existing cohorts to increase power, and we are adding new cohorts to better understand these candidates and underlying biology. We intend to report additional results later in 2026.
Speaker #3: We believe this is the first RNAi therapeutic to show adipocyte gene target silencing in a clinical trial, and we've seen dose-dependent reductions in adipose ALK7 mRNA, with a mean reduction of 88% at the 200 mg dose at week eight, and a maximum reduction of 94%.
Speaker #3: While these are very intriguing data, they are early and incomplete, so we have substantial work ahead of us before we get too excited about how these candidates could eventually be used.
Speaker #3: We will continue to run both Phase 1 and 2 studies. We are expanding existing cohorts to increase power, and we are adding new cohorts to better understand these candidates and underlying biology.
Christopher Anzalone: ARO-DIMER-PA is being developed as a potential treatment for atherosclerotic cardiovascular disease, or ASCVD, due to mixed hyperlipidemia, where both LDL cholesterol and triglycerides are elevated. We believe there are approximately 20 million people in the US with mixed hyperlipidemia, and this is a patient population without adequate treatment options. We recently announced that we dosed the first patients in the phase 1/2 clinical trial of ARO-DIMER-PA, which is a dual functional RNAi therapeutic designed to silence expression of the PCSK9 and APOC3 genes, thus designed to reduce both LDL cholesterol and TGs. This represents an important step forward for the RNAi field, as we believe it is the first clinical candidate to target two genes simultaneously in one molecule, and an important step forward for preventative cardiology, as both LDL and TGs have epidemiologic support as being important drivers for ASCVD risk.
Chris Anzalone: ARO-DIMER-PA is being developed as a potential treatment for atherosclerotic cardiovascular disease, or ASCVD, due to mixed hyperlipidemia, where both LDL cholesterol and triglycerides are elevated. We believe there are approximately 20 million people in the US with mixed hyperlipidemia, and this is a patient population without adequate treatment options. We recently announced that we dosed the first patients in the phase 1/2 clinical trial of ARO-DIMER-PA, which is a dual functional RNAi therapeutic designed to silence expression of the PCSK9 and APOC3 genes, thus designed to reduce both LDL cholesterol and TGs. This represents an important step forward for the RNAi field, as we believe it is the first clinical candidate to target two genes simultaneously in one molecule, and an important step forward for preventative cardiology, as both LDL and TGs have epidemiologic support as being important drivers for ASCVD risk.
Speaker #3: In ten, report additional results later in 2026. Aradigm, or PA, is being developed as a potential treatment for atherosclerotic cardiovascular disease, or ASCVD, due to mixed hyperlipidemia, where both LDL cholesterol and triglycerides are elevated.
Speaker #3: We believe there are approximately 20 million people in the US with mixed hyperlipidemia , and this is a patient population without adequate treatment options .
Speaker #3: We recently announced that we dosed the first patients in the phase one two clinical trial of aero , or PA , which is a dual functional RNAi therapeutic designed to silence expression of the Pcsk9 and Apoc3 genes .
Speaker #3: Thus designed to reduce both LDL cholesterol and TGS . This represents an important step forward for the RNAi field , as we believe it is the first clinical candidate to target two genes simultaneously in one molecule , and an important step forward for preventive cardiology .
Christopher Anzalone: We expect to have interim data for ARO-DIMER-PA in the second half of 2026. If we see good LDL and TG reduction in a well-tolerated manner, we may have something truly special for a very large and currently underserved patient population. Outside of cardiometabolic, we made important advances in our CNS portfolio, specifically in programs that utilize a new proprietary delivery system designed to achieve blood-brain barrier, or BBB, penetration, utilizing subcutaneous administration. In non-clinical studies across multiple animal models, we saw a deep target gene knockdown across the CNS, including deep brain regions. This underscores Arrowhead's leadership in the delivery of siRNA to multiple tissues and cell types throughout the body, utilizing the proprietary TRIM platform. Our first wholly-owned program using the BBB platform is ARO-MAPT, being developed as a potential treatment for tauopathies, including Alzheimer's disease.
Chris Anzalone: We expect to have interim data for ARO-DIMER-PA in the second half of 2026. If we see good LDL and TG reduction in a well-tolerated manner, we may have something truly special for a very large and currently underserved patient population. Outside of cardiometabolic, we made important advances in our CNS portfolio, specifically in programs that utilize a new proprietary delivery system designed to achieve blood-brain barrier, or BBB, penetration, utilizing subcutaneous administration. In non-clinical studies across multiple animal models, we saw a deep target gene knockdown across the CNS, including deep brain regions. This underscores Arrowhead's leadership in the delivery of siRNA to multiple tissues and cell types throughout the body, utilizing the proprietary TRIM platform. Our first wholly-owned program using the BBB platform is ARO-MAPT, being developed as a potential treatment for tauopathies, including Alzheimer's disease.
Speaker #3: As both LDL and TGS have epidemiologic support as being important drivers for ascvd risk . We expect to have interim data for PA in the second half of 2026 .
Speaker #3: If we see good LDL and TG reduction in a well-tolerated manner , we may have something truly special for a very large and currently underserved patient outside of population cardiometabolic .
Speaker #3: made important We advances in our CNS portfolio , specifically in programs that utilize a new proprietary delivery system designed to achieve blood brain barrier or bbv penetration .
Speaker #3: Utilizing subcutaneous administration in non-clinical studies across multiple animal models , we saw deep targeted gene knockdown across the CNS , including deep brain regions .
Speaker #3: This underscores leadership in the delivery of siRNA to multiple tissues and cell types throughout the body . Utilizing the proprietary trading platform . Our first wholly owned program using the platform is Arrow , being developed as a potential treatment for Tauopathies , including Alzheimer's disease .
Christopher Anzalone: During the last quarter, we announced that we dosed the first subjects in a phase 1/2 clinical trial that will include healthy volunteers and Alzheimer's patients. ARO-MAPT targets the tau protein in the brain, which has good biological validation as a potential driver of pathology and has emerged as a promising target for Alzheimer's disease and additional tauopathies. We anticipate interim clinical data from the healthy volunteer portion of the study, should be available in 2026, with data from the Alzheimer's patients to follow in 2027. This is a very exciting program for us. The second program to use our BBB delivery system is SRP-1005, formerly called ARO-HTT, for the treatment of Huntington's disease. This program is partnered with Sarepta, which recently announced the submission of its CTA for study SRP-1005-101, also known as INSIGHT, in approximately 24 participants.
Chris Anzalone: During the last quarter, we announced that we dosed the first subjects in a phase 1/2 clinical trial that will include healthy volunteers and Alzheimer's patients. ARO-MAPT targets the tau protein in the brain, which has good biological validation as a potential driver of pathology and has emerged as a promising target for Alzheimer's disease and additional tauopathies. We anticipate interim clinical data from the healthy volunteer portion of the study, should be available in 2026, with data from the Alzheimer's patients to follow in 2027. This is a very exciting program for us. The second program to use our BBB delivery system is SRP-1005, formerly called ARO-HTT, for the treatment of Huntington's disease. This program is partnered with Sarepta, which recently announced the submission of its CTA for study SRP-1005-101, also known as INSIGHT, in approximately 24 participants.
Speaker #3: During the last quarter, we announced that we dosed the first subjects in Phase 1 in a two-part clinical trial that will include healthy volunteers and Alzheimer's patients.
Speaker #3: Targets the T tau protein in the brain, which has good biological validation as a potential driver of pathology and has emerged as a promising target for Alzheimer's disease and additional tauopathies.
Speaker #3: We anticipate interim clinical data from the healthy volunteer portion of the study should be available in 2026 , with data from the Alzheimer's patients to follow in 2027 .
Speaker #3: This is a very exciting program for us . The second program to use our delivery system is SRP 1005 , formerly called arrow , for the treatment of Huntington's disease .
Speaker #3: This program is partnered with Sarepta , which recently announced the submission of its CTA for study . SRP 1005 101 , also known as Incyte , in approximately 24 participants .
Christopher Anzalone: While our cardiometabolic and CNS work by no means encompasses everything we are doing, they are areas of substantial focus and potential value drivers in the near, mid, and long term. Within these areas, we are addressing three of the greatest public health challenges of our time: obesity, cardiovascular disease, and neurodegenerative conditions. Now I'd like to move on to some key events during the recent period that have dramatically strengthened our balance sheet and give us the necessary resources to push multiple programs toward commercialization. We anticipate being funded through multiple potential independent and partner launches. These meaningfully increase revenue opportunities for the company and push us toward becoming cash flow positive and self-sustaining from commercial sales. Since our last reporting period, we have completed transactions with gross proceeds of $1.33 billion. Let's break that down.
Chris Anzalone: While our cardiometabolic and CNS work by no means encompasses everything we are doing, they are areas of substantial focus and potential value drivers in the near, mid, and long term. Within these areas, we are addressing three of the greatest public health challenges of our time: obesity, cardiovascular disease, and neurodegenerative conditions. Now I'd like to move on to some key events during the recent period that have dramatically strengthened our balance sheet and give us the necessary resources to push multiple programs toward commercialization. We anticipate being funded through multiple potential independent and partner launches. These meaningfully increase revenue opportunities for the company and push us toward becoming cash flow positive and self-sustaining from commercial sales. Since our last reporting period, we have completed transactions with gross proceeds of $1.33 billion. Let's break that down.
Speaker #3: While our cardiometabolic and CNS work by no means encompasses everything we are doing , there are areas of substantial focus and potential value drivers in the near , mid and long term .
Speaker #3: Within these areas , we are addressing three of the greatest public health challenges of our time obesity , cardiovascular disease , and neurodegenerative conditions .
Speaker #3: Now, I’d like to move on to some key events during the recent period that have dramatically strengthened our balance sheet and give us the necessary resources to push multiple programs toward commercialization.
Speaker #3: We anticipate being funded through multiple potential independent and partner launches . These meaningfully , these meaningfully increase revenue opportunities for the company and push us toward becoming cash flow positive and self-sustaining from commercial sales .
Speaker #3: Since our last reporting period , we have completed transactions with gross proceeds of $1.33 billion . Let's break that down . First , we completed a global licensing and collaboration agreement with Novartis for arrow Snca arrowheads preclinical stage siRNA therapy against alpha synuclein for the treatment of synucleinopathies such as Parkinson's disease .
Christopher Anzalone: First, we completed a global licensing and collaboration agreement with Novartis for ARO-SNCA, Arrowhead's preclinical-stage siRNA therapy against alpha-synuclein for the treatment of synucleinopathies such as Parkinson's disease. The collaboration includes a limited number of additional targets outside our pipeline that will utilize Arrowhead's proprietary TRIM platform. Arrowhead received a $200 million upfront payment and is also eligible to receive development, regulatory, and sales milestone payments of up to $2 billion. Arrowhead is further eligible to receive tiered royalties on commercial sales up to low double digits. Second, we earned a $200 million milestone payment from Sarepta following a drug safety community review and subsequent authorization to dose escalate and achievement of the second pre-specified patient enrollment target for ARO-DM1.
Chris Anzalone: First, we completed a global licensing and collaboration agreement with Novartis for ARO-SNCA, Arrowhead's preclinical-stage siRNA therapy against alpha-synuclein for the treatment of synucleinopathies such as Parkinson's disease. The collaboration includes a limited number of additional targets outside our pipeline that will utilize Arrowhead's proprietary TRIM platform. Arrowhead received a $200 million upfront payment and is also eligible to receive development, regulatory, and sales milestone payments of up to $2 billion. Arrowhead is further eligible to receive tiered royalties on commercial sales up to low double digits. Second, we earned a $200 million milestone payment from Sarepta following a drug safety community review and subsequent authorization to dose escalate and achievement of the second pre-specified patient enrollment target for ARO-DM1.
Speaker #3: The collaboration includes a limited number of additional targets outside our pipeline that will utilize arrows proprietary trim platform . Arrowhead received a $200 million upfront payment and is also eligible to receive development , regulatory and sales milestone payments of up to $2 billion .
Speaker #3: The collaboration includes a limited number of additional targets outside our pipeline that will utilize arrows proprietary trim platform . Arrowhead received a $200 million upfront payment and is also eligible to receive development , regulatory and sales milestone payments of up Arrowhead is further eligible to receive tiered royalties on commercial sales up to low double digits .
Speaker #3: Second , we earned $200 million milestone payment from Sarepta following a drug safety Committee review and subsequent authorization to dose escalate and achievement of the second pre-specified patient target for arrow Dm1 enrollment .
Christopher Anzalone: And third, we closed concurrent public offerings of $700 million aggregate principal amount of 0% coupon convertible senior notes and $230 million of common stock. Both offerings were several times oversubscribed and priced at company-friendly terms.
Chris Anzalone: And third, we closed concurrent public offerings of $700 million aggregate principal amount of 0% coupon convertible senior notes and $230 million of common stock. Both offerings were several times oversubscribed and priced at company-friendly terms.
Speaker #3: Third, we closed concurrent public offerings of $700 million aggregate principal amount of 0% coupon convertible senior notes, and $230 million of common stock.
James Hamilton: ... As I mentioned at the beginning of the call, we demonstrated strong execution across all areas of our business. We received regulatory approval in three different countries. We launched our first commercial product. We continued to grow our cardiometabolic portfolio. We had encouraging early results from our obesity programs. We advanced our TRIM Platform and CNS pipeline, and we meaningfully improved our financial position to push these and other programs forward. It has been a productive last few months at Arrowhead, with so much potential to continue this strong progress in 2026 and beyond. With that overview, I'd now like to turn the call over to Andy Davis. Andy?
Chris Anzalone: ... As I mentioned at the beginning of the call, we demonstrated strong execution across all areas of our business. We received regulatory approval in three different countries. We launched our first commercial product. We continued to grow our cardiometabolic portfolio. We had encouraging early results from our obesity programs. We advanced our TRIM Platform and CNS pipeline, and we meaningfully improved our financial position to push these and other programs forward. It has been a productive last few months at Arrowhead, with so much potential to continue this strong progress in 2026 and beyond. With that overview, I'd now like to turn the call over to Andy Davis. Andy?
Speaker #3: Both offerings were several times oversubscribed and priced at company-friendly terms. As I mentioned at the beginning of the call, we demonstrated strong execution across all areas of our business and received regulatory.
Speaker #3: approval in three different We countries . We first commercial product . We continued to grow our cardiometabolic portfolio . encouraging We had early results from our obesity programs .
Speaker #3: We advanced our CNS pipeline and platform, and we meaningfully improved our financials. We pushed these and other programs forward to position them for success. It has been a productive last few months at Arrowhead, with so much potential to continue this strong progress.
Andy Davis: Thank you, Chris, and good afternoon, everyone. It has been just over two months since the approval of Redemplo on 18 November 2025, and we are very pleased with the progress we are seeing. I'd like to share some early insights across healthcare provider engagement, patient dynamics, and payer developments. I'll start with healthcare provider engagement. As a reminder, we are targeting approximately 5,000 healthcare professionals through personal promotion, complemented by a much broader omni-channel effort. Early prescribing has been led by preventive cardiologists and endocrinologists, who together account for approximately 70% of total prescriptions, with the remainder coming from internal medicine physicians focused on lipid disorders. In addition, advanced practice providers, including nurse practitioners and physician associates, working within multidisciplinary care teams, are playing a meaningful role in patient identification and treatment decisions. Turning to patient dynamics.
Andy Davis: Thank you, Chris, and good afternoon, everyone. It has been just over two months since the approval of Redemplo on 18 November 2025, and we are very pleased with the progress we are seeing. I'd like to share some early insights across healthcare provider engagement, patient dynamics, and payer developments. I'll start with healthcare provider engagement. As a reminder, we are targeting approximately 5,000 healthcare professionals through personal promotion, complemented by a much broader omni-channel effort. Early prescribing has been led by preventive cardiologists and endocrinologists, who together account for approximately 70% of total prescriptions, with the remainder coming from internal medicine physicians focused on lipid disorders. In addition, advanced practice providers, including nurse practitioners and physician associates, working within multidisciplinary care teams, are playing a meaningful role in patient identification and treatment decisions. Turning to patient dynamics.
Speaker #3: In 2026 and beyond . With that overview , I'd now like to turn the call over to Andy Davis Andy . Thank you , Chris , and good afternoon , everyone .
Speaker #3: It has been just over two months since the approval of in November 18th , 2025 and we are very pleased with the progress we are seeing .
Speaker #3: I'd like to share some early insights across healthcare provider engagement , patient dynamics and payer developments . I'll start with healthcare provider . As a engagement reminder , we are targeting approximately 5000 healthcare professionals through personal promotion , complemented by a much broader omnichannel effort .
Speaker #3: Early prescribing has been led by preventive cardiologists and endocrinologists, who together account for approximately 70% of total prescriptions, with the remainder coming from internal medicine.
Speaker #3: Focused on physicians treating lipid disorders. In addition, advanced practice providers, including nurse practitioners and physician associates working within multidisciplinary care teams, are playing a meaningful role in patient identification and treatment decisions.
Andy Davis: As Chris mentioned, over 100 prescriptions for Redemplo have been received to date. We see this as a very strong start that exceeded our expectations for the early months of the launch. We are also seeing geographically balanced uptake across the United States. Early patient starts fall into three categories: patients transitioning from our expanded access program, patients naive to the APOC3 class, and patients switching from Olezarsen. Class-naive patients represent the overwhelming majority of starts, with expanded access and switch patients contributing evenly to the remainder. Patients receiving Redemplo include both clinically diagnosed and genetically confirmed FCS, with the majority not required to submit genetic testing to gain access. Importantly, a high proportion of patients are enrolling in the Rely on Redemplo patient support program, and in the fiscal Q1, patients eligible for co-pay assistance paid $0 out of pocket. Next, I'll touch on payer developments.
Andy Davis: As Chris mentioned, over 100 prescriptions for Redemplo have been received to date. We see this as a very strong start that exceeded our expectations for the early months of the launch. We are also seeing geographically balanced uptake across the United States. Early patient starts fall into three categories: patients transitioning from our expanded access program, patients naive to the APOC3 class, and patients switching from Olezarsen. Class-naive patients represent the overwhelming majority of starts, with expanded access and switch patients contributing evenly to the remainder. Patients receiving Redemplo include both clinically diagnosed and genetically confirmed FCS, with the majority not required to submit genetic testing to gain access. Importantly, a high proportion of patients are enrolling in the Rely on Redemplo patient support program, and in the fiscal Q1, patients eligible for co-pay assistance paid $0 out of pocket. Next, I'll touch on payer developments.
Speaker #3: Turning to patient dynamics, as Chris mentioned, over 100 prescriptions have been received to date. We see this as a very strong start that exceeded our expectations for the early months of the launch.
Speaker #3: We are also seeing launch geographically balanced uptake across the United States . Early patient starts fall into three categories patients transitioning from our expanded access program , patients naive to the ACO , C3 class and patients switching from Olezarsen class naive patients represent the overwhelming majority of starts that expanded access and switched patients , contributing evenly to the remainder .
Speaker #3: Patients receiving redemptio include both clinically diagnosed and genetically confirmed FXS , with not required to the testing genetic submit majority to gain access .
Speaker #3: Importantly, a high proportion of patients are enrolling in the ReLY on patient support program, and in the fiscal first quarter, patients eligible for co-pay assistance paid $0 out of pocket.
Andy Davis: While it is still early, we remain encouraged by positive payer feedback on both the clinical profile of Redemplo and our unified One Redemplo pricing approach. We are actively engaged with the largest payers, and discussions to date reflect a willingness to cover Redemplo to label, including access based on either genetic or clinical diagnosis of FCS. I'd like to conclude with a brief comment on execution. Within days of FDA approval, we had product available in the channel for FCS patients. Our Redemplo care coordinators, rare disease specialists, and field reimbursement navigators were deployed on day one to support prescribers and patients, and our payer account team continues to work closely with customers to minimize access barriers. The teams are off to a great start, and our teams are highly encouraged by early stakeholder feedback. This feedback further reinforces the key differentiating attributes of Redemplo.
Andy Davis: While it is still early, we remain encouraged by positive payer feedback on both the clinical profile of Redemplo and our unified One Redemplo pricing approach. We are actively engaged with the largest payers, and discussions to date reflect a willingness to cover Redemplo to label, including access based on either genetic or clinical diagnosis of FCS. I'd like to conclude with a brief comment on execution. Within days of FDA approval, we had product available in the channel for FCS patients. Our Redemplo care coordinators, rare disease specialists, and field reimbursement navigators were deployed on day one to support prescribers and patients, and our payer account team continues to work closely with customers to minimize access barriers. The teams are off to a great start, and our teams are highly encouraged by early stakeholder feedback. This feedback further reinforces the key differentiating attributes of Redemplo.
Speaker #3: Next, I'll touch on payer development. While it is still early, we remain encouraged by positive payer feedback on both the clinical profile of, and our unified one.
Speaker #3: approach . Pricing We are actively engaged with the largest payers in discussions to date , reflect a willingness to cover to label , including access based on either genetic or clinical diagnosis of FXS .
Speaker #3: I'd like to conclude with a brief comment on execution . Within days of FDA approval , we had product available in the channel for FXS patients .
Speaker #3: Our care coordinators , Rare Disease specialists , and field Reimbursement Navigators were deployed on day prescribers and one to support patients , and our payer account team continues to work closely with customers to minimize access barriers .
Speaker #3: The teams are off to a great start, and our teams are highly encouraged by early stakeholder feedback. This feedback further reinforces the key differentiating attributes of Redemptio.
Andy Davis: As a reminder, in the PALISADE study, Redemplo reduced triglycerides by 80% from baseline as early as month 1 and maintained this reduction with minimal variability through 12 months of treatment. In addition, the numerical incidence of acute pancreatitis was lower in Redemplo-treated patients than in placebo. The US-approved prescribing information includes no contraindications, no warnings, and no precautions. Redemplo can be self-administered at home once every 3 months, just 4 injections per year. With that, I'll turn the call over to James Hamilton to discuss the R&D portfolio.
Andy Davis: As a reminder, in the PALISADE study, Redemplo reduced triglycerides by 80% from baseline as early as month 1 and maintained this reduction with minimal variability through 12 months of treatment. In addition, the numerical incidence of acute pancreatitis was lower in Redemplo-treated patients than in placebo. The US-approved prescribing information includes no contraindications, no warnings, and no precautions. Redemplo can be self-administered at home once every 3 months, just 4 injections per year. With that, I'll turn the call over to James Hamilton to discuss the R&D portfolio.
Speaker #3: A reminder, in the ASPEN Palisade study, reduced triglycerides by 80% from baseline as early as month one and maintained this reduction with minimal variability through 12 months of treatment.
Speaker #3: In addition , the numerical incidence of acute pancreatitis was lower in treated patients than in placebo , and the US approved prescribing information includes no contraindications , no warnings , and no precautions , and can be self-administered at home once every three months .
James Hamilton: Thank you, Andy. I'd like to start with a review of the Redemplo FDA approval and information in the label and contained in the package insert. Redemplo is approved as an adjunct to diet to reduce triglycerides in adults with FCS. The recommended dose of Redemplo is 25 mg, and it can be self-administered at home by subcutaneous injection once every 3 months. Redemplo has no contraindications, warnings, or precautions in the US FDA-approved label. The most common adverse reaction includes hyperglycemia, headache, nausea, and injection site reactions. Redemplo was studied in patients with both genetic FCS and clinically diagnosed FCS in the phase 3 PALISADE study. Patients achieved deep and durable reductions in median triglycerides of around 80% from baseline, with reductions largely maintained below the guideline-directed threshold of 500 mg/dL throughout the year of treatment.
James Hamilton: Thank you, Andy. I'd like to start with a review of the Redemplo FDA approval and information in the label and contained in the package insert. Redemplo is approved as an adjunct to diet to reduce triglycerides in adults with FCS. The recommended dose of Redemplo is 25 mg, and it can be self-administered at home by subcutaneous injection once every 3 months. Redemplo has no contraindications, warnings, or precautions in the US FDA-approved label. The most common adverse reaction includes hyperglycemia, headache, nausea, and injection site reactions. Redemplo was studied in patients with both genetic FCS and clinically diagnosed FCS in the phase 3 PALISADE study. Patients achieved deep and durable reductions in median triglycerides of around 80% from baseline, with reductions largely maintained below the guideline-directed threshold of 500 mg/dL throughout the year of treatment.
Speaker #3: injections per year . With that , I'll Just four turn the call over to James Hamilton to discuss the R&D portfolio . Thank you Andy .
Speaker #3: I'd like to start with a review of the FDA approval and information in the label , and contained in the package for insert is approved as an adjunct to to diet reduce triglycerides in adults with FCS .
Speaker #3: The recommended dose of is 25mg , and it can be self-administered at home by subcutaneous injection . Once every three months , has no contraindications , warnings , or precautions .
Speaker #3: In the US , FDA approved label , the most common adverse reaction include hypoglycemia , headache , nausea , and injection site reactions was studied in patients with both genetic and FCS clinically diagnosed FCS .
Speaker #3: In the phase three PALISADE study, patients achieved deep and durable reductions in median triglycerides of around 80% from baseline, with reductions largely maintained below the guideline-directed threshold of 500 mg per deciliter throughout the year of treatment.
James Hamilton: Importantly, patients with genetic FCS versus clinical FCS showed similar reductions from baseline. We see the clinical FCS population as having the same high unmet need as the genetic FCS group, and as such, we think it's crucial to have shown that both patient populations showed similar large reduction from baseline in triglycerides. In PALISADE, treated patients also had a reduced rate of adjudicated acute pancreatitis events, a very welcome finding for FCS patients and their caregivers, and an important validation that reduction in triglycerides can in fact lead to reductions in pancreatitis. In addition to FCS, we are also investigating plozasiran in patients with severe hypertriglyceridemia, or SHTG.... We announced last quarter that the FDA granted breakthrough therapy designation to investigational plozasiran as an adjunct to diet to reduce triglycerides in adults with SHTG.
James Hamilton: Importantly, patients with genetic FCS versus clinical FCS showed similar reductions from baseline. We see the clinical FCS population as having the same high unmet need as the genetic FCS group, and as such, we think it's crucial to have shown that both patient populations showed similar large reduction from baseline in triglycerides. In PALISADE, treated patients also had a reduced rate of adjudicated acute pancreatitis events, a very welcome finding for FCS patients and their caregivers, and an important validation that reduction in triglycerides can in fact lead to reductions in pancreatitis. In addition to FCS, we are also investigating plozasiran in patients with severe hypertriglyceridemia, or SHTG.... We announced last quarter that the FDA granted breakthrough therapy designation to investigational plozasiran as an adjunct to diet to reduce triglycerides in adults with SHTG.
Speaker #3: Importantly , patients with FCS genetic versus clinical FCS showed similar reductions from baseline . see the We clinical FCS population as having same high the unmet need as the genetic FCS group , and as such , we think it's crucial to have to have shown that both patient populations should similar large reductions from baseline in triglycerides .
Speaker #3: In palisade treated patients also had a reduced rate of adjudicated acute pancreatitis events . A very welcome finding for FCS patients and their caregivers , and an important validation that reduction in triglycerides can , in fact , lead to reductions in pancreatitis .
Speaker #3: In addition to FCS , we are also investigating in patients with severe hypertriglyceridemia or htg . We announced last quarter that the FDA granted breakthrough therapy designation to investigational aceron as an adjunct to diet to reduce triglycerides in adults with StG breakthrough therapy designation is a process designed to expedite the development and review of drugs that are intended to treat a serious condition and where preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapies on clinically significant endpoints .
James Hamilton: Breakthrough therapy designation is a process designed to expedite the development and review of drugs that are intended to treat a serious condition, and where preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapies on clinically significant endpoints. This is another important step for the program. The global phase 3 studies of plozasiran designed to support the supplemental NDA filing to expand the label beyond genetic and clinical FCS are the Shasta-III and Shasta-IV studies, which enrolled approximately 750 patients, and NEAR-3, which enrolled 1,400 patients. We're also enrolling patients in Shasta-V to directly assess the ability of plozasiran to reduce the risk of acute pancreatitis as the primary endpoint. We remain on schedule to complete the blinded portion of the Shasta-III, Shasta-IV, and NEAR-3 phase 3 clinical studies in mid-2026.
James Hamilton: Breakthrough therapy designation is a process designed to expedite the development and review of drugs that are intended to treat a serious condition, and where preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapies on clinically significant endpoints. This is another important step for the program. The global phase 3 studies of plozasiran designed to support the supplemental NDA filing to expand the label beyond genetic and clinical FCS are the Shasta-III and Shasta-IV studies, which enrolled approximately 750 patients, and NEAR-3, which enrolled 1,400 patients. We're also enrolling patients in Shasta-V to directly assess the ability of plozasiran to reduce the risk of acute pancreatitis as the primary endpoint. We remain on schedule to complete the blinded portion of the Shasta-III, Shasta-IV, and NEAR-3 phase 3 clinical studies in mid-2026.
Speaker #3: This is another important step for the program . The global phase three studies of plus and designed to support the supplemental NDA filing to expand the label beyond genetic and clinical FCS by the Shasta three and Shasta four studies , which enrolled approximately 750 patients , and near three , which enrolled 1400 patients .
Speaker #3: We are also enrolling patients in Shasta, five to directly assess the ability of Azaserine to reduce the risk of acute pancreatitis as the primary endpoint.
Speaker #3: We remain on schedule to complete the blinded portion of the Shasta three Shasta four , year three . and Phase three clinical studies .
James Hamilton: We expect top-line data to be available in Q3 2026, with planned sNDA submission for SHTG before the end of the year. We presented the study design and baseline characteristics of the Shasta-III and Shasta-IV studies at the 23rd World Congress Insulin Resistance, Diabetes, and Cardiovascular Disease in December 2025. I'd like to spend a moment to go over a few key parts of that poster. The primary endpoint of the Shasta studies and the accepted regulatory endpoint is TG lowering versus placebo. Plozasiran has been highly active in all patient populations studied, so these studies are overpowered to show TG lowering. One of the additional objectives and key secondary endpoints of Shasta-III and Shasta-IV studies includes the assessment of acute pancreatitis rates.
James Hamilton: We expect top-line data to be available in Q3 2026, with planned sNDA submission for SHTG before the end of the year. We presented the study design and baseline characteristics of the Shasta-III and Shasta-IV studies at the 23rd World Congress Insulin Resistance, Diabetes, and Cardiovascular Disease in December 2025. I'd like to spend a moment to go over a few key parts of that poster. The primary endpoint of the Shasta studies and the accepted regulatory endpoint is TG lowering versus placebo. Plozasiran has been highly active in all patient populations studied, so these studies are overpowered to show TG lowering. One of the additional objectives and key secondary endpoints of Shasta-III and Shasta-IV studies includes the assessment of acute pancreatitis rates.
Speaker #3: In mid 2026 , we expect top line data to be available in the third quarter of 2026 , with planned Smda submission for StG before the end of the year .
Speaker #3: We presented the study design and Baseline Characteristics of the Shasta three and Shasta four studies at the 23rd World Congress . Insulin resistance , diabetes and cardiovascular disease .
Speaker #3: In December of 2025, I'd like to spend a moment to go over a few key parts of that poster. The primary endpoint of the Shasta studies, and the accepted regulatory endpoint, is TG lowering versus placebo, plus aspirin has been highly active in all patient populations studied.
Speaker #3: So these studies are overpowered to show TG lowering one of the additional objectives and key secondary endpoints of Shasta three and four studies Shasta assessment includes the of acute pancreatitis rates .
James Hamilton: To be clear, the study was not designed or prospectively powered to demonstrate AP rate reduction after just a year of treatment. However, there are a meaningful number of SHTG patients enrolled that would be considered at high risk for AP. Specifically, among the two studies, which will be pooled for AP event assessment, 37% of enrolled patients reported TGs greater than 880 mg/dL, an accepted high-risk threshold for AP. In addition, 20% of enrolled patients had a prior medical history of pancreatitis. Lastly, we are seeing AP events in the studies. We are, of course, still blinded and have about another 4 months before the last patient reaches the end of the blinded period, but overall, the studies are progressing as planned.
James Hamilton: To be clear, the study was not designed or prospectively powered to demonstrate AP rate reduction after just a year of treatment. However, there are a meaningful number of SHTG patients enrolled that would be considered at high risk for AP. Specifically, among the two studies, which will be pooled for AP event assessment, 37% of enrolled patients reported TGs greater than 880 mg/dL, an accepted high-risk threshold for AP. In addition, 20% of enrolled patients had a prior medical history of pancreatitis. Lastly, we are seeing AP events in the studies. We are, of course, still blinded and have about another 4 months before the last patient reaches the end of the blinded period, but overall, the studies are progressing as planned.
Speaker #3: be clear , the To study was not prospectively designed or to demonstrate AP rate reduction after just a year of treatment . However , there are a meaningful number of patients enrolled that would be considered at high risk for AP , specifically , among the two studies , which will be pooled for AP event assessment , 37% of enrolled patients reported TGS greater than 880mg per deciliter , accepted high risk threshold for AP .
Speaker #3: In addition, 20% of enrolled patients had a prior medical history of pancreatitis. Lastly, we are seeing AP events in the studies.
Speaker #3: We are , of course , still blinded and have about another four months before the last patient reaches the end of the blinded period .
James Hamilton: Chris mentioned the interim obesity results from our ARO-INHBE and ARO-ALK7 programs earlier, but I'd like to add some color and talk about what we're adding to these programs. First, these early results were very encouraging. The next steps would be to investigate whether and where there is a therapeutic benefit, and in the patient segments and treatment settings where it may be applicable. To review, the interim clinical trial results represent the first demonstration in humans that the Activin E ALK7 pathway, a genetically validated pathway that regulates adipose fat storage, may potentially be harnessed therapeutically to improve body composition and enhance weight loss versus tirzepatide treatment alone in obese patients with type two diabetes mellitus. This patient population typically experiences less weight loss with increased therapy. They're less likely to reach weight loss targets and need more effective, effective treatment options.
James Hamilton: Chris mentioned the interim obesity results from our ARO-INHBE and ARO-ALK7 programs earlier, but I'd like to add some color and talk about what we're adding to these programs. First, these early results were very encouraging. The next steps would be to investigate whether and where there is a therapeutic benefit, and in the patient segments and treatment settings where it may be applicable. To review, the interim clinical trial results represent the first demonstration in humans that the Activin E ALK7 pathway, a genetically validated pathway that regulates adipose fat storage, may potentially be harnessed therapeutically to improve body composition and enhance weight loss versus tirzepatide treatment alone in obese patients with type two diabetes mellitus. This patient population typically experiences less weight loss with increased therapy. They're less likely to reach weight loss targets and need more effective, effective treatment options.
Speaker #3: But overall, the studies are progressing as planned. Chris mentioned the interim obesity results from our ARROW and INHBE ARO-Oxidant programs.
Speaker #3: Earlier , but I'd like to add some color talk about what we're adding and to these programs . First , these early results were very encouraging .
Speaker #3: The steps would be next to investigate whether and where there is a therapeutic benefit, and in the patient segments and treatment settings where it may be applicable to review the interim clinical trial results. These represent the first demonstration in humans that the active and E-ALK7 pathway, a genetically validated pathway that regulates adipose fat storage, may potentially be harnessed therapeutically to improve body composition and enhance weight loss versus tirzepatide treatment alone in obese patients with type 2 diabetes mellitus.
Speaker #3: patient This population typically experiences weight loss with increasing therapy , they're less likely to reach weight loss targets and need more effective , effective treatment options .
James Hamilton: Importantly, ARO-INHBE, in combination with tirzepatide, achieved approximately twofold weight loss and approximately threefold reduction in visceral fat, total fat, and liver fat versus tirzepatide alone in obese diabetics. We saw signals that the pathway was active in the non-diabetics as well, but based on early data, the diabetic signal, particularly in combination with tirzepatide, appeared to be the clearest. We are planning-- We are already in the planning and execution stage of the following next steps: increasing numbers of patients in the phase 1 diabetic cohorts, including longer follow-up and better-- to better understand drug durability and activity out to 1 year, and initiating monotherapy cohorts in obese diabetic patients. We expect to have more data later in 2026 from these programs as we see data from the new expanded scope of the phase 1/2 studies.
James Hamilton: Importantly, ARO-INHBE, in combination with tirzepatide, achieved approximately twofold weight loss and approximately threefold reduction in visceral fat, total fat, and liver fat versus tirzepatide alone in obese diabetics. We saw signals that the pathway was active in the non-diabetics as well, but based on early data, the diabetic signal, particularly in combination with tirzepatide, appeared to be the clearest. We are planning-- We are already in the planning and execution stage of the following next steps: increasing numbers of patients in the phase 1 diabetic cohorts, including longer follow-up and better-- to better understand drug durability and activity out to 1 year, and initiating monotherapy cohorts in obese diabetic patients. We expect to have more data later in 2026 from these programs as we see data from the new expanded scope of the phase 1/2 studies.
Speaker #3: Importantly , and Inhibin combination with trizepatide achieved approximately two fold weight loss and approximately three fold reduction in visceral fat . Total fat , and liver fat versus Tirzepatide alone in obese diabetics .
Speaker #3: We saw signals that the pathway was active in the non-diabetics as well , but based on early data , the diabetic , particularly in combination with tirzepatide , appeared to be the clearest .
Speaker #3: are We planning . We are already in the planning and execution stage of the following next steps . Increasing numbers of patients in the phase one diabetic cohorts , including longer follow up and better to better understand drug durability and activity out to one year and initiating monotherapy cohorts in obese , diabetic patients .
Speaker #3: We expect to have more data later in 2026 from these programs . As we see data from the new expanded scope of the phase one two studies , I will now turn the call over to Daniel Apel .
James Hamilton: I will now turn the call over to Dan Apel.
James Hamilton: I will now turn the call over to Dan Apel.
Daniel Apel: Thank you, James, and good afternoon, everyone. I'll provide a brief outline of our financial picture. As we reported today, net income for the quarter ended 31 December 2025 was $30.8 million, for an income of 22 cents per share, based on 140.7 million fully diluted weighted average shares outstanding. This compares to a net loss of $173.1 million, or loss of $1.39 per share, for the quarter ended 31 December 2024, based on 124.8 million fully diluted weighted average shares outstanding at that time. Revenue for the quarter totaled $264 million, driven primarily by our licensing collaborations agreements with Sarepta and Novartis....
Dan Apel: Thank you, James, and good afternoon, everyone. I'll provide a brief outline of our financial picture. As we reported today, net income for the quarter ended 31 December 2025 was $30.8 million, for an income of 22 cents per share, based on 140.7 million fully diluted weighted average shares outstanding. This compares to a net loss of $173.1 million, or loss of $1.39 per share, for the quarter ended 31 December 2024, based on 124.8 million fully diluted weighted average shares outstanding at that time. Revenue for the quarter totaled $264 million, driven primarily by our licensing collaborations agreements with Sarepta and Novartis....
Speaker #3: Thank you , James , and good afternoon , everyone , and provide a brief outline of our financial picture . As we reported today , net income for the quarter ended December 31st , 2025 was 30.8 million , or an income of $0.22 per share , based on 140.7 million fully diluted weighted average shares outstanding .
Speaker #3: This compares to a net loss of 173.1 million , or loss of $1.39 per share , for the quarter ended December 31st , 2020 .
Speaker #3: based on For 124.8 million fully diluted weighted average shares outstanding at that time . Revenue for the quarter totaled 264 million , primarily by our license and collaborations agreements with Sarepta and Novartis .
Daniel Apel: Of this amount, approximately $229 million related to the Sarepta collaboration, and this included $181 million from the achievement of the second DM1 milestone, $32 million from the ongoing recognition of the initial Sarepta consideration, and $17 million related to reimbursement of incurred collaboration program costs. In addition, we recognized $34 million of the $200 million upfront payment we received from Novartis under our global licensing and collaboration agreement with them. The remainder of that $200 million will be deferred over time as we fulfill our preclinical collaboration obligations. Finally, on revenue, we also recorded our first commercial sale of plozasiran in FCS. As both Chris and Andy have mentioned, we are very encouraged with the feedback and uptake we are seeing with patients and providers.
Dan Apel: Of this amount, approximately $229 million related to the Sarepta collaboration, and this included $181 million from the achievement of the second DM1 milestone, $32 million from the ongoing recognition of the initial Sarepta consideration, and $17 million related to reimbursement of incurred collaboration program costs. In addition, we recognized $34 million of the $200 million upfront payment we received from Novartis under our global licensing and collaboration agreement with them. The remainder of that $200 million will be deferred over time as we fulfill our preclinical collaboration obligations. Finally, on revenue, we also recorded our first commercial sale of plozasiran in FCS. As both Chris and Andy have mentioned, we are very encouraged with the feedback and uptake we are seeing with patients and providers.
Speaker #3: Of this amount , approximately 229 million related to the collaboration , and this included 181 million from the of the achievement second Dm1 milestone , 32 million from the ongoing recognition of the initial sweat to consideration and 17 million related to reimbursement of incurred collaboration program costs .
Speaker #3: In addition , we recognized 34 million of the 200 million upfront payment we received from Novartis under our global licensing and collaboration agreement with them .
Speaker #3: The remainder of that 200 million will be deferred over time . As we fulfill our pre-clinical collaboration obligations . Finally , on revenue , we recorded our first commercial sale of aspirin in FCS as both Chris and Andy have mentioned , we are very encouraged with the feedback and uptake we are seeing with patients and providers .
Daniel Apel: For now, we are not disclosing specific sales numbers until such time as they become a meaningful driver to our financials. Turning to expenses, total operating expenses for the quarter were approximately $223 million, compared to $164 million in the prior year quarter, representing an increase of $59 million year over year. This increase was driven by $40 million of higher R&D expenses and $19 million of higher SG&A expenses. To break that down, the increase in R&D expense was primarily attributable to, as planned, higher clinical costs associated with the phase 3 registrational studies for plozasiran in SHTG, as well as increased clinical supply chain costs. Nearly half of our clinical trial spend in the quarter was associated with our three registrational SHTG studies, namely Shasta III, Shasta IV, and Europe, which again should read out in the summer.
Dan Apel: For now, we are not disclosing specific sales numbers until such time as they become a meaningful driver to our financials. Turning to expenses, total operating expenses for the quarter were approximately $223 million, compared to $164 million in the prior year quarter, representing an increase of $59 million year over year. This increase was driven by $40 million of higher R&D expenses and $19 million of higher SG&A expenses. To break that down, the increase in R&D expense was primarily attributable to, as planned, higher clinical costs associated with the phase 3 registrational studies for plozasiran in SHTG, as well as increased clinical supply chain costs. Nearly half of our clinical trial spend in the quarter was associated with our three registrational SHTG studies, namely Shasta III, Shasta IV, and Europe, which again should read out in the summer.
Speaker #3: We're now. We are not disclosing specific sales numbers until such time as we become a meaningful driver to our financials. Turning to expenses.
Speaker #3: Expenses for the quarter were approximately $223 million, compared to $164 million in the prior year quarter, representing an increase of $59 million year over year.
Speaker #3: This increase was driven by 40 million of higher R&D expenses and 19 million of higher G&A expenses . We break that down . The increase in R&D expense was primarily attributed to , as planned , higher clinical costs associated with our phase three registration of studies for possessor and StG , as well as increased clinical supply chain costs .
Speaker #3: Nearly half of our clinical trials spend in the quarter was associated with our three registrational studies , namely Shasta , three chefs to four in Europe , which should read again out in the summer as expenses increased year over year compared to the prior year's fiscal first quarter , primarily driven by investments to support the commercialization of exemplar as previously the US discussed advance of launch , we built commercial robust capabilities to fully support FCS and importantly , capabilities that were intentionally designed to be highly leverageable downstream .
Daniel Apel: SG&A expenses increased year-over-year compared to the prior year's fiscal Q1, primarily driven by investments to support the commercialization of Redemplo. As previously discussed, in advance of the US launch, we built robust commercial capabilities to fully support FCS, and importantly, capabilities that were intentionally designed to be highly leveragable downstream should we obtain approval for plozasiran in SHTG and zodasiran in HFH. Turning now to the balance sheet. Cash and investments totaled $917 million as of December 31, 2025; common shares outstanding at quarter end were 137.4 million. To be clear, the reported cash balance does not include the $200 million that we earned for the ARO-DM1 second milestone, which was received in January, nor does it include the $50 million anniversary payment that we expect to receive from Sarepta on or before February 10.
Dan Apel: SG&A expenses increased year-over-year compared to the prior year's fiscal Q1, primarily driven by investments to support the commercialization of Redemplo. As previously discussed, in advance of the US launch, we built robust commercial capabilities to fully support FCS, and importantly, capabilities that were intentionally designed to be highly leveragable downstream should we obtain approval for plozasiran in SHTG and zodasiran in HFH. Turning now to the balance sheet. Cash and investments totaled $917 million as of December 31, 2025; common shares outstanding at quarter end were 137.4 million. To be clear, the reported cash balance does not include the $200 million that we earned for the ARO-DM1 second milestone, which was received in January, nor does it include the $50 million anniversary payment that we expect to receive from Sarepta on or before February 10.
Speaker #3: Should we obtain approval for pedestrian and eshg and cedazuridine , a FH . Turning now to the balance sheet , cash and investments totaled 917 million as of December 31st , 2025 .
Speaker #3: Outstanding at quarter end, common shares were 137.4 million. To be clear, the balance does not include the $200 million in cash that we earned for the Dm1 second milestone, which was received in January.
Daniel Apel: Finally, and importantly, the cash balance of $917 million also does not include the financing transactions announced in early January, consisting of a concurrent offering of convertible senior notes and common stock, along with associated capped call transactions. As Chris mentioned, these were on company-friendly terms in the sense that the convertible was 0% coupon and the initial conversion premium was 35%. Said another way, the 0% coupon means the notes will not bear regular interest and the principal amount of the notes will not accrete. The initial conversion price represents a significant premium of approximately 35% over the public offering price per share of common stock in the common stock offering.
Dan Apel: Finally, and importantly, the cash balance of $917 million also does not include the financing transactions announced in early January, consisting of a concurrent offering of convertible senior notes and common stock, along with associated capped call transactions. As Chris mentioned, these were on company-friendly terms in the sense that the convertible was 0% coupon and the initial conversion premium was 35%. Said another way, the 0% coupon means the notes will not bear regular interest and the principal amount of the notes will not accrete. The initial conversion price represents a significant premium of approximately 35% over the public offering price per share of common stock in the common stock offering.
Speaker #3: Nor does it include the 50 million anniversary payment that we expect to receive from Sarepta on or before February 10th . Finally , and importantly , the cash balance of 917 million also does not include the financing transactions announced in early January , consisting of a concurrent and offering of convertible common stock associated call , along transactions .
Speaker #3: As mentioned , these were on company friendly terms in the sense that the was 0% coupon and the initial conversion premium was 35% .
Speaker #3: Said another way, the 0% coupon means the notes will not bear regular interest, and the principal amount of the notes will not accrete. The initial conversion price represents a significant premium of approximately 35% over the public offering price per share of common stock in the common stock offering.
Daniel Apel: Moreover, the private capped calls will prevent any dilution to existing shareholders up to 85% of the premium over the offering price, or roughly $119. We estimate that the total cost of capital of that convertible at any share price below that $119 to be very attractively below 1.5%. All that is to say is... All that is to say that we have very significantly and efficiently strengthened our balance sheet, which provides additional flexibility to support ongoing clinical development, current and future commercialization activities, and other long-term strategic priorities. With that brief overview, I will now turn the call back to Chris.
Dan Apel: Moreover, the private capped calls will prevent any dilution to existing shareholders up to 85% of the premium over the offering price, or roughly $119. We estimate that the total cost of capital of that convertible at any share price below that $119 to be very attractively below 1.5%. All that is to say is... All that is to say that we have very significantly and efficiently strengthened our balance sheet, which provides additional flexibility to support ongoing clinical development, current and future commercialization activities, and other long-term strategic priorities. With that brief overview, I will now turn the call back to Chris.
Speaker #3: Moreover, the private CAP calls will prevent any dilution to existing shareholders up to 85% of the premium over the offering price, or roughly $119.
Speaker #3: We estimate that the total cost convertible at any share price that $118 to very be attractively below 1.5% . All that is to say , is all that is to say have very significantly and efficiently strengthened our balance provides additional flexibility to support ongoing clinical development , current and future commercialization activities , and other long term strategic priorities .
Christopher Anzalone: Thanks, Dan. This is indeed an exciting time to be at Arrowhead or an Arrowhead shareholder. We're coming off an historic period for the company, where we executed extremely well, and all the hard work of the last several years is starting to pay off. While 2025 was productive, we look to the remainder of 2026 and the years ahead to be even more transformational. Let's look at some key 2026 events that we anticipate could be important value-creating events for the company and our shareholders. Commercial sales progress for Redemplo. Q3 2026 readout of phase 3, Shasta 3, and Shasta 4 studies of plozasiran in patients with SHTG, which we believe has the potential to be a $3 to 4 billion commercial opportunity.
Chris Anzalone: Thanks, Dan. This is indeed an exciting time to be at Arrowhead or an Arrowhead shareholder. We're coming off an historic period for the company, where we executed extremely well, and all the hard work of the last several years is starting to pay off. While 2025 was productive, we look to the remainder of 2026 and the years ahead to be even more transformational. Let's look at some key 2026 events that we anticipate could be important value-creating events for the company and our shareholders. Commercial sales progress for Redemplo. Q3 2026 readout of phase 3, Shasta 3, and Shasta 4 studies of plozasiran in patients with SHTG, which we believe has the potential to be a $3 to 4 billion commercial opportunity.
Speaker #3: With that brief overview, I will now turn the call back to Chris. Thanks, Dan.
Speaker #4: It is indeed an exciting time to be at Arrowhead or an Arrowhead shareholder. We're coming off an historic period for the company where we executed extremely well, and all the hard work of the last several years is starting to pay off.
Speaker #4: While 2025 was productive , we look to the of 2026 and the years ahead to even more transformational . Let's look at some key 2026 events that we anticipate could be important value creating events for the company and our shareholders , commercial sales progress for ejemplo , Q3 2026 readout of phase three , Shasta three and Shasta four studies of aspirin in patients with StG , which we believe potential to be a 3 to $4 billion commercial opportunity .
Christopher Anzalone: Second half 2026 readout for ARO-DIMER-PA, targeting PCSK9 and APOC3 for LDL and TG lowering, which may address mixed hyperlipidemia, a population of potentially 20 million patients in the US. Additional ARO-INHBE and ARO-ALK7 data presented in 2026, that may build on the already encouraging early data for this novel, non-incretin strategy, and early ARO-MAPT data in 2026, potentially providing validation for this drug candidate and our emerging CNS pipeline with systemic delivery via subcutaneous administration. These are just a few potentially important events in 2026 alone. If you fast-forward 1 to 3 years, we expect many more opportunities in our pipeline to build value and potential, commercial launches, both independently and with partners.
Chris Anzalone: Second half 2026 readout for ARO-DIMER-PA, targeting PCSK9 and APOC3 for LDL and TG lowering, which may address mixed hyperlipidemia, a population of potentially 20 million patients in the US. Additional ARO-INHBE and ARO-ALK7 data presented in 2026, that may build on the already encouraging early data for this novel, non-incretin strategy, and early ARO-MAPT data in 2026, potentially providing validation for this drug candidate and our emerging CNS pipeline with systemic delivery via subcutaneous administration. These are just a few potentially important events in 2026 alone. If you fast-forward 1 to 3 years, we expect many more opportunities in our pipeline to build value and potential, commercial launches, both independently and with partners.
Speaker #4: Second half 2026 readout or PA targeting Pcsk9 and Apoc3 for LDL and TG lowering , which may address mixed hyperlipidemia . Up population with potentially 20 million patients in the US .
Speaker #4: Additional ANGIOTENSIN and ARO-HB and ARO-7 data presented in 2026 may build on the already encouraging early data for this novel non-incretin strategy and early ARO data in 2026.
Speaker #4: Potentially providing validation for this drug candidate and our emerging CNS pipeline with systemic via delivery, subcutaneous administration. These are just a few potentially important events in 2026 alone.
Speaker #4: If you fast forward 1 to 3 years , we expect many more opportunities in our pipeline to build value and launches , potential commercial both independently and with partners .
Andy Davis: ... Thank you for joining us today. I would now like to open the call to your questions. Operator?
Chris Anzalone: ... Thank you for joining us today. I would now like to open the call to your questions. Operator?
Operator: Thank you. To ask a question, you need to press star one one on your phone and wait for your name to be announced. To withdraw your question, please press star one one again. Please limit to one question in the interest of time. Any additional questions, you can go back into the queue. Please stand by. We can find the Q&A roster in a moment for our first question. Our first question will come from the line of Mike Ulz from Morgan Stanley. Your line is open.
Operator: Thank you. To ask a question, you need to press star one one on your phone and wait for your name to be announced. To withdraw your question, please press star one one again. Please limit to one question in the interest of time. Any additional questions, you can go back into the queue. Please stand by. We can find the Q&A roster in a moment for our first question. Our first question will come from the line of Mike Ulz from Morgan Stanley. Your line is open.
Speaker #4: Thank you for today. I would now like to open the call to your questions. Operator.
Speaker #1: Thank you . To ask a question , you need to press star one one on your telephone and wait for your name to be announced .
Speaker #1: To withdraw your please press star one question , one again . Please . To one question . In the interest of any additional time , ?
Speaker #1: questions You can go back the queue . into Please stand by . The Q&A roster and moment for our first question . Our first question will come from Mike the line of Oles Morgan from line is Stanley .
[Analyst] (Morgan Stanley): Good afternoon, and thanks for taking the question. Maybe just one on Redemplo. Can you just give a little bit more color on the breakdown between the different categories of patients transitioning from expanded access, naive, and switch? And then maybe on the latter, in terms of switch, just any key reasons you're seeing a switch, and does it have anything to do with, you know, coverage and pricing? Thanks.
Mike Ulz: Good afternoon, and thanks for taking the question. Maybe just one on Redemplo. Can you just give a little bit more color on the breakdown between the different categories of patients transitioning from expanded access, naive, and switch? And then maybe on the latter, in terms of switch, just any key reasons you're seeing a switch, and does it have anything to do with, you know, coverage and pricing? Thanks.
Speaker #1: Your .
Speaker #1: open
Speaker #5: Good thanks for taking the
Speaker #5: afternoon and
Speaker #5: Maybe one on just . Can you Redemptio . little bit just give a color on the more between the breakdown categories of different patients transitioning from expanded access , switch .
Speaker #5: then naive and And latter , in maybe on the switch , terms of any reasons key a switch , and you're seeing does it have do anything to with coverage and pricing ?
Andy Davis: Thanks, Mike. This is Andy. Yeah, I can comment that the vast majority of patient origination is from APOC3 naive segment, with the remaining balance split roughly 50/50 between those that are coming from switch and those that are transitioning off of the expanded access program. As it relates to switch, we're seeing switch patients that are coming both from efficacy but also from safety as the two principal drivers for why physicians might be considering Redemplo as an alternative. I hope that helps.
Andy Davis: Thanks, Mike. This is Andy. Yeah, I can comment that the vast majority of patient origination is from APOC3 naive segment, with the remaining balance split roughly 50/50 between those that are coming from switch and those that are transitioning off of the expanded access program. As it relates to switch, we're seeing switch patients that are coming both from efficacy but also from safety as the two principal drivers for why physicians might be considering Redemplo as an alternative. I hope that helps.
Speaker #5: Thanks .
Speaker #4: Thanks , Andy . Yeah , Mike . comment This is I can vast that the patient of majority origination is Apoc3 naive segment with the from split remaining balance 5050 between coming switch roughly those that are and those from transitioning off of the that are access expanded .
Speaker #4: program relates to switch it . switch We're seeing that are As both coming from patients efficacy , but also from safety as , the two principal for why drivers might be physicians redemptio as an considering alternative .
Operator: Thank you. One moment for our next question. Our next question comes from the line of Maury Raycroft from Jefferies. Your line is open.
Operator: Thank you. One moment for our next question. Our next question comes from the line of Maury Raycroft from Jefferies. Your line is open.
Speaker #4: I hope that helps
Speaker #4: .
Speaker #1: Thank you. One moment for the next question. Our next question comes from Maori Line of Raycroft Jefferies. Your line is open.
[Analyst] (Jefferies): Hi, congrats on the progress, and thanks for taking my question. I'll ask one on obesity. Just wondering if you've had discussions with FDA about the development path, or when would it make sense to do this? And what could timelines for your phase 2 start look like? And do you need to have all the data, including combo data in hand, before you can determine next steps for the development path?
Maury Raycroft: Hi, congrats on the progress, and thanks for taking my question. I'll ask one on obesity. Just wondering if you've had discussions with FDA about the development path, or when would it make sense to do this? And what could timelines for your phase 2 start look like? And do you need to have all the data, including combo data in hand, before you can determine next steps for the development path?
Speaker #6: Hi . Congrats on the progress and taking my question . I'll ask thanks for one on wondering if you've discussions obesity . Just had with FDA about the development path or it make when would sense to this ?
Speaker #6: what could And do timelines for your phase two start look like ? you need to the including data , combo data And do have all , before you can in hand determine next steps for path development the ?
James Hamilton: Yeah, sure, Maury, I can take that. This is James. Probably middle of the year, we would be having some of those discussions with FDA. I don't think we need all of the data from all of the cohorts. As I mentioned in the prepared remarks, we expanded some of these cohorts, so they'll be going on, some of them, for a longer period of time. So, you know, FDA conversation is probably middle of around middle of the year, and then we'll be looking to file an IND shortly thereafter.
James Hamilton: Yeah, sure, Maury, I can take that. This is James. Probably middle of the year, we would be having some of those discussions with FDA. I don't think we need all of the data from all of the cohorts. As I mentioned in the prepared remarks, we expanded some of these cohorts, so they'll be going on, some of them, for a longer period of time. So, you know, FDA conversation is probably middle of around middle of the year, and then we'll be looking to file an IND shortly thereafter.
Speaker #2: Yeah , sure .
Speaker #3: Mario .
Speaker #4: is James that . This , probably can take middle of the .
Speaker #3: The year would be.
Speaker #3: Discussions with .
Speaker #3: think I don't we Having some of
Speaker #3: need We .
Speaker #3: .
Speaker #4: the from all of the cohorts . As I data mentioned in the those prepared FDA . All of remarks , we had expanded some of these cohorts .
Speaker #4: they'll be on some of them So for longer period of time And so , . you know , FDA probably conversations middle around middle would year .
Operator: Thank you. One moment for our next question. Our next question will come from the line of Andrea Newkirk from Goldman Sachs. Your line is open.
Operator: Thank you. One moment for our next question. Our next question will come from the line of Andrea Newkirk from Goldman Sachs. Your line is open.
Speaker #4: then be to And looking of the that to file an IND shortly thereafter .
Speaker #1: One moment for our next thank you. Question next from our line. Our question will come from Newkirk, from Andrea at Goldman Sachs.
[Analyst] (Goldman Sachs): Hi, guys. Good afternoon. Thanks for taking the question. Maybe I can ask you one here on the ARO-DIMER-PA asset. Just as we think about the data sets that's coming later this year, just curious if you might be willing to speculate or share what you are looking for or how you've defined a TPP, what level of reduction in LDL-C and trigs you're hoping to see, and then how that might inform a go, no-go decision for advancing the asset forward, and what extent of reduction would give you confidence that you could then see that translation to a benefit on MACE? Thanks so much.
Andrea Newkirk: Hi, guys. Good afternoon. Thanks for taking the question. Maybe I can ask you one here on the ARO-DIMER-PA asset. Just as we think about the data sets that's coming later this year, just curious if you might be willing to speculate or share what you are looking for or how you've defined a TPP, what level of reduction in LDL-C and trigs you're hoping to see, and then how that might inform a go, no-go decision for advancing the asset forward, and what extent of reduction would give you confidence that you could then see that translation to a benefit on MACE? Thanks so much.
Speaker #1: open . Hi ,
Speaker #7: Good guys . afternoon . Thanks
Speaker #7: for taking the Maybe I can question . ask you one here on the Aero Just as we think about dimer the the PA asset .
Speaker #7: Data set that are later, that's coming year, this. Just if you might be curious, be willing to speculate, share what or you are looking for, or how you've defined a TPP.
Speaker #7: What level of reduction in LDL-C and triglycerides are you hoping to see, and how might that inform a go/no-go decision for advancing the asset forward?
Speaker #7: And what extent of reduction would you could then give you see that translation to a benefit on Mace ? Thanks much so .
James Hamilton: Yeah, sure. We'll see. I think, you know, we probably don't have to reach the level of reduction in terms of APOC3 and triglycerides that we're seeing with zodasiran, for example, as something less than that, with the combination of the LDL cholesterol reductions would probably be sufficient. So, you know, I think if you look at some of the monkey data that we presented, in the dyslipidemic monkeys, we were seeing reductions in LDL and in triglycerides of around 40, 50%. So, I mean, so I think something like that, if you could do both of those, that would, that'd be really encouraging. But we'll see what the data show later this year.
James Hamilton: Yeah, sure. We'll see. I think, you know, we probably don't have to reach the level of reduction in terms of APOC3 and triglycerides that we're seeing with zodasiran, for example, as something less than that, with the combination of the LDL cholesterol reductions would probably be sufficient. So, you know, I think if you look at some of the monkey data that we presented, in the dyslipidemic monkeys, we were seeing reductions in LDL and in triglycerides of around 40, 50%. So, I mean, so I think something like that, if you could do both of those, that would, that'd be really encouraging. But we'll see what the data show later this year.
Speaker #3: sure . We'll we'll see .
Speaker #4: I think Yeah , we probably , you know , don't have level reach the of reduction in to apoc3 terms of we're and triglycerides that seeing with aspirin , for example , as something less than that with the combination of the LDL cholesterol reductions would probably be sufficient .
Speaker #4: So , you know , I think if you look at some we of the monkey presented data that in the monkeys , we seeing were Dyslipidaemic reductions in and in triglycerides of around LDL 50% .
Speaker #4: 40 , So think something like that , if you both of I those , that would that'd can do be really we'll see what the data encouraging .
Operator: Thank you. One moment for our next question. Our next question comes from the line of Luca Issi from RBC Capital Markets. Your line is open.
Operator: Thank you. One moment for our next question. Our next question comes from the line of Luca Issi from RBC Capital Markets. Your line is open.
Speaker #4: But later this show year.
Speaker #1: you . for our Thank next question One moment Next . question line comes from of Luca from RBC Capital Markets . Your line open is .
[Analyst] (Morgan Stanley): Well, thanks so much. Hi, team. This is Cassie on for Luca. Congrats on strong Redemplo launch and progresses. A question also on ARO-INHBE and ARO-ALK7, since we just talked about the regulatory path. Andy, appreciate early days, but how are you thinking about potential pricing for ARO-INHBE and ARO-ALK7? I mean, Lilly now offers Zepbound via Lilly Direct at $300 a month, and the compounders announced today that you can get oral Rybelsus basically at the same monthly price as Wegovy. So what is your latest thinking on pricing, and how should we think about COGS for ARO-INHBE and ARO-ALK7? Thanks so much.
Luca Issi: Well, thanks so much. Hi, team. This is Cassie on for Luca. Congrats on strong Redemplo launch and progresses. A question also on ARO-INHBE and ARO-ALK7, since we just talked about the regulatory path. Andy, appreciate early days, but how are you thinking about potential pricing for ARO-INHBE and ARO-ALK7? I mean, Lilly now offers Zepbound via Lilly Direct at $300 a month, and the compounders announced today that you can get oral Rybelsus basically at the same monthly price as Wegovy. So what is your latest thinking on pricing, and how should we think about COGS for ARO-INHBE and ARO-ALK7? Thanks so much.
Speaker #8: Thanks so much. Hi, team. This is Cassie on for Luca. Congrats on the strong launch and progress. My question is also on the HIV services.
Speaker #8: just talked We about the regulatory path and the early days . But how are you thinking about pricing for potential inhibition ALK seven .
Speaker #8: mean I literally now offers that direct at 300 a month the announced . And Compounders today that you can get oral basically at the wegovy same price monthly YouTube TV .
Andy Davis: Thank you. It is as you expected. It's way too early for us to think about that. You know, we're interrogating the biology here to see how these drug candidates could potentially, you know, work in various patient populations. Until we have a better understanding of that, you know, it's really too early to speculate on potential pricing.
Andy Davis: Thank you. It is as you expected. It's way too early for us to think about that. You know, we're interrogating the biology here to see how these drug candidates could potentially, you know, work in various patient populations. Until we have a better understanding of that, you know, it's really too early to speculate on potential pricing.
Speaker #8: latest So what is your thinking on pricing should we think about Cogs inhibition ? Thanks so much for
Speaker #8: .
Speaker #4: is you Thanks . as expected . It's way It as too early for us to to that . You think about know , we're inheriting the biology see here to these candidates how how drug could potentially work in various patient populations until we have a better understanding of that .
Operator: Thank you. One moment for our next question. Our next question comes from the line of Prakhar Agrawal from Cantor. Your line is open.
Operator: Thank you. One moment for our next question. Our next question comes from the line of Prakhar Agrawal from Cantor. Your line is open.
Speaker #4: You know, it's really too early to speculate on potential pricing.
Speaker #1: Thank you . One moment for our next question . Our next question comes line of Prakhar Agarwal from cancer . Your open line is .
[Analyst] (Cantor): Hi, thank you for taking my questions, and congrats on the quarter and the progress. So I think, James, you mentioned that about the pancreatitis event rates in the ongoing phase 3 SHARLOTTE 3-4 trials. Maybe if you can talk about the blinded AP events that you're seeing in those trials and whether it's in the same ballpark of what IONIS saw. And just a follow-up to that, would you expect the placebo event rate on AP reduction to perform similarly to Olezarsen core trials, given the population looks similar, or are there any nuances that we should be aware of? Thank you so much.
Prakhar Agrawal: Hi, thank you for taking my questions, and congrats on the quarter and the progress. So I think, James, you mentioned that about the pancreatitis event rates in the ongoing phase 3 SHARLOTTE 3-4 trials. Maybe if you can talk about the blinded AP events that you're seeing in those trials and whether it's in the same ballpark of what IONIS saw. And just a follow-up to that, would you expect the placebo event rate on AP reduction to perform similarly to Olezarsen core trials, given the population looks similar, or are there any nuances that we should be aware of? Thank you so much.
Speaker #9: Thank you for taking my the Hi . congrats on questions quarter and the progress . So I think mentioned James , you that about the pancreatitis event rates in the Three , ongoing phase three .
Speaker #9: trials . Maybe four talk about you can the blinded AP you are events that seeing in those whether it's in the trials , and ballpark same of what Ionis saw .
Speaker #9: just a to follow up you that , would expect the rate on event And reduction to placebo AP perform similarly to core Olezarsen , given the trials looks population similar ?
James Hamilton: Yeah. So on the first one, we're not gonna give any additional details on event rates, or the number of events that we've seen, other than to say that we are seeing events. On the second question, I mean, I think it's rational to look at the core 2 placebo rate, that the population was similar to ours. So they are obviously different studies, but the population was similar.
James Hamilton: Yeah. So on the first one, we're not gonna give any additional details on event rates, or the number of events that we've seen, other than to say that we are seeing events. On the second question, I mean, I think it's rational to look at the core 2 placebo rate, that the population was similar to ours. So they are obviously different studies, but the population was similar.
Speaker #9: Or are there any nuances that we should be aware of ? Thank you so much .
Speaker #4: Yeah .
Speaker #3: The first .
Speaker #4: we're One , So on . not going to give any details additional on event rates for the number of events that we've Other than to say that we are seeing events .
Speaker #4: On the second question , I mean , I think it's rational to look at the and the core . the core Two placebo the population rate that similar to was So ours .
Speaker #4: They are obviously different studies, but the population was similar.
Operator: Thank you. One moment for our next question. Our next question comes from the line of Jason Gerberry from Bank of America. Your line is open.
Operator: Thank you. One moment for our next question. Our next question comes from the line of Jason Gerberry from Bank of America. Your line is open.
Speaker #1: You one moment for our next question . Next question comes line of Jason Gerberry from Bank of America . Your line is open .
[Analyst] (Bank of America): Hey, guys. Thanks for taking my question. You mentioned payer feedback for Redemplo. I believe that was in the context of FCS. But I'm curious if in those discussions, SHTG came up at all, and whether that price point that you guys have for FCS is appropriate for a market the size of SHTG and the likely benefits that APOC3 would provide. It seems pretty de-risked at this point, but just kind of curious if those discussions came up and how the view was on the $60,000 price point. Thanks.
Jason Gerberry: Hey, guys. Thanks for taking my question. You mentioned payer feedback for Redemplo. I believe that was in the context of FCS. But I'm curious if in those discussions, SHTG came up at all, and whether that price point that you guys have for FCS is appropriate for a market the size of SHTG and the likely benefits that APOC3 would provide. It seems pretty de-risked at this point, but just kind of curious if those discussions came up and how the view was on the $60,000 price point. Thanks.
Speaker #10: Hey , guys , thanks for taking my question . You mentioned payer feedback for I believe that was in the context of FCS , but I'm curious if in those discussions came up at all .
Speaker #10: And whether that price point that you guys have for FCS is appropriate for a size QTc, and likely benefits that APOC3 provides, would that seem pretty—at this point.
Speaker #10: Pretty, but just de-risked. I'm kind of curious if those discussions came up and how the view was on the price point. Thanks.
James Hamilton: Thanks, Jason. This is Andy. Appreciate the question. I won't get into the details of any specific payer discussions, only to say that our team is laser-focused on ensuring we can gain coverage and access for those patients that have FCS, either genetically confirmed or clinically diagnosed. I would just add that the payers with whom we're discussing represent over 90% of US lives, and both the clinical teams and the economic teams recognize the clinical value and the economic value of Redemplo at the one Redemplo price that we've previously announced.
James Hamilton: Thanks, Jason. This is Andy. Appreciate the question. I won't get into the details of any specific payer discussions, only to say that our team is laser-focused on ensuring we can gain coverage and access for those patients that have FCS, either genetically confirmed or clinically diagnosed. I would just add that the payers with whom we're discussing represent over 90% of US lives, and both the clinical teams and the economic teams recognize the clinical value and the economic value of Redemplo at the one Redemplo price that we've previously announced.
Speaker #4: Thanks , Jason . This is Andy . the Appreciate won't get into question . I the details of payer any specific discussions , only to say that our team is is laser focused on ensuring we gain can coverage those and access for patients FCS , that have either confirmed genetically clinically diagnosed .
Speaker #4: I or add that the with whom we're would just payers discussing over represent us 90% of in clinical both the teams lives and the economic teams .
[Analyst] (Bank of America): Thanks.
Jason Gerberry: Thanks.
James Hamilton: You mentioned, you know, the size of the SHTG market. You know, we think there are, you know, somewhere around 3.5 million people with triglycerides above 500. But that market is not all created equal. You know, when we look at our at least initial target market there, and we look at how we price Redemplo, it is really focused on those very high-risk individuals, those maybe 750,000 to maybe a million people who have triglycerides above 880, you know, or a history of pancreatitis. You know, that's, at least initially, that is the real core market. That, you know, those are the patients who really need this new medicine.
Speaker #4: clinical the value and the economic value of Recognize at the one we've price that announced . previously Thanks . And you mentioned , you know , the the the size of the market .
James Hamilton: You mentioned, you know, the size of the SHTG market. You know, we think there are, you know, somewhere around 3.5 million people with triglycerides above 500. But that market is not all created equal. You know, when we look at our at least initial target market there, and we look at how we price Redemplo, it is really focused on those very high-risk individuals, those maybe 750,000 to maybe a million people who have triglycerides above 880, you know, or a history of pancreatitis. You know, that's, at least initially, that is the real core market. That, you know, those are the patients who really need this new medicine.
Speaker #4: We think there are around somewhere 3.5 million people with triglycerides above 500 . But that market is not all created equal . You know , when we when we look at , at our at least initial target there , and we market , look at how we price exemplo is really is it , it very on those high risk individuals , those maybe 750,000 to maybe a million people who are who have triglycerides above 800 or history of pancreatitis .
James Hamilton: So again, don't, you know, don't get lost in the 3 to 4 million people with trigs above 500. Really focus on the high risk group. That's who we're focusing on, at least initially.
James Hamilton: So again, don't, you know, don't get lost in the 3 to 4 million people with trigs above 500. Really focus on the high risk group. That's who we're focusing on, at least initially.
Speaker #4: That's at least initially . That real core is the market that those are the patients who really need medicine . this new And so again , don't get don't lost the in 3 to 4 million 500 .
Operator: Thank you. One moment for our next question. Our next question will come from the line of Patrick Trucchio from H.C. Wainwright. Your line is open.
Operator: Thank you. One moment for our next question. Our next question will come from the line of Patrick Trucchio from H.C. Wainwright. Your line is open.
Speaker #4: people that trades above Really focus group . on that high That's who we're on at focusing least initially risk .
Speaker #1: One moment for Thank you . question our next question comes from . Our next Patrick line of Your from H.C. Wainwright . Trujillo open line is .
[Analyst] (Cantor): Thanks. My question is on ARO-MAPT. I'm just curious, with the interim data from the healthy volunteer portion, and then with the patient data to follow, I'm wondering what specific elements of the healthy volunteer data, safety, CSF, tau knockdown, or downstream biomarkers, would most likely increase your confidence in this program, and as well, the data we should look for in patients to follow? And if you could also just talk about, you know, just the confidence this would give in the CNS targeting and platform overall, and how we should expect, you know, the CNS platform to develop from here.
Patrick Trucchio: Thanks. My question is on ARO-MAPT. I'm just curious, with the interim data from the healthy volunteer portion, and then with the patient data to follow, I'm wondering what specific elements of the healthy volunteer data, safety, CSF, tau knockdown, or downstream biomarkers, would most likely increase your confidence in this program, and as well, the data we should look for in patients to follow? And if you could also just talk about, you know, just the confidence this would give in the CNS targeting and platform overall, and how we should expect, you know, the CNS platform to develop from here.
Speaker #3: Thanks . My question is on I'm just aromatics . curious , with the interim from the data volunteer portion then with healthy the and patient data to follow , I'm specific wondering what the elements volunteer healthy of data safety CSF , knockdown or downstream would biomarkers likely increase your confidence in this most tau program .
Speaker #3: as well , the data we should look And in patients to follow for And . could also just if you about , you talk know , confidence just the this would the give in the in CNS targeting and platform overall and how we should expect , you know , the CNS here develop from platform to .
James Hamilton: Yeah, I can, I can take that, Patrick. This is James. So maybe I'll take the second question first. You know, we don't have any data in the clinic yet, any data from humans. But we do have data using the platform with multiple different targets in multiple different monkey studies, and they're pretty consistent in terms of the drug concentration that we get in various CNS regions and the knockdown we're able to achieve in even the deep brain. So you know, that is helpful and certainly enhances our confidence. But of course, the large leap in confidence will come once we see the clinical data.
James Hamilton: Yeah, I can, I can take that, Patrick. This is James. So maybe I'll take the second question first. You know, we don't have any data in the clinic yet, any data from humans. But we do have data using the platform with multiple different targets in multiple different monkey studies, and they're pretty consistent in terms of the drug concentration that we get in various CNS regions and the knockdown we're able to achieve in even the deep brain. So you know, that is helpful and certainly enhances our confidence. But of course, the large leap in confidence will come once we see the clinical data.
Speaker #3: Yeah .
Speaker #4: I can Patrick . This is James . take that . maybe I'll take the second question first . don't have any You know , we in in the clinic yet .
Speaker #4: Any data from humans . But we do have data . Using the platform with multiple different targets in multiple different monkey studies . And they're pretty consistent in terms of the drug concentration that we get in various CNS regions .
Speaker #4: And the knockdown we're able to achieve in even the deep brain . So that that is helpful . And certainly enhances our of confidence .
James Hamilton: To your first question, I think the key data that we anticipate being confidence building, of course, safety, and then the CSF knockdown will be key in the healthy volunteers. There's not a lot of other downstream biomarkers to measure in the healthy volunteers, but then going forward into the patient cohorts, we can measure some of the phospho-tau varieties in the blood, also in the CSF. Then, of course, we can look at tau PET, although those readouts will take a while to see the tau PET signals in the patients. I think seeing a reduction in tau PET signal will be really very encouraging.
James Hamilton: To your first question, I think the key data that we anticipate being confidence building, of course, safety, and then the CSF knockdown will be key in the healthy volunteers. There's not a lot of other downstream biomarkers to measure in the healthy volunteers, but then going forward into the patient cohorts, we can measure some of the phospho-tau varieties in the blood, also in the CSF. Then, of course, we can look at tau PET, although those readouts will take a while to see the tau PET signals in the patients. I think seeing a reduction in tau PET signal will be really very encouraging.
Speaker #4: of course , the large leap in confidence will come once we see the clinical data . And to your first question , I think the key data that we anticipate being confidence building , of course , safety .
Speaker #4: And then the the CSF knockdown will be in the healthy volunteers . There's not a lot of other downstream biomarkers to measure in the healthy volunteers .
Speaker #4: But then into the going forward cohorts , we can measure some of the the phospho tau varieties the blood . in Also in the CSF .
Speaker #4: And then of course , we can look at tau Pet . Although will take a readouts while see the the tau Pet signals in the patients .
Speaker #4: think seeing a I reduction in tau Pet signal will be really encouraging .
Operator: Thank you. One moment for our next question. The next question will come from the line of Edward Tenthoff from Piper Sandler. Your line is open.
Operator: Thank you. One moment for our next question. The next question will come from the line of Edward Tenthoff from Piper Sandler. Your line is open.
Speaker #1: Thank you. One moment for our next question. The question comes from the next line of Tenthoff from Piper Edward. Your line is open.
[Analyst] (Bank of America): Great. Thank you very much.
Edward Tenthoff: Great. Thank you very much.
[Analyst] (B. Riley Securities): ... So thanks for all the detail. I'm really excited to see the pipeline advancing. I'm wondering, when it comes to the recognition of revenues, at this point, are you guys anticipating breaking out a cost of goods sold line? I'm sure a lot of the manufacturing expenses already been expensed to R&D, but I'm just trying to think about how you're planning on reporting COGS going forward. Will you break out Redemplo product sales in the future too? Thanks.
Edward Tenthoff: ... So thanks for all the detail. I'm really excited to see the pipeline advancing. I'm wondering, when it comes to the recognition of revenues, at this point, are you guys anticipating breaking out a cost of goods sold line? I'm sure a lot of the manufacturing expenses already been expensed to R&D, but I'm just trying to think about how you're planning on reporting COGS going forward. Will you break out Redemplo product sales in the future too? Thanks.
Speaker #1: . Great .
Speaker #3: Thank you very much . I'm so thanks for all the detail . I'm excited to see the pipeline really advancing . wondering when it I'm comes to the recognition revenues at this point , are you guys anticipating breaking out of cost sold of goods ?
Speaker #3: ? Line I'm sure a lot of the manufacturing expenses already been expensed to R&D , but trying to I'm just think how you're planning on reporting Cogs going forward and will you break out redemption product sales in the future , too ?
Daniel Apel: Yeah. Thanks, Ted. Thanks for the question. Yeah, as you pointed out, the cost of goods sold, part of plozasiran are gonna be in the R&D. That's the majority of what we're gonna see in the short term. You know, we said in the prepared remarks, we're not, we're not gonna disclose this, you know, actively until such time as there are meaningful drivers. So we will at some point, not gonna hazard a guess as to when that will be. But then you would - at that point, you would normally see then sort of that traditional product growth and product cost of sales.
Dan Apel: Yeah. Thanks, Ted. Thanks for the question. Yeah, as you pointed out, the cost of goods sold, part of plozasiran are gonna be in the R&D. That's the majority of what we're gonna see in the short term. You know, we said in the prepared remarks, we're not, we're not gonna disclose this, you know, actively until such time as there are meaningful drivers. So we will at some point, not gonna hazard a guess as to when that will be. But then you would - at that point, you would normally see then sort of that traditional product growth and product cost of sales.
Speaker #3: Thanks . Yes .
Speaker #4: Thanks , Ted . Thanks for the question . Yeah . As you pointed out , the cost of goods sold prior to launch are going to be in the R&D .
Speaker #4: And that's the majority of what we're going to see in the short term . You know , we said in the prepared remarks , we're not we're not going to disclose this .
Speaker #4: You know , actively until such time as there are meaningful driver . So we will at some point not to hazard a guess as to when that will be .
Speaker #4: But then you would at that point , you would normally see then sort of that traditional product revenue and product cost .
Operator: Thank you. One moment for our next question. Our next question comes from the line of Joseph Thome from TD Cowen. Your line is open.
Operator: Thank you. One moment for our next question. Our next question comes from the line of Joseph Thome from TD Cowen. Your line is open.
Speaker #1: you Thank . next One moment for our question . question Our next comes from Joseph of from TD . Cavan . Your line is open .
[Analyst] (TD Cowen): Hi there. Good afternoon. Thank you for taking my question. Maybe just based on the, the differential biology of Activin E and ALK7, can you talk a little bit about your expectation to see monotherapy weight loss in obese, non-diabetic patients with the ALK7 program? And then a point of clarification, when you talk about the expansions of the studies in terms of, you know, including that, monotherapy diabetic population and expanding the overall size, was that for the Activin E and ALK7 programs already, both of them? Thank you.
Joseph Thome: Hi there. Good afternoon. Thank you for taking my question. Maybe just based on the, the differential biology of Activin E and ALK7, can you talk a little bit about your expectation to see monotherapy weight loss in obese, non-diabetic patients with the ALK7 program? And then a point of clarification, when you talk about the expansions of the studies in terms of, you know, including that, monotherapy diabetic population and expanding the overall size, was that for the Activin E and ALK7 programs already, both of them? Thank you.
Speaker #11: Hi there . Good afternoon . Thank you for taking my question . Maybe just the differential based on activity and biology of alk7 , can you talk little bit about your expectation to see monotherapy weight loss in obese non-diabetic patients with the program ?
Speaker #11: And then a point of clarification when you talk about the expansions of the studies in terms of including that monotherapy , diabetic population and expand the overall size , was that for the activin and E already , Alk7 both of them .
James Hamilton: Yeah, I think, well, we don't really have expectations in terms of monotherapy weight loss. We'll see what happens. I think we've said just a few times that we view these studies as hypothesis generating, so we'd like to see if there's an early signal and then potentially expand cohorts to confirm that signal. So, can't really predict ahead of time what we're going to see. Then on your second question, the addition of the monotherapy cohort, we did add that in the INHBE study. We will likely add that in the ALK7 study as well.
James Hamilton: Yeah, I think, well, we don't really have expectations in terms of monotherapy weight loss. We'll see what happens. I think we've said just a few times that we view these studies as hypothesis generating, so we'd like to see if there's an early signal and then potentially expand cohorts to confirm that signal. So, can't really predict ahead of time what we're going to see. Then on your second question, the addition of the monotherapy cohort, we did add that in the INHBE study. We will likely add that in the ALK7 study as well.
Speaker #11: Thank you programs .
Speaker #4: Yeah , I think well , we don't we don't really have in expectations terms of monotherapy We'll see happens . I weight loss .
Speaker #4: Yeah , I think well , we don't we don't really have in expectations terms of monotherapy We'll see happens . I weight loss . think what we've said to a few times we view that these studies as , as hypothesis generating .
Speaker #4: So we'd like to see if there's an early signal, and then potentially expand cohorts to confirm that signal. So we can't really, ahead of time, predict what we're going to see.
Speaker #4: Then on your second question , the the addition of the monotherapy cohort , we did add that inhibin in the study that we we will likely that in add the .
Operator: Thank you. One moment for our next question. Our next question comes from the line of Mani Foroohar from Leerink Partners. Your line is open.
Operator: Thank you. One moment for our next question. Our next question comes from the line of Mani Foroohar from Leerink Partners. Your line is open.
Speaker #4: study as well L-7 .
Speaker #1: Thank you . next One moment for our question . Our next question comes from money Foroohar from Partners . of Urine Leerink is open .
[Analyst] (Leerink): Hey, guys. Thanks for taking the question. I had a little bit of technical issue there earlier, so not sure if this was asked earlier. But could you give us a breakdown of the EAP versus non-EAP patients out of the 100+ prescriptions? And how should we think about the total pool of EAP patients rolling on to commercial drug? Is that - is there a tail of that remaining? And I have a follow-up question.
Mani Foroohar: Hey, guys. Thanks for taking the question. I had a little bit of technical issue there earlier, so not sure if this was asked earlier. But could you give us a breakdown of the EAP versus non-EAP patients out of the 100+ prescriptions? And how should we think about the total pool of EAP patients rolling on to commercial drug? Is that - is there a tail of that remaining? And I have a follow-up question.
Speaker #6: guys . Hey , Thanks for taking the question . I had a technical little bit of staff earlier , so this was with their not sure if asked earlier , could you give us a but breakdown of the EP versus non EP out of the 100 plus how should we think prescriptions , and about the total pool of rolling patients onto Is drug ?
James Hamilton: Thanks for your question, Manny. This is Andy. At this time, we're not gonna provide any further details aside from the previous remarks, which, you know, the vast majority of patients are APOC3 naive. That gives us a lot of optimism about our ability to identify and diagnose both genetically confirmed and clinically diagnosed FCS patients. And again, with respect to the balance, we do see that fairly evenly split between those patients transitioning off of the expanded access program and those that are coming via switch.
James Hamilton: Thanks for your question, Manny. This is Andy. At this time, we're not gonna provide any further details aside from the previous remarks, which, you know, the vast majority of patients are APOC3 naive. That gives us a lot of optimism about our ability to identify and diagnose both genetically confirmed and clinically diagnosed FCS patients. And again, with respect to the balance, we do see that fairly evenly split between those patients transitioning off of the expanded access program and those that are coming via switch.
Speaker #6: commercial that is tale of that there a remaining ? And I have a follow up question
Speaker #4: question .
Speaker #4: This is your this time , we're not Andy . At going to further provide details any aside . Thanks for previous which from the the remarks , majority of vast are patients apoc3 naive gives us .
Speaker #4: a lot of optimism about our That to ability identify and diagnose both confirmed and genetically clinically diagnosed FCS again , with patients . And to respect the balance , we do fairly see that evenly split those patients transitioning between off of the expanded access and those that program are coming via switch .
[Analyst] (Leerink): Okay, that's helpful. As a separate question, what are the expectations we should have over the next 12 to 18 months around potential data sets, admittedly perhaps early, on novel tissue types and further expansion of the platform?
Mani Foroohar: Okay, that's helpful. As a separate question, what are the expectations we should have over the next 12 to 18 months around potential data sets, admittedly perhaps early, on novel tissue types and further expansion of the platform?
Speaker #6: Okay . That's helpful . And then a separate question are the what expectations we should the have over next 12 to 18 months ?
Speaker #6: potential Around sets up ? data perhaps Admittedly , on , novel early types and tissue further expansion of the platform .
James Hamilton: That's a good question, Manny. We've not given any guidance to that at this point. I think we have enough exciting stuff, you know, with ARO-INHBE, ARO-ALK7 data, you know, with initial ARO-MAPT data, with initial ARO-DIMER data, with ARO-C3 and ARO-CFB reading out, you know, with Redemplo sales, you know, that we feel pretty good about those things. But you know us, Manny, we are always developing the platform, and we are always expanding it as such. So I can't-- It's possible that you may hear, you know, something about where we're going with the platform as well as maybe new candidates within the existing platform.
James Hamilton: That's a good question, Manny. We've not given any guidance to that at this point. I think we have enough exciting stuff, you know, with ARO-INHBE, ARO-ALK7 data, you know, with initial ARO-MAPT data, with initial ARO-DIMER data, with ARO-C3 and ARO-CFB reading out, you know, with Redemplo sales, you know, that we feel pretty good about those things. But you know us, Manny, we are always developing the platform, and we are always expanding it as such. So I can't-- It's possible that you may hear, you know, something about where we're going with the platform as well as maybe new candidates within the existing platform.
Speaker #4: That's a good question . We've any not given guidance for that at this point . I think we have enough exciting stuff , you know , with , you more know , with now inhibiting seven data with with initial initial data , with dimer data with and Shasta three four reading out know , with , you sales , you know , that we feel pretty good about those things .
Speaker #4: you But , know , know us , Manny you are always , we platform developing the and we are always expanding it . Besides .
Speaker #4: and so can't And it's possible that you may you may hear something about going with the platform as well as maybe new candidates new the existing platform .
James Hamilton: I just can't give you any guidance on when that might be. I apologize.
James Hamilton: I just can't give you any guidance on when that might be. I apologize.
Operator: Thank you. One moment for our next question. Our next question will come from the line of Madison El-Saadi from B. Riley. Your line is open.
Operator: Thank you. One moment for our next question. Our next question will come from the line of Madison El-Saadi from B. Riley. Your line is open.
Speaker #4: can't give you I just any guidance on I might be . apologize .
Speaker #1: Thank you . One moment for our next question . Our next question from the will come line of Madison , al-Saadi from V Riley .
[Analyst] (B. Riley Securities): Hi, guys. Thanks for taking our question. On the 100 prescriptions you mentioned, I'm curious, how many of those do you expect to be converted to paid drug? And how long does it take to get from prescription to drug in body? And then relatedly, how should we think about the pace of both patient onboarding and competitive switching? Is this kind of a leading indicator for SHTG dynamic? Thanks.
Madison El-Saadi: Hi, guys. Thanks for taking our question. On the 100 prescriptions you mentioned, I'm curious, how many of those do you expect to be converted to paid drug? And how long does it take to get from prescription to drug in body? And then relatedly, how should we think about the pace of both patient onboarding and competitive switching? Is this kind of a leading indicator for SHTG dynamic? Thanks.
Speaker #1: The line is open .
Speaker #12: Hi , guys . Thanks for taking our question . On prescriptions , you the 100 mentioned , I'm curious how many of those do you expect to be converted to ?
Speaker #12: To pay and drug how long does it take to get from prescription to drug ? body Drug and ? And then , relatedly , how should we think about the pace of both patient onboarding and competitive switching ?
Speaker #12: Is this kind of a leading indicator for a dynamic ? Thanks .
James Hamilton: Thanks, Madison. Happy to comment.
James Hamilton: Thanks, Madison. Happy to comment.
Andy Davis: ...What we're seeing are really high quality prescriptions in the sense that we believe these prescriptions truly represent either genetically confirmed or clinically diagnosed FCS patients. So we do have high confidence that a significant proportion of those prescriptions will, in time, translate into, you know, drug shipments and drug in patients. As far as the time it takes from prescription to drug shipment, again, that does vary by patient, by insurance, and by prior authorization. But I would say in general, we're able to do that within just a couple of weeks from prescription to patient receiving drug. So I've been incredibly pleased with the patient identification, including both genetic and clinically diagnosed, and incredibly pleased with the operational execution from the team in converting prescriptions to shipped medicine.
Andy Davis: ...What we're seeing are really high quality prescriptions in the sense that we believe these prescriptions truly represent either genetically confirmed or clinically diagnosed FCS patients. So we do have high confidence that a significant proportion of those prescriptions will, in time, translate into, you know, drug shipments and drug in patients. As far as the time it takes from prescription to drug shipment, again, that does vary by patient, by insurance, and by prior authorization. But I would say in general, we're able to do that within just a couple of weeks from prescription to patient receiving drug. So I've been incredibly pleased with the patient identification, including both genetic and clinically diagnosed, and incredibly pleased with the operational execution from the team in converting prescriptions to shipped medicine.
Speaker #6: Thanks , Madison .
Speaker #4: Happy to to comment are . What we're really high quality seeing prescriptions in the sense that we believe these prescriptions truly represent either genetically confirmed or clinically diagnosed FCS patients .
Speaker #4: So we do have high confidence that a significant proportion of those prescriptions will , in time , translate into drug shipments and in in drug .
Speaker #4: As far patients as the time takes it prescription from to drug shipment , again , that does by vary By insurance and and by But I would general , say in we're able to do authorization .
Speaker #4: within that just a couple of weeks from prescription to patient receiving drug . So I've been incredibly pleased with the patient identification , including genetic and clinically both diagnosed and incredibly pleased with the operational from the team in converting prescriptions medicine .
Christopher Anzalone: I also just broadly, you know, be careful about reading too much into where we are right now. We've only been actively in market for 10 weeks now. And so we're still working with payers, we're still working with, you know, with physicians to get comfortable prescribing this. We're still, you know, informing and educating patients and prescribers about the medicine. So we have a long way to go. You know, let's see where we are more towards the end of the year. We have a pretty small sample set at this point.
Chris Anzalone: I also just broadly, you know, be careful about reading too much into where we are right now. We've only been actively in market for 10 weeks now. And so we're still working with payers, we're still working with, you know, with physicians to get comfortable prescribing this. We're still, you know, informing and educating patients and prescribers about the medicine. So we have a long way to go. You know, let's see where we are more towards the end of the year. We have a pretty small sample set at this point.
Speaker #4: also just And broadly , you know , be careful about about reading too much into where we are right now . We've only been in actively in market for ten weeks now .
Speaker #4: And so so we're still working with with payers . We're still we're still working with , you know , with physicians to get comfortable prescribing this .
Speaker #4: still , We're know , you know , informing and educating patients and prescribers about the medicine . So we have way to a long You know , let's see where we are .
Operator: Thank you. I'm not showing any further questions in the queue. I would now like to turn it back over to Chris for any closing remarks.
Operator: Thank you. I'm not showing any further questions in the queue. I would now like to turn it back over to Chris for any closing remarks.
Speaker #4: go . More towards the end of the year . We have a pretty small sample set at this point .
Andy Davis: Thanks, everyone, for joining us today. We look forward to speaking with you next quarter.
Andy Davis: Thanks, everyone, for joining us today. We look forward to speaking with you next quarter.
Speaker #1: you . I'm not Thank showing further questions in any the queue . I would now like to turn it back over to Chris for any closing remarks .
Operator: Thank you for your participation in today's conference. This does conclude the program. You may now disconnect. Everyone, have a great day.
Operator: Thank you for your participation in today's conference. This does conclude the program. You may now disconnect. Everyone, have a great day.
Speaker #4: Thanks , everyone for joining us today . We look forward to speaking with you next quarter .
Speaker #1: Thank you for your participation in today's conference . This does conclude the program . You may now disconnect . day a great Everyone have .
