Q1 2024 Cytokinetics Inc Earnings Call
Operator: Good afternoon and welcome, ladies and gentlemen, to Cytokinetics' first quarter 2024 conference call. At this time, I would like to inform you that this call is being recorded and that all participants are in a listen-only mode. At the company's request, we will open the call for questions and answers after the presentation. We will allow only one question per participant and ask that you adhere to this request. I will now turn the call over to Diane Weiser, Cytokinetics' Senior Vice President of Corporate Affairs. Please go ahead.
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Speaker Change: Good afternoon, and welcome ladies and gentlemen, societal kinetics first quarter 'twenty 'twenty four conference call.
Speaker Change: At this time I would like to inform you that this call is being recorded and that all participants are in a listen only mode.
Diane Weiser: At the company's request, we will open the call for questions and answers after the presentation.
Speaker Change: We will allow for only one question per participant and ask that you would hear to this request.
Operator: I will now turn the call over to Diane Weiser Cytogenetics Senior Vice President of Corporate Affairs. Please go ahead.
Diane Weiser: Good afternoon, and thanks for joining us on the call today. Robert Blum, President and Chief Executive Officer, will begin with an overview of the corridor and recent developments. Fady Malik, EVP of R&D, will provide updates related to AFI-CAMPTN focused on Sequoia HCM, Forrest HCM, and recent interactions with FDA. Stuart Kupfer, SVP and Chief Medical Officer, will provide additional updates regarding the ongoing clinical trials of AFI-CAMPTN, Maple HCM, and Acacia HCM, and will also discuss the progression of CK586 and our emerging pipeline.
Diane Weiser: Good afternoon, and thanks for joining us on the call today, Robert Blum, President and Chief Executive Officer will begin with an overview of the quarter and recent developments Daddy Malik E. V. P of R&D will provide updates related to IP campaign focus just acquire HCM as far as HCM and recent interactions with FDA.
Diane Weiser: Kutzer SVP and Chief Medical Officer will provide additional updates regarding the ongoing clinical trials of assay Camden Maple HCM and Acacia HCM and will also discuss progression of CK sided sex and our emerging pipeline, Andrew Kellogg's, EVP and Chief commercial officer will speak about commercial readiness activities.
Diane Weiser: Andrew Callos, EVP and Chief Commercial Officer, will speak about commercial readiness activities for AFI-CAMPTN. Sung Lee, our new EVP and Chief Financial Officer, is with us today, but just listening alongside us, as this is his first day at Cytokinetics. Robert Wong, VP and Chief Accounting Officer, will provide a financial overview of the past quarter.
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Diane Weiser: Lee, our new EVP and Chief Financial Officer is with US today, but just listening alongside US as this is his birthday in cytogenetics, Robert Wong VP and Chief Accounting Officer will provide a financial overview of the past quarter and finally, Robert Brown will review, our corporate development strategies before closing the call by reviewing.
Expected key milestones for the year. Please note that portions of the following discussion including responses to questions contain statements that relate to future events and performance rather than historical facts and constitute forward looking statements. Our actual results may differ materially from those projected in these forward looking statements additional information concerning.
Diane Weiser: And finally, Robert Blum will review our corporate development strategies before closing the call by reviewing expected key milestones for the year. Please note that portions of the following discussion, including our responses to questions, contain statements that relate to future events and performance, rather than historical facts and constitute forward-looking statements. Our actual results might differ materially from those projected in these forward-looking statements. Additional information concerning factors that could cause our actual results to differ materially from those in these forward-looking statements is included in our SEC filings, including our current report regarding our first quarter 2024 financial results filed on Form 8K that was furnished to the SEC today. We undertake no obligation to update any forward-looking statements after this call. Now, I will turn the call over to Robert. Thank you, Diane.
Speaker Change: Factors that could cause actual results to differ materially American. These forward looking statements contained in our SEC filings, including our current report regarding our first quarter 2024 financial result filed on form 8-K that was furnished to the SEC stay we undertake no obligation to update any forward looking statements. After this call.
Now I will turn the call over to Robert Thank you Diane and thanks for joining us on the call today and.
Robert I. Blum: In addition, we are laser-focused on regulatory submissions in the second half of the year, continued conduct of the ongoing clinical trials program, accelerating our commercial readiness activities, and further expanding our pipeline. While the top-line results of Sequoia HCM announced last December included a comprehensive, high-level view of the safety and efficacy of afecamtin, a priority of ours is to present more fully the results from Sequoi Recently, we announced that that forum will be the European Society of Cardiology's Heart Failure 2024 Congress in Lisbon next week, Monday, May 13, where we will have three late-breaking clinical trial presentations related to Sequoia HCM. Along with the primary results, we have two other late breakers with data from additional analyses from Sequoia HCM, on which Fady will elaborate.
Robert I. Blum: Thank you, Diane. And thanks for joining us on the call today. In the first quarter, we made substantial progress executing on our muscle biology-focused portfolio, anchored by the broad development program of AFI-CAMP-2. On the heels of positive top-line results from Sequoia HCM, our pivotal Phase III clinical trial of afecamptin in patients with obstructive hypertrophic cardiomyopathy, we are running at full throttle in preparation for an ambitious set of presentations and publications scheduled to occur over the course of the year.
Robert: In the first quarter, we made substantial progress executing on our muscle biology focused portfolio anchored by the broad development program of Alfie Camden.
Robert I. Blum: On the heels of positive topline results from Sequoia HCM, our pivotal phase III clinical trial of Alfie Camden in patients with obstructive hypertrophic cardiomyopathy, we are running at full throttle in preparation for an ambitious set of presentations and publications scheduled to occur over the course of the year.
Robert: In addition, we are laser focused on regulatory submissions in the second half of the year continued conduct of the ongoing clinical trials program.
Robert: Celebrating our commercial readiness activities and further expanding our pipeline.
Robert: While top line results of the core HCM announced last December included a comprehensive high level view of the safety and efficacy of Alfie Camden, a priority of ours is to present more fully the results from Sequoia HCM at a major medical meeting.
Robert: Lee, we announced but that form will be the European Society of Cardiology Heart failure 2020 for Congress in Lisbon next week Monday May 13, where we will have three late breaking clinical trial presentations related to Sequoia HCM.
Robert: Along with the primary results we have two other late breakers with data from additional analyses from Sukhoi HCM on which study will elaborate.
Robert I. Blum: He will also lay out the steady stream of presentations and publications that we anticipate sharing related to Affey-Campden throughout 2024, as we believe will nicely elaborate on its Next in Class profile. During the first quarter, we also engaged meaningfully with FDA ahead of our planned submission of an NDA for Aficamptin in the third quarter of this year. We convened two meetings with FDA in the month of February, including a first meeting to review the results of Sequoia HCM and a second pre-NDA meeting to cover specific topics related to our submission.
Robert: He will also lay out the steady stream of presentations and publications that we anticipate sharing related to <unk> throughout 2024, because we believe we're nicely elaborate on its next in class profile.
Robert I. Blum: During the first quarter, we also engage meaningfully with FDA ahead of our planned submission of an NDA for <unk> Camden in the third quarter of this year, we convened to meetings with FDA in the month of February including our first meeting to review the results of Sukhoi HTM and a second pre NDA meeting to cover specific.
Robert: Topics related to our submission we are pleased with the agency's feedback and we look forward to additional discussions to occur this quarter, which will address specific questions. We are posing relating to potential rems scenarios. We've also been preparing for our planned submission of an MAA.
Robert I. Blum: We are pleased with the agency's feedback, and we look forward to additional discussions to occur this quarter, which will address specific questions we are posing relating to potential REMS scenarios. We've also been preparing for our planned submission of an MAA to EMA, expected in the fourth quarter of this year. Moreover, in 2024, many other work streams and activities were pushed into the next phase of execution, notably commercial readiness activities, which Andrew will speak about in more detail.
Robert: With EMA expected in the fourth quarter of this year.
Robert I. Blum: Moreover, in 2020 for many other work streams and activities were pushed into the next phase of execution now.
Robert: Notably commercial readiness activities, which Andrew will speak about in more detail.
Robert I. Blum: And while Sequoia HCM represents the forward edge of the Phase III development program for AFI-CAMPTN, we now have two other ongoing Phase III clinical trials evaluating AFI-CAMPTN, which each represent additional opportunities to expand the clinical evidence use case and hopefully reach more patients in need. Of course, we're also continuing our ongoing open-label extension study, FOREST-HCM, to collect longer-term data on the safety and efficacy of AFI-CAM-2. And, as Stuart will elaborate, MAPLE-HCM, the Phase III clinical trial of afecamtin as monotherapy compared to metoprolol as monotherapy, is expected to complete enrollment in the third quarter. This trial will provide an evidence-driven answer to the question clinicians are beginning to ask with the emergence of cardiac myosin inhibitors, that is, which drug should we initiate first?
Robert: And whilst the core HCM represents the Ford edge of the Phase III development program for <unk> Camden, We now have two other ongoing phase III clinical trials evaluating <unk> kimpton, which each represent additional opportunities to explain expand the clinical evidence used case and hopefully reach more patients.
Robert I. Blum: The need of.
Robert I. Blum: Of course, we're also continuing our ongoing open label extension study forest HCM to collect longer term data on the safety and efficacy of Alpha Kempton.
Robert I. Blum: And as Stuart will elaborate maple HCM, the phase III clinical trial of FP, Camden as monotherapy compared to metoprolol as monotherapy is expected to complete enrollment in the third quarter. This trial will provide an evidence driven answer to the question clinicians are beginning to ask with the emergence.
Robert: Of cardiac myosin inhibitors that is which drug do we initiate first maple HCM may be an important opportunity for <unk> kimpton as we hope it may inform a critical change in treatment practice.
Robert I. Blum: MAPLE-HCM may be an important opportunity for afecamtin as we hope it may inform a critical change in treatment practice, as could be outlined in emerging guidelines. Stuart will also share an update on ACACIA-HCM, our Phase 3 clinical trial of afecamtin in patients with non-obstructive HCM. And he'll also describe a third late-stage clinical trial called CEDAR-HCM, which is now open to enrollment in a pediatric population of patients with OHCM.
Robert: B outlined in emerging guidelines.
Robert I. Blum: Stuart will also share an update on Acacia HTM, our phase III clinical trial of <unk> in patients with non obstructive HCM and I'll also describe a third late stage clinical trial called Cedar HCM, which is now open to enrollment in a pediatric population of patients with O HCM.
Robert I. Blum: We're especially pleased to maintain a strong financial position at the end of the first quarter alongside our maturing R&D program. As I'll speak to in more detail later in this call, we believe that we are in an advantageous position in terms of our options for accessing diversified capital to further fuel our science for the benefit of patients and to deliver increasing shareholder value. And lastly, we were pleased to recently announce that we had finalized our search for a new CFO, and as you hopefully saw from the 8K we issued, I'm pleased to welcome Sung Lee to our team.
Robert I. Blum: We were especially pleased to maintain a strong financial position at the end of the first quarter alongside of our maturing R&D programs as I'll speak to in more detail later in this call. We believe that we are in an advantaged position in terms of our options for accessing diversified capital.
Robert I. Blum: To further fuel our science for the benefit of patients and to deliver increasing shareholder value.
Robert I. Blum: When we began our search, we had several key criteria for the ideal candidate, including previous experience in both large and mid-sized biopharma companies, global commercial finance experience, and a track record of innovative financing deals. Sung brings all of that and more, and we couldn't be happier to have him join our team. Prior to cytokinetics, Sung was CFO of Vir Biotechnology and also of Morphosis AG and Sangamo Therapeutics. Previously, he built his career expertise over 14 years at Gilead, where he served in various roles obtaining leadership expertise in tax, accounting, operations, and IR.
Robert: And lastly, we were pleased to recently announce that we had finalized our search for a new CFO and as you hopefully saw from the 8-K, we issued I am pleased to welcome sung Lee to our team. When we began our search we had several key criteria for the ideal candidate.
Robert: Including previous experience in both large and midsized biopharma companies.
Robert: Global commercial finance experience and a track record of innovative financing financing deals. Some brings all of that and more and we couldnt be happier to have him join our team prior to side of kinetic sung was CFO of Vir Biotechnology and also morphosis AG and Sangamo third.
Robert I. Blum: <unk>.
Robert: Previously he built his career expertise over 14 years at Gilead, where he served in various roles obtaining leadership expertise in tax accounting operations and IR.
Robert I. Blum: You will hear from Sung on our Q2 earnings call, and for those of you who don't already know him, you'll soon have a chance to meet him at upcoming conferences and one-on-one meetings. And with that, I'll turn the call over to Fady, please.
Robert: You will hear from song Lin, Our Q2 earnings call and for those of you who don't already know him you will still have a chance to meet him at our upcoming conferences and one on one meetings and with that I'll turn the call over to fatty please.
Fady Ibraham Malik: Robert, top of mind for all our late-breaking clinical trial presentations next week at Heart Failure 2024 in Lisbon. The presentations include, of course, the primary results of Sequoia HCM, which will provide a complete view of the results related to the baseline characteristics, primary and secondary endpoints, subgroup analyses, and select exploratory endpoints. There will also be two additional late breakers on other data analyses from the trial.
Fatty: Thanks, Robert top of mind for all our late breaking clinical trial presentations next week at heart failure 2024 in Lisbon.
Fatty: Presentations include of course, the primary results of Sequoia, HCM, which will provide a complete view of the results related to the baseline characteristics primary and secondary endpoints subgroup analyses and select exploratory endpoints.
Fady Ibraham Malik: And there will also be two additional late breakers.
Fady Ibraham Malik: One will elaborate on the dosing and safety experience with Sequoia HCM, and another is a deep dive into the improvements in exercise capacity affected by affecantin based on a detailed review of the CPET data. These presentations will not only expand on what was in the top-line press release but go further in providing an in-depth look at this very rich data set and its implications for clinical practice. As Robert mentioned, our presentations next week represent only the beginning of our plan to fully dissect the results from Sequoia HCM and build a comprehensive picture of the differentiated profile that continues to emerge for apicamptin.
Fatty: Another data analysis from the trial.
Robert: John will elaborate on the dosing and safety experienced since equate Sam and another is a deep dive into the improvement in exercise capacity effected by assay Campton based on a detailed review of the C pet data.
Robert: These presentations will not only expand on what was in the top line press release, but go further and providing an in depth look at this very rich dataset and its implications for clinical practice.
Fady Ibraham Malik: As Robert mentioned, our presentations next week represent only the beginning of our plan to fully this sector results from Sequoia ACM and build a comprehensive picture of the differentiated profile that continues to emerge for Abbvie campton.
Fady Ibraham Malik: We have an aggressive plan that starts with HFA and lays out, over the course of the year, a series of presentations and publications that will further elaborate on the efficacy and safety of AfiCampus. You can expect presentations at the European Society of Cardiology, the Heart Failure Society of America, and the American Heart Association meeting, as well as accompanying publications in leading medical journals. These include deeper dives into the echocardiographic, Kansas City cardiomyopathy questionnaire data, CMR data, cardiac remodeling, and biomarker data, as well as an integrated analysis of efficacy.
Speaker Change: We have an aggressive plan that starts with HSA and lays out over the course of the year a series of presentations and publications that will further elaborate on the efficacy and safety of Appia Captain you can expect presentations at the European Society of Cardiology Heart failure Society of America, and American Heart Association meet.
Robert: <unk> as well as the accompanying publications in leading medical journals.
Robert: These include deeper dives into the Echocardiographic, Kansas City Cardiomyopathy questionnaire data.
Fady Ibraham Malik: <unk> data cardiac remodeling and biomarker data as well as an integrated analysis of efficacy, we look forward to sharing more as the year progresses.
Fady Ibraham Malik: We look forward to sharing more as the year progresses. Shifting to Forest HCM, last month at ACC, we shared additional 48-week data from 46 patients with obstructive HCM and Forest HCM. We show that treatment with avicamptin is associated with sustained improvements in resting and val-salvo left ventricular outflow tract gradient, NYHA functional class, NT-ProBNP, and measures of cardiac structure and function. Furthermore, occasions of low left ventricular ejection fraction were very infrequent and were not associated with treatment interruptions or heart failure events.
Fady Ibraham Malik: Shifting to forest HCM last month at ACC, we shared additional 48 week data from 46 patients with obstructive HCM in forest HCM.
Robert: It showed that treatment with Abbvie Camden is associated with sustained improvements in resting in valsalva left ventricular outflow tract gradient.
Fady Ibraham Malik: <unk> functional class NT pro BNP and measures of cardiac structure and function.
Robert: Furthermore, occasions of left ventricular low leptin curricular ejection fraction.
Fady Ibraham Malik: Very infrequent and we're not.
Robert: He added with treatment interruptions or heart failure events.
Fady Ibraham Malik: These data continue to reinforce the safety and efficacy of longer-term treatment with apicamptin and OHCM. With nearly 300 patients currently enrolled in forced HCM, we look forward to reporting more longer-term data in the future. Importantly, during the quarter, we had many opportunities to engage with KOLs and treating clinicians in the community to discuss the results generated to date from the Program for Aficampus. Feedback has been incredibly positive, and we believe that once presented and published, the full results will reaffirm this enthusiasm. Specifically, our therapeutic medical science liaisons have interacted with over 500 healthcare professionals and completed physician practice profiling with HCM treatment programs.
Robert: These data continue to reinforce the safety and efficacy of longer term treatment with Abbvie Captain and O HCM with nearly 300 patients currently enrolled in forced HCM, we look forward to reporting more longer term data in the future.
Robert: Importantly, during the quarter, we had many opportunities to engage with kols.
Robert: And trading conditions in the community to discuss the results generated to date from the program for <unk> in Campton feedback.
Robert: Feedback has been incredibly positive and we believe that once presented and published the full results will reaffirm this enthusiasm.
Robert: Specifically, our therapeutic medical science liaisons have interacted with over 500 health care professionals and completed profiling of physician practices with HCM treatment programs at.
Fady Ibraham Malik: At the same time, our managed health care medical science liaisons engaged integrated delivery networks, or IDNs, around the results of Sequoia HCM, and finalized work to build our payer clinical value narrative. On the regulatory front, as Robert mentioned, during the first quarter, we held two meetings with FDA ahead of our NDA submission. During the second quarter, we plan to engage FDA once again, this time for a type B meeting focused specifically on potential risk mitigation.
Robert: At the same time, our managed healthcare medical science liaisons engaged integrated delivering networks are idms around the results of Sequoia HCM and Finalised work to build our payer clinical value narrative.
Fady Ibraham Malik: On the regulatory front as Robert mentioned during the first quarter, we held two meetings with FDA ahead of our NDA submission.
Robert: During the second quarter, we plan to engage FTA. Once again this time for a type b meeting focused specifically on potential risk mitigation.
Fady Ibraham Malik: While this is a topic that came up in both of our previous meetings, this next engagement affords us the opportunity to elaborate on the safety and pharmaceutical properties of Aficam, discuss their potential impact on different approaches to manage risk, and gain insight into FDA's perspective on these matters. In these discussions with FDA, our position is that the benefit-risk profile of apicamptin merits an approach to risk mitigation that's reflective of the safety profile of apicamptin as demonstrated in Sequoia HCM and Forest HCM.
Robert: While this is a topic that came up in both of our previous meetings. This next engagement affords us the opportunity to elaborate on the safety and pharmaceutical properties of Abbvie Captain.
Robert: Discuss their potential impact on different approaches to manage risk and gain insight into fda's perspective on these matters.
Robert: In these discussions with FDA, our position is that the benefit risk profile of Appia campton merits and approach to risk mitigation. That's reflective of the safety profile of <unk> Kimpton is demonstrated and so core HCM and forest HCM.
Fady Ibraham Malik: Altogether, we remain optimistic and believe these discussions will inform our proposal for a differentiated approach to risk mitigation in our NDA. As we've previously shared, we're planning for a rolling submission for the NDA, which will provide FDA the opportunity to begin their review of completed modules. We expect to begin and complete the submission during the third quarter. As I'm sure you'll appreciate, there is a tremendous amount of work that goes into an NDA, and our teams are working diligently towards this very important milestone for the company.
Robert: Together, we remain optimistic and believe these discussions will inform our proposal for a differentiated approach to risk mitigation and our NDA.
Robert: As we've previously shared we are planning for a rolling submission for the NDA, which will provide FDA the opportunity to begin their review of completed modules, we expect to begin and complete the submission during third quarter.
Robert: As I'm sure you will appreciate there is a tremendous amount of work that goes into an NDA and our teams are working diligently towards this very important milestone for the company.
Fady Ibraham Malik: Meanwhile, we're also preparing our marketing application, which we expect to submit to EMA in the fourth quarter of this year, and are coordinating with our partners Yixing and China to support their plans to support an NDA as well. During the quarter, we're pleased to welcome a new head of regulatory for Europe, who will lead regulatory strategy and product registration in Europe and support the launch readiness activities. While SCCOI HCM is taking center stage at the moment, it's followed and supported by additional clinical trials and development programs for apicamptin that we believe have the potential to expand the utility of cardiac myosin inhibitors. I'll hand it over to Stuart to elaborate more on these additional clinical trials, as well as provide an update on our earlier stage clinical development pipeline.
Robert: Meanwhile, we're also preparing our marketing application.
Robert: We expect to submit to EMA in the fourth quarter of this year and are coordinating with our partner <unk> Sheng in China to support their plans to support an NDA as well.
Robert: During the quarter, we are pleased to welcome a new head of regulatory for Europe will lead regulatory strategy and product registration in Europe and support the launch readiness activities.
Fady Ibraham Malik: While its core HCM is taking center stage at the moment, it's followed and supported by additional clinical trials and development program for rapid Kimpton that we believe have the potential explained to expand the utility of cardiac myosin inhibitors.
Robert: I'll hand, it over to Stuart to elaborate more on these additional clinical trials as well as provide an update on our earlier stage clinical development pipeline.
Stuart Kupfer: I'll start with our two ongoing Phase 3 clinical trials, MAPLE-HCM and AKESHA-HCM. We believe both of these trials have the opportunity to expand the potential benefit of atucampin to additional patients with obstructive and non-obstructive HCM and to improve affect and treatment guidance. Maple HCM, which is evaluating the potential superiority of apicampin as monotherapy compared to metoprolol as monotherapy in patients with obstructive HCM, is on track to complete enrollment in the third quarter. Over 75% of our global sites are activated, and we're expecting sites in China and South America to join Maple HCM.
Stuart: Thanks Patty.
Stuart: I'll start with our two ongoing phase III clinical trials Maple HCM and Acacia HCM. We believe both of these trials have the opportunity to expand the potential benefit of that to Camden, two additional patients with obstructive and non obstructive HCM and to elevate asking Camden and treatment guidelines.
Stuart Kupfer: Naval HCM, which is evaluating the potential superiority of asking Camden as monotherapy compared to <unk> monotherapy in patients with obstructive HCM is on track to complete enrollment in the third quarter over.
Robert: Over 75% of our global sites are activated and we are expecting sites in China, and South America to join Naval HCM.
Stuart Kupfer: By the end of the second quarter, we expect to be at approximately 50% of target enrollment and foresee a large increase in enrollment in the third quarter. The results from Sequoia HCM have provided a strong tailwind for enrollment and our ongoing trials, including Maple HCM. And we've observed strong enthusiasm from sites for completing the trial and getting us across the finish line.
Robert: By the end of the second quarter, we expect to be at approximately 50% of target enrollment and foresee a large increase in enrollment in the third quarter.
Stuart Kupfer: The results from Sequoia HCN and provided a strong tailwind for enrollment in our ongoing trials, including Maple HCM.
Stuart Kupfer: And we have observed strong enthusiasm from sites for completing the trial and getting us across the finish line.
Stuart Kupfer: We expect MapleHCM to read out in 2025, around the time when we hope to be commercially launching it, and a positive result will potentially position afecampin in guidelines for use as first-line therapy in obstructive HCM. Acacia HCM, the pivotal phase three clinical trial of aficampin in patients with symptomatic non-obstructive HCM, is also entering what we will expect to During the second quarter, we meet with many of our investigators and study staff at their sites around the world, and we recently held a successful investigator meeting in San Francisco.
Robert: We expect Naples, San to read out in 2025 around the time, when we hope to be commercially launching at the camp.
Stuart Kupfer: And if positive will provide the evidence space potentially positioned Camden and guidelines for use as first line therapy in obstructive HCM.
Robert: Acacia HCM the pivotal phase III clinical trial of <unk> in patients with symptomatic non obstructive HCM.
Robert: Also answering what we will expect to be a rapid phase of site activation and enrollment.
Robert: During the second quarter or meeting with many of our investigators and study staff at their sites around the world and we recently held a successful investigator meeting in San Francisco.
Stuart Kupfer: Given positive results from Cohort 4 of Redwood HCM, clinicians are enthusiastic about the promise of apicampin for the potential treatment of patients with non-obstructive HCM, and we look forward to continuing enrollment this year towards a goal of completion in 2025.
Robert: Given the positive results from cohort four of Redwood HCM <unk>.
Robert: Clinicians are enthusiastic about the promise of that be Camden for the potential treatment of patients with non obstructive HCM.
Robert: And we look forward to continuing enrollment this year towards the goal of completion in 2025.
Stuart Kupfer: Turning our attention to another important patient population, just this morning, we announced the start of yet another clinical trial of apicampin called Cedar-HCM, a randomized, double-blind, placebo-controlled trial and open-label extension evaluating the efficacy, pharmacokinetics, and safety of aficamptin in a pediatric population with symptomatic obstructive HCM. Cedar H&M will enroll two cohorts. Adolescents aged 12-17 years will be enrolled in the initial cohort.
Robert: Turning our attention to another important patient population just this morning, we announced the start of yet another clinical trial of <unk> called Cedar ACM.
Robert: Randomized double blind placebo controlled trial and open label extension evaluating the efficacy and pharmacokinetics and safety of ASIC Camden in a pediatric population with symptomatic obstructive HCM.
Robert: Okay, Cedar HCM will enrolled two cohorts.
Stuart Kupfer: A second, younger cohort of up to 10 children aged 6-11 years will begin enrollment after data from at least 20 adolescents supports safety and dose selection in the younger cohort. In the cohort of adolescents, the treatment regimen for apicampin will be the same as that for adults, that is once daily doses of 5 to 20 milligrams individually selected based on echocardiographic parameters.
Robert: Also only 40 adolescents, aged 12 to 17 years will be enrolled in the initial cohort.
Robert: Younger cohort of up to 10 children, aged six to 11 years will begin enrollment at the data from at least 20 adolescent patients supports safety and dose selection and the younger cohort.
Robert: In the cohort of adolescence with treatment regimen for Camden will be the same as that for adults.
Robert: That is once daily doses of 5% to 20 milligrams individually selected based on echocardiographic parameters.
Stuart Kupfer: Primary and secondary endpoints will be evaluated after 12 weeks of double-blind treatment. The primary endpoint is change in valsalva, the left ventricular outflow tract gradient, and secondary endpoints include change in resting gradient, pharmacokinetic measures, cardiac biomarkers, and symptoms. After 12 weeks of double-blind treatment, all patients will roll over into an open-label phase. While pediatric HCM may be rare, it's associated with a high risk of heart failure and serious arrhythmia. HCM tends to present similarly in children and adolescents as it does in adults, and is associated with shortness of breath, fatigue, and poor exercise tolerance, impacting overall quality of life.
Stuart Kupfer: Primary and secondary endpoints will be evaluated after 12 weeks of double blind treatment.
Robert: The primary endpoint is change in valsalva left ventricular outflow tract gradient.
Robert: And secondary end points include change in resting gradient pharmacokinetics current measures cardiac biomarkers and symptoms.
Robert: After 12 weeks of double blind treatment, all patients will roll over into an open label extension.
Robert: While <unk> pediatric HCM may be rare.
Robert: Associated with a high risk of heart failure and serious a revenues.
Robert: HCN tends to present similarly in children and adolescents as it does in adults and is associated with shortness of breath fatigue, and poor exercise tolerance impacting overall quality of life.
Stuart Kupfer: We're optimistic, given the results from Sequoia HCM in adults, that epicanthin may prove promising for this key segment of the HCM population. During the second quarter, we'll also be starting a phase one study to evaluate the pharmacokinetics, safety, and tolerability of apicampin in healthy Japanese participants, to advance our global clinical program in that geography. Additionally, during the first quarter, we advanced CK5A and another cardiac myosin inhibitor in development for the potential treatment of a subgroup of patients with heart failure with preserved ejection fraction or HEF test.
Robert: We're optimistic given the results from Sequoia HCM in adults that ft. Camden may prove promising for this key segment of the HCM population.
Robert: During the second quarter will also be starting a phase one study to evaluate the pharmacokinetics safety and tolerability of that the Camden in healthy Japanese participants to advance our global clinical program in that geography.
Robert: Additionally, during the first quarter, we advanced CK 586, and other cardiac myosin inhibitor in development for the potential treatment of a subgroup of patients with heart failure with preserved ejection fraction or <unk>.
Stuart Kupfer: Earlier today, we announced top-line data from the Phase 1 study of CK586, which showed that CK586 was safe and well tolerated in healthy participants with generally linear pharmacokinetics. These findings are supportive of advancing the program to a Phase II clinical trial, which we expect to begin in the fourth quarter of this year. We plan to present the data from the phase one study in more detail at a medical congress in the second half of the year. We believe CK586, which has a different mechanism of action than affecansin, has the potential to further unlock the potential biology of myosin modulation for patients with high unmet needs.
Stuart Kupfer: Earlier today, we announced top line data from the Phase one study of CK 586, which showed that CK 586 was safe and well tolerated in healthy participants with generally linear pharmacokinetics.
Robert: These findings are supportive of advancing the program into phase two clinical trial, which we expect to begin in the fourth quarter of this year.
Robert: We plan to present the data from the phase one study in more detail at the medical Congress in the second half of the year.
Robert: We believe CK 586, which has a different mechanism of action. The nasty Camden has the potential to further unlock the potential biology of myosin modulation for patients with high unmet need.
Stuart Kupfer: Finally, further work continued in the past quarter on our earlier stage programs that we expect to mature from our labs into the clinic later this year and next, extending beyond muscle contractility to the areas of muscle metabolism and energy. These emerging programs are an important part of our long-term vision as we continue to pioneer the field of muscle biology and pharmacology. And toward that end, we're pleased to be sponsoring a dedicated muscle biology symposium here in South San Francisco next Friday called CLIMB.
Robert: Finally further work continued in the past quarter on our earlier stage programs that we expect to mature from our labs into the clinic later this year and next extending beyond muscle contractility for the areas of muscle metabolism and energetics.
Robert: These emerging programs are an important part of our long term vision as we continue to pioneer this field of muscle biology and pharmacology.
Robert: And toward that end, we're pleased to be sponsoring a dedicated muscle biology symposium here in South San Francisco next Friday call Quad <unk>.
Stuart Kupfer: Contemporary Landscapes and Muscle Biology. This one-day event will bring together scientists, researchers, and emerging professionals to share innovative research in the field of muscle biology. The goal of this gathering is to foster collaboration, facilitate networking opportunities, and promote scientific interdisciplinary dialogue, with the ultimate goal of driving advancements in the understanding and treatment of muscle-related diseases and disorders. With that, I'll turn the call over to Andrew.
Robert: Contemporary landscapes in muscle biology.
Robert: This one day event will bring together scientists researchers and emerging professionals to share innovative research in the field of muscle biology.
Robert: The goal of this gathering is to foster collaboration facilitate networking opportunities and promote scientific interdisciplinary dialogue.
Robert: With the ultimate goal of driving advancements in the understanding of treatment and muscle related diseases and disorders.
Robert: With that I'll turn the call over to Andrew.
Andrew Callos: Thanks Stuart. After sharing top line results from Sequoia HCM at the end of last year, in the first quarter, we began the design and build phases of our go-to-market strategy. We worked to refine our market development campaign, which we plan to launch at HFSA later this year, and initiated the design and build of our comprehensive patient support services program, as well as our specialty distribution strategy. We continue to strengthen our commercial team with new hires, including a U.S. patient marketing lead and a head of Germany, which, if approved, is the first European country where we plan to launch Happy Camp.
Andrew: Thanks, Stuart after sharing top line results from Sequoia HCM at the end of last year and the first quarter. We began the design and build phases of our go to market strategies, we worked to refine our market development campaign, which we plan to launch at HSA later this year and.
Andrew Callos: And initiated the design and build of our comprehensive patient support services program as well as our specialty distribution strategy.
Andrew: We continue to strengthen our commercial team with new hires including a U S patient marketing lead and ahead of Germany.
Robert: Which if approved as the first European country, where we plan to launch happy Camden.
Andrew Callos: In 2024, hiring in our commercial organization and in Europe will remain modest, with more hiring expected in 2025 and 2026, gated to regulatory milestones in the US and EU and reimbursement levels on a country by country basis in the EU.
Andrew Callos: In 2020 for hiring in our commercial organization.
Robert: And in Europe will remain modest with more higher expected in 2025, and 2026 gated to regulatory milestones in the U S and EU and reimbursement levels on a country by country basis in the EU.
Andrew Callos: On that topic, we are pleased to continue to see more positive health technology assessments for the CMI class in the last several quarters, including from Germany, France, and the UK, which signals a potentially positive forecast for the future reimbursement of Aficampin across key countries if approved in Europe. During the quarter, we continued to build the value proposition for AFI-CAMPTN for various stakeholders, including payers and health plans, by generating data around Health, Economics, and Outcomes Research, or ATOR.
Robert: On that topic, we are pleased to continue to see more positive health technology assessments for the CMI class during the last several quarters, including from Germany, France, and the UK, which signals that potentially positive forecast for the future reimbursement of assay campaign across key countries if approved in Europe.
Andrew Callos: During the quarter, we continued to build the value proposition profit captain for various stakeholders, including payers and HCA is by generating data around health economics, and outcomes research or <unk> and.
Andrew Callos: In the first quarter and recent weeks, our team published and presented eight abstracts on topics including the impact of ethnicity, sex, region, and payer health care coverage on outcomes in HCN, medical therapy usage post septal myectomy, and long-term cost of care for patients with symptomatic obstructive HCN. Our findings around patient outcomes and costs related to obstructive HCM further underscore the clinical and economic unmet need for this growing Looking ahead, after we present and publish the primary results from Sequoia HCM next week, we will continue engagement with U.S. payers.
Robert: In the first quarter in recent weeks, our team published and presented 888.
Andrew Callos: <unk> abstracts on topics, including the impact of ethnicity checks region, and payer health care coverage on outcomes and ATM medical therapy usage post septal myectomy and long term cost of care for patients with symptomatic obstructive HCM.
Robert: Our findings around patient outcomes and costs related to obstructive HCM further underscore the clinical and economic unmet need for this growing patient population.
Andrew Callos: Our payer and medical account team began dialogue with every major payer in 2023, and beginning in Q3 of this year, we plan to initiate pre-approval information exchange with every major payer to review the results of Sequoia HCM so the payers understand the clinical meaningfulness of the results as well as the cost and outcome burden of obstructive HCM. I'm pleased with our progress for commercial readiness so far in 2024. As I've spoken about before, the obstructive ACM market has a highly concentrated customer base, which is typical of specialty cardiology.
Robert: Looking ahead after we present and publish the primary results from Sequoia ATM next week, we will continue engagement with U S. Payers are payer and medical account team began dialogue with every major payer in 2023 and beginning in Q3 of this year, we plan to initiate pre approval information exchange with every major payer.
Robert: To review the results of Sequoia HCM, so the payers understand the clinical meaningfulness of the results as well as the cost and outcome burden of obstructive HCM.
Robert: I am pleased with our progress for commercial readiness, so far in 2024 as I spoken to before the obstructive HCM market has a highly concentrated customer base, which is typical of a specialty cardiology. Unlike biopharma companies that have come before us who did not performed a market expectations. We believe our focus on specialty cardiology.
Andrew Callos: Unlike biopharma companies that have come before us who did not perform to market expectations, we believe our focus on specialty cardiology, anchored by Affie Kampen, should enable us to successfully reach the subset of cardiologists who treat approximately 80% of obstructive HCM patients. We believe that we are uniquely advantaged for success, and we're keeping our foot on the gas across our commercial readiness preparations this year. Just as our company successfully built a formidable R&D organization that cultivated the robust pipeline we have discussed today, we're well on our way to building an equally outstanding commercial organization poised to stand shoulder to shoulder with our highly respected R&D colleagues. Together, we are pioneering new frontiers of success driven by our shared vision and mission to help patients. And with that, I'll turn the call over to Robert Wong.
Robert C. Wong: Anchored by ASIC Hampton.
Robert C. Wong: <unk> successfully reached the subset of cardiology cardiologists to treat approximately 80% of the obstructive HCM.
Robert C. Wong: We believe that we are uniquely advantaged for success and we're keeping our foot on the gas across our commercial readiness preparations this year.
Robert: Just as our company successfully build a formidable R&D organization that cultivate a robust pipeline. We have discussed today, we're well on our way towards building an equally outstanding commercial organization poised to stand shoulder to shoulder with our highly respected R&D colleagues together, we're pioneering new frontiers of success driven by our shared vision.
Andrew Callos: <unk> and mission to help patients and with that I'll turn the call over to Robert Wong.
Robert C. Wong: Thanks, Andrew. We end the quarter with approximately $634.3 million of cash on the balance sheet, which represents two years of forward cash runway, including capital we expect to be available to us under our deal with Royalty Pharma upon satisfaction of conditions. Our first quarter 2024 R&D expenses increased to $81.6 million from $79.4 million in the first quarter of 2023, primarily due to spending on our cardiac myosin inhibitor programs offset by lower expenses for our skeletal muscle programs in the prior year.
Robert C. Wong: Thanks, Andrew we ended the quarter with approximately $634 3 million of cash on the balance sheet, which represents two years of forward cash runway, including capital, we expect to be available to us under our deal with royalty pharma upon satisfaction of conditions, our first quarter 2020 for R&D.
Robert C. Wong: <unk> increased to $81 6 million from $79 4 million in the first quarter of 2023, primarily due to spending on our cardiac myosin inhibitor programs offset by lower expenses for our skeletal muscle programs in the prior year, our first quarter 2020 for G&A expenses were <unk> 44.
Robert C. Wong: Our first quarter 2024 G&A expenses were $45.5 million, down from $49.7 million in Q1 2023, due primarily to higher pre-commercial expenses in the prior year. With that, I'll hand it back over to Robert Blum. Thank you, Robert.
Robert C. Wong: $5 5 million down from $49 7 million in Q1, 2023, due primarily to higher pre commercial expenses in the prior year with that I'll hand, it back over to Robert Blum.
Robert I. Blum: As you've heard, our cohesive biology, anchored by cardiac myosin inhibition, continues to drive shareholder value and growth as we execute on our plan to advance to the next tier of biopharmaceuticals. In the near term, we remain focused on elaborating on the positive results from Sequoia HCM in presentations and publications, as well as preparing for the successful launch of afecampin, if approved, in 2025 In 2024, we've been focused on parallel opportunities to sustain and grow our company by diversifying access to capital and strengthening our balance sheet, and we're prepared to execute on a series of primarily non-dilutive transactions, such as those associated with partnering and structured financial engineering.
Robert I. Blum: Hey, Robert.
Robert I. Blum: As you've heard our cohesive biology anchored by cardiac myosin inhibition continues to drive shareholder value and growth as we execute on our plan to advance to the next tier of biopharmaceutical companies in the near term we remain focused on elaborating on the positive results from Sequoia HCM in press.
Robert I. Blum: Patients in publications as well as preparing for the successful launch of <unk> Kimpton if I'm.
Robert I. Blum: Proved in 2025, while also continuing to invest in expanding our promising pipeline directed to our plans to build a specialty cardiovascular franchise.
Robert I. Blum: In 2024, we've been focused on parallel opportunities to sustain and grow our company by diversifying access to capital and strengthening our balance sheet and we're prepared to execute on a series of primarily non dilutive transactions such as comes with partnering and structured financial engineering.
Robert I. Blum: We hope to have more to say about these initiatives as they come to closure and commit to ensure not only access to diversified sources of capital but that we also focus on capital efficiencies as we deploy capital towards advancing our pipeline and emerging commercial business. Our priority remains our ongoing business development campaign for AFI Campton in Japan. During the quarter, we continued discussions with multiple parties, and with momentum, we hope to consummate a deal.
Robert I. Blum: We hope to have more to say about these initiatives as they come to closure and commit to ensure not only access to diversified sources of capital, but we also focus to capital efficiencies as we deploy capital towards advancing our pipeline and emerging commercial business.
Robert I. Blum: Our priority remains our ongoing business development campaign for <unk> in Japan during the quarter, we continued discussions with multiple parties and with momentum we hope to consummate a deal. We're also looking at restructuring and expanding current financial instruments and deals to lower our overall cost.
Robert I. Blum: We're also looking at restructuring and expanding current financial instruments and deals to lower our overall cost of capital and potentially monetize additional R&D progress. We expect to remain diligent about the different levers we can pull, as it could enable us in a principally non-equity dilutive manner to continue to augment shareholder value, and may also consider equity financing at the right time as part of a broader capital access strategy.
Robert I. Blum: Capital and potentially monetize additional R&D progress.
Robert I. Blum: We expect to remain diligent about the different levers, we can pull as could be enabling of us in our principally non equity dilutive manner to continue to augment shareholder value.
Robert I. Blum: And May also consider equity financing at the right time as part of our broader capital access strategy.
Robert I. Blum: Shaping our business conduct today and into the future is our Pledge to Corporate Responsibility. And recently, we were proud to release our second annual Corporate Responsibility Report, which highlights our actions and progress against our goals of keeping patients at the center of our work, advancing a high-integrity, diverse, and inclusive culture, and supporting sustainable communities. As we look at our longer-term goals and vision, which will be articulated in our Vision 2030 early next year, the company we aspire to be is a sustainable commercial enterprise with an enduring R&D organization. Our vision is to maintain our pioneering leadership in muscle biology and pharmacology, grounded in financial stewardship and doing what's right for the patients we serve. Now, I'll recap our upcoming milestones.
Robert I. Blum: Shaping our business conduct today and into the future is our pledge to corporate responsibility and recently, we were proud to release, our second annual corporate responsibility report, which highlights our actions and progress against our goals of keeping patients at the center of our work advancing our high integrity <unk>.
Robert I. Blum: First an inclusive culture and supporting sustainable communities.
Robert I. Blum: As we look at our longer term goals and vision, which will be articulated in our vision 2030 early next year. The company, we aspire to be is a sustainable commercial enterprise with an enduring R&D organization. Our vision is to maintain our pioneering leadership.
Robert I. Blum: In muscle biology, and pharmacology and grounded in financial stewardship and doing what's right for the patients we serve.
Robert I. Blum: For AFI Campton, we expect to present the primary results from Sequoia HCM at the European Society of Cardiology Heart Failure 2024 Congress next week. We expect to submit an NDA to the FDA in Q3 2024 and an MAA to the EMA in Q4 2024. We expect to complete enrollment in Maple HCM in Q3 2024 and continue enrollment in Acacia HCM throughout 2024. We expect to continue enrollment in Cedar HCM in 2024 and to begin a Phase I study of Affie Campton in Japanese Healthy Volunteers in Q2 2024.
Robert I. Blum: Now I'll recap our upcoming milestones for.
Robert I. Blum: Rafi Campton, we expect to present the primary results from Sukhoi HCM at the European Society of Cardiology Heart failure 'twenty 'twenty four Congress next week we.
Robert I. Blum: We expect to submit an NDA to the FDA in Q3, 2024, and an MAA to the EMA in Q4, 'twenty 'twenty four we expect to complete enrollment in Maple HCM in Q3, 2024 and continue enrollment in Acacia HCM throughout 2024.
Robert I. Blum: We expect to continue enrollment in Cedar HCM in 2024 and to begin a phase one study of <unk> Camden in Japanese healthy volunteers in Q2, 2024, and we expect to continue advancing our go to market strategies for <unk> Kimpton.
Robert I. Blum: And we expect to continue advancing our go-to-market strategies for Affie Campton. For CK586, we expect to present primary data from the Phase I study at a medical meeting in the second half of this year and to start a Phase II clinical trial in Q4 2024. And for preclinical development and ongoing research, we expect to initiate clinical development with another muscle-directed compound later this year, as well as continue our research and expanded muscle biology activities. Operator, with that, we can now open up the call, please, to questions. Thank you.
Robert I. Blum: For CK 586, we expect to present primary data from the phase one study on a medical meeting in the second half of this year and to start a phase II clinical trial in Q4, 2024 and for preclinical development and ongoing research, we expect to initiate clinical development.
Robert I. Blum: <unk> with another muscle directed compound later this year as well as continue our research and expanded muscle biology activities.
Speaker Change: Operator with that we can now open up the call pleased to questions.
Operator: Thank you. As a reminder, to ask a question, please press star 11 on your telephone and wait for your name to be announced. To withdraw your question, please press star 11 again.
Speaker Change: As a reminder to ask a question. Please press star one on your telephone and wait for your name to be announced to withdraw your question. Please press star one again.
Operator: In the interest of time, we do ask that you kindly limit yourself to one question at this time. Please stand by while we compile the Q&A roster. And our first question will come from Charles Duncan from Cancer Fitzgerald. Your line is open.
Speaker Change: In the interest of time, we do ask that you kindly limit yourself to one question at this time please.
Speaker Change: Please standby, we compile the Q&A roster.
Speaker Change: And our first question will come from Charles Duncan from Cantor Fitzgerald. Your line is open.
Charles Cliff Duncan: Hello Charles.
Operator: Sorry, Hi team this is STL on for Charles.
STL: Thank you for taking our questions and congrats on all the progress made in the quarter. So we have one question on CK five 8%.
Charles Cliff Duncan: On the Q1 results.
STL: This morning, well, let me the starting dose escalation strategy for the phase II clinical study and also how do you plan to monitor first safety and efficacy in this study.
Unknown Speaker: So it's a bit premature in light of the fact that we haven't announced the design for the Phase 2 study, but maybe Fady and Stuart can speak in general terms about how we think about dose escalation and the types of assessments that we might be considering for the Phase 2 study.
Charles Cliff Duncan: So it's a bit premature in light of the fact that we haven't announced the design for the phase II study, but may be fatty and Stuart can speak in general terms about how we think about dose escalation and the types of assessments that we might be considering for the phase II study.
Unknown Speaker: Yeah, I mean, I'll just say that all the doses that are in the press release were well tolerated, we didn't find a maximally tolerated dose, we didn't need to push it that high, we had a clear pharmacodynamic response, and that enabled us to decide on how to proceed to phase two. I think we'll have more to say on specific doses and things, but you can expect phase two to be really a dose finding study and patients with heart failure and preserved ejection fraction, those that will resemble patients that will study in phase three.
Stuart: Yes, I mean, I will just say that all of the doses that are in the press release were well tolerated we didn't find a maximally tolerated dose.
Stuart: I didn't need to push it that high we've had a clear pharmacodynamic response.
Speaker Change: That was enabling of us to decide on how to proceed to phase III.
Speaker Change: We will have more to say on specific doses and things, but you can expect phase III.
Speaker Change: Really a dose finding study in patients with heart failure preserved ejection fraction of those that will resemble patients that will study in phase III.
Unknown Speaker: And right now, in terms of monitoring and the dosing schedule and how we're going about it, I'm going to probably remain quiet on that until we are ready to be more forthcoming with those details. But what you can expect.
Unknown Speaker: And.
Speaker Change: Right now in terms of monitoring in the dosing schedule and how we're going about it I'm going to probably remain.
Speaker Change: Quiet on that until we are ready to.
Speaker Change: Be more forthcoming with those details.
Unknown Speaker: But what you can expect is that we're borrowing from learnings as it relates to affecamtin in NHCM for application to CK586 in HEF-PAF. And we believe that there are similarities in patient populations as well as endpoints for clinical evidence of effect. So we'll have more to say about that later in the year.
Unknown Speaker: But what you can expect is that we're borrowing from learnings as it relates to <unk> in <unk> HCM for application to CK 586, and half path and we believe that there are similarities in patient populations as well as endpoints for.
Unknown Speaker: Evidence of effect. So we will have more to say about that later in the year.
Speaker Change: Okay. Thank you so much.
Operator: Thank you. And our next question will come from Paul Choi from Goldman Sachs. Your line is now open.
Speaker Change: Thank you.
Speaker Change: Thank you and our next question will come from Paul Choi from Goldman Sachs. Your line is now open.
Unknown Attendee: Hi Robert. Good afternoon. And nice to see Sung again here and continue working with him. My question is about Maple.
Paul Choi: Hello, Paul.
Paul Choi: Hi, Robert Good afternoon, and nice to see stocking again here.
Paul Choi: When youre working with him.
Paul Choi: My question is on Maple and with regard to your.
Paul Choi: Timing comments with that trial to be enrolled by third quarter. This year are you presumably will have access to.
Paul Choi: I cover the data, let's say by as late as.
Paul Choi: Middle of next year. So my question here is.
Paul Choi: Do you have any plans to submit any interim or partial cut of the data or maybe even the full data as part of either safety or efficacy set and consideration for you as part of your NDA filing either to bolster your case for your proposed ramps and or label. If you could comment on that that would be great. Thank you.
Unknown Attendee: And with regard to your timing comments, with the trial, you know, to be enrolling by the third quarter this year, you presumably will have access to a cut of the data, let's say by as late as the middle of next year. So my question here is, do you have any plans to submit any interim or partial cuts of the data or maybe even the full data as part of either a safety or efficacy set in consideration for your as part of your NDA filing, either to bolster your case for your proposed RAMS and or label? If you could comment on that, that would be great. Thank you.
Robert I. Blum: Good question. I'll ask Fady to comment.
Speaker Change: Good question and I'll ask <unk> to comment.
Fady Ibraham Malik: Yeah, so, you know, the trial will remain blinded until it reads out in 2025. But that said, during an NDA review, there's something called a 120-day safety update. And so we'll be submitting aggregate safety data from ongoing trials like MAPLE and ACACIA, as well as updated safety and efficacy data from FOREST-HCM. So there will be more data, if you will, to consider and to cement the safety profile of AFI Campton during the NDA review.
Speaker Change: Yes, so the trial will remain blinded until until it reads out in 2025, but that said.
Fady Ibraham Malik: During an NDA review, there's something called a 120 day safety update and so we will be submitting.
Fady Ibraham Malik: Aggregate safety data from ongoing trials like Maple and Acacia as well as updated safety and efficacy data from from Forest HCM. So there will be more data if you will to consider.
Paul Choi: And to <unk>.
Paul Choi: Cement.
Fady Ibraham Malik: Safety profile of MP Kimpton.
Paul Choi: During the NDA review.
Fady Ibraham Malik: Okay, great. Thank you. Thank you, Paul. Thank you. And our next question will come from Srikripa Devarakonda from Truist Securities.
Speaker Change: Okay, great. Thank you.
Speaker Change: Thank you Paul.
Operator: And our next question will come from Srikripa Devarakonda from Truist Securities. Your line is now open.
Srikripa Devarakonda: Thank you.
Srikripa Devarakonda: And our next question will come from Crypto endeavor Khanda from curious Securities. Your line is now open.
Operator: Your line is now open. Good afternoon. Thank you so much. Good afternoon, Robert. Thank you so much for taking my question.
Unknown Attendee: You know, David Campton is still in the early stages of
Srikripa Devarakonda: Good afternoon, and thank you good afternoon, Robert Thank you so much for taking my question.
Unknown Attendee: You may begin.
Khanda: In early stages of launch and maybe jumping the gun here in terms of we have to wait for at the counter indicated through but just thinking a little bit long term if approved.
Robert I. Blum: Do you still have a significant proportion of the market to penetrate but was wondering about the potential of patients.
Srikripa Devarakonda: Switch from NEVA Camden topic Hampton is very they're concerned about the safety or not satisfied with the efficacy that may not be a base case, but was just wondering if there is a possibility.
Robert I. Blum: So it's interesting, I think, that analysts have been polling opinion leaders about that very matter. And we'll leave that to the equity research community to make its own conclusions from those surveys. But it's certainly our primary strategy to be expanding the category for Affy Kampton, so that it is applicable to a broader array of patients and physicians who prescribe for those patients, and not otherwise to focus on patient switches. But maybe I could ask Andrew to speak about how he's thinking about the broader category, growth, and penetration.
Speaker Change: So it's interesting I think.
Robert I. Blum: Analysts have been polling.
Andrew Callos: Opinion leaders about that very matter and we'll leave that to the equity research community to make its own.
Andrew Callos: Conclusions from those surveys.
Andrew Callos: But it is certainly our primary strategy to be expanding the category for ft, Camden as could be applicable to a broader array of patients and physicians who prescribed for those patients.
Robert I. Blum: Although there was to focus on patient switches, but maybe I could ask Andrew to speak about how he's thinking about.
Andrew Callos: The broader category growth and penetration.
Andrew Callos: Yeah, Robert, maybe just to build on what you described. I mean, we're expecting the vast majority of the market, the addressable market, to still be available. And really just educating physicians and broader cardiology on aficampin so they can inform and make an informed decision about the treatment of a patient. We're not going to be communicating or marketing switches at all. If that happens, it would be through the physician-patient dialogue. You know, we're trying to be good students.
Andrew Callos: Yes, Robert maybe just to build on what you described I mean, we're expecting the vast majority of the market addressable market to still be available.
Andrew Callos: Really just educating physicians and broader cardiology.
Andrew Callos: On after you Kempton.
Andrew Callos: And then so they can inform and Nick.
Andrew Callos: Informed decision around treatment of our patients were not going to be key.
Andrew Callos: Communicating or marketing switches at all if that happens that would be through the physician patient dialogue.
Andrew Callos: You know, we're trying to be good students, sorry. We're trying to be good students of other cardiovascular brand launches and next-in-class strategies and how that ultimately serves patients and shareholders. And I think it's incumbent upon us to be focused on where cardiac myosin inhibition still has application to a broader array of patients, ultimately, in order to be able to fulfill our science and mission.
Andrew Callos: We're trying to be good students.
Andrew Callos: As we're trying to be good students of other cardiovascular brand launches a next in class strategies and how that ultimately serves patients and shareholders and I think it's incumbent upon us to be focused on where cardiac myosin inhibition still has application to a broader array of patients ultra.
Andrew Callos: <unk> in order to be able to fulfill our science and mission.
Speaker Change: Got it thank you very much.
Operator: Thank you. Our next question comes from Tess Romero from JP Morgan. Your line is open.
Andrew Callos: You.
Tessa Thomas Romero: Thank you. Our next question comes from tests Romero from Jpmorgan. Your line is open.
Tessa Thomas Romero: Good afternoon.
Unknown Attendee: Good afternoon. Hey, Robert, and team.
Romero: Good afternoon, Hi, Robert and team. Thanks, so much for taking our question.
Unknown Attendee: Thanks so much for taking our question. So, you know, we saw that you have an upcoming meeting with the FDA this quarter. Curious about your latest thinking on what you will provide us all in terms of how that meeting goes on your interactions around a risk mitigation plan here. And like, will there be a disclosure before you begin the NDA filing or not? And what could that look like?
Tessa Thomas Romero: We saw that you have an upcoming meeting with the FDA this quarter.
Unknown Attendee: On your latest thinking on.
Unknown Attendee: What you will provide us.
Unknown Attendee: In terms of how that meeting goes on your interactions around or a risk mitigation plan here and like.
Unknown Attendee: Well there'd be a disclosure before you begin the NDA filing or not and what could that look like.
Robert I. Blum: And then, second quick question is just on the epidemiology side for HCM. You know, we attended ACC, and one of our key takeaways was around the focus on improving diagnosis. Where are you today on the right number? What is the patient number that could be addressable at the time of a launch? And how does this number expand if one includes the pediatric population? Thanks.
Unknown Attendee: And then second quick question is just on the epidemiology side for HCM. We attended ACC in one of our key takeaways was around the focus on improving diagnosis.
Robert I. Blum: Where are you today on the right number.
Robert I. Blum: Patient number is that could be addressable at the time of the lines and how.
Robert I. Blum: How does this number expand if one includes the pediatric population. Thanks.
Robert I. Blum: So I'll tackle the first one, maybe Fady will add something to that, and then we'll ask Andrew to comment on numbers two and three. As far as our strategy is concerned, we do not intend to provide feedback on a play-by-play basis with regard to regulatory interactions, especially as it relates to REMS. We do expect to come out of the meeting with FDA with clarity on what we might choose to do in connection with the potential REMS, and we'll give some general updates on our next earnings call, but not otherwise after the meeting itself.
Speaker Change: Sure. So I'll tackle the first one maybe thought he will add something to that and then I'll ask Andrew to comment on numbers two and three.
Robert I. Blum: As it relates to our strategy, we do not intend to provide.
Robert I. Blum: Feedback on a play by play basis with regard to regulatory interactions, especially as it relates to Rems, we do expect to come out of the meeting with FDA with clarity on what we might choose to do in connection with the potential Rems and we will give some general.
Robert I. Blum: Update with our next earnings call, but not otherwise after the meeting itself. We believe that it's in the interest of transparency to at least provide some general updates, but not so specifically as that may affect the actual review and disc.
Robert I. Blum: We believe that it's in the interest of transparency to at least provide some general updates, but not so specifically as that may affect the actual review and discussions we're having with FDA. And as you know, ultimately, it won't be until perhaps a mid-cycle review that the FDA gives us a sense of what they're thinking, and that's ultimately what's most actionable. And it'll be at that point, if this drug, hopefully, will be approvable, that we'll be in negotiations around a potential REMS or other risk mitigation strategies. So I don't think it's in anyone's interest to foreshadow that before we've even submitted the NDA. Fady, anything you want to add to that?
Fady: <unk>, we're having with the FDA and as you know ultimately it wont be until perhaps a mid cycle review that the FDA gives us a sense of what they're thinking and that's ultimately what's most actionable.
Fady: And it'll be at that point, if this drug hopefully will be approvable that will be in a negotiation around a potential rems or other risk mitigation strategies. So I don't think its in anyone's interest two.
Fady: For Shadow that before we've even submitted the NDA fabienne.
Fady Ibraham Malik: Yeah, I'll just add that, you know, these conversations in general are mostly just direction. They're not commitments in any sense.
Fady: <unk> anything you want to add to that.
Fady: Yes, I'll just add I think that these conversations in general are mostly just directional theyre not.
Fady: Not commitments in any sense, they are helping FDA get familiar with the data as we see them Bill obviously need to do their own review and so.
Fady: Any disclosures would not necessarily be that useful because.
Fady: Things will change over the course of a review and.
Fady: I don't think we want to be confusing.
Fady Ibraham Malik: They are helping FDA get familiar with the data as we see it. They'll obviously need to do their own review. And so, you know, any disclosures would not necessarily be that useful because things will change over the course of a review. And I don't think we want to be confusing folks in terms of, you know, what directions things are moving.
Fady: Confusing folks in terms of.
Robert I. Blum: To be clear, however, we do expect to float different scenarios, different risk mitigation strategies, as could be read on a potential REMS, and it won't be until after that meeting that we make a decision about how to approach that in the course of submitting an NDA in Q3. And with that, maybe I'll turn to Andrew to answer questions two and three.
Fady Ibraham Malik: What direction things are moving.
Fady Ibraham Malik: To be clear however, we do expect to float different scenarios different risk mitigation strategies as could read on a potential rems.
Robert I. Blum: And.
Robert I. Blum: It won't be until after that meeting that we make a decision about how to approach that in the course of submitting an NDA in Q3.
Robert I. Blum: And with that maybe I'll turn to Andrew to answer questions two and three.
Andrew Callos: So on the diagnosed, so I'll just stick to obstructive HCM, there are 200,000 patients today diagnosed, and of those, 130,000 would be eligible for treatment as defined by New York Heart Class 2 or 3. But like most rare diseases, that number of terms of the true prevalence is likely underrepresented where many patients are not diagnosed, but probably the true prevalence is three or four times that number. It's also not uncommon that once an available treatment is on the market, guidelines are updated to include that available treatment.
Andrew Callos: Sure so on the.
Andrew Callos: Diagnose so I'll just stick to obstructive HCM.
Andrew Callos: 200000 patients today diagnosed in.
Andrew Callos: Of those 130000 would be eligible for treatment is defined by New York Heart class two or three.
Andrew Callos: Like most rare diseases that number in terms of the true prevalence is likely underrepresented.
Andrew Callos: Many patients are not diagnosed the probably the crude prevalence is.
Andrew Callos: There is education and publications by pharmaceutical companies. You'll start to see those rates go up, and we're expecting that to be the case. In terms of the pediatric population, so of the 200,000 obstructive HCM, you know, diagnosed patients in the U.S. and Europe are similar, there are around six to 8,000 pediatric patients with HCM, you know, so I'm assuming that probably we don't have a breakout of New York heart class for the pediatric population. But I assume that probably 50 to 60% of that population would be eligible for treatment.
Andrew Callos: Three or four times that number.
Andrew Callos: So not uncommon Watson available treatment.
Andrew Callos: The market guidelines are updated to include that available treatment, there's education and publications by pharmaceutical companies, you'll start to see those rates go up and we're expecting that to be the case.
Andrew Callos: In terms of the pediatric population so of the 200000 in obstructive HCM.
Andrew Callos: Diagnose patients in the U S and Europe is similar.
Andrew Callos: It was around six to 8000 pediatric patients.
Andrew Callos: With HCM.
Andrew Callos: So I am assuming that it's probably we don't have a breakout of New York Heart class for the pediatric population.
Andrew Callos: But I'm, assuming it's probably 50% to 60% of that population would be eligible for treatment.
Andrew Callos: So hopefully, that answers your question. Thank you. Thank you. And as a reminder, we do ask that you kindly limit yourself to one question at this time. And our next question.
Speaker Change: So hopefully that answers your question.
Speaker Change: Thank you.
Operator: Thank you. And as a reminder, we do ask that you kindly limit yourself to one question at this time. And our next question will come from Roanna Ruiz from Lerink. Your line is open.
Speaker Change: Thank you.
Speaker Change: Thank you and as a reminder, we do ask that you kindly limit yourself to one question at this time.
Operator: And our next question will come from Roanna Ruiz from Leerink. Your line is open.
Roanna Clarissa H. Ruiz: Good afternoon.
Roanna Clarissa H. Ruiz: Good afternoon, everyone. So a follow up on the Cedar HCM trial in pediatric patients could you talk a bit about the unmet need there for an agent like assay canton and in this particular segment of the HCM market and how long might it take to actually complete that trial and do you have any thoughts on the regulatory path forward assuming that.
Roanna Clarissa H. Ruiz: You have positive data from Cedar.
Fady Ibraham Malik: Yeah, so, you know, I think the pediatric population is not as large as the adult population, but there's a significant unmet need. You heard Andrew comment on it about the size.
Operator: Okay.
Roanna Clarissa H. Ruiz: Yes so.
Fady Ibraham Malik: I think the pediatric populations not as large as the adult population, but there is a significant.
Fady Ibraham Malik: Unmet need yard Andrew comment on it on the size.
Fady Ibraham Malik: Don't have.
Fady Ibraham Malik: Generally the same they have the same types of treatment options available to them that adults do but.
Roanna Clarissa H. Ruiz: Surgery is much less preferred option in that age group if you will.
Fady Ibraham Malik: They don't generally have the same types of treatment options available to them that adults do, but surgery is a much less preferred option in that age group, if you will. The availability of a myosin inhibitor, I think, would be quite meaningful as an option for these patients, who when they manifest disease at an early age like this, it can be quite aggressive. So we may also, over time, be able to generate information that looks at how the disease is potentially stabilized as opposed to progressive.
Fady Ibraham Malik: The.
Roanna Clarissa H. Ruiz: Availability of a myosin inhibitor I think would be quite meaningful as an option for these patients who when they manifest disease at an early age like this it can be quite aggressive.
Fady Ibraham Malik: So we may also over time be able to generate.
Fady Ibraham Malik: Information that looks at how potentially the disease has stabilized as opposed to progressive.
Fady Ibraham Malik: And in this subgroup, that may be more amenable because their disease may progress more rapidly. I think when you talk about any pediatric patient population, the FDA, in general, is very accommodating to trying to move those things quickly as possible through the review process. We would expect these data to be positive. Just given that, you know, the main endpoint is reduction of the left ventricular outflow tract gradient. And so once we have these trials done, we're not really guiding it as to how long it'll take, but we think it'll go fairly quickly.
Fady Ibraham Malik: And then this.
Fady Ibraham Malik: Subgroup that may be more amenable because their disease may progress more rapidly.
Fady Ibraham Malik: No.
Fady Ibraham Malik: I think when you talk about any pediatric patient population the FDA in general.
Fady Ibraham Malik: Is very accommodating to trying to move those things quickly as possible to the review process.
Fady Ibraham Malik: We would expect.
Fady Ibraham Malik: These data to be positive.
Fady Ibraham Malik: Just given the.
Fady Ibraham Malik: The main endpoint is a reduction of the reluctant tricolor outflow tract gradient.
Fady Ibraham Malik: And so once we have these trial then we're not really guiding at to how long it will take but we think it will go fairly quickly.
Fady Ibraham Malik: We can we can file this as part of a supplemental NDA.
Speaker Change: Got it thanks.
Speaker Change: Thank you Ron.
Fady Ibraham Malik: We can we can file this as part of a supplemental NDA. Got it, thanks. Thank you, everyone. Thank you. Our next question will come from Salim Syed from Mizuho. Your line is open.
Speaker Change: Thank you.
Salim Qader Syed: Our next question will come from Salim Sayed from Mizuho. Your line is open.
Salim Qader Syed: Closely hey, Robert Good afternoon, everybody.
Salim Qader Syed: And I'd like to also extend my welcome and congrats a song it's good to reconnect him.
Salim Qader Syed: I guess on <unk>, if I can I know you are not.
Salim Qader Syed: Maybe this is for fat, providing any details exactly on the phase two design, but is there anything in particular fatty from the phase one trial.
Salim Qader Syed: You learn that how this compound could potentially differentiate from the myocardial <unk> two to four a compound in.
Salim Qader Syed: The read across there into how a trial could differentiate.
Salim Qader Syed: From their current phase II study.
Operator: Hello Salim. Hey, Robert. Good afternoon, everybody. And I'd like to extend my welcome and congratulations. This song gets
Salim Qader Syed: It's a challenging one to answer silly because.
Salim Qader Syed: We don't really know very much about two to four and we haven't told you very touchy about 586.
Operator: Thank you. Our next question will come from Salim Syed of Mizuho. Your line is open.
Salim Qader Syed: I guess I'll, just say and maybe I can ask Stuart to expand a little bit but our goal is to make.
Salim Qader Syed: Dosing of 586 simpler than inactive Camden, and we think it has some.
Speaker Change: <unk> unique properties that may enable that as well.
Speaker Change: We have looked at.
Unknown Speaker: It's a challenging one to answer Salim because we don't really know very much about 224, and we haven't told you very much about 586. Um, you know, I guess I'll just say, and then maybe we can ask Stuart to expand a little bit, but our goal is to make dosing 586 simpler than aficamptin, and we think it has some unique properties that may enable that, as we have seen in phase one. Stuart, do you want to maybe elaborate a little bit? I think the only thing I'll really add is that...
Speaker Change: Seen in phase one.
Operator: Stewart do you want to maybe elaborate a little bit.
Stuart Kupfer: I think the only thing I'll really add is that CK5H6 was designed to target what we consider more vulnerable populations. You know, these patients have had many comorbidities, perhaps more risk of, www.cdc.gov.au Okay, got it.
Stuart Kupfer: I think the only thing I would really add that CK six was designed to target what we consider more vulnerable population.
Stuart Kupfer: These patients have had many comorbidities.
Stuart Kupfer: Yeah, perhaps at more risk.
Stuart Kupfer: Some decreases in ejection fraction.
Stuart Kupfer: For example than.
Stuart Kupfer: We think 65 properties.
Stuart Kupfer: That make would make these patients.
Stuart Kupfer: More manageable and less prone to.
Stuart Kupfer: That or other risks and so again those details will be more forthcoming.
Speaker Change: Okay got it thank you very much.
Speaker Change: Thank you.
Operator: Thank you very much. Thank you. Thank you. And our next question will come from Jeff Hung from Morgan Stanley. Your line is open.
Operator: Thank you, and our next question will come from Jeff Hung from Morgan Stanley. Your line is open.
Speaker Change: Thank you.
Operator: And our next question will come from Jeff Hung from Morgan Stanley. Your line is open.
Jeff Hung: Thank you Robert and team Hi, Robert and team. Thanks for taking my question and congratulations on your new role proceed or what are the expectations for the dose range that is likely to be sufficient for most pediatric and adolescent patients.
Jeff Hung: We end up being similar dose range of the patients from Sequoia would you expect a higher rate of ejection fraction excursion because of the differences related to H. Thanks.
Unknown Speaker: Stuart, do you want to take that one? Hi Jeff.
Stuart Kupfer: Sure. Thanks for the question, Jeff.
Operator: Stewart do you want to take that one Jeff.
Stuart Kupfer: So, as you mentioned, the doses in adolescent patients are exactly the same as the doses that we're studying in adults, between 5 and 20 milligrams. And again, what's important is that the dose selection is individualized based on achievement of the target gradient and maintenance of a normal ejection fraction. In terms of younger children, the key question will be really, you know, what is a safe starting dose? And the plan will be to, you know, as I mentioned, evaluate, do an interim analysis, evaluate 20 patients, adolescent patients, and safety, and determine for younger children what is a safe starting dose. That's the basic strategy. And so, with that in mind and individualized dose selection, we anticipate that the benefit-risk ratio in children will be just as good as we're observing in adults.
Stuart Kupfer: Sure. Thanks for the question Jeff.
Stuart Kupfer: As I mentioned the other doses in adolescent patients are the same as it does.
Stuart Kupfer: Doses, we're studying in adults between.
Stuart Kupfer: 120 milligrams and.
Stuart Kupfer: Again, what's important is that the dose selection as individualized based on achievement of target gradient and maintain maintenance.
Stuart Kupfer: Our normal.
Stuart Kupfer: Jackson for action.
Stuart Kupfer: In terms of younger children the.
Stuart Kupfer: The key question will be really well.
Stuart Kupfer: What does it say starting dose.
Stuart Kupfer: The plan will be to analyze.
Stuart Kupfer: I mentioned evaluate.
Stuart Kupfer: To do an interim analysis evaluate 20 patients adolescent patients.
Stuart Kupfer: The pharmacokinetics and safety and determine.
Stuart Kupfer: For the younger children, what does this saved starting dose that's.
Stuart Kupfer: The basic strategy and so with that in mind and individually individualized dosing election, we don't anticipate we anticipate that the benefit risk ratio.
Stuart Kupfer: Children will be just as good as we are observing on the call.
Speaker Change: Great. Thank you.
Stuart Kupfer: Again.
Speaker Change: Thank you.
Operator: Our next question will come from Mayank Mamtani from B. Reilly Securities. Your line is now open.
Mom Tani: Our next question will come from my Mom Tani from B Riley Securities. Your line is now open.
Unknown Attendee: Good afternoon, team. Thanks for taking our questions. I'm also pleased to see some join the team.
Mayank Mamtani: Good afternoon team. Thanks for taking my questions and also pleased to see some joined the team just maybe on the next Monday's ESC Heart failure Congress data could you quantify if you could get the patient level safety analysis relative to maybe what we saw on the top line and also obvious.
Unknown Attendee: This may be on the next Monday's ESC Heart Failure Congress data. Could you clarify if we could get the patient level safety analysis relative to, perhaps, what we saw on the top line? And also, obviously, curious to see how much of the dose titration and the responder rate of patients staying at higher efficacious doses is helpful in having that TVO2 number that seems to be on the higher side and not connected to the baseline characteristics that we had initially thought. If you could clarify that, that would be great.
Unknown Attendee: Curious to see.
Unknown Attendee: How much of.
Unknown Attendee: The dose titration and the responder rate of patients staying at high.
Unknown Attendee: Efficacious doses.
Unknown Attendee: Hopefully do having that DVR do number.
Unknown Attendee: It seems to be on the higher side and not connected to the baseline.
Unknown Attendee: The stakes that we had initially thought if you could quantify that that'd be great.
Fady Ibraham Malik: Yeah, hi, Mike. I mean, I think most of your questions will be addressed in those presentations. Remember, there are three of them.
Speaker Change: Yes, Hi, Mike I mean, I think most of your questions will be addressed in those presentations are among the three of them one will speak.
Speaker Change: A lot about dosing and safety and so we will.
Speaker Change: Show the characteristics of patients at the various doses.
Speaker Change: And and how safety was related to that.
Fady Ibraham Malik: One will speak a lot about dosing and safety. And so we'll, you know, show the characteristics of patients at the various doses and, and, and how safety was related to that. But, you know, with regard to peak VO2, I can't really comment.
Fady Ibraham Malik:
Fady Ibraham Malik: With regards to peak <unk> cant really comment I'm not sure that there is a dose specific analysis, but.
Speaker Change: Got to remember these.
Fady Ibraham Malik: These patients were not randomized the dose they were randomized to a dosing strategy and they achieved a dose based on their individual response to the drug.
Fady Ibraham Malik: And so in that way, we and a lot of waste consider them just all part of the same dosing strategy.
Speaker Change: Got it thanks for taking questions.
Fady Ibraham Malik: Okay.
Speaker Change: Thank you.
Fady Ibraham Malik: Our next question will come from Jason Butler from citizens JMP. Your line is open.
Fady Ibraham Malik: Hi, Thanks for taking the question.
Speaker Change: Congrats on all the progress.
Speaker Change: Just wondering if you could give us an update on regulatory progress for China and what next steps are there. Thanks.
Fady Ibraham Malik: I'm not sure that there is a dose-specific analysis. But, you know, you have to remember these patients were not randomized to doses. They were randomized to a dosing strategy, and they achieved a dose based on their individual response to the drug. And so in that way, we, in a lot of ways, consider them just all part of the same dosing strategy.
Speaker Change: Hey, Jason I'll take that one.
Speaker Change: In light of that we're making very good progress, but we arent in a position yet to be specific until such time as we engaging our partner are aligned on setting those expectations. So I do hope that we'll be able to say something with the next earnings call.
Operator: Got it. Thanks for the inquiries. Thank you. Our next question will come from Jason Butler.
Speaker Change: Okay, great. Thank you.
Operator: Our next question will come from Jason Butler from Citizens JMP. Your line is open. Hi, thanks for taking the question.
Operator: Yeah.
Operator: Our next question will come from Jason Zemansky from B of A. Your line is now open. Hello, Jason. Hey, good afternoon.
Jason Nicholas Butler: Thank you.
Unknown Attendee: Hey, Jason, I'll take that one. You know, in light of that, we're making very good progress, but we aren't in a position yet to be specific until such time as we and Zijing, our partner, are aligned on setting those expectations. So I do hope that we'll be able to say something on the next earnings call. Okay, great. Thank you. Thank you. Our next question will come from Jason Zemansky from B of A. Your line is now open. Hello Jason. Hey, good to have you here.
Operator: Our next question will come from Jason Symanski from Bofa. Your line is now open.
Jason Eron Zemansky: Hello, Jason.
Speaker Change: Good afternoon, Kevin <unk> on for Jason.
Jason Eron Zemansky: So you've discussed the potential of our novel Rasp and is based on a risk algorithm I mean could you kind of elaborate a little bit on what this could look like have you received any feedback from regulators on this point and then if you could maybe touch on your base case assumptions currently for number of iqos likely to be required in the tie.
Unknown Attendee: And then in the maintenance setting as well thank you.
Operator: Sure, it's premature to speak to the second part of your question. The number of echoes is a function of conversations that we're still going to be having with FDA. But I think there's no scenario by which we can expect a similar REMS. I think in light of the fact that the dosing strategy employed in Sequoia HCM and continuing in Forest HCM is itself differentiated, and therefore, that ties to the risk mitigation strategy, it's, I think, going to be clear that there will be a different kind of REMS program.
Unknown Attendee: Sure it's premature to speak to the second part of your question. The number of echoes is a function of conversations that we're still going to be having with FDA, but I think there is no scenario by which we expect a similar rems.
Operator: I think in light of the fact that the dosing strategy employed in <unk>.
Operator: Sequoia HCM and continuing enforced HCM.
Operator: Is.
Operator: Itself with differentiated.
Operator: Therefore.
Operator: That ties to the risk mitigation strategy.
Operator: I think going to be clear that there will be a different kind of rems program.
Operator: And with that, we expect to have conversations with FDA about what that could look like in a concrete way, as we'll be informed by how we might approach risk mitigation in an NDA submission. And, as you know, there are different ways to approach risk mitigation. Some of that can be handled by labeling. Some of that can be handled in an informational REMS. Some of that may require an ETASU REMS system. The current cardiac myosin inhibitor is the subject of an Etosu REMS, and our objective is to understand how FDA thinks about risk in light of not just the EF excursions, as seems to be the focus of Wall Street, but also as regards ADME properties, drug-drug interaction properties, and pharmacokinetic properties that speak to half-life and shape of the curve with regard to pharmacodynamics.
Operator: And.
Operator: With that we expect to have conversations with FDA about what that could look like.
Operator: In a concrete way as will be informed by.
Operator: How we might approach risk mitigation in an NDA submission and there are different ways to approach risk mitigation as you know.
Operator: Some of that can be handled by labelling some of that can be handled in an informational rems. Some of that may require <unk> passu rems.
Operator: The current cardiac myosin inhibitor is the subject of.
Operator: And at Tulsa rooms at our objective is to understand how FDA thinks about risk in light of not just the U F excursions as seems to be the focus of wall Street, but also as pertains to add me properties drug drug interaction proper.
Operator: <unk>.
Operator: Pharmacokinetic properties that speak to half life and shape of the curve with regard to pharmacodynamics. All these things factor into how one approaches risk mitigation.
Operator: All these things factor into how one approaches risk mitigation. And I do think we're going to be in a position, as is our base case, to have a differentiated profile for risk mitigation. I think that's the best I can do today, but we do hope to have more to say down the road.
Operator: And I do think we're going to be in a position as is our base case to have a differentiated profile for risk mitigation.
Operator: I think thats the best I can do today, but we do hope to have more to say down the road.
Speaker Change: Got it thank you.
Speaker Change: Thank you.
Robert I. Blum: Thank you. Our next question comes from Carter Gould from Barclays. Your line is open.
Operator: Thank you. Our next question comes from Carter Gould from Barclays. Your line is open.
Unknown Attendee: Hey, Robert, and team. Good afternoon. Thanks for taking the question. I'm going to ask you a bit of an unfair one in that the AHA and ACC HCM guidelines are dropped this afternoon, but I'm going to go out on a limb and say that you probably had a good sense of what was going to be in there. So I guess, at a high level, is there anything in there that's surprising? Maybe just at the risk of provoking Diane and the operator, when you think of, I guess, for Andrew, when you think then about the importance of, you know, guidelines down the road conveying that differentiation between Mavicampton and Aficampton, how important is that based on all the prior kind of case study work you've done? Thank you.
Robert I. Blum: Okay.
Unknown Attendee: Hey, Robert team. Good afternoon. Thanks for taking the question I'm going to ask you a bit of an unfair one in that PHA at ACC HCM guidelines drops this afternoon, but I'm going to go out and allow me to say that you probably had a good sense of what was going to be in there. So I guess at a high level is there anything in there that's surprising.
Unknown Attendee: And.
Unknown Attendee: Maybe just at the risk of provoking Diane in the operator, when you think.
Unknown Attendee: I guess for Andrew when you think then about the importance of guidelines down the road conveying that differentiation between mavic Hampton and assay Camden, how important is that based on all the prior kind of case study work you've done. Thank you.
Unknown Attendee: Yes, so good on you for noting that those guidelines dropped just today. I will ask Fady and Andrew both to comment from their perspectives, in light of your questions, and you did get a grin out of Diane, by the way.
Speaker Change: Yes, so good on you for noting that those guidelines dropped just today.
Speaker Change: I will ask fatty and Andrew both to comment from their perspectives.
Speaker Change: Light of your questions and you did get a grand out of Diane and by the way.
Fady Ibraham Malik: I saw the guidelines drop into my inbox two hours before this call, and actually didn't see it until... in the middle of this call.
Unknown Attendee: Well.
Fady Ibraham Malik: I saw the guidelines drop into my Inbox two hours for this call and actually didn't see that until.
Fady Ibraham Malik: So just I'll do my best to answer your question, which having scanned them briefly, you know, I think they number one, they are referring to a class of myosin inhibitors. And there's nothing really surprising, I would say they follow sort of the same approach that the EESC guidelines did, kind of placing cardiac myosin inhibitors as following first-line therapy of beta blockers. So for patients who fail first-line therapy like that, that's why we are conducting MAPLE potentially to show that there are advantages to starting chronic myosin inhibitors first, and hopefully, those data will inform future guidelines. You know, there are some specific things where they overlap. Issues with cardiac myosin inhibitors that are actually compound specific and not cardiac myosin inhibitor specific.
Fady Ibraham Malik: In the mid in the middle of this call so just to.
Fady Ibraham Malik: Do my best to answer your question, which having scan them briefly.
Fady Ibraham Malik: I think.
Fady Ibraham Malik: One they are preferring until class myosin inhibitors.
Fady Ibraham Malik: Nothing really surprising I would say.
Fady Ibraham Malik: They follow the same.
Fady Ibraham Malik: Approach that the EMA.
Fady Ibraham Malik: The ESC guidelines did kind of placing a cardiac myosin inhibitors as.
Fady Ibraham Malik: Following first line therapy of beta blockers.
Fady Ibraham Malik: So the.
Fady Ibraham Malik: Are patients who fail first line therapy like that and Thats why we are conducting maple potentially to show that there are advantages to starting cardiac myosin inhibitors first and hopefully those data will inform future guidelines.
Fady Ibraham Malik: There are some specific.
Fady Ibraham Malik: Things, where they can play.
Fady Ibraham Malik: I won't delineate those, but I think if you read them, you'll figure that out. And so some of that may in the future need to be sorted out. And, you know, we'll just have to do our best to ensure that information is in the hands of the people who make the guidelines. We certainly weren't aware of what was in the guidelines. We don't process, you know, participate in that process. But we can make sure that the data is available to them, and I expect, and I'm sorry.
Fady Ibraham Malik: Issues with cardiac myosin inhibitors that are actually compound specific and not cardiac myosin inhibitor specific.
Fady Ibraham Malik: Eliminate those but I think if you read them.
Fady Ibraham Malik: That out.
Fady Ibraham Malik: And so some of that may in the future need to be sorted out.
Fady Ibraham Malik: And.
Fady Ibraham Malik: We'll just have to do our best to answer.
Fady Ibraham Malik: Sure that debt.
Fady Ibraham Malik: Information is in the hands of the people who do the guidelines, we certainly werent aware of what within the guidelines we don't.
Fady Ibraham Malik: Yes.
Fady Ibraham Malik: Participate in that process, but we can make sure that the data is available to them.
Andrew Callos: and I expect you to, and I'm sorry. Go ahead, Andrew.
Fady Ibraham Malik: And I expect.
Andrew Callos: Oh I'm sorry.
Andrew Callos: Yeah, the only thing I maybe was going to add to your question was around guidelines, certainly that's credibility when talking to physicians about evidence, it certainly adds credibility to payers to cover, especially if it's a first-line therapy, additional studies certainly give more strength to a guideline. As well, and I think it's probably extremely important, if a guideline, as an example, talks about, say, first-line therapy because of data like MAPLE, having that data and that evidence and being able to talk to physicians about it, as compared to not having that evidence and not being able to talk to physicians about it, I think that's a big difference.
Andrew Callos: So go ahead Andrew.
Andrew Callos: Yes, the only thing I would maybe it was going to add to your question was around the guidelines certainly thats credibility when.
Andrew Callos: Talking to physicians around evidence certainly adds.
Andrew Callos: Our credibility to payers to cover, especially if it's.
Andrew Callos: A first line therapy.
Andrew Callos: Additional.
Andrew Callos: Ah studies, certainly give more strength to our guideline.
Andrew Callos: As well and I think probably extremely important is.
Andrew Callos: <unk> as an example talks about say first line therapy because of data like maple, having that data and that evidence and being able to talk to physicians about it as compared to not having that evidence and not being able to talk to physicians about it I think the big difference.
Andrew Callos: So, you know, I think the guideline helps maybe to summarize the evidence with credibility to various stakeholders, and probably most importantly, it lets us, as a pharmaceutical company, talk to physicians and payers about the evidence relative to aficampin if we get approved.
Andrew Callos: So.
Andrew Callos: So I think the guideline helps maybe to summarize with credibility to various stakeholders.
Andrew Callos: And probably most employees.
Andrew Callos: US as a pharmaceutical company.
Andrew Callos: Talk to physicians and payers about the evidence relative to Abbvie Camden, if we get approved.
Operator: Thank you. Thank you. Our next question will come from Yasmeen Rahimi on behalf of Piper Sandler. Your line is open. Thank you, team, for all these great details. I guess as we're going into the...
Speaker Change: Thank you.
Yasmeen Rahimi: Thank you.
Operator: Our next question will come from Yasmeen Rahimi of Piper Sandler. Your line is open. Thank you, team, for all these great details.
Yasmeen Rahimi: Our next question will come from Yasmin Rahimi from Piper Sandler Your line is open.
Operator: Okay.
Operator: Thank you Tim for Ali.
Yasmeen Rahimi: Great details guys.
Yasmeen Rahimi: Going into the Cardiology heart failure Congress here on Monday.
Yasmeen Rahimi: You have spoken about how the more you look at the data at the better at gap do you think on like I guess the question that people have going into Monday data release or full data disclosure surrounding it.
Yasmeen Rahimi: Will it become even more clear that Avi can Ken Wednesday efficacy not only Jeff on safety product profile with.
Yasmeen Rahimi: Would love to kind of getting a color around that and I'll jump into that actually occur.
Robert I. Blum: So to be clear, we did not conduct a head-to-head comparison of Affie Campton with MAVA Campton, and it would not be appropriate to imply that we did. With that said, we believe the profile of Affie Campton in Sequoia, as it relates to efficacy, safety, convenience, and all those things that factor into its next-in-class profile, will be supported by the evidence, as would be indicative of our belief that aficamptin can become the cardiac myosin inhibitor of choice, ultimately if approved.
Yasmeen Rahimi: So to be clear, we did not conduct a head to head comparison of <unk> with Maverick Hampton and it would not be appropriate to imply that we did with that said, we believe the profile of the RFE kimpton in Sequoia.
Robert I. Blum: It relates to efficacy safety convenience and all of those things that factor into its next in class profile.
Robert I. Blum: Will be supported by the evidence.
Robert I. Blum: As would be indicative of our belief that ft campton can become the cardiac myosin inhibitor of choice ultimately if approved.
Robert I. Blum: With that said, that's ultimately for you to determine once you see the data; the results will be available, not just in presentations, but in publications, and not just at the European Heart Failure Meeting, but throughout other meetings later this year. And I do believe that these results are reaffirming our expectations. Thank you so much. Looking forward to Monday. Thank you.
Robert I. Blum: With that said that's ultimately for you to determine once you see the data the results will be.
Robert I. Blum: Available not just in presentations, but in publications and not just at the European Heart failure meeting, but throughout other meetings later this year.
Robert I. Blum: And I do believe that these results are reaffirming of our expectations.
Robert I. Blum: Thank you so much looking forward to Monday.
Speaker Change: Thank you.
Speaker Change: Thank you.
Operator: Our next question comes from Akash Tewari from Jeffries. Your line is now open. Hey, this is Amy on behalf of Akash. Thanks.
Speaker Change: Our next question comes from a cost to worry from Jefferies. Your line is now open.
Operator: This is Amy on for <unk>. Thanks, so much for taking our question just one on <unk>.
Amy: We think the drug will need.
Amy: Monitoring and what are you seeing in your healthy volunteer data that either supports and Douglas will point. This requirement. Thanks, so much.
Unknown Speaker: Oh, I'll maybe take that one. I think we developed CK586 as potentially the means to dose this in HFF patients without echo monitoring, routine echo monitoring. That said, we need to produce the data that eventually supports that, and I think it's premature to know for sure whether we'll achieve that or not. The profile that we saw in Phase 1 was contributory toward that, and so we'll know more as we explore this drug more in Phase 2.
Amy: I'll, maybe take that one I think.
Unknown Speaker: We develop CK 586 in the us.
Unknown Speaker: Potentially the mean to doses.
Unknown Speaker: In <unk> patients without echo monitoring routine echo monitoring.
Unknown Speaker: That said, we need to produce the data that eventually supports that and I think it's premature to know for sure whether we will achieve that or not the profile that we saw in phase one.
Unknown Speaker: Was contributory toward that.
Unknown Speaker: And so.
Unknown Speaker: We'll know more as we as we explore this drug more in phase II.
Operator: Got it. Thanks so much. Thank you. Our next question will come from Serge Belanger from Needham. Your line is open.
Speaker Change: Got it thanks, so much.
Serge D. Belanger: Thank you.
Operator: Our next question will come from Serge Belanger from Needham Your line is open.
Serge D. Belanger: Hello, Serge and good afternoon, good afternoon Robert.
Serge D. Belanger: <unk> had two meetings with the FDA, so far and another one coming up I guess later this quarter.
Serge D. Belanger: What are your current thoughts regarding the potential for a priority review or an ad com.
Operator: Thanks.
Serge D. Belanger: Yeah. So.
Unknown Speaker: Neither of those are in our base case assumptions. We believe that as a next in class profile, it would be more exceptional to assume priority review.
Serge D. Belanger: Neither of those are in our base case assumptions.
Unknown Speaker: <unk>.
Serge D. Belanger: We believe that as a next in class profile it would be more exceptional to assume priority review, it's certainly possible.
Serge D. Belanger: But that's not something that we're banking on so to speak.
Robert I. Blum: It's certainly possible, but that's not something that we're banking on, so to speak. And as it relates to an ADCOM, we don't expect one, but one can be surprised by those things. And for the fact that the data are quite compelling, there is already a very effective cardiac myosin inhibitor on the marketplace that does not have an ADCOM. I think it would be unusual to now have an ADCOM for a next-in-class compound, but I guess it's theoretically possible. But it's not our base case assumption.
Serge D. Belanger: And as it relates to an AD com.
Robert I. Blum: We don't expect one, but one can be surprised by those things and for the fact that.
Robert I. Blum: The data are we believe quite compelling.
Robert I. Blum: Already.
Robert I. Blum: Very effective cardiac myosin inhibitor on the marketplace that did not have an outcome I think it would be unusual to now have an outcome for a next in class.
Robert I. Blum: Compound but.
Robert I. Blum: I guess, it's theoretically possible, it's not our base case assumption.
Speaker Change: Got it thank you.
Operator: And our next question will come from Ash Verma from UBS. Your line is open.
Speaker Change: Thank you.
Robert I. Blum: And our next question will come from Ash Verma from UBS. Your line is open.
Unknown Attendee: Hi, thanks for taking our questions. I had to ask, so regarding just the presentation on Monday, from your perspective, what do you think is the bar for the LVF less than 40% event from the Sequoia study? What level of LVF, less than 40%, would give you the conviction that the FDA could actually indeed be discerning in viewing your safety profile as differentiated from ChemDial's? And then second, on the OHCM market, just in the long run, do you think fundamentally there is a lot of diagnosis expansion that you could drive? OHCM is sometimes compared to ADDR by investors, and that has seen pretty significant diagnosis expansion. Just so you can frame what the difference is between those.
Ashwani Verma: Hey, thanks, Thanks for taking our questions I had two.
Unknown Attendee: Regarding just the presentation on Monday like from your perspective, what do you think is the bar for the <unk> Midland 40% events.
Unknown Attendee: From the Sequoia study.
Unknown Attendee: Level of via <unk> would give you the conviction that the FDA could actually indeed be discerning and viewing your safety profile.
Unknown Attendee:
Unknown Attendee: Initiated from Kim Dias.
Unknown Attendee: Then second.
Unknown Attendee: On the OSV market just in the long run do you think fundamentally flawed.
Unknown Attendee: A lot of diagnosis expansion that you could die.
Unknown Attendee: Tim in terms of day to ADR by investors and that has seen pretty significant diagnosis expansion. Yes. If you can frame what's the difference between the two.
Unknown Attendee: Okay.
Fady Ibraham Malik: So I might ask Fady to speak to the comments about ES below 40, but recognizing that our top line press release already speaks to some of those matters. Yeah.
Speaker Change: So I might ask <unk> to speak to the comments about es below 40, but recognizing our topline press release.
Fady Ibraham Malik: Already speaks to some of those matters.
Fady Ibraham Malik: Yeah, I was going to say that, as we said, our top line was that we didn't have any treatment discontinuations for EF less than 40%. And, you know, the protocol mandated those if they were observed at the site. We'll expand on that in the presentation, I suppose. But again, I don't think, you know, N of zero or one is going to be any. The things that don't happen are good to know, and they are certainly supportive of the safety profile.
Fady: Yes, I would say on that.
Fady Ibraham Malik: Say that.
Fady Ibraham Malik: We said in.
Fady Ibraham Malik: In our topline that we didn't have any treatment discontinuation for es.
Fady Ibraham Malik: Less than 40%.
Fady Ibraham Malik: And the protocol mandated those if they were observed at the site.
Fady Ibraham Malik: We will expand on that in the presentation I suppose but.
Fady Ibraham Malik: Again, I don't think.
Fady Ibraham Malik: Zero or one is going to be any.
Fady Ibraham Malik: We've never had an event of heart failure due to Aftecampton, and treatment interruptions have been almost non-existent. I think overall, those are all very promising things with regard to potential labeling down the road. And I don't think we understood your second question, which I guess we'll entertain.
Fady Ibraham Malik: All of that contributory if you will.
Fady Ibraham Malik: Things that don't happen or a good to know and there are certainly supportive of the safety profile, we've never had.
Fady Ibraham Malik: Advent of heart failure due to that the canton.
Fady Ibraham Malik: And treatment interruptions have been almost non existent and so I think overall those are all very promising things.
Fady Ibraham Malik: With regards to.
Fady Ibraham Malik: The potential labeling down the road.
Fady Ibraham Malik: And I don't think we understood your second question, which.
Speaker Change: So I guess, we'll entertain.
Unknown Attendee: Yeah, just like quickly, I mean, in terms of just the diagnosis expansion of OHCM, right? One could argue that yes, it has a high unmet need, but the mortality and morbidity burden are not as high as something like ATTR, which has seen a significant diagnosis expansion. So do you believe that in the long run, you could potentially drive a lot of diagnostic expansion? I mean, Bristol has been doing a lot of DTC and other marketing campaigns, but it is still not driving any kind of meaningful inflection on ChemGyos. So just trying to understand if this OHCM market is sort of where it is, or could we see material expansion in the long run?
Fady Ibraham Malik: Yes, just quickly in terms of just the diagnose with expansion with CN right. I mean, one could argue that yes. It has a high unmet need but the mortality and morbidity.
Unknown Attendee: Burden is not as high as something like ADP are we just seeing that significant diagnose or expansion do you believe that in the long run you could potentially drive a lot of that.
Unknown Attendee: This expansion of ambition has been doing a lot of D C.
Unknown Attendee: Other marketing campaign was still not driving any kind of a meaningful inflection on <unk>. So just trying to understand if this was your market is sort of.
Unknown Attendee: Or could we see like a material expansion in the long run.
Robert I. Blum: I don't think the disease burden here is defined by mortality incidence as much as symptom burden and other matters that read on functional life and quality of life. And it's very clear that patients who receive a cardiac myosin inhibitor are very adherent and compliant with their therapy. Look at the data for ChemZio. These are patients who are benefiting substantially from receiving a new therapy and want to stay on that therapy. And I do believe that that's ultimately going to be defining what could be the opportunity for category expansion. Andrew, anything you want to add to that? Yeah, I would say that's probably part of the remit of
Speaker Change: Yes, I don't think the disease burden here is defined by mortality incidents as much.
Andrew Callos: Symptom burden and other matters that read on functional life and quality of life.
Andrew Callos: And it's very clear that patients who receive a cardiac myosin inhibitor.
Andrew Callos: Our very adherent and compliant with their therapy look at the.
Andrew Callos: Data for at <unk> <unk>. These are patients who are benefiting substantially from receiving a new therapy and want to stay on that therapy and I do believe that thats ultimately going to be defining of what could be the opportunity for category expansion.
Robert I. Blum: Andrew anything you want to add to that.
Andrew Callos: Yeah, I would say that's probably part of the remit of what we're doing in AKOR. If you think about some of the secondary endpoints like improvement in New York Heart Class Association, improvement in KCCQ, reduction in the need for septal therapy and surgery, all those speak to improvement in health outcomes and improvement in cost savings. So I think you'll start to see some more publications and us talking about that and then merging that with large data sets to show the impact it can have on larger populations.
Andrew Callos: Yes, I would say that's probably part of the remit of what we're doing in AQR. If you think about some of the secondary endpoints like improvement in New York Heart Class Association improvement in case, you see Q.
Andrew Callos: Reduction for the need of.
Andrew Callos: Subtle.
Andrew Callos: Therapy.
Andrew Callos: Surgery, all of those speak to improvement in health outcomes and improvement and cost savings. So I think youll start to see some more publications and I was talking to that and then merging that with large datasets.
Andrew Callos: So the impact it can have on larger populations.
Andrew Callos: So it may not be as obvious as something like TTR, but it's certainly something I think you're going to see over time that the expertise that we have in AKOR around this area will certainly lead to what you're describing. Thank you. And our next question will come from Sean McCutcheon from Raymond James. Your line is now.
Sean McCutcheon: So it may be not as obvious as something STR, but it's certainly something I think youre going to see over time that.
Sean McCutcheon: The expertise that we have an AQR around this area will certainly lead to what Youre describing.
Andrew Callos: Thanks.
Sean McCutcheon: Thank you.
Operator: And our next question will come from Sean McCutcheon from Raymond James. Your line is now open.
Sean McCutcheon: And our next question will come from Schon Mccutcheon from Raymond James Your line is now open.
Sean McCutcheon: Hello, Sean Hi, guys Hi.
Sean McCutcheon: Thanks for squeezing me in can you comment on the patients enrolled in the phase two study for 586 and maybe what does that do you think has the clearest.
Sean McCutcheon: Catherine physiological rationale and how youre thinking about that.
Sean McCutcheon: Phase II in terms of parsing.
Sean McCutcheon: Subsets and maybe broad strokes on the size of the opportunity in those respective populations.
Operator: Yes, sure, I'll ask Stuart to answer that, but in sort of general terms. I'll just mention ahead of time that, as Robert said earlier, you know, the NHCM patients that responded so well in the Phase 2 study kind of provide a human model of the type of HFPEP patients we'd want to study with CK586, and maybe Stuart can expand on those characteristics generally.
Sean McCutcheon: Yes sure.
Stuart Kupfer: I'll ask Stewart to answer that but in sort of general terms.
Stuart Kupfer: I'll just mentioned ahead of time that as Robert said earlier, the NH cm patients that.
Stuart Kupfer: Responded so well in the phase II study kind of provide the human model.
Stuart Kupfer: Of of the type of patients who would want to study with CK 586, maybe.
Operator: Maybe Stuart can expand on those characteristics generally.
Unknown Speaker: Yeah, thanks, Sadie. I think that's right.
Stuart Kupfer: Yeah. Thanks, I think that's right.
Stuart Kupfer: We believe that for a subgroup of patients.
Stuart Kupfer: We believe that for a subgroup of patients with severe diastolic dysfunction, maybe in large part driven by patients with hypercontractility, and some of these patients develop ventricular wall thickening just like patients with but non-obstructive HCM. And so the outcome in patients that we observe in Phase 2 in the Redwood HCM, the non-obstructed HCM, really reads, Unknown Speaker Improvement of Diastolic Function. Subgroup of Patients with Hepatitis P and so we'll be looking, we'll be evaluating patients who have, you know, relatively high ejection traction, some degree of intricate wall thickening or symptomatic, and having functional and symptomatic www.cdc.gov.au, Andrew, do you want to speak in general terms of
Stuart Kupfer: With severe diastolic dysfunction Navy in large part driven by.
Andrew Callos: By patients with hyper contractility in some of these patients.
Stuart Kupfer: <unk>.
Andrew Callos: And then tricolor wall that can I add just like patients with non obstructive HCM.
Stuart Kupfer: So.
Andrew Callos: The outcome in patients that we observe in.
Stuart Kupfer: Phase two in the Redwood HCM, the non obstructive HCM really reads.
Andrew Callos: Informs potential benefit.
Andrew Callos: The improvement of diastolic function.
Stuart Kupfer: Function.
Andrew Callos: In page in the subgroup of patients with path and so.
Andrew Callos: We will be looking.
Andrew Callos: We will be evaluating patients who have.
Andrew Callos: A relatively high ejection fraction.
Andrew Callos: Some degree trigger a wall.
Andrew Callos: Can I now or who are symptomatic.
Andrew Callos: And having functional and symptomatic.
Andrew Callos: Heart failure symptoms related to their to their disease.
Andrew Callos: But those patients could benefit from treatment. So that's generally population will be targeting.
Andrew Callos: Andrew, do you want to speak in general terms about how we're thinking about the subsets of HFPAF from a prevalence standpoint? Andrew, you might be on mute.
Stuart Kupfer: Andrew do you want to speak in general terms approximately around how we're thinking about.
Andrew Callos: The subset so half path from a prevalent standpoint.
Andrew Callos: Andrew you might be on mute.
Andrew Callos: Yeah, HFPEF is probably, from our press releases, around half of the overall market. We're describing here as a subset of HFPEF in the upper range of ejection fraction. I think once we start to learn more in phase two, we'll probably hone in more on exactly the size of that subset where the cutoff is.
Andrew Callos: Yes.
Andrew Callos: <unk> just saw probably from our press releases around half of the overall market.
Andrew Callos: We're describing here is a subset of <unk> paas and the upper range of ejection fraction and I think once we start to learn more in phase III, we will probably hone in more of exactly the size of that subset where the cutoff is.
Operator: Thank you. Thank you. Thank you. And I am showing no further questions from our phone lines. I'd now like to turn the conference back to Robert Blum, President and CEO, for closing remarks.
Speaker Change: Thank you.
Robert I. Blum: Thank you.
Robert I. Blum: Thank you and.
Operator: And I am showing no further questions from our phone lines I'd now like to turn the conference back to Robert Blum, President and CEO for closing remarks.
Robert I. Blum: Thank you, operator. And thank you to the analysts for some excellent questions. We're excited to share this update with you, especially as regards our expanded focus on the development program for AFI Campton. And at the same time, we believe that we are being good financial stewards as we think about capital diversification, capital deployment, and efficiencies. And we're looking forward to updating you on continued progress, including with our presentations next week, Monday, May 13, at the European Heart Failure Meetings and afterwards. We thank you for your interest in cytokinetics, and we'll look forward to the next earnings call. Operator, with that, we can now conclude the call.
Robert I. Blum: Thank you operator, and thank you to the analysts for some excellent questions.
Robert I. Blum: We're excited to share this update with you, especially as it pertains to our expanded focus on the development program for <unk> Canton and at the same time.
Robert I. Blum: We believe that we're being good financial stewards, as we think about capital diversification and capital deployment and efficiencies and we're looking forward to updating you on continued progress, including with our presentations next week Monday May 13 at the European Heart failure meetings and afterwards.
Robert I. Blum: We thank you for your interest in Cytogenetics, and we'll look forward to the next earnings call operator with that we can now conclude the call.
Operator: Thank you. This concludes today's conference call. Thank you for your participation. You may now disconnect.
Speaker Change: Thank you. This concludes today's conference call. Thank you for your participation you may now disconnect.
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