Q4 2023 Cytokinetics Incorporated Earnings Call
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Operator: Good afternoon and welcome, ladies and gentlemen, to Cytokinetics' fourth quarter 2023 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.
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Speaker Change: Good afternoon, and welcome ladies and gentlemen, two sided kinetics fourth quarter 2023 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 question and answers. After the presentation, we will allow for only one question.
Operator: At the company's request, we will open the call for questions and answers after the presentation. We will allow for 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 Communication and Investor Relations. Please go ahead.
Speaker Change: Per participant and ask that you adhere to this request I went I will turn the call over to Diane Weiser Fido kinetics, Senior Vice President of corporate communication and Investor Relations. 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 quarter and recent developments. Patty Malik, EVP of R&D, will provide updates related to AFI Campton, focusing on Sequoia HCM and Forrest HCM. Stuart Kupfer, SVP and Chief Medical Officer, will provide additional updates for AFI Campton relating to Acacia HCM and Maple HCM, and will also discuss CK586 and CK136. Andrew Kalos, EVP and Chief Commercial Officer, will speak about commercial readiness activities for AFTI-CAMPDEN. Robert Wong, VP and Chief Accounting Officer, will provide a financial overview of the past quarter.
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 at all.
Diane Weiser: That email like E V. P of R&D will provide updates related to IP campaign focused just acquire HCM and forests HCM.
Diane Weiser: It cuts, our SVP and Chief Medical Officer will provide additional updates for IP Camden relating to Acacia HCM and Maple H C. M and will also discuss Teekay 586, and CK 136, and Joe Keller EVP and Chief commercial officer will speak about commercial readiness activities for Camden.
Diane Weiser: Robert One VP and Chief Accounting Officer will provide a financial overview of the past quarter, and finally, blah blah blah, and we'll discuss our 'twenty 'twenty four financial guidance and corporate development strategies before closing the call by reviewing expected key milestones for the year.
Diane Weiser: And finally, Robert Blum will discuss our 2024 financial guidance and 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 contained in our SEC filings, including our current report regarding our fourth quarter 2023 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.
Diane Weiser: 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.
Diane Weiser: To differ materially from those in these forward looking statements is contained in our SEC filings, including our current report regarding our fourth quarter 2023 financial results filed on form 8-K that was furnished to the SEC today, we undertake no obligation to update any forward looking statements. After this call and now I will turn the call.
Robert I. Blum: And now I will turn the call over to Robert. Thank you, Diane, and thanks for joining us on the call today. The fourth quarter of 2023 represented a transformational inflection point for our company as we turned the card on Sequoia HCM, our Phase III clinical trial of afecamtin for the potential treatment of patients with hypertrophic cardiomyopathy, or HCM. The results exceeded our already high expectations, and we began 2024 firing on all cylinders, with renewed commitments to preparing for regulatory interactions and submissions with urgency, executing On today's call, Fadi will discuss our plans for presentation and publication of primary and other results of Sequoia HCM, which will further elaborate on the efficacy and safety of our next-in-class cardiac myosin inhibitor, and as well as how we're moving swiftly to regulatory submissions across the globe.
Robert: Over to Robert.
Robert: Thank you Diane and thanks for joining us on the call today.
Robert: The fourth quarter of 2023 represented a transformational inflection point for our company as we turn the card on the Sequoia Hcl, our phase III clinical trial about the Camden for the potential treatment of patients with hypertrophic cardiomyopathy or HCM.
Robert: <unk> exceeded our already high expectations, and we began 2020 for firing on all cylinders with renewed commitments to preparing for regulatory interactions and submissions with urgency execute.
Robert: Using a broad clinical development program always behind its quite aged.
Robert: Activating the next phase of commercial readiness activities.
Robert: But today's call Sandy will discuss our plans for presentation and publication of primary and other results of Sequoia ACM, which will further elaborate on the efficacy and safety of our next in class cardiac myosin inhibitor.
Sandy: And as well.
Sandy: We're moving swiftly directly short regulatory submissions across the globe and then Andrew will comment on how we are prudently planning for differentiated commercial positioning for our next Gen class opportunity.
Robert I. Blum: And then Andrew will comment on how we're prudently planning for differentiated commercial positioning for our next-in-class opportunity. As important as Sequoia HCM is to our path to commercialization, there's much more in our pipeline that we believe will meaningfully unlock shareholder value as the company continues to mature. Stuart will provide an update on the progress of Maple HCM and Acacia HCM, the additional Phase III clinical trials of Afecamton, which will provide an on-ramp to potentially expanding the utility of cardiac myosin inhibitors as a first-line treatment for obstructive HCM, as well as potentially provide a new treatment option for the growing number of patients with non-obstructive HCM. As you'll hear, there's been increased enthusiasm and activity surrounding these trials on the heels of the positive results from Sequoia HCF.
Andrew: As important as the core HCM as to our path to commercialization. There is much more in our pipeline that we believe will meaningfully unlock shareholder value.
Andrew: Company continues to mature.
Andrew: <unk> will provide an update on the progress of Maple HCM and Acacia HCM. The additional phase III clinical trials of <unk>, which will provide an on ramp to potentially expanding the utility of cardiac myosin inhibitors as a first line treatment for obstructive HCM as well as potentially provide.
Andrew: A new treatment option for the growing number of patients with non obstructive HCM.
Andrew: Youll hear there's been an increased enthusiasm and activity surrounding these trials on the heels of the positive results from Sequoia ACF.
Robert I. Blum: However, much still remains ahead of us to bring Afikampton to patients, but I'm confident in our ability to execute on our ambitious strategies. As you'll hear in more detail, we ended 2023 with a strong balance sheet thanks to reduced spending during the year. In the last quarter and earlier this year, we also added to our cash balance with an infusion of capital from our at-the-market, or ATM, equity vehicle. We're pleased to be reporting our financial guidance today with approximately two years of cash runway. When accounting for both our cash on hand and cash available to us, and despite projecting an increase in our expected operating expenses in 2024. Recently, we announced the retirement of Ching Jha from his position as CFO in order to attend to his personal health. King's departure is unrelated to our business prospects, and we are on mutually good terms with the company.
Andrew: However, much still remains ahead of us to bring out the campaign to patients, but I am confident in our ability to execute on our ambitious strategies.
Andrew: As you'll hear in more detail. We ended 2023 with a strong balance sheet. Thanks drew reduce spending during the year in the last quarter and earlier. This year. We also added to our cash balance with an infusion of capital from our aftermarket or ATM equity vehicle, we're pleased to be reporting our <unk>.
Andrew: The annual guidance today with approximately two years of cash runway when accounting for both our cash on hand and cash available to us.
Andrew: And despite projecting an increase in our expected operating expenses in 2024.
Speaker Change: Recently, we announced the retirement of Ching jaw from his position as CFO in order to return to his personal health changed.
Speaker Change: <unk> departure is unrelated to our business prospects and on mutually good terms with the company.
Robert I. Blum: On behalf of our senior leadership team, we support his decision for his well-being, and we express our gratitude for his many contributions to our company. Ching built a strong team that many of you may know, including Robert Wong, VP and Chief Accounting Officer, as well as Matt Yang, VP of Corporate Finance and FP&A. Together with the teams they have assembled, we're confident that Ching's departure will have minimal impact on day-to-day financial operations.
Speaker Change: On behalf of our senior leadership team, we support his decision for us will be and we express our gratitude for his many contributions to our company.
Speaker Change: <unk> built a strong team that many of you may know, including Robert Wong VP, and Chief Accounting officer, as well as Matt Yang VP of corporate finance and SG&A together with the teams. They have assembled we're confident that <unk> departure will have minimal impact to day to day financial operations.
Robert I. Blum: Libby Schneiders, our SVP of business development, who has served our company for well over 20 years, continues to lead business development, so we do not foresee any impact on that front. Furthermore, in anticipation of Ching having to attend to his health in the fourth quarter of 2023, we brought on a seasoned consultant who previously served as CFO of a public biopharma company and who has extensive experience in financial planning and structured finance transactions. He'll continue to work with me through this transition, as we've already initiated a national search for Ching's replacement that we expect will bring international commercial and capital allocation experience compatible with maturing operations. Cytokinetics is looking ahead to a bright future in 2024 and also beyond, and as we turn the page towards potential commercialization of the first medicine arising from our pioneering and leading muscle biology research, with more to come as we continue to pursue our R&D programs. We're fortunate to be where we are today, but it is not by coincidence.
Speaker Change: <unk>, our SVP of business development, who has served our company for well over 20 years continues to lead business development. So we do not foresee any impact on that front.
Speaker Change: Furthermore, in anticipation of cheating having to attempt to resolve in the fourth quarter of 2023, we brought on a seasoned consultants, who previously served as CFO of a public Biopharma company and who has extensive experience in financial planning and structured finance transactions.
Speaker Change: You need to work with me through this transition as we've already initiated a national search for <unk> replacement that we expect will bring international commercial and capital allocation experience compatible with maturing operations.
Speaker Change: So that'll kinetics as looking ahead to a bright future in 2024 and also beyond that as we turn the page towards potential commercialization of the first medicine arising from our pioneering and leading muscle biology research with more to come as we continue to prosecute our R&D programs.
Speaker Change: We're fortunate to be where we are today, but it is not by coincidence. It is a result of meticulous planning risk mitigation strategic foresight perseverance and dedication.
30: We're building a specialty cardiology company, leading without the Camden as the foundation and we have the pipeline the passion and the people to make happen that vision as we believe will further reward shareholders with that I'll turn the call over to 30. Please.
Robert I. Blum: It's a result of meticulous planning, risk mitigation, strategic foresight, perseverance, and dedication. We're building a specialty cardiology company, leading with Affy Campton as the foundation, and we have the pipeline, the passion, and the people to make that vision come true, as we believe will further reward shareholders. With that, I'll turn the call over to Fatty, please.
30: Thanks, Robert during the quarter, we shared top line results from Sequoia, HCM, which as Robert said exceeded our expectations. It was a truly extraordinary milestone reflective of an incredible commitment from so many including our investigators study staff patients and of course, our teams etcetera kinetic.
30: <unk>.
30: The results of Sequoia HCM showed that treatment with Abbvie kimpton significantly improved exercise capacity compared to placebo.
Patty Malik: Thanks, Robert. During the quarter, we shared top-line results from Sequoia HCM, which, as Robert said, exceeded our expectations. It was a truly extraordinary milestone, reflective of an incredible commitment from so many, including our investigators, study staff, patients, and, of course, our teams at Cytokinetics. The results of the Sequoia HCM showed that treatment with aficamptin significantly improved exercise capacity compared to placebo, increasing peak oxygen uptake, or peak VO2, measured by cardiopulmonary exercise testing by a least square mean difference of 1.74 milliliters per kilogram per minute, with a p-value of 0.000002.
30: Increasing peak oxygen uptake or peak via two <unk>.
30: Measured by cardiopulmonary exercise testing by lease square mean difference of 174 milliliters per kilogram per minute.
30: A P value of zero point zero zero zero zero zero too.
30: The treatment effect with Abbvie campton was consistent across all prespecified subgroups reflective of patient baseline characteristics and treatment strategies, including patients receiving are not receiving background beta blocker therapy.
30: Statistically significant and clinically meaningful improvements with a P value of less than 1.0001.
30: Observed across all 10 pre specified secondary endpoints, including the Kansas City Cardiomyopathy questionnaire clinical summary score at weeks 12, and 24, the proportion of patients with a greater than or equal to one class improvement in New York Heart Association functional class at.
Patty Malik: The treatment effect with apicamptin was consistent across all pre-specified subgroups, reflective of patient baseline characteristics and treatment strategies, including patients receiving or not receiving background beta blocker therapy, statistically significant and clinically meaningful improvements with a p-value of less than 0.0001, were observed across all 10 pre-specified secondary endpoints, including the Kansas City Cardiomyopathy Questionnaire Clinical Summary Score at Weeks 12 and 24, the proportion of patients with a greater than or equal to one class improvement in New York Heart Association Functional Class at Weeks 12 and 24, the change in provoked left ventricular outflow tract gradient and proportion of patients whose gradient fell below 30 millimeters of mercury at Weeks 12 and 24, as well as exercise workload and guideline eligibility for septal reduction therapy. These positive results reflect rapid and sustained improvements of symptoms, functional capacity, and heart failure status.
30: <unk> 12, and 24 the change in provoked left ventricular outflow tract gradient and proportion of patients whose gradient fell below 30 millimeters of Mercury at weeks 12, and 24 as well as exercised workload and guideline eligibility for septal reduction therapy.
30: These positive results reflect rapid and sustained improvement of symptoms functional capacity and heart failure status.
30: <unk> was well tolerated with an adverse event profile comparable to placebo treatment emergent serious adverse events occurred in eight patients or five 6% on epic Hampton and 13 patients or nine 3% on placebo core echocardiographic left ventricular ejection.
30: Traction or Lv app was observed to be less than 50% five patients or three 5% on a captain compared to one patient or 0.7% on placebo.
30: Importantly, there were no increases worsening heart failure or treatment interruptions due to low <unk>.
Patty Malik: Apicamptin was well-tolerated, with an adverse event profile comparable to placebo. Treatment-emergent serious adverse events occurred in 8 patients, or 5.6% of those on apicamptin, and 13 patients, or 9.3% of those on placebo. Core echocardiographic left ventricular ejection fraction, or LVEF, was observed to be less than 50% in five patients, or 3.5% on apicampin, compared to one patient, or 0.7% on placebo. Importantly, there were no instances of worsening heart failure or treatment interruptions due to low LVEF.
30: While these top line results provide a relatively comprehensive look at the overall effect of treatment with App at Canton, we have an extensive plan to share. The primary results in additional analysis in more detail in a series of published manuscripts and presentations. We hope the first of these to occur at heart failure 2020.
30: For the annual meeting of the Heart failure Association of the European Society of Cardiology, taking place in May and Elizabeth.
30: Along with the primary results from Sukhoi HCM over the coming months, we plan to present and publish key data that may lend support to the differentiated profile back in Canada.
30: One important analysis is to examine the clinical effectiveness of Asti Camden in patients across several end points in aggregate examining the breath of improvements patients experienced in Sequoia HCM.
30: And another we plan to elaborate on the dosing and safety experience from Sukhoi, HCM, which we believe informs the potential safety and monitoring needed in the clinical environment.
Patty Malik: While these top-line results provide a relatively comprehensive look at the overall effect of treatment with afecamptin, we have an extensive plan to share the primary results and additional analyses in more detail in a series of published manuscripts and presentations. We hope the first of these will occur at Heart Failure 2024, the annual meeting of the Heart Failure Association of the European Society of Cardiology taking place in May in Lisbon. Along with the primary results from Sequoia HCM, over the coming months, we plan to present and publish key data that may lend support to the differentiated profile of African-Canadians. One important analysis is to examine the clinical effectiveness of apicampin in patients across several endpoints in aggregate, examining the breadth of improvements patients experienced with Sequoia HCM.
30: In addition to these important analyses from Sequoia HCM.
30: We have a robust scientific communications plan over the coming year that includes manuscripts in Congress presentations that will further elaborate on the effective appy campton another metrics of exercise capacity cardiac remodeling from the CMS sub study echocardiographic measures of systolic and diastolic.
30: Function in cardiac structure symptoms and quality of life and cardiac biomarkers.
30: We look forward to sharing these analyses with you in 2024, starting in Q2.
30: I'd like to acknowledge the tremendous efforts by our steering committee and internal teams to support and execute on this ambitious plan for publications and presentations.
30: Shifting over to forest HCM. The open label extension clinical trial that be Camden, We can report that over 90% of the patients eligible for Redwood HCM and Sequoia HCM have enrolled in forest HCM, representing nearly 300 patients that will contribute to our understanding of it.
Patty Malik: In another, we plan to elaborate on the dosing and safety experience with Sequoia HCM, which we believe informs the potential safety and monitoring needed in the clinical environment. In addition to these important analyses from Sequoia HCM, we have a robust scientific communications plan over the coming year that includes manuscripts and Congress presentations that will further elaborate on the effect of apicamptin on other metrics of exercise capacity, cardiac remodeling from the CMR sub-study, echocardiographic measures of systolic and diastolic function and cardiac structure, symptoms and quality of life, and cardiac biomarkers. We look forward to sharing these analyses with you in 2024, starting in Q2. I'd like to acknowledge the tremendous efforts by our Steering Committee and internal teams to support and execute on this ambitious plan of publications and presentations.
30: <unk> of long term treatment with Abbvie Kempton.
30: Please note that this does not include patients from the China cohort of Sequoia HCM. These patients will eventually make their way into a China specific open label extension clinical trial.
30: In April at the American College of Cardiology annual scientific sessions, we will present, the efficacy and safety of Abbvie Camden in the first cohort of patients with symptomatic obstructive HCM that have completed one year of follow up in four states.
30: Last month, we shared data at <unk> 2024 from the cardiac cardiac magnetic resonance our CMO our sub study of four states.
Patty Malik: Moving over to FOREST-HCM, the Open Label Extension Clinical Trial of Aficamptin, we can report that over 90% of the patients eligible from Redwood HCM and Sequoia HCM have enrolled in FOREST-HCM, representing nearly 300 patients that will contribute to our understanding of the effects of long-term treatment with Aficamptin. Please note that this does not include patients from the Chinese cohort of SCCOI HCM.
30: The results showed that treatment with <unk> for 48 weeks resulted in cardiac structural remodeling improvements in cardiac function and stabilization of myocardial fibrosis, demonstrating that that be Camden has potential disease modifying effects and ability to improve the architecture of the heart in patients with obstructive.
30: <unk> HCM.
This analysis is small only 16 patients who are eligible at the time of this data cut.
30: We plan to expand on these data in the future as more patients reached the one year Mark and beyond.
Patty Malik: These patients will eventually make their way into a China-specific open-label extension clinical trial. In April, at the American College of Cardiology Annual Scientific Sessions, we will present the efficacy and safety of affecampin in the first cohort of patients with symptomatic obstructive HCM that have completed one year of follow-up in forest HCM. Last month, we shared data at CMR 2024 from the Cardiac Magnetic Resonance, or CMR, sub-study of forest HCM. The results showed that treatment with Aficamtin for 48 weeks resulted in cardiac structural remodeling, improvements in cardiac function, and stabilization of myocardial fibrosis, demonstrating that Aficamtin has potential disease-modifying effects and the ability to improve the architecture of the heart in patients with obstructive HCM.
30: Regarding regulatory engagements during the month of February we held two meetings with the FDA ahead of our expected submission of an NDA in the third quarter of this year.
30: A first meeting to review the results of Sequoia HCM and a second pre NDA meeting, providing an opportunity to align on the content and format of the NDA.
30: We're pleased with the Fda's feedback supporting the sufficiency of our proposed NDA submission package and their receptivity to our rolling submission plan.
30: We believe that the positive readout for Sukhoi HCM, along with a favorable pharmacologic and Adi profile of that campaign.
30: To support the opportunity to achieve differentiated labeling and risk mitigation.
30: We look forward to providing future updates regarding our interactions with FDA this year.
30: As to regulatory planning and interactions in Europe were similarly ready for submission of our marketing application in the fourth quarter of this year and plan to meet with the EMA in Q2 to inform preparations. Similarly, we're coordinating with our partner <unk> Sheng in China.
Patty Malik: While this analysis is small, only 16 patients were eligible at the time of this data cut. We plan to expand on these data in the future as more patients reach the one-year mark and beyond. Regarding regulatory engagements, during the month of February, we held two meetings with FDA ahead of our expected submission of an NDA in the third quarter of this year, a first meeting to review the results of Sequoia HCM, and a second pre-NDA meeting, providing an opportunity to align on the content and format of the NDA. We're pleased with the FDA's feedback supporting the sufficiency of our proposed NDA submission package and their We believe that the positive readout for sequoia HCM, along with the favorable pharmacologic and DDI profile of apicampin, continue to support the opportunity to achieve differentiated labeling and risk mitigation.
30: To collaboratively support <unk> plans to submit an NDA.
30: We're all our proactive planning in 2023 has enabled us to capitalize on the positive results from <unk> HCM and to proceed with urgency towards global regulatory submissions as well as to consider expedited pathway is consistent with our aggressive planning scenarios.
30: Alongside all of this from a medical affairs perspective during the quarter, our therapeutic medical science liaisons continued profiling of HCM treatment programs, while our managed health care Medical Science Liaisons began development of our payer clinical value proposition.
30: We also continued our support of medical education activities at medical conferences.
30: As Robert said the strength of the specialty Cardiology company. We are building is anchored in the power of our research engine and development pipeline.
30: With acid Kimpton, if approved leading the way.
30: The topline results from <unk> have been well received by the HCM community of physicians and patient advocates who foresee that they represent what can be a meaningful advance in care. We're grateful for their support and we look forward to continuing to engage with them as we work to establish <unk> as a potential next in class.
Patty Malik: We look forward to providing future updates regarding our interactions with FDA this year. As to regulatory planning and interactions in Europe, we're similarly ready for submission of our marketing application in the fourth quarter of this year and plan to meet with EMA in Q2 to inform preparation. Similarly, we're coordinating with our partners Yixing and China to collaboratively support Yixing's plans to submit an NDA.
30: <unk> treatment option for patients with HCM.
30: Now I'll hand, it over to Stuart to elaborate more on additional clinical trials progress, we're making with Abbvie kimpton and provide an update on our earlier stage clinical development pipeline.
Patty Malik: Overall, our proactive planning for 2023 has enabled us to capitalize on the positive results from Sequoia HCM and to proceed with urgency towards global regulatory submissions as well as to consider expedited pathways consistent with our aggressive planning scenarios. Alongside all of this, from a medical affairs perspective, during the quarter, our therapeutic medical science liaisons continued profiling of HCM treatment programs, while our managed health care medical science liaisons began developing our payer clinical value proposition. We have also continued our support of medical education activities at medical conferences.
Stuart: Thank you Patty.
In addition to sharing positive results strong Sequoia HCM during the fourth quarter, we continued enrollment in our building momentum with our two ongoing phase III clinical trials, so that the cancer.
Stuart: <unk> HCM and Acacia hei.
Stuart: We're pleased to say that screening and enrollment is accelerating had been catalyzed by the announcement of results from <unk>.
Stuart: Enable HCM, which is evaluating the potential superiority of that the Camden as monotherapy compared to <unk> <unk> as monotherapy in patients with obstructive HCM nearly all U S sites are now activated and we've now activated sites in the U K, France, Italy, the Netherlands and Israel.
Stuart: We expect to complete enrollment in Maple HCM in the third quarter of this year, which would enable results from maple HCM to be available in 2025 concurrent with when we hope to be commercially launching an app in cancer.
Stuart Kupfer: As Robert said, the strength of the specialty cardiology company we are building is anchored in the power of our research engine and development pipeline, with Atkin Kempton, if approved, leading the way. The top-line results from Sequoia HCM have been well-received by the HCM community of physicians and patient advocates who foresee that they represent what can be a meaningful advance in care. We're grateful for their support, and we look forward to continuing to engage with them as we work to establish Affy Kempton as a potential next-in-class treatment option for patients with HCM. Now, I'll hand it over to Stuart to elaborate more on the additional clinical trial progress we're making with Affy Kampton and provide an update on our earlier stage clinical development pipeline. Thank you, Patty.
Stuart: We believe that if maple HCM, where to read out positively it will provide support for the positioning of Abbvie Camden is a first line therapy for patients with obstructive HCM.
Stuart: We're developing anti Camden to capitalize on this next in class potential and Maple HCM factors importantly into that strategy.
Stuart: Acacia HCM the pivotal phase III clinical trial of anti cancer in patients with symptomatic non obstructive HCM is currently focused on study startup, but we have the necessary IRB approvals in the U S and the EU, we're progressing our clinical trial application through the new harmonized procedure.
Stuart: We plan to hold investigator meetings for North America, South America, and Europe in the second quarter and look forward to continuing enrollment in this trial through 2024 with the objective to complete enrollment in 2025.
Stuart Kupfer: In addition to sharing positive results from Sequoia HCM, during the fourth quarter, we continued enrollment and are building momentum with our two ongoing phase three clinical trials of aficampin, Maple HCM, and Acacia HCM. We're pleased to see that screening and enrollment are accelerating and have been catalyzed by the announcement of results from Sequoia HCM. In MAPLE-HCM, which is evaluating the potential superiority of apicampin as monotherapy compared to metoprolol as monotherapy in patients with obstructive HCM, nearly all U.S. sites are now activated, and we've now activated sites in the U.K., France, Italy, the Netherlands, and Israel.
Stuart: Acacia HCM represents an important opportunity for ASIC, Camden and test the key therapeutic hypothesis for expanding the evidence to support cardiac myosin inhibition and a growing population of patients underserved by current treatment options.
Stuart: We believe that the results from supply HCM and Redwood HCM.
Stuart: Basically add confidence to what we can expect from Acacia HCM.
Stuart: And our optimism is shared by clinical investigators.
Stuart: Both clinical trials have the potential to expand the utilization of Abbvie Camden in the HCM patient population and impact treatment guidelines.
Stuart: At the same time, we're pleased with the progress of our clinical trials of that the Camden in the past quarter. We also advanced our earlier stage development pipeline, notably with CK 586, our cardiac myosin inhibitor in development for the potential treatment of a subgroup of patients with heart failure preserved ejection fraction or half path.
Stuart Kupfer: We expect to complete enrollment in Maple HCM in the third quarter of this year, which would enable results from Maple HCM to be available in 2025, concurrent with when we hope to be commercially launching in Appicanton. We believe that if Maple HCM were to read out positively, it would provide support for the positioning of aficampin as a first-line therapy for patients with obstructive HCM. We're developing Aficampin to capitalize on its next-in-class potential, and Maple HCM factors importantly into that strategy. Acacia HCM, the Pivotal Phase III clinical trial of athecampin in patients with symptomatic non-obstructive HCM, is currently focused on study start-up, but we have the necessary IRB approvals in the U.S. and the E.U.
Stuart: Having completed analysis of the single ascending dose data, we proceeded to the multiple ascending dose portion of the study.
Stuart: We expect to complete the phase one study in this quarter and subsequently shared data in the second quarter of this year with plans to begin a phase II clinical trial in the second half of the year.
Stuart: We believe that the positive phase II data, we generated for App at Camden in patients with non obstructive HCM support the development of CK 586, and a population of patients with Pep path is condition has many parallels to non obstructive HCM.
Stuart: Currently in the United States. There are about $3 6 million people have path, which is expected to increase of $4 8 million by 2033.
Stuart Kupfer: We are progressing our clinical trial application through the new harmonized procedure. We plan to hold investigator meetings for North America, South America, and Europe in the second quarter and look forward to continuing enrollment in this trial through 2024 with the objective of complete enrollment in 2025. Acacia HVM represents an important opportunity for aficampin and tests a key therapeutic hypothesis for expanding the evidence to support cardiac myosin inhibition in a growing population of patients underserved by current treatment options. We believe that the results from Sequoia HCM and Redwood HCM faithfully add confidence to what we can expect from Acacia HBM.
Stuart: Approximately 30% to 40% of these patients present with characteristics that may make their condition amenable to cardiac myosin inhibition.
Stuart: Even with <unk> inhibitors. The first evidence based therapies, demonstrating some benefit in doses half path. There is still a high residual risk for cardiovascular events and a clear need for additional effective therapies.
Stuart: Additionally, during the past quarter for CK 136, our cardiac troponin activator. We continued analysis of the single and multiple ascending dose cohorts in the phase one study in healthy participants and expect to complete the study in the second quarter of this year.
Stuart: In 2024, we plan to share more updates from our early stage pipeline and our research.
Stuart: As more programs mature from our labs into the clinic, we will report on how in recent years, we've extended our focus in muscle biology beyond muscle contractility to also include other programs entering development, representing a potential innovations and muscle metabolism and energetics.
Stuart Kupfer: And our optimism is shared by clinical investigators. Both clinical trials have the potential to expand the utilization of apicampin in the HCM patient population and impact treatment guidelines. At the same time, we're pleased with the progress of our clinical trials at Vaticanpton. In the past quarter, we also advanced our earlier stage development pipeline, notably with CK586, our cardiac myosin inhibitor in development for the potential treatment of a subgroup of patients with heart failure with preserved ejection fraction, or HFPEF. Having completed analyses of the single ascending dose data, we proceeded to the multiple ascending dose portion of the study. We expect to complete the Phase I study this quarter and subsequently share data in the second quarter of this year with plans to begin a Phase II clinical trial in the second half of this year. We believe that the positive phase 2 data we generated for Affy Kampden in patients with non-obstructive HVM support the development of CK586 in a population of patients with PEPPF, because this condition has many parallels to non-obstructive HVM.
Stuart: Well I think Camden represents the most important near term value driver for our company.
Stuart: Our early stage pipeline and emerging programs from ongoing research demonstrate productivity against our vision 2025.
Stuart: Our long term goal is to cement our leadership in muscle biology, adjacent two exciting developments in treatments for cardio metabolic syndromes.
Stuart: We look forward to sharing more progress soon.
Stuart: With that I'll turn the call over to Andrew.
Andrew: Thanks Stuart.
Andrew: During the quarter.
Andrew: Head of our announcing the results from Sequoia HCM, we continued commercial readiness activities for Alphacat them throughout 2023, our focus was on learning the FERC phase in our go to market approach. We commissioned multiple market research studies seeking insights from nearly 850 health care professionals and more than 160 patients with <unk>.
Andrew: Our active HCM, which revealed that the HCM patient journey can be complex and challenging.
Andrew: Furthermore, HCM can also negatively impact patients overall mental health social engagement and other aspects of everyday life.
Andrew: We also tested a range of product profiles and market research prior to our receipt of the results from Sequoia HCM the.
Andrew: The tested attributes most closely resembling the actual Sequoia HCM results revealed that health care professionals would perceive the results from Sequoia ATM favorably that would drive use of after captain if approved across a broad range of obstructive HCM patients.
Stuart Kupfer: Currently, in the United States, there are about 3.6 million people with HEP PATH, which is expected to increase to 4.8 million by 2033. Additionally, approximately 30-40% of these patients present with characteristics that may make their condition amenable to cardiac myosin inhibition. Even with SGLT2 inhibitors, the first evidence-based therapies demonstrating some benefit in those with PEF-PEF, there is still a high residual risk of cardiovascular events and a clear need for additional effective therapy. Additionally, during the past quarter, for CK136, our cardiac troponin activator, we continued analyses of the single and multiple ascending dose cohorts in the phase one study of healthy participants and expect to complete the study in the second quarter In 2024, we plan to share more updates from our early stage pipeline and our research.
Andrew: These learnings are informing the strategic decisions, we're making around them a target product profile positioning potential customer profiles at our patient services hub access in particular is a key focus for us aligned with our company values of keeping patients at the forefront of all we do we're designing a comprehensive patient support program to facilitate and such.
Andrew: Port patients and transitioning to treatment with actually canton inclusion inclusive of patient education resources, and reimbursement support and affordability programs for eligible patients.
Andrew: In the fourth quarter of 2023, we held initial conversations with specialty pharmacies and patient service hub providers to support our build of a differentiated patient services hub.
Andrew: Now that we have results from Sequoia ATM. This year, we are advancing to the second and third stage phases of our go to market strategy design and build.
Andrew: This year, we plan to build our patient support services access strategy inclusive of distribution model contracting approach and pricing.
Andrew: Our seasoned account manager team is fully staffed and interacted with every major payer in 2023 to introduce cytogenetics. This field based payer account team will continue to further engage in 2024 to share the results from Sequoia ATM and insured payers understand the clinical meaningfulness of the results in 2010.
Stuart Kupfer: As more programs mature from our labs into the clinic, we will report on how, in recent years, we've extended our focus in muscle biology beyond muscle contractility to also include other programs entering development representing potential innovations in muscle metabolism and energetics. Well, I think Hampton represents the most important near-term value driver for our company, and our early stage pipeline and emerging programs from ongoing research demonstrate productivity against our vision 2025. The long-term goal is to cement our leadership in muscle biology adjacent to exciting developments and treatments for cardiometabolic syndromes. We look forward to sharing more progress soon. With that, I'll turn the call over to Andrew.
Andrew: Four we also plan to finalize our product positioning and launch our market development and education campaign, while we develop our branded marketing campaign in support of a potential 2025 promotional launch.
Andrew: As we previously shared we began building our commercial capabilities in the United States prior to the potential FDA approval and launch of OMA captive mccarville after receiving the <unk> from the FDA, we maintain the infrastructure that <unk> build and further refine that team and activities to prepare for what now will be our first commercial launch without <unk>.
Andrew: <unk>.
Andrew: Today, we have onboard the majority of U S based headquarter personnel that we expect to need including marketing field sales leadership and individuals leading insight generation market analysis commercial strategies systems and operations, we expect to expand the team in 2025 with ATP customer facing positions gate it appropriately.
Andrew: Alongside the regulatory process for App you Kathryn.
Andrew: In Europe, having hired key leadership positions last year, including our head of Europe and head of market access we plan to only modestly modestly expand our EU staff in 2024, while maintaining a disciplined <unk> spending and gaining cost of regulatory submissions and feedback from health technology assessments or HCA.
Andrew Kalos: Thanks, Stuart. During the quarter, ahead of our announcing the results from Sequoia HCM, we continued commercial readiness activities for Aficantin. Throughout 2023, our focus was on learning, the first phase in our go-to-market approach. We commissioned multiple market research studies, seeking insights from nearly 850 healthcare professionals and more than 160 patients with obstructive HCM, which revealed that the HCM patient journey can be complex and challenging. Furthermore, HCM can also negatively impact patients' overall mental health, social engagement, and other aspects of everyday life.
Andrew: It is encouraging to see favorable benefit assessment from HCA is in both Germany, and France related to cardiac myosin inhibitor as a new treatment option for patients with obstructive HCM and we believe this bodes well for future reimbursement of assay Campton, if approved across major European markets.
Andrew: Overall, I'm very pleased with our foundational commercial readiness work that we've completed ahead of sharing our results with Sequoia HCM last year, which sets us up to nimbly advance into the next phase of our planning in 2024 and can enable us to capitalize on what we believe will be differentiated positioning for abbvie Camden in the treatment of patients with obstructive HCM.
Andrew Kalos: We also tested a range of product profiles and conducted market research prior to our receipt of the results from Sequoia HCM. The tested attributes most closely resembling the actual Sequoia HCM results revealed that health care professionals would perceive the results from Sequoia HCM favorably and would drive use of athicampin if approved across a broad range of obstructive HCM patients. These learnings are informing the strategic decisions we're making around the target product profile, positioning, potential customer profiles, and our patient services hub. Access, in particular, is a key focus for us. Aligned with our company values of keeping patients at the forefront of all we do, we're designing a comprehensive patient support program to facilitate and support patients in transitioning to treatment with ATSI-Canton, inclusive of patient education resources and reimbursement support and affordability grant programs for eligible patients.
Andrew: With that I'll turn the call over to Robert Wong.
Robert Wong: Thanks, Andrew we ended the fourth quarter with $655 4 million in cash and investments, which included a $162 9 million that we raised through our at the market equity vehicle in the quarter. Following the quarter close we raised approximately $83 million net through yesterday.
Robert Wong: With our ATM equity vehicle, which is not reflected in our year end balance our fourth quarter 2023, R&D expenses increased to $85 million from $75 million in the fourth quarter of 2022, primarily due to spending on our cardiac myosin inhibitor programs.
Robert Wong: Our fourth quarter 2023, G&A expenses were $44 1 million down from $54 million in Q4, 2022, due primarily to lower outside spending on commercial activities offset by higher personnel related costs, including stock based compensation.
Robert Wong: Overall, our net cash burn in 2023 was $414 million relative to what was our initial 2023 guidance of $4 20 to $4 $50 million.
Andrew Kalos: In the fourth quarter of 2023, we held initial conversations with specialty pharmacies and patient service hub providers to support our build of a differentiated patient services hub. Now that we have results from the Sequoia ATM this year, we are advancing to the second and third stage phases of our go-to-market strategy, design, and build. This year, we plan to build our patient support services, and access strategy inclusive of a distribution model, contracting approach, and price. Our seasoned account manager team is fully staffed and has interacted with every major payer in 2023 to introduce cytokinetics. This field-based payer account team will continue to further engage in 2024 to share the results from Sequoia HCM and ensure payers understand the clinical meaningfulness of the results. In 2024, we also plan to finalize our product positioning and launch our market development and education campaign, while we develop our branded marketing campaign in support of a potential 2025 promotional launch.
Robert Wong: We believe we proved to be good stewards of shareholder capital by reducing spending ahead of the results of Sequoia HCM at the end of the year, which puts us in a stronger financial position to begin 2020 for now I'll hand, it over to Robert <unk> to review, our financial outlook 2020 for guidance and corporate development strategy.
Robert Wong: These.
Robert Wong: Thank you Robert today, we announced our financial guidance for 2024.
Robert: The company anticipates revenue will be in the range of $3 million to $5 million operating expenses will be in the range of $4 20 to $4 $50 million and net cash utilization will be approximately $3 $90 million to $420 million in terms of capital allocation. Our priorities are focused on benefiting patients and enriching shared.
Robert: Order value anchored by a prudent spending plan that balances advancing our commercial strategy with investment in our pipeline. Our foremost priority is advancing regulatory submissions for <unk> in obstructive HCM and ensuring commercial preparedness in key markets like the U S and Europe.
Robert: Secondly, and continuing Maple HCM and Acacia HCM, we plan to maximize the therapeutic potential of <unk> Kimpton and finally, we remain focused on growing our pipeline inclusive of CK 586, and bolstering our R&D platforms to sustain future innovation.
Robert: Inclusive of the approximately $83 million net raised in recent weeks through our ATM as well as cash available to us under our loan agreement with royalty pharma. Our current cash balance of 655 billion at the end of last year, representing approximately two years of forward cash.
Andrew Kalos: As we previously shared, we began building our commercial capabilities in the United States prior to the potential FDA approval and launch of Omicamptin-McCarbol. After receiving the CRL from the FDA, we maintained the infrastructure that had been built and further refined the team and activities to prepare for what now will be our first commercial launch with Aficamptin. Today, we have on board the majority of U.S.-based headquarters personnel that we expect to need, including marketing, field sales leadership, and individuals leading insight generation, market analysis, commercial strategy systems, and operations.
Robert: Cash runway based on our financial guidance and projected 2020 for operating expenses and net cash utilization with.
Robert: With positive results from the Sequoia HCM in hand, we've been looking at the arc of capital requirements, leading up to potential approvals and the global commercial launch of <unk> kimpton as well as sustaining and growing R&D through profitability as such we continue to focus on a multi pronged approach.
Andrew Kalos: We expect to expand the team in 2025 with HCP customer-facing positions gated appropriately alongside the regulatory process for apicampin. In Europe, having hired key leadership positions last year, including our head of Europe and head of market access, we plan to only modestly expand our EU staff in 2024 while maintaining a disciplined eye to spending and gating costs of regulatory submissions and feedback from health technology assessments, or HTAs. It is encouraging to see favorable benefit assessments from HTAs in both Germany and France related to cardiac myosin inhibitors as a new treatment option for patients with obstructive HCM.
Robert: To accessing capital that enables us to pull several different levers over time as has been our history, we plan to monetize our R&D progress and preserve shareholder value via partnering as well as structured finance engineering and other non dilutive approaches are prior.
Speaker Change: <unk> remains focused on business development and as you know we've been focused on the Japan deal for RP Kimpton, we're in active discussions with multiple parties and I am pleased with how that deal campaign is looking.
Speaker Change: Moreover, we're considering partnering CK 586 for the potential treatment of half path. While also preserving key rights for cytogenetics and both co development and co commercialization, we believe that our longstanding leadership in the area of cardiac myosin modulation for the treatment of severe.
Andrew Kalos: And we believe this bodes well for future reimbursement of acecantin if it is approved across major European markets. Overall, I'm very pleased with the foundational commercial readiness work that we completed ahead of sharing our results at Sequoia HCM last year, which sets us up to nimbly advance into the next phase of our planning in 2024 and can enable us to capitalize on what we believe will be differentiated positioning for azecamptin in the treatment of patients with obstructive HCM. And with that, I'll turn the call over to Robert Wong. Thanks, Andrew. We ended the fourth quarter with $655.4 million in cash and investments, which included $162.9 million that we raised through our at the market equity vehicle during the quarter.
Speaker Change: Cardiovascular diseases has enabled us to look at partnering in an advantaged way as would benefit shareholders. While also preserving important shareholder value in major markets of value for athlete Kempton.
Speaker Change: And Additionally, we may consider restructuring our debt and other novel ways to build on the momentum from Sequoia HCM and structured finance transactions that we believe would augment shareholder value and importantly would not subtract from it.
Speaker Change: I'll remind you that through our transaction with royalty pharma, we remain eligible for two additional loan tranches under our development funding agreement. The first tranche of $75 billion became available upon our sharing positive results from Sequoia HCM and we remain eligible to draw down.
Robert Wong: Following the quarter close, we raised approximately $83 million net through yesterday with our ATM equity vehicle, which is not reflected in our year-end balance. Our fourth quarter 2023 R&D expenses increased to $85 million from $75 million in the fourth quarter of 2022, primarily due to spending on our cardiac myosin inhibitor program. Fourth quarter 2023 G&A expenses were $44.1 million, down from $54 million in Q4 2022, due primarily to lower outside spending on commercial activities, offset by higher personnel-related costs, including stock-based compensation.
Speaker Change: For one year following our receipt of the results which occurred in late December.
Second tranche of $100 million will become available to us where we to choose to draw on it subject to the satisfaction of certain conditions. Most notable of which is the acceptance of an NDA submission for up the Camden in the United States.
Speaker Change: As we are now a company valued between.
Speaker Change: And $10 billion, we have an ambitious yet practical and realistic plan to increase shareholder value over the next three to five years to that which would be upwards of $15 billion to $20 billion. How do we get there by unlocking the value in our pipeline and maximizing our opportunities at <unk>.
Speaker Change: <unk> without the Camden in North America, and Europe based on the results from Sequoia Hcl from there, we hope to increase value as Maple HCM and Acacia HTM readout results that now have higher probability of technical success at <unk>, We expect CK 586.
Robert Wong: With positive results from Sequoia HCM in hand, we've been looking at the arc of capital requirements leading up to potential approvals and the global commercial launch of Affy Campton, as well as sustaining and growing R&D through profitability. As such, we continue to focus on a multi-pronged approach to accessing capital that enables us to pull several different levers over time. As has been our history, we plan to monetize our R&D progress and preserve shareholder value via partnering, as well as structural financial engineering and other non-dilutive approaches. Our priority remains focused on business development, and as you know, we've been focused on a Japan deal for Affy Campton. We're in active discussions with multiple parties, and I'm pleased with how that deal campaign is looking.
Speaker Change: To advance through proof of concept.
Speaker Change: With additional upside coming from our earlier stage pipeline.
Speaker Change: I also want to take a moment to address recent M&A speculation relating to subtle kinetics.
Speaker Change: Since sharing the positive results from Sequoia HCM subtle kinetics has been rumored to be an acquisition target.
Speaker Change: While we will not and cannot comment on specific speculations. Let me please be clear about one thing we.
Speaker Change: We did not initiate door, nor do we have a sale process ongoing however.
Speaker Change: However, as responsible fiduciary to our shareholders, but I can assure you that we thoroughly evaluate options that are presented and as you heard me say a moment ago. We continue to advance business development discussions we are optimistic about how those discussions are proceeding and we're committed to building a sustained.
Robert Wong: Moreover, we're considering partnering CK586 for the potential treatment of HFPEF, while also preserving key rights for cytokinetics in both co-development and co-commercialization. We believe that our longstanding leadership in the area of cardiac myosin modulation for the treatment of severe cardiovascular diseases has enabled us to look at partnering in an advantaged way, which would benefit shareholders, while also preserving important shareholder value in major markets of value As we are now a company valued between $8 and $10 billion, we have an ambitious yet practical and realistic plan to increase shareholder value over the next three to five years to that which would be upwards of $15 to $20 billion. How do we get there?
Speaker Change: <unk> specialty cardiology company as starts with executing against our goal of bringing new medicines to patients.
Speaker Change: We take great care in fulfilling our duty to shareholders by ensuring that we're doing the right thing to deliver maximum value and controlling that which we can control. We believe that can be best achieved by advancing our innovative science and ensuring operational efficiencies and thoughtfully.
Speaker Change: Prudently, managing and deploying capital to maximize shareholder value.
Speaker Change: We're beginning 2024 and an advantaged position.
Speaker Change: We are turning the page to the next chapter of the cyber kinetic story and the work. We're now undertaking will set the stage for our first potential regulatory approvals, we hope within the next 18 months in the second half of this year, we expect to submit regulatory filings in both the United States and Europe for <unk>.
Robert Wong: By unlocking the value in our pipeline and maximizing our opportunities. It starts with Affy Campton in North America and Europe based on the results from Sequoia HCM. From there, we hope to increase value as Maple HCM and Acacia HCM read out results that now have a higher probability of technical success. Next, we expect CK586 to advance through proof of concept and half path with additional upside coming from our earlier stage pipeline. Since sharing the positive results from Sequoia HCM, cytokinetics has been rumored to be an acquisition target.
Speaker Change: Hampton you heard from <unk> in Q3, an NDA with FDA and in Q4 and MAA with ebay. We expect can be more precise timing goes through our Q1 earnings call and especially as it relates form positioning.
Speaker Change: Contained within the content of those submissions.
Robert I. Blum: We're beginning 2024 in an advantaged position. We have turned the page to the next chapter of the cytokinetic story, and the work we're now undertaking will set the stage for our first potential regulatory approvals, we hope, within the next 18 months. In the second half of this year, we expect to submit regulatory filings in both the United States and Europe for AFI Camden. You heard from FADI in Q3, an NDA with FDA, and in Q4, an MAA with EMA. We expect to be more precise as timing goes to our Q1 earnings call, and especially as it relates to positioning and other aspects contained within the content of those submissions. For AFI-CAMPTEN, we expect to present primary results from Sequoia HCM at a medical conference in Q2 2024. 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. We expect to complete enrollment in Acacia HCM in 2025 but continue its enrollment in 2024.
Speaker Change: And we have ambitious goals beyond obstructive HCM as well as beyond RP campton, including expanding our development pipeline with new compounds arising from our research extending beyond the contractility of muscle to the energetics growth and metabolism of muscle each of our.
Speaker Change: Programs in muscle biology has been designed to potentially address severely ill and underserved populations in need of new therapies and our vision of building a specialty cardiology company is now being realized and we are well positioned for success.
Speaker Change: With that I'd now I'd like to share our expected 2024 milestones for <unk> Kimpton, we expect to present primary results from Sequoia HCM at a medical conference in Q2 2024.
Speaker Change: 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 and Maple HCM in Q3, 2024, we expect to complete enrollment of Acacia HCM and <unk>.
Speaker Change: 25, but continue its enrollment in 2024, and we expect to continue advancing go to market strategies for <unk>.
Robert I. Blum: And we expect to continue advancing go-to-market strategies for AFI-CAMPTEN. For Omicampt and McCarville, we expect the CHMP to issue an opinion regarding the MAA in Q2 2024. For CK5A6, we expect to share data from the Phase 1 study in Q2 2024. And finally, for CK136, we expect to complete the Phase 1 study in the second quarter of this year.
Speaker Change: For <unk>, we expect to see HMP to issue an opinion regarding the MAA in Q2 2024 for CK 586, we expect to share data from the phase one study in Q2 2024, and finally for CK 136, we expect to complete.
Speaker Change: The phase one study in the second quarter of this year.
Operator: And, Operator, with that, we can now open up the call, please, to questions. Thank you. 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.
Operator: Operator with that we can now open up the call pleased to questions.
Thank you to ask a question. Please press star one one on your telephone and wait for your name to be announced to withdraw your question. Please press star one again.
Operator: As a reminder, the company has requested that each participant please adhere to the allowance of one question per person. Please stand by, we compile the Q&A roster. And our first question comes from MyInc., Ma'am Tani with B. Reilly Securities. Your line is open. Good afternoon, team.
As a reminder, the company has requested that each participant please adhere to the allowance of one question per person. Please.
Operator: These standby, we compile the Q&A roster.
Operator: And our first question comes from My Inc name, Tony with B Riley Securities. Your line is open.
MyInc, Ma'am Tani: Thanks for taking our questions, and I hope for Ching's well-being too. So, just quickly on the pre-NDA meeting that occurred, I was just curious how much of the Forest HCM data, you know, would be part of that submission? I believe by the ESC Heart Failure Congress, you'd probably have about 150 patients go through the titration phase.
Tony: Good afternoon, Deane, thanks for taking our questions and hopefully change well being too. So just quickly on the pre NDA meeting that occurred I was just curious how much of the potash at cm data.
Tony: It would be bottled that submission I believe.
Tony: By the ESC heart failure Congress, you'd probably have about 150 patients.
To go through the titration phase of this unit.
Patty Malik: I'm just curious about how much of that is going to be important as part of the, you know, pre-NDA meeting and eventually make it into the NDA submission. So I'll turn that call, that question, rather, over to Fadi. Thanks.
Tony: How much of that is going to be important outside of the.
Tony: Pre NDA meeting in <unk>.
Tony: Eventually make it into the NDA submission.
30: So I'll turn that call that question rather over to 30.
Tony: Thanks.
Patty Malik: So I can't give you the exact number of patients that will be available. But we also, besides the number of patients who have extended follow-up in FOREST at the time of NDA submission will also have the opportunity to submit an update at the day 120 time point. There's a day 120 safety update where we can expand the data set. So, you know, we fully expect the number of patients with, you know, up to a year of follow-up to be perfectly adequate for their review. I understood. And a related question, just quickly, the ACACIA at CM: I'm glad you narrowed the timelines there.
30: So I can't give you the exact number of patients that will be available, but we also besides of the number of patients.
30: Who have extended follow up in and.
30: Forest the time of NDA submission, we will also have the opportunity to submit an update at the day 120 time point, there's a day 120 safety update where we can expand the data set so we fully expect the number of patients with up to a year of follow up to be perfectly add.
30: Quit for their review.
Speaker Change: I understood and a related question just quickly.
Speaker Change: Acacia <unk> I'm glad you narrowed the timelines there.
Patty Malik: I was just curious, based on everything we know about dosing, safety, and cardiac remodeling data, just your confidence level for that, you know, what you could report there, also recognizing that a threshold could be set from the ODC at CM study early part of next year. Thanks again for taking. Well, I think based on the results that we saw in Phase 2 and the predictability of dosing and the safety profile we saw with apicampin and sequoia, we feel very confident with the outcomes and potential outcomes of Acacia. So, I think all of them read well on the likelihood of a trial reading out positively, and, you know, we'll continue to conduct it in a way that helps us get to the best answer. Thank you for the question. Just a reminder, please.
Speaker Change: Just curious there.
Speaker Change: <unk> done everything we know on dosing safety <unk> modeling data.
Acacia: Your confidence level for that.
Acacia: You could report there.
Acacia: Also recognizing that obsession could be set from the OTC SDN study next early part of next year. Thanks again for taking my questions.
Speaker Change: Well I think based on the results that we saw in phase two and the predictability of dosing and the safety profile, we saw that the Camden in Sequoia.
Speaker Change: We feel very confident with the outcomes.
Speaker Change: And potential outcomes in Acacia so I think all of them really well on the likelihood of that trial reading out positively in.
Speaker Change: We will continue to conduct it in a way that helps us get to the best answer.
Speaker Change: Thank you for the question just a reminder, please one question per.
Operator: One question per analyst, please. Next question, please. Our next question comes from the line of Salim Saeed with Museo.
Speaker Change: And analysts. Please next question please.
Speaker Change: Our next question comes from the line of Salim Syed with Mizuho. Your line is open.
Salim Saeed: Your line is open. Good afternoon, Cytokin. Hey, good afternoon, Robert, and hello, everybody. Robert, I just wanted to focus on, maybe this is for Patty as well, but on the rolling submission. What exactly is going in for it? I believe that's the first time you guys are using that terminology, if I'm not mistaken, rolling submission for AFI camp. And so what exactly is going on?
Salim Syed: Good afternoon.
Salim Syed: Hey, good afternoon, Robert and Hello, everybody.
Salim Syed: Robert I, just wanted to focus on maybe thats for that as well, but on the rolling submission what exactly is going in first I believe is the first time you guys are using that terminology, if I'm not mistaken rolling submission for <unk> Kimpton, So what exactly is going in.
Salim Syed: And then what is the piece that you plan to submit.
Salim Syed: Later and then.
Salim Syed: Curious also if the rolling submission if that in any way encompasses potential too.
Robert I. Blum: first, and then what is the piece that you plan to submit? later, and then curious also if the rolling submission, if that in any way encompasses the potential to submit the MAPLE HCM data, because you're going to get that before an approval decision. Yes, so I'll answer that question.
Salim Syed: The Maple HCM.
Salim Syed: <unk>.
Salim Syed: Because you're going to get that before an approval decision. Thank you.
Speaker Change: Yeah. So I'll answer that question. So you are right. We are using that terminology for the first time and thats coming out of our recent meetings with FDA. We're very encouraged by what appears to be a leaning forward.
Robert I. Blum: So, you're right, we are using that terminology for the first time, and that comes out of our recent meetings with FDA. We're very encouraged by what appears to be a leaning forward and willingness to consider these data sooner. We're looking at how we might accelerate submission, and in that regard, they were amenable to our recommendation that we do it on a rolling basis.
Speaker Change: Willingness to consider.
These data sooner, we're looking at how we might accelerate.
Speaker Change: Submission and in that regard they were amendable to a recommendation that we do it in a rolling basis, we're going to be submitting the most of the modules earlier than what's going to be the long pole in the tent. So to speak in terms of what will be coming in later will be some of the CMC data that we're still in the process of fine.
Speaker Change: <unk>.
Speaker Change: In light of the fact that there are certain time points, we need to collect in order to be able to finalize those submissions, but all of this speaks to we think.
Robert I. Blum: We're going to be submitting most of the modules earlier, and what's going to be the long pole in the tent, so to speak, in terms of what will be coming in later, will be some of the CMC data that we're still in the process of finalizing, in light of the fact that there are certain time points we need to collect in order to be able to finalize those submissions. But all this speaks to, we think, a focus forward in connection with a submission that could be started sooner. Keep in mind that the actual filing doesn't change. That ultimately depends on the final modules as they are then submitted, but it certainly allows the review to begin. As it relates to MAPLE, no, we don't expect that those data will be contained in this submission. MAPLE won't be read out until next year.
Speaker Change: Our focus forward in connection with a submission that could be started sooner keep in mind that the actual filing doesn't change that ultimately depends on the final mark.
Speaker Change: <unk> as they are then submitted but it certainly allows the review to begin.
Speaker Change: As it relates to Maple no. We don't expect that those data will be contained in this submission may four won't read out till next year.
Speaker Change: Okay got it thank you very much.
Speaker Change: Thank you Celine.
Speaker Change: Our next question comes from the line of Aswany Verma with UBS. Your line is open.
Aswany Verma: Hi, good afternoon.
Aswany Verma: Hi, good afternoon, Bob on behalf of Ashbury from UBS just two.
Salim Saeed: Okay, I got it. Thank you very much. Thank you, Salim.
Aswany Verma: Real quick question in terms of cardiology in general just a high level.
Ashwani Verma: Our next question comes from the line of Ashwani Verma with UBS. Your line is open. Hi there. Hi Fatma, on behalf of Ash Horma from UBS. Just a general quick question in terms of cardiology in general, just a high level. Is portfolio level contracting common?
Aswany Verma: Portfolio level contracting comment like what such a practice.
Aswany Verma: Companies with a broad set of offerings may get favorable favorable benefit of scale in the long run.
Speaker Change: I don't understand that question.
Speaker Change: Might you repeat it please.
Speaker Change: Sure, Yes, Im just trying to understand.
Robert I. Blum: Would such a practice mean that companies with a broad set of offerings may get favorable benefits of scale in the long run? Um, I don't understand that question. Might you repeat it, please? So yeah, I'm just trying to understand, in the cardiology space, is portfolio level contracting common? Like, do you think companies that have a broad set of diverse portfolios and cardiology have a larger benefit compared to companies that are focused on single assets?
Speaker Change: Cardiology.
Speaker Change: In fact for you on that little contracting call me like do you think companies that have like a broad set of diverse portfolio in cardiology. They have like a larger benefit from Baird company, they're focused on single asset.
Speaker Change: I see your question is are we going to be potentially disadvantaged because we will be launching with the single product relative to payers I assume who might have multiple who might be contracting around multiple products is that right.
Speaker Change: Yes.
Speaker Change: Sure.
Speaker Change: A powerpoint.
Speaker Change: Yeah from a payer point of view no I don't believe we're at a disadvantage payers aren't managing this category is a rare disease, there's over 120 payers in the U S. Each payer.
Robert I. Blum: I see. Your question is, are we going to be potentially disadvantaged because we'll be launching with a single product relative to payors, I assume, who might have multiple, who might be contracting around multiple products? Is that right?
Speaker Change: <unk> has very limited number of patients so it's not a big budget impact.
Speaker Change: It's a high unmet need there is one product in the market our second product. So usually arent payer managed categories from a cardiology point of view.
Robert I. Blum: Yeah. So from a payer point of view. From a payer point of view, no, I don't believe we're at a disadvantage. Payers aren't managing this category. It's a rare disease.
Speaker Change: The focus of of <unk>.
Speaker Change: Subset of Cardiologists, we will focus on those that.
Speaker Change: That diagnose and treat HCM as a subset of overall cardiology and highly motivated to treat the disease. So they're going to be more focused on the products that patients relative to the disease. So.
Robert I. Blum: There are over 120 payers in the U.S. Each payer, you know, only has a very limited number of patients. It's not a big budget impact. It's a high unmet need. There is, you know, one product in the market, and a second product. So these usually aren't payer-managed categories.
Speaker Change: I all means I think we will compete fine and it's not going to be at a disadvantage.
Speaker Change: Okay, great. Thank you so much for taking our question.
Speaker Change: Thank you.
Speaker Change: Our next question comes from the line of Mike <unk> with Barclays. Your line is open.
Andrew Kalos: From a cardiology point of view, you know, the focus of a subset of cardiologists was what we would focus on, those that diagnose and treat HCM as a subset of overall cardiology and are highly motivated to treat the disease. So they're going to be more focused on the products, and the patients relative to the disease. So by all means, I think we'll compete fine, and it's not going to be at a disadvantage. Okay, great. Thank you so much for taking our questions. Thank you. Our next question comes from the line of Maya Iskandarani with Barclays. Your line is open. Good afternoon. Hi, this is Carter.
Mike: Good afternoon.
Mike: Hi, This is Carter thanks for taking the question Robert.
Carter: Maybe for <unk>.
Robert and fatty.
Mike: The commentary on the Ram sort of referenced Sequoia PK in the DDI profile, all of which we've known for a while I guess now but can you comment if the FDA interactions in any way shifted or reaffirmed your belief in that differentiated label ramps. Thank you.
Speaker Change: Yeah, I'll just comment very briefly it's premature because those conversations will continue but reaffirming our view that.
Speaker Change: Between positioning labeling lesser rooms, and other things that read on safety and risk mitigation that we will be in a position to have a very constructive fruitful conversation with FDA.
Paul Choi: Thanks for taking the question, Robert. Maybe for Robert and Fatty, commentary on the REMS sort of reference Sequoia PK and the DDI profile, all of which we've known for a while, I guess now, but can you comment if the FDA interactions in any way shifted or reaffirmed your belief in that differentiated label or REMS? Yeah, I'll just comment very briefly, it's premature because those conversations will continue, but reaffirming our view that between positioning, labeling, lesser REMS, and other things that read on safety and risk mitigation, we'll be in a position to have a very constructive, fruitful conversation with FDA. Next question, please. Our next question comes from the line of Paul Choi with Goldman Sachs. Your line is open. Good morning, good afternoon, folks. Hi Robert.
Speaker Change: Next question please.
Speaker Change: Our next question comes from the line of Paul Choi with Goldman Sachs. Your line is open.
Paul Choi: Good morning, good afternoon.
Paul Choi: Hi, Robert Good afternoon team.
Paul Choi: My question is.
Paul Choi: With regard to your recent meeting with the <unk> can you maybe just confirm for US if there were any either preclinical or clinical testing requirements.
Paul Choi: We're specified by the agency and the security there are any.
Paul Choi: How much of this needs to be coordinated with EMEA requirements ahead of a potential MAA filing and our physicians to NDA. Thank you for taking our questions.
Speaker Change: We don't believe there are any other such studies that are required.
Speaker Change: Okay, great hopefully that addresses your question well thank you.
Yes.
Speaker Change: Our.
Speaker Change: Next question comes from the line of Tess Romero with J P. Morgan Your line is open.
Speaker Change: Okay.
Speaker Change: Yes.
Robert I. Blum: Good afternoon, team. My question is, with regard to your recent meeting with the FDA, can you maybe just confirm for us if there were any either preclinical or clinical testing requirements that were specified by the agency, and to the degree there are any, how much of this needs to be coordinated with EMA requirements ahead of a potential MAA filing in addition? Thank you for taking our questions. We don't believe there are any other such studies that are required.
Tess Romero: Hello, Good afternoon.
Tess Romero: Yes.
Tess Romero: So we just wanted to ask is there any data from Sequoia that you're yet to see at this point.
Speaker Change: I'll turn that over to <unk>.
Speaker Change: We've seen the full set of data I wouldn't I wouldn't say every analysis you can imagine has been complete that may go on for years to be honest as we think of new analyses, but all.
Speaker Change: All of the pre specified analyses.
Robert I. Blum: Hopefully, that addresses your question well. Thank you. Our next question comes from the line of Tess Romero with J.P. Morgan. Your line is open. Okay. Hello, good afternoon. I'm Adion from TEST.
Speaker Change: For for example, the clinical study reported then completed and reviewed in detail.
Speaker Change: Got it thank you.
Speaker Change: Youll also see this laid out in full glory in the second quarter, we expect multiple presentations multiple publications.
Speaker Change: Next question please.
Tess Romero: So, we just wanted to ask, is there any data from Sequoia that you are yet to see at this? I'll turn that over to Fadi. You know, we've seen the full set of data. I wouldn't say every analysis you could imagine has been complete. That may go on for years, to be honest, as we think of new analyses. But all the pre-specified analyses for, for example, the clinical study report have been completed and reviewed in detail. Thank you. You'll also see this laid out in full glory in the second quarter.
Speaker Change: Our next question comes from the line of Yasmin Rahimi with Piper Sandler Your line is open.
Yasmin Rahimi: Good afternoon, and thank you for all the thoughtful remarks.
Yasmin Rahimi: It's been now two months since the top line data.
Yasmin Rahimi: Have you had a chance to complete all your.
Yasmin Rahimi: <unk> preparation by hiring the consultants are reviewing the data on integrated basis coming up with a package and kind of laying out sort of the proposal like could you maybe give us a color on which of these activities have been completed what is left to do.
Patty Malik: We expect multiple presentations and multiple publications. Next question, please. Our next question comes from the line of Yasmeen Rahimi with Piper Sandler. Your line is open. Good afternoon, team. And thank you for all the thoughtful remarks. It's been now two months since the top line data. Have you had a chance to complete all your... preparation by hiring the consultants, reviewing the data on an integrated basis, coming up with a package and kind of laying out the proposal? Could you maybe give us a color on which of these activities have been completed, what is left to do, how much of the proposal, I guess, is in... And thank you again for allowing me to ask my question I mean, you know, I'll take that one.
Yasmin Rahimi: Of the proposal I guess.
Yasmin Rahimi: Finishing that.
Speaker Change: So again for allowing me to ask my question.
Speaker Change: Thank you guys.
Speaker Change: I'll take that one.
Speaker Change: We are in the process of doing just what you are asking I don't think thats the kind of thing that we're going to choose to elaborate on publicly that's obviously something that it performs.
Speaker Change: Competitive positioning.
Speaker Change: Do believe to elaborate on Carter's question that we're going to be potentially able to consider how we may be differentiated and advantaged in ways that could be enabling <unk> to be.
Yasmeen Rahimi: And we are in the process of doing just what you are asking. But I don't think that's the kind of thing that we're going to choose to elaborate on publicly. That's obviously something that informs competitive positioning.
Speaker Change: Accessed by a greater number of physicians.
Speaker Change: For greater number of patients.
Speaker Change: And as it relates to how that Rems may.
Speaker Change: It may be.
<unk> situated what would be the elements of it that's something that I think will be the subject of negotiation at the end of the day during the review period.
Robert I. Blum: I do believe, to elaborate on Carter's question, that we're going to be potentially able to consider how we may be differentiated and advantaged in ways that could be enabling Avafi-Campden to be accessed by a greater number of physicians for a greater number of patients. And as it relates to how that REMS may be positioned, situated, and what its elements would be, that's something that I think will be the subject of negotiation at the end of the day during the review period of an NDA after it's on file. So that's not something I expect we'll be providing play-by-play visibility for until such time as we believe that there's certainty around what that looks like. I got it.
Speaker Change: Of of an NDA after it's on file.
Speaker Change: So that's.
Speaker Change: That's not something I expect we will be providing play by play visibility to until such time as we believe that there is certainty around what that looks like.
Speaker Change: Got it thank you so much Robert.
Speaker Change: Keep up the amazing work.
Speaker Change: Thank you so much.
Speaker Change: Okay.
Speaker Change: Our next question comes from the line of Jason Butler with JMP Securities. Your line is open.
Jason Butler: Hey, Jason.
Jason Butler: Hi, Robin this is Jose for Jason Thanks for taking our question you mentioned that the data from forest HCM demonstrate favorable favorable structural in your modeling.
Yasmeen Rahimi: Thank you so much, Rob, for the amazing work. I'll be back. Thank you so much.
Jose: Could you just speak to the potential long term functional benefits of social digital winning HCM patients and as a quick follow up on <unk>.
Jason Butler: Thank you. Our next question comes from the line of Jason Butler with J&P Securities. Your line is open. Hey Jason, this is Joseph.
Jose: Your discussions with payers do you have any insights into the potential west pricing versus the standard of care. Thank you.
Joe Pantginis: Hi Rob and Tim, this is Joseph Ruggies with a question. You mentioned that the data from Forrest HCM demonstrate favorable structural remodeling. Can you speak to the potential long-term functional benefits of structural remodeling?
Jose: So I'll ask Saudi or Stuart to address the first question.
Jose: Yes.
Saudi: This is really a really key question that we are anxious to investigate.
Saudi: Certainly in the long term because what we hypothesize is that.
Stuart Kupfer: Transcripts provided by Transcription Outsourcing, LLC. Do your discussions with payers give any insights into potential risk pricing versus the standard of care? So I'll ask Fadi or Stuart to address the first question. This is really a key question that we are anxious to investigate, certainly in the long term, because what we hypothesize is that cardiac myocin inhibition will result in a long-term, very favorable remodeling and, hopefully, normalization of cardiac structure and function.
Saudi: Correct margin. In addition will result in long term very favorable remodeling and hopefully normalization.
Saudi: Cardiac structure and function and we anticipate that that will mediate.
Saudi: Confer.
Saudi: Lifelong benefit for patients in terms of symptomatic and functional improvement.
Saudi: We could also hypothesized hypothesize reduced risk of <unk>.
Saudi: Cardiovascular events so.
Speaker Change: I might just add that the data we reported in January it really just the first taste of the.
Stuart Kupfer: And we anticipate that that will mediate, confer a lifelong benefit for patients in terms of symptomatic and functional improvement, and we could also hypothesize a reduced risk of cardiovascular events. I might just add that the data we reported in January are really just the first taste of the CMR cohort, which I think is the largest cohort of patients that will undergo serial CMRs at one, you know, yearly or every other yearly intervals. And so, you know, the number and length of time those data will mature as the years go on and give us a very good sense of how the cardiac structure changes over time.
Speaker Change: <unk> cohort, which I think is the largest cohort of patients.
Speaker Change: We will undergo serial <unk> that one.
Speaker Change: Yearly or every other year the intervals.
And so there are a number that the length of time those data will mature.
Speaker Change: As the years go on and I think gives us a very good sense of how.
Speaker Change: The cardiac structure changes over time.
Speaker Change: From a pricing point of view.
Speaker Change: We will be sharing Sequoia ATM results with Payors.
Speaker Change: Once the Chipotle ATM results are published.
Speaker Change: Sure.
Speaker Change: The there is one product on the market today, obviously that has a list price will be in the range of that list price where there were.
Speaker Change: At slightly above slightly below we haven't finalized where we'll be in pricing.
Jason Zemanski: From a pricing point of view, we will be sharing the Sequoia HCM results with payers once the Sequoia HCM results are published. There is one product on the market today, obviously, that has a list price and will be in the range of that list price, whether we're at, slightly above, slightly below. We haven't finalized where we'll be in pricing, but we'll finalize that as we get closer to launch next year. And those are not the kinds of things that one talks about at this point in time.
Speaker Change: We'll finalize that as we get closer to launch next year.
Speaker Change: And those are not the kinds of things that one talks about at this point in time.
Speaker Change: Thanks.
Speaker Change: Thanks, So much next question please.
Speaker Change: Our next question comes from the line of Joe Pat Guinness with H C. Wainwright. Your line is open.
Speaker Change: Hey, Joe Hey, everybody. Good afternoon. Thanks for taking the question. So I think my question is more suited for our most suited for Andrew.
I know you guys from an internal standpoint don't want to talk about the competitive profile of what youre thinking, but curious from your marketing research and talking to physicians and pulling et cetera. The profile data from Sequoia has it led to any data that you can discuss regarding physicians willingness to.
Andrew Kalos: Thanks. Thanks so much. Next question, please. Our next question comes from the line of Joe Pantginis with HC Wainwright. Your line is open. Hey Joe.
Joe Pantginis: Hey everybody. Good afternoon. Thanks for taking the question. So I think my question is most suitable. I know you guys from an internal... The Bulletproof Executive 2013, etc. Profile data... The Bulletproof Executive 2013, or,... We haven't focused on that.
Speaker Change: Switch from <unk> early on or needing more real world data in order to get educated about the.
I have a camden.
Speaker Change: We havent focused on that we're completely focused on making sure that patients who were on beta blockers calcium channel blockers not on standard of care not on disease modifying therapy are activated towards the CMI educate those physicians on.
Andrew Kalos: We're completely focused on making sure that patients who are on beta blockers, calcium channel blockers, not on standard of care, not on disease-modifying therapy, are activated towards a CMI, and educate those physicians on, hopefully, the label and REMS program associated with affecanthin to suit it for approval, and we're going to focus on activating and getting patients on therapy. We're not going to focus on patients who are already That's really up to the physician-patient dialogue if they choose to go that route. For a next-in-class opportunity like we believe Affy Campton represents, it's incumbent upon us to think about how this might lead to an expansion of the category for the benefit of more patients at the end of the day, and that's where our focus is.
Speaker Change: Filippo label, and Rems program associated with Abbvie, Camden or should it be approved and we're going to focus on.
Speaker Change: Activating and getting patients on therapy, we're not going to focus on.
Speaker Change: Patients who are already on an existing therapy, that's really up to a physician patient dialogue if they choose to go that route.
Speaker Change: Very fair underscore.
Speaker Change: For our next in class opportunity like we believe Effie Camden represents it's incumbent upon us to think about how might this.
Speaker Change: Lead to an expansion of the category for the benefit of more patients.
Speaker Change: Under the day, and Thats, where our focus is.
Speaker Change: Do think that theres over well over 100000 patients who will be eligible our best guesses.
Speaker Change: The vast majority of those will still be.
Andrew Kalos: We do think that there are over, you know, well over 100,000 patients who will be eligible. And our best guess is that the vast majority of those will still be available for HCMI treatment when we go to market. Our next question comes from the line of Jeffrey Hung with Morgan Stanley. Your line is open. Good afternoon. Hi, good afternoon. This is Catherine on for Jeff.
Speaker Change: Available for CMI treatment, when we come to market.
Speaker Change: Fantastic. Thank you.
Speaker Change: Our next question comes from the line of.
Speaker Change: Jeffrey hung with Morgan Stanley Your line is open.
Jeff Hung: Hi, good afternoon.
Jeff Hung: Hi, Good afternoon. This is Katherine on for Jeff. Thank you. So much for taking a question. We just had a quick one on just curious in your conversations or interactions with providers.
Jeff Hung: Thank you so much for taking our question. We just had a quick one. I'm just curious, from your conversations or interactions with providers, have they expressed any enthusiasm about the potential for a less restrictive friend program, or have they indicated whether they feel the data might be supportive of that? Well, we've certainly spoken to providers, and they're optimistic, given the safety profile that we've shared with them in the Sequoia data, but they really, you know, obviously don't contribute to the FDA's evaluation of that So I would just stay tuned. So, thank you. Thank you. Our next question comes from a line from Rona Ruiz with Lee Ring Partners. Your line is open. Good afternoon, everyone. Thanks. I was curious if you could discuss the European market opportunity for AFI Campton a little bit and if there are any similarities or differences that we should think about versus the U.S., and a possible timeline for ramping up more of a commercial presence in Europe.
Jeff Hung: Expressed any enthusiasm about the potential for a less restrictive rems program or have they indicated whether they feel the data might be supportive of that.
Speaker Change: Well, we certainly spoken to providers and they are optimistic given the safety profile that.
Speaker Change: We've shared with them and this is quite a data, but they really obviously don't contribute to the fda's evaluation of that question.
Speaker Change: We will.
Speaker Change: We'll be proposing something based on the findings that we have in the accumulated data that we have.
Speaker Change: So I would just stay tuned.
Speaker Change: Understood. Thank you so much.
Speaker Change: Thank you.
Speaker Change: Our next question comes from the line of Roanna Ruiz Lee with Leerink Partners. Your line is open.
Speaker Change: Good afternoon, everyone.
Speaker Change: Thanks, So I was curious if you could discuss the European market opportunity for Etsy, Camden, a little bit and if there are any similarities or differences that we should think about versus the U S and possible timeline for ramping up more of a commercial presence in Europe.
Speaker Change: Our European point of view.
Speaker Change: You heard the filing dates the prevalence in terms of the number of patients.
Speaker Change: Slightly higher just because of the overall population a slightly higher price point.
Speaker Change: Lower than the U S probably in the range of 15% to 25% depending on the country.
Speaker Change: From an opportunity point of view once approval has occurred we will launch in Germany that allow for launch at approval other markets need to submit.
Andrew Kalos: From a European point of view, you heard the filing dates. The prevalence in terms of the number of patients is slightly higher just because the overall population is slightly higher. The price point is lower than the U.S., probably in the range of 15 to 25 percent, depending on the country.
Speaker Change: For reimbursement and pricing negotiation, so in general Europe will probably be about a year behind in launch.
Speaker Change: Terms of market opportunity outside of Germany.
Speaker Change: Got it thanks.
Speaker Change: Thank you.
Speaker Change: Our next question comes from the line of Sweet crude that Debra <unk> with <unk> Securities. Your line is open.
Rona Ruiz: From an opportunity point of view, once approval has occurred, we will launch in Germany. That allows for launch at approval, while other markets need to submit for reimbursement and pricing negotiation. So, in general, Europe will probably be about a year behind in launch, in terms of market opportunity outside of Germany.
Sweet crude: Good afternoon and below good afternoon. This is <unk> on for Clipper is there anything in your data analysis to help predict who would be more susceptible to LDF drops in patients treated with alpha campaign and would that be incorporated in your Rems program.
Andrew Kalos: Thanks. Thanks. Thank you. Our next question comes from the line of Sri Kritha Devarakonda with Truist Securities. Your line is open. Good afternoon, this is Bilal on behalf of CRIPA.
Sweet crude: And are you seeing stabilization of Lvs overtime.
Sweet crude: Well the answer to the second part of your question is yes, and we've looked at those data and we've actually presented those data you'll see some of that in forest where.
Sweet crude: You will see over time for up to a year.
Sri Kritha Devarakonda: Is there anything in your data analysis to help predict who would be more susceptible to LVF drops in patients treated with africantin? And would that be incorporated into your REMS program? And are you seeing stabilization of LVF over time? Well, the answer to the second part of your question is yes, and we've looked at the data, and we've actually presented those data. You'll see some of that in the forest where, you know, you'll see EF over time for up to a year. It looks very stable.
Sweet crude: It looks very stable.
Sweet crude: <unk>.
Sweet crude: Question is two susceptibility factors.
Sweet crude: I think you can kind of predict.
Sweet crude: Obviously, who might have susceptibility those are patients that have <unk>.
Sweet crude: After you finish titration there, yes are 50%, 55% there may be at more risk because they are closer to the cutoff, it's kind of obvious.
Sweet crude: And I think when you look at any sort of monitoring strategy. That's employed in medicine generally look at patients that are closer to.
Patty Malik: I think the question as to susceptibility factors is, I think you can kind of predict, obviously, who might have susceptibility. Those are patients that have, you know, after you finish titration, their EFs are 50, 55 percent; they're maybe at more risk because they're closer to the cutoff. It's kind of obvious.
Sweet crude: Certain thresholds you look at the more carefully than other patients who are more distant from it so well.
Speaker Change: We will certainly incorporate that into our thinking.
Speaker Change: Thank you very good thank you.
Speaker Change: Our next question comes from the line of.
Speaker Change: Jason <unk> with Bank of America. Your line is open.
Jason Butler: Hello, Jason.
Jason Butler: Good afternoon. This is Cameron those Doug on for Jason Congrats on the quarter and thanks for taking my question how important to your commercial outlook is used in the community setting, especially when looking at the frontline opportunity. What do you think is necessary to make Canadian prescribers feel more comfortable administering assay, especially given it.
Patty Malik: And I think when you look at any sort of monitoring strategy that's employed in medicine, you generally look at patients that are closer to certain thresholds. You look at them more carefully than other patients who are more distant from them. So we'll certainly incorporate that into our thinking. Very good. Thank you. Our next question comes from the line of Jason Zemanski with Bank of America. Your line is open. Hello, Jason.
Speaker Change: It's likely to have a run and then how long do you expect this transition to take.
Speaker Change: Much.
Speaker Change: Youre, describing community I'm, assuming you're assuming cardiology community cardiology.
Jason Butler: Good afternoon, this is Cameron Bozdog on for Jason. Congratulations on the quarter and thanks for taking our question. How important to your commercial outlook is use in the community setting, especially when looking at the frontline opportunity?
Jason Butler: Yes, exactly there is there is a little over 1000 prescribers now of CMI. When you look at claims data there is probably around 10000 cardiologists to drive.
Andrew Kalos: What do you think is necessary to make community prescribers feel more comfortable administering AFI, especially given it's likely to have a REM? And then how long do you expect this transition? When you're describing community, I'm assuming you still mean cardiology, community cardiology. Yes, exactly.
Jason Butler: Diagnosis about 80% of diagnosis.
Jason Butler: Smaller subset of general cardiology.
Jason Butler: Typically specialty product launches or we go from a core outward.
Jason Butler: That outward will occur over time, I do think the rent probably slows it down a bit education continues to occur as patients start to show up in general cardiology already on a CMI.
Andrew Kalos: There are a little over 1,000 prescribers now of CMI. When you look at claims data, there are probably around 10,000 cardiologists who drive diagnosis. You know, about 80% of diagnosis is a smaller subset of general cardiology. Typically, specialty product launchers always go from a core outward; that outward will occur over time.
Jason Butler: That will accelerate especially in the maintenance phase so.
Jason Butler: Hard to say exactly with at that point, we will be but it will certainly occur over time, given the number of patients that need to be treated.
Speaker Change: Thanks, so much for that color.
Speaker Change: Thank you.
Speaker Change: Our next question comes from the line of Serge Belanger with Needham Your line is open.
Andrew Kalos: I do think the REMS probably slows it down a bit as education continues to occur. As patients start to show up in general cardiology already on a CMI, that'll accelerate, especially in the maintenance phase. It's hard to say exactly what that point will be, but it'll certainly occur over time, given the number of patients that need to be treated. Thank you. Our next question comes from the line of Serge Bellinger with Needham. Your line is open. Good afternoon. Hi, good afternoon.
Serge Belanger: Good afternoon.
Serge Belanger: Hi, good afternoon.
Serge Belanger: I guess my question is.
Serge Belanger: Regarding the upcoming Sequoia presentations.
Serge Belanger: Maybe just talk about the.
Serge Belanger: Decision too.
Serge Belanger: Presenting at the heart failure meeting rather than the upcoming ACC meeting next month.
Serge Belanger: And maybe what kind of additional data and analysis, we will see in those results.
Speaker Change: Sure I'll take that one so we knew there was a risk when we top line. The results of Sequoia HCM that we may run a foul of a policy that ACC is regarding acceptance.
Serge Bellinger: I guess my question is regarding the upcoming Sequoia presentation. Maybe just talk about the, ®MD-BO The U.S. Department of Health and Human Services, USA. This document is a transcription and a copyrighted document presented at the heart failure meeting rather than. We will have an upcoming ACC meeting next month, and maybe we'll see what kind of additional data and analyses we'll see in those results. Thanks. Sure, I'll take that one. So we knew there was a risk when we top-lined the results of Sequoia HCM that we may run afoul of a policy that ACC has regarding acceptance of abstracts and what would be otherwise contained in the public domain when they had that abstract to review. We also knew we had a material obligation as shareholders to disclose that which we did choose to disclose.
Speaker Change: The abstracts.
Speaker Change: And what would be otherwise contained in the public domain when they have that abstract to review. We also knew we had a material obligation to shareholders to disclose that which we did choose to disclose so.
Serge Belanger: <unk>.
Serge Belanger: Our abstract for Sequoia was not accepted by ACC, we assumed for the reasons that it was.
Serge Belanger: Ready.
Serge Belanger: Closed in large detail in our press release and therefore, the next meeting.
Serge Belanger: Is the HSA meeting a month later in May.
Serge Belanger: Elizabeth So that's where these data we hope will be presented that's still to be determined.
Serge Belanger: But at the same time, we expect that it will be presented and potentially published concurrently in and around the second quarter. So I hope that answers your question, but we expect.
Serge Belanger: Not just will these primary efficacy and safety results.
Serge Belanger: Presented and published but more so in connection with other secondary analyses and also some post hoc analyses.
Robert I. Blum: So, our abstract for Sequoia was not accepted by ACC. We assume for reasons that it was already disclosed in large detail in a press release, and therefore, the next meeting is the HSA meeting a month later in May in Lisbon. So that's where these data will be presented, but that's still to be determined. But at the same time, we expect that they will be presented and potentially published concurrently in and around the second quarter. So I hope that answers your question, but we expect not just these primary efficacy and safety results to be presented and published but more so in connection with other secondary analyses and also some post hoc analyses.
Serge Belanger: We believe we've got an aggressive communications plan with regard to presentations and publications and you'll see multiple presentations.
Serge Belanger: Presentations and publications during that period.
Serge Belanger: Before it just seemed the additional data.
Speaker Change: Thank you.
Speaker Change: Our next question comes from the line of Charles Duncan with Cantor Fitzgerald. Your line is open.
Charles Duncan: Hey, good afternoon, Hey, Robert and team Congrats on all the progress thanks for taking our questions ACC seems a little shortsighted on that last point I wanted to ask you a couple of.
Robert I. Blum: We believe we've got an aggressive communications plan with regard to presentations and publications, and you'll see multiple presentations and publications during that period. Thanks. Look forward to seeing the additional data. Thank you. Our next question comes from the line of Charles Duncan with Kantor Fitzgerald. Your line is open. Hey, good afternoon. Hey, Robert, and team.
Charles Duncan: Quick questions that you may not be able to answer one is kind of simple and that is do you anticipate an AD com to be called.
Charles Duncan: The second is harder to answer and that is what compels EMR farmer.
Charles Duncan: Pharmacopeia economic modeling supportive say premium pricing.
Charles Duncan: Hey, Curt to standard of care I know you are not really talking about pricing, but just your perspective.
Charles Duncan: Congratulations on all the progress. Thanks for taking our questions. ACC seems a little shortsighted on that last point. Wanted to ask you a couple of quick questions that you may not be able to answer. And one is kind of simple.
Charles Duncan: Okay.
Speaker Change: So as it relates to an outcome, we can't know that.
Speaker Change: What we do know is that.
Speaker Change: There was not an outcome for amount of Camden.
Speaker Change: That doesn't necessarily mean, we wouldn't have an outcome, but we will be prepared if there is an outcome.
Robert I. Blum: And that is, do you anticipate an adcom to be called? And the second is harder to answer. And that is what compels you to do more pharmacoeconomic modeling, supportive of, say, premium pricing or an anchor to a standard of care? I know you're not really talking about pricing, but just your perspective. Thank you. As it relates to an ADCOM, we can't know that. What we do know is that there was not an ADCOM for Mavicampton.
Speaker Change: That's not something that one can know at this juncture and certainly not until.
Speaker Change: Our submission is accepted for filing.
Speaker Change: As it relates to what were compelled by in connection with pricing I'm not going to.
Speaker Change: Speak to the pricing, but I will say, we're quite compelled by what are the H E. R. Data sets that are being prepared and analyzed and how they relate to value associated with kimpton and that alongside of a number of other factors ultimately.
Robert I. Blum: That doesn't necessarily mean we won't have an ADCOM. We'll be prepared if there is an ADCOM, but that's not something that one can know at this juncture, and certainly not until a submission is accepted for filing. As it relates to what we're compelled by in connection with pricing, I'm not going to speak to pricing, but I will say we're quite compelled by the HEOR data sets that are being prepared and analyzed and how they relate to value associated with Aficamptin. And that, alongside of a number of other factors, ultimately contribute to what will be determinants of pricing.
Speaker Change: Two what will be determinants of pricing I think that's the best I can do for you today.
Speaker Change: Perfect.
Speaker Change: So it's an interesting year, thanks for taking the questions.
Speaker Change: Thanks Charles.
Speaker Change: And I'm showing no further questions at this time I would now like to turn the conference back to Robert Blum, President and CEO for closing remarks.
Robert I. Blum: Thank you operator, we covered a lot of ground today.
Robert I. Blum: Both in our prepared comments and also in our Q&A. So I'll be brief I'll simply say that with Sequoia results. We do believe we have turned an important page on to the next chapter for sort of kinetics and will remain vigilant with regard to execution and doing right by shareholders at the same time, we're humble.
Robert I. Blum: I think that's the best I can do for you today. Perfect. It's been an interesting year.
Charles Duncan: Thanks for taking the time to answer the question. Thanks, Charles. And I'm showing no further questions at this time. I would now like to turn the conference back to Robert Blum, President and CEO, for closing remarks. Thank you, Operator. We covered a lot of ground today, both in our prepared comments and also in our Q&A, so I'll be brief. I'll simply say that with the Sequoia results, we do believe we've turned an important page on the next chapter for cytokinetics, and we'll remain vigilant with regard to execution and doing right by shareholders. At the same time, we're humbled by the science, but we're also leaning forward on the opportunity that AFI Campton presents, not just as it relates to OHCM but also as it opens the door, we believe, to other value creation.
Robert I. Blum: By the science, but also we're leaning forward on the opportunity that <unk> presents not just as it relates to <unk>, but also because it opens the door. We believe on other value creation, we will look forward to keeping shareholders abreast of that progress.
Speaker Change: With that operator, we can now conclude the call. Please.
Speaker Change: This concludes today's conference call. Thank you for participating you may now disconnect.
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Charles Duncan: We'll look forward to keeping shareholders abreast of that progress, and with that, Operator, we can now conclude the call, please. This concludes today's conference call. Thank you for participating. You may now disconnect. Thomas Yip, Harry Jenq, Joe Pantginis, Carter Gould, Gil Blum, Robert Blum, Cytokinetics Inc Thomas Yip, Harry Jenq, Joe Pantginis, Carter Gould, Gil Blum, Robert Blum, Cytokinetics Inc Thomas Yip, Harry Jenq, Joe Pantginis, Carter Gould, Gil Blum, Robert Blum, Cytokinetics Inc Thank you for watching. Thomas Yip, Harry Jenq, Joe Pantginis, Carter Gould, Gil Blum, Robert Blum, Cytokinetics Inc Thank you for watching.
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