Cytokinetics at RBC Conference: Strategic Insights on Drug Development

On Wednesday, 21 May 2025, Cytokinetics (NASDAQ:CYTK) participated in the RBC Capital Markets Global Healthcare Conference 2025, providing a comprehensive update on its drug development pipeline. The company shared optimistic developments, particularly around its lead drug candidate aficamtan, while also addressing regulatory hurdles and strategic plans for commercialization.

Key Takeaways

• Cytokinetics is preparing for a potential 2026 launch of aficamtan, contingent on FDA approval.
• The FDA requires a REMS for aficamtan, leading to a major amendment and extension of the review process.
• The company holds a strong financial position with $1.1 billion in cash and investments as of Q1 2025.
• Positive results from the MAPLE HCM study were announced, comparing aficamtan to metoprolol.
• Cytokinetics is advancing other promising drugs, including omecamtiv mecarbil and CK-586.

Regulatory Updates on Aficamtan

• The FDA’s requirement for a REMS has extended the review timeline for aficamtan.
• Cytokinetics anticipates a differentiated REMS profile based on clinical study results.
• The MAPLE HCM study showed positive outcomes, with data presentations expected this year.
• The Acacia study in NHCM has completed enrollment ahead of schedule, with data expected in 2026.
• Regulatory feedback prompted changes to the Acacia study endpoints to align across jurisdictions.

Commercial Strategy for Aficamtan

• A potential launch of aficamtan is targeted for 2026, pending FDA approval.
• A dedicated commercial team is in place, focusing on category penetration beyond centers of excellence.
• Engagement with payers and medical education programs is ongoing.
• The launch strategy will differ from that of mavacamten by BMS, aiming for broader market penetration.

Pipeline Updates

• Omecamtiv Mecarbil is undergoing a Phase 3 study, with significant potential noted by the CEO.
• The AMBER Phase 2 study for CK-586 is enrolling, targeting HFpEF patients with higher ejection fractions.
• Cytokinetics aims to complete at least two cohorts of the CK-586 study this year, with data expected next year.

Financial Position

• Cytokinetics ended Q1 with $1.1 billion in cash and investments.
• The company has potential access to an additional $425 million from Royalty Pharma.
• These funds will support the launch of aficamtan and further pipeline advancements.

Q&A Highlights

• CEO Robert emphasized the adaptability of workflows to REMS and the importance of aficamtan’s pharmaceutics profile.
• He expressed confidence in aficamtan’s potential to reach patients by 2026, pending approval.
• Robert also highlighted the enthusiasm for omecamtiv at cardiology meetings, which is not yet reflected by Wall Street.

Cytokinetics remains optimistic about its pipeline and strategic plans. For more details, please refer to the full transcript below.

Full transcript - RBC Capital Markets Global Healthcare Conference 2025:

Unidentified speaker: We’re really happy to have Cytokinetics with us, here represented by their CEO, Robert, and their chief financial officer, Sung. So thanks for being here.

Robert, CEO, Cytokinetics: Thank you.

Unidentified speaker: I think we’ll jump right into things. You know, I think one of the biggest updates lately, what has been the regulatory discussions around aficamtin, and the potential, major or the major amendment. So I guess, you talk a little bit more about what led to that, what drove the major amendment, and ultimately, the confidence that even with the REMS, you’re still gonna see a differentiated label?

Robert, CEO, Cytokinetics: Sure. So I’ll go back into 2024 a little bit as well to answer your question. So Cytokinetics had multiple meetings, three meetings with FDA once we had the results from SEQUOIA HCM, the pivotal clinical study of aficamtan in patients with O HCM, and these three meetings preceded the NDA submission. At all three of these meetings, we had conversations with FDA officials, both from the review division and also from the division that would consider a potential REMS. And in those meetings, and as they were focused to matters relating to safety, tolerability, risk mitigation, and informed our consideration of submission.

In all three of those meetings, were minuted, we got feedback around how we would approach a potential submission. We considered it reasonable that we deemed it reasonable to submit without a REMS, but knowing that there were considerations around safety and risk mitigation, we included language within proposed label that would address those matters. FDA accepted the submission without a REMS, which should tell you something. But for as is reasonable, FDA determined during its review of the application that a REMS should be required. So we effectively, knowing that that was a risk, took language that was in the proposed label and moved it to a proposed REMS and submitted with that proposed REMS very promptly after receiving that feedback from FDA.

And upon its receipt, FDA considered that and thought it necessary to associate with a major amendment and an extension of the review clock. I wouldn’t conclude from any of that that there was anything in terms of shift of strategy or change of meaningful plans. We’ve been all along the way referring to our expectation, if approved, that aficamtan would be associated with a differentiated risk mitigation profile. We weren’t using the words REMS, but now we know that a REMS is required. We do believe, if approved, aficamtan will be associated with a differentiated REMS.

And I don’t consider it to be so different that I would anticipate that will have meaningful effect to the way it’s positioned in the marketplace. If approved, I think aficamtan would be approved with a dosing regimen, with a DDI profile, with associated convenience and other aspects of positioning that should be enabling of all of the things we’ve been talking about all along, which should be good for category growth and category penetration and, hopefully, as could be for Affy Camden, preferential share of category.

Unidentified speaker: Understood. You know, there’s can be a lot of difference between REMS programs. So can you maybe talk about, you know, whether we’re too narrowly focused on REMS itself and maybe the differences that REMS programs can have and how even if you have a REMS, there can be a a gap between what a REMS for a competitor may look like. And I understand you can’t go into specifics, but sort of what are some of the differences in how REMSes can be structured that we may not be appreciating that can enable epicanthin to have differentiation?

Robert, CEO, Cytokinetics: Thank you for asking that question. It’s a very good question. I do think we’re over indexing on REMS because in the marketplace, I think, ultimately, workflows adapt to REMS, and what should ultimately matter is the pharmaceutics profile, the PKPD profile, the clinical safety, efficacy, tolerability profile of drugs in a category with and without consideration of REMS. What do we know so far? We know that mavacamten, the first in class drug in this category, is doing quite well, adding about fifteen hundred patients per quarter.

It’s due to generate, I believe, over a billion dollars in sales for BMS this year, but for which the use of mavacamten continues to be somewhat concentrated, mostly in centers of excellence, roughly 600 physicians in The United States accounting for about 80% of the prescription, but with a high adherence and compliance, Patients who are on the drug tend to stay on the drug. BMS is getting its price. It doesn’t look like there’s a lot of payer pushback. And I think, ultimately, this category, cardiac myosin inhibitors, are making a meaningful dent in the treatment of patients, but where there’s still about eighty to eighty five percent of eligible patients not being treated with a cardiac myosin inhibitor today. So we see there to be an opportunity where another compound coming into the category can be lifting the tide as would be enabling of growth of cardiac myosin inhibitors for patients, and I think REMS is a component of that.

But to your question, and I’m sorry it took me a while to get here, to your question, there are major differences in REMS programs as exist today. And I think if you look at the way aficamtan is studied, the uptitration regimen, the DDI profile, all the things that are now presented and published for benefit of aficamtan to potential patients, what do we know from Sequoia, what do we know from Forrest, All of these things point to the possibility that if approved, FDA would approve the drug based on its clinical study in ways that would be differentiated in the marketplace. We think that would be to the advantage of physician convenience, patient convenience, a lot of things that we think ultimately speak to market penetration.

Unidentified speaker: Got it. And, you know, as you think about sort of the differentiation of the drug and what physicians want to see, are there things that aren’t necessarily in the label, wouldn’t appear in the label that are properties of ethicamtin that may resonate with physicians and help uptake?

Robert, CEO, Cytokinetics: Yeah. The ongoing clinical evidence. Our team is doing a superb job of continuing to study aficamtan in ways that should further the knowledge base for how a cardiac myosin inhibitor could ultimately be used. Case in point, we recently announced positive results from MAPLE HCM, a head to head study of aficamtin versus first line metoprolol in patients with OHCM. Believe it or not, metoprolol and other beta blockers and calcium blockers are used in the treatment of OHCM as first line therapy, but for which there’s scant clinical evidence to support their use nor is there broad FDA review or approval, but yet the guidelines call for it because these are drugs that have been out there for a long time, and they’re cheap, but for which we’ve done now the first randomized head to head study of afecamtin versus metoprolol, and we announced recently that that study was positive.

I’m looking forward to our presenting and publishing those data as I think we’ll continue to further the evidence to support aficamtan in ways that this goes beyond the REMS. This goes beyond even what ultimately may be knowable about aficamtan today. And I think investing in ongoing studies like MAPLE, another study called Acacia in NHCM. We recently announced that we concluded enrollment ahead of schedule. You’ll see those data in 2026.

These are the kinds of things that I think further the evidence to support aficamtan if positive.

Unidentified speaker: Got it. So on Maple, you know, you’re to present that data. How soon do you think Maple can be part of guidelines? Is this something that you can use to interact with payers now? And then, you know, maybe make it a two part question.

Is MAPLE and the data from MAPLE something that you’re going to show to the FDA now as part of the review process or for the European regulators?

Robert, CEO, Cytokinetics: There’s no expectation that we’ll be submitting MAPLE as part of the ongoing review. It would be potentially presented and published this year. That’s our hope. And as to your question about guidelines, that could mean enablement of expansion of guidelines to consider MAPLE in 2026. So it will be in the public domain.

It should be informing guidelines, but it won’t be the subject of our interactions with regulatory authorities nor do we expect that we’d be promoting in accordance with that until it’s ultimately incorporated into label.

Unidentified speaker: Got it. Maybe we can talk a little bit about the commercial strategy for aficamtan. I guess, how are you thinking about how to target physicians, where you are with payer outreach? And ultimately, do you think you’re gonna be targeting mostly new patients? Are you gonna be targeting switch patients?

Robert, CEO, Cytokinetics: So if approved, aficamtan would be available and hopefully in the hands of patients in 2026 because we have a PDUFA date end of twenty twenty five. But make no mistake about it, we’ve been preparing for this for quite some time. Our head of sales is in the audience here, and he’s been overseeing a group of field managers that have been on payroll for a couple of years now. These are folks that have been thinking about how we go to market. What does that mean for sales, learning, and development?

What does that mean for territories, configurations, incentive compensation? How do we be ultimately optimizing for the best type of commercial launch? And they’ve been working side by side with other colleagues for several years as we do all of the market analytics, all the segmentation, all the omnichannel planning, all the work that goes into a successful launch. We’ve been approaching this not just from the standpoint of a first launch, but what would be for enduring value through multiple launches, label expansion, life cycle management, and aficamtan as it opens the door onto other products like omecamtiv and CK five eighty six. So how we think about a commercial business has been the subject of planning for many, many years at our company, how we incorporate our commercial colleagues alongside of our r and d colleagues into one culture, making it happen.

So I think when Cytokinetics shows up in the marketplace, it’ll be with a tailored bespoke experience for physicians, payers, and patients that enables, we would hope, somewhat of a differentiated commercial story as well. We’ve been interacting with payers for a long time. We’ve got medical affairs colleagues that have been in territory for multiple years engaging with all sorts of opinion leaders around medical education programs, etcetera. So our plans here have been in the works for quite a while, and I expect that that’ll show up in the way we go to market.

Unidentified speaker: Got it. And I think investors are probably gonna use the Camxious launch as sort of a benchmark for how afacamten’s launch may go. So, you know, I’m curious your thoughts. Do you expect the launch of avacamten to potentially be more rapid because the market’s built out? Is there gonna be pressure because there’s some competitive dynamics?

Then, I guess, you know, as you think about how Bristol launched that drug, I mean, are there any learnings that you think you’d things you’d do differently, that you wanna take away?

Robert, CEO, Cytokinetics: Yeah. I’m gonna answer that, and I’ll also ask Sung to speak to it from his perspective. But we’ve been good students of not only the, BMS mavacamten launch, but also many of us come from experiences with cardiology and next in class launches that go back decades, and we’re trying to best take learnings from those and apply them here for aficamtan. A second drug to market typically has, for some period of time, a similar trajectory, but then what drives launch velocity? Those are the kinds of things that ultimately are informing our decisions.

How we, as a new commercial entrant, come to market speaks to building something that’s tailored to our pipeline, our strategy. What does that mean for distribution? What does that mean for how we segment and prioritize the physicians we call upon, with what reach and what frequency? These are all things that you can borrow learnings from next in class drugs in cardiology, and I think it’s reasonable to use a benchmark of the mavacamten launch, but things should be different. I’m not gonna speak today to what would be metrics to measure, not yet.

You’ll hear those from us in due course, but I do think that a second drug in the same category typically deploys a different strategy. We’re not going to be focused as much on switches. We may see some, but it’s more important for us to drive category penetration beyond centers of excellence to the community and ultimately as we’ll be benefiting the category where there are currently ample opportunities for patients not being presently treated or treated well.

Sung, Chief Financial Officer, Cytokinetics: Yeah, Leon, I’ll just add to that. We have a PDUFA date, obviously, ahead of us, and we’d like to see ultimately the label that we arrive at. But based on our clinical data, we certainly have an aspiration to want to do better. But as Robert said, in the cardiovascular space, in the early years, certainly, you could

Robert, CEO, Cytokinetics: see a linear pattern. Yeah. There’s, I think, a lot of history around cardiology launches where they typically take longer to get payer traction reimbursement. This is going to be a category where, based on what we know so far, you shouldn’t expect a lot of Medicare contracting. This is a roughly fifty-fifty split Medicare, non Medicare, but we anticipate that this will all be handled primarily under Medicare exception.

So time to reimbursement factors into how quickly we can see the asymmetric phase of revenue generation post launch, but there’s other things that we’re doing that also should drive launch velocity, and they speak to patient experience. How can we create a patient hub that ultimately is distinguishing of aficamtan in the marketplace? How can we be enabling of, from the standpoint of positioning and driving where there are pockets of demand to a preferential place? What strategies might we employ that are bespoke to the Cytokinetics experience. These are things that I’m not going to speak to in detail today, but they’re top of mind for us.

Unidentified speaker: That makes sense. I wanted to ask on Acacia and the nonobstructive market. We saw some data from or we didn’t see a lot of data from Ristal, but there was an update. I guess, how are you thinking about Acacia? You made some changes to the study, to the study endpoints.

Were those internally driven? Were those driven by regulators? Were those driven in any way by what you thought may have come out of the Bristol data? Can you talk about that?

Robert, CEO, Cytokinetics: Yeah. So in the audience, we have, the lead, from our clinical team who’s better suited to do that than I am, but I’ll do my best. So firstly, to answer your last question, nothing that we did was driven by any specific knowledge of the Bristol data But for feedback we received to harmonize across regulatory jurisdictions, we wanted to make certain that the endpoints and statistical plans were cohesive across territories. So we made minor modifications to the way in which the study design will ultimately be implemented but for which the same endpoints are being measured. We’re just making what was previously the first amongst the hierarchy of secondary endpoints a co primary, and we’ve altered the statistical plan to be accommodating of that, and we’ve increased enrollment to increase our statistical power to measure both co primaries.

All of these things are in line with regulatory feedback and have nothing to do with the BMS study design, but for which we do believe it’s to our advantage, Acacia can be positive were we to hit on one or both of the coprimaries. And for now, having increased the enrollment, we think we’re in a good position to test the hypotheses that are being studied in Acacia and as could be enabling of something very meaningful for aficamtan if positive in Acacia. BMS reported that their study, OHDSI, was negative in NHCM, and we don’t know why. We have speculations like everybody else, but we can’t know until we see the data. We don’t think it reads on mechanism.

We don’t think it reads on the population, but for which we’ll ultimately know the answer based on the study we have designed and are conducting, which is going quite well. So if acacia is positive, it will be imperative that we move swiftly to get those data submitted to regulatory authorities as promptly as possible because as we’re seeing now in claims data, the population of patients with NHCM may represent now about fifty percent of the total. So this is a larger and more rapidly growing segment of the population than we would have expected even a couple of years ago, and that could be quite enabling of the commercial profile of aficamtan to be even further differentiated upon a potential approval. But again, subject to Acacia being positive, and we’re excited about that possibility.

Unidentified speaker: Got it. Got few minutes left, and I wanted to touch on some of the other drugs that Cytokinetics is is working on, maybe starting with omecamtiv. You guys are running a second phase three there, and assuming that it ends up reading out positively, I guess I’m curious how you see that drug fitting into the landscape for HFrEF, especially maybe towards the end of the decade as guideline directed therapy continues to evolve? So are you seeing that drug? Is it going to be used primarily in the population that it’s studied?

Do you think that there’s maybe opportunity to expand that, that the study is just sort of run-in that population because that’s where it’s most likely to show a clinical benefit, but the drug could actually be used more broadly?

Robert, CEO, Cytokinetics: Yeah. I think this is one of the great opportunities for our science. We’ve studied omecamtiv six ways from Sunday, and we’ve done this in over 30 clinical trials. It’s apparent that omecamtiv mecarbil has a very profound pharmacodynamic effect that’s translated to clinical benefit in phase two and phase three studies. So here, omecamtiv already the subject of a positive 8,000 patient phase three study where that effect size was doubled in a patient population with more severe disease.

So the ongoing confirmatory phase three study is a roughly 2,000 patient study meant to replicate a finding from a prior 8,000 patient study. And if positive in the ongoing COMET study, I do think it’ll be potentially quite meaningful for patients with more severely advanced heart failure with ejection fractions below 30. These are not patients that are seemingly benefiting from existing standard of care. The event rate of death and hospitalization for these patients remains unacceptably high, and I do think that if positive and common, the heart failure specialists tell us that this will be a very meaningful driver of guideline adoption of this new innovation. I think that when we go to cardiology meetings, like was the case just a few days ago at the European Heart Failure Meetings, there’s a very high level enthusiasm for omecamtiv that, frankly, is not matched right now with Wall Street interest, and I think that could be a mismatch and a potential arbitrage for investors.

I think there’s a significant opportunity with omecamtiv that’s being largely underappreciated.

Unidentified speaker: Yeah. I also wanna touch on c k five eight six and the AMBER study.

Robert, CEO, Cytokinetics: And we should also talk about our financials when we can, so I want to make sure that’s appreciated We’ll

Unidentified speaker: we’ll we’ll make those the last two questions then. Okay. So, you know, five eighty six for AMBER, can can you talk about how recruitment’s progressing? And then I’m also curious on the patient population. I think we hear questions on why you’ve chosen the patient population with sixty percent as the LVF cutoff.

I guess, can you talk about why you think that’s the right patient population to go after here?

Robert, CEO, Cytokinetics: Yeah. So much like HFrEF, patients with a reduced ejection fraction can be broken into cohorts, and we’re studying those with more severely advanced systolic dysfunction with omecamtiv. With CK five eighty six, an inhibitor of cardiac myosin, we are looking at a subset of HFpEF patients, patients with preserved ejection fraction. And there, those with higher EFs have anatomies that more resemble the NHCM patients that we’re studying with aficamtan, and the clinical evidence will hopefully support movement into phase three. The preclinical data already demonstrates that these patients are not more likely to benefit from existing standard of care, but a cardiac myosin inhibitor could be addressing that which ails them in ways that could benefit them.

So we’re looking at the other extreme side of heart failure with a subset of patients with higher EFs, thicker anatomies, and where, while they don’t have a pressure gradient, we do think that they are different from the other HFpEF patients that maybe are more metabolic. So we are not focused to the more obese metabolic patients. We are focused to those with more hyperactive contractility, and we think they will benefit from an inhibitor. So the Phase II study, AMBER, is enrolling. We hope to have at least two cohorts completed this year, data hopefully next year.

Unidentified speaker: Got it. And you guys have a lot going on at the company, gearing up for a launch, a lot of R and D, so you wanted to mention something on the financial position. Love to hear

Sung, Chief Financial Officer, Cytokinetics: your help. So we finished quarter one, as you know, with 1,100,000,000 in cash and investments. So we’re in a real solid position with regard to the balance sheet. Importantly, we have access to further capital. We could be eligible for up to an additional $425,000,000 and this would be provided to us by our partner, Royalty Pharma.

So this puts us in a great position to not only launch aficamtan, but also to advance our pipeline and importantly, the label expansion opportunities for aficamtan. Appreciate it. I think that’s all the

Unidentified speaker: time we had. Thank you so much for joining us.

Robert, CEO, Cytokinetics: Thanks very much. Appreciate it.

Sung, Chief Financial Officer, Cytokinetics: Thank you.

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