Cytokinetics at Leerink’s Global Healthcare Conference: Strategic Pipeline Insights

On Monday, 10 March 2025, Cytokinetics (NASDAQ: CYTK) presented at Leerink’s Global Healthcare Conference 2025. The company’s leadership discussed its strategic initiatives, focusing on pipeline advancements, regulatory updates, and commercial readiness. While the outlook for its cardiac myosin inhibitor, aficamten, appears promising, challenges from competitors like Bristol Myers Squibb remain. Cytokinetics highlighted its robust financial position and emphasized its commitment to expanding market reach.

Key Takeaways

• Cytokinetics is advancing aficamten through FDA review, targeting a differentiated label.
• The company maintains a strong cash position of $1.2 billion, prioritizing non-equity dilutive capital.
• Plans include launching aficamten independently in North America and Europe, with partnerships in Japan and China.
• Ongoing trials aim to expand aficamten’s market potential and influence treatment guidelines.
• Cytokinetics is confident in its ability to offer a competitive alternative to Bristol Myers Squibb’s CAMZYOS.

Financial Results

• Cytokinetics reported cash and cash equivalents of approximately $1.2 billion.
• The company has enhanced its balance sheet through deals with Royalty Pharma, avoiding equity dilution.
• It positions itself as a leader among peers in balance sheet strength.

Operational Updates

• Aficamten is on track for a late cycle FDA meeting in June, with no AdCom expected.
• The company launched a disease awareness campaign targeting healthcare professionals and patients.
• Commercial readiness activities are underway, focusing on medical affairs and payer engagement.

Future Outlook

• Cytokinetics aims to have at least two approved products by 2030, expanding its pipeline in muscle biology.
• The company targets over 100,000 patients by 2030, with plans for global launches.
• It seeks to broaden the cardiac myosin inhibitor market beyond specialized centers.

Q&A Highlights

• Aficamten aims to provide more confidence, ease of use, and flexibility for patients.
• The MAPLE trial compares aficamten to beta blockers, while the ACACIA trial focuses on non-obstructive HCM.
• Cytokinetics believes its infrastructure supports market expansion efforts.

Readers are encouraged to refer to the full transcript for more in-depth insights.

Full transcript - Leerink’s Global Healthcare Conference 2025:

Operator: Okay. Okay.

Ruana Ruiz, Senior Biotech Analyst, Lyric: Welcome, everyone, to the Lyric Global Healthcare Conference. I’m Ruana Ruiz, one of the senior biotech analysts here at Lyric. And just want to welcome everybody, and I’m really excited to introduce Cytokinetics, a member of the management team. So with me here today, I have Robert Blum, CEO, and Andrew Callos, EVP and CCO. So thanks for joining us.

Operator: Thank you.

Ruana Ruiz, Senior Biotech Analyst, Lyric: And I think I’ll kick it to Robert. You have a couple slides you wanted to go through for intro?

Operator: Yes. Just to create a level set, we put together a few slides that hopefully will provide a nice backdrop for the conversation we’re going to be having. I’ll be talking about the company and as we talk about our company, it’s patients like Eric, who inspire us to do the good science that we do for the benefit of ultimately potential new medicines. I’ll be making some forward looking statements. I’ll refer you to our SEC filings with regard to caveats to those statements.

And of course, we’re not gonna undertake any obligation to update those statements, but encourage you to take a look at those things. So Cytokinetics is a company that has been pursuing a mission since I started the company, now over 25 ago. We’re focused to new medicines that improve health span of people with devastating cardiovascular and neuromuscular diseases by sticking to our roots, our expertise in muscle biology, and we’re addressing where we believe that expertise translates to a novel pharmacology of muscle function. Here’s our pipeline. It’s a pipeline of potential new medicines, all of which have been discovered by our scientists.

This is organic to how we think about science and biology and pharmacology. And as you can see, cardiac myosin, which is a mechanochemical enzyme that drives the mechanics of cardiac muscle contractility, forms the cornerstone of our pipeline. We’ve got multiple, programs in later stages of development, all of which read on cardiac myosin, foremost amongst which is an inhibitor of cardiac myosin called affikamten, which is pending FDA regulatory review for the potential treatment of obstructive HCM. Afikamten is also the subject of other clinical trials, including one that should read out in q two called MAPLE also in OHCM and also other studies ongoing in NHM and pediatric OHCM. But cardiac myosin also forms the basis of our interest in activating cardiac muscle for severe forms of advanced heart failure and inhibiting it in the form of HFpEF, heart failure with preserved ejection fraction.

So here you’ve got three programs all directed to one molecular target and that forms the cornerstone of not only our pipeline, but also our business strategy, a franchise emerging in specialty cardiology as we’ll talk more about. But it’s not the whole story. We also have other programs underneath the hood. I doubt we’ll have time to talk about them, but they relate to other interests in cardiovascular and neuromuscular medicine. Here’s our vision 02/1930.

It speaks not only to where we’ve been, but where we’re going. We expect by 02/1930 to have brought not one, but at least two, potentially more products into, the armamentarium of patient care. And our goal is to be advancing approved products, at the same time, and this perhaps distinguishes us from many companies today, At the same time, we’re significantly augmenting pipeline in muscle biology. And how we think about go to market matters, you’ll hear more from Andrew, our chief commercial officer in a moment, but we expect to be launching our own medicines in North America and Europe at the same time through partnerships in Japan and China, and reaching over a hundred thousand patients by the time 2,030 rolls around, and all while still leading in the way of culture, with focus to equitable access, as you’ll hear more about our company over time. Ours is a company that thinks about not only science and pipeline, but also how you get there and relying principally on non equity dilutive capital to do it.

We’ve set a high watermark, we would argue, amongst peer group companies in terms of how we’ve augmented balance sheet. And you can see here that Sato Kinetics recently reported cash and cash equivalents of approximately $1,200,000,000 on our balance sheet with still ample access to additional capital as would not come at the expense of equity shareholders through deals we’ve done including, I believe, more deals with Royalty Pharma than any other company and on terms that we think are very much in the interest of low cost of capital and high capital efficiency. So we think about being good stewards of shareholder capital and, we can speak more to that if you have questions. Here’s the news flow. This is a company because of pipeline that has quite ample news flow this year and the next couple of years as we build out on our business.

And you can see highlighted here in green things that you can expect of us this year, specifically as relates to a potential regulatory approval here in The United States for afacamten in OHCM, additional clinical trial data in Maple this year, completing enrollment, of acacia, as would be occurring later next year and things like that that we can speak to as you may have interest. Last year, at the end of the year, we announced some key deals. We announced a deal in Japan for Afikampton with Bayer. We announced a deal in China with Sanofi as they stepped into the shoes of a prior partner, a company called Xijin, or Korzell as it was renamed. And I think this is enabling of us to think about the global presence of Afikamten in over eighty to ninety percent of eligible patients with OHCM.

Cytokinetics is thinking boldly and broadly as it relates to ensuring access to affecamten promptly upon approvals. And as we prepare to go to market, we’ve been in preparation with commercial readiness activities, not only medical affairs, but also as we engage with payers. And we recently launched a unbranded disease awareness campaign initially to healthcare professionals, more recently to patients. And I encourage you to take a look at these websites and knowing that we’re investing in market development through these initiatives. I think it’s really important to know Cytokinetics to understand how we think about go to market and Andrew will speak to this in great more detail.

But we have been a good student of learnings of other companies including first time biotech launch companies. And we believe we’ve already implemented strategies that have demonstrated to have been successful for biotech companies that are their first time commercial launch companies. This is not our first rodeo, me and others, we’ve done this already several times before and we’ve taken learnings from those experiences to be enabling of a specialty cardiology approach to building our franchise. We’re thinking about multiple programs. We’re thinking about how we approach market access differently.

How we’re thinking about omnichannel precision marketing differently. How we’re thinking about the patient experience and ultimately, nurse navigators and other ways that we engage with HCPs in their offices, specialty distribution channels and other fit for purpose ways of enabling a very focused disciplined way of addressing a highly concentrated customer segment as we would believe could translate into initial launch velocity and enduring commercial sales. So, happy to get into more detail about that. And as I mentioned, it’s not just about Afikampton in its first studies, but other ongoing studies we’re investing quite amply in additional clinical trials, some of which you might even say sound a bit like phase four studies that we’ve accelerated into earlier time frames so that we can hit the market at a stride and demonstrate already, we hope, category growth and hopefully preferential share. And Maple should be contributing to that as well as could Acacia in non obstructive HCM in 02/2020, ’5 and completing enrollment, later this year as depicted on this slide.

So here are the milestones for this year. I won’t go into each of these individually. I think I’ve already touched on most of them. But to know Cytokinetics again is to recognize how we expect to go to market in The United States with approval hopefully anticipated in Europe, next year, in China later this year, and at the same time with other catalysts that could be contributing to augmenting of the story with maple and acacia to follow. And that’s at the same time we’re advancing omecamtiv in phase three, c k five eight six in phase two, CK089 in phase one.

So Cytokinetics is a story of not only, we hope, impact in terms of commercialization this year, but also pipeline growth and enduring value for shareholders as we build out our franchise verticals. And with that, I’ll turn it back to you for Q and A.

Ruana Ruiz, Senior Biotech Analyst, Lyric: Sure. Sounds great. That was a really good overview. I guess to kick off, I did notice you put out a bit of a release earlier this morning with some regulatory updates. So just want to level set here for the audience.

Could you just talk about some of those updates and where things stand for Afikampton in particular?

Operator: Yes. So knowing we were going to have lots of one on one meetings this week and also three investor conferences this week, Our lawyers counseled us that it would be good to level set from a Reg FD standpoint to ensure that everybody has the same access to the same information. So we announced this morning that we recently did convene a meeting with FDA, a mid cycle meeting. And during that meeting, a couple things. One is FDA has made clear that it does not expect, an AdCom, for Afikampton, that we are on timeline for a late cycle meeting in June.

And, coming out of the meeting, same as going into the meeting, Sato Kinetics continues to believe that if afacamten is approved, that we should expect a differentiated label and risk mitigation profile. So we are pleased and, you know, that’s about it in terms of how we’re going to be able to comment on, what’s been occurring with ongoing FDA interactions.

Ruana Ruiz, Senior Biotech Analyst, Lyric: Yep. Got it. Okay. Great. And so just thinking about afikamten, since it’s a big driver of a lot of the investor conversations I’ve had over time.

So in terms of SEQUOIA and the Phase three trial and putting out more granular data updates, more interesting updates later this year. I mean, what’s your plan in terms of publications, medical meeting presence, and how does that tie in well to commercial education, etcetera, ahead of a potential AFI launch?

Operator: So this is one of those things that I think, is incredibly important at Cytokinetics and frankly is a competitive advantage. Cytokinetics is a company that’s both been a pioneer and a leader in this space, and it’s evidenced not just in the clinical trials that we’re doing, but the scientific evidence that’s been published. Last year, we published over a dozen manuscripts in support of Afikampton’s, evidence to support go to market. And, we do believe that, this is somewhat unusual and provides us support as our medical affairs colleagues and our eventual commercial colleagues will be calling on customers. And you should expect more of that from us, not just with regard to additional analyses from Sequoia, but from Forest.

We now have hundreds of patients in Forest HCM, the open label extension that are out beyond one in two years. And those data continue to demonstrate, we believe, the same properties that we engineered into Afikampton as could be potentially differentiating. Moreover, Maple reading out in Q2, you’ll see those data presented and published hopefully soon. I think it’s a hallmark of how we do business that Sato Kinetics is continuing to advance the science.

Ruana Ruiz, Senior Biotech Analyst, Lyric: Yep. Got it. And drilling down a bit more, there has been a lot of discussion around Kim Zios’ REMS from Bristol and how that might be have been a limiting factor in the uptake of that program. Can you just talk about what are your view of the REMS requirements for that product? And, you know, could that be generalized to the CMI class?

Or just remind us of some of the differentiation points for AFI where things could be different.

Operator: Yeah. So I’m gonna ask Andrew to speak to this primarily, but what I’ll say is this. We think that, BMS is doing a great job with regard to cardiac myosin inhibition is a very effective therapeutic strategy. There are roughly five fifty to 600 physicians accounting for over 80% of the CAMZYOS prescription volume, and those numbers are impressive, especially as it relates to repeat prescriptions and patient adherence. So these are things that are demonstrating that for their first in class drug, they’re doing all the kinds of things you’d expect of a new cardiovascular medicine.

It’s incumbent upon Sato Kinetics upon a potential approval and launch to expand the category. And that’s where our strategy has always been. And with that, I’m gonna turn it over to Andrew to talk about where the REMS fits into this and how we might perceive to be differentiated. Sure.

Andrew Callos, EVP and CCO, Cytokinetics: So I mean, as Robert alluded to, there’s a little over five fifty cardiologists who are really driving mavacam in over 80% of their volume. And to get out to beyond the specialized centers into general cardiology, our research tells us that the REMS is a major factor that that’s a slow process. There’s elements of REMS that are echoes that there are a number of echoes required to get up to a maintenance dose. There’s a percentage of patients who actually have to go down in dose from five to two point five. There’s a drug to drug interaction monitoring required.

There’s a pretty narrow window between when an echo occurs, when a patient can get the next prescription. So collectively, it does add some level of administrative challenge to offices to get a patient on steady state dose. That said, I think what we hear is that patients and cardiologists in general, say that mavil works well, that you do it does take some time as I alluded to. If you could lower that administrative burden, if you can get a patient from an efficacy point of view faster to maintenance dose, if there’s less requirements relative to a risk management program, that should unlock more of the market. The market is pretty linear right now.

I think there’s about 1,400 patients per quarter being added, maybe about 15,000 our estimate number of total patients on MAVA. The market opportunity is probably over 100,000. They are New York Heart Class IIIII patients. So our expectation is about eighty percent or more of the market for obstructive HCM will be available. And if we go to the cardiologist, we’ll start in those same $550,000,000 likely.

But if we can expand to another several hundred physicians, then you’ll start to see maybe the market grow in terms from this linear growth stage more to a high growth stage over time.

Ruana Ruiz, Senior Biotech Analyst, Lyric: Yes. Got it. And in the context of the CMI class growing with potential approval of affine, I know you cannot speak directly to FDA input yet, but I was curious going into continuing this review process for AFE, what are your goals in terms of presenting information to the FDA? Or what do you hope that they, garner from all the package that you’ve provided them so far?

Operator: So, you know, we announced with our earnings call that, we recently submitted the day one twenty update, and you’re seeing in real time how the forest, open label study of Aficamton is a real world experience. And what I’m especially encouraged by is we’ve yet to see any excursions relating to ejection fraction that contribute to a heart failure diagnosis or hospitalization. That to me underscores the fact that we’re doing the right thing by how we approached dose up titration and dose maintenance and that this is, in affecamten if approved. Hopefully, a potential medicine that can be enabling with more confidence, and ease of use and flexibility, but it can’t stop there. We have to demonstrate to physicians, nurses, pharmacists, and patients that the experience with Afikampton, like in the open label extension, is one that can be engendering confidence.

We have to design something that’s tailored for patients and that is recognizing of how patients identify with their disease. And that’s what I think ultimately matters. I hope that our label will be reflective of this experience that we’re trying to create for patients that would ultimately be enabling of more physicians, more patients to get comfortable with a medicine sort of this. And again, it’s all as driven by science.

Ruana Ruiz, Senior Biotech Analyst, Lyric: Yep. I hear you. So you also mentioned two phase three trials coming up that we should watch for both MAPLE and ACATIA. So could you just give us an overview of what do you hope to see in these two different trials and how would it be important for expanding afacamten’s opportunity in the future?

Operator: So I’ll turn to Andrew about this, but firstly, I’ll say we were very encouraged by data from Sequoia, which demonstrated that for patients both on and off beta blockers, that those receiving affikamten did better than placebo counterparts. So that’s indicative of what we hope will be, confidence going into the rate readout of maple where we’re doing a head to head comparison of patients on alpha campton versus those on beta blockers. Beta blockers, for those of you don’t know, tend to be used first line, for these patients, a, because they’re cheap and buzz they’ve been around for a long time, but there’s no clinical evidence to support their use in terms of, increased exercise capacity. So we do believe that this could be meaningfully incremental if affecamten shows superiority to beta blockers, especially, as it relates to guideline directed treatment and how payers might see, afecamten. So we’re looking to maple to be, enabling of increased confidence.

In Acacia, we’re studying in a population called non obstructive HCM, whereas Andrew can comment, we’re seeing a more rapid growth of prevalence. So Andrew, might you speak to both where you see maple and acacia fitting in?

Andrew Callos, EVP and CCO, Cytokinetics: So it’s important to understand from a market point of view, New York Heart Class IIIII is the patients that we’ve studied, and they’re the ones that would be candidates for a CMI. Over ninety percent of patients today are on a beta blocker or calcium channel blocker, most on a beta blocker. We talked about the importance of unlocking cardiology, general cardiology outside the specialized centers where eighty percent of prescribing is today. There are those physicians. So in market research, there’s a couple kind of key findings.

One, there is a segment of physicians who feel like beta blockers are good enough. They’re not educated on CMIs or they’re not convinced. And when they would see head to head data, if you put a profile in front of them that says a head to head data that shows superiority, it unlocks more physicians in terms of activating them to prescribe. The impact that has on that broader set of cardiology as well as those that are already prescribing, it does two things. One is it expands the market.

So if you look at peak penetration of what a CMI market will be for eligible patients, that increases with the addition of a second data set. And when you look at a preference share of available treatments, the preference share for APTICamp increases as well. A second data set confers more assurance that efficacy, safety, real world in a second study provides a little more confidence that it just wasn’t a single study. So it really grows the market and it increases share and it should unlock more prescribers from a Maple point of view. From a payer point of view, Maple should also increase or influence guidelines.

Once guidelines come into play, then that’s when we can start to hopefully unlock payers over time, but that takes more time. From a non obstructive, so when we looked at this market maybe two or three years ago in terms of epi, the obstructive versus non obstructive was about a sixtyforty with obstructive being 60% of the population. Obstructive population is growing with the population, so it’s fairly flat, very, very small level of increase. Non obstructive is increasing at double digit rates. So non obstructive is probably approaching now fiftyfifty in terms of the market and likely will overtake obstructive in terms of the overall number.

So non obstructive is turning out to likely be a larger opportunity than we were originally anticipating and we’re going to be publishing from an ATOR point of view later this year, the ADEPI data, that analysis and what those numbers look like overall. So but it’s the same I think another critical factor point is these are the same prescribers. So we’re building a field force to focus on 10,000 cardiologists who are 80% of diagnosis and prescribing of all therapies, including generics. They would be the same prescribers, obstructive, not obstructive. So our go to market strategy, when you look at Sequoia, when you add on Maple, when you add on a different population of Acacia, it’s all the same infrastructure and same fuel force expanding this market.

Operator: I’m going to sing Andrew’s praises for a minute because one of the reasons why he was so attractive to join us as our Chief Commercial Officer is, and this was four years ago, is that he had co led the development and co promotion of Eliquis when he was then at Pfizer after twenty three years working together with BMS, understands what a next in class drug coming into a market can do for a category. And Eliquis, is one of those archetypal examples of a cardiovascular medicine that made a huge impact, with the introduction of a new medicine as a second entrant and how that grew the category for the benefit of all patients.

Ruana Ruiz, Senior Biotech Analyst, Lyric: Got it. I know, Andrew, you alluded to some possible guideline updates that could be favorable after MIPL and or Acacia readout. Could you just help frame for the audience timelines of that? If there’s any way to know when those could kick in? And I know it’s a moving target, but and how are you thinking about this possibly happening for only AFI or could there be a CMI class update?

Like what are the pushes and pulls there?

Andrew Callos, EVP and CCO, Cytokinetics: Yes. I mean, if it’s a little bit crystal balling then, I mean, I’ll go from our colleague, Dan, who’s in the with us today or VP of Clinical Development that tells me that guidelines are trying to move forward towards annual updates as compared to maybe every three or four years. With this data set, is it conceivable that guidelines could be updated in early twenty twenty six to include? My experience is what they would probably would say CMIs, but with an asterisk pointed data. The other difference is if guidelines do say CMIs with an asterisk pointing to Maple, from a promotional point of view, a pharmaceutical company needs evidence to promote a given claim.

So we would have that evidence to promote that claim. And to my knowledge, BMS is not running such a study. And so I think from a preference point of view or from an execution point of view, we would be able to show both data sets. This is a confirmatory data set, maybe with higher levels of peak VO2, but confirmatory safety, confirmatory efficacy. And I think that speaks volumes for cardiologists in my experience.

I’ve been in the cardiology market for sales and marketing for a long time. And when you see evidence over evidence, especially with long term data, this really does give assurance to some cardiologists. Cardiologists generally take longer to get on board with newer therapies, and this certainly can accelerate

Ruana Ruiz, Senior Biotech Analyst, Lyric: that. Got it. And I know this is more than just a one product story. So it’s not all about Afi. So I did want to ask a bit about some of your pipeline development products as well.

We’ve got five eighty six, which is advancing. We’ve got omecamtiv. Maybe could you give us a little bit of highlights about where those stand and what to look forward to next?

Operator: Yeah. So heart failure is the number one reason why people in The United States are hospitalized. It’s growing, with the aging demographics, and it represents already six to seven million patients in The United States. And there are new medicines that are making a dent in heart failure, certainly, SGLT twos and GLP ones and otherwise Entresto’s a $7 or $8,000,000,000 a year drug. But for which on the extremes, those patients with severely reduced ejection fraction or those patients with supernormal ejection fraction hypercontractility, It’s our assertion that those medicines are not demonstrating effects that would address the high unmet need.

And we’ve positioned omecamtiv for severely ill heart failure patients with reduced ejection fraction. It was already the subject of a positive phase three study in over 8,000 patients, but admittedly where the effect size in a less sick population was more modest. So we’re now doing a confirmatory study as requested by FDA in a sicker sicker population. And as that study is now in phase three and enrolling, we’re very excited about where that represents upside. On the other side of the spectrum are those patients with hypercontractile ventricles and heart failure with preserved ejection fraction.

And there, we think a cardiac myosin inhibitor could also play a meaningful role. These patients look like the non obstructive HCM patients in a lot of ways, And we have very encouraging data to support the use in in HCM. So we’ve advanced a second cardiac myosin inhibitor, CK five eight six, into phase two, and that study is now enrolling. So we have omecamtiv in phase three, CK five eighty six in phase two, and it’s those three programs that represent three legs of a stool for a franchise strategy that speaks to who we are at Cytokinetics in terms of business. And we do believe that we are in a position by 02/1930 to be advancing three potential medicines to patients and as would be addressing, unmet needs across different patients with impaired cardiovascular muscle function, but all as would be generally prescribed by the same concentrated cardiology physician base.

So we don’t have to scale sales and marketing necessarily because we’re talking about relatively the same customers, and we think that’s core to strategy.

Ruana Ruiz, Senior Biotech Analyst, Lyric: Yep. Got it. So I know we’re close at time. I just want to pause here and see if anybody in the audience has any questions that they might want to jump in with.

Operator: Edgewise as a competitor.

Ruana Ruiz, Senior Biotech Analyst, Lyric: I think the question was, do you see Edgewise as a possible competitor? What are your thoughts there?

Operator: So I don’t want to talk about, you know, competitors or competitors. What I will say is we’re very, science and data driven, and we’ll look forward to seeing data from both Camzyos and the Edgewise seven thousand five hundred compound as they come forward. We believe that the bar is very high that was set by Afikampton in terms of what would be expected clinical profile. And one needs a lot of data here in order to be able to make statements about where one would be positioned. What I will say is with afacamten, we have a five and a ten milligram dose for which if we only studied those two doses, we would see rapid gradient reductions, no EF excursions, and one might conclude that those drugs were sufficient, those dose strengths sufficient to address a majority of patients without the need for echo monitoring.

We don’t believe that addresses the diversity of patients that some require higher doses and we do believe that FDA is not likely to consider no echo monitoring, for patients with OHCM, especially since these patients get, echo monitored once or twice a year already. So we think that Afikampton occupies the sweet spot for what would be, positioning as would, set the bar high for any other competitive threat.

Ruana Ruiz, Senior Biotech Analyst, Lyric: I don’t see any other questions, so I’ll probably wrap it there so we can get to other sessions. But thanks again, Robert and Andrew, for joining us. It was a really great discussion.

Operator: Thank you very much. Thank you.

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