Cytokinetics at Citi’s Biopharma Conference: Aficamten’s Promising Path

Published 02/09/2025, 21:06
Cytokinetics at Citi’s Biopharma Conference: Aficamten’s Promising Path

On Tuesday, 02 September 2025, Cytokinetics Inc. (NASDAQ:CYTK) presented at Citi’s Biopharma Back to School Conference, detailing its strategic advancements and challenges. The company highlighted its commitment to muscle biology and specialty cardiology, focusing on aficamten for hypertrophic cardiomyopathy. Positive study results and a strong financial position were emphasized, though competition and regulatory hurdles remain.

Key Takeaways

  • Cytokinetics is preparing for the potential FDA approval of aficamten for obstructive hypertrophic cardiomyopathy (OHCM) with a PDUFA date set for December 26.
  • The company maintains a robust financial position with approximately $1 billion in cash and access to additional capital.
  • Aficamten’s clinical profile is positioned to differentiate it from Bristol Myers Squibb’s mavacamten.
  • The MAPLE study showed aficamten’s superiority over beta-blockers, potentially influencing treatment guidelines.
  • Cytokinetics aims to expand the cardiac myosin inhibitor market rather than compete directly with existing products.

Financial Results

  • Cytokinetics reported a strong cash position with $1 billion in cash and investments at the end of Q2.
  • The company has access to a $100 million loan from Royalty Pharma, with an additional $175 million available upon FDA approval of aficamten, and $150 million for pipeline development.
  • Previous financings included equity and non-dilutive deals, strengthening its financial foundation.

Operational Updates

  • The SEQUOIA study for aficamten in OHCM is under regulatory review, with a decision expected by the end of the year.
  • The MAPLE study results, published in the New England Journal of Medicine, demonstrated positive outcomes for aficamten.
  • The ACACIA study for non-obstructive HCM (NHCM) is fully enrolled, with results anticipated in the first half of 2026.
  • Cytokinetics is building a specialty cardiology franchise with a sales team targeting high-volume prescribers in the US and Europe.
  • The COMET study is enrolling patients to confirm previous findings for omecamtiv mecarbil in advanced heart failure.

Future Outlook

  • Cytokinetics plans to launch aficamten post-approval, focusing on centers of excellence and community cardiologists.
  • The company anticipates its REMS program for aficamten will be less burdensome, aiding in physician adoption.
  • There is potential for label expansion based on MAPLE study results, which could enhance market penetration.
  • Cytokinetics is developing CK-586 for heart failure with preserved ejection fraction (HFPEF).

Q&A Highlights

  • Aficamten’s differentiation from mavacamten includes a favorable titration profile and fewer drug-drug interactions.
  • The MAPLE study’s strong data may lead to aficamten being considered as a first-line therapy over beta-blockers.
  • Cytokinetics aims to grow the cardiac myosin inhibitor market, leveraging existing referral patterns.

For further details, readers are encouraged to refer to the full transcript below.

Full transcript - Citi’s Biopharma Back to School Conference:

Unidentified speaker, Conference Organizer: Everybody for coming to the City Bio Pharma Conference. Next on the docket, we have with us Cytokinetics. They had some interesting news today. I’m sure we’ll discuss it. Before we jump into that, if you could all just introduce yourselves and give us a kind of the brief history of Cytokinetics, and then we’ll jump in.

Robert Blum, President and CEO, Cytokinetics: Good afternoon. Thanks to the folks at City for inviting us to the conference today. It’s a big day for us. I’m joined by an all-star team of our senior executives. I’m Robert Blum. I’m President and CEO of Cytokinetics. I’ll ask them to introduce themselves. I’ll maybe for just a few minutes provide some background and then jump into a presentation that we made and try to do all that in about five minutes so that we leave plenty of time for conversation.

Fady Malik, Head of Research and Development, Cytokinetics: I’m Fady Malik. I’m the Head of Research and Development at Cytokinetics. I joined the company when we launched in 1998. I’m a physician scientist and cardiologist and lead our both discovery and development efforts.

Sung Lee, CFO, Cytokinetics: I’m Sung Lee. I joined Cytokinetics about 16 months ago as CFO.

Isaac Ciechanover, Chief Business Officer, Cytokinetics: Hello there. I’m Isaac Ciechanover. I’m the Chief Business Officer, and I also joined around 16 months ago.

Robert Blum, President and CEO, Cytokinetics: As mentioned, I’ll just provide a quick overview, but then I’ll jump into a few slides. Fady and I started this company 27 plus years ago, and over these many years, we’ve built out a new pharmacology rooted in one biology. He, as he is a visionary and physician scientist, has led us all along the way. Over the course of these many years, we’ve built out a portfolio. You’ll hear about that more in a minute. We’ve added to our team, including, as you see here, Sung and Isaac, who joined us within the last year or so. I think that reflects the maturity and the evolution of the company. There are many of us who have been with the company for 10, 20, or more years. Others who have joined more recently.

All fit for purpose as we’re seeking now to turn a page on the company as we move from R&D to commercialization over the next several months. With that as a background, I’ll jump into some more detail. I’ll be making some forward-looking statements. I’ll point you to these slides and also to our SEC filings as it relates to caveats to those statements. We don’t undertake an obligation necessarily to update those statements. I will be talking to you about the company in broad brush strokes. Our mission has always been to mine this area of biology for new medicines. In particular, as that biology reads on muscle and translating muscle biology into a new muscle pharmacology, in particular around diseases of cardiovascular and neuromuscular impairment.

As you can see on this slide, our commitment to that science has translated into a pipeline, a pipeline of potential medicines, all of which have been discovered and developed at Cytokinetics and for which we’re quite pleased with progress, including progress announced even just this past weekend at the European Society of Cardiology. As I mentioned, our focus has been on muscle biology, in particular, the mechanics or machinery that drives the contractility of muscle and where one particular molecular target, cardiac myosin, a mechanical chemical enzyme that translates ATP hydrolysis into mechanical force that drives contractility of muscle.

Ours is the first company to industrialize research around the sarcomere, the fundamental unit of muscle contractility that we all probably studied in high school, but for which we now have a pharmacology rooted in myosin modulation, inhibitors, and an activator of myosin that we believe hold great promise to the build of a specialty cardiology franchise. Lead amongst these compounds is aficamten, a potential next-in-class cardiac myosin inhibitor. It’s currently pending FDA review for potential approval in obstructive hypertrophic cardiomyopathy based on a study called SEQUOIA that read out over a year ago. That study provides, we believe, ample support and evidence for what we hope will be an approval later this year, PDUFA date December 26. At the same time, we’re pending regulatory review in China and in Europe based also on SEQUOIA. That informs the leading edge of our pipeline and go-to-market strategy.

This past weekend at the European Society of Cardiology, investigators presented results from a second Phase III study called MAPLE. That study was positive, and we believe that could enable a potential label expansion, but not initially will we go to market based on MAPLE, instead on SEQUOIA. As you may have questions, the team here, we can respond to how MAPLE factors into our lifecycle management plans. A third study is completed enrollment and will read out in the first half of 2026. That’s a study called ACACIA. ACACIA is a study of aficamten in patients with non-obstructive HCM. You begin to understand how aficamten is itself a pipeline-in-a-pill, OHCM followed by NHCM, but for which there are two other drug candidates in later stage development.

Omecamtiv mecarbil, a cardiac myosin activator, already the subject of over 30 completed clinical trials, including one 8,000 patient study called GALACTIC that was positive and for which we’re now doing a second confirmatory study and how that may enable us to extend the franchise to advanced heart failure treated by the same physician group. There’s another cardiac myosin inhibitor called CK-586. It acts by a different mechanism. It’s being developed for the potential treatment of heart failure with preserved ejection fraction. Three drug candidates, one molecular target, one strategy oriented towards building an enduring business in specialty cardiology. We have other compounds in earlier clinical development. They’re depicted on the pipeline. I won’t speak to them today.

You begin to see how this all begins to play out in terms of what could be multiple specialty cardiology launches that could be occurring over successive time, starting with Sequoia by the end of this year, followed by MAPLE, followed by ACACIA, followed by HFREF, followed by HFPEF, and there you begin to understand why we think a specialty cardiology business model, the likes of which really don’t exist in biopharma, but for which we believe could build uncommon enduring value for patients and shareholders. Over the weekend, I mentioned we presented results from MAPLE. These are the primary efficacy data as they were presented on Saturday in a late breaking session, simultaneously published in the New England Journal of Medicine. What you can see here depicted is patients on aficamten did better by the primary efficacy endpoint of peak VO2. This is a measure of exercise capacity.

Patients on the standard of care beta-blocker, metoprolol, actually did worse. How could that be standard of care, you might ask? This was the first randomized controlled study of a beta-blocker in patients with obstructive hypertrophic cardiomyopathy (OHCM), despite the fact that beta-blockers have been a mainstay standard of care in this disease for 60 years. Absent having done a proper study like this, we do believe now that these data are presented and published, they may turn heads and call into question the use of the first-line treatment in this population. I think the cardiology community very enthusiastically embraced these data when presented on Saturday. If you’re interested, we also convened an IR webinar this morning, and the principal investigator presented again, and those data in that presentation are archived on our website alongside the publication. Here are the design elements of the ACACIA study that I mentioned.

As you may have questions, we’ll get into that, I’m sure. ACACIA is due to read out in the first half of next year, as I mentioned. This is really interesting. Non-obstructive HCM represents probably half the population with HCM, and where the prevalence would suggest it’s growing at a faster rate than obstructive HCM. The first-in-class compound in this category, a compound called mavacamten, was recently the subject of a study called Odyssey in patients with NHCM. Those data were also presented on Saturday at the same late breaker. Unfortunately, that study was not successful. The study failed to demonstrate an effect on either of the two coprimary endpoints, but with trends that are really quite provocative and for which we believe they may illuminate that aficamten in ACACIA could represent a positive outcome.

As you may have questions about what was presented and why we remain optimistic, Fady can speak to that in our question and answer session. We are building a specialty cardiology franchise. You see here how they all fit together. This is a concentrated customer segment base where we believe with roughly 125 sales professionals in each of the United States and Europe, we can get to the high-volume prescribers who treat these advanced populations where there’s a very high unmet need. We believe we can build a specialty distribution network, and with what we believe to be both payer leverage and differentiation amongst products, we believe we can build a very valuable business for shareholders with all of these products. We have a strong financial position. We recently reported our Q2 earnings, over $1 billion, approximately $1 billion in cash.

To the point, given deals we’ve done, and I do believe Cytokinetics has created a high watermark amongst peer group companies in terms of combining both equity dilutive, but especially leaning into non-equity dilutive capital with partnerships, royalty monetizations, and the proper balance of debt, we believe we have access to still additional capital. We have access to $100 million in a loan from Royalty Pharma that we have already qualified for, another $175 million that we may qualify for if aficamten is approved by the end of the year, and an additional $150 million that may be available to us for the development of one of our pipeline programs.

These are loans that come with deals we’ve done with Royalty Pharma and for which we believe the cost of capital is competitive and advantageous to us as we continue to hopefully be good financial engineers, much in the same way we’ve developed our R&D pipeline. Sung can speak to how we’re thinking about not only access to capital, but how we can be efficient with regard to deployment of capital. With that as an introduction, I’ll turn it back to you for question and answer, and thank you for your interest in Cytokinetics.

Unidentified speaker, Conference Organizer: Thank you so much for that, and I appreciate the seminar this morning. It was very interesting. I guess to start out, when we look at this space overall, OHCM and NHCM, naturally people are looking at Ching Jaw, which is out there, mavacamten, and they’re trying to measure that up against the profile of aficamten. Thus far, we can compare them on the actual data in OHCM, which is under the FDA review, but there’s also these other emerging data sets with respect to MAPLE and ACACIA coming online, Odyssey from their end. How do you see the profiles of these drugs ultimately being compared? I mean, there’s the REMS program. Yes, there is the data that’ll be on label, but they also, all the doctors know that there’s other things going on that differentiate them.

Robert Blum, President and CEO, Cytokinetics: Yeah, that’s a very good question. There’s a lot to unpack. I’m going to turn it over to Fady, but I’ll just start by saying we played a hand in the discovery of mavacamten as it was discovered in a company that incubated within our laboratories before we spun it out as a separate entity. We know that compound quite well. We understand its positives, but also where there could be opportunities to engineer a next-in-class compound as we did. Aficamten was engineered with certain properties in mind, and we’ve studied it both preclinically and clinically to elaborate on what could be a differentiated profile. We do believe the clinical studies that we’ve conducted in later stage development, REDWOOD, SEQUOIA, now MAPLE, elaborate on its profile. With that, maybe I’ll turn it over to Fady to answer your specific question.

Fady Malik, Head of Research and Development, Cytokinetics: Sure. You know, as Robert said, and we worked to discover aficamten, we had a particular profile in mind. We wanted a drug that could be titrated easily, a drug that was reversible. If you like with any drug you titrate to effect, you want it to be able to down-titrate simply, a drug that’s devoid of significant drug-drug interactions that complicate its use. Ultimately, to take those properties, including a shallow PK/PD relationship, and develop a best-in-class beta package for them. Through the conduct of our Phase II trial, Redwood, followed by Sequoia, which is now followed by MAPLE in obstructive HCM, we think that the safety profile of aficamten is exemplary, with showing essentially that titration works. You can titrate the drug as needed. You don’t have to interrupt treatment. You can down-titrate the drug if necessary. Patients respond to down-titration.

We haven’t observed heart failure events, significant heart failure events, as a consequence of the use of aficamten. As was presented at the European Society of Cardiology, in addition to MAPLE, was some very long-term follow-up data in our open label extension. Those things, those attributes that we intentionally designed in aficamten now are manifesting themselves in our development program. Ultimately, we hope that leads to a label and practical, everyday experience in using aficamten that make it something that physicians want to choose.

Unidentified speaker, Conference Organizer: Understanding that your REMS program is still in the works and we don’t have a submission, where do you see, given the recent changes that happened on Bristol Myers Squibb’s end, as potential areas of differentiation for your program?

Fady Malik, Head of Research and Development, Cytokinetics: Yeah, I think, you know, the properties, again, the way that we have employed aficamten in our clinical trials, we hope would be reflected in the label as well as REMS. Titration would occur on a more frequent schedule, so patients could potentially get to target dose within six to eight weeks if they so chose, perhaps maybe longer than that if they decide to go more slowly, if they don’t have to worry about significant drug-drug interaction effects. That’s another aspect of REMS that physicians might be educated on the label, but patients don’t have to every month kind of go through their medication list with a pharmacist because there aren’t as many or meaningful clinically significant drug interactions. All of these things I think help, if you will, differentiate a potential REMS from another.

The REMS are reflective of your label, and the label is reflective of the use of the drug in the clinical trials as well as obviously the data that you generate with them. I think if you look at our clinical data, they support a meaningfully differentiated REMS down the road.

Unidentified speaker, Conference Organizer: In terms of once it’s approved and launches, what’s the lift in getting into the new to therapy patients versus potentially getting some switches? It strikes me that certainly given some changes regarding for Ching Jaw, a switch is a higher lift, a much heavier lift than would be a new to patients, a new to therapy. Towards that end, how penetrated is the market at this point?

Robert Blum, President and CEO, Cytokinetics: Yeah, so Bristol Myers Squibb is doing a nice job educating and building market awareness for the category of cardiac myosin inhibitors. They’re adding about 1,500, 1,600 patients per quarter, and they’re going to do north of $1 billion worldwide in sales this year. For which in the U.S., there’s still about 650 physicians who are accounting for over 80% of the prescriptions. It’s incumbent upon us to work to grow the category and to hopefully have preferential share of the category, but to ensure that more physicians are comfortable prescribing a cardiac myosin inhibitor for their patients. Right now, we believe they’ve penetrated about 15% of the symptomatic diagnosed population and where there’s even more who are undiagnosed and perhaps going to be available ultimately for treatment, and that’s in the U.S.

We believe that a next-in-class opportunity, if aficamten were to be approved, could be opening the aperture on the market in ways that the differentiation could drive wider adoption. Cardiology launches generally take longer. There’s both that period under which you have to go from free drug to revenue-producing drug, but also just adoption takes longer, but they have long tails as well. We do believe that there’s quite a substantial TAM here and enduring value that could make this one of the leading cardiovascular categories. It’s important that Cytokinetics, when we go to market, we’re not seeking to compete with Bristol Myers Squibb. That would be perhaps bringing us both down, but rather as could be enabling of more patients to benefit from this innovation.

While there may be some switches and we’re hearing that there are high-volume prescribers who are intending to switch their patients, that’s not going to be core to our strategy. Rather, instead, what’s core to our strategy is building on the momentum and speaking to the integrity of our data, our science, and its implication for patients with obstructive hypertrophic cardiomyopathy.

Unidentified speaker, Conference Organizer: On the question of the high-volume prescribers, because of the burdensome REMS program, has there been kind of a conglomerate of people who will just pass the patients on because they don’t want to deal with the program toward these high-frequency prescribers? Can the drug that gets kind of has a more simple or easier program push beyond that group?

Robert Blum, President and CEO, Cytokinetics: Yeah, so we’ve been out in the field, if you will, for a couple of years seeking to answer that question, and I think we’ve got a lock on it. We understand referral patterns very well locally, metro area by metro area, and we understand how they translate to payers. We have had 15 Area Business Managers and many MSLs deployed for a long time readying for our commercial launch. We believe we understand it well enough to know this, that right now a lot of community-based cardiologists are choosing to refer patients to centers of excellence, but reluctantly, and that if there is a potential new medicine that could be available with a less burdensome REMS, that they may be more likely interested in prescribing themselves. We believe we can activate them if aficamten is approved with the differentiated label that we expect of it.

Our strategy will be to focus to centers of excellence and also where there may be low-lying fruit in the peripheral community of cardiologists who also see a lot of HCM patients. Our intention is to be focused to where we can influence both those high-volume prescribers who have themselves already been warehousing patients awaiting the potential of aficamten, as they’ve told us, and at the same time grow the category beyond where currently cardiologists are comfortable prescribing.

Unidentified speaker, Conference Organizer: You presented details of MAPLE this past weekend, and the data was very, very strong. It was clearly superior. How does that translate ultimately into a market opportunity? How difficult is it going to be to move into the first line? How should we think about that when we’re building our models? Secondly, do you see as there being a benefit just from an optics perspective of eventually having on label that might help after beta-blockers, even though the trial is for a front line?

Robert Blum, President and CEO, Cytokinetics: The question you just asked, I asked that of many opinion leader cardiologists over the weekend. I do believe for the fact that beta-blockers are a mainstay standard of care and they’re cheap, that it’s not going to be overnight that we see practice changing. To my surprise, many cardiologists indicated that when they get back to their offices today, they will be looking at certain patients differently than they would have last week for knowing these data, and that the time that they may remain on beta-blockers could be shortened. At the end of the day, this ultimately has to factor into guidelines. To be clear, we won’t be promoting MAPLE data upon launch. It won’t be in our label.

We will be compliant to label, but for which we could imagine that there may be some physicians who are more likely going to be looking more skeptically or critically at beta-blockers. Ultimately, this will take some time, but I do think it could be enabling of aficamten to become a first-line therapy for these patients. Fady, I think, has a better insight into this, and maybe he should also comment.

Fady Malik, Head of Research and Development, Cytokinetics: Yeah, obviously, beta-blockers have been entrenched for decades, and people have a lot of comfort with using them. If our data weren’t so compelling, it’d be, I think, a long road to displace them. The fact that aficamten was better than metoprolol was not surprising to me. I expected that to be the case. What was, I think, surprising to the physician community was how ineffective metoprolol was. Obviously, that should cause one to question its use. Now, remember, these things can always be tried, and one can switch from one to the other. I think the data over time, as they get incorporated into timelines, as physician practice begins to evolve, you’ll begin to see, not in year one, year two, but out in the outlying years, you’ll begin to see a greater proportion of physicians that decide to go straight to cardiac myosin inhibitors.

Payer practices will probably evolve over time to support that as well. This is a trend that’s been seen not once, but many times before in medical practice where the newer therapy displaces something that maybe has been entrenched for quite some time.

Unidentified speaker, Conference Organizer: It’s very interesting. I’m curious to see how that ultimately evolves. If we move on now to non-obstructive, and obviously ACACIA is going on, and there is data presented today with respect to Ching Jaw from Odyssey or presented this past weekend, could you run us through what your takeaways from that data are and what are the deltas that from the Ching Jaw experience that could be different for you? Because they came close. They just were a little short.

Fady Malik, Head of Research and Development, Cytokinetics: Yeah, no, absolutely. We were pleasantly surprised to see that there were positive trends on their endpoints. The underlying data, you know, the rationale for using a drug in NHCM was also positively impacted. It reduced anti-ProBNP, which presumably reflects improved filling pressures. It reduced or improved diastolic function and the relaxation of the heart. You saw peak VO2 move in the right direction. You saw KCCQ, their other primary endpoint, move in the right direction. That one was confounded by an unusually large placebo effect that is not typically observed to that magnitude with KCCQ. What was clearly evident is that the way that the drug was dosed confounded the interpretation of the results. They had upwards of 25% of patients that had to interrupt treatment at some point during study. Some of those went on and discontinued treatment permanently.

We had patients that developed heart failure as a result of some of the EF excursions. Obviously, that impacts patients’ assessment of their health status as well as their exercise performance. There were doses that were utilized that haven’t been studied before for their effectiveness, like 1 milligram or 2.5 milligrams. What we put together with that is that perhaps there was a dilution of the treatment effect. We’ll have to wait and see if they publish a non-treatment analysis that excludes patients whose treatment were interrupted. They indicated as much as that they were planning to do so, and you would think that if that was the case, there’s probably something there.

You know, aficamten, we’ve in our Phase II and open label extension experience, we’ve been able to dose aficamten with an EF-guided algorithm that has been well tolerated, hasn’t led to discontinuations of treatment or treatment interruptions or severe heart failure events. In the background of that, we’ve seen improvements in diastolic function. We’ve seen improvements in anti-ProBNP, even to a larger extent than they observed. I think at least the fundamental basis for believing that this drug should work in NHCM is still intact. Perhaps something that is less prone to confounding will help us get to the right answer.

Unidentified speaker, Conference Organizer: Obviously, we’ll find that out not too far from now. Getting back kind of to a question I had asked earlier, if you’re a doctor out there and you’re prescribing and you’re trying to compare the two data sets that are on label, which is always fraught with difficulties and it gets noisy, but you have a label that one of the therapies has MAPLE on it and does have non-obstructive, assuming the trial works out, what does that mean for the on-label indication right now that eventually it can turn out there? Does that mean that the profile of aficamten in the head-to-head indication right now is actually improved, or do you think doctors will view them all as completely separate indications?

Fady Malik, Head of Research and Development, Cytokinetics: I think that remains to be seen, but you invest a lot of time learning how to use a particular drug. If you can use one for everything, then there’s no reason to necessarily pick the other because it’s superior in some aspect. I think the rational thing is that people will simplify their practice of medicine and kind of focus on the one that has the most uses. ACACIA could provide, if you will, a rationale for focusing on the use of aficamten if ACACIA were positive. I think physician practice ultimately might reflect that.

Robert Blum, President and CEO, Cytokinetics: I think there’s two ways to answer your question. It’s, does the properties of the molecule lend itself to the potential approval across different indications, or do the indications ultimately drive the use of the molecule? I think they’re, in some ways, one and the same. This is a bit of a reciprocal. We’ve designed and engineered into aficamten certain properties that may lend it, able to be studied in such a way that it could translate to positive outcomes in OHCM and NHCM. That was the intention. I believe enabling of flexible dosing, flatter PK/PD relationships, and the opportunity to achieve greater dose density is hopefully going to be serving us well, not only in OHCM but also in NHCM.

Unidentified speaker, Conference Organizer: We’ve got five minutes left here. What do you think we should, I mean, we could talk about a lot of other things. We could talk about what your marketing effort will look like, or if you want to move into omecamtiv mecarbil, what do you think that the street is not paying attention to that we should be?

Robert Blum, President and CEO, Cytokinetics: Maybe I’ll answer that real quick, but I also want to make sure that we get a chance for you to learn a little bit more about how we’re thinking from a corporate development and a finance standpoint, because I think those things matter also to ultimately inform the full picture of Cytokinetics. To your question, omecamtiv mecarbil is the subject of a study called COMET, which is designed to confirm what we’ve already seen in GALACTIC. In GALACTIC, approximately 4,000 patients were enrolled in each of two arms. This was a study of a broader, more heterogeneous group of heart failure patients with ejection fractions below 40. We saw a statistically significant effect on death and readmission, but admittedly, it was a more modest effect. In the more advanced patients with EF below 30, it was more than double.

The FDA and EMA have asked us to now go back and do a smaller study just in that population, confirm the effects. If COMET is positive, which we have every reason to believe it can be, that could be really meaningful for patients who right now don’t have a good treatment for the more severe forms of heart failure. In effect, we’re doing a 2,000-patient study to replicate something we’ve already seen in an 8,000-patient study, of which 4,000 of those patients match up to the inclusion criteria in the 2,000-patient study. We hope this is going to be like being on the five-yard line, first and goal, and an opportunity to bring this forward to the end zone for patients and shareholders. That study, COMET, is already enrolling. Hopefully, it completes enrollment next year and would be another pillar in our franchise development strategy.

Fady Malik, Head of Research and Development, Cytokinetics: Sure, so we think about business development from two perspectives. Our primary focus is obviously the launch of aficamten and making sure that patients everywhere in the world get access to it. As you know, late last year, we did a deal in Japan. We have a partner in China with Sanofi, Bayer in Japan. There’s still the rest of the world territories. We are obviously building our own franchise in Europe, but there’s been considerable interest in being able to work with us in those territories. That’s something that is of great focus to aficamten. Separate to that, there’s in-house expertise, as you can imagine, both from a biology perspective, chemistry, in muscle diseases in general, not just cardiac. We are going to leverage that. From a business development perspective, we look at multiple programs, both preclinical as well as clinical, to augment our pipeline.

We’ll do that in a cost-effective way, but we really are in a unique situation to bring best of breed. We did a deal last year from an equity perspective with Imbria, where we think that it’s a very unique molecule. We continue to do that across the board, and that’s something that you should pay attention to and expect from us.

Robert Blum, President and CEO, Cytokinetics: Sure, so obviously in building a cardiovascular franchise, we want to make sure that whatever we do, we can leverage our sales force. I will say that our focus has been on small molecules. We look to expand outside of small molecules into other modalities, but ones that are already well established. Skeletal muscle and cardiac are probably the primary focus for us. None of this happens without a strong balance sheet and good financial discipline. Maybe Sung could speak to sort of how we’re approaching those matters.

Fady Malik, Head of Research and Development, Cytokinetics: Sure, happy to. Just to reiterate what Robert said, we ended quarter two with a very solid balance sheet position with $1 billion in cash and investments. Of course, this was made possible through the financings that we did about a year ago, partly equity, but also partly non-dilutive capital provided by Royalty Pharma. I also want to focus you on the capital that lies ahead of us, in addition to the $1 billion in cash and investments already on our books. As Robert mentioned, we’re eligible to take down a $100 million loan from Royalty Pharma this year. We’ve already qualified for that. We could further become eligible for an additional $175 million should aficamten be approved this year in the U.S. Those will be two very important levers for us in terms of adding capital to our balance sheet. Also, we have pipeline funding.

Should CK-586 move into a pivotal study, Royalty Pharma has the option to opt in up to $150 million. That could be potentially an important source of capital to further advance our pipeline.

Unidentified speaker, Conference Organizer: Excellent. I think we’ve run off to the end of our time. Thank you so much. Always great and look forward to seeing you again soon.

Robert Blum, President and CEO, Cytokinetics: Thanks very much. Appreciate it.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.

Latest comments

Risk Disclosure: Trading in financial instruments and/or cryptocurrencies involves high risks including the risk of losing some, or all, of your investment amount, and may not be suitable for all investors. Prices of cryptocurrencies are extremely volatile and may be affected by external factors such as financial, regulatory or political events. Trading on margin increases the financial risks.
Before deciding to trade in financial instrument or cryptocurrencies you should be fully informed of the risks and costs associated with trading the financial markets, carefully consider your investment objectives, level of experience, and risk appetite, and seek professional advice where needed.
Fusion Media would like to remind you that the data contained in this website is not necessarily real-time nor accurate. The data and prices on the website are not necessarily provided by any market or exchange, but may be provided by market makers, and so prices may not be accurate and may differ from the actual price at any given market, meaning prices are indicative and not appropriate for trading purposes. Fusion Media and any provider of the data contained in this website will not accept liability for any loss or damage as a result of your trading, or your reliance on the information contained within this website.
It is prohibited to use, store, reproduce, display, modify, transmit or distribute the data contained in this website without the explicit prior written permission of Fusion Media and/or the data provider. All intellectual property rights are reserved by the providers and/or the exchange providing the data contained in this website.
Fusion Media may be compensated by the advertisers that appear on the website, based on your interaction with the advertisements or advertisers
© 2007-2025 - Fusion Media Limited. All Rights Reserved.