Good afternoon, everyone. My name is Gena Wang. I'm a biotech analyst at the Barclays 7th Global Healthcare Conference. It's my pleasure to introduce our next presenting company, Cytokinetics. On the stage with me, we have Robert Blum, President and Chief Executive Officer. We also have Andrew Callos, EVP Chief Commercial Officer. Rob, I think you wanted to give a brief introduction before we dive into the questions.
Yes, thank you. Thanks to Gena for the introduction. Thanks to Barclays for inviting us back for us to provide an update on all the good things happening at Cytokinetics. I'll be making a short presentation. We should start with this picture. This is Eric. Eric is a patient with OHCM, and it's people like Eric that inspire us to do the work we do as we seek to, through our science, our portfolio, potential medicines, empower muscle, empower lives. I'll be making some forward-looking statements. I'll draw your attention to our SEC filings with regard to elaborations and caveats to those statements. We do not take initiative and undertake any obligation with regard to updating those statements. We point you to those risk factors and filings. Here's our mission. It's a mission that's been in place for Cytokinetics for a good while.
We are pioneers and leaders in an area of biology for which we've maintained a discipline and focus. We hope to bring forward new medicines to improve the healthspan of people with devastating cardiovascular and neuromuscular diseases as are rooted in the etiology of muscle function. Our expertise is in muscle biology and translating that to a new pharmacology. On this slide, you see depicted all of those drug candidates that have emerged from our own research in muscle biology over 20 years, with focus to those that are activators or inhibitors of muscle contractility or the mechanics of muscle performance. In particular, advanced aficamten, now pending review for potential approval by the US FDA, as well as in China and in Europe for the potential treatment of obstructive hypertrophic cardiomyopathy. We will have more to share, I'm sure, about that.
Also, as we're developing it for non-obstructive HCM, and also, as you see depicted here, an activator, omecamtiv mecarbil, an inhibitor, CK-586, also of cardiac myosin. Somewhat uncommon, three compounds, modulators of one molecular target, validated for new pharmacology and has formed the cornerstone of our business strategy to build a franchise commercially that leverages our expertise in R&D. All directed to the same concentrated customer segment. We also have other compounds in our pipeline, earlier stage, and over time, we'll have more to say about those. That's where we are. This is where we're going. This is our vision 2030. Cytokinetics is committed to advancing these potential new medicines to patients. As we expect to have at least two of these medicines approved for patients over the next several years, we're building out our commercial business as a forward-integrated company. That's not new.
That's always been our intention. As we see to 2030, we hope to bring that forward to over 100,000 patients globally with our own footprint commercially in North America and Europe, leveraging partnerships in other territories, including, as we recently announced last year. We expect to do that at the same time we maintain our ingenuity and innovation, expanding our pipeline, expanding our interest in muscle biology beyond small molecules, and also maintaining the same commitment to culture and values and purpose, and especially addressing where we can inequities in care. Ours is a company that's always thought about being good stewards of capital, and in particular, thinking smartly about how we access and deploy capital. We currently have, as reflected in our Q4 earnings call, over a billion dollars, $1.2 billion approximately, in cash and cash equivalents on the balance sheet at the end of December.
You can see we also have access to quite substantial additional capital at, we would argue, lower than market cost of capital for a company in our peer group. We can draw on that to support our continuing business imperatives. We do believe we've been good engineers of the financials to support the business we aspire to build. With that, you can see here depicted on this slide quite ample news flow, call them catalysts, if you will, for what could be increasing shareholder value. I'll be speaking to a few of these, in particular this year, with a potential FDA approval, approval in China, we hope, and also additional clinical trial readouts like MAPLE in Q2 and completing enrollment in Acacia later this year.
You can see ours is a story that is not without its ample news flow to support what we hope can be enhanced and continuing growing shareholder value. First and foremost, priority number one at the company is to bring aficamten to patients. To do that in what amounts to at least 80-90% of where we think the business lies, ourselves in North America and Europe, Europe, albeit in a gated way as we go country by country and based on not just regulatory review, but investing as we get reimbursement approvals. In China, partnered with Sanofi. In Japan, partnered with Bayer. We believe that is the best way to do that, which we can ourselves muster while still leaning on others in areas beyond our own reach.
You can see we've already begun with awareness campaigns directed to healthcare professionals and patients, unbranded as is important and compliant, as we do our part to develop a market that we think is substantially larger than is currently penetrated by the first-in-class cardiac myosin inhibitor. Companies like ours don't always go commercial, and those that do sometimes are successful and oftentimes are not. We've been good students of those companies who are first-time launchers, first-time commercial. What are the things that they do well? As depicted in a recent McKinsey study, we think we've already been doing all of these things well and not recently, but over time, meaning over the last couple of years. We're developing a program comprised of multiple drug candidates in an enduring pipeline and portfolio.
We're investing meaningfully in our launch year and not otherwise, being bashful about investment spending to support subsequent years. We're also thinking about ways we can leverage digital marketing and omnichannel marketing so as to be smart and efficient at how we deploy marketing dollars. We're thinking about ways we can onboard market access sooner. We've been out there talking to payers already for two years, making certain that they're aware of we're coming soon and doing it in a compliant way. We're thinking about how best we can build a custom bespoke experience for healthcare professionals in their offices and also to support patients, knowing that you only get to show up once and build an impression. We want that to be a white-glove service for Cytokinetics in support of healthcare professionals and patients who may benefit from our medicines.
That also speaks to a fit for purpose, customized distribution strategy unique to Cytokinetics and aficamten. We also believe in evidence. Cytokinetics has a reputation for being strong in clinical research, strong and credible, and we're continuing to do clinical studies, some of which you might even think look like phase four studies and accelerating them into this year. MAPLE is a study of aficamten head-to-head with metoprolol, as could be supportive of potential first-line treatment of a cardiac myosin inhibitor as we hope to grow the category. Acacia is a study of aficamten in non-obstructive HCM. MAPLE should read out Q2 this year, Acacia next year. CEDAR is a pediatric study. FOREST is a study that's open-label extension that's already been presented and published with hundreds of patients going out beyond one and two years.
We think all of these together create ample evidence to support aficamten use. My last slide speaks to our milestones this year. Here they are. First and foremost, as relates to aficamten, go to market in the US, prepare for launch in the US, and do that successfully, focusing to uncommon launch momentum and velocity and how do we build category penetration and hopefully, as ultimately may enable best-in-class preferential share. At the same time, read out MAPLE, continue and complete enrollment in Acacia and also in CEDAR, and support omecamtiv mecarbil in phase three, CK-586 in phase two, CK-089 in phase one, and demonstrate that we can continue to innovate and, as could be, advancing multiple medicines to patients over the next couple of years. With that, I'll bring this presentation to a close and invite conversation and questions.
Thank you. Maybe I will start with the commercial launch preparation. PDUFA for September 26. Did the FDA definitively give an answer that there will be ADCOM or no ADCOM?
Thank you for asking. Earlier this week, we issued an 8K just to ensure level-set playing field for all conversations we'd be having here at this and other conferences in Miami. As we wanted to be Reg FD compliant, we communicated prior and at our earnings call that we expected a mid-cycle meeting in March. We have now communicated that meeting occurred. At that meeting, FDA indicated we should not expect an ADCOM. We should expect late-cycle review to occur in June. Therefore, we believe we're on timeline relative to a September PDUFA date. We also came out of that meeting, much as we went into that meeting, with a belief that if aficamten is approved, we should expect a differentiated label and risk mitigation. That's not a different statement than the one we had been making, but purposefully meant to be the same.
Okay. Very helpful. At what point do you think FDA would discuss with you regarding the label, potential label?
I probably shouldn't comment too much more on that meeting, but I do believe that the kinds of conversations we had even before submitting the NDA with regard to label and risk mitigation and those that have continued, including through our recent mid-cycle meeting, all read on label and therefore enable us to make the statement that we did.
Okay. Very helpful. Maybe for Andrew, the pricing strategy, and maybe any thoughts there, what could be the good benchmark out there?
Yeah. There is a product in the market today that kind of establishes a baseline price. I think typical would be to be in proximity of that price. We do not have to finalize our price until we launch, and that is the list price or the WAC price. We will publish that at launch.
Okay. I think in my calculation, Camzyos is about $100,000.
A little north of that, right?
Yeah. Yeah. You think it will be north?
We'll be in proximity to that price.
Okay. WAC price?
They are a little north of $100,000 per patient per year presently in the United States. Andrew and his team are doing a nice job of thinking about how we might ultimately, if approved, also be priced. What I can say is they're doing a fine job. They're pointing to what could be sales north of a billion dollars in the United States and Europe this year. At the same time, as we calculate, and Andrew can elaborate on this, we think that they're demonstrating where there's opportunity for category growth and great awareness, great patient adherence, great repeat prescriptions, but all as is predominantly within a very concentrated number of prescribers. Andrew, you may want to comment.
Sure. I mean, we've talked about business model. This is a specialty cardiology business model. It's a concentrated market. The vast majority of prescriptions are in specialized centers and centers of excellence and academic centers. The community is starting to get involved. There's 35,000 cardiologists or so in the US, and I think we're just, as a category, scratching the surface and 1,000-2,000 physicians writing, the vast majority of writing being maybe the top 5,000-6,000 physicians. The market availability, based on our estimation of EPI, will be probably at least 80% of the market available when we go to the market. We'll be focused on new patients and growing the market alongside BMS as a second option, where we do think we have some levels of differentiation that Robert spoke to and that our 8K spoke to as well.
We're continuing to progress the launch. We did kind of the strategy and design over the last 18 months, and we're really in build phase right now. I can elaborate there if you'd like.
Of course.
In the build phase, we do have an account management team focused on payers, interacting with payers for reimbursement. We've done our pricing and contracting work, which we spoke to. We've made many HEOR publications that kind of speak to EPI, that link Peak VO2 to outcomes, that speak to cost associated with HCM. We'll continue those publications this year. We've sized our field force, drawn territories. We'll start recruiting in April. That takes several months. We'll hire post-late cycle in proximity to approval, making sure that we're ready for launch. We're building a custom patient support program. We've signed our strategic partners, things like Nurse Navigators, Copay Card, strategic partners in both distribution and specialty pharmacy. We're testing our visual aid now. As you saw on a slide, we've launched our marketing campaign in terms of non-branded to raise awareness both to physicians and patients.
have built our digital infrastructure in support of that campaign. Everything is progressing as is. We have our headquarter-based employees. It is just a field force which we will add, and that will be about 125-150 people later this year.
Okay. That's great. Maybe should we, this is September 26th. Realistically, should we see any revenue in 2025, or would that be more likely a 2026 story?
I mean, I think we'll have a full year in 2026 from a revenue point of view. If you consider late September with getting inventory and implementing with two holidays, with getting payers on board, we're really focused more on patient acquisition. We'll bridge patients through our support program. I think you'll start to see there may be a little revenue this year, but the revenue really starting to pick up in 2026.
Okay. And then the launch trajectory, are there any should we use Camzyos as a benchmark the first, say, few quarters, or you think you can do much better than that?
That's the best analog we have of launch trajectory. It's something that Robert and I debate constantly in terms of what does that launch trajectory look like. We're pretty bullish in terms of that BMS has done a wonderful job of creating awareness. Awareness levels are very high. Again, as I mentioned earlier, it's a very concentrated market. It's going to start in the specialized centers and academic centers. We have relationships. So we're bullish that the launch trajectory, when I say launch, I'm speaking 12-24 months, that we will do at least as good, if not better, than BMS.
Just to add, I think Andrew's right. Cardiovascular launches are somewhat of a unique animal. This one has shown with BMS and Camzyos similar trends. They go linear for what amounts to sometimes two or more years before they start to go exponential or hyperlinear. In our case, with this being potential next-in-class, there are good proxies and comps that one can look at in similar specialty markets for what may be possible.
Okay. Why are the first two years linear and become exponential after two years?
I myself have been part of many cardiovascular launches, but we've studied the last, say, 8-10 cardiovascular launches. In general, it's 2-4 years linear to get a high growth. I think there's several reasons for that. One is it takes a little bit of time for payer uptake. Generally, you start with a sub-specialty, if you will, within a specialty in a market like cardiology. In this instance, specialized centers, and then you grow out to the community. That growth out to the community in terms of expanding prescribers, building awareness, getting on board, educating physicians, having them prescribe meaningfully beyond a few patients to really penetrate the market.
I think it's a combination of payer adoption, physician adoption, patient adoption that collectively, once that experience collectively is there, you start to see if the experience is good and meaningful in kind of the balance of safety efficacy with reimbursement. That's when you really start to see a meaningful change. I think a second player in the market certainly helps. Usually, you do see categories grow faster when you have multiple companies engaged with multiple medicines.
I was involved in my first commercial cardiovascular launch in 1985, my second one in 1998, and there are many others that have occurred in between. It's rare that a first-in-class drug becomes the category leader in cardiology. It's oftentimes a second-in-class that becomes the category leader, and oftentimes, as is determined by safety, even more sometimes than efficacy. At the same time, what's somewhat unique to cardiovascular drug launches relative to others is the tail is much longer. It takes while longer to get to peak sales, the tail is longer. You see this, for instance, classically with drugs like Eliquis that Andrew co-led as he was then at Pfizer or Entresto.
If you look at how Novartis has done a nice job with Entresto, I remember many on the street questioning whether it was ever going to get to the forecasted sales of $4 billion-$6 billion for Entresto, and it blew way past that now as they've done a nice job with Entresto. That is sort of a story amongst cardiovascular drug launches that repeats itself pattern after pattern.
I think the other thing that gives us confidence of a successful trajectory, not only if we get approved with SEQUOIA in September, as Robert showed, MAPLE, which is a head-to-head study in the same population, will top line in the second quarter. If that's positive, we would be adding that about a year after launch. A year after that, a case shall hold different population. So in a way, this is a portfolio and a pill that have three data sets with two populations in a very, very short time frame that should hopefully accelerate launch beyond just centers of excellence.
The last few minutes, I want to ask MAPLE, since you also mentioned this is next quarter. Maybe the expectation there, maybe share with us the initial study design thoughts and how confident you think that that will be a positive study.
Yeah. Maybe I'll talk about it from the clinical standpoint if you want to talk about it from the commercial.
Sure.
MAPLE is a study that borrows from learnings, or learnings, I should say, from SEQUOIA. In SEQUOIA, the first pivotal study of aficamten in OHCM, we noted that patients, whether they were on beta blockers or not as background therapy, they in both cases saw increases, clinically meaningful increases in change from baseline Peak VO2. That was a wonderful thing to see for aficamten. In the continuing open label extension study, physicians who have this discretion were increasingly comfortable taking patients off of beta blockers, which absent evidence became a first-line treatment, but for which there's never really been a study conducted with them in this population to test effects on Peak VO2. As that was occurring in the open label extension, it was clear that patients were still benefiting and physicians were still quite comfortable. We thought, why not do a head-to-head study, aficamten versus metoprolol?
One could argue it may not be a fair fight, but for which we do believe that aficamten has demonstrated effects with and without beta blockers in this population of OHCM patients. There are reasons to believe that beta blockers may be neutral to negative when studied this way in this population. This is going to be a real clinical study to test that hypothesis, and we're looking forward to seeing those data in Q2.
Yeah. I mean, quickly, MAPLE, from a commercial point of view, a second data set gives more assurance to especially community cardiology around efficacy, safety as confirmatory to what was seen in SEQUOIA. It should grow the market, influence guidelines in terms of first-line therapy. Based on our research, expecting that MAPLE for the OHCM category would expand category penetrations of more patients treated than would have otherwise, as well as expand share preference in favor of aficamten given that second data set.
Okay. Great. Thank you very much. We look forward to all the updates later this year.
Thank you very much.
Thank you.
Thank you.