Cytokinetics Inc. Citigroup’s Biopharma Back to School Summit 2026
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Welcome again to Citi's Biopharma Back to School Summit 2026. I'm Eric Joseph, a senior biotech analyst with Citi, and our session this afternoon is with Cytokinetics. I'm delighted to be joined by Robert Blum and several members of the Cytokinetics team. Thank you all for joining us today. Rob, just before hopping into Q&A, I could have you start out with some high-level remarks, and then we'll go from there.
Thank you, and thanks for inviting us to participate in this Back to School event. It's our first opportunity to interact with investors in person after returning from Munich, where data from ACACIA-HCM were presented to a very large congress hall, very well-attended, and positive reception on the part of cardiologists there attending that conference in Munich. Those results from ACACIA, we believe, provide a very important milestone and catalyst for what could be additional value creation that may come our way, as that could be data incorporated into a submission for FDA and other regulatory authorities' review and potential approval, and ultimately enable MYQORZO to be the first cardiac myosin inhibitor approved across the full spectrum of HCM patients. Why does that matter? Because we're already demonstrating with our commercial launch of MYQORZO in OHCM that it is rapidly ascending, and where we've pointed to, by the end of the year, we expect to see preferential share of new prescriptions, something quite unusual for a next product launched in a category that its predecessor had already been for four years.
I do think the market is embracing aficamten for the properties that we engineered into it, and the business is off to a great start with a commercial launch we're very pleased with that continues to exceed expectations. We're NHCM to factor into the label together with OHCM. We believe that redefines the way of treatment of patients with HCM, and we're very excited about that. I expect we'll talk a lot about aficamten and MYQORZO, OHCM, and NHCM today.
As time permits, hopefully, we can also speak about our financial picture and our outlook for the pipeline.
Great. Yeah. Well, hopefully, we'll touch on all of those points. Let's pick up from ESC and full presentation of the ACACIA results. Actually, let's take a step back and just think about the non-obstructive HCM market. If we're looking at estimates, the prevalence would seem to be roughly an even split between obstructive and non-obstructive disease if you're looking at the total HCM market. But non-obstructive disease tends to have a lower rate of diagnosis. Can you speak a little bit to why that's the case and whether that has anything to do with how prominent symptomatology or how symptomatic patients often present with an HCM?
Sure. To my right are colleagues, Tricia, who leads U.S. marketing, and Dan, who oversees clinical research, and maybe I'll ask them both to comment on that.
Yeah. Thanks. Can you hear me? Is this on? Okay. Yeah.
Yeah. Thanks. Dan Jacoby, clinical research over here. Yes. This is, yeah, Dan Jacoby, clinical research here at Cytokinetics, and I'm also a hypertrophic cardiomyopathy physician. For many years, this was a major part of my practice. I ran clinical trials, and I still have a small HCM clinic. So, I think I can kind of say what we're seeing is an evolution in the understanding of HCM as therapies come online and as the potential for treatment comes online. In fact, my practice when I was practicing, I had over 1,000 patients, and I was more 70/30 non-obstructive to obstructive because we didn't have an active myectomy center. I think Tricia can speak more knowledgeably to what we're seeing currently in the community, but I think we're still trying to understand the full scope of how this disease presents.
It's been years of collecting data from myectomy centers and specialty centers that have prioritized the evaluation of obstructive patients. The second part of your question is really an interesting one, which is: how do you know when an HCM patient is symptomatic? How do you diagnose them? Are they harder to diagnose? Where are they in the community? What we're seeing is that physicians who take care of large numbers of cardiology patients have these patients in their practice. They're actually not that hard to diagnose. But because there's no specific treatment right now, other than just beta blockers or calcium channel blockers, they're just sitting on them, and these are not the typical patients that end up getting referred to these high-volume practices. But in fact, there's a number of them, and when you talk to individual practitioners, they say, "Oh, I have 10 in my practice.
I have 12 in my practice that I'm just sitting on." As we see therapy come online that's effective, aficamten come online that's effective for these patients, it becomes available in the commercial setting, there's going to be a shift, and these practices are going to start to really look at how they diagnose and treat these patients. I think that's going to lead potentially to a shift in them wanting to engage more with this treatment rather than refer out. At that point, I'll hand it over to Tricia and see what your thoughts are.
Yeah, I think what Dan really hit on was the evolution that we're anticipating to happen within the marketplace, and how HCM is being treated, and where HCM is being treated. We have seen over recent years there has been a shift even in the epidemiology and the understanding of the diagnosis rates for HCM, for O and for N. I think historically there was an assumption that there was more OHCM patients, whether that was a 60/40 or 70/30 split. But in more recent years, with the recent data that we have available to us, we see that it's more of a 50/50 split. Of that 50% that is NHCM, a large proportion of those patients would be viable for a CMI treatment based on what we understand today. A significant opportunity exists within the NHCM space.
I do think we'll continue to see evolution in the space in diagnosis rates, as well as how these patients are being treated once there is a medicine that is available for NHCM.
Coming back to the full phase III data themselves, can you talk a little bit about what the clinician feedback has been now since the broader clinical profile has been presented, and has it all been enthusiasm? Any reservations, and what data points, I guess, are resonating most with the treating community?
Yeah, we had an opportunity to engage with a number of physicians following the full data readout at ESC, and the receptivity is largely positive. There is a lot of excitement, a lot of recognition that prior to ACACIA-HCM, there were no trials that successfully demonstrated superiority. So to have the first positive NHCM trial and the hope for the first medicine that could be available for NHCM was largely communicated back to us around the anticipation to be able to treat 50% of their HCM population.
Just as we got to see more of the safety profile at ESC, I want to just pick up on the slight imbalance in heart failure events that were observed in the study. Despite them only occurring in a small minority of subjects, this may have given investors a little bit of pause. Were there any common characteristics in these patients that might have predicted for their heart failure risk or being at higher risk of heart failure? More broadly, can you help put these SAEs in perspective? How significant are they, and how would they be managed in the real-world context?
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