Edited Transcript of MYOK earnings conference call or presentation 11-Nov-19 1:30pm GMT – Yahoo Finance

SOUTH SAN FRANCISCO Dec 2, 2019 (Thomson StreetEvents) -- Edited Transcript of MyoKardia Inc earnings conference call or presentation Monday, November 11, 2019 at 1:30:00pm GMT

MyoKardia, Inc. - SVP of Clinical Development

MyoKardia, Inc. - SVP of Medical Sciences

MyoKardia, Inc. - Senior Director of Corporate Communications & IR

MyoKardia, Inc. - President, CEO & Director

Wedbush Securities Inc., Research Division - MD & Head of Healthcare of Equity Research

Wells Fargo Securities, LLC, Research Division - MD and Senior Biotechnology Analyst

Ladies and gentlemen, thank you for standing by, and welcome to the MyoKardia AHA 2019 Data Call. (Operator Instructions) I would now like to hand the conference over to your speaker today, Michelle Corral. Thank you. Please go ahead, ma'am.

Michelle Corral, MyoKardia, Inc. - Senior Director of Corporate Communications & IR [2]

Thank you, Shannon. Good morning, and thank you for joining us for today's call. I'm Michelle Corral, MyoKardia's Head of Corporate Communications and Investor Relations. Today, we will be reviewing 2 sets of data: top line data from our Phase II MAVERICK-HCM study of mavacamten in nonobstructive HCM population and the 48-week results from our PIONEER open-label study of mavacamten in obstructive HCM patients.

On the MyoKardia website, we have posted the 2 press releases issued this morning. Leading today's call is MyoKardia's, CEO, Tassos Gianakakos. Tassos is joined today by Jay Edelberg, our SVP of Clinical Development; and Marc Semigran, our SVP of Medical Sciences. Following their prepared remarks, we will open the lines for Q&A.

As a reminder, the information discussed during this call will include forward-looking statements, which represent the company's view as of today, November 11, 2019. We undertake no obligation to update or revise any forward-looking statement to reflect new information or future events, except as required by law. Please refer to today's press release as well as our filings with the SEC for information concerning risk factors that could cause actual results to differ materially from those expressed or implied by these statements. I'd like to now hand the call over to our CEO. Tassos?

Tassos Anastasios Gianakakos, MyoKardia, Inc. - President, CEO & Director [3]

Hi, everyone, and thanks for joining us today. It's an important day for our mavacamten program and for where MyoKardia is heading in HCM and beyond with our precision medicine strategy. Mavacamten continues to generate robust clinical responses in patients with HCM, as evidenced by a growing body of data. So this morning, we'll be going over new data from 2 studies that continue to paint an increasingly exciting picture for mavacamten's role in HCM patient care. First, we'll discuss top line results from our Phase II MAVERICK study in patients with nonobstructive HCM. MAVERICK is one of the largest and most comprehensive studies conducted to date in this patient population. The successful study has provided us a treasure trove of data and new insights about this population, whose therapeutic treatment options are virtually nonexistent. We'll also review the 48-week data from our PIONEER-OLE study. After one year of chronic treatment with a once-daily pill, we're seeing sustained and durable improvements among all patients in the study as well as evidence of some remarkable changes to the structure of the heart.

On behalf of the company, I want to thank the patients, investigators and site staff, whose dedication and commitment made these studies a success. We're going to be covering a lot of new information on this call, which we'll try to do efficiently with some brief headlines before we get into the details, starting with MAVERICK.

MAVERICK'S primary study objective was demonstrating safety and tolerability we've achieved. In addition to demonstrating safety, we identified a patient profile with diastolic dysfunction that we believe most likely to achieve benefit from mavacamten. This is a big deal. It's what MyoKardia was formed to do. To give some perspective, 3 million people in the U.S. have diseases of diastolic dysfunction, referred to as HFpEF, who historically have been addressed as a single group and managed in an undifferentiated way with no approved therapies. With data emerging from MAVERICK, we believe we can now subtype these patients, both those with HCM and those with HFpEF. As a result, we'll be advancing Mavacamten's development both in nonobstructive HCM as well as in our first expansion beyond HCM into a well-defined subgroup of patients with HFpEF in a precision-efficient fashion.

Turning to the PIONEER-OLE study. At one year of doses, mavacamten safety and tolerability profile remain solid, and the treatment benefits are sustained, gradient is reduced, EF remains above normal with little change from baseline. And patients are feeling better, and we're seeing a wide range of biomarkers moving towards normal levels, supportive of important improvements in their hearts. Beyond our decisions to advance in nonobstructive HCM and expand into HFpEF, the data and insights from both studies have also increased our confidence in EXPLORER in a number of ways.

First, the safety and tolerability in MAVERICK was very consistent with what we've seen to date. MAVERICK also gave us an opportunity to confirm our expectations for anticipated placebo response and further validate our powering assumptions in EXPLORER. The evidence of long-term benefit and potential for mavacamten to slow or reverse HCM's progression in our obstructive patient population continues to grow. And the diastolic disease benefit observed in MAVERICK, adds to our belief that we will see significant clinical benefit in EXPLORER. I know we're excited to get into it. So let me hand the call over to Jay, who will take us through the top line data from our MAVERICK Phase II trial in nonobstructive patients. Jay?

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Jay Edelberg, MyoKardia, Inc. - SVP of Clinical Development [4]

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Thank you, Tassos. And good morning to everyone. I'm excited to be here today to talk about the top line results from MAVERICK. Hypertrophic cardiomyopathy, or HCM, is a chronic, progressive disease that changes over time. While some people with HCM might not experience much in the way of symptoms, others struggle with ordinary activities and far too many develop atrial fibrillation, stroke, heart failure and sudden cardiac death. Both the obstructive and nonobstructive forms of the disease, genetic mutations that lead to excessive contractions are the same, but the ways that the hypertrophy or thickening of the heart muscle manifest can vary.

In obstructive patients, we typically see significant thickening of the septum near the mitral valve that results in an obstruction to blood flow in the left ventricular outflow track.

In nonobstructive HCM, the thickening of the heart muscle tends to be more symmetric and the disease manifests are driven by the inability of the left ventricle to expand and fill with blood. MAVERICK enrolled a total of 59 patients representing one of the largest controlled clinical trials in nonobstructive patients ever conducted. Considering the unmet medical need and prior limited clinical trial data in this population, MAVERICK represents a groundbreaking opportunity for mavacamten and our patients. Mavacamten -- sorry MAVERICK is a dose-ranging study. Participants were assigned to 1 of 3 groups, each either to receive placebo for once-daily doses of mavacamten targeting 2 EXPLORER concentrations, centigram 200 or 500 nanograms per ml. By design, some patients were above and others below target concentrations in order to provide a full picture of the right dosing approach. This is notable because prior to MAVERICK, there was no biomarker equivalent of the obstructive gradient to guide dose titration.

Based on our results, we think we have uncovered the markers to identify and dose these patients, offering us an important opportunity to make meaningful difference in how nonobstructive HCM patients are treated. The entry criteria for MAVERICK were a diagnosis of nonobstructive disease, as defined by echocardiographic guidelines; a baseline ejection fraction of at least 55%; an elevated NT-proBNP and symptoms; New York Heart Classification Class II or III. Baseline characteristics, such as gender, genetics, age, fitness level, degree of symptoms were evenly distributed between the active and placebo arms. A threshold for diastolic dysfunction was not required for enrollment. One of the first things we noted in this study was a spectrum measures of diastolic dysfunction amongst the patients. Indeed, only about half the patients had elevated filling pressures and rest is measured by elevated E/e' prime.

Now let's turn to the data. First and foremost, mavacamten appears to be well tolerated. Adverse events noted in MAVERICK were consistent with prior studies of mavacamten and the majority of AEs were mild or moderate and reversible. Serious adverse event rates were twice as high amongst the placebo patients compared to the 2 active treatment groups combined. The most serious adverse events in the study were cardiac related. There were 5 patients in the treatment -- with on treatment reductions in ejection fraction below the prespecified protocol levels. These were all transient events and all patients recovered without harm. We are pleased with this safety profile.

We have now added 40 patients treated with mavacamten for over 16 weeks to our safety database, more than double what we had coming out of our PIONEER Phase II study.

Turning to the readout of our exploratory efficacy endpoints. When we look at all patients valuable for treatment effect, there was no meaningful separation between the active and placebo arms for peak VO2, New York Heart Association Class or echo measures of diastolic parameters. There was a dramatic change in several biomarkers of cardiac stress for patients receiving mavacamten versus placebo. Specifically, we saw a 50% reduction in NT-proBNP levels across both treatment cohorts versus placebo, which was highly statistically significant. As early as week 4, we observed a separation from placebo, even amongst the patients treated with the low concentration. The NT-proBNP reductions were sustained through week 16. This is an important result given mavacamten's mechanism of action and relieving the excessive contraction in the heart. This is where we wanted to be to see a treatment effect and tells us that our drug is working as hypothesized.

As a reminder, NT-proBNP is an important marker of cardiac wall stress and elevated NT-proBNP levels are associated with increased rates of heart failure-related death, hospitalization and progression to end-stage disease and stroke. Equally important was the observation of clear clinical benefit in 2 patient subgroups, a prespecified subgroup with high-scope morbidity and mortality associated with their disease and amongst those with greater degree of diastolic dysfunction.

We have long hypothesized that one reason mavacamten would work well in nonobstructive HCM is due to the positive effects and diastole compliance and filling we have seen in animal models and in the clinic with obstructive HCM. As could be expected with mavacamten, those with elevated left ventricular filling pressures, a marker of more pronounced diastolic disease, we saw trends differentiating these patients on treatment from those on placebo across the majority of our exploratory endpoints. Peak VO2, NT-proBNP, E/e' prime and other diastolic measures all improved in the active treatment group. In a prespecified population of high-risk HCM patients treated with mavacamten resulted in similar trends of clinical benefit, amongst multiple parameters of symptoms, function and diastolic improvement versus placebo.

What are these data telling us? The wide range of concentrations teaches us where we want to be in a starting dose in the nonobstructive for the diastolic disease populations moving forward. We now have a strategy for dosing based on clinical parameters just like we are doing in EXPLORER. Differentiating activity amongst the subgroup now informs potential enrollment criteria and endpoints for future clinical trials. We expect an identification of these patient disease markers will also be key in developing effective therapies for HFpEF subpopulations. This is precision medicine in action.

Next, we're going to continue our analysis of MAVERICK results and are preparing abstracts to be submitted to upcoming medical scientific congress. We are aiming to present the additional results from this study by the end of the first half of next year. Based on these results, our team is excited in developing and optimizing protocols for our next set of studies. We're looking forward to discussions with the FDA in the first half of next year to discuss potential paths to registration in nonobstructive HCM. We're also excited about launching our first Phase II study in patients with HFpEF, who, we believe, can benefit from Mavacamten's mechanism of action. And with that, I'll turn the call over to Marc Semigran, who will walk us through the highlights from PIONEER-OLE 48-week data that will be presented at the AHA next week.

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Marc Semigran, MyoKardia, Inc. - SVP of Medical Sciences [5]

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As someone who's treated these patients for many years, hearing the MAVERICK data is really exciting. As it's a potential game-changer for patients with cardiovascular disease. This morning, the embargo lifted on the data being presented at the upcoming AHA scientific sessions. There, Dr. Steve Heitner, Director of the OHSU HCM program, will present new findings that have emerged from our PIONEER-OLE study. Not only is this study providing us a continued look at Mavacamten's long-term benefits, it is giving us a glimpse into what may be happening in EXPLORER. I'm excited to describe how safety, durability and consistency of effect epitomize the results we're seeing in the 48-week OLE data. 12 patients are included in this dataset and remain on the study. The marked reduction in LVOT obstruction reported at 12, 24 and 36 weeks is maintained at 48 weeks. The safety profile remains unchanged. Patients are feeling better and across multiple and diverse biomarkers, we're seeing signs of their hearts regressing towards a more normal state.

In all 12 patients, LVOT gradient was consistently and meaningfully reduced, whether measured at rest during the Valsalva maneuver or after exercise. A particular note is the postexercise gradient, which decreased from 128 millimeters of mercury at baseline to 40 at 48 weeks. This is new information as it is the first report of Mavacamten's effect on postexercise gradient in the OLE. It is a dramatic clinically and statistically significant indication that we are continuing to alleviate obstructions to blood flow with mavacamten therapy.

Obstructive HCM is a disease that's most apparent with activity. Patients will be able to supply their peripheral organs with adequate nutrients and oxygen when they need it the most while exerting themselves or with other forms of stress. Importantly, LV ejection fraction remained above normal in all patients at all-time points. Mavacamten is restoring to normal the frequency of myosin binding to actin and force generation in the heart. This is a direct improvement from the constant cardiac hypercontractility these patients have as the cause of their disease, the equivalent of being constantly in fighter flight mode.

The beneficial effect of mavacamten normalizing the heart's ability to contract at rest and to increase appropriately when necessary is an excellent example of the results of MyoKardia's precision approach to cardiovascular disease. Mavacamten was well tolerated with no drug-related adverse events observed and no dose adjustments needed. Of note, no cardiac AEs have been associated with treatment over the entire OLE study. Patients in the OLE study report improvements in how they're feeling and their ability to function on a day-to-day basis. When we look across several of the study's symptom and function measurements, it is clear that patients on mavacamten are finding durable and meaningful improvements in their symptoms and in the impact of HCM on their daily lives. The majority of patients are now NYHA Class I or asymptomatic. Among the few patients who remain at Class II, other parameters, including LVOT gradient and NT-proBNP are showing persistent improvements. At week 48, we also asked patients to complete the Kansas City Cardiomyopathy Questionnaire, commonly referred to as the KCCQ. Patients' responses to the questions are converted to a scale of 1 to 100, with a higher score being better and an increase of 6 or more being considered clinically significant. KCCQ scores rose from a mean of 74 at baseline to 87 at 48 weeks, a 13 points improvement.

We've previously presented compelling biomarker data as well. These have included NT-proBNP and E/e' prime, which you've heard a lot about from Jay in the MAVERICK study, also left atrial volume a measure of filling pressure that is associated with an increased risk of atrial fibrillation. Each of these measures, NT-proBNP, E/e' prime and left atrial volume, have decreased by statistically significant amounts approaching or achieving normal levels. While we view all of these data points as exciting signs of the heart failure -- of the heart functioning better, particularly noteworthy is the reduction in the mean NT-proBNP from 1,836 picograms per mL at baseline to only 136 at week 48. By way of context, a normal NT-proBNP plasma level is 125 or lower. These changes in parameters of ventricular filling and cardiac wall stress may indicate that treatment with mavacamten is lessening the stiffness of the left ventricle associated with hypertrophy over time. This would improve the left ventricle's ability to relax and fill with oxygenated blood during diastole.

We are reporting a new finding with this dataset that joins these other biomarkers as an exciting indicator of a change in cardiac structure for the better. The abnormally thickened interventricular septum, which is a defining feature of obstructive HCM, progressively decreases from baseline. Specifically, when measured by echocardiography at baseline, the mean septal thickness in PIONEER-OLE patients was 17 millimeters. After 48 weeks of mavacamten, it was 15 millimeters. It is noteworthy that septal thickness was observed in the OLE to progressively decrease at each time point it was measured. This longitudinal change supports our hypothesis that normalizing contractility with mavacamten therapy in oHCM patients will lead to beneficial changes in cardiac structure. By way of context, a normal septal wall thickness ranges from 6 to 10 millimeters. According to the AHA/ACC guidelines, HCM is diagnosed based on a maximum wall thickness of at least 15 millimeters. Wall thicknesses of 13 to 14 are considered borderline.

In addition, the risk of sudden cardiac death in HCM patients has been observed in the literature to increase progressively as wall thickness increases above 15 millimeters. As data emerged from our other studies, we will continue to monitor these and other biomarkers to enable us to build a more complete picture of the long-term effects of mavacamten on the heart and its potential for slowing disease progression and reverse remodeling. The prospect of making a meaningful positive impact in the lives of patients is what brought me to industry. I am exhilarated by the improvements associated with the mavacamten treatment that we have observed in our HCM patient symptoms, physiology and biomarkers. The directionally consistent and durable results seen in the OLE study across diverse parameters are nothing short of remarkable. Tassos?

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Tassos Anastasios Gianakakos, MyoKardia, Inc. - President, CEO & Director [6]

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Thank you, Marc and Jay for the data review. So before we open the line up to your questions, let me summarize what these data mean for the big picture at MyoKardia and how it sets up our company over the next 6 to 9 months. We're moving mavacamten forward in 2 patient segments in addition to obstructive HCM. As Jay mentioned, MAVERICK gives us confidence to advance in nonobstructive HCM and in a subset of HFpEF. Much like PIONEER guided our design of EXPLORER, MAVERICK has generated the data we needed for nonobstructive patients, including identifying the analogous biomarkers to LVOT gradient. We're ready to finalize our nonobstructive HCM plans and aim to discuss them with the FDA in the first half of next year.

In parallel, we'll be taking our first thoughtful step into an adjacent subgroup of HFpEF patients roughly estimated to be about 10% to 20% of all HFpEF, bringing our precision medicine approach to one of the largest remaining areas of medical need. Our confidence in the successful EXPLORER trial has also gone up a lot with today's data. Our safety database just grew substantially, and mavacamten continues to be consistently well tolerated. We've looked at the placebo arm in MAVERICK and found that our estimates for placebo response in EXPLORER is spot on. So we're feeling really good about EXPLORER's design and powering assumptions. And our confidence in Mavacamten's efficacy in EXPLORER is supported by both studies today. The OLE, of course, in the same patient population and the subgroup data from MAVERICK tells us mavacamten is improving left ventricular compliance enabling the heart to fill at lower pressures, which should help obstructive HCM patients as well. Finally, while early, the evidence is mounting that mavacamten may be enabling structural changes in the heart that could ultimately be disease modifying, with multiple and diverse markers all improving and moving in concert towards normal.

So let's take a look at where MyoKardia will be in 6 to 9 months. For mavacamten, we'll be through EXPLORER and moving towards our first NDA filing in obstructive HCM. The SRT study will be underway looking at obstructive HCM patients, who have been referred for surgery. The MAVERICK insights you heard about today will have positioned us for registration path and nonobstructive HCM, and we'll have started a Phase II study in a targeted HFpEF, our first non-HCM indication with mavacamten.

For 224, we expect to be reading out data from our Phase I program, and we'll be moving into Phase II study in HCM and/or targeted HFpEF. And for MYK-491, we'll be underway in a Phase II genetically defined DCM population and gearing up to drive a precision registration program in patients with diastolic dysfunction. And all the while, we'll be mining the proprietary clinical insights from our studies, including MAVERICK and EXPLORER, which will enable important patient profiling and novel research programs. It's really an exciting time for us here at MyoKardia, seeing the vision come into focus, which our team is incredibly motivated to make a reality. So with that, operator, let's open the lines up for questions.

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Questions and Answers

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Operator [1]

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(Operator Instructions) Our first question comes from Anupam Rama with JP Morgan.

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Anupam Rama, JP Morgan Chase & Co, Research Division - VP and Analyst [2]

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Just on the EF reduction observed here in MAVERICK, I believe in the PIONEER study, you saw EF dip below 50% at drug concentrations that were 1,000 nanograms per ml. And here in MAVERICK, you chose drug concentrations that are lower, but it still appears that 5 patients kind of dip below 50%. How are you thinking about EF here heading into the 2Q EXPLORER results? Was there anything particular about these 5 patients' baseline characteristics worth noting when it comes to EF?

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Jay Edelberg, MyoKardia, Inc. - SVP of Clinical Development [3]

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So in this study, it was a dose-ranging study. So we specifically pushed the dose based solely on concentration without regard to any clinical response. And so therefore, by definition, we expected some patients to be at upper levels and that their ejection fraction may come down. And that's exactly what we learned. Though what this tells us now coming out of MAVERICK is that we have physiologic, we have biomarkers that we can follow to guide our dosing in our future studies, just like we're doing in EXPLORER.

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Tassos Anastasios Gianakakos, MyoKardia, Inc. - President, CEO & Director [4]

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And Anupam, just to add, what we're seeing here really lines up with all of our modeling, our preclinical modeling, our earlier studies around PK and effect on EF. And the thing to keep in mind, too, because your question was specifically around thinking about EXPLORER. Look at that OLE data, the EF is really hasn't moved at all for these patients. So dosing in the obstructed patients, we feel really good about, and MAVERICK is supporting our strategy to dose in EXPLORER. And separately, as we think about moving forward in the nonobstructed patient population, we feel really good that this data has given us the ability to identify a well-tolerated dose that's going to keep nonobstructed patients in the normal range.

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Operator [5]

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Our next question comes from Marty Auster with Crdit Suisse.

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Martin Douglas Auster, Crdit Suisse AG, Research Division - Research Analyst [6]

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I had a couple. I was wondering if you could provide more detail on the placebo group performance in the ITP versus baseline on some of the functional endpoints you looked at NYHA and VO2 max. And then also, I was wondering if you could maybe provide a little more information on the subgroup with greater diastolic dysfunction where some efficacy measures were observed. What's the size of that subgroup? And how was that defined?

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Tassos Anastasios Gianakakos, MyoKardia, Inc. - President, CEO & Director [7]

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Marty, so let me hit the placebo response rate. Yes, this was a key takeaway that increased even further our confidence in EXPLORER. So we had modeled an estimated 25% placebo response. We're looking at the responder definition in EXPLORER, and we saw 21%. And so this supports the assumptions in EXPLORER validating the power assumptions that we've got in that study. And we're feeling even better about it after MAVERICK. In terms of the subpopulations, this, I have to say, I'm glad you asked that question, it is fundamental to our strategy and to MyoKardia's competitive advantage. This idea of profiling and subtyping patients' clinical trials that we're doing with targeted drugs like mavacamten, 491, 224, they're generating such important information, often proprietary information that we got to be very careful about disclosing for competitive reasons. We've learned a lot about this patient population from MAVERICK. We're going to apply that knowledge going forward to nonobstructed development and to HFpEF studies next year. Some of the things that we're learning from these populations and from the biomarker and imaging profiles are, of course, how to dose, how to select them, what measurements are going to matter the most, how to dose adjust to a physiological effect, which is really important. In MAVERICK, we dosed the PK. So it's all really, really good. Jay, maybe you want to paint a clinical profile of these patients a bit.

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Jay Edelberg, MyoKardia, Inc. - SVP of Clinical Development [8]

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Yes. So we -- so that we actually looked at 2 different subgroups. One, we found, very interestingly, that many of the -- that half the patients that we enrolled actually had, at rest, lower levels of E/e' prime. So focusing on those who are actually abnormal, we're really, really pleased to see that those parameters were able to start to normalize over the 16 weeks. So we treat out further. There is opportunity potentially for the others. Similarly, as Tassos mentioned, we've been able to identify a high-risk population, the details of which we'll disclose later on, which got a consistent benefit as well with improvements in function and physiology and biomarkers as well.

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Martin Douglas Auster, Crdit Suisse AG, Research Division - Research Analyst [9]

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Maybe you could clarify, Tassos, on the comment you made about a 20% response rate in the treatment in the placebo group. What were the treatment group response rates? Was there any dose range? And what is the mean VO2 change from baseline in the placebo group? If you could offer any more detail on any of those questions.

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Tassos Anastasios Gianakakos, MyoKardia, Inc. - President, CEO & Director [10]

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Yes. So we -- in these groups that have -- appeared to have more congestion, whose symptoms are really impacting and limiting their function that Jay just described, we saw solid trends in Peak VO2 and New York Heart Association, they would qualify for a responder analysis. The magnitude of those changes were similar to what we saw in PIONEER actually. But many other measures in those groups improved as well. So we'll be thinking about that as we consider the design of the next study for sure.

In MAVERICK, unlike EXPLORER, wasn't designed to show optimal effect on the EXPLORER responder definition, as we all know. And it's in a different patient population as well with a different dosing scheme. But nevertheless, in these populations, which is why we're really excited, right, is that we're seeing a lot of concordant movements in really important endpoints, including peak VO2 and New York Heart. So really what's great to take away is the placebo response rate, as you've highlighted here; the fact that NT-proBNP is dropping in everybody, and that we're able to really frame out how to think about dosing, patient selection and measurements of the right markers for these groups with diastolic disease. So we're talking about being able to help patients with diastolic disease, which is really awesome.

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Operator [11]

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Our next question comes from Ritu Baral with Cowen.

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Ritu Subhalaksmi Baral, Cowen and Company, LLC, Research Division - MD & Senior Biotechnology Analyst [12]

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I just want to clarify a little bit about those 5 patients that the ejection section drops. Were -- can you confirm that they were at the highest, essentially, plasma exposures in the range that you were looking at, even above the 500 target? And what did you do to resolve them? You said they were transient. Did you dose reduced these patients or did that resolve on its own?

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Edited Transcript of MYOK earnings conference call or presentation 11-Nov-19 1:30pm GMT - Yahoo Finance