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Introducing Leading Through Setbacks with Cytokinetics’ Robert Blum from Clinical Trialblazers. Follow the show: Clinical Trialblazers In this episode of Clinical Trial Blazers, host Alberto Grignolo speaks with Robert Blum, president and CEO of Cytokinetics, about his journey from the biology classroom to leading a biopharmaceutical company focused on muscle biology. Robert shares the values that guide his leadership style and how to move forward after failing. DISCLAIMER: Please note, this is an independent podcast episode not affiliated with, endorsed by, or produced in conjunction with the host podcast feed or any of its media entities. The views and opinions expressed in this episode are solely those of the creators and guests. For any concerns, please reach out to team@podroll.fm.
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- [00:03]
Alberto Grignolo
Hello and welcome to Clinical Trial Blazers, a podcast by Parkside Biotech, where we explore the human stories behind breakthrough clinical research. In this series, we take you inside the world of clinical development, bringing you conversations with top voices from biotech. Together, we will explore the pivotal choices, bold risks and remarkable achievements that define these trailblazers legacies uncovering the heart, grit and ingenuity driving the future of scientific innovation. I am Alberto Grignolo, your host on this journey. Whether you're a rising leader in biotech or simply curious about the future of clinical trials, this series is here to inform, inspire, and spark your imagination. Today I'm talking with Robert Blum, President and CEO of Cytokinetics in South San Francisco, a biopharmaceutical company that develops therapies for heart failure, hypertrophic cardiomyopathy, and ALS based on the science of muscle biology. Robert, welcome. Delighted to be with you.
- [01:08]
Robert Blum
Thank you. It's a pleasure to be with you.
- [01:10]
Alberto Grignolo
Let's start from the beginning. I understand that you are a native of Asheville, North Carolina. So I want to ask you, what was it like growing up in the mountains considering that you have moved to the left coast of the United States at some point in your life?
- [01:23]
Robert Blum
So I love Asheville, North Carolina. It is still, to this day my home. It is a little town nestled in the Blue Ridge Mountains, surrounded by environmental beauty. But more importantly, even are the people, people who are good to their core. And I think growing up there afforded me a window onto the rest of my life that frankly, still to this day is very meaningfully impactful. I go back there often. I have dear friends who are there and the place, the music, the culture, everything about Asheville, I think, is defining to who I am.
- [02:08]
Alberto Grignolo
Take me back to when you realized you wanted to pursue the path that led you to where you are today, a biotech CEO. And specifically, was it all very intentional on your part or was it just accidental and maybe serendipitous?
- [02:21]
Robert Blum
So I would say it was very intentional, if not myopic to things that were presented to me from my very beginnings as I came to love science and biology. I've always wanted to pursue the business of biology. I was afforded experiences and have been exposed to mentors who helped refine that thinking. And that was a perfect storm, if you will, convergence of things I was exposed to and people that I became exposed to, all having a shape and influence on who I became.
- [03:04]
Alberto Grignolo
So did you have a vision of actually becoming a CEO of a biotech company?
- [03:08]
Robert Blum
I think so. It's A little difficult to say because I don't know exactly that when I first developed a want to be part of this industry, I necessarily could say I knew what it meant to be a CEO. But the people who were inspiring me were in fact CEOs. When I was a student in high school and I came to love biology and I had a wonderfully influential high school teacher. He introduced me and exposed me to the wonderful things that were happening on the west coast, in California, in the Bay Area, as the biotechnology revolution was occurring. We back then referred to it as recombinant DNA and genetic engineering and things like that. But I came to learn about what people like Bob Swanson, the founder of Genentech, were doing in and around San Francisco, in the Bay Area. And that had everything to do with my want to pursue a career in the business and biology and to launch that in the Bay Area by pursuing my next level academic studies at Stanford.
- [04:28]
Alberto Grignolo
You mentioned Bob Swanson with a degree of admiration, as I heard it. Was he a role model for you and how so?
- [04:35]
Robert Blum
Absolutely. Bob Swanson, for those who may not know, is perhaps the farther patriarch of our industry as the founder of Genentech, somebody who had great impact on not only my professional development and how I think about my career, but also the values that come alongside of that. He was extremely motivated by the power of biology and science and what that could mean for new medicines as well as new companies and a new industry. And I'm very fortunate to have known him and for his having been a participant in our company, Cytokinetics. He was our first angel investor and I had the good fortune of spending time with him until his tragic passing, much too young to a glioblastoma.
- [05:37]
Alberto Grignolo
Very tragic indeed. Do you consider him to have been a mentor to you and perhaps there were other mentors in your life as well that you'd like to acknowledge?
- [05:46]
Robert Blum
Absolutely. He was in fact a mentor for the fact that he also helped develop and pioneer what it means to do business development in our industry. Other mentors included Roy Vagelos, who was also an angel investor in Cytokinetics Series A funding. A former manager, Lee Douglas, who was instrumental in shaping my career as he was one of the early business development executives in biopharmaceuticals and was the first CEO of a company, Core Therapeutics, where I worked immediately after graduate school. But I'd say the most influential person, mentor, coach, inspiration in my life was my father. My father, who was a small business entrepreneur, who himself came to this country with nothing he was a survivor of the Holocaust, liberated from Auschwitz, came to this country to start a new life, and despite having witnessed the worst of human atrocities, had always a very positive spirit, energy, outlook, commitment, high conviction and great heart, compassion and values. And I do believe that he infused in me and my two brothers a love of science and how science for medicine could do so well for the benefit of humanity.
- [07:23]
Alberto Grignolo
What a remarkable role model. What a lesson in life. Thank you for sharing that. You mentioned Roy Vagelos. If I'm not mistaken, he was a very senior R and D executive at Merck. Am I right?
- [07:35]
Robert Blum
Yeah. So Roy was perhaps one of the most important CEOs in Merck's corporate history. I had the good fortune of speaking to Roy just a week ago and we talked about Cytokinetics, which he invested in over 25 years ago. But also we talked about similarities between the company we still aspire to be and what was Merck. Merck a company maybe still is a company that leads with its hearts and minds and values where science has benefited humanity. And there are many examples under Roy's leadership and since then where Merck has done the right thing by peoplehood and how that is good for business.
- [08:26]
Alberto Grignolo
Has that inspired you to do the right thing by people and by patients?
- [08:31]
Robert Blum
I hope so. I think Cytokinetics is a company that also leads with hearts and minds. And we've made lots of decisions along the way in the interest of science for the benefit of people. And not all of them have necessarily in the short term translated to good business, but certainly over the medium to long term have, and we believe over the long arc of the corporate development of our company, we'll be in a good position to have demonstrated how those decisions reward all stakeholders, including patients and all shareholders. I should mention that another important mentor to me who also has inspired in me the importance of mission, culture, purpose and values is Ewing Kaufman. Ewing Kaufman, the founder of Marion Laboratories. And he, perhaps more than any other, demonstrated to me the significance of company culture and how that affords continuity of team, continuity of values and how that translates ultimately from science to clinical research and ultimately to competitive advantage for commercial enterprises.
- [09:58]
Alberto Grignolo
Can we do a little self reflection? You have grown as a leader to the highest levels in the industry. So what type of leader are you? How do you inspire the people you work with?
- [10:08]
Robert Blum
I think it starts with humility and values. I do believe that as I've matured as a leader and as our company has perhaps evolved alongside, we've taken the definition of servant based leadership to New levels. I hopefully am a leader that is an exemplar of of what it means to be team oriented, collaborative and inspirational. Empowering of others to do what they know how to do best and demonstrating that we across the organization can be transparent. It's important that we are all accessing information to be enabling of us to do what we do at our company so we don't use information as a superpower segregated to some and not to others. Ours is an organization and hopefully I as a leader and am an example of this, who can be enabling with the sharing of information and decision making authority to let people do what they know how best to do. And in that way we're all rowing in the same direction. Where I think I still can do more and better as a leader is to be inviting of more criticality of thought, more dynamic tension in order to be enabling of us to always be at our very best. Challenging assumptions. For instance, as a younger company, I think we did that quite well. Certainly that's something scientists do every single day. But as we've become a more mature organization, it's incumbent upon us to be reminded of that being a necessity, an essential to doing the right things all the time and being the best organization we can be.
- [12:19]
Alberto Grignolo
The journey continues, right? Your journey is by no means over as a leader. As you said, turning to the science that you've started to mention, you and cytokinetics have long focused on therapeutics that are based on muscle biology. So why muscle biology? What is the origin of that particular focus?
- [12:36]
Robert Blum
Well, to be quite candid, that was not the original vision of the company. When we started this company in 1998, we were committed to an area of biology called the cytoskeleton, where we believed we could be best suited to pioneer and lead and industrialize that biology for the benefit of new medicines. And our scientific founders were all academic leaders in that space. But we channeled that biology initially into areas focused to oncology. And we were quite successful discovering and developing in the early days of the company new antimitotic drug candidates as could be effective new chemotherapies. But I led a strategic planning process in 2005 and 2006 and around that it became abundantly clear that for us to be a majorly impactful biopharmaceutical company, it wasn't going to come with our oncology programs. They were certainly interesting, but maybe not as compelling around which we could build a platform for leadership in the biopharmaceutical space as would provide enduring value and we redirected our research, taking actually a step backwards in order to ultimately take steps forward in muscle biology. So we frankly re engineered the company. It was a difficult time because it meant we had to say goodbye to certain of our colleagues who were expert scientists and physicians in clinical research in oncology, and refocused the company to muscle biology, a space where we thought we could both pioneer and lead. And it was with a longer term vision, frankly. We saw how companies in areas of bone health and in metabolic syndromes were building adjacent franchises around which we thought if we could pioneer and lead and build a franchise in muscle biology, they would come to our door seeking partnerships because we had a leadership position as would be complementary to their own. And that would enable for us, with non equity dilutive capital, to support the build of a portfolio or pipeline of drug candidates around which we could build the leading company in muscle and muscle biology as would translate to franchise businesses that would be affording of us sustainable and enduring value and we could build hopefully the next great biopharmaceutical company. That was over 20 years ago. And I would argue that we have been achieving everything we set out to do at that time. It's taken us a while, but now in 2025, we're on the cusp of translating that pharmacology and a pipeline of multiple drug candidates directed to muscle to what will be now a new business with application in cardiovascular medicine and also ultimately in neuromuscular medicine.
- [16:14]
Alberto Grignolo
Very interesting illustration of how a strategic change of direction some two decades ago has led you to where you are today. Now, I've had the pleasure of knowing you for over a decade and I know that you have had a long commitment, both personally and corporately, to finding a cure for als. Amyotrophic lateral sclerosis. Why als?
- [16:35]
Robert Blum
So I would say that ALS found us more than we, in fact, with any foresight, pursued our platform in als. But as a company devoted to the mechanics and contractility of muscle biology, it became clear that there were opportunities where augmenting muscle function and with small molecules increasing skeletal muscle force and power and endurance could provide a new therapeutic window to potential treatments of als. This is a most devastating condition, yes, but where patients live their lives and ultimately experience this disease is in the loss of muscle function. Where neurons innervate muscles, muscles become weaker and ultimately patients experience loss of function and activities of daily living ultimately succumbing to the disease because of respiratory muscle loss of function. So we looked at this and thought, who better than us? A company with expertise in muscle biology, we could turn ALS on its head and potentially bring forward new medicines as could be enhancing of muscle function, slowing the loss of function in patients with als.
- [18:07]
Alberto Grignolo
So where are you now in your journey on als? Towards a potential cure.
- [18:12]
Robert Blum
So, unfortunately we tried and admittedly failed. It was perhaps the worst days of my professional career to be acknowledging, especially in front of the ALS patients who so much dependent on us, that we had not delivered a potential new medicine for the potential treatment of als. But it's had indelible impact to the company we are for having traveled that journey with ALS patients, caregivers and physicians, knowing how much they depend on biopharmaceutical innovation and for having met and experienced those patients. It has influenced the company culture and values and how we think about our corporate responsibilities more than anything else. We've gone back to research in terms of als, and we're thinking now about how we can advance new medicines that are focused less to the contractility of muscle for the potential treatment of ALS, and more as relates to energetics, growth and metabolism of muscle as could be affording a new path forward in als. So we have not given up, but we do acknowledge that we're not yet on the cusp of a new medicine in ALS at this point in time.
- [19:39]
Alberto Grignolo
Well, best wishes for achieving a measure of success in that very, very difficult space. For a horrible dise. Let me turn to money, because drug development, of course, is expensive and we all know that all new ventures and established companies need funding. I noticed that in your Q3 2024 press release you boasted cash of 1.3 billion. So clearly there has been a very huge, hugely successful fundraising effort. So I want to ask you, how did you prepare to ask for capital way back then, and did you run into any particular obstacles or roadblocks?
- [20:16]
Robert Blum
Yes and always. But I do believe we've demonstrated tenacity and resilience and more importantly, creativity in the way we go about accessing capital. I think to be a CEO in the biopharmaceutical space is about how does one not only access capital, but deploy capital in efficient ways to demonstrate return on those investments? And even as a company that's pre commercial, we have to constantly be disciplined to think about how we're accessing capital and demonstrating that we can monetize our science and research and development in increasingly meaningful ways for the benefit of investors, whether they be venture capital investors, mezzanine investors, crossover investors, public equity investors, or even more recently, as we've demonstrated, investors in debt capital from the beginning Cytokinetics has set forward to show investors that we can monetize investment in ways that reward them, but primarily through non equity dilutive sources. Cytokinetics, I believe, has set a high watermark amongst peer group companies to demonstrate through business development and corporate development that we can access even more capital along the way through partnerships and collaborations through financial engineering transactions like we've done with Royalty Pharma. I think we've done more deals with royalty pharma than any other company, including more earlier stage deals that's afforded us opportunities to advance medicines, to retain rights to those medicines and to build shareholder value along the way. We talked about our ALS medicines. What we haven't yet talked about is our cardiovascular medicines.
- [22:20]
Alberto Grignolo
That's right.
- [22:21]
Robert Blum
And we have demonstrated through partnerships like originally with Amgen in 2006, more recently with Bayer and Sanofi in 2024 and other deals in between, like with Astellas, that we've been able to access not just capital, but other resources, competencies, skills and experiences to be enabling of with each successive deal, our ability to advance programs and pipeline and mature the company in a corporate development sense, leaning on these deals and the occasional equity financing in order to not only augment balance sheet, but also boost and advance pipeline such that as we have this conversation today, Cytokinetics is hopefully demonstrating with three later stage programs, all as our modulators of cardiac myosin, that we're on the cusp of delivering on the promise of our science for investors, but also for patients, as we hopefully will go to market later this year with our first cardiovascular medicine.
- [23:43]
Alberto Grignolo
Congratulations on such successful fundraising efforts and achievements over the past couple of decades, turning to clinical trials without which one cannot get a drug registered and onto a market. Your clinical trial journey in als, but also in heart disease, also in hypertrophic cardiomyopathy. In these trials, did you all at Cytokinetics blazed uncharted trails or did you follow more traditional paths?
- [24:09]
Robert Blum
Absolutely. I think we've been, to use your terminology, a trailblazer. A trailblazer, if you will, in clinical research, but it starts actually with translational biology and pharmacology, where you're measuring typically a number of different things. We from the beginning have thought about quantifiable endpoints, those things that are able to be measured even in cell biology and in vitro and ex vivo in vivo sciences. As we go from preclinical research into early clinical research, and the same things that we're measuring about muscle response preclinically, we take into the clinic. So we've pioneered some of those measurements and assessments and we've even developed some of the instruments that enable us to translate that preclinically into the clinic. So in phase one healthy volunteers, and subsequently into phase two patients, you're assessing with high fidelity, test retest reliability, some of the same endpoints that you're going to take all the way through into pivotal clinical trials. So that's enabling of a through line, a cohesive way of assessing impact, initially in test tubes and petri dishes and ultimately in patients looking at organ response. So that's one aspect of pioneering clinical research. Another is the way you elevate patient voice in the conduct of clinical research. And I think cytokinetics has demonstrated things there too that are uncommon. Cytokinetics has met patients where they are often taking assessments that would otherwise be performed in clinic and bringing them to patients homes. So that's enabling of higher adherence and compliance and ensuring that we're not perhaps becoming victims of loss to follow up or lost data along the way. So cytokinetics, from an operational standpoint, has thought about clinical research and clinical sciences failure modes and where one can improve upon them and in ways that I don't think are norms in clinical sciences. Across the biopharma sector.
- [26:43]
Alberto Grignolo
Was cytokinetics, in a way, a pioneer in what we now know or call decentralized clinical trials?
- [26:50]
Robert Blum
I'd like to think so. Time will tell if that translates into something that does produce the outcomes that would suggest that that has worked for us. But yes, we have. We do rely very importantly on our strategic partners and CROs. But at the same time we've built in house some of the competencies, we've decentralized that in terms of how it's manifest and deployed. Cytokinetics has also, I think more than is typical, gone international. We do large global studies. You have to in cardiology and frankly in als. And we've gone into remote corners of the world, but always with a high quantification of the things that can cause interruption. So we very mindfully focus on training. We very thoughtfully engage assessors at the local sites to ensure that they are managing and monitoring and that we're, we have the right dashboards to assess the quality of data, albeit, as is often blinded all the way through the conduct of clinical trials.
- [28:08]
Alberto Grignolo
We all know that clinical trials can succeed or not succeed. Were you ever tempted to just give up?
- [28:15]
Robert Blum
Never tempted to give up. I think to be in this business you have to accept failure and you have to Confront it in an honest way. But I do think the way one approaches clinical trials is essential. This is the intersection of good science and medicine. And clinical research, like any other research, is about hypothesis generation and hypothesis testing. So you have to be doing the right things by both and they speak to different principles in guiding the design and conduct and operationalizing of clinical trials. We do single dose studies to assess endpoints ultimately as could be then hypothesis generating before we do hypothesis testing. We do studies that look for evidence of effect in earlier smaller studies before we then proceed to do the more meaningfully and statistically robust later stage studies in phase two. And ultimately the connective tissue between phase two and phase three studies is essential so that you're assessing in phase two both inclusion and exclusion criteria and endpoints that you intend to study in phase three. These are things that seem obvious but at the same time are not often practiced. For instance, in our heart failure study with Omecamtiv, we conducted over 30 clinical trials from 2006 until 15 years later before we and Amgen conducted the pivotal 8000 patient phase 3 study. That study, even as it was positive, still left open certain questions that now need to be confirmed in yet another phase 3 clinical trial. So you have to be patient, you have to be persistent, but you also have to be a good student of what you can learn from one study to the next. You have to be objective. And that's, I think the elegance of and clinical research is, I think the fulcrum around which our businesses are built.
- [30:43]
Alberto Grignolo
Have you ever felt that the world or the circumstances were stacked against you? And as I'm asking you that question, I'm thinking back of your father, his story of resilience and survival and rebirth in a different country. But did you ever feel that the world was stacked against you? And if you felt that way, how did you stay motivated?
- [31:02]
Robert Blum
I think all of us in the business of discovering and developing new medicines feel like the world is stacked against us. It is because science is about continual learning. And most drugs, as you know, that enter clinical studies fail if you don't acknowledge that going in one is naive to the reality of the business we're in. But isn't that the beautiful thing about this? Because despite the odds being stacked against us, we all still persevere because we know it's important and we know that patients depend on us and we are self selecting. I think all of us for being in this business because we know it's difficult, it's an arduous task, but the gratification and the fulfillment at the end of the day makes it all worthwhile.
- [31:59]
Alberto Grignolo
Robert, in closing I'd like to ask you how do you foresee the future of cytokinetics? Recently you announced Vision 2030, so I'd like to ask you what are your corporate trajectory and your personal trajectory?
- [32:12]
Robert Blum
So I foresee for cytokinetics that we will hopefully continue to execute on the same plan we set forward back in the day when we did that original strategic planning that cytokinetics will be counted amongst the great biopharmaceutical companies for being a pioneer and a leader in our area of muscle biology, maintaining discipline and focus to it through both organic and inorganic programs. As we may ourselves become a partner of choice to others who are innovating. Also in this space and through in licensing and sponsored research and even M and A, we augment that which we're doing in our own laboratories that we will be developing new medicines rooted in muscle biology and directed to these two verticals, cardiovascular medicine and neuromuscular medicine. In each case building specialty franchises that afford a high return on investment, concentrated sales and marketing to support a high yield on shareholder equity and as that will translate to sustainable and enduring growth businesses in the biopharmaceutical space. Specialty as it is, but still very high value, delivering medicines that are important. That's going to be enabling of us not only to be addressing diseases that are rooted in the pathophysiology of muscle dysfunction, but as we age and baby boomers are increasingly now the predominant users of these new medicines that we're affording higher health span and more longevity, more active lifestyle and functional quality of life for patients that may ultimately blur the lines between that which is a pharmaceutical and that which is what I call a consumerceutical and where medicines or products rooted in muscle will enable higher productivity life. That's the vision. And as we executed on our Vision 2025 and now set forward on Vision 2030, I hope that cytokinetics will be an exemplar, albeit it takes us a while to do it, an exemplar amongst other great companies as to how best to achieve those objectives.
- [34:59]
Alberto Grignolo
We're wishing you great success later this year and beyond as you prepare hopefully to launch your very first product. Robert, this wraps up this episode of the clinical trial blazers. Big thanks to you, Robert Blum, President and CEO of Cytokinetics for sharing your personal journey and offering valuable insights on how clinical trials are driving better outcomes for patients. With respect to your leadership style, I was impressed by your mention of humility and values, being a servant leader and sharing information and enabling decision making by others, and importantly, as you said, inviting criticality of thought, which I think, as you mentioned, leads to better decisions overall.
- [35:41]
Robert Blum
Thank you Alberto.
- [35:44]
Alberto Grignolo
We hope that today's discussion provided meaningful perspectives on navigating the complexities of clinical development while staying focused on patient centered results. Stay tuned for more episodes of Clinical Trial Blazers, where we will continue to explore human stories behind the ideas and innovations shaping biotech. And please do take a moment to rate and review this podcast wherever you listen. Clinical Trial Blazers is created by Paraxel Biotech and hosted by me, Alberto Grignolo. Special thanks to our production partner, Wonder Media Network. Thank you for listening. Until next time, stay curious and let's keep pushing the boundaries of clinical research.