Cytokinetics is a specialty cardiovascular biopharmaceutical company, building on its over 25 years of pioneering scientific innovations in muscle biology, and advancing a pipeline of potential new medicines for patients suffering from diseases of cardiac muscle dysfunction.
You've spent nearly three decades building Cytokinetics, but your journey into biotechnology began much earlier. What first inspired your interest in the field?
I grew up in a small town in western North Carolina, where I had the good fortune of having a biology teacher who, although he had never left that mountainous town, was very well read. He spoke about the advances in biotechnology emerging in the late 1970s through genetic engineering and recombinant DNA technologies around the San Francisco Bay Area, where Genentech was just getting started. He inspired a love of biology—not just as a science, but for its potential to transform medicine—which led me to pursue both biology and the business of biology, and how scientific innovation could translate into new medicines for patients.
When I arrived in San Francisco, I met Genentech founder Bob Swanson at a shareholder meeting, who became a mentor, an early angel investor in Cytokinetics and helped shape the way we think about translating biology into medicines. At the same time, my father, a Holocaust survivor who was liberated from Auschwitz, had an enormous influence on me. His generosity of spirit and belief in using science and medicine to benefit people inspired my brothers and me to pursue careers that could make a difference. Those influences came together when I studied biology and economics at Stanford and began my career in biotechnology while the industry was still finding its footing.
Muscle has traditionally been viewed simply as the tissue that helps us move, but it's now recognised as central to overall health. What changed, and why did Cytokinetics choose to focus on muscle biology so early?
I remember studying the sarcomere in high school biology—an elegant biophysical structure that gives muscle the ability to contract and relax. It's a multi-protein complex whose proteins work together to generate mechanical force. Later, at Stanford, I studied with one of the pioneers who helped decipher how those proteins are organized to produce that force. Even then, I began thinking about how that science might one day translate into a biopharmaceutical company.
Several years later, I reconnected with that professor when he and three other scientists were looking to commercialize their research. Two venture capitalists independently approached me because they believed the scientists needed someone with business development experience to help build the company. We founded Cytokinetics around the biology of the cytoskeleton, which governs how cells move, divide and how muscles contract. Over time, we narrowed our focus to muscle biology because we believed it offered the greatest opportunity to transform medicine.
More than 20 years ago, we believed muscle biology would become central not only to treating disease but also to healthy aging, long before terms like "health span" and "longevity" entered the mainstream—and that's exactly the direction the field has taken.
After nearly three decades of research, Cytokinetics has reached its first commercial milestone. What does this moment represent for the company?
To get from where we started to where we are today, we identified applications of our science that could generate earlier impact, create cash flow and fund research programs that would take longer to mature. Given my background in cardiovascular medicine, we focused initially on medicines that activate or inhibit cardiac muscle in diseases where its function is impaired. That allowed us to build a sustainable commercial enterprise while continuing to invest in skeletal muscle, which represents the frontier of healthy aging and longevity.
Our priority was developing medicines targeting cardiac myosin, the protein responsible for the heart's pumping function. In 2005, we began developing a cardiac myosin activator for heart failure and, after 33 clinical trials, including a positive 8,000-patient study, we're conducting one additional confirmatory study requested by the FDA and EMA. In parallel, we developed the cardiac myosin inhibitor MYQORZO, recently approved in the United States, Europe and China for symptomatic obstructive hypertrophic cardiomyopathy in adults. While MYQORZO is our first commercial medicine, it is funding the next generation of medicines, including Phase I skeletal muscle programs targeting neuromuscular diseases, frailty and potentially improving muscle function alongside GLP-1 therapies.
What's been the hardest part of building an entirely new field of medicine?
We've had successes and setbacks. We had a Phase 3 trial fail with a skeletal muscle activator we believed had potential for ALS, or motor neuron disease, but it never diminished our commitment to persevere. One hallmark of our company has been the continuity of our convictions and our resilience.
The bigger challenge has been pioneering an entirely new field. We haven't just had to discover new medicines—we've had to define new mechanisms of action, new biomarkers and new ways of demonstrating meaningful clinical benefit. That has meant developing new tools to measure muscle response, from preclinical models assessing endurance and isolated muscle fiberes to clinical measures regulators recognize as meaningful for patients. Building that scientific and regulatory pathway has been every bit as pioneering—and at times as frustrating—as discovering the medicines themselves.
You've watched biotechnology evolve over more than four decades, and technologies like AI are accelerating that progress. Looking ahead 10 years, how do you see muscle biology shaping the future of medicine and healthy aging?
I've seen many technologies over the past 40 years that were expected to be transformational. They've all improved efficiency and productivity, but there's no substitute for good science and dedicated scientists. AI will be meaningfully important, particularly in helping us reach human studies more efficiently, but I don't believe it will replace the need to test medicines in people. In silico models, algorithms, and machine learning will help us get there faster, but human studies will remain essential.
Ten years from now, I believe we'll have an abundance of medicines directed specifically at muscle. Today, although we have many treatments for heart disease, very few act directly on the proteins that drive the heart's function. Most target downstream consequences such as blood pressure or cholesterol. Medicines like MYQORZO represent a new generation of therapies that work directly on the heart's machinery, and that same approach will extend to smooth and skeletal muscle. As genetic precision improves, muscle will become a common pathway for improving health span and longevity, with many more companies focusing on muscle biology.
