After years in the doldrums, the biopharma sector is showing renewed momentum, with breakthroughs and investment building across rare disease, oncology and neuroscience. IPO activity is beginning to return , with biotech listings raising $1.7 billion in Q1 2026, the strongest quarter since 2021. It is into that selective rebound that Cytokinetics arrives with something rare in biotech: a first FDA approval after nearly three decades in development.

The company recently secured approval for Myqorzo (aficamten), marking the culmination of a 27-year program that began as early-stage muscle biology research and has now become a commercial therapy for hypertrophic cardiomyopathy. The condition affects as many as 200,000 patients in the U.S., and a similar number in Europe. The milestone is not just a product launch; it is also a test case for whether long-horizon, highly focused biotech strategies can survive an industry defined by failure.

For CEO Robert Blum, the approval is validation of a deliberate model of company-building that prioritizes scientific depth, financial discipline, and long-term persistence over breadth or speed. “It’s not unprecedented, but it’s uncommon, and for so many of us it is all we know, which makes it that much more gratifying for what we’ve accomplished,” he says.

Surviving Biotech’s 90% Failure Rate

Biotech is notoriously unforgiving. More than 90% of drugs fail as they move from research into development, and even in late-stage trials, roughly half never make it to approval. Rather than diversify widely, the company chose to go deep, focusing on how cells, particularly heart muscle cells, move.

Over time, Cytokinetics built expertise in enhancing and suppressing muscle contraction. Its latest therapy targets hypertrophic cardiomyopathy, where the heart muscle becomes hyperactive.

“This particular medicine is a drug that inhibits a protein in your heart that is responsible for its pumping function,” Blum explains. “In certain patients that system can become hyperactive, and that can create symptoms, and risk of mortality. The goal of this therapeutic strategy is to relax that contractility, and in so doing, restore a quality of life that allows them to exercise, to feel better, to live more normal lives.”

Survival by Design, Not Luck

Scientific focus alone does not explain Cytokinetics’ survival. Blum says that its longevity depended as much on financial strategy. At a critical juncture, the company sold off its first major drug program. “We had to sell it off to finance the company,” he says. “It was a means by which we were able to maintain the team, the science and continue to build on our business with a portfolio of medicines that act in similar ways.”

For most founders, decades of uncertainty would bring doubt. Blum insists it never did. “There never was a time when I doubted it would happen,” he says. “It was always a question of how.”

That certainty became a stabilizing force, particularly during setbacks. From failed molecules to regulatory setbacks, the company developed scenarios for success and failure and updated them constantly. “Nothing that has happened at Cytokinetics wasn’t something we could have anticipated,” says Blum. “In every instance, we had a contingency plan.”

Culture as Infrastructure

If strategy kept the company alive, culture kept it together. From early on, Blum treated culture as a design choice, built around continuity of vision and reinforced through hiring.

“I try to get to the humanity of these candidates,” he says. “That’s the best arbiter of whether they have the resilience and the convictions to be part of a journey like the one we’ve been traveling.”

Today, the company employs more than 700 people across North America and Europe, with partnerships in Asia. Despite that scale, Blum emphasizes alignment. “There has to be a through line, something that unites us, whether it’s a financial executive, a scientist, a lawyer; they all have to feel valued,” he says. “I went from being a young CEO, thinking I was supposed to have the answers, to thinking about my role more as an ambassador to mission, culture, values, and enabling and empowering others.”

A distinctive aspect of Cytokinetics’ approach is its engagement of patients as collaborators. “The patients that we serve are partners to us in a journey,” says Blum. “These are patients who are in our laboratories, engaging with our scientists, poring over data with us, helping us in the design of clinical trials.”

They are also present in moments of failure. After a late-stage ALS (amyotrophic lateral sclerosis) drug trial failed, Blum describes it as ‘the worst moment of my professional career.’ “It was those patients and their caregivers who were there to elevate, lift us,” he says.

The industry faces many challenges, and as pharmaceutical companies create new therapies, ensuring access remains critical. As Lisa Salberg, president of the Hypertrophic Cardiomyopathy Association, points out, vertical integration can impact access.

“This can limit access to patients getting cutting-edge therapies that meet their needs rather than the cheapest option, which we know doesn’t always work,” she says. “That’s not to say that inexpensive drugs do not have a role in the treatment of HCM, but it's important to note that we never had labeled indication drugs until 2022. We have the ability to improve health and improve cardiac function before advanced heart failure strikes.”

Cytokinetics is now entering commercialization, with approvals secured across the U.S. Europe, and China, and plans to launch simultaneously. According to investor Robin Boldt, Europe mirrors the U.S. in patient numbers but will represent about 15% of peak market economics due to pricing differences.

He says: “Some companies have deprioritized launching drugs in Europe given the less favorable pricing environment. Robert Blum and team would never forgo launching such an important drug just because of a lower ROI calculus, which speaks to the culture at Cytokinetics and its leadership.”

The challenge now is execution without compromise. “Good companies get bought and never sold, and this will apply to Cytokinetics as well,” adds Boldt. “Specialty cardiology is very much on strategy for many large pharmaceutical companies, and I would not be surprised to see the company being acquired at some point, more so because Cytokinetics still has full ownership of the rights to its lead drug in the U.S. and Europe.”

At the same time, the company must continue to innovate. “It’s not enough that we have our first approval,” Blum says. “We have to deliver more medicines again and again and again.”

For Blum, the company’s purpose is inseparable from his own story. His father, a Holocaust survivor, shaped his outlook. “He had a magnanimous and generous spirit of compassion,” he says. “He inspired in me a love of science, for medicine, for humanity. That enabled me to combine a love of science, medicine and entrepreneurship, to build and lead a company that would make a big impact on personal lives.”

Three decades on, that ambition remains intact. “We have tasted the sweet success of our first approval,” Blum says. “And now we want to build on that, even if that takes longer to do it right.”