Robert Blum and Fady Malik, MD, have been involved with Cytokinetics since its founding nearly 30 years ago. As they sat in a conference room Monday afternoon discussing the overwhelmingly successful trial results for Myqorzo (aficamten), it was a feeling of excitement and humility in equal measure.
By meeting both primary endpoints in the Phase 3 ACACIA-HCM study, Cytokinetics is on track to become the first company to win approval for the treatment of non-obstructive hypertrophic cardiomyopathy (nHCM).
Myqorzo was approved by the FDA last December to treat obstructive diseases, offering the potential for a blockbuster hit. According to claims data collected by Cytokinetics, the opportunity presented by nHCM will double the number of potential patients for cardiac myosin inhibitors.
Winning the first approval for Myqorzo was an important milestone five months ago, but CEO Blum and head of research and development Malik seemed particularly thrilled that Cytokinetics was the first company to achieve positive trial results in this indication and is on track to bring the drug to nHCM patients for the first time.
“We have a very robust and consistent effect here, which is one of those things that those of us in this industry aspire to but rarely achieve, and that’s very gratifying,” Blum said of the study’s findings in an interview with Fierce.
In this study, Myqorzo produced statistically significant improvements from baseline to week 36 compared to placebo. The primary endpoint was measured using the patient-completed Kansas City Cardiomyopathy Questionnaire (KCCQ) and maximal exercise performance criteria.
The company said the improvements with KCCQ were “strong and consistent” throughout treatment with Myqorzo. Regarding exercise testing, participants were assessed by measuring peak oxygen uptake. Malik compared the improvement in intake observed in this study to being equivalent to “a few weeks of hard training” provided by Myqorzo.
No secondary endpoints were reported for cell dynamics, but statistically significant improvements were consistently seen across all measurements.
Bloom said the company will discuss the results with the FDA as soon as possible.
At Monday’s steering committee meeting, the results were discussed, with members using words such as “transformative, amazing and a home run,” Blum said.
“No studies have ever shown positive results in this population,” Bloom said. “In some ways, in designing and conducting this study, we were trying to do that without a lot of benchmarks or reference standards, but we believed in the drug candidate and the best way to approach designing the right study, and it worked.”
In the indication of obstructive hypertrophic cardiomyopathy, Cytokinetics competes with Bristol-Myers Squibb’s cardiac myosin inhibitor Camdios. Camzios was approved in 2022 and had sales of $1.07 billion last year.
BMS’s trial of Camzyos in nHCM fell short last year, making Cytokinetics’ success in the same indication with a drug with the same mechanism of action even more remarkable.
“Not all biopharmaceutical companies are as rooted in novel biology and science as we are. We are blazing a trail,” Bloom said. “It took more than 25 years to make this kind of innovation possible.”
