Robert Blum and Fady Malik have been with Cytokinetics since its inception almost 30 years ago. By achieving both primary endpoints in the phase 3 ACACIA-HCM study, Cytokinetics is on its path to becoming the first company to achieve approval for a drug to treat non-obstructive hypertrophic cardiomyopathy (nHCM).
In December of last year, the FDA approved Myqorzo to treat the obstructive type of the condition with blockbuster potential that comes with it. The opportunity, which comes with nHCM, would have doubled the potential number of patients that would have been served by the cardiac myosin inhibitor, based on the claims data that had been gathered at Cytokinetics.
With the trial results, Cytokinetics’ share price increased by 21% on Monday morning.
While scoring its first approval in Myqorzo was a significant milestone five months ago, CEO Blum and R&D chief Malik sounded particularly moved that Cytokinetics has become the first company to provide positive trial results in the indication and is on the verge of delivering medicine for the first time for those with nHCM.
“There’s a very robust, consistent effect here that is—for those of us in this business—one of those things that you aspire towards, but you rarely achieve, and that’s incredibly gratifying,” Blum said of the study results in an interview with Fierce.
Myqorzo showed statistically significant improvement in baseline to Week 36 as compared to placebo. The Kansas City Cardiomyopathy Questionnaire (KCCQ) was used to measure primary endpoints, which is completed by patients, and the maximal exercise performance standard.
The enhancement on the KCCQ was strong and consistent during the treatment period of those on Myqorzo, the company said. Concerning the exercise test, the subjects were evaluated using the consumption of peak oxygen uptake. Malik compared the uptake improvement that was observed in the study as equal to “weeks of hard training,” provided by Myqorzo.
Cytokinetics reported no secondary endpoints, but indicated statistically significant changes were observed consistently in all of the measures. According to Blum, the company will, as quickly as possible, discuss the results with the FDA.
Blum said there had never been a study that had demonstrated any positive results in this population. We were going at it in the design and conduct of this study, without a lot of benchmarks and reference standards, but we believed in the drug candidate, and how we could best approach designing a proper study, and it worked.
Competing with Bristol Myers Squibb in its obstructive hypertrophic cardiomyopathy indicator, Cytokinetics is competing against Bristol Myers Squibb’s cardiac myosin inhibitor Camzyos, which was approved in 2022 and had sales of 107 billion dollars last year.
The failure of BMS in a trial of Camzyos in nHCM last year made the achievement of Cytokinetics with the same indication of a drug with the same mechanism of action all the more remarkable.
Not all biopharma companies are as established in novelty biology and science as we are. We’re forging a path,” Blum said. It has taken us more than 25 years before we were able to enable this type of innovation.
Cytokinetics has announced groundbreaking clinical results for Myqorzo, a promising therapy developed for cardiomyopathy treatment. The historic trial success positions Myqorzo as a major advancement in cardiovascular medicine and highlights Cytokinetics’ growing role in innovative heart disease therapies.
Overview of the Historic Trial
The landmark clinical trial evaluated Myqorzo in patients suffering from cardiomyopathy, a condition that weakens the heart muscle and reduces its ability to pump blood effectively. Myqorzo demonstrated significant improvements in cardiac function, exercise capacity, and symptom reduction compared to standard treatment approaches.
Researchers reported that Myqorzo achieved its primary and secondary endpoints, making the study one of the most significant recent developments in cardiomyopathy research.
Key Findings from the Myqorzo Trial
Improved Heart Function
Myqorzo significantly enhanced cardiac performance by improving the efficiency of heart muscle contractions. Patients receiving Myqorzo showed measurable gains in overall heart function during the study period.
Better Exercise Capacity
Patients treated with Myqorzo experienced improved physical endurance and exercise tolerance. This outcome is especially important for individuals with cardiomyopathy, who often struggle with fatigue and limited activity levels.
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