Cytokinetics Maps MYQORZO Expansion, Heart Failure Pipeline at Bernstein Conference

Key Points

• MYQORZO adoption is accelerating: Cytokinetics reported approximately 2,500 new patient starts in the first quarter and 3,000 in the second, while prescriptions increased to nearly 1,500. The company held slightly more than 40% of new prescriptions and is targeting 50% by year-end.
• The company plans to seek approval for MYQORZO in non-obstructive hypertrophic cardiomyopathy based on ACACIA study data, potentially making it the only cardiac myosin inhibitor approved for both major HCM subtypes.
• Cytokinetics is advancing its heart-failure pipeline, with Phase III omecamtiv mecarbil results expected around 2028 and Phase II ulacamten results anticipated in 2027. Management said near-term business development will prioritize earlier-stage technologies rather than late-stage acquisitions.

Cytokinetics NASDAQ: CYTK executives outlined the company’s commercial expansion plans, cardiovascular pipeline and business-development priorities during a Bernstein investor symposium, highlighting the launch of MYQORZO and clinical programs targeting several forms of heart failure.

Isaac Ciechanover, executive vice president of corporate development and chief business officer, said the company was founded in 1997 and began operating in 1998 in South San Francisco. While its original focus included oncology and antifungal treatments, Cytokinetics later shifted toward cardiovascular disease following research into the sarcomere, the functional unit of muscle contraction.

The company now describes itself as a commercial-stage developer of cardiovascular medicines, particularly cardiac myosin inhibitors, while continuing research in skeletal-muscle conditions. Ciechanover said Cytokinetics’ near-term goal is to become a leading specialty cardiology company.

Vision 2030 and Commercial Expansion

Ciechanover described the company’s “Vision 2030” strategy, which includes goals of having two approved therapies across three indications, maintaining 10 new molecular entities in development and operating in more than 15 countries by 2030. The company also aims for its medicines to reach at least 100,000 patients through clinical trials and commercialization.

MYQORZO has launched in the U.S. for obstructive hypertrophic cardiomyopathy, or HCM, and has also launched in Germany, according to Ciechanover. He said the company has partners including Sanofi in China and Bayer in Japan, while Cytokinetics intends to commercialize directly in the U.S. and Europe where possible.

“No one is going to focus on a company and on a product better than yourself,” Ciechanover said, explaining the company’s preference for direct commercialization in major markets. He added that partnerships remain appropriate in certain territories where establishing operations would not be practical.

The company’s cardiovascular strategy centers on three advanced programs:

• MYQORZO for obstructive HCM;
• Omecamtiv mecarbil, a cardiac myosin activator in Phase III development for heart failure with reduced ejection fraction, or HFrEF; and
• Ulacamten, a cardiac myosin inhibitor in Phase II development for heart failure with preserved ejection fraction, or HFpEF.

MYQORZO Launch Metrics and HCM Expansion

Ciechanover said Cytokinetics has seen growing adoption of MYQORZO despite entering the market as the second treatment in its class, four years after the first launch. He said the company reported approximately 2,500 new patient starts in the first quarter and 3,000 in the second quarter, compared with roughly 2,000 patients per quarter before the launch.

Prescriptions dispensed rose from fewer than 400 in the first quarter to nearly 1,500 in the second quarter, he said. Cytokinetics has reached more than 700 physicians who can potentially prescribe cardiac myosin inhibitors, with more than half of those physicians either not prescribing such therapies regularly or never having prescribed one previously, according to Ciechanover.

The company said it held slightly more than 40% of new prescriptions when physicians selected between available therapies and has an aspirational target of reaching 50% by year-end.

Ciechanover said Cytokinetics is filing a supplemental new drug application based on data from the ACACIA study in non-obstructive HCM. If approved, he said MYQORZO would be the only cardiac myosin inhibitor approved in both obstructive and non-obstructive HCM.

He estimated that 110,000 to 120,000 U.S. patients have diagnosed obstructive HCM, of whom about 20,000 to 25,000 have received therapy. Cytokinetics believes the non-obstructive HCM population is similarly large, he said.

Heart Failure Pipeline

Dan Jacoby, Cytokinetics’ vice president of clinical research, discussed the company’s Phase III COMET trial of omecamtiv mecarbil in patients with progressive HFrEF. The study was designed based on a subgroup from the prior GALACTIC study, focusing on patients with lower left ventricular ejection fraction, recent hospitalization and elevated NT-proBNP levels despite guideline-directed medical therapy.

Jacoby said the COMET trial includes a run-in period intended to confirm that participants are taking the therapy and to reduce early, unmodifiable events. He said the study is expected to read out around 2028.

Unlike cardiac myosin inhibitors used in HCM, omecamtiv mecarbil is intended to increase cardiac contractility. Jacoby said the drug is designed to increase force generation without disrupting calcium cycling, increasing oxygen demand or causing cellular injury.

For ulacamten, Cytokinetics is conducting the Phase II AMBER study in a subset of HFpEF patients whose disease is predominantly driven by diastolic dysfunction. Jacoby said the company is in the dose-ranging portion of the trial and expects to complete enrollment this year. Initial results from the Phase II cohort are expected in 2027, after which the company will determine whether another cohort is needed before moving into Phase III.

Business Development Focus

Ciechanover said Cytokinetics does not expect to pursue late-stage Phase II or Phase III acquisitions in the near term, citing the company’s existing pipeline and commercial priorities. Instead, management plans to focus on earlier-stage opportunities, including novel targets and modalities such as RNA interference and biologics.

He said the company may pursue rest-of-world licensing collaborations while retaining a focus on direct commercialization in key developed markets. Cytokinetics also expects to consider collaborations in which it shares development costs before potentially assuming development and commercialization responsibilities.

About Cytokinetics (NASDAQ:CYTK)

Cytokinetics, Incorporated is a biopharmaceutical company focused on discovering and developing medicines that target the biology of muscle function. The company applies expertise in muscle physiology and contractility to develop potential treatments for cardiovascular and neuromuscular diseases.

Its leading programs include aficamten, an investigational cardiac myosin inhibitor being developed for conditions involving abnormal heart-muscle contraction, including hypertrophic cardiomyopathy.

This instant news alert was generated by narrative science technology and financial data from MarketBeat in order to provide readers with the fastest reporting and unbiased coverage. Please send any questions or comments about this story to contact@marketbeat.com.