Can CYTK Clinch a Potential FDA Nod for Its Cardiovascular Drug?

It has been a roller coaster ride for Cytokinetics, Inc. CYTK so far in 2025.

While the delay in FDA approval of its lead candidate aficamten for the treatment of patients with obstructive hypertrophic cardiomyopathy (oHCM) was a setback for the company, the recent positive data on this cardiovascular drug at the European Society of Cardiology Congress 2025 has relieved investors.

Recent Positive Data on Aficamten Boost CYTK Stock

Aficamten is an investigational selective, small molecule cardiac myosin inhibitor.

In August, Cytokinetics presented primary results from the late-stage MAPLE-HCM study in a Hot Line Session at the European Society of Cardiology Congress 2025 in Madrid, Spain.

Results demonstrated the superiority of aficamten to the standard-of-care beta-blocker metoprolol on all clinically relevant efficacy endpoints. Aficamten also showed superiority to metoprolol in five of six secondary endpoints. It had a larger effect on measures of symptoms, functional class, and left ventricular outflow tract (LVOT) gradients as compared to first-line standard-of-care metoprolol. Aficamten substantially improved functional class and reduced patient symptom burden.

The results were a huge boost for CYTK as they raised hopes for a plausible FDA approval.

Cytokinetics noted that these effects were achieved in a broader patient population with oHCM than previously studied in SEQUOIA-HCM.

Cytokinetics also presented additional data on aficamten. The additional data from MAPLE-HCM showed that aficamten improved cardiac structure and function compared to metoprolol. New analysis showed that the annual incidence rate of atrial fibrillation with aficamten is 1.5%. Cytokinetics also presented longer-term data, which were consistent with the previously reported safety profile of aficamten.

CYTK had earlier entered into a collaboration and license agreement with Bayer BAYRY for the exclusive development and commercialization of aficamten in Japan for the treatment of patients with obstructive and non-obstructive HCM.

The deal included an upfront payment of €50 million and milestone payments of €90 million. Cytokinetics is also eligible to receive up to €490 million in commercial milestone payments upon Bayer's achievement of certain sales milestones and tiered royalties on net sales of aficamten in Japan.

Delay in Potential FDA Approval Hit CYTK

In May 2025, CYTK announced that the FDA has extended the target action date for the new drug application (NDA) seeking approval of aficamten for oHCM to Dec. 26, 2025.

The FDA had earlier set a target action date of Sept. 26, 2025. CYTK submitted the NDA for aficamten in obstructive HCM without an accompanying REMS, and the FDA accepted the same following pre-NDA discussions (wherein safety and risk mitigation were discussed).

During the NDA review, the FDA requested that Cytokinetics submit a REMS consistent with the inherent characteristics of aficamten, which the company subsequently provided. The submission of a REMS has now been determined by the FDA to be a major amendment to the NDA, resulting in a standard three-month extension to the original target action date. Nonetheless, no additional clinical data or studies have been requested by the FDA.

This delay was a huge setback for CYTK as it does not have any approved product in its portfolio.

Upon approval, aficamten will face competition from Camzyos (mavacamten), a first-in-class cardiac myosin inhibitor marketed by Bristol Myers Squibb BMY.

BMY obtained FDA approval of the drug in 2022 for the treatment of adults with symptomatic New York Heart Association class II-III obstructive HCM to improve functional capacity and symptoms.

Camzyos has put up a stellar performance. BMY also has a promising candidate, milvexian, in its cardiovascular pipeline.

CYTK’s Other Pipeline Candidates

Other pipeline candidates include omecamtiv mecarbil, a cardiac muscle activator, in patients with heart failure. Cytokinetics is enrolling in the confirmatory phase III multi-center, double-blind, randomized, placebo-controlled trial, COMET-HF, to assess the efficacy and safety of omecamtiv mecarbil in patients with symptomatic heart failure with severely reduced ejection fraction. Enrollment is expected to be closed in late 2026.

Additionally, Cytokinetics initiated AMBER-HFpEF, a phase II randomized, placebo-controlled, double-blind, multi-center, dose-finding clinical trial on ulacamten (CK-586) in patients with symptomatic heart failure with preserved ejection fraction (with left ventricular ejection fraction [LVEF] ≥ 60%). Cytokinetics expects to complete patient enrollment of the first two cohorts in the second half of 2025.



 

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