Cytokinetics, a specialty biopharmaceutical company focusing on cardiovascular diseases in advanced stages, has joined forces with Bayer through an exclusive collaboration and licensing arrangement. This partnership aims to advance the development and marketing of aficamten for patients in Japan suffering from both obstructive and non-obstructive hypertrophic cardiomyopathy (HCM). The agreement includes certain reserved development rights for Cytokinetics. Aficamten, which represents a new class of cardiac myosin inhibitors, offers potential therapeutic benefits for individuals with HCM.
In this strategic collaboration, Cytokinetics will receive an initial €50 million payment. Additionally, the company stands to gain up to €90 million contingent upon achieving various milestones up to commercial launch, with €20 million expected shortly. Further financial prospects include up to €490 million in commercial milestone payments from Bayer, dependent on certain sales targets, accompanied by tiered royalties on aficamten’s net sales in Japan.
The synergy between Cytokinetics’ extensive development framework for aficamten and Bayer’s local acumen and specialization in cardiovascular disease management aims to support patients in Japan with unmet medical needs.
The unified development strategy involves Bayer spearheading a Phase 3 clinical study with Japanese patients experiencing obstructive HCM, while Cytokinetics widens its ongoing ACACIA-HCM Phase 3 trial for non-obstructive HCM patients in Japan. This step is geared towards securing marketing authorization for aficamten within the region. Concurrently, the CEDAR-HCM study evaluates the drug’s impact on pediatric patients with obstructive HCM.
Cytokinetics’ President and CEO, Robert I. Blum, remarked, “While we push forward with aficamten’s commercialization in the U.S. and Europe, entering this alliance with Bayer allows us to benefit from their cardiovascular focus and know-how, aiming to extend our offering to more HCM patients.” Blum emphasized the importance of collaborations to expand access to their groundbreaking innovations.
Juergen Eckhardt, M.D., from Bayer’s Pharmaceuticals Division, expressed enthusiasm about the promising advancements aficamten has shown in prior studies and the opportunity to rapidly deliver this therapy to Japanese patients. He highlighted this partnership as a reflection of Bayer’s dedication to providing groundbreaking treatments for unaddressed cardiovascular needs, leveraging their comprehensive expertise from discovery to commercialization.
Aficamten, identified as a selective small molecule cardiac myosin inhibitor following extensive chemical refinement, was designed to decrease actin-myosin cross-bridge activity during the cardiac cycle, thus moderating myocardial hypercontractility related to HCM. Preclinical assessments demonstrated aficamten’s ability to bind selectively to an allosteric site on cardiac myosin, reducing myocardial contractility.
The aficamten development venture is investigating its effectiveness in increasing exercise capacity and alleviating symptoms in HCM patients and assessing its long-term impact on cardiac function and structure. The SEQUOIA-HCM trial, a pivotal Phase 3 trial, evaluated aficamten’s safety and efficacy in patients experiencing symptomatic obstructive HCM. The US FDA and China’s NMPA granted aficamten Breakthrough Therapy status for obstructive HCM treatment. Cytokinetics presented an NDA to the FDA in Q3 2024, with plans to submit an MAA to the EMA by Q4 2024.
In addition to the SEQUOIA-HCM study, aficamten is under evaluation in various other trials: MAPLE-HCM compares it against metoprolol for obstructive HCM, ACACIA-HCM focuses on non-obstructive HCM, CEDAR-HCM looks at pediatric cases, and FOREST-HCM is an open-label extension trial scrutinizing aficamten’s performance in HCM.
Hypertrophic cardiomyopathy (HCM) is a condition where the heart muscle thickens abnormally. This thickening detracts from the heart’s ability to relax, reducing its pumping efficiency, which leads to exercise intolerance and symptoms like chest discomfort, dizziness, breathlessness, and fainting. HCM is the most predominant inherited cardiovascular disorder, affecting approximately 280,000 diagnosed patients and an additional 400,000-800,000 undiagnosed individuals in the U.S. The majority of HCM patients have obstructive HCM, leading to outflow tract obstructions, while the rest have non-obstructive variants, which impact blood flow differently. Those with HCM face heightened risks of cardiovascular issues, such as atrial fibrillation, stroke, mitral valve disorders, and life-threatening arrhythmias. HCM significantly contributes to sudden cardiac deaths among youth and athletes and increases the likelihood of advancing into dilated cardiomyopathy and heart failure, necessitating potential transplants.
Cytokinetics is committed to the discovery, advancement, and marketing of muscle biology-directed pharmaceuticals intended for debilitating diseases impairing cardiac muscle function.
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