An experimental Cytokinetics drug has Phase 3 data showing it improved the ability of the heart to work during exercise. In addition to meeting this main goal of its pivotal test in a rare cardiovascular disease, the results look competitive with a Bristol Myers Squibb product already on the market..

一种实验性细胞动力学药物的3期数据显示，它改善了心脏在运动过程中的工作能力。除了满足其在一种罕见心血管疾病中进行关键测试的主要目标外，该结果看起来与市场上已经上市的百时美施贵宝产品具有竞争力。。

The Cytokinetics drug, aficamten, is a potential treatment for obstructive hypertrophic cardiomyopathy (oHCM), a condition that leads to thickening of heart muscle that makes it harder for the organ to pump blood. Patients experience fatigue and shortness of breath. The condition can progress to heart failure.

细胞动力学药物aficamten是阻塞性肥厚型心肌病（oHCM）的潜在治疗药物，这种疾病会导致心肌增厚，使器官更难抽血。患者出现疲劳和呼吸急促。这种情况可能会发展为心力衰竭。

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Cytokinetics reported preliminary Phase 3 results on Wednesday showing that treatment with its pill after 24 weeks led to a 1.74 point increase in peak oxygen uptake compared to a placebo. The study had a targeted enrollment of 270 patients. South San Francisco-based Cytokinetics said the treatment effect was consistent across patient subgroups, including those receiving or not receiving beta blockers, a type of blood pressure drug also used to manage oHCM..

细胞动力学周三报道了初步的第三阶段结果，结果显示，与安慰剂相比，24周后服用避孕药导致峰值摄氧量增加1.74点。该研究有针对性地招募了270名患者。位于南旧金山的细胞动力学研究中心表示，治疗效果在患者亚组中是一致的，包括那些接受或不接受β受体阻滞剂的患者，β受体阻滞剂是一种也用于治疗oHCM的血压药物。。

Secondary goals included assessments of cardiomyopathy and heart function at weeks 12 and 24. Cytokinetics reported statistically significant and clinically meaningful improvement on these measures, but did not disclose specific figures.

次要目标包括在第12周和第24周评估心肌病和心脏功能。细胞动力学报告了这些措施的统计学显着性和临床意义的改善，但未披露具体数字。

In oHCM, genetic mutations affect myosin, a heart muscle protein. Mutated myosin lead to excessive contractions that thicken the heart muscle. Aficamten is a small molecule designed to bind to myosin, stabilizing the protein in a state in which it is unable to turn chemical energy into the movement of heart muscle contractions..

在oHCM中，基因突变会影响肌球蛋白（一种心肌蛋白）。突变的肌球蛋白导致过度收缩，使心肌增厚。Aficamten是一种设计用于与肌球蛋白结合的小分子，可将蛋白质稳定在无法将化学能转化为心肌收缩运动的状态。。

BMS drug Camzyos works similarly to aficamten. The pharmaceutical giant added its oHCM molecule to its pipeline via a $13.1 billion acquisition and the drug is projected to become a blockbuster seller. In the nine months ended Sept. 30, BMS reported the product accounted for $143 million in revenue compared to $8 million in the same period in 2022, the year of the medication’s FDA approval..

BMS药物Camzyos的作用类似于aficamten。这家制药巨头通过131亿美元的收购将其oHCM分子添加到其管道中，预计该药物将成为畅销药。在截至9月30日的9个月内，BMS报告称，该产品的收入为1.43亿美元，而2022年（该药物获得FDA批准的那一年）同期为800万美元。。

One problem for Camzyos is it can also reduce the volume of blood the heart’s left ventricle pumps with each contraction, potentially leading to heart failure. The drug’s label flags this left ventricular ejection fraction (LVEF) risk, which must be managed by a Risk Evaluation and Mitigation Strategy (REMS), a program that imposes strict oversight and monitoring of the use of a drug..

Camzyos的一个问题是它还可以减少每次收缩时心脏左心室泵送的血液量，从而可能导致心力衰竭。该药物的标签标记了这种左心室射血分数（LVEF）风险，必须通过风险评估和缓解策略（REMS）进行管理，该策略对药物的使用进行严格的监督和监测。。

Cytokinetics said aficamten was well tolerated with an adverse event profile comparable to placebo. The company reported that five patients (3.5%) in the treatment group developed low LVEF compared to one (0.7%) in the placebo group. But there were no reports of worsening heart failure or treatment interruptions.

细胞动力学研究表明，阿非卡姆汀耐受性良好，不良事件发生率与安慰剂相当。该公司报告说，治疗组有5名患者（3.5%）出现低LVEF，而安慰剂组只有1名（0.7%）。但没有关于心力衰竭恶化或治疗中断的报道。

Cross-trial comparisons can be misleading, but Camzyos’s Phase 3 test posted results showing seven patients in the treatment group and two in the placebo arm experienced reductions in LVEF..

交叉试验比较可能会产生误导，但Camzyos的3期试验结果显示，治疗组有7名患者和安慰剂组有2名患者的LVEF降低。。

“A therapy like aficamten that improves exercise capacity in a clinically meaningful manner, absent low LVEF events that interrupt treatment, should be a welcome addition for HCM patients as well as the clinicians who treat them,” Martin Maron, director of the Hypertrophic Cardiomyopathy Center, Lahey Hospital and Medical Center in Burlington, Massachusetts, said in a prepared statement..

马萨诸塞州伯灵顿Lahey医院和医学中心肥厚型心肌病中心主任Martin Maron在一份准备好的声明中说：“像aficamten这样的疗法，可以以临床上有意义的方式提高运动能力，并且不存在中断治疗的低LVEF事件，应该是HCM患者以及治疗他们的临床医生欢迎的补充。”。。

In a research note sent to investors Wednesday, Leerink Partners analyst Roanna Ruiz wrote that aficamten’s consistent treatment effect across patient subgroups could support broader use of the drug in oHCM. She added that the safety profile could support a less restrictive REMS program compared to Camzyos.

Leerink Partners分析师罗安娜·鲁伊斯（RoannaRuiz）在周三发给投资者的一份研究报告中写道，aficamten在患者亚组中的一致治疗效果可能支持该药物在oHCM中的更广泛使用。她补充说，与Camzyos相比，安全配置文件可以支持限制较少的REMS计划。

That potential has reportedly attracted takeover interest from at least two big pharma companies..

据报道，这一潜力吸引了至少两家大型制药公司的收购兴趣。。

Cytokinetics said full results from aficamten’s Phase 3 study will be presented at an upcoming medical conference.

细胞动力学表示，aficamten第三阶段研究的全部结果将在即将举行的医学会议上公布。

Image: Magicmine, Getty Images

图片：Magicmine，Getty Images

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Gabby Everett, the site director for BioLabs Pegasus Park, offered a tour of the space and shared some examples of why early-stage life science companies should choose North Texas.

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