Cytokinetics' Market Potential

In the news: Cytokinetics (NASDAQ:CYTK) has initiated phase I clinical trial to investigate the pharmacokinetic profile of CK-2017357. Cytokinetics has also initiated the second part, or "Part B", of a previously initiated, Phase I clinical trial of CK-2017357 in healthy male volunteers.

CK-2017357 is a small molecule drug that offers hope to patients suffering from detrimental and life-threatening muscle wasting and neuromuscular dysfunction. These diseases include amyotrophic lateral sclerosis (ALS), myasthenia gravis, sarcopenia, claudication, cachexia, and shortcomings associated with aging.

The primary objective of the multi-dose phase I clinical trial is to determine the safety and tolerability of CK-2017357 after multiple oral doses to steady state in healthy male volunteers. The secondary objective is to evaluate the pharmacokinetic profile of CK-2017357 after multiple oral doses to steady state.

Cytokinetics has initiated Part B of phase 1 trials while Part A is still ongoing. Despite the fact that Part 1 trial remains blinded, investigators could not miss that all the doses given have been tolerated, including the dose that produced CK-2017357 blood levels associated with increased skeletal muscle function in preclinical models.

Part 1 single-dose trial is meant to assess safety, tolerability, and pharmacokinetic profile in healthy male volunteers and determine its maximum tolerated dose and plasma concentration. The objective of the “Part B” trial of phase I trial is to assess the pharmacodynamic effect of CK-2017357 when administered orally to healthy male volunteers. In this trial, the force-frequency relationship of an externally stimulated nerve-muscle pair and its relationship to CK-2017357 plasma concentration will be determined.

Interested in the trial details click here.


Cytokinetics has two programs; both deal with life-threatening conditions that have yet to find satisfactory treatments. The first program focuses on developing cardiac myosin activators for heart failure. In this program, the drug, omecamtiv mecarbil, has reached phase 2 clinical trials with promising results. The second program, which is the topic of the above news, is focused on the discovery and development of small molecule sarcomere activators that are expected to boost skeletal muscle (voluntary muscle) contractility. CK-2017357 is the first small molecule sarcomere activator to reach clinical trials.

How does CK-2017357 work?

Skeletal muscle contractility is believed to be driven by the sarcomere, which is composed of several key proteins. Myosin, one of the sarcomere’s proteins interacts with a second protein, protein called actin, to convert chemical energy into mechanical force. A set of regulatory proteins, tropomyosin, and three troponin make the actin-myosin interaction dependent on changes in intracellular calcium levels.

CK-2017357 selectively activates the troponin complex, hence increases its sensitivity to calcium, leading to an increase in skeletal muscle force. The results are encouraging, bringing hope for patients suffering from amyotrophic lateral sclerosis (ALS), myasthenia gravis, sarcopenia, claudication, cachexia, and shortcomings associated with aging.


- ALS: Affects between 20,000 and 30,000 people in the United States. The disease is associated with a 3-year mortality rate of 50%. There are no specific effective treatments.

- Myasthenia gravis: A chronic disease characterized by a defect in the transmission of nerve impulses to skeletal muscles afflicts approximately 60,000 patients in the United States.

- Cachexia, Is characterized by a drastic and unintentional loss of body mass. It is prevalent in 15%-35% of heart failure patients and in approximately 50% of cancer patients.

- Intermittent claudication: Cramping pains in the legs caused by peripheral arterial disease. It impacts between 1million to 3 million people in the United States each year.

- Sarcopenia: This condition characterized by loss of muscle mass, strength, and function impacts the lives of around 25-30% of the U.S. population over the age of 65.

So, the market could be huge. Add to it the heart failure drug market, then we would be talking about billions of dollars in revenues for Cytokinetics when these drugs reach the market.

Cytokinetics discovered and developed its own breakthrough molecules. Its drugs and their targets are unique and might offer hope for patients suffering from devastating and life-threatening diseases. The rationale behind its treatments is scientifically sound and satisfying and the small molecule drugs developed for skeletal and involuntary muscles have not demonstrated any serious side effects.

Cytokinetics’ cardiac myosin program attracted Amgen. At first, Amgen invested a large sum of money as a reservation of priority rights. Then, after the announcement of positive results, Amgen took a closer look at the drug, its rationale and the results and decided to officially partner on the program. Amgen’s enthusiasm added more validation to our enthusiasm, which grew much bigger for Cytokinetics breakthrough molecules, thus, for its stock. We wish this firm all the best.

Disclosure: No positions