Cytokinetics, Defying the Fashion in Biotech, Sticks with Gradual Science-Based Strategy

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was to pursue new drug development based on growing understanding of the cell’s skeleton, or cytoskeleton, or basically a network of proteins involved in mechanical aspects of cell behavior. This approach has evolved over the years into a focus on how cells act during muscle contraction.

This scientific foray has led Cytokinetics in a number of interesting directions. I spoke to Blum mainly about CK-357, a compound the company is developing for ALS, otherwise known as Lou Gehrig’s disease. This is a neurodegenerative disease that gradually paralyzes and kills people. The basic biological cause is unknown, and there aren’t really any effective treatments on the market today.

Cytokinetics isn’t really seeking a cure for this condition, but it hopes its basic biology approach will be useful for treating major symptoms of the disease. The drug is designed to increase muscle force, and delay fatigue in fast-twitch muscles. That’s the muscle type that enables sprinters to run their races, or ordinary people to go up a flight of stairs. They also deteriorate more quickly than other muscle types as people age, and the downhill slide is worsened by neurodegenerative diseases like ALS.

Cytokinetics has structured its clinical trial program to provide a clear answer on both whether the drug is working at a biological level, and whether it’s doing anything that matters for patients. The patient goes in for a series of visits to the doctor, and gets a sequence of placebo or different doses of the drug, so each patient can serve as his or her own control for the experiment. Researchers are monitoring the patient’s speaking ability, handwriting, and lung volume—which are all movements controlled by fast-twitch muscle that deteriorates in patients with ALS. So far, the Cytokinetics drug looks to be well-tolerated. More details on whether the drug has any effect on muscle function are expected to be available by the end of the year, Blum says.

If this drug shows an ability to help ALS patients, then the company will move on to further clinical trials that could show things like whether the drug is able to keep patients’ diaphragms strong enough so they can stay off a ventilator for a longer period of time than without the drug, Blum says. Even without treating the underlying cause of disease, such a drug could help patients live longer.

“If you can get patients out of the hospital sooner, or prevent hospitalization, or prevent expensive procedures, we think there’s a strong [health economics] benefit,” Blum says.

It will take time for Cytokinetics to follow through on that kind of vision. And it will probably take even longer for the company to show some evidence that its muscle biology hypothesis is useful in another program, supported by Amgen, to help patients with heart failure.

How long this will take, Blum isn’t really saying. But he told me he understands what a few biotech entrepreneurs have said in these pages lately, lamenting the trend toward more cautious investment in biotech. By venturing out on the edge, Cytokinetics may toil in obscurity for years and then one day hit it big with something that matters.

“We’re building what we think will be a more durable business. Every day we see another specialty pharma company pivot on a single program, misfiring. Our business is not built on that model,” Blum says. “Our investors have bought into a longer-term vision.”

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