Bristol Myers stumbles in bid to widen heart drug’s use

Camzyos’ failure in a form of hypertrophic cardiomyopathy dampened its commercial outlook and spurred debate as to whether other drugs like it would similarly struggle in testing.

Bristol Myers stumbles in bid to widen heart drug’s use

Dive Brief:

• Bristol Myers Squibb on Monday said its drug Camzyos failed a Phase 3 trial in people with a progressive heart condition, closing off an opportunity to expand use of a medicine it sees as a future blockbuster.

• According to Bristol Myers, Camzyos missed the dual main goals of a study focused on the non-obstructive form of “HCM,” or hypertrophic cardiomyopathy. It failed to meaningfully improve peak oxygen consumption as well as scores on an assessment of heart health. The company didn’t provide study details, but said more information will be shared “with the scientific community in the future.”

• Camzyos was acquired through the $13 billion buyout of MyoKardia in 2020 and two years later became the first drug cleared for use in the “obstructive” and more common form of the disease. Biotechnology companies Cytokinetics and Edgewise Therapeutics are developing similar medicines that are both in the advanced stages of clinical testing. Cytokinetics’ drug, aficamten, could be approved in the U.S. later this year.

  Dive Insight:

  Camzyos’ setback carries implications for Bristol Myers as well as the companies trying to develop better medicines for HCM, a chronic condition characterized by a potentially deadly stiffening of the heart muscle.

  Bristol Myers is bracing for the losses of billions of dollars in yearly revenue when the patents expire for its cancer immunotherapy Opdivo and blood thinner Eliquis. The company already announced a cost-cutting plan under CEO Chris Boerner, and is looking to a handful of medicines, Camzyos among them, to grow sales in the years ahead.

  Camzyos generated $602 million in sales in 2024. But Bristol Myers has said that number could reach $4 billion in 2029, and one way to get there was a clearance in the non-obstructive setting, which is estimated to account for about a third of the people with the condition. The setback is “a disappointment,” wrote Leerink Partners analyst David Risinger, though Bristol Myers could rebound if a series of coming Phase 3 readouts are successful.

  The study failure also stirred debate among analysts as to whether drugs like Camzyos, known as cardiac myosin inhibitors, can help all people with HCM. Camzyos has proven beneficial for the obstructive form that impedes blood flow and can lead to heart failure as well as other potentially serious complications. The non-obstructive type doesn’t, but can still put people at risk of poor health outcomes. The new study tested whether Camzyos might be similarly helpful for those people as well.

  The fact that Camzyos wasn’t suggests obstructive and non-obstructive HCM are “two unique diseases,” said Milind Desai, the director of the Cleveland Clinic’s HCM Center, in a statement provided by Bristol Myers.

  Analysts were mixed on whether the cardiac myosin inhibitors Cytokinetics and Edgewise are developing might similarly struggle in non-obstructive HCM. Cytokinetics’ drug, aficamten, is under Food and Drug Administration review in obstructive HCM and in a Phase 3 trial in non-obstructive disease. Edgewise’s EDG-7500 could join it in late-stage testing next year.

  Matt Phipps, of the investment bank William Blair, wrote that Camzyos’ shaky performance in the latest study — as well as Phase 2 testing in non-obstructive HCM patients — “may be more attributed to disease biology,” which has negative implications for the whole drug class.

  That form of the disease is also more heterogeneous, making it tougher for a drug to succeed in testing, added RBC Capital Markets’ Leonid Timashev.

  Still, there are key differences between the drugs and their development plans that leave some analysts optimistic about Cytokinetics and Edgewise’s chances. Aficamten appeared to perform better in earlier testing in non-obstructive patients, and Cytokinetics’ Phase 3 study is designed differently, wrote Mizuho Securities’ Salim Syed. RBC’s Timashev added that EDG-7500’s “unique mechanism” could “overcome the limitations” of other medicines in non-obstructive HCM.

  “We now see a 2-handed market” split between Cytokinetics and Edgewise, Timashev wrote.