A closely watched heart drug from Amgen and Cytokinetics has both hit the primary endpoint on a major trial and dramatically disappointed investors.
In the 8,250-person study, the drug reduced the odds of hospitalization or other urgent care for heart failure by 8%, a statistically significant result. But the drug failed to help high-risk patients live longer, an endpoint that analysts had been following as the key marker for whether the drug could make a large impact.
“Trial technically worked,” Mizuho’s Salim Syed wrote in a note to investors, “but failed on what was really needed here to be a foundational medicine, in my view, which is CV death.”
Cytokinetics shares fell 43% on the news, from $23.66 to $15.33, wiping away a major spike the company had seen in May, when a MyoKardia drug with similarities proved successful on multiple endpoints. Amgen was down 4.6%, from $257.67 to $245.82.
The two companies have collaborated on cardiovascular R&D since 2007.
The drug, omecamtiv mecarbil, works by targeting myosin, a protein that converts chemical energy into mechanical force in the heart. The drug was supposed to activate the protein, helping the heart contract.
MyoKardia’s drug, mavacamten, also targeted myosin but inhibited it, helping correct a rare cardiomyopathy. The company, acquired this week by Bristol Myers Squibb, also has a myosin activator in Phase II.
This would not be the first significant disappointment for Cytokinetics, a company that has worn the vicissitudes of biotech over its 23-year history. The South San Francisco-based company invested in a series of ALS drugs over the last decade, only to see several fail major trials in the notoriously difficult-to-treat conditions.
In public statements, neither executives from Amgen and Cytokinetics touted the study as a success nor indicated they would submit for FDA approval. Cytokinetics’ R&D chief Fady Malik said the trial “provides insight” and that “they look forward to further data analyses.” Amgen R&D chief David Reese said simply they “further the understanding of treating heart failure.”
“The outcomes observed in GALACTIC-HF further the understanding of treating heart failure, a devastating disease in which half of heart failure patients will die within five years of initial hospitalization,” Reese said. “At Amgen, we remain committed to developing and delivering transformative medicines that improve the lives of patients with cardiovascular disease.”
Full data will be presented at the American Heart Association in November, but the early readout also spells bad news for one other company, Royalty Pharma. In 2017, the company paid $90 million for a 4.5% royalty.
Correction: An earlier version of this article misstated the terms of a royalty deal with RTW. It does not cover omecamtiv mecarbil.