Alnylam encouraged by trial
Blood pressure drug has positive results
An experimental medicine from Cambridge-based Alnylam and the Swiss drug giant Roche lowered high blood pressure when used in combination with current treatments in a clinical trial, the companies said Tuesday.
Full results of the study, which tested the medicine called zilebesiran, will be presented at the American College of Cardiology’s Annual Scientific Session, which starts on April 6 in Atlanta, the companies said in statements. The treatment calls for the medicine to be injected twice a year.
Simon Fox, an Alnylam vice president, said zilebesiran demonstrated “clinically significant” benefits to recipients in a study of 672 adults with mild to moderate hypertension that was not adequately controlled by one of three current generic medicines.
The benefits on top of the three medicines were sustained for up to six months after a single injection.
Roche expressed high hopes for the experimental treatment.
“With twice-yearly dosing in combination with standard of care medication, zilebesiran has strong potential to sustain lower blood pressure and reduce the risk of stroke, heart attack, and death that can result from inadequate treatment,” said Dr. Levi Garraway, Roche’s chief medical officer.
The business partners are beginning another trial of the drug as an add-on treatment in adults who take two to four standard medications for high blood pressure but still have uncontrolled hypertension and high cardiovascular risk.
Myles Minter, an analyst for William Blair, said in a note to investors that Al
nylam’s press release was “light on details.” Nonetheless, he wrote, “We view zilebesiran as a potentially significant opportunity given estimates that one in three adults are living with hypertension worldwide.”
About 1.3 billion people worldwide live with hypertension, according to the World Health Organization, or roughly one in three adults. Up to 80 percent of people with high blood pressure don’t have the condition under control despite the availability of several classes of medicines to treat it.
High blood pressure is a leading risk factor for cardiovascular disease and causes over 10 million deaths worldwide each year but is often overlooked because it typically doesn’t produce symptoms, according to the Centers for Disease Control and Prevention. Left untreated, it can lead to heart disease, stroke, and kidney failure.
In July, Roche agreed to develop and commercialize Alnylam’s zilebesiran for hypertension, and paid Alnylam $310 million upfront. The deal is potentially worth up to $2.8 billion to Alnylam if the medicine reaches certain goals.
Like five other medicines developed by Alnylam and approved by the Food and Drug Administration since 2018, the hypertension drug relies on RNA interference, or RNAi, to correct mistakes in the genetic code that lead to disease. Craig Mello, a professor at the University of Massachusetts Medical School, and another American scientist, was given the Nobel Prize in 2006 in medicine or physiology for pioneering research into RNAi, which can be programmed to find a genetic defect and silence it.
Until zilebesiran, Alnylam has mostly focused on drugs that treat rare diseases. If the business partners succeed in getting regulators to approve a twice-a-year injectable treatment for high blood pressure, it would represent a significant pivot for Alnylam.