NEW YORK — A handful of drugmakers are racing to develop a new class of medicines that they believe could be the biggest weapon against heart disease since statins were introduced in the 1980s.
The contest is taking place largely out of public view, but researchers and industry analysts say excitement is building over the injectable medicines. They work by blocking PCSK9, a protein whose natural function is to preserve levels of "bad" LDL cholesterol in the bloodstream.
Researchers in Paris, Montreal and Dallas, through experiments with thousands of patients that began in 1999, discovered the gene behind PCSK9 and deciphered its function.
Biotechnology company Regeneron and its big pharmaceutical partner Sanofi are now ahead of the pack in developing a drug that goes after PCSK9. A single injection of their medicine, called REGN727, slashed LDL levels by more than 60 percent for a month in an early trial. Results from mid-stage studies are expected later this year.
"The PCSK9 mechanism is perhaps one of the best examples we have in all of medicine of how the study of genetics can identify new targets for developing new therapies," said Dr. Daniel Rader, a noted heart researcher at the University of Pennsylvania.
Although no serious side effects have been seen with REGN727, larger trials are needed to rule out problems such as liver toxicity or raised blood pressure that have wrecked other promising heart drugs, including Pfizer Inc.'s experimental torcetrapib.
Rader, who is a consultant to several of the companies developing PCSK9 inhibitors, said most drugmakers are working on once-a-month injections that would likely be self-administered by patients using very small needles. Some drugs might eventually be administered every two to three months, he said.
Amgen Inc., Merck & Co., Alnylam Pharmaceuticals Inc. and a partnership between Bristol-Myers Squibb Co. and Isis Pharmaceuticals Inc. are in hot pursuit with rival drugs of their own.
The anti-PCSK9 medicines are expected to be used alongside statins, such as Pfizer's Lipitor and AstraZeneca's Crestor, for patients who do not achieve cholesterol-lowering goals with statins alone.
They would also be used on their own for a much smaller group who cannot tolerate statins — oral drugs with global annual sales of $20 billion that inhibit the liver's production of LDL cholesterol.
In the United States, more than 10 million patients could ultimately be candidates for the new family of medicines. Industry analysts say the products could command $1,000 a month or more because of their potential importance to high-risk patients. Statins cost about $130 a month for branded pills like Lipitor.