Drugmakers desperate for innovative medicines are courting a tiny French company whose lab holds a product that may transform the treatment of heart attacks.
Cerenis Therapeutics SA, a closely held company, is testing a compound in a new class of drugs that mimic high-density lipoprotein, also known as good cholesterol, to purge arteries clogged by plaque.
Cerenis is the brainchild of Jean-Louis Dasseux, a biochemist who spent the past 25 years studying HDL’s ability to ferry fatty substances away from artery walls. His most advanced medicine, dubbed CER-001, last month entered mid-level clinical trials. Cerenis is overseeing the tests, having turned down collaboration offers from bigger drugmakers.
“If it works, this will be huge,” said Denis Lucquin, managing partner and chairman of Sofinnova Partners, a venture capital firm that owns a stake in Cerenis. “Lots of pharmaceutical companies are sniffing around.”
Some of the suitors were among the world’s largest drugmakers, Dasseux said in an interview in Paris. Three approached Cerenis with a written proposal, he said. He declined to name the companies, as did Lucquin.
Dasseux figures Cerenis will be on firmer footing to negotiate a deal or sell shares if it can show its way of imitating the body’s purging mechanism works to combat cardiovascular disease, the world’s biggest killer. When fatty substances coat the arteries, a process called atherosclerosis, they narrow the passage and pieces of the plaque lining can come loose, interrupting blood flow to the brain or the heart.
“We think we can go a lot further with this product,” the 52-year-old executive said in an interview over a cup of espresso at Cafe de la Paix, near the Paris Opera house. “At the end of the phase 2, if we have plaque regression, this will be a major event. There won’t be too many difficulties at that point to raise funds, either through a stock market listing, private financing, or through partnerships and alliances.”
Dasseux estimates the drug could reach pharmacy shelves in 2017 as a temporary medicine to avert repeat heart attacks and stroke, and fetch between $5 billion and $8 billion in sales annually by 2020 or 2021 in that indication. He figures the mid- stage tests will be over by the end of next year.
Cerenis said last month it began testing CER-001 on more than 500 people in the U.S., Canada and Europe in the second of three stages of research needed for approval to sell a drug. The researchers are trying to show that taking six infusions of the man-made protein over five weeks after a heart attack or stroke will raise the number of HDL particles in the blood, reducing the risk of another incident, Dasseux said.
An early clinical test designed to check the drug’s safety on 32 volunteers also showed cholesterol moving away from the artery walls. The compound mimics the most efficient good- cholesterol particles, small discs known as nascent HDL that swirl around in the bloodstream, harvesting fatty deposits from within the artery wall. As the discs swell with the lipid load, they turn into a more mature form of HDL that the liver can recognize, ensuring the fatty harvest gets recycled or eliminated by the body.
The rewards may be big. Worldwide sales in the cholesterol market topped $35 billion in 2009, according to the research firm IMS Health Inc. Atherosclerosis is the main cause of cardiovascular disease, which may kill 23.6 million people a year by 2030 by World Health Organization estimates.
If CER-001 can reverse the buildup of plaque, “this could change the paradigm for the treatment of atherosclerosis,” said Jean-Claude Tardif, director of the research center at the Montreal Heart Institute. Tardif is the lead investigator for the mid-stage clinical tests.
‘Next Big Thing’
Dasseux, a biochemist with an MBA from the University of Michigan, founded Cerenis in 2005 to develop compounds inspired by HDL’s mechanism after working on a rougher version at another startup, Esperion Therapeutics Inc. Esperion was bought by Pfizer Inc. (PFE) for $1.3 billion in 2004. The maker of the best- selling pill Lipitor has since cut back on cholesterol research and the Dasseux compound’s development was dropped in 2008.
Still, Cerenis isn’t the only one pursuing HDL. After years spent working on drugs that reduced so-called bad cholesterol, drugmakers including Merck & Co. are investigating ways to boost the good type instead.
“HDL is the next big thing” in the field of cholesterol research, Les Funtleyder, a health-care portfolio manager at Miller Tabak & Co. in New York, said in a telephone interview.
Merck is developing anacetrapib, an experimental medicine in the final stages of testing billed as a potential blockbuster after data presented in November showed it could boost good cholesterol by an unprecedented 138 percent.
Dasseux says Cerenis won’t compete with anacetrapib or a rival product by Roche Holding AG (ROG) called dalcetrapib because they work differently, targeting a protein that helps good cholesterol go bad. Also, his drug targets patients who, in the weeks after having suffered a heart attack or stroke, must avert another.
People who survive a heart attack face the greatest risk of dying from sudden cardiac death during the first month after leaving the hospital, according to a study by the Mayo Clinic.
After a few weeks, patients will probably stop using CER- 001 and turn to other drugs to prevent further cholesterol accumulation, according to Dasseux.
Aside from Sofinnova, Cerenis investors include HealthCap, Alta Partners, TVM Capital, EDF Ventures, OrbiMed and Daiwa Corporate Investment. The company last year raised 50 million euros ($71 million) to fund CER-001’s development. It has enough cash until the beginning of 2014, according to Dasseux.
Cerenis, based in Toulouse, France, and Ann Arbor, Michigan, in July added former GlaxoSmithKline Plc (GSK) Chief Executive Jean-Pierre Garnier to its board. The company is also studying CER-001 for the treatment of a genetic disorder characterized by high cholesterol, a rare disease that Dasseux says may warrant an accelerated regulatory review, and has five other HDL therapies in its labs.
The medicine, if it works in combination with other heart drugs, may become “a multi-billion-dollar treatment,” said Funtleyder of Miller Tabak.
Others are more sceptical because the anti-cholesterol market is getting crowded, experienced drugmakers like Pfizer have stumbled and the Cerenis medicine is only just entering mid-stage tests.
“It’s much too early to assess the likelihood of success,” Steven Nissen, head of cardiology at the Cleveland Clinic in Ohio, said in e-mailed comments. The field “is getting more crowded. Cerenis will need strong Phase 2 data to stay competitive.”
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