Cerenis Therapeutics
Based: Toulouse, France and Ann Arbor, MI
Founded: 2005
Website: www.cerenis.com
CEO: Jean-Louis Dasseux

The Scoop: Who says the blockbuster is dead? Certainly no one at Cerenis Therapeutics. The French-American biotech has its sights set on an ongoing mid-stage study of its own carefully crafted high-density lipoprotein. It's an HDL mime designed to do what natural HDL can do: Scour arteries of plaque and reduce mortality in acute cardio patients facing a second or third heart attack. If CER-001 works without killing anyone along the way, a new class of drugs like this could tackle a mega-blockbuster market. "Huge" was the word Denis Lucquin, managing partner of Sofinnova and a committed investor, chose when Bloomberg came calling some months ago.

What Makes It Fierce: CEO Jean-Louis Dasseux, who's been working in this field for more than two decades, might think "huge" doesn't quite do justice to the potential of the lead drug. And this is a topic he's devoted considerable thought to. He set up Fournier's research center in Heidelberg and then went on to work as VP of chemistry and technology at Esperion Therapeutics.

At Esperion, he worked with a team that produced enough HDL particles to run a proof-of-concept study that spurred plaque regression in weeks. But their production costs were unrealistic and the program never made it past the proof-of-concept stage. After Pfizer ($PFE) bought out Esperion for $1.3 billion, later killing the HDL project, he went back to the drawing board and launched Cerenis in 2005.

"We started to think what could be done," recalls Dasseux. "Now we know much more about the protein and HDL, so we started from scratch, integrating the knowledge, and produced the protein in a different organism, a mammalian cell. Now it's easier to purify and the cost of production is therefore much lower."

Last spring, Cerenis launched a mid-stage clinical trial of CER-001, planning to enroll 500 patients with acute coronary syndrome--right after a heart attack--at 50 sites around the globe. The objective is simple: Demonstrate a reduction in atherosclerotic plaque, with 6 weekly intravenous infusions of three different doses.

Success and a clean safety profile would poise Cerenis to do a major deal with one of the Big Pharma companies eyeing the biotech's progress. And Dasseux clearly wants to be involved in pivotal late-stage trials. After all these years laboring in the lab with early-phase studies, he believes he can hatch a major new drug.

"Now," says the CEO, "I want to go all the way to the market."

He has the money to make it work. The biotech's last round raised $72 million, giving it enough cash to fund its work into 2014, finishing Phase II next year and advance some of its other programs along the way.

Cerenis isn't the only company at work on HDL. Despite some serious missteps along the way at Pfizer, which experienced a catastrophic failure with torcetrapib, both Merck ($MRK) (anacetrapib, a promising CETP inhibitor) and Roche (dalcetrapib, positive in Phase II) have been patiently at work in the clinic on HDL therapies. But they work on a different mechanism and won't likely interfere with Dasseux's goal of providing a first-in-class intervention therapy for heart attack victims.

Venture Backers: IRDI-IXO, Strategic Investment Fund, Sofinnova Partners, HealthCap, Alta Partners, TVM Capital, EDF Ventures, OrbiMed and DAIWA Corporate Investment.