Ambitious HDL program joins a growing list of high-profile clinical failures

Cerenis raised about $60 million to undertake an ambitious mid-stage program of its lead therapy: an agent that was designed to work just like HDL, capable of scouring arteries of the plaque that threatens to trigger cardiovascular events in high-risk patients. CEO Jean-Louis Dasseux was so confident he had found the magic HDL bullet that he told reporters and a group of investors that included Sofinnova and OrbiMed that he would hold off on any pharma deal for CER-001 until he could prove that the drug worked in a key trial.

But like other high-profile HDL therapies that came before it, CER-001 failed.

The French biotech reports that in the trial--which recruited more than 500 acute-coronary patients--the mimetic therapy failed to hit the primary endpoint: reducing the lipid-carrying plaque in the arteries by a statistically significant amount compared to a placebo. Touching only on top-line results, the biotech said that one of three doses in the study did achieve "nominal statistical significance versus placebo" in the "modified Per Protocol population" of 295 patients.

Dasseux, an experienced investigator with decades of work in the field, had gathered an impressive group of backers for the company. But HDL cardiovascular remedies have defied even the biggest companies, like Roche ($RHHBY) with dalcetrapib and Pfizer ($PFE) with torcetrapib. The NIH failed as well with the HDL-boosting Niaspan. Merck ($MRK) and Eli Lilly ($LLY), though, have remained committed to their programs, despite significant skepticism that an HDL approach can be both safe and effective in reducing the threat of a cardiovascular incident.

The potential rewards, though, have been painted with megablockbuster colors. Peak sales projections have started around $5 billion and range up to $10 billion--a holy grail figure for the R&D field. Dasseux, who's been featured in the Fierce 15 and Red Herring, has put the number at $8 billion.

Cerenis isn't walking away, either.

"The apparent demonstration of benefit at one dose level versus placebo in our analysis is encouraging," said investigator Stephen Nicholls in a statement. "CER-001 warrants further study in clinical trials to evaluate its potential benefits for patients with cardiovascular disease."

- here's the release

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