One day this month, Cambridge, MA-based Cerulean Pharma will get the answers to two important questions that few private, venture-backed biotech companies ever get far enough to answer.
It will find out if it has a legit new technology platform for fighting cancer, and whether it has a drug that helps cancer patients live longer.
The moment of truth is days away for Cerulean, the developer of nanoparticle-based cancer drugs, founded in 2006 with technology from MIT and Caltech. Cerulean, after raising $84 million in venture capital, is preparing to rip off the blind from a mid-stage clinical trial of 157 patients with lung cancer who were randomly assigned to get its drug (CRLX101) and best supportive care, or best supportive care alone.
While many private biotechs at this early stage of the game use squishy, subjective “surrogate” goals designed to save time and money while offering a hint of whether the new drug works, Cerulean is asking the most important and toughest question straight away. The study will tell the company whether its drug can improve survival time for very sick lung cancer patients whose tumors have spread after receiving one or two prior therapies.
If Cerulean can help those patients, that will have big implications for its business. The company will be in position to race ahead with unusual confidence in the third and final phase of clinical trials normally required for FDA approval of new drugs. It will also clearly be in a bargaining position of strength, with options to strike a big new partnership, go public, or raise a lot more private money to plow into its R&D pipeline. A positive trial would also clearly be a big win for Cerulean’s venture backers, which include Polaris Venture Partners, Venrock, Lilly Ventures, Lux Capital, Crown Venture Management, and Bessemer Venture Partners.
By the same token, if it fails in this study, it will have a lot of explaining to do, and will be in a position to make some hard choices.
“There’s no question, this is a big value inflection point for us,” says Cerulean CEO Oliver Fetzer.
If the Cerulean drug passes this test, it will also be a validating event for a nanoparticle drug delivery platform that the firm believes has potential in treating a wide variety of tumor types. Cerulean wouldn’t be the first company striving to deliver potent anti-tumor drugs through nanoparticles—Celgene’s protein-bound paclitaxel (Abraxane) is one example on the market—but its technology could enable Cerulean to bring nanoparticles to a bigger audience of physicians and patients in the cancer world.
For those new to the story, Cerulean’s nanoparticle is a cyclodextrin-based polymer that’s linked to a highly potent chemo agent called camptothecin. Although the prefix “nano” in nanoparticle makes it sounds super-small, the whole package is actually large by cancer drug standards. The injectable
