UPDATED: Cerulean Pharma's lead nanodrug crashes in lung cancer study

Oliver Fetzer, CEO of Cerulean Pharma

After raising more than $80 million in venture money, Cerulean Pharma failed its big test. The biotech's lead candidate crashed and burned in a Phase IIb study, missing an ambitious main endpoint of overall survival in patients with non-small cell lung cancer. With the most advanced study of its key compound CRLX101 ending in failure, the Cambridge, MA-based company has a lot riding on four other clinical trials of the experimental therapy.

Oliver Fetzer, CEO of Cerulean (a 2011 Fierce 15 company), set out to make a statement about the potential of CRLX101 with overall survival--rather than weaker endpoints such as progression-free survival--as the main goal of the Phase IIb trial in lung cancer patients. Success would have raised the profile of CRLX101, a feat of nanotechnology that marries trace mounts of the anticancer toxin camptothecin with a polymer molecule, designed to infiltrate leaky tumor vessels and kill cancer while limiting effects on healthy tissues.

In the Phase IIb study of 157 patients with previously treated lung cancer, those on CRLX101 showed in available top-line data signs of anticancer activity such as reduced tumor sizes. The trial took place in the Ukraine and Russia, comparing treatment with Cerulean's candidate and best supportive care (BSC) with BSC alone, according to the company. The company measured but did not disclose detailed results on the other main endpoints of safety and tolerability and secondary measures such as progression-free survival and objective tumor response.

"Late-stage lung cancer is a very difficult disease to treat, and we are disappointed that we did not achieve our ambitious goal of improving survival for patients who have limited treatment options," Fetzer, a former Cubist Pharmaceuticals ($CBST) executive, stated.

Any signal of activity might raise hopes for other studies of CRLX101, which Cerulean is trialing in combination with Roche's ($RHHBY) Avastin for kidney cancer and as a standalone treatment for ovarian, gastric and small cell lung cancers in collaboration with academics. Next month the company plans to report data from the study of the drug teamed with Avastin in a form of kidney cancer known as renal cell carcinoma at the annual meeting of the American Association of Cancer Research.

The failure of the midstage study means Cerulean's plans to secure a partner to advance its lead drug into Phase III in NSCLC patients have fallen through. It's backed by the venture outfits Polaris Venture Partners, Lux Capital, Lilly Ventures, Bessemer Venture Partners, Venrock Associates and CVF. And Fetzer expects their continued support as he plots out the future of the company. 

"Clearly we need to do an additional financing and we're going to do that in the not-too-distant future," Fetzer told FierceBiotech in an interview. "So that's clearly going to be done. I think we have to look at it in the context of analyzing the data out of the Phase II trial, looking at how partnership discussions are going, and we have a very small investor syndicate. So what I'm doing is looking at the different options of how we put that together."

Despite the setback in the NSCLC study, Fetzer says that his faith in CRLX101 hasn't faded and he noted that many successful oncology drugs failed in studies before finding success in later trials.

- here's the release

Editor's note: This story has been updated with comments from CEO Oliver Fetzer.

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