Cerulean Announces First Patient Dosed with CRLX301 in Phase 1/2a Trial in Advanced Solid Tumors

December 17, 2014 08:30 AM Eastern Standard Time

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Cerulean Pharma Inc. (Nasdaq: CERU), a leader in Dynamic Tumor Targeting™, today announced that the first patient has been dosed with CRLX301 in Cerulean's Phase 1/2a trial in patients with advanced solid tumor malignancies. CRLX301 is a nanoparticle-drug conjugate, or NDC, with a docetaxel payload, and it is the second clinical candidate from Cerulean's Dynamic Tumor Targeting Platform.

"CRLX301 was superior to docetaxel in seven of seven animal models, with a statistically significant survival benefit in five of those preclinical models," said Paul Friedman, M.D., Executive Chairman of Cerulean. "We also saw preclinical tolerability that is consistent with our expectations of the platform -- specifically, improved tolerability relative to published data with docetaxel. We look forward to learning if these preclinical results will translate in the clinic."

Edward Garmey, M.D., Chief Medical Officer of Cerulean added, "During the course of the CRLX301 clinical development program, we will carefully assess the investigational spaces in which currently approved taxanes can be improved through the targeted delivery of the cytotoxic payload to tumor cells and the relative sparing of healthy cells. We also are interested in those settings where taxanes haven't been approved but where our NDC technology might facilitate their use, either alone or in combination."

This is the first clinical trial of CRLX301. The Phase 1/2a trial is commencing in Australia and is expected to expand to the U.S. The Phase 1 portion is a single-arm, open-label, dose-escalation study of CRLX301 in up to 36 patients with advanced solid tumor malignancies that is designed to identify the maximum tolerated dose and establish the recommended Phase 2 dose of CRLX301 when administered by intravenous infusion once every three weeks. The Phase 2a portion will be an expansion cohort that will enroll an additional 24 patients. The primary endpoints are pharmacokinetics, safety and tolerability of CRLX301. Preliminary evidence of efficacy also will be evaluated.

About CRLX301

CRLX301 is a dynamically tumor-targeted nanoparticle-drug conjugate (NDC) designed to concentrate in tumors and slowly release its anti-cancer payload, docetaxel, inside tumor cells. In preclinical studies, CRLX301 delivers up to 10 times more docetaxel into tumors, compared to an equivalent milligram dose of commercially available docetaxel and was superior to docetaxel in seven of seven animal models, with a statistically significant survival benefit seen in five of those seven models. In addition, preclinical data show that CRLX301 had lower toxicity than has been reported with docetaxel in similar preclinical studies.

About CRLX101

CRLX101 is a dynamically tumor-targeted NDC designed to concentrate in tumors and slowly release its anti-cancer payload, camptothecin, inside tumor cells. CRLX101 inhibits topoisomerase 1 (topo 1), which is involved in cellular replication, and hypoxia-inducible factor-1α (HIF-1α), which research suggests is a master regulator of cancer cell survival mechanisms thought to promote drug and radiation resistance. CRLX101 has shown activity in late-stage disease in three different tumor types, both as monotherapy and in combination with Avastin®. CRLX101 is currently in Phase 2 clinical development and has been dosed in more than 250 patients.

About Cerulean's Dynamic Tumor Targeting™ Platform

Cerulean's Dynamic Tumor Targeting Platform creates NDCs that are designed to provide safer and more effective cancer treatments. We believe our NDCs concentrate their anti-cancer payloads inside tumors while sparing normal tissue because they are small enough to pass through the "leaky" vasculature present in tumors but are too large to pass through the wall of healthy blood vessels. Once inside tumors, our NDCs enter tumor cells where they slowly release anti-cancer payloads from within the tumor cells.

About Cerulean Pharma

The Cerulean team is committed to improving treatment for people living with cancer. We apply our Dynamic Tumor Targeting Platform to create a portfolio of NDCs designed to selectively attack tumor cells, reduce toxicity by sparing the body's normal cells, and enable therapeutic combinations. Our first platform-generated candidate, CRLX101, is in multiple clinical trials in combination with other cancer treatments, all of which aim to unlock the power of combination therapy. Our second platform-generated candidate, CRLX301, is in a Phase 1/2a clinical trial. For more information, please visit www.ceruleanrx.com.

Cautionary Note on Forward Looking Statements

Any statements in this press release about our future expectations, plans and prospects, including statements about the clinical development of our product candidates, statements about our estimated research and development expenses and sufficiency of cash to fund specified use of cash and other statements containing the words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "would," and similar expressions, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: the uncertainties inherent in the initiation of clinical trials, availability and timing of data from ongoing and future clinical trials and the results of such trials, whether preliminary results from a clinical trial will be predictive of the final results of that trial or whether results of early clinical trials will be indicative of the results of later clinical trials, expectations for regulatory approvals, availability of funding sufficient for our foreseeable and unforeseeable operating expenses and capital expenditure requirements and other factors discussed in the "Risk Factors" section of our Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on November 13, 2014 and in other filings that we make with the Securities and Exchange Commission. In addition, any forward-looking statements included in this press release represent our views only as of the date of this release and should not be relied upon as representing our views as of any subsequent date. We specifically disclaim any obligation to update any forward-looking statements included in this press release.

Avastin® is a trademark of Genentech Inc.

Cerulean Pharma Inc.
Aurora Krause, 617-551-9627
Corporate Communications
[email protected]