Release Details

Trial Conducted in Collaboration with the National Cancer Institute and AstraZeneca

WALTHAM, Mass.--(BUSINESS WIRE)-- Cerulean Pharma Inc. (NASDAQ:CERU), a clinical-stage company developing nanoparticle-drug conjugates (NDCs), today announced that the first patient has been dosed in an open-label, single center Phase 1/2 clinical trial of its lead NDC candidate, CRLX101, in combination with LYNPARZA™ (olaparib) in patients with advanced solid tumors. This trial is being conducted by the National Cancer Institute (NCI), in collaboration with AstraZeneca and Cerulean. It is under the direction of NCI investigators, Anish Thomas, MBBS, M.D., Staff Clinician at the Thoracic and Gastrointestinal Oncology Branch of the Center for Cancer Research at the NCI, and Yves Pommier, M.D., Ph.D., an expert in DNA repair, and Chief of the Developmental Therapeutics Branch at the NCI.

There is strong scientific rationale for combining CRLX101, a topoisomerase inhibitor (Top1) NDC, with inhibitors of DNA damage response, including PARP inhibitors such as LYNPARZA. In preclinical studies, CRLX101 causes transient DNA damage in bone marrow and sustained DNA damage in tumors, consistent with the prolonged circulation and tumor targeting of CRLX101. By having a proper dosing schedule of CRLX101 and LYNPARZA, it may be possible to mitigate much of the combined bone marrow toxicity in the clinic, while achieving a combination benefit.

"Preclinical data on the use of PARP inhibitors in combination with chemotherapy supports the concept that PARP inhibitors intensify the effect of DNA damaging therapies. However, typically these combinations have been unsuccessful in previous clinical trials due to toxicities, specifically bone marrow toxicity," stated Dr. Anish Thomas. "The next step is to advance CRLX101 with LYNPARZA into the clinic, and we will gain valuable insights on the potential of this combination."

"AstraZeneca has the world's leading DNA Damage Response portfolio, a position bolstered by the first FDA-approved PARP inhibitor, LYNPARZA," said Susan Galbraith, Head of the Oncology Innovative Medicines Unit at AstraZeneca. "LYNPARZA has great potential beyond treating patients with BRCA mutations, and we are eager to explore possible synergistic combinations with other anti-cancer therapies. Based on data observed to date, we believe LYNPARZA in combination with CRLX101 has the potential to improve patient outcomes and toxicity concerns typically associated with PARP-Top1 inhibitor combinations."

"We currently are combining CRLX101 with angiogenesis inhibitors, chemotherapy, and now a PARP inhibitor, which speaks to the broad potential applicability and combinability of our NDCs," said Christopher D.T. Guiffre, President and Chief Executive Officer of Cerulean. "This trial represents our first step in a new frontier for CRLX101: DNA Damage Response. We look forward to top-line data from this trial in the first half of 2017."

The Phase 1 portion of this study will enroll up to 30 patients with advanced solid tumors that are resistant or refractory to standard therapy. The trial is designed to identify the maximum tolerated dose, or recommended Phase 2 dose of CRLX101 when combined with LYNPARZA, and to provide additional data on pharmacokinetics, pharmacodynamics and safety.

For patients interested in enrolling in this clinical trial, please call NCI's toll-free number: 1-800-4-Cancer (1-800-422-6237) (TTY: 1-800-332-8615) and/or visit: http://www.cancer.gov/.

About CRLX101

CRLX101 is a nanoparticle-drug conjugate (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 also inhibits hypoxia-inducible factor-1α (HIF-1α), which research suggests is a master regulator of cancer cell survival mechanisms. CRLX101 has shown activity in four different tumor types, both as monotherapy and in combination with other cancer treatments. CRLX101 is in Phase 2 clinical development and has been dosed in more than 350 patients. The U.S. FDA has granted CRLX101 Orphan Drug designation for the treatment of ovarian cancer and Fast Track designation in combination with Avastin^® in metastatic renal cell carcinoma.

About LYNPARZA (olaparib)

LYNPARZA is an innovative, first-in-class oral poly ADP-ribose polymerase (PARP) inhibitor that exploits tumour DNA repair pathway deficiencies to preferentially kill cancer cells. This mode of action gives LYNPARZA the potential for activity in a range of tumour types with DNA repair deficiencies. LYNPARZA is the first PARP inhibitor to be approved for patients with germline BRCA-mutated advanced ovarian cancer, and has been launched in the U.S. and Europe. In addition to ovarian cancer, AstraZeneca is investigating the full potential of olaparib in multiple tumour types, with Phase III studies in second line gastric cancer, BRCA-mutated pancreatic cancer and adjuvant and metastatic BRCA-mutated breast cancers underway.

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 NDC clinical 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 NDC clinical candidate, CRLX301, is in a Phase 1/2a clinical trial. For more information, please visit www.ceruleanrx.com.

About AstraZeneca

AstraZeneca is a global, innovation-driven biopharmaceutical business that focuses on the discovery, development and commercialisation of prescription medicines, primarily for the treatment of cardiovascular, metabolic, respiratory, inflammation, autoimmune, oncology, infection and neuroscience diseases. AstraZeneca operates in over 100 countries and its innovative medicines are used by millions of patients worldwide. For more information please visit: www.astrazeneca.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 the sufficiency of cash and cash equivalents to fund our operations, debt service and other scheduled expenditures and other statements containing the words "anticipate," "believe," "continue," "could," "estimate," "expect," "hypothesize," "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 and completion 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 May 2, 2016, 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 registered trademark of Genentech, Inc.

LYNPARZA is a trademark of the AstraZeneca group of companies.

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For Cerulean Pharma Inc.:
Nicole P. Jones, 781-209-6385
Director, Investor Relations and Corporate Communications
[email protected]

Source: Cerulean Pharma Inc.

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