CASI Pharmaceuticals Announces First Patient Dosed In Phase 2 Trial For ENMD-2076 In Fibrolamellar Carcinoma (FLC)

Nov 04, 2015, 07:00 ET from CASI Pharmaceuticals, Inc.

ROCKVILLE, Md., Nov. 4, 2015 /PRNewswire/ -- CASI Pharmaceuticals, Inc. (Nasdaq: CASI), a biopharmaceutical company dedicated to innovative therapeutics addressing cancer and other unmet medical needs, announces the dosing of the first patient in a Phase 2 clinical trial in patients with advanced fibrolamellar carcinoma (FLC) at Memorial Sloan-Kettering Cancer Center. The trial entitled "A Phase 2 Multi-center, Open-label Study of Oral ENMD-2076 for the Treatment of Patients with Advanced Fibrolamellar Carcinoma (FLC)" will be evaluating the safety and efficacy of ENMD-2076, an orally-active Aurora A/angiogenic kinase inhibitor with a unique kinase selectivity profile and multiple mechanisms of action. More information about the trial can be found at

Rong Chen, M.D., CASI's Chief Medical Officer, commented, "Fibrolamellar carcinoma is a rare malignant neoplasm of the liver and is often advanced when diagnosed due to lack of symptoms. It has distinct clinical, histological and prognostic features from conventional hepatocellular carcinoma (HCC).  Because of the current absence of first line treatment options, we feel ENMD-2076 can be a viable candidate to potentially fill a currently unmet medical need and become a first line therapy in the treatment of FLC patients."

Ken Ren, Ph.D., CASI's Chief Executive Officer, commented, "We are excited to further evaluate ENMD-2076 in the treatment of FLC. In our previous Phase 1 trial, we observed therapeutic effects of ENMD-2076 with a FLC patient.  More recently, we also observed its strong anti-tumor activities in a FLC patient derived xenograft (PDX) mouse model, in which tumor volume in the untreated group increased 500% compared to a 60% decrease in tumor volume in the ENMD-2076 treatment group during the 31-day study period.  We believe there is a good correlation between a clearly identified tumor genesis drive gene mutation and the potential sensitivity of the patients to ENMD-2076 therapy, which targets Aurora A, VEGF, and FGFR.  We are optimistic about the drug's potential in the treatment of FLC and look forward to advancing its development."

About ENMD-2076

ENMD-2076 is an orally-active, Aurora A/angiogenic kinase inhibitor with a unique kinase selectivity profile and multiple mechanisms of action. ENMD-2076 has been shown to inhibit a distinct profile of angiogenic tyrosine kinase targets in addition to the Aurora A kinase. Aurora kinases are key regulators of mitosis (cell division), and are often over-expressed in human cancers. ENMD-2076 also targets the VEGFR, Flt-3 and FGFR3 kinases, which have been shown to play important roles in the pathology of several cancers. ENMD-2076 has shown promising activity in Phase 1 clinical trials in solid tumors including ovarian, breast, liver, renal and sarcoma, as well as in leukemia and multiple myeloma. ENMD-2076 is currently in Phase 2 clinical trials in multiple indications, including triple-negative breast cancer, soft tissue sarcoma, ovarian clear cell carcinomas and fibrolamellar carcinoma. ENMD-2076 has received orphan drug designation from the U.S. FDA for the treatment of ovarian cancer, multiple myeloma, acute myeloid leukemia, and hepatocellular carcinoma.  ENMD-2076 has received orphan drug designation from the European Medicines Agency Committee for Orphan Medicinal Products for the treatment of hepatocellular carcinoma, including fibrolamellar carcinoma.

About CASI Pharmaceuticals, Inc.

CASI is a biopharmaceutical company dedicated to the acquisition, development and commercialization of innovative therapeutics addressing cancer and other unmet medical needs for the global market with a commercial focus on China. CASI's product pipeline includes exclusive rights to ZEVALIN® (ibritumomab tiuxetan), MARQIBO® (vinCRIStine sulfate LIPOSOME injection) and EVOMELA™ (CE-Melphalan HCI for injection) for greater China (including Taiwan, Hong Kong and Macau). CASI's development pipeline also includes its proprietary drug candidate ENMD-2076, a selective angiogenic kinase inhibitor currently in multiple Phase 2 oncology studies, and 2ME2 (2-methoxyestradial) currently under reformulation development. CASI is headquartered in Rockville, Maryland and has a wholly owned subsidiary and R&D operations in Beijing, China. More information on CASI is available at and in the Company's filings with the U.S. Securities and Exchange Commission.

Forward Looking Statements

This news release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act with respect to the outlook for expectations for future financial or business performance, strategies, expectations and goals, including without limitation with respect to our clinical trials and anticipated results.  Forward-looking statements are subject to numerous assumptions, risks and uncertainties, which change over time. Forward-looking statements speak only as of the date they are made, and no duty to update forward-looking statements is assumed.  Actual results could differ materially from those currently anticipated due to a number of factors, including: the risk that we may be unable to continue as a going concern as a result of our inability to raise sufficient capital for our operational needs; the possibility that we may be delisted from trading on the Nasdaq Capital Market; the volatility in the market price of our common stock; the difficulty of executing our business strategy in China; our inability to enter into strategic partnerships for the development, commercialization, manufacturing and distribution of our proposed product candidate or future candidates; uncertainties relating to preclinical and clinical trials, including delays to the commencement of such trials and their expected outcomes; risks relating to the need for additional capital and the uncertainty of securing additional funding on favorable terms; risks associated with our product candidates; risks associated with any early-stage products under development; the risk that results in preclinical models are not necessarily indicative of clinical results; uncertainties relating to preclinical and clinical trials, including delays to the commencement of such trials; the lack of success in the clinical development of any of our products; dependence on third parties; risks relating to the commercialization, if any, of our proposed products (such as marketing, safety, regulatory, patent, product liability, supply, competition and other risks); risks relating to interests of our largest stockholders that differ from our other stockholders; and the risk of substantial dilution of existing stockholders in future stock issuances.  Such factors, among others, could have a material adverse effect upon our business, results of operations and financial condition.  We caution readers not to place undue reliance on any forward-looking statements, which only speak as of the date made. Additional information about the factors and risks that could affect our business, financial condition and results of operations, are contained in our filings with the U.S. Securities and Exchange Commission, which are available at

ZEVALIN® and MARQIBO® and EVOMELA™ are proprietary to Spectrum Pharmaceuticals, Inc. and its affiliates. 



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