CASI Pharmaceuticals Receives EU Orphan Drug Designation For The Use Of ENMD-2076 To Treat Hepatocellular Carcinoma Including Fibrolamellar Carcinoma

ROCKVILLE, Md., Oct. 14, 2015 /PRNewswire/ -- CASI Pharmaceuticals, Inc. (Nasdaq: CASI), a biopharmaceutical company dedicated to innovative therapeutics addressing cancer and other unmet medical needs, announces that its oncology drug candidate, ENMD-2076, has received Orphan Drug designation from the European Medicines Agency (EMA) for the treatment of hepatocellular carcinoma (HCC), including fibrolamellar carcinoma (FLC), a rare type of HCC.

The designation provides CASI with 10 years of market exclusivity in EU after ENMD-2076 receives marketing authorization there. The product was also granted Orphan Drug designation for the treatment of HCC by US Food and Drug Administration (FDA) in 2014, which provides CASI with a 7-year market exclusivity in the country after its New Drug Application approval.

Dr. Ken Ren, Chief Executive Officer of CASI, commented, “We are very pleased with the Orphan Drug designation in the EU for HCC. This is an important regulatory milestone for us to enhance the commercial value of ENMD-2076 for global market. We are in the process of initiating our phase 2 clinical trials in the US for the treatment of FLC and expecting to start patient recruitment very soon. We will seek regulatory approvals and expand the trial into EU once a signal of clinical benefits is indicated in our US trial. As a part of our global development plan, we also have clinical trial application filed with Chinese FDA pending for approval for FLC. We are quite optimistic and committed to the development of ENMD-2076 as a first line therapy for the treatment of FLC and look forward to advancing its development to the next step.”

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.

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 www.casipharmaceuticals.com 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 the closing of the private placement offering and the anticipated use of the net proceeds. 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 the closing of the private placement offering does not occur, 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; risks relating to interests of our largest stockholders that differ from our other stockholders; the risk of substantial dilution of existing stockholders in future stock issuances, including as a result of the closing of the private placement offering; 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; 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; and risks relating to the commercialization, if any, of our proposed products (such as marketing, safety, regulatory, patent, product liability, supply, competition and other risks). 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 www.sec.gov.

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

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