CASI Pharma Met Endpoint in Phase II ENMD-2076 in Fibrolamellar Carcinoma

ROCKVILLE, Md., Aug. 16, 2016 /PRNewswire/ -- CASI Pharmaceuticals, Inc. (Nasdaq: CASI), a biopharmaceutical company dedicated to innovative therapeutics addressing cancer and other unmet medical needs, today announced that its Phase 2 trial of ENMD-2076 in fibrolamellar carcinoma (FLC) has met its Stage 1 endpoint of objective response for non-futility based on the pre-specified interim analysis criteria and will advance to Stage 2 development.

Rong Chen, M.D., CASI's Chief Medical Officer, commented, "The trial is to evaluate safety and efficacy of ENMD-2076 as a single agent in a total of 29 locally-advanced or metastatic FLC patients. It uses a Simon's optimal two-stage design to allow for an early determination of potential futility in the 16 patients of Stage 1. An additional 13 patients will be recruited in Stage 2 to allow an overall final analysis with sufficient power. Having met the Stage 1 endpoint, we will now advance the trial to Stage 2 development. Meanwhile, the Stage 1 remains ongoing with approximately one-third of the patients still under treatment. A full interim analysis will be conducted and reported after all 16 patients have reached study endpoint of responses."

Ken Ren, Ph.D., CASI's Chief Executive Officer, commented, "We are pleased to see our FLC trial advance into Stage 2, which upon completion will increase the amount of data of ENMD-2076 in FLC patients.  FLC is a life-threatening disease that typically affects young adults and is currently without any systemic treatment. Based on results to date, we are encouraged by the potential of ENMD-2076 to address the unmet medical needs of these patients.  We are thankful to our investigators for their dedication to the program and to our FLC patients who make the trial meaningful and drive our purpose."

More information about the trial can be found at www.clinicaltrials.gov.

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 MARQIBO^® (vinCRIStine sulfate LIPOSOME injection), EVOMELA^® (melphalan) for Injection and ZEVALIN^® (ibritumomab tiuxetan) for the greater China market (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 remaining closing or closings 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 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 candidates 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.

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

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SOURCE CASI Pharmaceuticals, Inc.