CASI Pharmaceuticals Granted Type C Meeting With U.S. FDA For ENMD-2076 In Fibrolamellar Carcinoma

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Dec 17, 2014, 07:00 ET

ROCKVILLE, Md., Dec. 17, 2014 /PRNewswire/ -- CASI Pharmaceuticals, Inc. (Nasdaq: CASI), 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 primary commercial focus on China, today reported that it has been granted a Type C meeting with the U.S. Food and Drug Administration (FDA) and that the meeting will be held in February 2015. During this meeting the Company will seek guidance on the clinical trial protocol design and the regulatory path forward to obtain marketing approval for ENMD-2076 for the treatment of fibrolamellar carcinoma.

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Ken K. Ren, Ph.D., CASI's Chief Executive Officer, stated, "We look forward to advancing our investigational drug ENMD-2076 for fibrolamellar carcinoma (FLC), a rare malignant neoplasm of the liver that represents a subset of hepatocellular carcinoma, and often times affects adolescents and young adults with no history of primary liver disease. There is currently no standard first-line systemic treatment option available for FLC, which has a distinct clinical and histological prognosis. We believe that our proprietary drug candidate ENMD-2076, which targets Aurora A, VEGF and FGFR, has potential to treat FLC."

Dr. Ren continued, "In a previous ENMD-2076 Phase I solid tumor trial, we observed an FLC patient achieve stable disease at cycle 2, partial response at cycle 12 and stable disease for 17 months after treatment. Interestingly, research published this year by a third party in Science¹ reported the detection of a chimeric transcript protein that is expressed and detected in 100% of patient FLC tumor tissue examined but not in adjacent normal liver, suggesting strongly that this genetic alteration contributes to tumor pathogenesis of FLC. The chimeric transcript is the catalytic domain of protein kinase A, which results in Aurora A kinase being overly expressed in FLC patients. Aurora A kinase is therefore believed to be a promising therapeutic target for FLC. "

"Our initial clinical trial plan was to conduct a Phase II trial for FLC, for which we have obtained IND clearance from the FDA. However, based on our recent investigator's meeting that included key opinion leaders in the field of fibrolamellar carcinoma, we will seek to conduct a direct NDA registration trial based on our belief that the drug candidate may address an important unmet medical need in this orphan indication. We are currently preparing our briefing package and look forward to receiving guidance from the FDA and finalizing our study design to move forward on the regulatory path towards market approval. We are also pleased that the Company has received Orphan Drug designation earlier this year from the FDA for the treatment of hepatocellular carcinoma including FLC," he added.

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 primary commercial focus on China. CASI's product pipeline includes exclusive rights to ZEVALIN^® (ibritumomab tiuxetan), MARQIBO^® (vinCRIStine sulfate LIPOSOME injection) and Captisol-Enabled^TM (propylene glycol-free) melphalan (CE melphalan) 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. In addition, CASI will be updating its Corporate Presentation to be available on its website at www.casipharmaceuticals.com/investor-relations.

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. 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; 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 www.sec.gov.

ZEVALIN^® and MARQIBO^® are registered trademarks of Spectrum Pharmaceuticals, Inc. and its affiliates. Captisol-Enabled™ is the trademark property of its owner.

COMPANY CONTACT:
CASI Pharmaceuticals, Inc.
240.864.2643
[email protected]

INVESTOR CONTACT:
LHA
Kim Sutton Golodetz
212.838.3777
[email protected]

¹ "Detection of a Recurrent DNAJB1-PRKACA Chimeric Transcript in Fibrolamellar Hepatocellular arcinoma," Joshua N. Honeymoon et al., Science 343, 1010 (2014)

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