ROCKVILLE, Md., Feb. 9, 2015 /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 commercial focus on China, is pleased to announce that it has received formal meeting minutes from the U.S. Food and Drug Administration (FDA) regarding the clinical and regulatory path leading to market approval of ENMD-2076 for the treatment of Fibrolamellar Carcinoma (FLC). The meeting minutes stem from a recent informative meeting between CASI and the FDA.
"We are strongly encouraged by the FDA's feedback and appreciate their guidance and support of our development program," commented Ken K. Ren, Ph.D., CASI's Chief Executive Officer. "Based on our meeting, we believe the FDA has provided us with a clear direction for the clinical and regulatory path forward, particularly regarding the design of our clinical trial, which may support a New Drug Application (NDA) of ENMD-2076 as the first line therapy for FLC."
Dr. Ren commented further, "As confirmed by the FDA, below are the key elements of our clinical and regulatory path:
- We plan to conduct a single arm trial to evaluate the overall response rate (ORR) of ENMD-2076 in FLC patients as the primary endpoint.
- The trial will be conducted as a Simon 2-stage study with interim futility analysis released after 16 patients reach a clinical endpoint. Futility would be declared if none of the patients experience a response and would result in the termination of the study.
- If, however, one or more patients experience a response according to the RECIST 1.1 criteria, the study would continue with additional patients. At that time, we would expect to meet with the FDA to discuss what tumor response criteria would be considered reasonably likely to provide clinical benefit and support an accelerated approval based on the surrogate endpoint of tumor response.
- Assuming that the results of the proposed study show that ENMD-2076 improves overall tumor response, CASI would expect to propose a confirmatory clinical trial and, in parallel, submit an NDA under subpart H for accelerated approval of new drugs for serious or life-threatening illnesses based on overall tumor response rates.
- CASI will submit to the FDA a request for Breakthrough Therapy designation whenever clinical data meets the appropriate criteria."
"While fulfilling the FDA's other regulatory requirements, including a food effect study of ENMD-2076 in healthy human subjects, we intend to move forward and are working with our dedicated investigators and the FLC patient community to initiate the trial as soon as possible and will update our study protocol at www.clinicaltrials.gov once a final version of the protocol is available. We have received orphan drug designation for hepatocellular carcinoma, from the FDA in the U.S. and intend to file for orphan drug designation for FLC in the EU."
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 Captisol-EnabledTM (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.
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.
SOURCE CASI Pharmaceuticals, Inc.