CASI Pharmaceuticals, Inc. (NASDAQ:CASI) is a clinical-stage biopharmaceutical company focused on the acquisition ,development and commercialization of proprietary therapeutics for the treatment of cancer and other diseases for the global markets, with a focus on China. The Company employs a global drug-development strategy that leverages our expertise and resources in North America and China. By conducting trials under both North American and Chinese regulatory pathways, the The Company intends to reach clinical efficacy inflection points with drug candidates at greater cost-efficiencies. CASI’s lead compound, ENMD-2076, a selective kinase inhibitor is currently in multiple Phase II studies in triple-negative breast cancer, soft tissue sarcoma, and ovarian clear cell carcinoma in U.S. and Canada, with plans to expand trials into China. Its second compound, 2ME2 (2-methoxyestradiol), has demonstrated promising preclinical results in autoimmune disorders including rheumatoid arthritis and multiple sclerosis, and was previously under clinical investigation for various cancers. CASI intends to continue its evaluation of 2ME2 for further internal development. In addition, the Company is actively pursuing in-licensing opportunities and internal development to enrich its product pipeline. CASI is headquartered in Rockville, MD and has established a R&D center with clinical and regulatory staff in Beijing.
The Company underwent a name change to CASI in 2014 to reflect the Company’s shift to a new development model that includes a focus on the Chinese pharmaceutical market.
Lead Drug Candidate
ENMD-2076 for Oncology
CASI’s lead program is 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 I clinical trials in many different solid tumor cancers including ovarian, breast, liver, renal and sarcoma, as well as in leukemia, and multiple myeloma, and is completing a Phase II clinical trial for ovarian cancer. CASI is conducting a Phase II study of ENMD-2076 in triple-negative breast cancer, a Phase II study in advanced/metastatic soft tissue sarcoma, and a Phase II study in advanced ovarian clear cell carcinomas in North America. Pending approval by the CFDA, the Company intends to expand these trials into China. ENMD-2076 has received orphan drug designation from the U.S. Food and Drug Administration for the treatment of ovarian cancer, multiple myeloma and acute myeloid leukemia.
ENMD-2076 has strong global intellectual property protections worldwide with patents approved or pending for approvals in the US, Canada, Japan and many other countries. On May 6, 2013, the Chinese State Intellectual Property Office issued a patent for ENMD-2076 covering composition of matter and uses to treat various cancers.
Other Drug Candidates
2ME2 (2-methoxyestradiol) for Rheumatoid Arthritis and Multiple Sclerosis. 2ME2 is an orally active compound that has anti-proliferative, anti-angiogenic and anti-inflammatory properties. 2ME2 attacks tumor cells through multiple mechanisms of action including the inhibition of angiogenesis. 2ME2 inhibits production of the transcription factor hypoxia inducible factor-1 alpha (HIF-1a), which is a protein required for angiogenesis and cell survival. The inhibition of angiogenesis is an important approach to the treatment of both cancer and rheumatoid arthritis. 2ME2 has potential as both a single agent or in combination with standard cares in the treatment of rheumatoid arthritis based on its anti-angiogenic, anti-inflammatory, and anti-osteoclastic (bone resorption) properties. 2ME2 has also shown positive preclinical results in multiple sclerosis.
The U.S. Patent & Trademark Office issued on March 19, 2013 patent #8,399,440—“Disease Modifying Anti-Arthritic Activity of 2-Methoxyestradiol for composition and method claims covering the combination of 2ME2 and methotrexate for the treatment of rheumatic diseases, including rheumatoid arthritis”.