Celek Pharmaceuticals Receives National Cancer Institute Award to Develop CEL-031 for the Treatment of Bladder Cancer

Pilot study demonstrates CEL-031 efficacy in animal bladder cancer model

ROCKVILLE, Md.--(BUSINESS WIRE)-- Celek Pharmaceuticals today announced that the National Cancer Institute (NCI) has awarded the Company a Phase I Small Business Innovation Research (SBIR) Contract to support development of CEL-031, Celek’s targeted, first-in-class oncology drug candidate for the treatment of bladder cancer. Celek was selected for this $176,266 award based on a competitive review following NCI’s request for proposals on the development of novel anticancer agents.

CEL-031 is a small molecule compound that selectively induces apoptosis, or programmed cell death, in cancer cells by targeting phosphodiesterase enzymes, which are overexpressed in human tumor cells. The drug candidate showed evidence of efficacy and a favorable safety profile in earlier clinical studies as an oral treatment for several advanced cancers. In this setting, however, CEL-031’s efficacy was limited by the compound’s oral bioavailability. Celek plans to maximize CEL-031’s commercial potential by developing it for non-muscle invasive bladder cancer (NMIBC), where the compound will be instilled directly into the bladder by catheter, the standard route for delivering drugs to treat this disease. There is a significant need for safer and more effective NMIBC treatments that can prevent recurrence and progression of this cancer.

Celek and its collaborators, Drs. Seth Lerner and Jonathan Levitt in the Scott Department of Urology at Baylor College of Medicine, recently completed a pilot study of CEL-031 in an animal model of NMIBC. In this study, mice with bladder cancer tumors were treated twice over a three-week period with CEL-031 at two dose levels, while control animals were treated with the formulation vehicle alone. At the end of the treatment period, the average tumor size in both groups of CEL-031-treated animals was reduced by approximately 50% compared to the control mice. In addition, twice as many animals survived through 3 weeks in the CEL-031-treated groups compared to the control group. These results from a small pilot study support CEL-031’s potential as an effective, targeted therapy for NMIBC. During the Phase I SBIR project period, Celek will build on these results by further optimizing the CEL-031 formulation and testing the drug’s efficacy in a larger study using the NMIBC animal model.

Dr. Lerner, who holds the Beth and Dave Swalm Chair in Urologic Oncology at Baylor College of Medicine, said: “There is a pressing need for more effective treatments for non-muscle invasive bladder cancer. The results of the pilot study are very encouraging and we look forward to testing this compound further under the NCI SBIR contract, with the goal of moving it towards a proof-of-concept clinical trial in bladder cancer patients.”

About non-muscle invasive bladder cancer

Bladder cancer is the 5th most common cancer in the U.S., with over 70,000 new diagnoses and 14,000 deaths in 2010. The incidence of this disease continues to rise worldwide. The majority (70%) of bladder cancer cases are diagnosed at the non-muscle invasive stage, where tumors are confined to the bladder lining or have started to grow into the connective tissue, but have not yet penetrated into the muscle layer. Current treatments for non-muscle invasive disease involve transurethral surgical removal of the tumors via cystoscopy, usually followed by intravesical BCG immunotherapy or chemotherapy with mitomycin C or other non-specific cytotoxic agents. However, BCG and mitomycin C often cause significant side-effects, which limit their frequency of dosing. Furthermore, these therapies often fail, with tumors recurring in approximately 70% of patients. Patients with higher grade non-muscle invasive disease on initial diagnosis have a particularly poor prognosis, with a 30-40% rate of progression to more serious disease. Recurrence in these patients is usually treated by surgically removing the bladder. The need for regular monitoring and treatment of NMIBC makes bladder cancer the most expensive cancer to treat on a per-patient basis. There is a strong demand for new targeted drugs for NMIBC that reduce rates of tumor recurrence and progression.

About Celek Pharmaceuticals

Celek Pharmaceuticals, LLC is a privately-held biotechnology company based in Rockville, MD, developing targeted medicines for specific cancers and inflammatory disorders that urgently need new therapies. The Company is initially focusing primarily on diseases of the bladder. Celek has exclusive rights to CEL-031 from OSI Pharmaceuticals, a member of the Astellas group of companies. The new NCI SBIR contract will support 100% of the Phase I SBIR project studies described above.


Celek Pharmaceuticals
Graham Allaway, 301-461-7934
[email protected]

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