Geron Corporation Receives NCI Award to Continue Development of Telomerase Inhibitors to Treat...

Business Editors & Health/Medical Writers

MENLO PARK, Calif.--(BUSINESS WIRE)--Sept. 10, 2002

Geron Corporation (Nasdaq:GERN) today reported receipt of a National Consortium Drug Discovery Group (NCDDG) grant award of $796,189 to support the development of a potent and safe

telomerase inhibitor for the treatment of multiple types of cancer. This award constitutes the eighth consecutive year of funding (cumulatively $4.2 million) through the National Institutes of Health for the research collaboration between Geron Corporation, Memorial Sloan Kettering Cancer Center and the National Cancer Institute, which began in September 1995.

The focus of the grant, entitled "Telomerase: A Molecular Target for Cancer Therapy," is to develop compounds that inhibit telomerase and establish pre-clinical models for demonstrating safety and efficacy of these compounds. A safe and effective telomerase inhibitor has the potential to treat all types of cancer with minimal side effects. Cancer kills through uncontrolled tumor cell growth caused by genetic mutations. All types of cancer rely on telomerase to allow the uncontrolled growth to continue indefinitely, instead of stopping when the cancer cells' telomeres become critically short. Geron's lead anti-cancer compound, GRN163, is a telomerase inhibitor that has shown safety and efficacy in multiple, pre-clinical in vitro and in vivo studies.

GRN163 RESULTS TO DATE

In vitro testing: Geron and collaborators so far have tested GRN163 in vitro on 13 different types of cancers, including myeloma, lymphoma, and cancers of the breast, pancreas, kidney, cervix, brain, lung, prostate and ovary, and demonstrated inhibition of telomerase activity in every cancer type. Treatment with GRN163 resulted in senescence and apoptosis (death) of the tumor cells, with the required duration of treatment related to the initial telomere length of the cancer cells. In contrast, even higher concentrations of GRN163 showed no toxicity to a number of normal cell lines.

In vivo testing: Geron and collaborators have also tested GRN163 in animal models of human malignant glioblastoma (brain cancer), prostate cancer, lymphoma and multiple myeloma, and demonstrated anti-tumor efficacy in all models. In three separate studies, human malignant glioblastoma (brain cancer cells) were implanted under the skin in mice and the resulting tumors were treated with GRN163. In all three studies, short-term treatment (over one to three weeks) with GRN163 resulted in tumor reduction in all treated animals. On average the tumors in the treated animals were 70% - 80% smaller than untreated animals. Also, the treated animals showed no signs of toxicity. Importantly, tumor growth was slowed in all of the mice that received GRN163, and in one case, the tumor completely disappeared after this very short-term treatment. Additionally, two studies examined delivery of GRN163 into the brains of rats bearing human glioblastoma tumors, and demonstrated that GRN163 was taken up and retained by the tumor cells, indicating appropriate bioavailability (uptake into tumor cells) for the treatment of human brain cancer cells.

Memorial Sloan Kettering Cancer Center has reported data from several in vitro and in vivo studies of human lymphoma and myeloma, two forms of blood cancer. The in vitro studies confirmed that GRN163 effectively inhibited telomerase activity in the tumor cells and caused cancer cell death. The in vivo studies, in which human tumor cells were implanted in immunodeficient mice and then injected with GRN163 or a control, demonstrated that short-term treatment with GRN163 inhibited the growth of both tumor types. Dr. Malcolm A. S. Moore, Department Head of Developmental Hematopoiesis at Memorial Sloan Kettering Cancer Center, New York, presented these data at the European Haematology Association 7th Annual Meeting in June 2002.

"We are very pleased that our progress in the development of telomerase inhibitors as novel anti-cancer agents has been judged by the National Cancer Institute to warrant continued funding," said Thomas B. Okarma, Ph.D., M.D., Geron's president and chief executive officer. "Because telomerase activity is key to the malignancy and spread of all types of cancer cells, we believe telomerase inhibition will be effective against a very wide range of cancer types, while avoiding toxicity to normal cells."

INTELLECTUAL PROPERTY

Geron has an extensive patent portfolio relating to telomerase, including issued patents covering the sequence of the GRN163 compound. Earlier this year, Geron acquired various patents and patent applications owned by Lynx Therapeutics that cover oligonucleotides with phosphoramidate backbone linkages and methods of synthesizing them; these patents provide Geron with rights needed to manufacture GRN163.

CLINICAL DEVELOPMENT

Geron expects to complete pre-clinical toxicology studies by the first quarter of 2003, and then file an IND in support of a Phase 1 clinical trial. After FDA approval, the Phase 1 trial will be conducted at two or more cancer centers, and will focus on glioblastoma (brain cancer). Geron expects to begin patient enrollment for the trial in the first half of 2003.

Geron is a biopharmaceutical company focused on developing and commercializing therapeutic and diagnostic products for applications in oncology and regenerative medicine, and research tools for drug discovery. Geron's product development programs are based upon three patented core technologies: telomerase, human embryonic stem cells, and nuclear transfer. The company's oncology development program directed against telomerase rests on three synergistic approaches: direct telomerase inhibition (GRN163); telomerase immunotherapy; and anti-tumor gene therapy/oncolytic therapy utilizing the telomerase promoter.

This news release may contain forward-looking statements made pursuant to the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Investors are cautioned that such forward-looking statements in this press release regarding future applications of Geron Corporation's technology constitute statements involving risks and uncertainties, including, without limitation, risks inherent in the development and commercialization of potential products, dependence on collaborative partners, and the maintenance of our intellectual property rights. Actual results may differ materially from the results anticipated in these forward-looking statements. Additional information on potential factors that could affect our results and other risks and uncertainties are detailed from time to time in Geron's periodic reports, including the quarterly report on Form 10-Q for the quarter ended June 30, 2002.

Additional information about the company can be obtained at http://www.geron.com.