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Geron Announces Initiation of Clinical Trial for Its Telomerase Inhibitor Drug GRN163L in Patients with Chronic Lymphocytic Leukemia

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MENLO PARK, Calif.--(BUSINESS WIRE)--July 27, 2005--Geron Corporation (Nasdaq: GERN ) announced today that it has initiated clinical testing of its lead anti-cancer compound, GRN163L, in patients with chronic lymphocytic leukemia (CLL) at two clinical sites in the New York metropolitan area.

This initial Phase I/II, dose-escalation trial of GRN163L will be conducted in patients with advanced CLL. The study is primarily designed to demonstrate the safety and tolerability of GRN163L administered intravenously on a weekly basis. Importantly, CLL also provides a unique opportunity to measure both the magnitude and time course of telomerase inhibition in tumor cells achieved at various doses of GRN163L. By serially assessing the effects of the drug on the target enzyme in CLL cells, insights will be gained regarding the dose and dosing interval that optimally inhibits telomerase activity in the tumor. In this way, pharmacokinetic and pharmacodynamic parameters can be correlated with any observed reduction in patients' tumor burden.

"This first trial is open for patient enrollment at Long Island Jewish Medical Center and North Shore University Hospital, under the respective leadership of principal investigators Kanti Rai, M.D. and Steven Allen, M.D.," said Melissa Kelly, Geron's vice president of oncology. "These sites and investigators were chosen because of their extensive clinical research experience in CLL as well as other hematologic malignancies. A third investigational site will be coming online in the near future."

"We are excited to be participating in the clinical evaluation of this first-in-class drug, GRN163L, directed against the novel and specific cancer target telomerase in patients with CLL," said Kanti Rai, M.D., Chief of the Division of Hematology and Oncology at Long Island Jewish Medical Center. "We know from collaborative work recently reported by our institution and North Shore University Hospital that telomerase is highly expressed and that telomeres are relatively short in many patients with advanced CLL. Studies have shown that these telomere-related markers correlate with a poor prognosis. We are, therefore, very hopeful that our patients may benefit from this new drug."

Dr. Rai is a recognized leader in CLL research and is also currently serving as the President-Elect of The American Society of Hematology (ASH). For more information about this initial Phase I/II trial, visit the U.S. National Library of Medicine website at http://www.ClinicalTrials.gov to access a brief study synopsis. A link to this site is also provided on Geron's website at http://www.geron.com.

About GRN163L

GRN163L is a potent inhibitor of telomerase. Inhibition of telomerase activity by GRN163L in cancer cells results in telomere shortening, and leads to cell cycle arrest or apoptosis. GRN163L is a 13-mer oligonucleotide N3'-- P5' thio-phosphoramidate (NPS oligonucleotide) that is covalently attached to a C16 (palmitoyl) lipid moiety. GRN163L binds directly with high affinity to the template region of the RNA component of human telomerase (hTR), which lies in the active or catalytic site of hTERT, the telomerase reverse transcriptase. GRN163L binding to hTR results in direct, competitive inhibition of telomerase enzymatic activity. The mechanism of action of the drug is not antisense mediated.

GRN163L has been characterized preclinically and shown to inhibit telomerase in human tumor cells of many cancer types, in both cell culture systems and animal models. Studies of this agent alone, and in combination with chemotherapeutics and other targeted therapies, indicate the importance of telomerase as a target for the treatment of cancer, and the potential utility of GRN163L in the treatment of patients with hematologic and solid tumor malignancies.

About Chronic Lymphocytic Leukemia (CLL)

CLL is the most common form of leukemia in adults in the Western world. According to the American Cancer Society, an estimated 9,700 patients in the United States will be diagnosed with CLL in 2005. CLL results from an acquired mutation in a single cell in the bone marrow. This change in the cell's DNA causes it to become malignant, and results in the uncontrolled growth of abnormal lymphocytes in the bone marrow leading to an increase in the number of abnormal lymphocytes in the blood. Lymphocytes are a type of white blood cell that normally produce antibodies and play an important role in fighting infections. Patients with CLL typically develop symptoms that may progress over a number of years and include a decrease in immunity, marked increase in the size of lymph nodes, spleen, and liver, and impaired production of other normal blood cells. Eventually, these changes may cause life-threatening complications, such as profound infections and fatal bleeding. For more information about CLL, visit http://www.leukemia-lymphoma.org.

Geron is a biopharmaceutical company developing and commercializing three groups of products: i) therapeutic products for oncology that target telomerase; ii) pharmaceuticals that activate telomerase in tissues impacted by senescence, injury or degenerative disease; and iii) cell-based therapies derived from its human embryonic stem cell platform for applications in multiple chronic diseases.
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