|Geron Presents Positive Results from Phase 2 Study of Imetelstat in Essential Thrombocythemia at the American Society of Hematology Annual Meeting|
MENLO PARK, Calif., December 10, 2012 - Geron Corporation (Nasdaq: GERN) today announced positive clinical results from the Phase 2 trial of imetelstat, the company's first-in-class telomerase inhibitor, in patients with essential thrombocythemia (ET). ET is a chronic blood disorder that is representative of a group of diseases known as myeloproliferative neoplasms (MPNs). The data, which showed rapid and durable hematologic and molecular responses in patients treated with imetelstat, were presented Sunday evening in an oral session at the 54th Annual Meeting of the American Society of Hematology (ASH) in Atlanta, GA, by Prof. Dr. med. Gabriela M. Baerlocher of the University Hospital and University of Bern, Switzerland, and a principal investigator of the trial. To view the presentation slides, please visit www.geron.com/PDFs/Geron-Imetelstat-ETPh2-ASH-2012.pdf.
"The observed hematologic response rate of 100%, accompanied by a molecular response rate of 86% among the patients who had a JAK2 V617F mutation, is very impressive considering that these were patients who did not respond to or tolerate other therapies," said Prof. Baerlocher. "The 60 to 90% reduction in JAK2 V617F allelic burden in patients who had the molecular responses, and the rapidity with which these responses were observed, exceeded our expectations. It is also encouraging that all of the patients who were eligible to remain on imetelstat beyond a year have elected to do so."
Study Rationale and Design
Hematologic responses were measured by reductions in platelet counts. Molecular responses were measured by reductions in mutant JAK2 allelic burden in circulating granulocytes. A decrease in the relative proportion of mutant JAK2 to wild type JAK2 is consistent with selective inhibition of the neoplastic progenitor cells responsible for the disease. The European LeukemiaNet criteria were used to grade both hematological and molecular responses.
Imetelstat was initially administered weekly by intravenous infusion during an induction phase. After achieving a complete hematologic response, which occurred in a median time of approximately six weeks, a maintenance phase was begun in which dosing frequency was modified based on a patient's individual response profile. In 11 out of 13 (84.6%) patients who attained a complete hematologic response, the frequency with which imetelstat was administered to maintain the response was reduced to every two weeks or less, generally decreasing over time. Six out of seven (85.7%) eligible patients have chosen to remain on treatment beyond one year.
Imetelstat Development in Hematologic Malignancies
Based on the results from the ET study, Dr. Ayalew Tefferi, M.D., at the Mayo Clinic has begun an investigator-sponsored pilot study to evaluate safety and efficacy of imetelstat in patients with myelofibrosis, a myeloproliferative neoplasm in the same spectrum of diseases as ET. For more information about this study, please refer to http://clinicaltrials.gov/ct2/show/NCT01731951. Geron is in the initial planning stages of a company-sponsored Phase 2 study in myelofibrosis, which will be informed, in part, by data from the Mayo Clinic study. In addition, Geron intends to expand its directed program of investigator-sponsored trials in 2013 to other hematologic myeloid indications, including acute myelogenous leukemias.
About Essential Thrombocythemia (ET)
ET is a chronic blood disorder characterized by increased numbers of platelets in the blood. These platelets may have abnormal function, which can lead to an increased risk of thrombotic or hemorrhagic complications. Currently used treatments, such as hydroxyurea and anagrelide, can be effective in reducing platelet counts in patients with ET, but do not alter the underlying biology of the disease, and clinical resistance or intolerance to these agents occurs in a proportion of patients. The utility of interferon-alpha, which can induce molecular responses in some patients, is limited by tolerability.
Imetelstat (GRN163L) is a potent and specific inhibitor of telomerase. This first-in-class compound is a specially designed and modified short oligonucleotide, which targets and binds directly and with high affinity to the active site of telomerase. Unique and proprietary oligonucleotide chemistry improves binding affinity and stability in plasma and tissues. A lipid modification enables cellular and tissue penetration and biodistribution. To date, clinical data from Phase 2 studies indicate that the compound has activity against hematologic malignancies and in solid tumors with short telomeres.
Update on Phase 2 Trial in Multiple Myeloma
Geron is a biopharmaceutical company developing first-in-class therapies for cancer, including its telomerase inhibitor, imetelstat. For more information about Geron, visit www.geron.com.
Use of Forward-Looking Statements
Except for the historical information contained herein, this press release contains forward-looking statements made pursuant to the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Investors are cautioned that statements in this press release regarding Geron's plans or expectations for or of: dates to obtain or present data or other results from any clinical trials; and clinical development plans or success of imetelstat, including imetelstat possibly having applicability for the treatment of other progenitor cell-driven hematologic malignancies, including myelofibrosis, constitute forward-looking statements. These statements involve risks and uncertainties that can cause actual results to differ materially from those in such forward-looking statements. These risks and uncertainties, include, without limitation: (a) regarding dates for the availability of data or other results - delays in enrollment, delays caused by institutional review boards or regulatory agencies, shortage of supply, dependence on clinical trial collaborators and safety issues; and (b) regarding the development of imetelstat - those risks and uncertainties inherent in the development of potential therapeutic products, including without limitation, results from the ET trial may not mean that imetelstat has applicability for the treatment of other progenitor cell-driven hematologic malignancies, including myelofibrosis; successful clinical trial results and the protection of Geron's intellectual property rights. Additional information and factors that could cause actual results to differ materially from those in the forward-looking statements are contained in Geron's periodic reports filed with the Securities and Exchange Commission under the heading "Risk Factors," including Geron's quarterly report on Form 10-Q for the quarter ended September 30, 2012. Undue reliance should not be placed on forward-looking statements, which speak only as of the date they are made, and the facts and assumptions underlying the forward-looking statements may change. Except as required by law, Geron disclaims any obligation to update these forward-looking statements to reflect future information, events or circumstances.
Anna Krassowska, Ph.D.
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