"The data in the ET and MF study publications in
In the Phase 2 clinical study evaluating imetelstat in ET, all patients achieved a hematologic response, with the majority achieving a hematologic complete response. Rapid and substantial molecular responses observed in the study suggested therapeutic activity of imetelstat on the growth of malignant progenitor cell clones that drive ET. The safety and efficacy results reported in the NEJM publication are consistent with the preliminary data from this study previously presented in part at the annual meetings of the
"This study was a first look at what happens when you treat ET patients with a drug that has a totally novel mechanism of action," said Dr.
"The molecular responses suggest that imetelstat may have broad activity across hematologic myeloid malignancies which warrants further clinical study in other myeloproliferative neoplasms," noted Prof. Dr. med.
In the Phase 2 pilot study evaluating imetelstat in MF patients, unprecedented complete and partial remissions, including reversal of bone marrow fibrosis and molecular responses, were observed. These results suggest the potential value of telomerase-targeting strategies for the treatment of MF, and identify imetelstat as an active drug in this disease. The safety and efficacy results reported in the NEJM publication are consistent with the preliminary data from this study previously presented in part at the annual meetings of the
"The clinical and molecular remissions seen in the study suggest selective anti-clonal activity, not previously documented in current drug treatment of MF," commented Dr.
A Phase 2 clinical study in MDS, to be conducted by Janssen, is expected to open for patient enrollment by the end of 2015.
Imetelstat (GRN163L; JNJ-63935937) is a potent and specific inhibitor of telomerase that is administered by intravenous infusion. This first-in-class compound, discovered by Geron, is a specially designed and modified short oligonucleotide, which targets and binds directly with high affinity to the active site of telomerase. Preliminary data suggest that imetelstat shows disease-modifying activity by affecting the malignant clones associated with hematologic myeloid malignancies. Most commonly reported adverse events in imetelstat clinical studies conducted to date include fatigue, gastrointestinal symptoms and cytopenias. Patients in these studies also experienced elevated liver enzymes, which resolved to normal or baseline in the majority of patients followed after imetelstat treatment was withdrawn. Imetelstat has not been approved for marketing by any regulatory authority.
Geron is a clinical stage biopharmaceutical company focused on the collaborative development of a first-in-class telomerase inhibitor, imetelstat, in hematologic myeloid malignancies. 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: (i) the initiation, timing, size and completion of planned and potential clinical trials of imetelstat to be conducted under the collaboration agreement with Janssen, including those planned for MF, MDS and AML; (ii) the potential for, and safety and efficacy of imetelstat treatment for patients with hematologic myeloid malignancies; and (iii) other statements that are not historical facts, 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, risks and uncertainties related to: (i) the uncertain, time-consuming and expensive product development and regulatory process, including whether Geron and Janssen will succeed in overcoming all of the clinical safety and efficacy, technical, scientific, manufacturing and regulatory challenges in the development and commercialization of imetelstat; (ii) Janssen's ability to enroll patients in any of the planned clinical trials; (iii) whether any efficacy or safety results may cause the benefit-risk profile of imetelstat to become unacceptable; and (iv) whether imetelstat can be applied to any or to multiple hematologic myeloid malignancies. Additional information on the above risks and uncertainties and additional risks, uncertainties 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
Anna Krassowska, Ph.D. Investor and Media Relations 650-473-7765 [email protected] [email protected]