Geron Corporation (NASDAQ:GERN) announced the dosing of the first patient in a Phase 2 clinical trial to evaluate imetelstat in patients with myelofibrosis (MF). This clinical trial, also referred to as the IMbarkTM study, is being conducted by Janssen Biotech, Inc. (Janssen), under the terms of the exclusive worldwide imetelstat license and collaboration agreement between the companies.
Phase 2 Clinical Trial Design
The purpose of the Phase 2 clinical trial is to assess the efficacy, safety and tolerability of two dose levels of single-agent imetelstat in patients with MF. The trial is designed to enroll approximately 200 patients (approximately 100 patients per dosing arm) with DIPSS intermediate-2 or high risk MF who have relapsed after or are refractory to Janus Kinase (JAK) inhibitor treatment. At the time of enrollment, patients must have measurable splenomegaly and symptoms of MF. Patients will be assigned randomly on a blinded basis on a 1:1 ratio to one of two dosing arms – 9.4 mg/kg every three weeks or 4.7 mg/kg every three weeks. Dose reductions for adverse events are allowed and will follow protocol-specified algorithms. An interim review of data from the trial is planned after approximately 20 patients per arm have been randomized and followed for at least 12 weeks, in order to assess the adequacy of one or both of the initial dosing arms. As a result of this interim review, one or both dosing arms could continue as planned, be stopped or modified, or alternative doses could be selected.
The co-primary efficacy endpoints for the trial are spleen response rate and symptom response rate. Spleen response rate is defined as the percentage of patients who achieve ≥ 35% reduction in spleen volume from baseline at the Week 24 visit, as measured by imaging scans and assessed at a central imaging facility and by an Independent Review Committee. Symptom response rate is defined as the percentage of patients who have ≥ 50% reduction in Total Symptom Scores from baseline at the Week 24 visit, based on patient-reported outcomes on a modified Myelofibrosis Symptom Assessment Form version 2.0 electronic diary. The primary efficacy analysis of the co-primary endpoints will occur after all treated patients have been followed for at least 24 weeks.
Secondary efficacy endpoints include the number of patients achieving complete remission (CR) or partial remission (PR), clinical improvement (CI), and anemia, spleen and symptom responses as assessed using the modified 2013 International Working Group-Myeloproliferative Neoplasms Research and Treatment (IWG-MRT) criteria. These secondary endpoints will be assessed at the time of the primary efficacy analysis. Exploratory endpoints include cytogenetic and molecular responses, as well as leukemia-free survival.
Safety outcomes will be monitored throughout the trial and will include enhanced data collection and reporting for adverse events of interest, including hepatobiliary-associated laboratory findings and hepatic adverse events. (Original Source)
Shares of Geron closed yesterday at $3.55. GERN has a 1-year high of $4.67 and a 1-year low of $1.76. The stock’s 50-day moving average is $3.44 and its 200-day moving average is $3.84.
On the ratings front, Geron has been the subject of a number of recent research reports. In a report issued on September 3, Oppenheimer analyst Ling Wang reiterated a Buy rating on GERN, with a price target of $5.50, which represents a potential upside of 54.9% from where the stock is currently trading. Separately, on August 10, Piper Jaffray’s Charles Duncan reiterated a Buy rating on the stock and has a price target of $5.
According to TipRanks.com, which ranks over 7,500 financial analysts and bloggers to gauge the performance of their past recommendations, Ling Wang and Charles Duncan have a total average return of -7.3% and 12.9% respectively. Wang has a success rate of 28.6% and is ranked #3512 out of 3759 analysts, while Duncan has a success rate of 56.3% and is ranked #399.
Geron Corp is a clinical stage biopharmaceutical company engaged in the discovery and development of therapeutic products for oncology such as telomerase inhibitor, imetelstat and in hematologic myeloid malignancies, among others.