Geron Announces Initiation Phase 2/3 Clinical Trial of Imetelstat in MDS and ODD designation for MDS

[Fishermangents]

Geron Announces Initiation of Janssen Phase 2/3 Clinical Trial of Imetelstat in Myelodysplastic Syndromes

MENLO PARK, Calif., Jan. 14, 2016 (GLOBE NEWSWIRE) -- Geron Corporation (Nasdaq:GERN) today announced the dosing of the first patient in a Phase 2/3 clinical trial to evaluate imetelstat in patients with myelodysplastic syndromes (MDS). This clinical trial, also referred to as the IMergeTM study, is being conducted by Janssen Research & Development, LLC, and is the second study to be initiated under the terms of the exclusive worldwide imetelstat license and collaboration agreement between Geron and Janssen Biotech, Inc. (Janssen).

Phase 2/3 Clinical Trial Design
The purpose of the Phase 2/3 clinical trial is to evaluate imetelstat in transfusion dependent patients with International Prognostic Scoring System (IPSS) Low or Intermediate-1 risk MDS who have relapsed after or are refractory to prior treatment with an erythropoiesis-stimulating agent (ESA).

As designed, the trial consists of two parts, and a total of approximately 200 patients are expected to be enrolled. Part 1 of the trial is planned as a Phase 2, open-label, single-arm design to assess the efficacy and safety of imetelstat. Up to 30 patients are expected to be enrolled in Part 1, all of whom will receive imetelstat and be followed for safety, hematologic improvement and reduction in transfusion requirement. Before proceeding to Part 2, the data from Part 1 must support a positive assessment of the benefit/risk profile of imetelstat in these patients. Part 2 of the trial is planned as a Phase 3 double-blind, randomized, placebo-controlled design to compare the efficacy of imetelstat against placebo. Approximately 170 patients are expected to be enrolled in Part 2, who will be assigned randomly, in a 2:1 ratio, to receive either imetelstat or placebo. The inclusion criteria for both parts of the trial require that at the time of enrollment each patient must be red blood cell transfusion-dependent. Imetelstat (or placebo in Part 2) will be administered as an intravenous infusion. The starting dose will be 7.5 mg/kg every 4 weeks and may be escalated according to certain protocol-specified conditions. Dose reductions for adverse events may follow protocol-specified algorithms. All patients may receive supportive care, including transfusions or myeloid growth factors, as needed per investigator discretion and according to local standard practices.

The primary efficacy endpoint is designed to be the rate of red blood cell transfusion-independence lasting at least 8 weeks, defined as the proportion of patients without any red blood cell transfusion during any consecutive 8 weeks since entry to the trial. A primary efficacy analysis is planned to occur 12 months after the last patient is enrolled.
Secondary efficacy endpoints in the trial include the proportion of patients achieving red blood cell transfusion-independence lasting at least 24 weeks, the time to and duration of red blood cell transfusion-independence, the proportion of patients achieving hematologic improvement, the proportion of patients achieving complete remission (CR) or partial remission (PR) per 2006 International Working Group (IWG) criteria for MDS, the proportion of patients requiring red blood cell transfusions and the amount, the proportion of patients requiring the use of myeloid growth factors and the dose, as well as assessments of the change in the patients’ quality of life using several different validated instruments. Patients are also planned to be followed for an assessment of overall survival and time to progression to acute myeloid leukemia (AML). These secondary endpoints are expected to be assessed at the time of the primary efficacy analysis.

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.

Multiple medical centers across North and South America, Europe and Asia are planned to participate in the trial. For more information about the IMergeTM study being conducted by Janssen, please visit https://clinicaltrials.gov/ct2/show/NCT02598661.

Orphan Drug Designation
On December 23, 2015, the United States Food and Drug Administration (FDA), granted orphan drug designation to imetelstat for the treatment of MDS. The FDA has previously granted orphan drug designation to imetelstat for the treatment of myelofibrosis (MF). Orphan drug designation is granted by the FDA’s Office of Orphan Drug Products in order to support development of medicines for underserved or rare diseases and patient populations that affect fewer than 200,000 people in the United States. Orphan drug designation qualifies the sponsor of the drug for various development incentives of the ODA, including, if regulatory approval is received, seven years of market exclusivity with certain limited exceptions, exemption from FDA application fees, and certain tax credits for qualified clinical testing. The granting of orphan drug designation does not alter the standard regulatory requirements and process for obtaining marketing approval. The safety and effectiveness of a drug must be established through adequate and well-controlled studies. Orphan drug exclusivity does not prevent the FDA from approving a different drug for the same disease or condition, or the same drug for a different disease or condition.

link to the press release: http://ir.geron.com/phoenix.zhtml?c=673 ... id=2129160

[Greg E]

The table is set. It's kind of amazing that everything is coming together at a moment when the market is selling off. I've been in Geron for years, and it's taken amazing patience to wait for the opportunity the company now has. Some investors are just coming to Geron now, and they have no idea how lucky they are to be able to buy into this opportunity at this moment at this price.

Two trials are underway,- likely there will be significant news in the next 12 months even though the primary analysis aren't scheduled to be done in the next 12 months. Just the advancement to Phase 3 in MDS will be huge when it comes. BTD is possible, as well as just unofficial news coming out of either trial.

JNJ just highlighted Imetelstat at JP Morgan on Monday. Every indication we have, and admittedly they are just anecdotal, is that things are proceeding as the Mayo Trial results suggest they would in the MF and MDS trials, though clearly enrollment numbers are still very low in the MF trial. To me, JNJ's confidence, and their deliberation in setting up the MF trial and proceeding with great care in enrollment, are excellent signs of the likelihood of success.

Good luck to all, and my best to patients. Thanks to Fishermangents for setting up this site.

Greg E

[scottmayhew]

It's kind of amazing that everything is coming together at a moment when the market is selling off.

...yeah, I was thinking about this the other day and was kind of hoping they wouldnt have anything to announce until the overall market correction was over...that a jump in pps would most likely be dampened by an overall exit from the market. that being said, thank God for positive news!

[Fishermangents]

Now into Phase 3 according to the CT site: https://clinicaltrials.gov/ct2/show/study/NCT02598661