Geron PR confimrs: Both IMerge and IMbark Continue in Lower Risk MDS and Relapsed or Refractory MF

[Fishermangents]

Geron Announces Completion of Second Internal Data Reviews for Imetelstat Trials Being Conducted by Janssen
Conference Call Scheduled for 8:00 a.m. EDT Today, April 10

Both IMerge and IMbark Continue in Lower Risk Myelodysplastic Syndromes and Relapsed or Refractory Myelofibrosis

MENLO PARK, Calif., April 10, 2017 (GLOBE NEWSWIRE) -- Geron Corporation (Nasdaq:GERN) today announced that Janssen Research & Development, LLC has completed the second internal data reviews of IMerge and IMbark, the clinical trials of the telomerase inhibitor imetelstat in lower risk myelodysplastic syndromes (MDS) and relapsed or refractory myelofibrosis (MF), respectively. For IMerge, the benefit/risk profile of imetelstat in the treated patients supports continued development in lower risk myelodysplastic syndromes. A data package and proposed trial design refinements are planned to be provided to the FDA. For IMbark, the current results suggest clinical benefit and a potential overall survival benefit associated with imetelstat treatment in relapsed or refractory myelofibrosis; the trial will continue unchanged to evaluate maturing efficacy and safety data, including an assessment of overall survival.

IMerge
IMerge (NCT02598661) is a Phase 2/3 clinical trial evaluating imetelstat in transfusion dependent patients with Low or Intermediate-1 risk MDS who have relapsed after or are refractory to prior treatment with an erythropoiesis stimulating agent (ESA). The clinical trial is in two parts: Part 1 is a Phase 2, open-label, single-arm design in approximately 30 patients and Part 2 is designed to be a Phase 3, randomized, controlled trial in approximately 170 patients. The primary efficacy endpoint is the rate of red blood cell transfusion independence lasting at least 8 weeks. Key secondary endpoints include the rates of red blood cell transfusion independence lasting at least 24 weeks and hematologic improvement. Part 1 of the trial is fully enrolled.

The second internal review of IMerge included data from the approximately 30 patients enrolled in Part 1. Based on this second internal review, the Collaboration’s Joint Steering Committee has determined the following:

- The safety profile was consistent with prior clinical trials of imetelstat in hematologic malignancies, and no new safety signals were identified.
- The benefit/risk profile of imetelstat, including assessments of 8-week and 24-week transfusion independence and hematologic improvement by erythroid (HI-E) response, across multiple MDS sub-types, supports continued development in lower risk MDS.
- Part 1 of the trial will continue unmodified, and patients remaining in the treatment phase may continue to receive imetelstat.
- A data package, as well as proposed refinements to the trial design for Part 2 of IMerge, is planned to be provided to the FDA.
- Data from Part 1 are expected to be submitted for consideration for presentation at a medical conference in the future.

Geron expects that FDA feedback and the totality of imetelstat program information, including an assessment of the evolving treatment landscape in MDS and the potential application of imetelstat in multiple hematologic malignancies, will inform Janssen’s decision to initiate Part 2 of IMerge. If Part 2 of IMerge is initiated, Geron expects this Phase 3 stage of IMerge to be opened for patient enrollment in the fourth quarter of 2017.

IMbark
IMbark (NCT02426086) was originally designed as a Phase 2 clinical trial to evaluate two dose levels of imetelstat (either 4.7 mg/kg or 9.4 mg/kg administered every three weeks) in approximately 200 patients with Intermediate-2 or High risk MF who have relapsed after or are refractory to prior treatment with a JAK inhibitor. The co-primary efficacy endpoints for the trial are spleen response rate (≥35% reduction of spleen volume assessed by imaging) and symptom response rate (³50% reduction in Total Symptom Score) at 24 weeks.

- The second internal review of IMbark included data from the approximately 100 patients who were enrolled in the trial, with each dosing arm analyzed separately. Based on this second internal review, the Collaboration’s Joint Steering Committee has determined the following:
- The safety profile was consistent with prior clinical trials of imetelstat in hematologic malignancies, and no new safety signals were identified.
- The data support 9.4 mg/kg as an appropriate starting dose for the relapsed or refractory MF patient population.
- In these relapsed or refractory MF patients treated in the 9.4 mg/kg dosing arm, the spleen volume response rate observed to date was less than that reported in front-line MF patients treated in trials with other drugs. However, activity within multiple outcome measures was observed with imetelstat treatment, which suggests clinical benefit in this relapsed or refractory MF patient population. These outcome measures included a range of spleen volume reductions, decreases in Total Symptoms Score, and improvements in hematologic parameters, such as anemia and peripheral blood counts. In addition, the data suggest a potential overall survival benefit associated with imetelstat treatment in these patients.
- The trial will continue without any modifications, including conduct of all safety and efficacy assessments as planned in the protocol, including overall survival. Patients remaining in the treatment phase may continue to receive imetelstat.
- Enrollment of new patients to the trial will remain suspended because the total number of patients enrolled to date is adequate to assess longer-term outcome measures when the data are fully matured.

During the next year, Geron expects Janssen to evaluate maturing efficacy and safety data from the trial, including an assessment of overall survival. Geron expects the longer-term data from the trial, potential health authority feedback, and the totality of imetelstat program information, including an assessment of the evolving treatment landscape in MF and the potential application of imetelstat in multiple hematologic malignancies, including MDS, will inform Janssen’s decision whether to continue development of imetelstat in relapsed or refractory MF.

Conference Call
At 8:00 a.m. EDT on April 10, 2017, Geron’s management will host a conference call to review outcomes from the second internal data reviews of IMbark and IMerge. Participants can access the conference call live via telephone by dialing 877-303-9139 (U.S.); 760-536-5195 (international). The conference ID number is 6116409. A live audio-only webcast is also available through the company’s website at www.geron.com in the Investors section under Events and at http://edge.media-server.com/m/p/w5mtfw9k. The audio webcast of the conference call will be available for replay approximately one hour following the live broadcast through May 11, 2017.

Link: http://ir.geron.com/phoenix.zhtml?c=673 ... ID=2260919

[irishtrader52]

Today is our anniversary and we received a wonderful gift- the imetelstat good news made official. John has lived a very good, completely normal life for 4 years thanks to Imetelstat scientists, researchers, investors, and believers. He was one of the very sick patients (high or intermediate-high-risk imetstat R/R to either jakafi approximately 50% or hydroxurea approximately 50%) in the Mayo pilot. Now same in IMbark and IMerge it seems.

John's prognosis in April 2013 was -when he started imetelstat - 1 year of miserable, painful deterioration, given his complex/negative karotype, DPSS score, and rapidly progressing marrow fibrosis. Yet, imetelstat stopped the cancer in it's tracks with very minimal side effects. Imetelstat killed the progenitor cancer cells with subsequent recovery of peripheral blood counts - why I often called it a chemical stem cell/marrow transplant.

Now after 4 years of imetelstat treatment in Rochester MN every 3 weeks, he needs the optimal dose of 9.4 but sadly cannot get it until imet is approved. So we leave the "pilot" imetelstat trial after 4 years with this IMbark/IMerge good news fueling our hope the good imet effect will last long enough for John to be one of the 1st MF patients to infuse imetelstat at the optimal dose as an approved treatment right here at home.

That is our plan. For 4 years, I have researched every high risk MF drug in development and I concluded nothing else comes close to imetelstat (there are 2 drugs in very early stages with some promise - but they are not imetelstat). And I found the Mayo pilot study myself. So we are really "all in".

Thank you for traveling the long, frustrating, and miraculous Imetelstat road with us. On this long, long 'watch and wait', many investors feel the same fear, uncertainty, and frustration of the physicians and cancer patient.

John and I think that you can rest easier now my friends as the science unfolds slowly but surely (but as always due your own due diligence). In the meantime, enjoy life. It is always too short. (Insert Shamrock Here).

PS John and I defintely plan to join all of you on Turtle Island for the celebration party!! Then you will know we were always 'real'. More importantly, we are both alive!

[biopearl]

Irish, sincerest congratulations to you and John and thank you for continuing to share the details of your journey. You have been a voice of great reassurance for those of us "believers" who have gathered bread crumbs and traveled a long road. I hope you have not felt alone as we have gathered information and made important decisions together in many ways. I look forward to the day you can travel 20 minutes for John's infusion at the optimal dose. If the FDA had half a brain that day would be today. bp

[irishtrader52]

Thank you, bio. It is a great day for the "believers" and cancer patients. Our burden was much lightened by all the excellent information shared by all and kind words offered. Celebrate!!!

[biopearl]

Irish, is John actually leaving the pilot study? I know it was scheduled to close in May. I certainly hope some provision has been made for these patients and they will be offered continued therapy. Can you please clarify what happens? Scarlett said no new patients will be enrolled in the R/R study which initially I took to be a very good sign since the statistical power of a smaller study speaks for itself but what of patients like John or newly identified patients in the R/R group? They will fall outside the ongoing R/R study. Have provisions been made for them? I certainly hope so. What are your thoughts?? My own guess is that after further observation the R/R group will provide enough data (three more months?) to conclusive prove survival benefit. That should be the gold standard for FDA approval. I am concerned that patients will fall into a "gap" situation which ethically should not be allowed to happen. This makes me think that there must be some path to early approval. Do you agree? Regards, bp

[irishtrader52]

Yes, he is leaving pilot study. Multiple factors none related to effect of imetelstat. Include but not limited to JJ not approving 9.4 at this time for some pilot patients (since he was not on that dose when he left study and so had to resume at same lower dose). Not wanting to travel to MN for 5th year for less than 9.4.

I cannot help but also suppose that at this juncture JJ also wants the longest "pilot" study in medical history to end so as not to have any loose threads hanging at this important time in development - hence May date you mention. However, you remember that we left the pilot study for 15 months mid-study and are fully prepared to ride it out one more time.

I will make sure John is 1st in line when Imetelstat is available to all MF patients. He was an original MF trial patient who helped get imetelstat studies this far and I have contact for JJ external patient ombudsman I found if needed.

However, we expect smooth sailing as we plan to meet you all on Turtle Island someday.

[biopearl]

Irish, best wishes for continued good health to you both. Please stay in touch. bp

[Fishermangents]

Best wishes, Irish. Thanks for keeping us updated here.

[huntingonthebluffs]

Irishtrader52, yes best wishes and continued good health. I can’t remember any posts that are more poignant, hopeful, germane and of interest to all who follow what this is all about, than when you provide your updates and thoughts. While on one hand, I am still sad knowing what might have been for my deceased brother-in-law if he could have had access to Imetelstat, but also very hopeful for John, those in current CT’s and those who will eventually have the opportunity to receive treatment with Imetelstat. Your posts are certainly helping others know there is a drug that could allow them to live life, knowing now more than ever, just how special each day should be.

[irishtrader52]

Thank you all. I will stay in touch as we move closer and closer to the light at end of our long tunnel.