Geron Corporation (GERN) Q3 2015 Earnings Report, Transcript and Summary

Good day ladies and gentlemen, and welcome to the Geron Third Quarter 2015 Earnings Conference Call. At this time all participants are in a listen-only mode. Later we will conduct a question-and-answer session and instructions will follow at that time. [Operator Instructions] I'd now like to turn the conference over to Anna Krassowska, Head of Investor Relations. You may begin.

Thank you, Sania. Good afternoon, everyone, and thank you for joining us for the Geron third quarter 2015 earnings call. With me this afternoon are Dr. John Scarlett, our President and Chief Executive Officer; and Olivia Bloom, our Executive Vice President, Finance and Chief Financial Officer. Today we issued a press release that reported results for the third quarter ended September 30, 2015. This release can be found on our website at www.geron.com. Today's call is also being webcast live on our website and will be available for replay through December 05. Before we begin, please note that except for statements of historical facts, the statements during this conference call are forward-looking statements under the Safe Harbor provisions of the Private Securities Litigation Reform Act of 1995. These include, without limitations, statements regarding the potential payments under the Janssen collaboration agreement, the timelines, prospects and plans for imetelstat, including clinical study initiation and enrollment, the therapeutic potential and safety of imetelstat, Geron desire to diversify and financial or operational projections or requirements. These statements include 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 that imetelstat is safe and efficacious in multiple indications, regulatory agencies commence the clinical trials to begin or continue to proceed, clinical trials to proceed without delays and Geron will receive continuation, milestone and royalty payments from Janssen. 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 reports on Form 10-Q for the quarter ending June 30, 2015. 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 and circumstances. We'll begin today's call with a summary of the third quarter operating results from Olivia, and then Chip will review recent events and highlights from upcoming activities through the end of the year. Olivia?

Thanks Anna, good afternoon. For the third quarter of 2015, we reported net income of $27.2, or $0.17 per share, compared to a net loss of $9.5 million, or $0.06 per share, for the comparable 2014 period. For the first nine months of 2015 we reported net income of $8.5 million, or $0.05 per share, compared to a net loss of $26.7 million, or $0.18 per share, for the comparable 2014 period. Revenues for the third quarter of 2015 were $35.4 million compared to $160,000 for the comparable 2014 period. Revenues for the first nine months of 2015 were $36.2 million compared to $975,000 for the comparable 2014 period. The increase in revenues for the 2015 period compared to the same period in 2014 was primarily due to recognition of revenue for the $35 million upfront payment from Janssen. We received the upfront cash payment from Janssen in December 2014 upon the effectiveness of the collaboration and license agreement with them, which we recorded as deferred revenue at that time. Under accounting rules to recognize the upfront payment of revenues, we had to deliver imetelstat license rights to Janssen and complete the technology transfer related activities as outlined under the collaboration agreement. In the third quarter of 2015 we completed our performance of the technology transfer related activities to Janssen. Combining this performance, with the delivery of the imetelstat license rights, we were eligible to fully recognize the upfront payment and collaboration revenue in the third quarter of 2015 in accordance with generally accepted accounting principles. Total operating expenses for the third quarter of 2015 were $8.3 million compared to $10.1 million for the comparable 2014 period. Research and development expenses for the third quarter of 2015 were $4.1 million compared to $6 million for the comparable 2014 period. General and administrative expenses for the third quarter of 2015 were $4.3 million compared to $4.1 million for the comparable 2014 period. Total operating expenses for the first nine months of 2015 were $28.1 million compared to $28.3 million for the comparable 2014 period. Research and development expenses for the first nine months of 2015 were $13.8 million compared to $16.4 million for the comparable 2014 period. General and administrative expenses for the first nine months of 2015 were $12.9 million compared to $11.9 million for the comparable 2014 period. Year-to-date operating expenses for 2015 also included restructuring charges of $1.3 million related to the organizational resizing announced in March 2015. The decrease in research and development expenses for the 2015 periods, compared to the same periods in 2014, was primarily the net result of lower manufacturing cost for imetelstat and reduced personnel-related costs resulting from the organizational resizing, which was partially offset by higher cost for the clinical development of imetelstat in collaboration with Janssen. The increase in general and administrative expenses for the 2015 periods, compared to the same periods in 2014, was primarily the result of higher non-cash stock-based compensation expense and increased legal costs associated with our patent portfolio. We ended the third quarter of 2015 with $151.8 million in cash and investments. We've not incurred any impairment charges on our marketable securities portfolio. I will now turn the call over to Chip to review recent company events. Chip?

Thanks, Olivia, good afternoon, everyone and thanks for joining. During the third quarter there were two important events for imetelstat that I would like to highlight. First, two papers on imetelstat were published as back to back articles in the September 3 issue of the New England Journal of Medicine. The paper reported results on Geron's proof of concept study in patients with essential thrombocytopenia and the Mayo Clinic pilot study in myelofibrosis where unprecedented molecular and bone marrow responses were observed in the studies. These data suggest imetelstat has disease modifying activity through the selected innovation of the malignant progenitor cell of the malignant clones responsible for the underlying diseases. The papers are available online in the New England Journal of Medicine website. Also in September the first patient was dosed in the IMbark myelofibrosis study. This study is the first to be initiated under the collaboration with Janssen. As we previously described IMbark is a one to one randomized single blind multicenter Phase 2 clinical study to evaluate the activity of two dose levels of imetelstat either 9.4 or 4.7 mgs/kg. Approximately 200 patients a 100 patients per dosing arm, with DIPSS intermediate-2 or high-risk myelofibrosis who have relapsed after or are refractory to a JAK inhibitor are planned to the enrolled in the study. The co-primary efficacy endpoints are spleen response and symptom response rate. The endpoints -- or these endpoints for the basis for the approval of JAK. Secondary efficacy endpoints include the number of patients achieving CR or PR, CI and a linear spleen and symptoms responses as well as an assessment of overall survival. We believe the depth of response captured by the secondary outcomes is clearly unique to imetelstat and is further validated in the trial will enable clear differentiation of imetelstat effects compared to JAK inhibitors. Exploratory endpoints for the study includes cytogenetic and molecular response as well as Leukemia free survival. We hope these evaluation will lead to more insight into the underlying molecular pathology myelofibrosis and how imetelstat may affect such diseases processes. I would like to use the remainder of our time today to discuss the unmet medical need and myelodysplastic syndromes or MDS and the designers Janssen plan to Phase 2-3 study of imetelstat in patients with lower risk MDS that is expected to be uploaded to clintrials.gov shortly. The term MDS describes a group of disorders associated with malignant progenitor cell clones where both the bone marrow and profile blood cells may have abnormal or dysplastic cell morphology and this is of course with the disorder was originally named for. MDS is characterized clinically by the ineffective production of red blood cells frequently resulting in severe anemia. In addition, low numbers of white blood cells and other cytopenias may cause life-threatening infections or bleeding. Transformation to secondary acute myeloid leukemia or ML occurs in up to 30% MDS cases and results in poor overall survival. MDS is the most common of the myeloid malignancies and is primarily a disease of the elderly. It is believed that approximately 12,000 reported new cases in the U.S. every year and the approximately 60,000 people estimated to be living with the disease may be an underestimate of the incidence in prevalence. The majority of patients approximately 70% fall into what is considered to be lower risk groups at diagnosis. According to the International Prognostic Scoring System or IPSS that takes into account the presence of a number of disease factors such as side cytopenias, cytogenetic and blast counts and assigns relative risks of progression to acute leukemia and overall survival. When initially diagnosed with MDS approximately 80% of patients have anemia or low red cell counts and low hemoglobin. Chronic anemia is a predominant clinical problem in lower risk MDS. Many of these patients become dependent on blood transfusions which can lead to elevated levels of iron in the blood and the other tissues that the body has no normal way to get rid of. Iron overload is potentially dangerous condition. Studies in patients with MDS have shown that iron overload resulting from regular blood cell transfusions is associated with poor overall survival and a higher risk of developing leukemia. This negative effect on survival depends on the number of red cell transfusion received per month. Two units per month may reduce life expectancy by up to 50%. The primary goal of therapy for lower risk patients with anemia is to restore or improve the production of red blood cells so that patients can become transfusion independent. Current treatment options for anemia are inadequate. Development of targeted therapies for MDS is not yet been successful and no new drug has been approved for MDS in the United States since at least 2006. Erythropoiesis Stimulating Agents or ESAs such as Epo improve anemia in approximately 60% of patients but the effect is transient with a median duration of responsible of approximately three years. Those patients who do not respond or who relapse quickly are predicted to have a poor survival. When Lenalidomide or Revlimid is approved for transfusion dependent anemia in the 10% to 20% of MDS patients who have a particular cytogenetic abnormality known as the LIP5Q and which a portion of the cells in their bone marrow have a dilution of long arm as chromosome 5. In a recent clinical trial of linomide in patients without 5Q and who are resistant to ESAs, approximately 27% became transfusion independent with a medium duration of just eight months. The initial testing of imetelstat patients in MDS occurred in the Mayo Clinic pilot study conducted by Dr. Ayalew Tefferi. Preliminary data from these patients have been accepted as an oral presentation at the 57th American Society of Hematology or ASH and the Annual Meeting to be held in Orlando, Florida from December 5 to December 8. Accepted abstract were published this morning on the ASH website. In Dr. Tefferi study nine patients with MDS-RARS were treated with imetelstat. MDS-RARS stands for refractory anemia with ringed sideroblasts and is a subtype of MDS in which patients have an increased risk of progression to AML and shortened survival. Seven of the patients in Dr. Tefferi’s studies were classified by IPSS as intermediate one risk and two patients were intermediate two risk. At the start of the study all patients were anemia with hemoglobin of less than 10 g/dL and eight of the nine patients were considered to be transfusion dependent according to the 2006 IWG criteria for MDS. Seven of the nine patients have received prior treatments including six previously received ESAs. All nine patients were given imetelstat as a starting dose of 7.5 mg/kg every four weeks. This was lower than the dose of 9.4 mg/k every three weeks given to the MF patients and Dr. Tefferi’s other cohort MDS study and that was in order to minimize the risk per myelosuppression in patients with already compromised bone marrow. The abstract used made the 2015 as a cutoff date for data and reported the medium duration of treatment of imetelstat to be 13.7 months with a range of 6.6 to 17.9 months. During treatment with imetelstat three of the eight patients or 38% who had been deemed transfusion dependent at the start of the study became transfusion independent, which was defined as not requiring transfusions for at least eight weeks. Among these patients transfusion independent started between weeks 9 and 14 of treatment and the medium duration was 28 weeks with a range of 9 to 37 weeks. As of the data cutoff abstract four patients remained on active treatment. Five patients discontinued treatment for the following reasons. One patient died during active therapy deemed unrelated to imetelstat. One patient was diagnosed with a second malignancy. One patient progressed into acute leukemia and two patients were deemed to have an insufficient response. Safety data are consistent with prior imetelstat study and information is reported in the abstract. Additional and updated efficacy and safety data will be reported during the oral presentation in December. The planned MDS study of imetelstat to be conducted by Janssen named emerge is a Phase 2-3 clinical study to evaluate imetelstat in transfusion dependent patients with IPSS low or intermediate one risk MDS following prior treatment with an erythropoiesis stimulating agent or ESA. Multiple medical centers across North and South America Europe and Asia are planned to participate in this clinical trial with the first side expected to be open for patient enrollment by the end of 2015. Details of the study designed and eligibility criteria as well as participating medical centers that will be updated on an ongoing basis will be made available to the public on the clintrials.gov website. Today, I would like to describe the basic design of the Emerge study and comment on the rational for several of the design elements. The study will consist of two parts and approximately 200 patients will be enrolled. Part one is a Phase 2 open label single arm design to assess the safety and efficacy of imetelstat. Up to 30 patients will be enrolled in part one and all will receive imetelstat. Janssen will review and asses data in part one and if supportive of a satisfactory benefit risk profile part two of the study will be initiated. Part two is a Phase 3 double blind randomized placebo controlled design to compare the efficacy of imetelstat against placebo. Approximately 170 patients will be enrolled in part two and will be assigned randomly in a 2:1 ratio to receive either imetelstat or placebo. Part two has been designed to enable registration of the drug for approval if the data from the trail are positive. The inclusion criteria for the study require, excuse me, the inclusion criteria for the study require that at the time of enrollment, each patient must be red blood cell at transfusion dependent. imetelstat for placebo in part two will be administered as an intravenous infusion. The starting dose will be 7.5 milligrams for kilogram every four weeks. The dose may be escalated according to certain conditions laid out in the protocol. Dose reductions for adverse events will follow protocol cost specified algorhythm. All patients will receive supportive care including transfusion or myeloid growth factors as needed per investigator discussion and according to local standard practices. The primary efficacy endpoint for both parts of this study is the rate of red blood cell transfusion independents lasting at least eight weeks defined as the proportion of patients without any red blood cell transfusion during any consecutive eight weeks since entry to the study. Excuse me, transfusion independents were eight weeks is a clinically meaningful and robust endpoint, which is included in the 2006 IWG response criteria for MDS and has been the basis for regulatory approvals in MDS. The secondary efficacy endpoints for both parts of this study included proportionate patients achieving red blood cell transfusion independents lasting at least 24 weeks as well as the time to and duration of red blood cell transfusion independents. In addition the proportion of patients achieving hematologic improvement including what is referred to as hematologic improvement erythroid and the proportion of patients achieving CRs or PRs both for 2006 IDVG criteria will be assessed. Assessment of CR and PR will capture the depth of the response. Other secondary endpoints include the proportion of patients requiring red blood cell transfusions and the amount, the proportion of patients requiring the use myeloid growth factors in the dose as well as assessments of the change in the patient's quality of life using several different validated instruments. Patients will be followed for an assessment of overall survival and time to progression to AML. The time to progression to AML and overall survival are important endpoints considering the natural history of the MDS includes transformation to acute leukemia in a meaningful proportion of patients. Safety outcomes will be assessed by procedures and measures consistent with previous imetelstat studies and include enhanced data collection and reporting for hepatobiliary associated laboratory filings and adverse events. The primary analysis of efficacy from both parts of the study is schedule to occur 12 months after the last patient is enrolled. The decision to advance from part one to part two of the study is not dependent on the primary analysis and may occur on the basis of an internal review by Janssen of the open label data to determine whether the data is supportive of the satisfactory benefit of this profile. The primary analysis will also evaluate secondary endpoints. We expect to report topline results from the primary analysis from part two of the study and that detailed information will be subsequently submitted for presentation at a future medical conference. We remain very enthusiastic about imetelstat and are pleased with the progress that Janssen has been making with the drug. The launch of two big clinical trials within one year of announcing collaboration is quite a remarkable accomplishment even for a company with the resources and expertise of Janssen. As we've indicated since announcing our collaboration with Janssen, imetelstat is our sole product and we would therefore like to diversify our pipeline. We're seeking new oncology products, programs or companies that we believe to be exciting and a strategic fit both scientifically and from business perspective to maximize the potential of our company for shareholders. We continue to conduct a substantial and meaningful effort to evaluate a number of potential candidates and we cannot say whether this will result in a transaction. However performing a rigorous and comprehensive process takes time and we're not in a position to make further comments about future timing of such transactions if any on the call today. Before we open the call to questions, I wanted to finish by conforming that there will be two oral presentations at ASH related to imetelstat. In addition to Dr. Tefferi's oral presentation of the data that we just described for the MDS large cohort of the male pilot study there will also be an oral presentation of data from the proof of concept study in ET investigating the dynamics of additional mutations besides JAK2, V617F CALR and nipple mutations following imetelstat treatment. Both presentations are scheduled to occur in the same session entitled Myeloproliferative Syndromes Clinical Early and Late Stage Trials in MPMs. This will be held on Saturday December 5, 2015, between 9:30 and 11 AM Eastern Time. A third imetelstat abstract containing nonclinical data evaluating the activity of the imetelstat in a non-clinical model of AML was selected for presentation as a poster in a Saturday evening poster session. This information is also contained in a press release we issued this morning. We are planning to host a webcast event for investors and analysts to discuss the data presented at ASH as well as the designs of the Phase 2 embark and Phase 2-3 emerge studies being conducted by Janssen. Stay tuned for more information and we look forward to seeing you there. Thank you for listening. I would be very pleased to answer questions in the time we have remaining. So with the operator let's open the call to questions please.

[Operator Instructions] And our first question comes from Charles Duncan of Piper Jaffray. Your line is now open.

Hi this is Sarah Weber on for Charles. Congratulations on the progress. Could talk a little bit more about you believe Janssen would like to see in terms of advancing imetelstat from part one to part two of that study.

I think it’s a little hard to really comment on that today other than to make the comments directly about having a favorable benefit risk ratio. The specifics of that obviously are going to depend on the data and I think we're not really in a position to comment to strongly about that. Obviously, we would have to believe that a double blind placebo controlled phase 3 study would be likely to be successfully, but beyond that I don’t think we have any specific targets right now that we've established.

Okay. And then just one additional question in terms of endlicensing and additional asset what kind of attributes are you looking for in an ideal asset?

Well, I think we've talked about that previously a bit. It has to make business sense to. so one of the criteria that we have commented on previously is that it would need to be oncology focused and we certainly have been looking predominantly and hematology-oncology focus because we really know that space pretty well and of course with imetelstat were deeply meshed in the myeloid side of the hem-malignancies. Beyond that we have been pretty open about whether it would be an in-licence or whether it would be a platform that we would acquire or even potential a company, but beyond that we haven’t made any further characterizations.

Thank you for taking my question.

Our next question comes from Thomas Yip of FBR & Company. Your line is now open.

Hi, guys, congrats on the progress on imetelstat and look for very much to ASH in the month. The first question about the new MD as an occasion, can you perhaps outline how you see imetelstat to set into the current MDS driven landscaped.

We can’t think Thomas for the question. The big problem with low risk MDS is anemia, severe anemia and in particular anemia that requires transfusions and as we commented on the call and I just elaborated a bit, transfusions although they are relatively inexpensive they really not entirely benign by any means. A side from issues of actually getting into transfusions, which we all know can carry some degree of infectious disease risk in the like. The real issue with repeated transfusion is accumulation of the iron in internal organs and the blood and bone marrow for that matter and that causes toxicity, which causes actually a diminished long term survival. So that’s why transfusion independent is really a well-established regulatory outcome. Current therapies beyond transfusion are really pretty much related to ESAs or erythrocyte stimulating agents EPO, but as we commented they don’t ordinarily give a very prolonged improvement and in patients who have relatively high levels of EPO already, obviously they don’t have a lot of -- they are actually not indicated or don’t have a lot of utility. The hypomethylating agents are used in some cases in the United States they're on level for low risk. But in Europe, they're actually not but again they have a relatively limited period of time that they usually improve this. Now Lenalidomide Revlimid is approved for use in the relatively small number of patients with 5Q delete syndrome as it’s called and it's certainly we know that there is some data out there that I sighted that there is a 27%, they have 27% transfusion independence rate and in a study of Lenalidomide in non-type 2 delete syndrome patients. And just that that will be filed eventually as a SNDA but I think the point is that this really would fit very nicely into that space, into that low risk space because we believe that the malignant progenitor cell clones that are responsible for MDS have a lot of similarities to the same type of mechanism of action in MS. And so we have a lot of hope that we'll actually be improving the underlying disease characteristic of MDS. That’s still to be proven, but I think we would see it sitting in certainly in lieu of transfusions and if ESAs have either not worked or patients have become resistant to it. That's sort of where it's aimed at the moment.

Okay. That sounds good. Thanks for the details. That makes a lot of sense. Since one of the topic of ASH I see that one of the ASH abstracts are focused on genetic mutation, can you just perhaps quickly align what is the significance of specific G mutations and either MF or may be MDS or even AML?

Well that’s the 64 gazillion dollar question that a lot of sequence basements of various hematology oncologists hospitals are churning away at. I think we really can’t go outside of the four corners of the abstracts under the embargo rules. So really can’t make any further comments on that particular abstract. But I will just simply say this is a major effort on the part of the community to understand what are driver mutations both in the sense of the first mutations to be seen but also those that are thought to actually be causative of the underlying disease but what we now know is that there are used really numerous clones with numerous mutations involved in most of these Myeloid hematologic malignancies. I think it really is uncertain today other than knowing some mutations have certain prognostic factors associated with them in sort of a statistical arrangement. I think beyond that we really don’t know how individual mutations cause or associated with particular elements of the disease. Understanding that will obviously give us tools for ultimately in the future, may be patient selection and certainly that are prognostic opportunities and may be in the development of new very targeted therapies, but for today we're still very much, the community is still very much in a search and research mode. So I don't think we can say a whole lot more about that moment. Long winded explanation so I am not really sure.

Okay got it, thanks again for taking my questions and looking forward to ASH.

Thank you.

And next question comes from [indiscernible] of Stifel. Your line is now open.

Hey can you hear me now. Okay, thank you taking my question. So you mentioned that you're going to leverage or expand in oncology but you also have extensive experience in endocrinology. Are you looking for in this space as well?

No, good question, it is true for those who don’t know on the call, I am in an endocrinologist and I have a couple of companies previously that were endocrine companies, but no the company as a whole Geron has really been focused from many years now on encology and I would like to say that we are very much in a clinical and regulatory flow associated, the information flow associated with in particular hematologic malignancies because of our continuing collaboration with Jannsen and work with imetelstat. So I think that’s really then natural place for us to be focused. It does not rule out there may be certain types of technology platforms for example or even particular products that might have application outside of oncology, but really our focus has been oncology with hematologic oncology as the principal focus.

Okay. Thank you very much.

Our next question comes from Ling Wang of Oppenheimer. Your line is now open.

Thank you. Congratulations on the progress. Couple of questions here so first may be already mentioned that, but I wanted clarify for the phase 2-3 emergency study what are any other patient even if you used the mutation status to as they are on a criteria such as review one mutation.

Yes, we are not, it’s too early to draw conclusions about the applicability of individual mutations or even constellations of mutations as patient selection tools as you undoubtedly know Ling that’s a lengthy development process in and of itself. I don’t think we really have enough information about these mutations to go there especially with such important study. So the purpose of this study is actually are to define the clinical phenotypes that are appropriate and going to be used to define the patients enrolment studies and then with respectively evaluate the apparent associations between certain mutations and outcomes of the study and outcomes of the individual patient outcomes. That hopefully will get us a little bit closer to understanding what are important mutations, what may be bi-standard mutations in the like, but it’s certainly too early to be using anything as patient selection tool.

Got it. And also it looks like Janssen decides to do a pretty small Phase two trial for MDS and then move to Faze three versus myelofibrosis trial a start up pretty big full blown Phase two trial. Can you share maybe with us what are the thoughts and considerations here?

So it’s good observation. I think the observation that obviously we have a Phase 3 component to the MDS study. Certainly suggest that the Janssen has some confidence based on results today to be planning such as significant study. I think that’s a real built of confidence. It has also build confidence in the drug as a whole. On the MF we have always said and would continue to say that a key purpose in the MF study is to really refine the dose and I think that’s really important and that’s a key plan for that study. So that doesn’t lend itself to placebo controlled phase three study and hence the MF study remains a single agent but multi dose finding study.

Okay. And lastly at ASH presentation I just want to clarify we are going to get updated results and this is what have been positive today on that abstract.

Well that’s historically been the fact, but these are academic collaborators and they are going to at the end of the day have the dispositive view as to exactly what they will present. So we don’t have control over that and actually don’t really know for sure, but that has historically been the fact. So I guess we'll all go and see what comes out.

All right. Okay. Thank you. And congratulations again.

Thank you.

Thanks Ling.

And this concludes our question-and-answer session. I would now like to turn the call back over to Dr. Scarlett for any further remarks.

Well, thanks a lot everybody for this call. I think we're feeling very good about how the program is shaping up and we look forward to further discussions and seeing many of you at ASH. Thank a lot.