Geron Corp (GERN) 2013 Q2 法說會逐字稿

Good day, ladies and gentlemen, and welcome to the second-quarter 2013 Geron earnings conference call. My name is Derek, and I will be your operator for today. At this time, all participants are in a listen-only mode. We shall facilitate a question-and-answer session at the end of the conference. (Operator Instructions) I would now like to turn the conference over to Anna Krassowska, head of Investor Relations.

Thank you, Derek. Good afternoon, everyone, and thank you for joining us for the Geron second-quarter 2013 earnings call. With me today are Dr. John Scarlett, President and Chief Executive Officer; Olivia Bloom, Senior Vice President Finance and Chief Financial Officer; and Craig Parker, Senior Vice President and Corporate Development.

This afternoon, we issued a press release that reported results for the second quarter ended June 30, 2013. 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 until September 9.

Before we begin, please note that, except for statements of historical fact, the statements during this conference call are forward-looking statements under the Safe Harbor provision of the Private Securities Litigation Reform Act of 1995. These include, without limitation, statements regarding the timelines, prospects, and plans for imetelstat including anticipated timelines for a clinical study of enrollment, clinical results and data, the therapeutic potential and safety of imetelstat, and financial or operational projections or requirements including spending guidelines. These statements involve risks and uncertainties that can cause actual results to differ materially from those in such forward-looking statements. Information concerning factors that could cause actual results to differ materially from those in the forward-looking statements is contained in Geron's periodic reports filed with the Securities and Exchange Commission under the heading risk factors, including Geron's quarterly report on Form 10-Q for the quarter ended June 30, 2013. 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 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, or circumstances.

We will begin today's call with a summary of the second-quarter operating results from Olivia, and then Chip will review the recent events. Olivia?

Thanks, Anna. Good afternoon. For the second quarter of 2013, the Company reported a net loss of $8.9 million, or $0.07 a share, compared to $18.3 million, or $0.14 per share, for the comparable 2012 period. Net loss for the first six months of 2013 was $20.8 million, or $0.16 per share, compared to $37.1 million, or $0.29 per share, for the comparable 2012 period. The Company ended the second quarter of 2013 with $71.6 million in cash and investments.

Revenues for the second quarter of 2013 were $112,000, compared to $130,000 for the comparable 2012 period. Revenues for the first six months of 2013 were $877,000, compared to $1.4 million for the comparable 2012 period. Total operating expenses for the second quarter of 2013 were $9.1 million, compared to $18.6 million for the comparable 2012 period. Research and development expenses for the second quarter of 2013 were $4.8 million, compared to $12.8 million for the comparable 2012 period.

General and administrative expenses for the second quarter of 2013 were $3.4 million, compared to $5.8 million for the comparable 2012 period.

Operating expenses for the 2013 second quarter also included restructuring charges of $838,000, in connection with the Company's decisions in April to discontinue its discovery research program and companion diagnostic program based on telomere length. As well as close its research laboratory facility, which resulted in a reduction of the Company's workforce from 64 to 44 positions.

Total operating expenses for the first six months of 2013 were $21.8 million, compared to $38.8 million for the comparable 2012 period. Research and development expenses for the first six months of 2013 were $12.7 million, compared to $27.9 million for the comparable 2012 period.

General and administrative expenses for the first six months of 2013 were $8.2 million, compared to $10.9 million for the comparable 2012 period.

The year-to-date operating expenses for 2013 also included restructuring charges of $916,000.

The decrease in research in development expenses for the three- and six-months periods ending June 30, 2013, compared to the same period in 2012, primarily reflected reduced personnel-related costs resulting from the recent restructuring, lower cost for the manufacturing of imetelstat and GRN1005 drug products, and reduced clinical trial expenses with the wind-down of the imetelstat trials in solid tumors and GRN1005 trials in patients with brain metastases.

The decrease in general administrative expenses for the three- and six-month periods ending June 30, 2013, compared to the same periods in 2012, primarily reflected reduced personnel-related costs resulting from the recent restructuring and lower legal costs associated with the Company's patent portfolio.

Non-cash operating expenses, which primarily included stock-based compensation and depreciation, were approximately $1.6 million and $3.2 million for the three- and six-month periods ending June 30, 2013, respectively, compared to $2.1 million and $4.4 million for the comparable 2012 period.

Interest and other income for the second quarter of 2013 amounted to $56,000, compared to $165,000 for the comparable 2012 period. Interest and other income for the first six months of 2013 was $137,000, compared to $341,000 for the comparable 2012 period. The decline in interest and other income primarily reflected a decrease in cash and investment balances due to the use of cash for operations. The Company has not incurred any impairment charges on its investment portfolio.

At the beginning of the year, we projected an operating expense burn of approximately $33 million for 2013. Given current and anticipated activities for the second half of 2013, I expect an increase in total operating expenses of approximately $4 million for a projected total 2013 operating expense burn of approximately $37 million. The increase in projected 2013 operating expenses reflects additional costs associated with imetelstat manufacturing and anticipated transaction costs related to the divestiture of the stem cell assets.

We expect to end 2013 approximately with $50 to $55 million in cash and investments.

I will now turn the call over to Chip, who will review our recent company events. Chip?

Thanks, Olivia. Good afternoon, everybody. I'll begin with the updated clinical results from the Phase II trial of imetelstat in patients with essential thrombocytopenia, or ET, that was presented at the European Hematology Association conference in June. Then I'll provide a brief update on the recent progress as it's been reported to us by Dr. Tefferi on his investigator response trial in myelofibrosis ongoing at the Mayo Clinic.

Our rationale for studying imetelstat in ET was to provide a proof of concept for further development of imetelstat in a broader range in the hematologic myeloid malignancies, including myelofibrosis, where there is a clear unmet medical need for a product that could be disease-modifying. The updated results from the ET trial presented at EHA included data for an additional six months of treatment and follow-up to the 14 patients presented at the original American Society of Hematology, or ASH, annual meeting in December of last year. And also included data from four additional patients enrolled in the trial after the data cutoff for the ASH presentation.

As of the May 2013 data cutoff for EHA, the median time on imetelstat treatment was 14 months, ranging from three months to 2.5 years. The updated data were consistent with the high hematologic and molecular response rates reported at ASH. In addition, since the hematologic and molecular responses were contained in patients on treatment, imetelstat appears to have good durability in its effects on disease.

The ET trial has been closed to further patient enrollment, but we continue to treat and follow patients previously enrolled in the trial.

Long-term administration of imetelstat was generally well-tolerated in this trial. There were no new safety signals observed in the six-month update and no patients discontinued from the trial due to drug-related adverse events.

On our last quarterly call, we discussed our internal investigation into liver function test observations in the ET trial. The investigation was conducted in collaboration with external liver experts that we engaged as consultants. We have now completed the current investigation process. Based on the data from the investigation, the liver-function test abnormalities do not appear to progressively worsen over time and have not resulted in any clinical swelling. No patients discontinued study treatment due to the liver biochemistry abnormalities.

Based on the conclusions from the investigation, the external liver experts recommended no changes to the use or administration of imetelstat.

I'd like to comment now on Dr. Tefferi's investigator-sponsored trial in myelofibrosis at Mayo Clinic. Because MS is also, like ET, a myeloproliferative neoplasm driven by malignant myeloid progenitor cells, we and Dr. Tefferi reasoned that imetelstat might have a similar disease-modifying effect in patients with MS. And therefore, he initiated the study at Mayo Clinic late last year.

This study is an open-label trial in patients with intermediate or high risk of primary myelofibrosis, post-essential thrombocythemia MF, or post-polycythemia [vera MF]. The study is designed to evaluate the safety and efficacy of the imetelstat, as well as determine an appropriate dose and schedule for further evaluation. The primary endpoint is overall response rate, which is defined by the proportion of patients who were classified as responders, according to the International Working Group for Myelofibrosis Research and Treatment, or IWG, criteria.

This means that they have achieved either clinical improvement, CI; partial remission, a PR; or complete remission, a CR. Secondary endpoints for this study include reduction of spleen size, transfusion independence, safety, and tolerability.

Dr. Tefferi has provided the following communications to us regarding the enrollment status of his MF trial. First, enrollment of the first 11 patients, in which the dose of imetelstat was given once every three weeks, was completed at the end of March 2013.

Second, enrollment of the second cohort of 11 patients, in which Dr. Tefferi increased the dose intensity of imetelstat to levels similar to those used in ET trial, was completed at the end of May of this year. Third, in both cohorts, the pre-specified criteria and the clinical protocol of at least two responders, according to IWG criteria, were met and enabled expanded enrollment into those cohorts.

In addition, Dr. Tefferi communicated to us that the Mayo Clinic Institutional Review Board has recently approved a protocol amendment to add a new cohort of 11 patients with MS that has transformed into acute leukemia, known as blast-phase MF. BLast-phase MF is largely refractory to the conventional induction chemotherapy that is used for acute myelogenous leukemia. As a consequence, these patients have a dire prognosis with a median survival of less than six months, and are in great need of novel and effective therapies.

We continue to be pleased by the progress that Dr. Tefferi has made, and expect the data from the study, if positive, will enable us to design and initiate a Geron-sponsored multi-center study in MF.

We believe a disease such as MF and possibly acute myelogenous leukemia, or AML, and myelodysplastic syndrome, or MDS, represent the greatest value-creating opportunity for Geron because many patients have significant unmet medical needs. And, unlike existing therapies, we believe imetelstat has the potential to be a disease-modifying treatment.

Before we move on to questions, I would like to comment, because the MF study is ongoing and because the intention is for a mature data set to be presented in an appropriate peer-reviewed medical conference in the future, we're not going to comment further today on any safety or efficacy observations from Dr. Tefferi's ongoing IST.

So with that operator, let's open the call to questions.

(Operator Instructions) Charles Duncan, Piper Jaffray.

This is Roy in for Charles. Thanks for taking my questions. Sounds like good progress with the MS study. Had a few questions now on the safety or efficacy if you could possibly address them. I guess, what may be the possible criteria for the third cohort? And it's not clear to me, are there two different cohorts at blast phase MF, and then a third cohort of, I guess, non-blast phase MF. If you could address that, thanks.

Sure. So there currently two cohorts of patients with non-blast phase, that is MF, alone. The first cohort was started with Q3 weekly dosing. The second cohort had dosing very similar to what we gave in the ET study, which was closer to weekly dosing; at least initially. And both of those cohorts are now approved because of the responses seen for additional patients to go into those cohorts. Then there was a third cohort. Maybe we should stop saying first, second, and third and just use cohort A, B, and C. So A and B were the ones that I just spoke to.

And then there is a third cohort, if you will. This third cohort, which is initially a cohort of 11 patients -- and these are patients who specifically -- where their MF is transformed into acute leukemia known as blast phase MF. So they are separate and distinct from the other two cohorts of non-blast phase MF. Does that answer your question, Roy?

Yes, I think so. I guess I'm not familiar with that. Have there been any recent studies with other agents where you could maybe get a sense of what those patients look like?

Blast phase patients, this is where the disease MF, which is bad enough, has transformed itself into an acute leukemia, meaning that there are greater than 20% blasts. Very, very, very immature cells -- white cells, and greater than 20% blasts. And it is considerably more difficult to successfully treat then de novo AML. It looks like AML, but it has a very, very poor prognosis. As we mentioned, in general, these patients have a survival -- median survival of less than six months once they transform into blast phase. So it's quite an acute illness. And it's mostly characterized by, again, a very AML-looking picture in the peripheral blood and, for that matter I guess, in the bone marrow. But it's quite difficult to treat. And they use a variety of different agents for this, but none of them have been particularly successful.

Okay. And then if I could get one last question. I guess kind of looking forward, maybe speculate a little bit. Let's assume that imetelstat continues to behave well, and you get a good clinical response. And, I guess, what other agents that are made be coming down the pike are you guys watching closely, in terms of either potential and competition or just defining the space? Thanks.

We don't see anything right now that has any kind of similar mechanism of action. I mean, this is really quite a unique drug. And I think, obviously, the clinical profile of imetelstat will define where it sits in the competitive landscape. And we're not in a position to talk about that today. But I don't think this is something where -- I think this is quite a different product. It is a product that is given by infusion. Most of the other example JAK inhibitors are oral agents.

As we've sort of harped on, we think that there's at least a potential for disease modification -- meaningful disease modification. I think the history of most of the JAK inhibitors -- not to throw cold water on them, if I had MF, I'd want to be taking one today, but nevertheless they haven't shown themselves to be very effective in addressing the underlying malignant clone and malignant disease. And so I think it's quite -- this is quite a unique drug. And so we'll have to see think -- I think we'll have to all see the full profile, and then I think it will become fairly evident where it sits in the competitive landscape.

Great, appreciate it. Thank you.

(Operator Instructions) Brian Klein, Stifel Nicolaus.

Thanks for taking my questions. So first, I know that you don't control Dr. Tefferi's data. But just wondering if it's fair to assume that we might get a clinical update at the upcoming ASH meeting in December?

Well, we said all along, Brian, that we expected that he would want to have results available at that time. And so I don't think it's our place to tell you whether that will or won't happen and under what circumstances. But that's always been the expectation and certainly been his goal.

Great. And then as more cohorts of patients are added to the study, how quickly do you think you can turn around and initiate your own internal Phase II program? Are you going to now wait until this blast phase cohort is fully enrolled and wait for that data? Or do you think you can move ahead and just the MF alone?

Well, to be determined based on data. But in principle, I don't think that we need to wait for blast phase MF data. I think that's actually a pretty different manifestation of the disease. And I'm not at all sure anyone would be enthusiastic about commingling patients with blast phase MF in a more conventional MF story.

I mean, the study that (inaudible) is doing as we speak has a very similar patient populations that, which all the JAK inhibitors in the study, which is dips intermediate two and high-risk patients. That protocol, much like theirs, never contemplated treating blast phase MF. So I think if we do --- if blast phase MF turns out to be attractive, which we don't know yet but if it does, I think that would be the subject, I would think, of a different program. Because it's quite different treatment -- presumably different treatment of rhythms. And certainly different treatment outcomes are being looked for.

But your expectation would be that in 2014 you could initiate a trial?

I would certainly hope so, yes. In blast phase MF?

No, in just MF.

In standard primary MF and post-PV/ET MF, yes. Absolutely.

Okay. And then you've talked in the past, and today as well, about how imetelstat might be disease-modifying. When you think about potential endpoints, is complete response the appropriate endpoint that we should be focused on? Or do you think that ultimately survival, or at least progression-free survival, is a better metric of disease modification?

Yes, thanks that's a great question. Well, both PRs and CRs, even with the newest criteria, are quite hard to achieve. I mean, as far as we can tell, none of the existing panoply of drugs out there have achieved any PRs and CRs. There have been some retrospective looks at some data here and there. But certainly, I don't think anyone believes that the existing JAK inhibitors, for example, much less the earlier products, actually produce real PRs and CRs.

So I think that if you were so fortunate as to have an agent that could induce a partial or complete remission, one would think that that would be a very attractive drug. I mean, obviously depending on the side effect profile. But that would be a very attractive drug. And I would find it quite hard to imagine that regulatory agencies would hold you to a standard today of having to show a survival benefit off the top. I also think that if you -- I mean, remember with the CR, you're clearing myelofibrosis from the bone marrow, right? And that's a pretty big deal; nobody has done that. So if you actually cleared it from the bone marrow, you know, and return the bone marrow to age-adjusted normal cellularity and lack of fibrosis, that would be a pretty big accomplishment. I wouldn't think that you would have to wait for a survival benefit to show up. I would certainly expect there would be one, but I don't think -- the regulatory standard would be another question. So that would have to be discussed at length with regulatory agencies. I'm talking off the top of my head, but that sort of my initial reaction to your question.

Okay, that's fair. One final question. You know, given the increased burn that you are expecting for this year, do you think that your current financial resources are sufficient to get you through a Company-funded Phase II trial?

Kind of depends on how big it is and how long it is, and what -- and we actually have to put into it. So I think I'm going to reserve commentary until we've got a little bit better feel for that, Brian. I mean, I think I'll just leave it at that. I think when we know what we're going to do, we'll be very happy to discuss our financial strategy with you that it will make a great deal of sense to do so.

Great, I appreciate the time. Thank you.

At this time, that will conclude the Q&A portion for today's conference. I would now like to turn the call back over to talk to John Scarlett for any closing remarks.

Well, thanks a lot guys. Appreciate you being on the call in a busy earnings season and a busy afternoon for earnings. So we look forward to telling you the story. And we look forward to the year progressing on as we see what happens towards the fourth quarter -- third and fourth quarter this year at Geron. Bye-bye.

Ladies and gentlemen, that concludes today's conference. We thank you for your participation and you may now disconnect. Have a great day.