GTx's CEO Hosts GTx POWER Trials Results Conference (Transcript)

Aug.19.13 | About: GTx, Inc. (GTXI)

GTx, Inc. (NASDAQ:GTXI)

GTx POWER Trials Results Conference Call

August 19, 2013 9:00 am ET

Executives

Mitchell S. Steiner – Vice Chairman and Chief Executive Officer

Jeffrey Crawford – Lead Investigator-POWER Trials and Chief-Division of Medical Oncology, Duke University School of Medicine

Marc S. Hanover – President and Chief Operating Officer

Analysts

Brian J. Klein – Stifel, Nicolaus & Co., Inc.

David M. Nierengarten – Wedbush Securities, Inc.

Ryan S. Martins – Lazard Capital Markets LLC

Eric T. Schmidt – Cowen & Co. LLC

Biren Amin – Jefferies LLC

Howard Liang – Leerink Swann LLC

Operator

Good morning, everyone and welcome to the GTx POWER Trial Results Conference Call. This call is being recorded. At the end of the Company's prepared remarks we will open the call for questions. Questions-and-answers - I’ll provide specific instructions at that point.

I will now turn the call over to Dr. Mitchell Steiner, CEO of GTx. Please go ahead sir.

Mitchell S. Steiner

Thank you, operator. Joining me on today's call is Dr. Jeff Crawford, the lead investigator of the POWER trials, who is the Chief of the Division of Medical Oncology at Duke University School of Medicine; and Marc Hanover, the COO and President of GTx.

Before we begin, let me remind you that we will be making forward-looking comments during today's call. And I direct you to the press release we filed this morning as well as the quarterly report filed on Form 10-Q with the SEC on July 22, 2013, where we discussed the risks and uncertainties that may affect our business. This morning GTx reported the results of the POWER1 and POWER2 clinical trials for the use of Enobosarm for the prevention and treatment of muscle wasting in patients with non-small cell lung cancer.

First, let me remind you about the trial's design. The POWER1 and POWER2 studies were each placebo controlled double-blind clinical trials, which randomize approximately 325 patients with stage III or IV non-small cell lung cancer to oral daily doses of placebo or Enobosarm 3 milligrams at the time they began their first line standard platinum doublet chemotherapy. The POWER trial is differed in the kind of first line chemotherapy that the patient received. In POWER1, they received a platinum plus paclitaxel or docetaxel whereas in POWER2, they received a platinum plus gemcitabine, pemetrexed or vinorelbine. This was done because the regulatory authorities wanted to be sure we controlled for the possible confounding effects of these chemotherapies. The POWER trials were designed to assess the co-primary endpoints by responder's analysis after three months of treatment with Enobosarm or placebo. Responders for lean body mass were defined as patients who maintained or showed improvement in total lean body mass muscle, assessed by DXA, while responders for physical function were those who showed a 10% or greater improvement in stair climb power. We also conducted pre specified continuous variable analyses of lean body mass and stair climb power for European regulatory authorities.

Overall, survival is being assessed as a safety endpoint, and GTx announced earlier this year that the FDA has designated Enobosarm for the prevention and treatment of muscle wasting in patients with non-small cell lung cancer as a fast track development program, because it qualifies as a drug candidate to treat a serious disease, and fill an unmet medical need.

Let me now review the top line results. The clinical trials failed to meet the overall pre-specified criteria for the responders’ analysis for the co-primary endpoints of lean body mass and physical function or stair climb power at Day 84 as agreed with FDA.

The responders’ analysis of the co-primary endpoint showed mixed results. P values for lean body mass was 0.036 and 0.113 for POWER1 and POWER2 respectively, and the p values for stair climb power was 0.315 and 0.289 respectively. As for the pre-specified continuous variable analysis discussed with the European regulators using the same end-points, Enobosarm demonstrated a consistently positive effect on lean body mass relative to placebo. In both studies p values were 0.0003 and 0.0227 for POWER1 and POWER2 respectively.

For stair climb power, the p value for POWER1 was 0.0336. However the p value for POWER2 was 0.7923. Notably missing data were well balanced between the arms in both trials for both endpoints.

Across both clinical trials and patients with non-small cell lung cancer receiving chemotherapy Enobosarm was well-tolerated with the occurrence of serious adverse events and overall incidence of adverse events was similar across placebo and treatment groups, but they differed between trials.

In the safety analysis of survival, there was no evidence of a difference between patients treated with Enobosarm versus placebo in either trial.

Published observational data suggests that lean body mass is related to survival outcome. This observational finding was replicated in each of the POWER trials based on exploratory analysis of current survival data from the trials using landmark analysis and time depending covariant Cox regression modeling that includes the lean body mass response and arm as covariance. The effect [size] [ph] and direction was similar in both of the trials demonstrating that lean body mass response is associated with longer survival.

Interestingly, in the preliminary data, the same association was not observed for responders on physical function meaning improvements in stair climb power were not associated with survival benefit. This encouraging survival signal for lean body mass was observed in both trials. While we are disappointed that both studies did not meet the pre-specified responders analysis criteria Day 84. We believe the overall results are compelling including the consistent positive effects on muscle, the fact that Enobosarm was well tolerated and the association of lean body mass with the survival signal.

Accordingly, we planned to initiate discussions with both the FDA and European Regulatory Authorities to discuss the path forward. Before I take questions, I would like to personally thank all the employees at GTx for their tremendous efforts in conducting these high-quality Phase 3 clinical studies as well as all of the principal investigators, and their staff at over 80 clinical sites in eight countries for their help in recruiting, and managing these patients. And most of all, I want to thank the patients with non-small cell lung cancer, who participated in the POWER1 and POWER2 clinical studies in order to make it possible for future patients to potentially have access to important therapies.

Operator, we're ready to take our first question.

Question-and-Answer Session

Operator

Ladies and gentlemen, we will now open the lines for the questions-and-answer portion of the conference. (Operator Instructions) Your first question comes from the line of Brian Klein from Stifel. Please proceed.

Brian J. Klein – Stifel, Nicolaus & Co., Inc.

Hi, guys sorry to hear about the disappointing news. Thanks for taking my questions. First, can you give us an update on the absolute number of survival events that have occurred between the two arms and when we might get an update on the final survival analysis?

Mitchell S. Steiner

Right. So the absolute events is roughly about 280 deaths have occurred at this point, and the survival assessment, safety assessment will be actionable at 450 deaths, which we expect to see in first quarter of 2014.

Brian J. Klein – Stifel, Nicolaus & Co., Inc.

And as before, the two arms you said were equally balanced in survival?

Mitchell S. Steiner

Yes, as reported at this point now there is again no difference in the two studies and two arms.

Brian J. Klein – Stifel, Nicolaus & Co., Inc.

Great. I wanted to ask specifically in the Day 84 results. There seems to be a discrepancy in that lean body mass you met the primary endpoint of less than 0.05 p value in POWER1. But then when you compare across to these stair climb power, it looks like you had a numerically worse outcome in terms of stair climb power versus POWER2. Can you comment on that discrepancy?

Mitchell S. Steiner

Yeah, versus POWER2. Yes, so what we are finding out now, so the discrepancy is you have two studies. The two studies are different studies, they are non-identical studies. We had to control for chemotherapy because we know that the chemotherapy itself can have effects on these same parameters.

And so the 504 study and the 505 study, again well controlled for their type of chemotherapy, and the 504 study, it appears that the patients did recent lean body mass they did similarly to 505. Whereas in the stair climb power, it appears that stair climb power the patients in 504 did better than 505. And we're trying to understand what those differences are. And one of the comments I made was that the safety of our compound looks very good in both studies.

I mean you really can't tell it from the placebo. They are very similar. However within each study, the patients are different. So in the 505 study, these patients are put on gemcitabine primarily vinorelbine or pemetrexed.

And these patients appear to be and again this is very preliminary. We are trying to work through it. It appeared to be a different set of patients. And is it because they put sicker patients on gemcitabine, because it has "less toxicity" than a taxane, but we also noticed that 45% to 50% of the patients in the 505 study have anemia whereas in the 504 study it's more like 24%. So we're going to learn a lot about the patients in each of these studies. This is just top line data right now.

Now I'm just trying to give you a flavor for there maybe a very reasonable explanation for why stair climb signal was what we wanted in 504 for continuous variable, whereas in 505 it was not. But I think the most important thing is the lean body mass effect was consistent in both and the survival signal that we saw related to lean body mass was consistent in both in direction and the effect [size] [ph].

Brian J. Klein – Stifel, Nicolaus & Co., Inc.

Thanks, and then just two final quick ones. Any comment on quality of life parameters? Number one and number two…

Mitchell S. Steiner

Yeah, it’s early, yeah go ahead.

Brian J. Klein – Stifel, Nicolaus & Co., Inc.

Number two was, if you could comment on whether you think stair climb power is inappropriate endpoint given what we're seeing here?

Mitchell S. Steiner

Yeah, so the first question is, what about quality of life. The answer is, we collected quality of life and we're still working through those data. That's a tertiary endpoint as you know FDA and European regulatory authorities don’t place much weight on it. So we're not, because of top line we kind of put it down the list of things to look at quickly.

The question related to stair climb power, look, we've done several studies using stair climb power. We did a 501 study in healthys where stair climb power was able to discriminate the effects of our drug. And in that study as you remember we increased lean body mass, we increased stair climb power. The 502 study, which was done in patients with cancer, five different cancers, including lung cancer, albeit they weren't all getting continuous chemotherapy. So some of them were on chemo, some were not. And in that, we did see an increase in lean body mass, and again we saw an improvement in stair climb power.

In 504 increase in lean body mass in patients on taxane, 91% of them were on paclitaxel and even with continuous poison, they showed a similar, if not more increase in lean body mass. As you know we met that endpoint and stair climb power by continuous variable worked. It's just the 505, we got lean body mass unequivocally increases, but stair climb power did not.

So I think what we're going to find is that stair climb power may not be a sensitive as a DXA machine, and there maybe reasons why stair climb power may not have a detected or not sensitive enough in a patient for example that has half of them with anemia.

So we know oxygen is important for physical function and we know lean body mass is also important for survival, and so maybe what we're seeing here is a very important signal from the standpoint of change in the character of the drug from being a drug that is just going after increasing lean body mass and physical function to a drug that also includes a potential for increasing lean body mass and having an impact on survival. So this is new information to us.

Quite frankly we were empowered to see this and the fact that we see it in each trial to the same degree I mean, this is really, really, really interesting from the standpoint of what we think we can do with this compelling evidence that unambiguously increases lean body mass.

Brian J. Klein – Stifel, Nicolaus & Co., Inc.

Thanks for taking my questions.

Mitchell S. Steiner

Thank you.

Operator

Your next question comes from the line of David Nierengarten from Wedbush Securities. Please proceed.

David M. Nierengarten – Wedbush Securities, Inc.

Hey, I had a couple of questions obviously, sorry to hear the news. When you talk about our survival benefit, I mean can you give any quantification of any differences in survival right now? Is there any top line data you can give us on that?

Mitchell S. Steiner

Yeah. So right now, because it's all preliminary. There is really not much more we can say than to say that we saw the effect in both studies. The effect was same degree and the same direction. The hazard ratio, I could probably make a comment about the hazard ratio, and that is that the hazard ratio difference was at least 20% in each study. So this is not a small signal. This is a strong signal that I want to tamper it by making sure that you understand it’s preliminary.

And again, we did not expect it from a standpoint that we weren’t powered for it. So we just need to understand it a little bit better. It’s not trivial, and so consequently we think we have something new. That lean body mass is "associated with" that may be responsible for, that we should move forward and have a discussion.

David M. Nierengarten – Wedbush Securities, Inc.

And have you – you haven’t had a discussion because the trial wasn’t powered for it or have you on the possibility of approval on a survival benefit with the regulatory agencies?

Mitchell S. Steiner

That’s right. And so we went to agencies. We were linking lean body mass as a clinical benefit in itself. So if somebody maintains or has an improvement in lean body mass, is that a clinical benefit and the agencies agreed to that.

And then next question is, can we measure physical function because what’s another clinical benefit of having an increase in lean body mass with some kind of physical function measure we chose stair climb power and the agreement was that a 10% or better improvement in that would be worthy.

They were trying to link stair climb power and lean body mass together. And survival, we just didn’t know whether or not we would see a signal. And it’s so funny is the literature is full of information that shows that if you have a reduction in lean body mass that you end up with shorter survival, nobody really has a tool to go back in there and make lean body mass go up. So it was purely a leap of faith that if you increase lean body mass, it would have anything to do with survival.

So we kind of stayed away from that, because that’s a different kind of trial. And so our thought was, well, we’ve got lots of evidence that increasing lean body mass would improve physical function. We have some of that evidence here as well. We have a unique patient population we need to understand in 505. But to have the ability to increase lean body mass and see a survival signal, which – it’s so funny, people say we know that lean body mass has something to do with survival, but this is the first time there’s an intervention that consistently builds muscle unambiguously to every study that was done and could be a real tool to test that hypothesis.

David M. Nierengarten – Wedbush Securities, Inc.

Thanks.

Operator

Your next question comes from the line of Biren Amin from Jefferies. Please proceed.

Biren Amin – Jefferies LLC

Yeah. I have a couple of questions regarding the data. So Mitch, maybe you could talk to us a little bit about the continuous variable analysis because I think in your prespecified analysis in POWER2, you missed on lean body mass with 0.113, but then were significant with this continuous variable analysis. So can you discuss that and whether the regulators may accept this analysis when you go to them? Thanks.

Mitchell S. Steiner

Yeah, great question. So, let me just make a comment about that. So it’s a very good point. So a responders analysis is an extremely conservative ITT analysis. The reason it’s conservative you give no credit to the patients that actually have some information, and so if you say you’re going to do a responders analysis at day 84 that means if a patient comes in at day 42 and has done stair climb and you’ve measured at DXA and they don’t come in for the day 84 visit, they’re zero. And so, you only give credit for those patients that make it to day 84. So if you die you get a zero. If you don’t commit to the assessment, you get a zero. If you drop out, you get a zero.

Whereas in continuous variable, you are allowed to look across the spectrum all times. So you’re looking at day 42, day 84, day 147. So you essentially are getting an average if you will of what’s happening to the patient over that period of time and you get partial credit for patients that at day 42 may have come in and whatever that data is, it’s in the evaluation. So by getting partial credit it takes you clearly over the top.

Now one interesting finding using a responders analysis, as you know in 504 using a responders analysis to lean body mass we hit with a P value of 0.036. But if you go to day 147, you hit again 0.026. So using the same responders analysis only looking at day 147, only giving credit to the patients of day 147, we hit and in 505, even though at day 84 was 0.113, P value at day 147 was 0.013.

So perhaps these patients from up to day 84 are getting their first line chemotherapy and by five months done with their first line chemotherapy and some of them do get a break. So it maybe that by not giving our drug concomitant with the poison or as much poison these patients are now turning around and even with the most conservative responders analysis we’re still hitting 0.013.

So the continuous variable gives you – and the continuous variable P values are even more dramatic in our favorite day 147. So for continuous, at day 147 for 504, our P value there was less than 0.0001 and for day 147 for 505 it’s 0.0036. So it’s not surprising that an anabolic agent need some time and we’re actually capturing that not only in the responders analysis but also in the continuous and that’s part of the reason we feel our data is compelling on muscle. And on stair climb power, it’s much more, it’s not as – the measurement is not as sensitive as a DEXA scan, but nonetheless, again it’s very similar to what we saw in the 502 study.

Biren Amin – Jefferies LLC

And, maybe if I could have a follow-up on the stair climb power. Do you think the reasons why you missed were because you had changed the criteria from the Phase 2 to the Phase 3 where you’re only allowing patients to climb eight steps versus 12 steps? Also were there any geographic differences in the trial?

Mitchell S. Steiner

Yes, there are no geographic differences in the trial. For the most part all of those covariates that could have interfered really – it was a very well done study with very little interference.

In terms of the difference in the studies, no I don’t think that’s the case. I think we are dealing with different patient populations. I think stair climb power is a good way to assess function. I think the challenge is that when you take a function measurement in different patients that have different issues going on, that it’s going to be sensitive or not sensitive, not that it’s not measuring what you wanted to measure. It’s whether the patients are capable of achieving what you’re asking during that period of time.

So that’s what we’re going to learn with 505. With 504, I mean the patients clearly were, we saw the controls. They were dropping and the treated were going up. I mean, stair climb power is a good measure of anabolic activity.

We need to learn now is why is it in a different patient population. I mean there is a reason why people have chosen to go onto gemcitabine. They tend to be patients that are different than patients who can quote take taxane and there’s also differences in how they’re given.

So, for example taxane, we tend to give them a dose at the beginning of the cycle and then they get a couple of weeks to recover, and mainly because they get a couple of weeks to recover that makes a difference in terms of how they respond to stair climb, where as gemcitabine, you’re not given that drug that drug as often. I mean you’re given that drug more often, and I don’t know. I’ve got Dr. Crawford with me. Dr. Crawford, would you want to comment on that, on how those drugs are given?

Jeffrey Crawford

So the taxane is given once every three weeks along with the platinum and the gemcitabine as a weekly regiment either two out of three or every week depending on how the physician prescribes it. So it’s a more frequent administration of chemotherapy clearly in that gemcitabine arm. That could have clearly influenced the results as well as in the fact you brought up about anemia. Clearly more anemia in that population which would make improvement in physical function more difficult in the [face] of anemia.

Mitchell S. Steiner

Okay?

Biren Amin – Jefferies LLC

Okay. Thanks.

Mitchell S. Steiner

All right. Thank you.

Operator

Your next question comes from line of Ryan Martins from Lazard Capital Markets. Please proceed.

Ryan S. Martins – Lazard Capital Markets LLC

Hi. Thanks for taking the questions. So Mitch, are you going to be awaiting the final survival data before you actually proceed with any filings or even discussions? Are you going to have discussions with the regulators before you see the final survival data?

Mitchell S. Steiner

Yeah, so remember there are different kinds of survival data. So the survival data that we’re waiting 450 deaths is a safety survival. And what that means is you’ve got patients that were treated with drug for five months and then after they’re treated for five months we basically follow their survival.

The survival data that we would go to the regulatory agencies with have more to do with answering a different question and the question is in those patients that are responders basically not just lean body mass, but to our drug and lean body mass that’s where we’re seeing the survival signal. So that is more of a discussion with the agencies about a strong survival signal and that survival signal is very different than “a safety signal” what happens when somebody comes after a period of time.

So we feel that the data that we have in both studies and pooled is sufficient to go to the regulatory agencies now. I mean, again we did not expect to have a drug that increases lean body mass, improves physical function also have a survival signal with these fewer event. We’re empowered for 844 events. And so this is very, very encouraging and we do believe that it’s sufficient to go to the agencies, in particularly the FDA, because for Europe we believe that all the prespecified endpoints for Europe are primarily met. And having a safe drug, I can’t say safe, well tolerated drug we did not see any safety signals. And having the potential for a survival signal really again changes the character of the drug.

And so for Europe, we’re going to meet with them and move ahead and with U.S. this is a different discussion. This is a different discussion. So the question is we already fast track with this indication. And now with this additional information what does it mean?

And, no, we’re not going to wait till first quarter of next year. That’s a safety endpoint and we feel pretty good now with almost 62%, 65% of the deaths reported that what we need to be focusing on is the value of lean body mass having a drug that can intervene i.e.; build lean body mass, and having evidence that needs to be confirmed that building lean body mass improves survival. Dr. Crawford, do you want to comment on that?

Jeffrey Crawford

And so I think in the field of lung cancer we’ve been – it’s been well known, since we overstated this disease that weight loss and functional status were bad prognostic signs for survival. Really until enobosarm we didn’t have an agent that potentially could reverse both of these situations. And now that we do, we see that increase in lean body mass may well be associated with prolonged survival. So reverse of a shorter survival.

I think it fits with this whole concept of providing good supportive care for our patients, providing maximum benefit to them while they are receiving chemotherapy, which has a lot of downside in the patient population. So if we are able to strengthen those patients, provide better muscle mass going into therapy and throughout therapy, it’s certainly likely that we’ll a survival difference and that’s what the data is showing us at this point.

Ryan S. Martins – Lazard Capital Markets LLC

Just a follow-up also, in terms of your discussion with regulators in Europe. So is the responder analysis not a part of that discussion? Was that not something that they wanted to see when you had designed the trials?

Mitchell S. Steiner

Our SAP pre-specifies that in U.S., the U.S. regulators wanted a responder analysis. Europe, they preferred a continuous variable analysis. So our SAP pre-specifies us to continuous variable analysis. So they’ll be happy to see the responders analysis, but they wanted the continuous variable one.

So for the continuous variable one, the 504 day 84 meets that need and 505, again the P value there for continuous day 84 is 0.0227 and we missed on stair climb. So, and we think we have a very good discussion about what stair climb means based on the patient population, and of course we do have 502, which was done in cancer patients and well controlled double-blind placebo study that was the one that was published in Lancet where we clearly again increased lean body mass and improved physical function stair climb.

So we think the package is there and it has been prespecified and again the other part that we didn’t expect is the fact that safety is so clean. And so with clean safety, the risk benefit works in our favor and again adding the survival signal I think – we’re encouraged, but we have to have the discussions.

Ryan S. Martins – Lazard Capital Markets LLC

Okay. Thank you. And maybe one follow-up. Can we have the updates on regulatory discussions then obviously prior to 1Q?

Mitchell S. Steiner

The regulatory discussions will be prior to 1Q. Yes. So we planned to have it by year-end. We planned to have information from both FDA and also EMEA. We are working very hard to get that because again these are new findings and for U.S. and for Europe we have an additional finding that is not just lean body mass physical function, but now survival.

Ryan S. Martins – Lazard Capital Markets LLC

Thanks.

Operator

Your next question comes from the line of Eric Schmidt from Cowen & Company. Please proceed.

Eric T. Schmidt – Cowen & Co. LLC

Thanks and thanks for taking my questions. Mitch, why does the press release refer to the continuous variable analysis as initial and exploratory? Was it post-hoc or was it part of the protocol?

Mitchell S. Steiner

It was part of the protocol, it was pre-specified and for the SAP it was pre-specified for Europe. So that would be the responders – that would be the package that we would send to Europe as agreed upon with Europe. So we said that only because some of the P values may change as we get more information.

I don’t think it will be materially so, but that was part of it. The other part of it is we want to clear that from an FDA standpoint we did miss our prespecified endpoints and then by definition for a continuous variable, that’s a secondary endpoint for the U.S. regulators. But for Europe, it’s actually our primary endpoints and the analysis that they requested.

Eric T. Schmidt – Cowen & Co. LLC

Okay. And then to play devil’s advocate, on potential approval on something around lean body mass, this may be for you or perhaps better for Dr. Crawford, why should a regulator approve a drug that improves lean body mass, but is not associated with demonstratably benefits in either survival or physical function that have been shown statistically significant on a progressive perspective randomized fashion?

Mitchell S. Steiner

So I’m going to let Dr. Crawford to answer that question.

Jeffrey Crawford

So I think we said a lot about this. As we’ve heard these are just top line data – different about this study. We started with patients who were previously untreated. So we are really trying to protect them through the course of chemotherapy. So let’s say I don’t think we can equate the lack of hitting the physical function benchmark as no effect on physical function.

I think in fact there is physical function in fact particularly in the first trial, I think also in the second. So I think there is a relationship clearly between with body mass and physical function well shown in prior trials and I think that will be supported here. So I think that’s an endpoint for our patient not only increasing lean body mass, but better physical function during therapy.

And obviously if there is a survival difference that’s going to be even more impactful for the regulators and certainly for us in lung cancer community. We provide the board of care for the (inaudible) quality life to our patients to be as good as possible, but we also provide supportive care because we expect the patients with good supportive care will in fact live longer than the patients who don’t have the supportive care and that’s been well shown in the landmark paper by Temel published in the New England Journal couple of years ago showing advanced lung cancer patients that provided support of care even without agents such as enobosarm one can see a survival improvement. So in fact, if one can empower these patients with more lean body mass, better physical function during therapy I think lung cancer committee would not find it unexpected to see a longer survival in that group.

Eric T. Schmidt – Cowen & Co. LLC

Thank you.

Operator

You have a follow-up question from the line of David Nierengarten from Wedbush Securities. Please proceed.

David M. Nierengarten – Wedbush Securities, Inc.

Hi. Thanks for taking the follow-up. Just to get back to the hazard ratio and overall survival, do you have any other parameters around survival? Have you reached a median survival or anything like that or have you just the rough hazard ratio? Thanks.

Mitchell S. Steiner

Yeah. If you don’t mind that’s all I can give you right now.

David M. Nierengarten – Wedbush Securities, Inc.

Okay.

Mitchell S. Steiner

What’s interesting to me is first of all it’s top line data, but what’s interesting to me is what I'm telling you. We didn't see in 504 and not 505. In other words this is not a single trial. So when you look at both well controlled studies and by the way the number of deaths of missing information, probably not number of deaths, but missing information for follow-ups. It’s not just deaths, but it’s also who is alive, is about 5%. So we have 95% of the information in 504 and 505 in terms of biostatistics. And so, in both the 504 and 505 studies, we see the same signal and basically the same effect and the effect is at least 20% different. So, this is encouraging.

David M. Nierengarten – Wedbush Securities, Inc.

All right. Thank you.

Mitchell S. Steiner

Thank you.

Operator

Your next question comes from line of Howard Liang from Leerink Swann. Please proceed.

Howard Liang – Leerink Swann & Co

Hi. Thanks for taking my questions. I just wanted to understand the 20% signal to survival that you refer to, I may be missing something. So, I think your press release you said in the safety analysis of survival there is no evidence of the difference between patients treated with Enobosarm and placebo either in clinical trial, is that not a overall survival in the safety analysis?

Mitchell S. Steiner

Yeah. So with that doesn’t in that analysis you are taking, the safety analysis you are taking lean body mass non-responders and lean body mass responders and adding them together. So, the overall survival as a safety analysis, the survival we're referring to has more to do with what does lean body mass mean to the patient in terms of survival. It's a different analysis.

Howard Liang – Leerink Swann LLC

So that analysis of looking at survival of patients with improvement in lean body mass versus those who didn't?

Mitchell S. Steiner

Correct. And between arms and both trials combined. So and the answer is yes.

Howard Liang – Leerink Swann LLC

Okay. But so that's not improvement in survival per se. It's just saying that in the population you're seeing there is improvement. They still are both longer that could have been due to their base line characteristics?

Mitchell S. Steiner

It's not due to baseline characteristics, but the answer is yes. That the difference is though, again we need to have enough events to prove and confirm this. So, in other words with the number of events that we have that we're powered for, it was for a safety analysis. And for the survival analysis, we're not going to able to come out of this study and tell you that patients are going to be cured.

We are going to come out of this analysis with two independent signals that suggest that these patients should be treated with drug longer. And also that lean body mass matters, and this is the first prospective study that it's looking at lean body mass, where you're looking at DEXA scans at baseline 42 days, Day 84, Day 147 and this is not an isolation because not just 504 and 505 that sees the signal and if you go and look at overall survival albeit we were empowered for it in our Lancet paper which is 502, at 1 milligram the hazard ratio was 0.8 and at 3 milligrams it was 0.7.

So the overall feeling is, the overall data shows that lean body mass must matter and that a study needs to be designed where patients are allowed to take the drug more than just five months, and to ask the very question where this lean body mass means to survival. So I would think of muscle is sort of targeted therapy just like with an outpatient really go after targeted therapy. And in lung cancer, the only strides that we are going to make today is to pull out the patients that matter, and Dr. Crawford do you want to comment on that?

Jeffrey Crawford

I think that our whole approach in lung cancer is really now personalized medicine. And we're trying to identify patients, who benefit from one strategy versus another. And what's really encouraging to me in the study is to see that the patients, the responders who improved their lean body mass or maintained it through therapy had a better long-term survival, and this is after five months of treatment. So in fact, clearly persisted beyond the five months of therapy. So I think it’s very intriguing.

Howard Liang – Leerink Swann LLC

Great. Just can you remind us the pre-specified p value hurdle for statistical significance for each of the co-primary endpoints with 0.05 or something apart from...

Mitchell S. Steiner

Yes, it was for the responders analysis it was 0.05 for each co-primary, you had to be less than 0.05 and for the continuous variable it's also basically the same but we're much more significant than that.

Howard Liang – Leerink Swann LLC

Then for your upcoming meeting with the FDA, what do you hope to achieve, I guess what would you propose to them as the next steps?

Mitchell S. Steiner

That's what we're working on right now. As you know, we have fast track status which means we can have an opportunity to meet as often as we want and have a great dialog with the FDA because that's what fast track means. So let us do that. And our goal is to have information by end of year.

Howard Liang – Leerink Swann LLC

Last quickly question, was there any difference in adverse events that was attributable to the treatment?

Mitchell S. Steiner

When you say treatment you mean to the chemo or to our drug.

Howard Liang – Leerink Swann LLC

To Enobosarm, sorry.

Mitchell S. Steiner

No, nothing at all. And in fact, we even looked at liver, liver was clean. I mean that was the only signal we saw in the early studies that we've said, we would focus on it, but we did not see any difference in liver adverse events in placebo versus and either 504 or 505. This is one of the cleanest drugs I’ve had an opportunity to work on.

Howard Liang – Leerink Swann LLC

Thanks very much.

Operator

We will now conclude the Q&A portion of the call. I would now like to turn the call back over to Dr. Mitchell Steiner for closing remarks.

Mitchell S. Steiner

We would like to thank you all for participating in the conference call today and we look forward to updating you on our progress with regulatory authorities in the coming months. Thank you, again.

Operator

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

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