Call Start: 09:00
Call End: 09:22
Exelixis, Inc. (NASDAQ:EXEL)
UBS Global Healthcare Conference
May 20, 2014 09:00 AM ET
Susan Hubbard - IR
Michael Morrissey - President and CEO
Andrew Peters - UBS
Andrew Peters - UBS
Good morning and welcome everyone to the 2014 UBS Global Healthcare Conference. My name is Andrew Peters and I'm the small midcap biotech analyst here at UBS. It's my pleasure today to introduce Exelixis, a leading player in oncology space. Speaking on behalf of the Company we have Susan Hubbard, Investor Relations; and Michael Morrissey the CEO. After the presentation there's a breakout session at 10 O'clock in the Carnegie East room. Thank you.
I am just going to read out brief forward looking statements. During the course of this presentation we will be making forward-looking statements regarding future events or the future performance of the company, including statements about possible future developments regarding clinical, regulatory, commercial, financial and strategic matters. Actual events or results of course could differ materially. We refer you to the documents that Exelixis' files from time to time with the SEC and in particular the company's Quarterly Report on Form 10-Q filed on May 1, 2014.
These documents contain and identify, under the heading Risk Factors, important factors that could cause actual results to differ materially from those contained in any forward-looking statement, including without limitation, the availability of data at the referenced times, risk and uncertainties related to the initiation, conduct and results of clinical trials; risks relating to the commercialization of COMETRIQ; risk and uncertainties related to regulatory approval processes and compliance with applicable regulatory requirements, the sufficiency of Exelixis capital and other resources and market competition.
And with that, I'll turn it over to you Mike.
All right. Thanks Susan. Good morning everybody. Thanks for coming out this morning. It’s my pleasure to tell you a bit about Exelixis today. It’s a big year for us in 2014. We have a lot going on with a variety of pivotal trials. As I am assuming many of you know we are focused on a single asset for the company called Cabozantinib. This is a compound that we have developed and discovered in house going back about 10 years now, very interesting compound has shown I think some encouraging and some provocative Phase II data over the last few years and I think our goal now is to really prove what the compound can do in a variety of indications in pivotal trials.
Our overall goal is to build the global franchise around Cabozantinib, we think it’s got potential to be a player that could impact a number of different tumor indications based upon our Phase II data and we’re really looking to maximize potential of this compound for patients and for shareholders from the standpoint of being able to build a broad focus around the compound.
So we actually have two assets in our stable of compounds, one that we focus on and with our development efforts, again Cabozantinib and the second compound called Cobimetinib. This is a compound that we again discovered, and brought to Phase I in house. We have partnered this compound; it’s on the right with Roche and Genentech. It’s a selective, if not specific, MEK inhibitor. It’s in a single pivotal trial right now in first line BRAF positive melanoma in combination with Zelboraf. It’s a very interesting compound in of its own right and you will hear more about that later on in the presentation.
But our main focus from a development point of view and from a stand point of view is on Cabozantinib. Again as Susan mentioned this is a compound that is now marketed for metastatic medullary thyroid cancer, MTC, we have very promising encouraging Phase III data at the end of 2011. We filed and got approved in this indication in 2012 in the U.S. We received approval as well in Europe in Q1 of this year. Small indication, we’ll talk more about that in a few minutes, but it’s the first step then and again building this franchise around the drug in the oncology study.
We have five ongoing pivotal trials for Cabozantinib currently and if you look at [indiscernible].gov you will see a very wide range of Phase I, Phase II clinical trials, single agent combinations across a variety of different indications, some that we’re sponsoring, the government through the NCI CTEP program at looking at 13 different trials, number of investigator sponsored or IST trails as well, looking to be able to broaden the possibilities of different indications again single agent Cabo combination with different drugs to be able to really understand the range of activity that we could see in this with this drug in the oncology setting. So very exciting time for us. Again, we have six ongoing pivotal trails between the two compounds which I think is rather rare for a company of our size and our stature, but I think it really underscores the commitment we have towards developing the agent and our confidence in the compound based upon it’s Phase II profile.
So 2014 is a big year for us again, we expect top line data from four pivotal trials, two for prostate cancer COMET-1 for overall survival COMET-2 for pain, talk more about those in a few minutes. The [indiscernible] trial was the label -- is the label enabling pivotal trial for MTC, the primary endpoint was progression free survival. Again we had a very strong signal there at the end of 2011 with a hazard ratio of 0.28, about a threefold increase in medium PFS compared to control. The overall survival endpoint is a secondary endpoint and it’s one that we expect to hit our events later this year, so we hope to have top line data there as well.
And again Roche is running the pivotal trial with Cobimetinib in combination with Zelboraf and they are guiding consistently they have top line data in a filing in 2014 with that combination. I’ll show you some data there towards the end.
So let’s talk about Cabozantinib. Again, this is a very interesting compound, very unique compound, it was designed to hit a variety of different receptors and kinases including VEGF axis and the MET axis, two important pathways which drive really all aspect to tumor biology and the vascularization that’s involved in helping tumors grow, survive and actually thrive. We had some I think early ideas based around how these two different pathways and two different targets have very redundant features as tumors are in this rapid growth phase and our goal was to block those effectively with a single molecule. And both pre-clinically and clinically we’ve seen very unique and I think very provocative data in terms of how this molecule can behave in different settings, different tumor types and obviously we’re looking now at pivotal trials to be able to prove that once and for all in a very definitive fashion to really establish efficacy and safety in a variety of different populations.
Our goal here is quite simple. It’s based upon the Phase II profile. If you look at the constellations of different indications, ideally we'll be able to build on the success of MTC, prostate cancer, renal cancer, liver cancer, other types of cancers, that we're interested in pursuing to be able to build a franchise around this molecule and that’s very rare. I think nowadays in the spirit of looking at single driver mutations for single subtypes of different tumor types, I think the ability of the molecule like cabozantinib to hit a variety of different tumor types by going after some of the key redundant pathways is a very important challenge for us and one that we're -- I think we're looking at very carefully now with our efforts in the development setting for the compounds.
So let’s talk about what we are doing so far to commercialize cabozantinib in the MTC area. Again we were approved for progressive metastatic MTC in November of 2012. This is a small micro niche - nano niche oncology population, by our estimates between 500 and 700 drug accessible patients in the first line and second line sitting in the U.S. So we've tried to right size, our commercial efforts to be able to, again not overspend but to get the -- be able to get the drug to patients who need the therapy. Because it’s a very-very terrible disease and one that we think we can bring benefit to. So we have a 15 rep sales force calling on about 1,400 or so different prescribers in the U.S. We’ve been booking about $5 billion or so in revenue. Certainly that was our Q1 number. So we’re getting off to reasonable start and we've been on the market now for about a year or so.
Our marketing dynamics, our prescribing dynamics are becoming a little bit more clear. For the patients who are currently on therapy, we have about 7.5 month duration of dosing which is good to see and about a quarter of those patients have been on drug for 12 months or more, really reinforcing the point that based upon our Phase III data, cabozantinib appears to have an impact on, again, delaying progression in this population.
So, again we're using MTC as a way to again, from a commercial point of view, to get the drug to patients that need it, first of all, and then learn how to commercialize as a company, to be able then to have really all the kinks worked out. So when we have success in other indications, we’re able to move rapidly and really make every day count in those larger, more commercially meaningful indications.
In terms of the EU, again we have a positive CHMP opinion that was received late last year. We received marketing authorization in March of this year. Again, we have orphan drug status maintained which is somewhat rare for a second drug in a small indication in Europe. And we have partnered with Sobi to support the EU MTC launch. That deal goes to the end of 2015, when again we would then be able to commercialize globally for other indications as well. So it's a very good way to work with Sobi to again monetize the asset in Europe, again, small indication but one that we want to be able to get working on in Europe, so that we’re ready then to go full force when we have positive data in other larger indications.
So again, Cabo is certainly ahead, again a variety of activity in a variety of different indications, most prominent and certainly most provocative user activity in the prostate cancer space. Again this is data that’s been now generated over the last three or four years. We've seen very provocative activity of the first compound showing the ability for a drug to either resolve or reverse very dramatic bone scan involvement with this drug. And again prostate cancer is known to have metastasis to the bone as the main driver for that morbidity and mortality with that disease. So, it’s the one that we looked at very carefully in the Phase II setting. We have two ongoing Phase III pivotal trials to be able to document a survival benefit and a (Pan) [ph] benefit with this drug. But the market is clearly one that is evolving rapidly with the advent and the introduction of six different compounds over the last few years.
With a survival benefit, we've seen a lot of moment there, a lot of success for patients, lots of new drugs, new options for patients with metastatic prostate cancer. And that’s a good news. The $1 billion number in the U.S and 2012 is an old number now, abiraterone is close to $2 billion this year, enzalutamide is close to $0.5 billion, so lots of movement there, lots of new options for patients with prostate cancer and that’s the good news right. I think the bad news is that, unfortunately no one with metastatic disease is being cured, which are prostate cancer. Every man that gets one of those drugs will progress eventually, some quick, some over time; and patients need new options to be able to then to address their emergent independent disease because once patients progress on abiraterone, enzalutamide, even [indiscernible], they often then have tumors that are emergent independent and have different drivers for that disease which we think cabozantinib can play an important role.
So again, we have worked very hard here as a company to (drop out) [ph] the compounds in Phase II, and we looked at about 350 patients in a variety of Phase II settings and trials to be able to understand the scope of limitations of the drug and obviously now we’re doing the appropriate Phase III trials to be able to really understand the activity in that setting.
We’re looking at a variety of other trials as well, with cabozantinib in the Phase II setting. And most notably the combinations of cabozantinib with abiraterone in the free chemo space, I'll talk about that in a few more minutes. But also we have plans to combine with enzalutamide with the idea that by combining [indiscernible] mechanisms, AR focus drugs like ab and enzalutamide with a compound like cabozantinib which can impact different disease processes, you might be able to extend the progression free survival with these patients for a long period of time.
So let’s talk about some of our Phase II data. Again, this is I think a one slide summary of several 100 patients worth of data to understand again what Cabozantinib does in this setting. Certainly we have again for the first time very clear evidence that we have a direct impact on metastatic bone disease by bone scan technology and other technologies as well. So we see, as you see on this one slide on the right very dramatic resolution of existing bone scans -- bone lesions by bone scan and we’ve been able to further validate that if you will with other technologies, various MRI techniques, various PET techniques as well as looking at bone biomarker.
So very I would say conclusive evidence that we have a direct impact on both the tumor in the bone and the bone itself, but again we have to prove that that actually can then lead to a benefit in terms of survival and pain in the ongoing pivotal trials. But we’ve seen in Phase II a reduction in pain, reduction in narcotics, a very clear reduction in circulating tumor cells, a property that’s been shared by others as well, so again good validation of the overall pharmacology in this disease setting. We’ve seen soft tissue disease shrink, nodal disease, liver disease, visceral disease all shrink. And we’ve seen very dramatic improvement in the progression free survival.
So, again overall very attractive profile in Phase II, one that we certainly have to validate further in Phase III but certainly has given and our coworkers and the academic area lots of interest and confidence to push this forward in a very I would say aggressive fashion.
So we again have two ongoing pivotal trials here, COMET-1 is focused on overall survival, this fully enrolled back at the end of last year, again randomized two to one Cabozantinib versus prednisone, we had interim analysis in March. We were told to go forward by the IDMC for the final analysis. We think that will happen later this year based upon the current event rate. So again stay tuned for that.
COMET-2 is focused on pain palliation, very important part of the story. Again these men with advanced prostate cancer as their disease progresses often suffer very debilitating pain as the bone disease gets worse. That drives a variety of comorbidities and ultimately their mortality. I would like to be able to again capitalize on what we saw on Phase II in terms of pain reduction or COMET reduction, COMET-2 is assigned to do that in a very regulated fashion. We’re still enrolling here and again expect to have top line data in 2014.
In terms of other trials, besides prostate cancer, we have three others ongoing. We talked about EXAM before. We have seen very interesting data in second line, renal cancer, second line liver cancer. We had data at ASCO in 2012 and we’ve used that data then as a spring board to then initiate other pivotal trials. Those are going (median) [ph] it's our second line RCC, this is our number one enrollment goal for 2014. I will get into that in a few minutes why that’s important and then CELESTIAL is for second line liver cancer. Again both are important large mature indications, lots of unmet medical needs, lots of patients in need of new therapies. So we’re really excited about these different indications and about our data from Phase II obviously you have to go forward now and get the pivotal Phase III trials done and let that data speak for itself.
So in terms of (median) [ph] again, we’ve seen very again interesting data in second line RCC, we have a paper and press that will come out shortly talking about that data that we had at ASCO few years ago in a very late line population with all the caveats that we have a single arm non-randomized collocations. We saw about a 30% response rates in this setting. We saw patients with bone disease have their bone disease shrink by various MRI techniques. We saw a PFS value that was 2.5 to three fold longer than what is normally seen in the Phase II setting with other VEGF targeting or MET targeting molecules. So very interesting data, obviously with all the caveats of being small non-randomized single arm study, but one that has driven a lot of interest in the compound in this indication.
So we have currently again, this is our highest enrollment parity for 2014, it’s one that we want to see get done enrolled and then read out as soon as possible, obviously having two (NYSE:GU) [ph] indications reading out back to back in ’14 and ’15 which we have the goals for (median) [ph] is a very important goal for us. Obviously a single (GU) [ph] focused sales force could handle both of those, so we’re very interested from the standpoint of having again this franchise ambition move forward having prostate and renal be the main drivers of that.
So stay tuned there. It’s enrolling well. We have most of the sites up and we’re at that steep part of the enrollment curve right now, so we’re excited about where this is at and where it’s going over the next year or so. With CELESTIAL again this is our Phase II trial in liver cancer. There is no current approved standard of care for second line liver post progression on Sorafenib, so large unmet medical need globally obviously, so one that we’re very excited about.
Again we have very I think very interesting data from Phase II and one that is going to enroll a little bit slower due to the kind of the complications of enrolling this trial in Asia but one that we’re doing I think at a pretty good cliff. Others ongoing, I mentioned before, I won’t deliver over the point here but again, the broad activity in terms of Cabozantinib has prompted again, 50 plus different trials in Phase Ib, Phase II.
Okay. So, let’s take a minute to wrap up talking about Cobimetinib, again this is our specific MEK inhibitor that we’re pursuing in collaboration with Roche Genentech. Again, this is a very hot space right now in terms of the idea that the available data both pre-clinically and clinically with other compounds, other systems shows that better efficacy and longer duration of therapy can be achieved by combining two different inhibition points on the MEK kinase pathway by going after the RAF target and the MET target with two different molecules. Certainly the GSK data that phase 1B 2 data looks very encouraging and really provide us some important proof-of-concept data.
Roche has put together a really nice story here and they had an update, had a meeting in Lithuania few weeks ago, and it’s summarized here. They have seen about a 90% confirmed response rate by combining cobimetinib with Zelboraf, again, they are RAF inhibitor. Again, in first line BRAF-mutant positive melanoma, the PFS data is new, so about 14 months of progression free survival which is very encouraging. Again, Zelboraf is 6.5 to seven months in the same setting, and about 85% -- approximately 85% estimate of survival at the one-year time point.
So again, looking very encouraging from the standpoint of single-agent Zelboraf, patients normally can progress to that within about six to seven months. Overall survival is about a year. So again looking pretty promising there from the standpoint of what this combination could do for patients in this setting.
So, again Roesch is guiding to have [indiscernible] data, again, that's the name of the pivotal trial and file in the second half of 2014. We’re very excited about that and looking forward to having that data be part of the overall data profile that we'll have out in 2014.
Okay, so in terms of upcoming event ASCO is right around the corner, in Chicago. We'll have nine different presentations for cabozantinib, focusing on a variety of different activity profiles and including trials in progress updates for RCC and HCC, as well as data from various IST and future programs in prostate cancer, bladder cancer, others. So I think it should be a pretty good show for us. Roche is presenting another update on cobimetinib that will repeat I think some of the data they got from Lithuania along with some PET data, so that should be interesting. And again all the abstracts are available now online. So take a look at those, please.
And my last slide is the financials. Again we had our Q1 call few weeks ago. We ended the quarter with 408 million or so in cash and our guidance remains the same. Looks like the year -- we expect to end the year with more than $200 million in cash.
So I will end with just a summary of our top priorities for the year. Obviously topline readouts are for the COMET studies, is our key number one priority, we’re excited about where that's going. And we’re ready to get that out when the data is available.
Certainly based upon success, we will be working hard to push forward regulatory filings on a global scale. We've got full filing teams in place right now for COMET1 and COMET2 doing a lot of data acquisition, data cleaning, all those kinds of things, all the blocking, and pathing you need to be ready to file based upon positive data, so I am feeling pretty good about that right now.
Certainly expediting enrolment from METEOR is our number 1 priority for enrolling trials. This is very important from the standpoint of having a second indication come online as quickly as possible out to prostate and certainly that’s one that's going very well right now, and then to complete our planning for the commercial build out in both the U.S. and Europe for prostate cancer and beyond. So a real commercial focus spent, once we have data in a larger indication.
So, with that, I will stop here. Thanks again for your time and interest and we'll be happy to take questions at the breakout. I think I'll kind of pause. Okay.
Yes, and if anybody who would like a copy of our slides emailed to you or want to be added to our email distribution list, please just catch me in the back of the room. Thank you.
All right. Thank you.
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