Lineage Cell Therapeutics, Inc.

Lineage Cell Therapeutics, Inc.

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Biotechnology

Lineage Cell Therapeutics, Inc. (LCTX) Q3 2016 Earnings Call Transcript

Published at 2016-11-03 22:13:39
Executives
Matthew Haines - Investor Relations at EVC Group, Inc. Adi Mohanty - Co-Chief Executive Officer Michael West - Co-Chief Executive Officer Russell Skibsted - Chief Financial Officer
Analysts
Kevin DeGeeter - Ladenburg Thalmann Keay Nakae - Chardan Capital Markets
Operator
Welcome to the BioTime Third Quarter 2016 Results Conference Call. As a reminder, this conference call is being recorded. I will now introduce your host for today’s conference, Matt Haines of BioTime Investor Relations. Matt, please go ahead.
Matthew Haines
Thank you, operator, and good afternoon, everyone. Thank you for joining us today for BioTime’s investor conference call and webcast to review the company’s results for the third quarter 2016 and recent corporate developments. There will be a tape replay of this call, which will be available approximately two hours after the call’s conclusion and will remain available for seven days. The operator will provide the replay instructions at the end of today’s call. With us today are Co-Chief Executive Officers, Adi Mohanty and Dr. Michael West; and Chief Financial Officer, Russell Skibsted. Each will make prepared remarks and then we will take questions from our covering analysts and institutional holders. Before we get started, we would like to remind you that during the course of this conference, the company will make some projections and forward-looking statements regarding future events. We encourage you to review the company’s filings with the SEC, including, without limitation to the company’s forms 10-K and 10-Q which identify the specific factors that may cause actual results or events to differ materially from those described in these forward-looking statements. These factors may include, without limitation, risks inherent in the development and/or commercialization of potential products, uncertainty in the results of clinical trials, or regulatory approvals, need and ability to obtain future capital, and maintenance of intellectual property rights. And with that, I’d like to turn the call over to Adi Mohanty, Chief Executive Officer of BioTime.
Adi Mohanty
Thank you, Matt. I’d like to thank everyone for joining us on the call today. During the third quarter and in recent weeks, we’ve made important clinical progress with both of our technology platforms, pluripotent stem cell technology and our HyStem three dimensional cell delivery matrix technology. We have released important positive clinical data from our OpRegen trial in dry-AMD and announced a presentation of some initial data from our Renevia pivotal trial and the medical aesthetic indication of HIV-related facial lipoatrophy. We expect these programs to generate numerous significant milestones in the coming months, which we expect will get these products closer to the patients that need them and this clinical progress should lead to increases in shareholder value. During our last conference call, we emphasized our commitment to our near-term focus on three primary objectives: first, advancing products like Renevia and OpRegens for clinical trials; second, simplifying the company’s structure to increase transparency and focus resources on the development of promising cellular therapies that address patient needs and large market opportunities; and third, unlocking value for our shareholders from our non-core subsidiaries and through the progress of our public subsidiaries. Clinical progress drives valuation in biotech. I’ll expand on this further, when I discuss our Renevia and OpRegen programs in a few minutes. Our second focus, simplification, helps us better manage our corporate structure in operations. Simplification will continue to help with the company focus on the higher priority activity. It also leads to improved communications with our current and potential shareholders as well as all other stakeholders. For example, when we deconsolidated Asterias’ financials from BioTime’s financials, this past May, it simplified how we reported our numbers and made our operating performance easier to understand. Regarding our third area of focus, unlocking value, our spin out of OncoCyte last December was a good example of unlocking value for our shareholders. Our holdings in our two publicly-traded subsidiaries, Asterias and OncoCyte, were together worth about $166 million as of September 30. Both companies have made excellent progress with their programs. I’ll discuss this further later in the call. So let’s begin with the clinical progress of BioTime’s two core therapeutic programs: Renevia for facial aesthetics and OpRegen for the treatment of the dry form of age-related macular degeneration or dry-AMD. Renevia is our product candidate that’s closest to the market. It is based on our proprietary cell delivery matrix, HyStem that is combined with the patient’s own cells in a single procedure done in the physician’s office. The HyStem platform is designed to allow for the stable engraftment of transplanted cells. Renevia is delivered directly to those parts of the patient face where there is fat loss, in order to promote facial tissue regeneration. The initial indication of it is that current clinical trial is lipoatrophy associated with HIV treatment, but we think there’s a much larger opportunity in facial lipoatrophy. Lipoatrophy is a term for fat loss. Facial fat loss is what happens in cases like when we age. It leads to wrinkles, laugh lines and other effects that create visible reminders that we’re all getting older. According to GBI Research, the global facial aesthetics market in 2016 is expected to be approximately $5.4 billion, which is more than double what it was only three years ago. The ongoing trial we have in Europe is a pivotal randomized evaluator blinded study in the HIV related lipoatrophy. We previously communicated that we would be fully enrolled by the end of this year. And I’m happy to say, we’re now, with nearly two months left in the year, almost completely enrolled. We expect top line data in the first-half of 2017. That dataset, if successful, should be sufficient to file for CE mark in Europe, which is generally a three month approval process. This means we could have an approved commercial product in Europe by the end of next year. Additionally, we’re looking to leverage the dataset from this trial in some key aesthetics markets such as South Korea and Brazil, two of the largest markets for aesthetics in the world. Earlier this week, we reported that our principal investigator for Renevia trial, Dr. Ramon Llull, will make a presentation on run-in patients for HIV associated facial lipoatrophy from the pivotal Renevia-02 trial at the 14th Annual International Federation for Adipose Therapeutics and Science Meeting on November 17 in San Diego. This meeting is also called the IFATS meeting. Dr. Llull is Director of Stem Europe Mallorca Center in Mallorca, Spain. And according to the abstract of the presentation that has been published, the data indicate that 3-D imaging suggest volumetric improvements were sustained through a one-year follow-up and that Renevia was safely administered with no serious adverse events. In addition, Dr. Llull will present the mechanistic rationale and data illustrating how Renevia may be generating new facial tissue following the procedure. We look forward to making Renevia available to the approximately 350,000 patients with HIV related lipoatrophy in Europe. However, we see the study in Europe as a gateway study to a much broader indication of facial lipoatrophy and tissue regeneration. The tissue regeneration market is a substantial one, with over one million augmentation procedures being performed each year in the U.S. alone and continues to grow rapidly. Our goal is to show that Renevia can result in new tissue growth. If this occurs, then we believe Renevia has the potential to become a superior alternative to synthetics and other existing techniques used in reconstruction of tissue. Facial aesthetics market is a self-pay market. There are already thousands of people in the U.S. that pay $8,000 to $10,000 for fat transfer procedures that don’t last long. We believe in the opportunity for a better long-lasting product. Now, moving to OpRegen which targets the major unmet medical need of dry-AMD, AMD is the leading cause of blindness in people over the age of 60. As a reminder, there are two types of AMD, the wet and dry forms. There are treatments in the market for wet AMD and together these generate over $5 billion in annual revenues. However there are no currently approved therapies available to treat dry-AMD, which is a much larger medical need and opportunity with 9 times as many patients in dry-AMD and there are for wet AMD. OpRegen has been progressing in a Phase I/IIa dose escalation clinical trial, which is evaluating the safety and efficacy of three different dose regimens in the advanced form of dry-AMD. We recently announced that data from the first cohort will be presented at a major conference on December 2 in Rome. The first patient cohort received a target dose of 50,000 cells. The primary focus of this cohort is safety. We have seen so far that RPE cells able to engraft successfully in the desired area of the retina in a nice monolayer. Also, OpRegen was safely administered with no significant adverse events reported to-date. We believe that for any cell therapy to potentially improve vision, it will be important for it to engraft as a single layer of cells in a similar fashion as the original RPE cells that are being replaced. Imaging from the first patient was just completed, one year of post-treatment clinical assessment shows evidence that the transplanted cells are engrafted and are alive at the 12 month assessment. Our principal investigator will present data from this and other patients at the upcoming conference in Rome in December. We’re very encouraged by these results, because as I mentioned before the risk profile of cell therapy products is different from other types of pharmaceuticals. We’re replacing cells that are missing. What is critical here is that the cell-stay and engraft at the desired location, because if they remain healthy then there is a higher probability of success in later stage trials. You’ll recall in - that in June we announced that the Data Safety Monitoring Board or DSMB before this trial had recommended moving forward with enrollment in the second patient cohort. The second cohort, which is ongoing it’s receiving a higher and more clinically significant 200,000 cell dose of OpRegen. It’s at these higher cell dose cohorts, where we believe OpRegen has the potential to demonstrate more meaningful therapeutic outcomes. We expect enrollment in the second patient cohort to be completed in 2016. And subject to DSMBs review of the second cohort. We expect to be allowed to begin administering the next dose to the third patient cohort before the year end. We expect to be able to begin sharing data from this second cohort early in 2017. As we continue to generate encouraging data from this trial. We have been encouraged to speed up the opening of the U.S. clinical trial sites the continued safety results in the opening up new sites should allow us to accelerate recruitment of future cohorts in the coming year. This in turn should allow us to generate more data faster and ultimately be closer to bringing OpRegen to the over 16 million American suffering from Dry-AMD. Now turning to our affiliate Asterias, in September Asterias announced positive interim efficacy data on AST-OPC1 in patients with complete cervical spinal cord injuries. The results were from the cohort of five patients in the trial treated with 10 million cells. The results while still early demonstrate meaningful improvement in motor function, specifically all five patients in this cohort achieve at least one motor level of improvement with two out of five patients achieving two motor levels of improvement on at least one side of their body. This level of improvement particularly in the use of a patient’s hands, fingers, and arms is very important for quality of life and ability to function independently. There are some video showing the impact of this improvement on Asterias’s website, and if you haven’t seen the patient videos, I encourage you to do so because it is quite remarkable. The difference in the lives of the young men highlighted is a compelling reminder of what could be possible with the therapies we’re developing. Asterias expect to report further data in January, and will provide more detail during that conference call on November 14. The data from OPC1, the data from OpRegen together with the recent data on some of our other programs have made us more confident about the overall potential of our pluripotent stem cell platform. Our other publicly traded subsidiary OncoCyte has been making good progress towards its first commercial product a confirmatory lung cancer test. OncoCyte’s partner the Wistar Institute presented positive data on this test at the recent CHEST Meeting, which took place in Los Angeles in October. OncoCyte is now completing its own study based on Wistar’s positive findings. OncoCyte expects to complete a study and report results later this year, if the data continues to be positive, OncoCyte plans to launch its lung cancer diagnostic test in the middle of 2017. Lung cancer kills more people in the U.S. than any other type of cancer and there are no good noninvasive options available today to confirm a lung cancer diagnosis. OncoCyte has been making good progress and tests for other types of noninvasive cancers too, and we’ll provide more detail in its third quarter conference call on November 10. Through the rest of this year and throughout 2017, we will remain committed to achieving our three key objectives. First, we will advance our programs with Renevia in HIV-related facial lipoatrophy and OpRegen for Dry-AMD. Or Renevia this includes completing the pivotal trial enrollment presenting top line data filing for European approval and hopefully obtaining a CE mark by the end 2017. We will also make progress with Renevia and other geographies. For OpRegen our goal include reporting data on Cohort I opening U.S. clinical sites, reporting data from Cohort II, completing and reporting data from Cohort III, as well as completing Cohort IV by the end of next year. Second, we’ll continue to implement additional actions to further simplify our structure. And third, we continue to participate in the progress of Asterias and OncoCyte, and we will pursue new opportunities to unlock value from our various subsidiaries. I would now like to turn the call over to Mike West, our Co-Chief Executive Officer to talk about our continued scientific leadership.
Michael West
Thank you, Adi. The research team at BioTime is acutely aware of the enormous potential of our company to create value for our shareholders. And simultaneously to improve the quality of life for those suffering from chronic degenerative disease. This potential is directly linked to the power of pluripotent stem cells to transform into the various cell types, they comprise the human body. This technology platform allows for the first time in history, manufacturer on an industrial scale, of all of the cellular building blocks of the heart, brain, or any other tissue that may be damaged by disease or trauma. In this respect, it is we believe a far more powerful platform than adult stem cells, which are allow the manufacturer only a limited number of potential products and generally speaking have a far more difficult time being manufactured to scale at a reasonable cost. We believe that the results, we’ve seen to date in patients by applying the technology to spinal cord injury with AST-OPC1 therapeutic candidate, and applying it to age-related macular degeneration through OpRegen are just too of numerous applications we expect to emerge in the coming years. So the goal of the research team at BioTime is to identify those additional future product opportunities with the greatest value and to advance them in preclinical studies. At the same time, we are diligently building a robust patent landscape, to ensure that we can protect our products exclusivity and increase the value to our shareholders in potential corporate partners. Most of the early stage were being performed by the BioTime research group is highly confidential, although we’ve provided from time to time a peek at some of our projects. In various scientific conferences we have for instance shown data on the production of various types of novel cartilage cell types. These may be useful in treating such disorders as low back pain and osteoarthritis. The leading complaint of an ageing population. We’ve shown data on the successful generation of what are called brown adipocytes that we and other groups believe could be important new therapeutic strategies for treating obesity and Type 2 diabetes. But this is just a tiny sampling of over 200 cell types for which we’ve created a scalable manufacturing process. I’m gratified to report that the work underway at BioTime is showing great promise in providing what we believe will be novel cost effective therapies for diseases for which drug, surgery and other current therapies are minimally effective. I’m also pleased to report that the pharmaceutical and biotechnology industries are becoming increasingly aware of the power of the technology to deliver first-in-class therapeutics and to we show results that are never before seen in medicine. We look forward to reporting on our progress in leading the field going forward and building value for our shareholders by advancing these products in the clinic and incorporate partnering. So with that, I’ll turn the discussion over to Russell Skibsted our CFO to report on the company’s financials, Russell?
Russell Skibsted
Thanks, Mike. Good afternoon, everyone. Or consolidated cash and cash equivalents totaled approximately $30 million as of September 30. This compares to about $42 million on December 31, 2015. It should be noted however that the 2015 cash position did include Asterias. In addition on September 30, there was approximately $2 million in restricted cash and available for sales securities, also on September 30, BioTime owned 21.7 million shares of Asterias common stock and 14.7 million shares of OncoCyte common stock, which represented an aggregate market value of $166 million as of that date. On August 31, OncoCyte strengthened its balance sheet and completed a successful equity raise that netted them about $10 million from both new and existing investments. The additional capital allowed them to continue development of their key cancer diagnostic programs in lung, breast, and bladder cancers through meaningful milestones including the critical data that is expected soon of lung cancer. Now I’ll turn the call back over to Adi.
Adi Mohanty
Thanks, Russell. As you heard from all of our members, it has been another very productive period for BioTime and our subsidiaries. With numerous value creating milestones ahead. For Renevia, we’re anticipating completing the pivotal clinical trial enrollment by the end of this year then completing the trial and reporting top line data in the first half of 2017. With these data in hand, we anticipate filing a CE mark - for a CE mark approval in Europe, and upon approval launching Renevia in Europe, and certain other countries in the second half of 2017. For OpRegen, we will report more detail data from Cohort I of our Phase 1/2a trial at ISOPT on December 2. Further, we expect to complete enrollment of patients in Cohort II, and gain approval from the DSMB to proceed to Cohort III this year. In early 2017, we expect to report complete Cohort I data and announced U.S. clinical trial site initiation. Also in the first half of 2017, we expect to report data from Cohort II and complete enrollment in Cohort III. Additionally in 2017, Asterias will be announcing data from its OPC1 trial in complete cervical cord spinal injury patients, while OncoCyte looks to launch its lung cancer diagnostic test in 2017. We look forward to updating you, as we advance our leadership position in pluripotent stem cell based therapies and our proprietary high stem delivery system, and utilize them to address major unmet needs in degenerative diseases. I would now like to open the call for questions. Operator, please go ahead.
Operator
At this time we’ll be conducting question and answer session. [Operator Instructions] Our first question comes from Kevin DeGeeter with Ladenburg Thalmann. Pleased proceed with your question.
Kevin DeGeeter
Hey, good afternoon, guys. Thanks so much for taking the question. Just quickly, with regard to OpRegen, as you open up some U.S. sites in the first-half of 2017, can you just give us some of your general thoughts as to how you’re thinking phase of enrollment and the number of patients per site you may be able to bring in and other metrics to help us think about the timeframe to get to some clinically relevant data on OpRegen?
Adi Mohanty
Hi, Kevin, so what we’ve said is we are accelerating the initiation of U.S. trial sites. We’ve been talking to some pretty key opinion leaders, who are very engaged with us. And we expect to be able to do that early in 2017, not really even middle, but the early in 2017. We expect to complete our second cohort this year. So that leads us into Cohort 3. And Cohort 3 next year will allow us to maybe have some U.S. sites treat Cohort 3 patient, while we still have the Israel site active. I think what we’re at this point saying is, we can finish definitely Cohort 3 in the first-half of next year. We were expecting by the middle of next year to even finish Cohort 4, which is a faster pace than before. But as we get the site on board, so as we initiate that site sometime in the beginning of next year I think we’ll have a much better sense of what kind of screening and pipeline of patients we have, to give you a more precise number. At this point to be able to complete all four cohorts by the middle of next year, seems like a pretty good start for us. I hope that helps.
Kevin DeGeeter
It does. That’s terrific. And then maybe just one more for me, then I’ll get back into queue, [and don’t worry, that’s my] [ph] last question. With regard to Renevia and the potential for CE mark in 2017, can you just talk about from a business development standpoint, your strategy to leverage potential CE mark for entry into other markets outside of Europe, including markets in Asia, potentially South America as well?
Adi Mohanty
Yes, so I don’t know if people have - remember, just recently about a week or two ago, we made an announcement that we’ve hired a Head of Corporate Development, Jim Knight, who has spent several years at Questcor. He was there from the early days in 2010 till - I think that the sales were around $100 million till they went past $1 billion. So he was there in a large part of helping them expand the indications of the product or the drug that they had. He is very well experienced. He was at Biogen. He’s been at Elan. So this is somebody we brought on that has a lot of experience with how to take advantage of multiple indications of a single drug, as well as how to in partnering work with a large pharma in a very good. And so, Jim’s responsibilities are going to include business development and strategy related to our marketing of many of these products. This we think is an appropriate time to bring somebody like that on. This data and maybe I’ll digress just a little bit. I’m not sure everybody quite got the earlier this week news where we talked about the early data on Renevia. So our principal investigator, Ramon Llull, is presenting data from the run-in patients which are the first few patients, because of it being a device trial, these first few patients don’t go into the main trial. But they are trained at - training patients at the sites that are running the trial. So essentially they’re the first few patients of the pivotal trial. The data from this trial, which shows that those people are at one year and at one year we still have sustained volume increases in the face, along with what I find most exciting is that, we might actually have proof that we grow new tissue. So that’s the kind of data that allows us already to start having Jim put into packages that we can work with companies across Europe as well as our previous connections that we’ve already made with South Korea and Brazil, particularly South Korea. I think at the last conference call we mentioned Russell and I had been out to South Korea. We met with several significant pharma partners who are very interested and in Renevia and its application in South Korea. So our intention now is that after November 14, when the data is presented, it will allow us to use that data in more substantial conversations with partners in geographies that might have pathway that we can explore or take advantage of. So give us a couple months. This is going to evolve pretty quickly. We will be able to come back and give you a more detailed explanation of what our strategy is for Europe and for the Asian countries. I think it’s a really exciting time that we actually have data, but there’s still time. So I think this is a good time for us. We will be ready by the time where we have to make - we have to pull the trigger on some of these actions next year.
Kevin DeGeeter
Terrific, thanks for all the detail.
Operator
Our next question comes from Keay Nakae with Chardan Capital Markets. Please proceed with your question.
Keay Nakae
Yes, thanks. First, with respect to the upcoming data presentation on the dry-AMD patients, will they be presenting any efficacy data on these three patients?
Adi Mohanty
Hi, Keay. So I think that we want to be careful not to make too much of efficacy data in the 50,000 cell dose. It’s a pretty small dose. We do see some encouraging signs, but we don’t think it’s appropriate to start looking at efficacy at this stage. I think though the surrogates for efficacy are very important and we see them now. And so, it’s really important that we be able to share things like engraftment, that even after one year the cells are alive and healthy in the back of the eye that they are well-integrated grafted in the back of the eye with the rest of the RPE cells, so many things that are surrogates. We do have some efficacy data, but I don’t think the intention here is to share a lot of that information. I think we want to try and do that with the second cohort, which is coming up pretty soon, right. So we think in early next year, with the second cohort, which gets the more clinically significant 200,000 cells, we should have information that we can start sharing that will be more clinically meaningful in the early part of 2017. So just trying to make sure we don’t set too many expectations for the 50,000 cell dose and that we don’t get too far ahead of ourselves. We’re pretty excited, but trying to control ourselves from sharing early data on a small dose cohort.
Keay Nakae
Okay, so then focusing on the second cohort, the 200K cells, when we get to early next year, how long does it take before you are able to measure these surrogates, such as the engraftment of the cells in a monolayer?
Adi Mohanty
We think it can be pretty early. So I think a meaningful data-point would be six months and we’re pretty close to reaching that with our first patient treated in that second cohort. So we’ll have the other patients reach that point in pretty early in 2017. So that’s why we’re feeling comfortable, saying that in early 2017. We should be able to share information on that second cohort.
Keay Nakae
Okay, very good. And then switching over to Renevia, with respect to the idea that you may actually be generating or growing new tissue, how do you go about advocating that?
Adi Mohanty
Yes. So it is one of those I think I’ve mentioned before. I mean, that to us are the most important thing that - we are in the business of regeneration and we believe we can regenerate new tissue or help by regenerating tissue, so animal studies have been done. So we had some animal studies already, where we were able to do histology of animals, and see that - it showed in the animal we grew new tissue. We’re doing further studies in larger animals to support the clinical trials, but there were instances that our PI was able to get some human data. And that’s the part that he is sharing, because the intention is not to have histology or cut a piece of the face to check what’s under there. But he did turn out to have an opportunity to get some human data. And I believe that’s what he will be sharing in two weeks. So that’s how you know, you can look underneath and you see if there’s tissue and blood vessels and…
Keay Nakae
Okay. And…
Adi Mohanty
Actually, maybe I’ll add something else. Sorry. The other part is though - is that there are well-established methods of assessing externally whether or not there is neo-tissue. There are measurements of elasticity. There are measurements of the feel of the tissue that is created. So there are techniques that we are now looking at, that will help supplement the histology to help decipher the difference between neo-tissue or not.
Keay Nakae
Okay. And then, just finally, with respect to that data coming up in a couple of weeks, how many patients were there in this, let’s call it the run-up cohort.
Adi Mohanty
So we know that we had nine run-in patients, which is three at each of our three sites. I’m not really sure if all nine are included or if six or seven or eight are included in his presentation. We’ll have to wait for the presentation to see that. But it’s somewhere between six and nine patients.
Keay Nakae
Okay, very good. Thanks.
Operator
Ladies and gentlemen, we have reached the end of the question-and-answer session. I’d like to turn the call back to Adi Mohanty for closing comments.
Adi Mohanty
Yes, thank you very much. Thanks for the questions. And thanks for calling in and listening. As I mentioned, it really has turned here into a very exciting time for us with very significant milestones coming up. So we look forward to sharing all that information in the coming weeks and months. Thank you.
Operator
Thank you for your questions. For additional information, please feel free to contact us or visit our website at www.biotimeinc.com. This concludes today’s conference. Thank you for your participation. You may disconnect your lines at this time.