BioTime, Inc (NYSEMKT:BTX) Q1 2016 Earnings Conference Call May 17, 2016 4:30 PM ET
Dan Lawrence - Director of Investor Relations and Corporate Communications
Adi Mohanty - Co-Chief Executive Officer
Michael West - Co-Chief Executive Officer
Russell Skibsted - Chief Financial Officer
Keay Nakae - Chardan Capital
Rohit Vanjani - Oppenheimer
Bruce Jackson - Lake Street Capital Markets
Good day, ladies and gentlemen, welcome to the BioTime First Quarter 2016 Results Conference Call. [Operator Instructions] As a reminder, this conference call is being recorded.
I'll now introduce your host for today's conference call, Dan Lawrence, BioTime’s Director of Investor Relations and Corporate Communications. Dan, please go ahead.
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 result and key accomplishments for the first quarter of 2016 and recent corporate developments. There will be a taped 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 members of BioTime's senior management team, including Co-Chief Executive Officers Adi Mohanty and Dr. Michael West; also Chief Financial Officer, Russell Skibsted. Adi and Russell will make prepared remarks and then we will take some questions from sell-side analysts and Portfolio Managers.
Before we get started, we would like to remind you that during the course of the conference call, the company will make some projections and forward-looking statements regarding future events. We encourage you to review the company's past and future 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, Co-Chief Executive Officer of BioTime.
Thanks, Dan, and good afternoon everyone. Thank you for joining us this afternoon. We’re off to a strong start in 2016. Since our last call six short weeks ago, we have continued to make progress on our core clinical therapeutic programs. These key programs leverage our proprietary pluripotent stem cells and our proprietary cell delivery platforms with important product candidates targeting large market opportunities in medical aesthetics and ophthalmology.
On the call today, we’ll provide a brief update on our two core development programs Renevia for medical aesthetics and OpRegen in dry age related vascular degeneration or dry AMD. We will also briefly touch upon recent developments in our important non-core programs. We continue to focus on our three primary objectives designed to drive increased shareholder returns; clinical progress, simplification, and unlocking value from our non-core subsidiaries.
In the area of clinical progress, BioTime and our subsidiary Asterias Biotherapeutics have a total of four therapeutic product candidates in human clinical trials. Renevia is our most advanced product candidate. It could be on the market in Europe and certain other geographies as early as the second half of next year. Renevia is a product candidate where we combine the patient’s own fat cells with our HyStem cell delivery platform which is injected into the places of the patient’s face where tissue is missing.
The entire process is a single outpatient procedure that can be easily performed in the doctor’s office. Our pivotal clinical trial assessing Renevia’s efficacy in HIV-related facial lipoatrophy is ongoing and progressing as expected. The efficacy measures of the trial include change in tissue thickness measured by 3D volumetric change and aesthetic scores that include patient reported outcomes. We expect to complete patient enrolment by the second half of this year where top line efficacy data is expected in early 2017.
If the data are positive, we will submit a CE mark filing in the first half of 2017 with approval potentially in the second half of next year. There are about 350,000 people in Europe with HIV related lipoatrophy and we look forward to making Renevia available to these patients next year. However, we consider this trial to be a gateway trial that could serve as the basis for expanding the label for Renevia to include medical aesthetic uses such as age related and trauma related facial fat loss, which represents a much larger multi-billion dollar market.
This broader indication is generally referred to as facial lipoatrophy. There is very strong interest for Renevia in markets beyond the U.S. and Europe, specifically Brazil and several Asian markets like South Korea represent significant opportunities.
We recently visited with several companies in South Korea that have shown strong interest in our Renevia program. We hope to progress these discussions in the coming weeks and months. We remain very encouraged by Renevia’s progress to-date, in terms of patient enrolment and clean safety profile.
We are in active discussions with the FDA, regarding the study and the approval pathway in the U.S. for the broader indication of facial lipoatrophy. We believe there are many other potential applications for Renevia and the HyStem platform in combination with a diverse range of autologous and allogeneic cell therapies.
OpRegen is our product candidate for dry AMD. AMD is the leading cause of severe vision loss in people over the age of 60 around the world. There are two types of AMD, wet and dry. Wet AMD therapeutics, a widely used and generate around $8 billion in annual revenue. There are nine dry AMD patients for every one wet AMD patient, but there are no currently approved therapies available for dry AMD.
It is a much larger opportunity than wet AMD and truly an unmet need that affects many people. OpRegen is in a Phase 1/2a dose escalation clinical trial for the dry form of AMD. The last patient in the first cohort was successfully treated earlier this year. All patients have not completed the minimum follow-up time required since treatment, and we’re preparing the report for the upcoming Data Safety Monitoring Board or DSMB meeting this quarter. We expect that the independent DSMB will recommend that we commence the second cohort.
Our goal is for this to happen very soon after the approval. The second patient cohort will receive a higher, more clinically significant dose of OpRegen. We expect to complete the second cohort and if the data are positive, begin the third cohort by the end of this year. There continues to be significant interest in dry AMD among Pharma and biotech companies.
For example, Ocata which had a Phase 2 asset in dry AMD was acquired by Astellas, a large Japanese Pharma company for approximately $400 million earlier this year. With both programs, we’re assessing several development strategies including potential corporate partnerships. The partnering environment with major international Pharma is particularly active right now because these companies are extremely focused on building their product pipelines.
Russell and I have just returned from meeting with several pharma companies that are actively exploring partnerships with companies like ours. They’re looking for products thought to have high commercial potential, a relatively rapid time to market, and excellent clinical results. We believe Renevia and OpRegen potentially have very high commercial value, while Renevia certainly meets the criteria for close to market with excellent clinical results.
With partnerships we look to create win-win relationships that maintain long-term value while creating none or minimally dilutive funding to get our products to market. We have a rich pipeline beyond the current two primary products, which we expect to generate value over time and as resources allow.
Let’s briefly turn to Asterias and the other two therapeutic candidate programs in the clinic. Asterias has presented promising clinical data from the Phase 2 study of their cancer immunotherapy AST-VAC1 in AML and from the Phase 1/2 clinical trial of AST-OPC1 in spinal cord injury.
Asterias had a successful end of Phase 2 meeting for VAC1 earlier this year, when the FDA indicated general agreement with Asterias’ proposed development plan for registration of VAC1 through a single Phase 3 trial to support an accelerated development pathway and BLA filing for VAC1. The company currently is evaluating plans for progressing the VAC1 program towards a pivotal Phase 3 trial, which would begin in 2017.
Asterias has other product in the clinic; OPC1 for the treatment of spinal cord injury has some big news reported just yesterday. The company announced plans to expand the OPC1 Phase 1/2a trial in spinal cord injury patients. The expansion means that more patients will now be treated in the trial. The management of Asterias believes this trial expansion should increase the statistical confidence of the safety and efficacy data, expand the range of spinal cord injury patients being evaluated and better position the product for a potential accelerated regulatory pathway.
Like OpRegen, which receives some non-diluted funding from the office of the Chief Scientist of Israel, the OPC1 trial is being funded in part by $14.3 million grant from the California Institute of Regenerative Medicine.
Asterias also recently added a very prestigious member to its Board of Directors with the appointment of Dr. Howard Scher. Dr. Scher was involved with the developments of two of the most important Prostate Cancer Therapeutics to be commercialized in recent years. Zytiga and Xtandi, both products have been major successes in the marketplace.
The Asterias team looks forward to doctor Scher’s insides and contributions. Asterias’s management provided an update on all of their clinical programs during a conference call yesterday, which is archived on their website.
Turning briefly to our cancer diagnostic subsidiary Oncocyte. They announced exiting news that is bladder cancer abstract has been selected for presentation in the post session including a light panel discussion at the prestigious ASCO meeting in early June.
The data being presented is a continuation of the data first presented at the American Association for Cancer Research Annual Meeting in 2015, which demonstrated a high level of sensitivity and specificity in the detection of urothelial carcinoma, which is the most common type of bladder cancer.
Oncocyte plans to host it’s conference call tomorrow at 5.00 pm eastern time to provide a more detailed update on its recent progress, including the ASCO bladder abstract that is being released tomorrow at 5.00 pm. I’d like to spend a few moments on our noncore assets. We are pleased with the significant progress within these operations as they mature towards standalone businesses.
In particular, we’re excited to see life absolutions, our digital health subsidiary gaining more traction with customers and world class institutions. Lifemap was the first commercial developer working with Apple on its game changing Researchkit technology platform that enables medical researcher’s together medical and health data using a patient’s iPhone.
LifeMap recently launched its new mobile health app design and development service to help research institutions and help companies worldwide to develop custom Smartphone apps and research studies. Through the new service, LifeMap offers clients its deep expertise in medical science, consumer behaviour, app analytics and design.
LifeMap’s innovative data driven mobile health or Mhealth apps are designed to recruit clinical study participants obtain patient concept through the iPhone and passively collect participant health information. LifeMap has developed innovative digital health apps in collaboration with some leading research institutions like Mount Sinai, Stanford School of Medicine, National Jewish Health, as well as with strategic partners like 23andMe.
LifeMap and Mount Sinai recently expanded the availability of its co-developed Asthma health app to the United Kingdom and Ireland in addition to the U.S. We are very pleased by the significant progress at LifeMap with several of its achievements being mentioned in the press sparking increasing interest from potential partners and customers.
So with that, I now turn the call over to Russell Skibsted, our CFO, who will discuss our financial results. Russell?
Thanks, Adi. Good afternoon everyone. Our consolidated cash and cash equivalents totalled $27.1 million as of March 31, 2016, compared to $42.2 million as of December 31, 2015. The $27.1 million of cash on hand at the end of March, included $16.4 million held by Biotime and its non public subsidiaries. As of March 31, 2016, the value of the common stock we owned in Oncocyte and Asterias was approximately $170 million.
Last week, Asterias announced a successful equity raise, the financed netted them a little over $16 million. This additional capital allows them to continue the development of their primary clinical programs, including the expanded OPC1 Phase 1/2a clinical trial.
The financing takes our ownership percentage in Asterias to below 50%, which means that Biotime may longer to be required to consolidate Asterias financial after May 13. This is important in our objective of simplification because our financials going forward would be much easier to read and understand and in our opinion, a step closer to be more representative of BioTime’s operations.
We believe with our available capital along with our grants and other non dilutive funding that we have the ability to execute our operating plans through 2016.
Now, I’ll turn on the call back over to Adi. Adi?
Thanks, Russell. So to summarize, we’re making steady clinical progress with our dry AMD and medical aesthetic therapeutic programs. We see increasing interest from partners across all our technologies, programs and noncore assets. At the same time, we’re committed to simplifying our structure, while unlocking the value of our more mature revenue generating subsidiaries for Biotime shareholders.
With that, operator we’re ready for questions.
Thank you. At this time, we’ll be conducting question and answer session. [Operator Instructions] And our first question comes from the line of Keay Nakae from Chardan Capital. Please proceed with your question.
Hi, thanks. Yes it’s Keay. First question is for Russell, with respect to the ability to eliminate the consolidation on your subs, beyond the ownership volume below 50%, are there other considerations that have to be met before you’re able to move in that direction?
Good question. There are in fact, the primary issue that we look at is effective control. So that can be either control of the Board of Directors or can be some sort of voting arrangement. What I would say with Asterias is we do not need any of those. So, I would expect that we should be deconsolidated going forward.
Okay. Very good. So that looks pretty straight-forward than at least with respect to Asterias?
Yes. Correct thanks.
Okay. And then, just a second question for Adi, as it relates to the OpRegen dry AMD study, so what if anything, will you be able to say about the first patient cohort once you get the approval to move into the second patient cohort? Obviously they are evaluating safety, but will you be able to say anything about efficacy for that group around the time that you transition to the enrolment of the second cohort?
Hi, Keay. Yeah, thanks for the question. So, the first cohort is a safety dose. Although, the patients that we’re treating, they’re not healthy volunteers, they’re actually dry AMD patients. There might be something that we see, but the intention here is to just assess safety. What we’d like to see is we’re preparing the report right now. We’re getting ready for the DSMB meeting. What the DSMB allows us to say will be important, but maybe worth reminding people that when you look at cell therapies or these types of programs, it is so much different from a more typical biologic or small molecule program where the early safety signal doesn’t give you that much more risk reduction compared to a cell therapy program.
When we’re replacing cells that are missing, what is more important to learn very early on is whether or not the cells are still there? Whether or not the body reacted and you had some immune or adverse reactions. There’s some pretty significant things that we expect to learn with this initial cohort. How much we will be allowed to say will depend on how our interaction with the DSMB goes, but anything that allows us to say that we had a successful cohort one and move on to cohort two, we think is a pretty substantial de-risking of the program unlike other typical clinical trials.
Okay. Great. Thanks for that. And then, just maybe looking a little further ahead, as you get into the second cohort, which is kind of begetting a different dose? Again kind of similar question as you transition to approval to launch into the third cohort, what might you be able to say at that time while it’s still around the end of the year about, say second cohort efficacy?
Yeah, that’s a good question. So the second cohort as you know that goes up to about 200,000 cell dose and just as a reference you know, Ocata used 150,000 cell as their clinical dose. So we expect for a 200,000 cell cohort to have some clinical signal. We will be measuring more efficacy and clinical signals.
How much we’ll be able to say will again depend on, this is still a clinical trial. I know its open label, but to maintain the integrity of the trial would be more important for us, but we should be seeing some signal and we should be able to say something we hope by the end of that second cohort before going into cohort three.
Okay, very good. Thank you.
And our next question comes from the line of Rohit Vanjani of Oppenheimer. Please proceed with your question.
Hi, thanks for taking my questions. Russell, I think last quarter, you were at 57% ownership of Asterias and 58% for Oncocyte. What is it for Oncocyte now and then with Asterias’ common stock and warrants offering? You said, you’re below that 50%, but what percentage is it exactly and what would it have been without that offering?
Without the offering, it would have been approximately 57%, which is what it was before. With the offering, as it was announced, we’re down to 49.5% of Asterias and then Oncocyte remains approximately 58%.
Okay. And then Adi, for the Renevia trial, I think you said you needed 56 completers. Do you know where you are with enrolment right now? I think last quarter, you said you were two/thirds enrolled.
Yeah. We don’t quite talk about numbers. We’ve treated more than 40 patients already and so we don’t get into specifics of numbers, but it is tracking towards where we thought which is full enrolment in a few months. So the second half of this year, we expect to be fully enrolled and still be able to have that top line readout at the beginning of next year. I think from the last time, we have the call to now we’ve added more patients. So it’s progressing well.
Okay. And then you said you’re in discussions with the FDA on facial lipoatrophy, any idea what would be involved there if you had to do a U.S. trial, would it be too well controlled studies for approval?
Yeah. So in this area, Rohit, I actually think we’ve got some really good news. We have yet to find the details, but let me tell you we’ve been interacting with the FDA over the last few months as I mentioned and they have told us now officially to follow the device approval pathway. So, in our interactions with them, while we were preparing the documents and going to type c meeting and they have come back and said, no we think it is a device approval pathway.
There are still certain hurdles that we have to pass through with people from [indiscernible] involved the assessment, but this is a significant, I think positive for us where a device approval pathway means a single pivotal study and it also means we can leverage a significant amount of the data that we’re generating in Europe.
So instead what you were just referring to, which is a typical biologics pathway of some previous study and then two confirmatory Phase 3 study just like seem to single pivotal study. Now, this is still early in the conversation as soon as we finished our meetings and have really confirm this pathway and the future of how we’re going to progress in the U.S. will be sure to share that as appropriate, but in deed over the last couple of weeks we’ve had. What I think pretty encouraging news on this front.
That’s great. And then, last one from me. I think there was a stem cell article that came out earlier this month that it relates to Biotime as competitor, can you discuss the relevant of that article and then the likelihood that investors can induce the right 3d genome confirmation in IPS cells and actually what factors are involved there?
Rohit, maybe Mike will answer this question.
All right, Rohit. So you’re probably referring to induce pluripotent stem cells for all the listeners let me just very quickly recap what we were talking about. So Biotime is really relatively unique here. Our profound focus on these cells, pluripotent stem cells they are able to make all the cells of the human body. There is a different version of these cells, they are called induced pluripotent stem cells or sometimes called IPS cells.
And so, in that technology and which we some years ago have reported on its possible to actually take an age cell like a skin cell. It’s sort of like a time machine take it back to beginning of life and reset the cell clock of aging [indiscernible] and we showed in that publication, I think it was back 2010 that can work, but one out of 10 attempts. Since that time, others who have study these cells embarked greater detail, the Japanese side has won a Nobel price, if some others work and it has become a big thing in the research industry, but these are sort of synthetic versions of the real normal cells, the pluripotent stem cells, the bio term works with.
And so they attract a lot of study and in the last week there was a study in cell stem cell, a prestigious scientific journal, which look very carefully at the DNA of these cells and showed abnormalities. This mere some of the other papers have been published in the last few years. So, BioTime’s position is, pluripotent is the future of regent of medicine, but we rely on the goal standard normal pluripotent stem cells. We do research on IPS as we’re not using them clinically in part because of results like that just came out that you are referring to showing abnormalities and safety of products made from these cells is front and foremost of importance to the FDA and therefore biotech companies, so it highlights the strategy of BioTime and focusing on normal pluripotent stem cells, does that help answer the question or I can get into more of science if you want.
No. That’s perfect. I’ll probably take it up offline with you, but thanks for that.
Before you - Rohit, never mind. We’ll take it offline because I was going to mention, we just made the strip all across Asia, where Japan and South Korea and other places where the IPS cells actually were being used quite a lot to a person everyone said, they’ve stopped using that and they don’t think that’s a great idea and that they actually were interested in the cells we have and it made sense to them to follow those. So I just ...
It’s important technology, it’s being widely used for research, for drug discovery and further things. BioTime has taken the position, the normality and safety is front and foremost and so we are focused on normal pluripotent stem cells.
Great. Thanks for taking the question.
And our final question comes from line of Bruce Jackson from Lake Street Capital Markets. Please proceed with your question.
Hi, congratulations on all the progress.
So, my first question is about the Renevia regulatory pathway in United States. So you’re already taking to the FDA, how would things play out here after the CE mark? Is this something that where you can parallel path the U.S. study or would you wait to fully complete the EU study before starting the U.S. study?
Well, we don’t have to wait for the EU study to be completed or registration. We do need a substantial portion of the data as you know the FDA cares a lot about safety and we’ve got a pretty large safety database already from the EU trial and it continues to grow. So there is a point at which we can get enough data from the trial without having to wait for completion of that trial and so we think, I think we previously said by the end of this year, we hope to have a protocol and a pathway and if we get approval, we’ll be ready to start this trial without having the wait for EU trial complete or the registration to complete in Europe.
Okay. And then, my other question was about OpRegen and with the varying dose levels in the trial. Can you just tell us a little bit about how you arrived at those dosing levels and why you think that the higher levels are going to be efficacious?
Well that’s a good question. I think this is a little bit of science and little bit of art. There is a significant amount of animal study that we’ve done. So I think for those who haven’t seen it, it’s a worthwhile look, several hundred animals worth of preclinical data. It was presented at ARBO last year. It’s the basis of a lot of the program that we are actually running and that combined with the experience of people like Ocata in our own experience, it indicates to us that we think that the dose is somewhere between the 200,000 and 500,000 and that Ocata was under dosing a little bit.
And our animal studies showed, you know so it is an extrapolation that’s why I said a little bit of science and a little bit of an art to come up with this dose and again as you can imagine there is differences in the size of the atrophy that we’re trying to address so somewhere in the 200,000 to 500,000 range and so we’re trying both of them in our third and fourth cohort. So, we’ll get data from both those dose arms.
Okay. Great. Thank you very much.
Okay. We’ve reached the end of our question and answer session. I’ll turn the conference back over to management for any closing remarks.
Thank you very much everyone joining us. We appreciate you taking the time and we’re look forward to coming back and reporting on further progress. Thanks.
Ladies and gentlemen, this does conclude today’s teleconference. We thank you for your time and participation today. You may disconnect your lines at this time and have a wonderful rest of your day.
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